FDA Matters Blog
Will the Real FDA Please Stand Up?
This e-mail grabbed my attention this week:TAKE ACTION: FDA Whistleblowers being Fired - HELP THEM.[Organization] has received an appeal for help to prevent the firing of courageous, honest FDA scientists who risked their careers to save lives by informing Congress about serious safety concerns involving dangerous, FDA-approved drugs and medical devices.FDA employees should not be afraid to speak honestly and freely about misconduct that threatens the health and safety of all Americans.This isn't the FDA that FDA Matters has known and followed for 30 years. Yet claims like the one in this e-mail are persistent and come from many sources.
This e-mail grabbed my attention this week:
TAKE ACTION: FDA Whistleblowers being Fired - HELP THEM.
[Organization] has received an appeal for help to prevent the firing of courageous, honest FDA scientists who risked their careers to save lives by informing Congress about serious safety concerns involving dangerous, FDA-approved drugs and medical devices.
FDA employees should not be afraid to speak honestly and freely about misconduct that threatens the health and safety of all Americans.
This isn't the FDA that FDA Matters has known and followed for 30 years. Yet claims like the one in this e-mail are persistent and come from many sources.
Many FDA critics—inside and outside the agency—believe the FDA is corrupt, industry-beholden, and arbitrary in its decisions. They allege that the agency is insufficiently concerned about safety and that managers have too much power to overrule staff and suppress dissent.
FDA Matters has itself raised questions about workplace problems at FDA. There are links below to columns on "Dissent and Efficiency: Difficult Trade-offs for FDA," "Why Do Some People Dislike the FDA?" and "FDA: A Hit and a Miss" (about Avandia). Nobody would take the position that all 12,000 FDA employees are happy, fulfilled or satisfied with their work.
Nonetheless, I have always found FDA employees to be committed to the American people and dedicated to the public health needs of patients and consumers.
After spending four years as a government manager in the 1980's and over a decade as a manager in the private sector, I believe FDA is like most companies or organizations: encompassing a broad range of competency and commitment and having its fair share of job dissatisfaction. From my government experience, I also remember how hard it can be to re-assign government workers who lack commitment or are incompetent or disruptive.
So, would the real FDA please stand up? Is it my very positive experiences or the dark accusations of ethical lapses, industry coziness and harmful suppression of disagreements? How can two such disparate views co-exist?
Forming a consensus and "speaking with one voice" are logical and sensible for FDA, but not an accurate reflection of what usually happens when well-trained, analytically-oriented people with different perspectives gather to make a decision. For example, reviewers focused on the risk-benefit of a medical product are often at odds with reviewers whose focus is safety.
The situation can become more confused when data is open to different interpretation. In such a clash, some people will feel they were not heard or that their views were not considered seriously enough. This is understandable and inevitable. Further, no one can deny that FDA has made some bad decisions and might have done better if it had listened to dissenting views.
However, aggrieved employees may also feel that malicious agency thinking and dictatorial managers have kept their views from becoming the FDA's position. It is this generalization--from a single instance to the entire agency--that fosters the corrupt image of FDA propounded by agency critics.
I am not persuaded that these critics are right. FDA is making progress in handling dissent and in encouraging managers to be more open-minded. As happened with the Avandia decision, the agency is trying to be honest about disagreements. I am still impressed by the FDA's self-evaluation of its poor performance in the ReGen medical device approval.
To me, these are hopeful signs, as well as indications that FDA values the nation's public health above all other interests. FDA makes mistakes, but there is no conspiracy. The agency is fully committed to serving the American people.
Steven
Some related columns:
Dissent and Efficiency: Difficult Trade-offs for FDA
May 9th, 2010
Why Do Some People Dislike FDA?
October 15th, 2009
Yesterday, I received separate posts from three organizations that are anti-industry, one of which dislikes FDA and one of which hates FDA. They are not alone in these feelings. There are many groups and individuals who believe that industry and physician professional societies run FDA. I don’t accept their premise or the “facts” from which they launch attacks. Read the rest of this entry
FDA: A Hit and A Miss
September 26th, 2010
FDA Matters has watched FDA handle the Avandia decision differently from any prior controversy. I like the new approach. In the same week, FDA provided a status report on its long-overdue social media and Internet communications policy. Because the agency’s efforts have been glacial, the prospect of useful guidance is dim. I think this is a serious problem. Read the rest of this entry
The State of the FDA—January 2011
FDA's touches the lives of every American at least 6 to 10 times each day. The agency oversees 80% of the nation's food supply, all of human/animal medical products and cosmetics, and almost all radiation-emitting devices. Altogether, the agency is responsible for about 20% of all consumer dollars spent in the United States.With the President set to deliver his State of the Union address to Congress in 10 days, it seemed a good time for FDA Matters to provide its view of the "State of the FDA." At the beginning of 2011, the agency is doing well, but has a lot of catching-up to do and faces a number of threats.
FDA's touches the lives of every American at least 6 to 10 times each day. The agency oversees 80% of the nation's food supply, all of human/animal medical products and cosmetics, and almost all radiation-emitting devices. Altogether, the agency is responsible for about 20% of all consumer dollars spent in the United States.
With the President set to deliver his State of the Union address to Congress in 10 days, it seemed a good time for FDA Matters to provide its view of the "State of the FDA." At the beginning of 2011, the agency is doing well, but has a lot of catching-up to do and faces a number of threats.
Strengths: FDA's most important strength is the dedication of the agency's staff and the leadership of Commissioner Hamburg's team. They have made great strides in advancing the public health mission of the FDA, aided by increasing appropriations and the momentum of a new presidency. The renewed emphasis on public health has become a core strength of the FDA.
The agency's commitment to science is another strength. Not every decision can be answered with science (e.g. policy on social media), but a substantial number can. The FDA is successfully building its scientific capabilities, although it is a work in progress. Similarly, the agency's new initiatives to establish an international presence represent a strong response to the challenges of globalization.
Weaknesses: While FDA has been doing better, it is still an organization with workplace issues. Within the agency, barriers still exist to sharing knowledge across offices and divisions. To the broader world, the whole agency seems insular. In particular, the FDA makes inadequate use of expertise that lies outside the agency, particularly in academia.
FDA's information technology (IT) systems are grossly inadequate for an agency with such large, far-flung responsibilities. Some major, badly needed databases aren't going to be available anytime soon, preventing the agency from making the best, most well-supported decisions.It also dooms staff to perform tedious work that could be done in seconds with better tools.
Opportunities: Within FDA, there is the potential for a 21st century regulatory and public health agency that could be the world's benchmark for decades. While such standing is a goal in itself, it is also true that the world-wide use of FDA standards opens up export markets and can contribute to our nation's economic growth.
To realize this opportunity, FDA needs to do a better job in explaining regulatory science to Congress, the media and the public. The tools and standards FDA uses every day are still largely anchored in the last century. Public investment in improving regulatory science is what will bring the agency into the new century.
Another opportunity for the agency is to break the secrecy of how decisions are made. Observers often wonder about the agency's rationale. Industry needs the benefit of understanding the agency's actions. Dr. Hamburg and other senior FDA leaders have taken an important step by publishing more articles in mainstream journals. The handling of internal dissent in FDA's ranks was accomplished deftly in last summer's Avandia decision. In doing so, honestly and forthrightly, FDA increased it credibility and was better able to defend its ultimate decision.
A very different type of opportunity is presented by FDA's ongoing consolidation of personnel at just two locations, White Oak and College Park. This provides the opportunity for a more cohesive workforce, as well as a more efficient one.
Threats: FDA's largest threat relates to its funding and the possibility of being dragged back by budget cutting. Several years of increases have greatly helped the agency, but it is not enough to offset decades of budgetary neglect or carry the agency forward without more money. The gap can widen quickly because FDA's responsibilities keep increasing: more complex science, globalization and two major new laws to implement, food safety and bio-similars.
Another looming threat comes from the ongoing negotiations for re-authorization of pharmaceutical and medical device user fees. FDA needs the funding, but not the likely hit on its public credibility. When Congress considers renewal legislation in 2012, the agency is likely to be caught in the crossfire between those who think FDA is too industry-friendly and those who think the agency impedes FDA-regulated industries in bringing new, sometimes life-saving products to market.
Compounding this threat, user fee re-authorization will probably be the only FDA legislation that must pass Congress over the next two years. Dozens of legislator are going to try to get their FDA-related concerns addressed in the legislation. No one knows how much damage the final legislation might do to FDA.
Steven
Will the New Congress Be Good for FDA-Regulated Industries?
FDA Matters is hearing that FDA-regulated industries will benefit from the 2010 election. It is assumed that a Republican-led House and more Republicans in the Senate will benefit drug, device and food companies. After all, aren't Republicans more business-friendly and more concerned about perceived regulatory excess?Those saying and thinking these things may be in for a rude awakening. Even worse, they may find themselves nostalgic for the "good old days" (whenever those were). Everybody—FDA, industry, patients and consumers—is going to have a rough time over the next two years. Industry will be heard more often, but not always have the winning position.
THIS IS THE LAST OF MY 2010 COLUMNS ON FDA AND THE NEW INCOMING CONGRESS. LINKS ARE AT THE BOTTOM OF THIS PAGE.
FDA Matters is hearing that FDA-regulated industries will benefit from the 2010 election. It is assumed that a Republican-led House and more Republicans in the Senate will benefit drug, device and food companies. After all, aren't Republicans more business-friendly and more concerned about perceived regulatory excess?
Those saying and thinking these things may be in for a rude awakening. Even worse, they may find themselves nostalgic for the "good old days" (whenever those were). Everybody—FDA, industry, patients and consumers—is going to have a rough time over the next two years. Industry will be heard more often, but not always have the winning position.
To start with, the Republican House of Representatives is going to be asking lots of questions about policies and programming initiated by the Executive Branch during the last two years, including FDA. Because of their backgrounds as heads of big-city public health department, Commissioner Hamburg and Principal Deputy Sharfstein are more prepared then most political appointees.
However, they have never experienced the volume or magnitude of these inquiries. Neither FDA nor any stakeholder benefits if FDA is busy answering Congressional letters or preparing for oversight hearings on the Hill…..instead of reviewing products, setting standards and conducting inspections.
Even in the Senate, oversight and investigations are going to make a big comeback. This is a byproduct of budget politics: if there is no money to spend and big divisions over authorizing legislation, then Members turn to investigations to fill the time and command national attention.
Beyond FDA, regulated industries are going to come under increased scrutiny. The incoming chairman of the House Oversight and Government Reform has already announced plans to investigate the over-use of expensive medical devices and probe the way food recalls are handled. Imported food, drugs and raw ingredients from China may be given oversight scrutiny. Current investigations are going to continue on quality manufacturing in drugs, biotech products and OTC drugs.
Senator Grassley, as Ranking Minority on the Senate Finance Committee, has held investigations of tax-exempt hospitals, non-profit advocacy groups, FDA and FDA-regulated industries. Senator Hatch, who will be Grassley's successor in the post, has already indicated his intention to continue many of Grassley's issues and to have a tough investigations staff.
The Alliance for a Stronger FDA has already established that consumer and patient groups, health professional societies, associations and industries have a common interest in a strong FDA through increased appropriations. In the face of the current budget-cutting fervor, it remains to be seen whether industry will be able to convince legislators that FDA needs more resources from general revenue.
Separate, but related: a year from now, the drug, biotech and medical device industries will be trying to limit the amount of new user fees they will be required to pay when user fees legislation is re-authorized in 2012. It is hard to see how business' complaining about excessive fees will prevail against Congress' need to increase FDA funding from sources other than general revenue.
From these examples, it is possible to see a larger theme. Republicans generally believe that industry, without too much government intervention, should be relied upon for job creation in the US. Most of the party rhetoric is focused on achieving these through reducing the federal budget, trimming federal regulations and regulatory agencies, and making sure that "the people" who voted for them in the last election feel they are being heard.
The FDA context is different. Most FDA-regulated companies want simplification of the regulatory requirements and more certainty in their implementation, but are not interested in eliminating FDA's regulatory structure or limiting its ability to assure public health and safety. Thus, industry would not benefit from efforts to starve, roll-back, harass or marginalize the agency. The worst-case for the next two years is that Congress might try all four and not listen to industry concerns about negative outcomes.
Steven
"Must-Pass Legislation" Key to FDA's Future
December 12th, 2010
FDA Matters believes that the 2010 election will profoundly affect the FDA's mission, priorities, funding, standards and work flow. Eighteen months from now, FDA's leadership team will probably be the same, but the agency won't be. At the moment, there is only one "must-pass" item on Congress' FDA agenda: the next round of user fee renewals that will come before Congress in the Spring of 2012. Read the rest of this entry
Two Strategies for FDA Legislation in 2011
December 5th, 2010
The current Congress has two primary FDA-related accomplishments: have been less visible: an abbreviated approval pathway for bio-similar drugs; and a food safety bill that may be enacted before Congress adjourns. FDA Matters believes that any FDA-related legislation will falter in 2011 if it does not follow the strategy behind one or the other of these efforts. Read the rest of this entry
FDA and Election 2010: Oversight and Investigations
November 13th, 2010
President Obama's election and the distraction of health reform have distracted us from the disruption that divided government imposes on FDA. With the new Republican majority, the agency will find itself buffeted by political forces that are as concerned about "scoring points" as they are about improving government. FDA Matters thinks this will have a large impact on FDA, as well as the agency's stakeholders. Read the rest of this entry
FDA and Election 2010: Deficit Reduction and Appropriations
November 6th, 2010
So-called "wave elections"–where one party overwhelms the other–are particularly hard to judge. The ground rules are going to change dramatically– in ways that no one can fully anticipate. At first, each side refuses to compromise. Then, something happens that sets the pattern for whether people will work together and on what issues. This may take months to resolve or may occur before the new Congress arrives. As things change, FDA Matters thinks there are some key issues for FDA-watchers to monitor. Read the rest of this entry
“Must-Pass Legislation” Key to FDA’s Future
FDA Matters believes that the 2010 election will profoundly affect the FDA's mission, priorities, funding, standards and work flow. Eighteen months from now, FDA's leadership team will probably be the same, but the agency won't be.Identifying and understanding the likely changes to FDA requires examining the meaning of "must-pass legislation" and its escalating importance as a quarrelsome Congress turns into a divided Congress. At the moment, there is only one "must-pass" item on Congress' FDA agenda: the next round of user fee renewals that will come before Congress in the Spring of 2012.
This is the fifth straight column looking at FDA and Congress. This reflects FDA Matters' belief that the 2010 election will profoundly affect the agency's mission, priorities, funding, standards and work flow. Eighteen months from now, FDA's leadership team will probably be the same, but the agency won't be.
Identifying and understanding the likely changes to FDA requires examining the meaning of "must-pass legislation" and its escalating importance as a quarrelsome Congress turns into a divided Congress. At the moment, there is only one "must-pass" item on Congress' FDA agenda: the next round of user fee renewals that will come before Congress in the Spring of 2012.
"Must-pass" legislation is a bill or resolution that Congress is compelled to pass in order to maintain the functions or functioning of government. The best examples are appropriations bills, Congressional budget resolutions, and legislation to increase the limits on the national debt. Sometimes, "must pass" is defined by the agenda of a political party or the President, as was the case with health care reform.
Looking at this Congress' two FDA-related legislative accomplishments, "must pass" played a role in both. Representative Anna Eshoo (D-CA) had the votes to advance her version of bio-similars in place of the version supported by House Energy and Commerce Committee Chair Henry Waxman (D-CA). Occasionally Committee Chairs don't have the votes to prevail; they re-gain leverage by not scheduling a mark-up. This was not an option for Waxman because Eshoo's bio-similars bill was offered as an amendment to the "must pass" health care reform bill.
Because of the breadth of mostly bi-partisan support for food safety legislation, it was possible for it to advance as regular legislation in both the House and Senate. However, procedural barriers have now stalled the Senate-passed version in the House and it might not become law. The House responded by adding food safety legislation to the FY 11 Continuing Resolution (CR) that will fund the government when the current one runs out at midnight on December 18. This tactic may not ultimately work, but the CR is definitely "must pass legislation."
With some exceptions, it is hard to anticipate what pieces of legislation become "must-pass." In theory, FDA might go an entire Congress without enactment of any major legislation. This can't be the case for the incoming Congress because authority to collect both prescription drug and medical device user fees expires on September 30, 2012. In the months prior to that date, Congress will face a choice: pass re-authorizing legislation or deprive the agency of $700 million in revenue each year. Thus, "user fee legislation" = "must-pass legislation."
User fees need to be renewed every five years. The last round of user fee re-authorizations resulted in the Food and Drug Administration Amendment Act (FDAAA). It is complicated and emerged after extremely tough negotiations. The final legislation was 155 pages long and had 11 separate Titles. It is at: http://www.gpo.gov/fdsys/pkg/PLAW-110publ85/pdf/PLAW-110publ85.pdf.
Commissioner Hamburg, Members of Congress, and most FDA stakeholders have wish lists of ways they would change FDA and the laws it implements. None of these--at least not any major ones--are likely to pass in 2011 without broad bi-partisan support.
But 2012 will be another story. In the process of adopting the "must pass" user fee reauthorizations, we can anticipate Congress considering dozens of provisions and programs that don't relate to user fees. Lawmakers and stakeholders will all have a chance to put their mark on the FDA in 2012.
Steven
Two Strategies for FDA Legislation in 2011 December 5th, 2010
The current Congress has two primary FDA-related accomplishments: an abbreviated approval pathway for bio-similar drugs; and a food safety bill that may be enacted before Congress adjourns. FDA Matters believes that any FDA-related legislation will falter in 2011 if it does not follow the strategy behind one or the other of these efforts. Read the rest of this entryy
FDA Funding and the Appropriations Drama November 28th, 2010
The process for passing appropriations bills should be similar from one year to the next. After all, it is a one-directional, regimented process. Yet, I have been through 30 or so appropriations cycles and each one seems to have its own unique story to tell. This year's efforts would make a riveting "made for television" movie "based on a true story." FDA Matters doesn't know how it will end, especially for FDA. Read the rest of this entry
FDA and Election 2010: Oversight and Investigations November 13th, 2010
President Obama's election and the health reform debate have distracted us from the disruption that "divided government" imposes on FDA. With the new Republican majority, the agency will find itself buffeted by political forces that are as concerned about "scoring points" as they are about improving government. FDA Matters thinks this will have a large impact on FDA, as well as the agency's stakeholders. Read the rest of this entr
FDA and Election 2010: Deficit Reduction and Appropriations November 6th, 2010
So-called "wave elections"–where one party overwhelms the other–are particularly hard to judge. The ground rules are going to change dramatically– in ways that no one can fully anticipate. At first, each side refuses to compromise. Then, something happens that sets the pattern for whether people will work together and on what issues. This may take months to resolve or may occur before the new Congress arrives. As things change, FDA Matters thinks there are some key issues for FDA-watchers to monitor. Read the rest of this entry
Two Strategies for FDA Legislation in 2011
The current Congress will be remembered for its 15-month battle to enact health reform legislation. The FDA-related accomplishments have been less visible: an abbreviated approval pathway for bio-similar drugs included in the health reform law; and a food safety bill that may be enacted before Congress adjourns later this month.In both cases, unanimity was never possible, but working majorities formed and prevailed. FDA Matters believes that any FDA-related legislation will falter in 2011 if it does not follow the strategy behind one or the other of these efforts.
The current Congress will be remembered for its 15-month battle to enact health reform legislation. The FDA-related accomplishments have been less visible: an abbreviated approval pathway for bio-similar drugs included in the health reform law; and a food safety bill that may be enacted before Congress adjourns later this month.
In both cases, unanimity was never possible, but working majorities formed and prevailed. FDA Matters believes that any FDA-related legislation will falter in 2011 if it does not follow the strategy behind one or the other of these efforts.
Bio-similars. The Democrats (Senator Kennedy and Representative Eshoo) leading the bi-partisan effort—and most of the Democratic rank and file who supported the legislation--come from high-tech, life sciences states and districts. Republicans joined the Democrats because of similar home-town support from life sciences companies and interest in promoting a pro-innovation, pro-economic development agenda.
The combination of Democrats with biotech interests and Republicans became a powerful force. It is also an approach that can be duplicated next year with a reasonable chance of success.
Indeed, another such effort is already underway regarding the medical device review process at FDA. Two weeks ago, 8 members of the Minnesota Congressional delegation sent Commissioner Hamburg a letter stating:
We support the FDA's work to improve the process for the approval of safe and effective Class II medical devices but we must work to ensure that we continue to foster life-saving innovation and growth….and a larger goal of saving and improving patients' lives….Changes that may jeopardize that goal should not be made unless there is clear evidence that the changes are necessary to address a public health problem.
The letter was signed by the state's 2 Democratic Senators, 3 of its Republican House members and 3 of its Democratic House members. Elsewhere, the letter mentions that Minnesota has over 500 medical device companies that employ almost 35,000 residents at average pay-levels almost 50% higher than the state average.
Food Safety. As with bio-similars, there was a widespread consensus that food safety reform legislation was needed. The similarities between the two efforts end there.
Hill staff and key constituency groups held extensive discussions about needed changes. After lots of disagreements and some very tough negotiating, most Democrats and Republicans found enough common ground to move forward in the House.
The Senate also evolved a compromise version—somewhat different from the House, but with a similar breadth of support. Action stalled in the Senate because of cost issues and concerns about small food producers. But the core of support never disappeared.
When those concerns were addressed in the Senate, the other extraneous issues and disagreements fell away. Even the House appeared to have signaled that they would accept the Senate bill, rather than face further negotiation that might delay action until next Congress. Such deference has become increasingly uncommon in Congress.
I don't want to minimize the difficulties in establishing and conducting negotiations on food safety. It may still fail. What made agreement possible was the willingness of constituency groups to work together, despite strongly held views that were often opposed. This approach can also work next year for other issues, as a broad-base of constituency groups lead Congress to a bi-partisan agreement on legislation.
The current Congress has not been known for its bi-partisanship. Some commentators think bi-partisanship may disappear altogether in the new Congress. Nonetheless, FDA is an area where getting the parties together is possible.
If consumer and patient groups, industry and associations and the Administration or Congress want to get anything done in 2011, they would do well to consider how the bio-similars and food safety strategies might apply to their cause.
Steven
The Minnesota delegation's letter to Commissioner Hamburg about changes in the process of approving medical device. http://www.hpm.com/pdf/MNLegConcerns.pdf
Some relevant past columns:
All FDA Stakeholders Affected by Medical Device Reforms October 31st, 2010
There are so many visible, contentious FDA issues right now….that reform of the medical device approval process has received only a fraction of the attention it deserves. Other centers at FDA and non-device stakeholders need to be watching more closely. FDA Matters is. Read the rest of this entry
FDA: An Honest Broker on the Slow Path to Bio-similars October 24th, 2010
FDA Matters enthusiasm for bio-similars is a matter of public record. The market will build slowly, but 10 years from now the new law will be seen as ushering in a new age of biopharmaceutical product development. Read the rest of this entry
Fall Scorecard for Follow-on Biologics September 11th, 2009
The creation of a regulatory pathway for follow-on biologics (FOB) has become a favorite topic of FDA Matters. The substance of the legislation is important and the politics are fascinating. It should get even better this fall. Read the rest of this entry
FDA Funding and the Appropriations Drama
The process for passing appropriations bills should be similar from one year to the next. After all, it is a one-directional, regimented process. It always starts with "total available funds" and a baseline of current spending programs and concludes when the President signs an appropriations bill into law.Yet, I have been through 30 or so appropriations cycles and each one seems to have its own unique story to tell. This year's efforts would make a riveting "made for television" movie "based on a true story." FDA Matters doesn't know how it will end, especially for FDA.
The process for passing appropriations bills should be similar from one year to the next. After all, it is a one-directional, regimented process. It always starts with "total available funds" and a baseline of current spending programs and concludes when the President signs an appropriations bill into law. And there are only 12 such bills that have to pass each year.
Yet, I have been through 30 or so appropriations cycles and each one seems to have its own unique story to tell. This year's efforts would make a riveting "made for television" movie "based on a true story." FDA Matters doesn't know how it will end, especially for FDA.
Here is a summary of the plot thus far:
The Democratic-controlled, but still badly divided Congress, failed to pass any of the 12 appropriations bills that fund the entire trillion-dollar federal government. Usually, Congress passes at least the Defense and Homeland Security bills. Past deadlocks have mostly been limited to the other ten bills.
In addition to disputes over total funding and specific program allocations, Congress has been bedeviled by a continuing fight over appropriations earmarks. In both Houses, there are Democrats and Republicans on both sides of the question. Some argue that earmarks are a wasteful, expensive process that epitomizes Congress' lack of commitment to deficit reduction. Others argue that earmarks are one of the few ways that Congress can reduce the discretionary prerogatives of the Executive Branch.
In late September, Congress passed a Continuing Resolution (CR) to fund government from October 1 (start of the new fiscal year) to December 3. The government will shut down at 12:01 a.m. on December 4 unless Congress passes another CR, an omnibus funding bill or individual appropriations bills. The current CR creates no priorities; virtually the entire government will have spent the first 9 weeks of the FY 11 fiscal year operating under the same ceilings as the FY 10 spending levels. While this postponed difficult decisions until after Election Day, a further short extension of the CR is possible to December 10 or 17.
Republicans picked up more than 60 seats in the House and will be the majority party starting in January. Democrats maintained control of the Senate for next year, but will go from a 59-41 advantage to a 53-47 margin. Understandably, Democrats want to accomplish as much as possible in the post-election session, while Republicans are inclined to defer most things to the new Congress. However, these do not reflect hard lines—what "might be possible" changes daily, sometimes hourly.
The House and Senate appropriations committees have tried to put together an omnibus FY 11 spending bill (i.e. decide on the content of all 12 funding bills separately, then roll them into one legislative package). Supposedly, they had leadership encouragement to do this, but there doesn't appear to be much enthusiasm outside of the appropriations committees. However, most of the hard staff work on this seems to be done. A less expensive version of the bill might have a chance to pass.
Where does FDA stand in this drama? Bad scenarios for the agency include a Continuing Resolution set at the FY 10 levels or, worse, a CR that imposes across-the-board cuts. The best scenario is the omnibus bill, on the assumption that this will incorporate decisions about priorities. Any funding bill in which some programs do better than others (which could be a CR as well as an omnibus), gives FDA the best chance to received increased resources for FY 11.
Steven
FDA and Election 2010: Deficit Reduction and Appropriations November 6th, 2010
So-called "wave elections"–where one party overwhelms the other–are particularly hard to judge. The ground rules are going to change dramatically– in ways that no one can fully anticipate. At first, each side refuses to compromise. Then, something happens that sets the pattern for whether people will work together and on what issues. This may take months to resolve or may occur before the new Congress arrives. As things change, FDA Matters thinks there are some key issues for FDA-watchers to monitor. Read the rest of this entry
Deficit Hawks in the New Congress Threaten FDA Funding October 17th, 2010
FDA Matters doesn't know who the majority party in the House and Senate will be next year. There seem to be a very large number of races where incumbents are vulnerable or are too close to call. The fate of FDA will be driven by the post-election tone of Congress, more than by the fates of individual Members or who holds the majority. Read the rest of this entry
FDA and Election 2010: Oversight and Investigations
The return of a Republican majority in the House of Representatives means an increase in Congressional oversight and investigations. This mirrors 2006, when the Democrats took back Congress and immediately started investigating the Bush administration.President Obama's election and the distraction of health reform have helped us forget the impact of divided government on FDA. Once again, the agency will find itself buffeted by political forces that are as concerned about "scoring points" as they are about improving government. FDA Matters thinks this will have a large impact on FDA, as well as the agency's stakeholders.
The return of a Republican majority in the House of Representatives means an increase in Congressional oversight and investigations. This mirrors 2006, when the Democrats took back Congress and immediately started investigating the Bush administration.
President Obama's election and the distraction of the health reform debate have helped us forget the impact of divided government on FDA. Once again, the agency will find itself buffeted by political forces that are as concerned about "scoring points" as they are about improving government. FDA Matters thinks this will have a large impact on FDA, as well as the agency's stakeholders.
In the House of Representatives, there are three committees that exercise "oversight and investigations" jurisdiction (O&I) over the FDA. The primary ones are the Energy and Commerce Committee and the Oversight and Government Reform Committee. The Appropriations Committee also conducts O&I.
The Energy and Commerce Committee intends to investigate the scope of work and expenditures under FDA's contract with McKinsey & Company, an international management consulting firm that has been asked to improve the productivity of the generic drug review office. Once the Energy and Commerce has reorganized itself for the new Congress and priorities are set, there are sure to be more areas under investigation.
The Oversight and Government Reform Committee is off to a faster start because outgoing chair Representative Edolphus Towns (D-NY) and incoming chair Representative Darrell Issa (R-CA) were already working together on manufacturing problems in the drug industry. This has now become an investigation of whether understaffing in FDA's Puerto Rico office led to inadequate oversight of industry.
FDA is under scrutiny all the time. Congress' watchdog arm, the General Accounting Office (GAO), regularly reports on areas where they believe FDA should do better. Likewise, the HHS Inspector General keeps a watchful eye on the agency. In a more helpful, but often no less critical mode, the Institute of Medicine also oversees many aspects of FDA programs.
What may make 2011 different?
Dr. Hamburg and Dr. Sharfstein can expect to spend countless more days preparing for and testifying at oversight hearings. Their involvement is the tip of the iceberg, with staff at every level being pulled from other work to respond to Congress. Some key staff that were able to focus on big picture issues this year...are likely to be in "damage control" mode most of next year.
FDA's competence will be questioned publicly, then magnified by the media. FDA has a lot of problems. Oversight can contribute to improvements. However, any loss of public confidence in FDA is a serious barrier to the agency doing its job better.
This may also lead to questions about whether the agency should receive more monies….when, in fact, many of the problems (not all of them) cannot be fixed without more resources. For example, unless you believe there are FDA staff sitting idle, how does understaffing in FDA's Puerto Rico office get resolved without a larger appropriation?
Oversight of food safety may lead to more effective use of resources, but cannot obviate the need for more inspectors. Oversight of medical products may lead to more conservative decisions by the agency, further slowing new therapies for patients.
As oversight of FDA ramps up, so too will oversight of industry. FDA Matters has exhorted CEO's to see quality controls, good manufacturing and improved sales and marketing practices to be integral to whether its products represent a public good. O&I is likely to be unflinching in its exposure of those who have not taken this to heart.
In the end, FDA will survive. Hopefully, it will be a better agency, while preserving its needed funding growth. Meantime, it is inevitable that FDA and industry will feel some pain.
Steven
My apologies to readers who may have received more than one mailing last week. The end of daylight savings time caused repeat distribution of several columns. Hopefully, it is now fixed.
FDA and Election 2010: Deficit Reduction and Appropriations November 7th, 2010
So--called "wave elections"–where one party overwhelms the other–are particularly hard to judge. The ground rules are going to change dramatically– in ways that no one can fully anticipate. At first, each side refuses to compromise. Then, something happens that sets the pattern for whether people will work together and on what issues. This may take months to resolve or may occur before the new Congress arrives. As things change, FDA Matters thinks there are some key issues for FDA-watchers to monitor. Read the rest of this entry
Quality Control Woes: What's a CEO to Do? June 2nd, 2010
Medical products companies are struggling to assure FDA and the American people that their products are "safe as manufactured and distributed." We don't know whether quality control has become lax, FDA is discovering more problems or industry has just had a run of bad luck. We do know that quality control relies on a lot of people maintaining tough standards…and that manufacturing is rarely a priority of a drug and device company CEO. FDA Matters asks: "what's a CEO to do?" Read the rest of this entry
Black, White, Shades of Gray November 13th, 2009
Civil and criminal investigations are becoming a more prominent feature in the world of FDA-regulated industries. People who never gave any thought to this….suddenly find themselves needing to understand how investigations work. Being FDA-regulated means "always worrying that you will have to say you're sorry." But it matters whether you are apologizing to FDA or trying to apologize to investigators. Read the rest of this entry
FDA and Election 2010: Deficit Reduction and Appropriations
So-called "wave elections"--where one party overwhelms the other--are particularly hard to judge. The ground rules are going to change dramatically-- in ways that no one can fully anticipate. At first, each side refuses to compromise. Then, something happens that sets the pattern for whether people will work together and on what issues. This may take months to resolve or may occur before the new Congress arrives. As things change, FDA Matters thinks there are some key issues for FDA-watchers to monitor.
As a result of the 2010 Mid-Term election, Republicans have netted more than 60 seats and taken control of the House of Representatives, effective in January. The Senate will remain in Democratic hands, but with a much slimmer majority.
So-called "wave elections"--where one party overwhelms the other--are particularly hard to judge. The ground rules are going to change dramatically-- in ways that no one can fully anticipate. At first, each side refuses to compromise. Then, something happens that sets the pattern for whether people will work together and on what issues. This may take months to resolve or may occur before the new Congress arrives.
As things change, FDA Matters thinks there are some key issues for FDA-watchers to monitor:
Regardless of how Democrats, Republicans and the President choose to interact in 2011, there will be a working majority in both House and Senate for significant deficit reduction.
- This is not a good budget environment for FDA or any federal agency with growing responsibilities.
- If deficit reduction is based on evaluating each agency and program and identifying national priorities, then the case for increased FDA funding is very strong.
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If deficit reduction is accomplished by "across the board" cuts, then FDA may still be treated as an exception. However, it will be far more difficult to make the case for FDA as a budget-cutting exception.
The direction of deficit reduction is yet-to-be determined.
- There is a large difference between campaign rhetoric and the realities of reducing the deficit.
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Congress has never been able to break "the iron triangle of deficit reduction:"
- Republicans don't want new taxes, making it hard to generate more revenue.
- Democrats don't want to change entitlement programs, making it hard to significantly decrease expenditures.
- Everyone's favorite punching bag--discretionary spending--isn't large enough or growing fast enough to produce deficit reduction on the scale required.
- Republicans don't want new taxes, making it hard to generate more revenue.
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Budget deficits are not calculated using ordinary math. For example:
- The Bush tax cuts expire at the end of this year. Re-instating them is an additional "cost" that will widen the deficit--even though tax rates will stay the same.
- Similarly, health care reform provided about $800 billion in savings to offset its $800 billion cost. Thus, repealing or defunding any parts of health care reform associated with cost savings….could wind up increasing costs and widening the deficit.
- The Bush tax cuts expire at the end of this year. Re-instating them is an additional "cost" that will widen the deficit--even though tax rates will stay the same.
The situation in both House and Senate is fluid with regard to committee assignments, chairmanships and leadership.
- Republicans in the House and Senate will be increasing their numbers on each Committee. A number of current Republican members will be moving up in seniority and may have new opportunities and choices to make.
- Democrats in the House and Senate will be decreasing their numbers on each committee. While this would normally tend to freeze committee positions, a number of very senior Democrats will not be back, opening up opportunities. One person's decision could affect the choices available to a dozen other members.
- In sum, expect changes in the committees that authorize, appropriate and oversee FDA.
The Alliance for a Stronger FDA has been preparing for this much tougher budget environment. The Alliance recognized a year ago that the federal budget situation was likely to deteriorate and has spent the intervening time building the case for FDA to be seen as an exception to budget-cutting. More recently, they have started talking about how a strong, well-resourced FDA is important to jobs and economic growth in the US.
For those readers who are not members, I strongly recommend joining. The membership includes consumer and patient advocacy groups, health professional societies, research advocacy groups, associations, companies, law firms and consulting firms, and individuals.
Every additional member strengthens the Alliance's position in advocating for increased funding for FDA.
Steven
For purposes of disclosure: I am one of the founders and Deputy Executive Director of the Alliance for a Stronger FDA. It is the only multi-stakeholder group devoted to education and advocacy to increase the appropriated resources available to FDA. For more information about the Alliance, go to www.StrengthenFDA.org or write to me at sgrossman@StrengthenFDA.org.
Deficit Hawks in the New Congress Threaten FDA Funding
October 17th, 2010
FDA Matters doesn't know who the majority party in the House and Senate will be next year. There seem to be a very large number of races where incumbents are vulnerable or are too close to call. The fate of FDA will be driven by the post-election tone of Congress, more than by the fates of individual Members or who holds the majority. Read the rest of this entry »
FDA Funding for FY 11: Back to the Future
October 3rd, 2010
Not so long ago, FDA's appropriation barely budged from year-to-year. A good year was a 2% to 3% increase. This changed four years ago, after the formation of the Alliance for a Stronger FDA. The agency's case for more resources—always a good one—finally had an independent, multi-stakeholder voice. Champions on the Hill and in the Executive Branch emerged.
The agency appropriation has grown 50% in the last three fiscal years. So far, FY 11 looks more like the past than it does the last few years. FDA Matters believes that the consequences could be severe. Read the rest of this entry »
All FDA Stakeholders Affected by Medical Device Reforms
There are so many visible, contentious FDA issues right now….that reform of the medical device approval process has received only a fraction of the attention it deserves. Other centers at FDA and non-device stakeholders need to be watching more closely. FDA Matters is.
There are so many visible, contentious FDA issues right now….that reform of the medical device approval process has received only a fraction of the attention it deserves. Other centers at FDA and non-device stakeholders need to be watching more closely. FDA Matters is.
The key area being examined is how FDA regulates Class II (medium risk) devices. Generally, they require a 510(k) Premarket Notification to be marketed. Under this process, a sponsor must show that a device is "substantially equivalent" to a device already marketed. The 510(k) is significantly less rigorous and time consuming than a full Product Marketing Application (PMA), the medical device equivalent of a drug NDA or a biologics BLA. A related area under review is the broad interpretation that "substantially equivalent" has been given.
Given the realities of the medical device industry, such an abbreviated process is necessary. In pharmaceuticals, product life cycles are typically 8 to 10 years or more after approval. In contrast, devices are being improved constantly. Products, as approved, might be on the market only 18 months to 2 years before the devicemaker is ready with improvements.
Thirteen months ago, FDA released an internal review of the agency's (mis)handling of the 510(k) device approval of a product from a company named ReGen. The report provided an inventory of issues to be re-evaluated and immediate impetus for FDA to improve the medical device approval process.
At the time, I expressed hope that FDA could formulate changes by itself, rather than give Congress a reason to consider amendments to the medical device statute. FDA also saw it this way and asked the IOM to formulate recommendations while the agency proceeded internally to develop its own proposals.
FDA released its recommendation in August, with public comments due in October. Industry agreed with some of the proposals, but was unhappy about the breadth of FDA's initiative and some specific recommendations. Some consumer groups thought the FDA proposals did not go far enough.
As a result of industry concerns, a bipartisan group of Members of the House Energy and Commerce Committee have written to Commissioner Hamburg asking FDA to slow down its implementation of changes. The group was careful not to endorse or criticize FDA's actions. The Members requested more transparency and reminded FDA of the importance to patients and the economy of medical device innovation. Similar caution and concerns are being expressed to FDA from the Senate side.
Why should other parts of FDA and non-device stakeholders care about these developments? First, FDA showed a rare capacity for self-criticism in the ReGen report. These types of inquiries will occur elsewhere at FDA when system failures occur.
Second, FDA has decided that it has the insight and statutory authority to substantially overhaul a major approval pathway. It has been doing so without close Congressional scrutiny. If food safety reform legislation isn't enacted this year, FDA may need to proceed in a similar manner. Other efforts may eventually be undertaken with drugs, biologics and other medical products.
Third, Congress may be taking a more bipartisan approach to FDA. A system of checks and balances may be evolving between Congress and FDA, where the agency is given room to develop policies under the arms-length scrutiny of Congress.
Fourth, the current effort is reminiscent of Representative Eshoo's successful efforts to add biosimilars legislation to health care reform. The key was that Democrats from states with biopharmaceutical companies were willing to join with Republicans in supporting changes in FDA's laws and actions. The current effort makes clear that medical device companies are capable of bringing together a similar coalition.
I recommend keeping an eye out for further developments in medical device reform. The lessons learned may well affect your own interests.
Steven
When Abbreviated May Not Mean Faster or Easier July 25th, 2010
FDA is working on an approval pathway for bio-similars, re-examining the way medical devices are reviewed, trying to upgrade the quality and speed of generic drug reviews and will soon be evaluating its process for granting accelerated approvals to drugs. These seemingly unconnected activities all have in common that they are supposed to be abbreviated processes to get new products to patients more quickly without risking safety or quality problems. Read the rest of this entry »
"No Surprise" That Medical Devices Are Under Scrutiny October 1st, 2009
Five weeks ago, I wrote a column entitled, "Re-Evaluating the Medical Device Approval Process." It was not widely-read. I assumed it was because everyone already knew that a review was underway at FDA with more activity coming. Apparently, I was wrong. Read the rest of this entry »
Re-evaluating the Medical Device Approval Process August 27th, 2009
Earlier this year, a GAO report concluded that many high risk medical devices have not been adequately reviewed. In June, the House Health Subcommittee held the first of what may be a series of hearings on medical devices. The media appears increasingly interested in medical devices and is raising more questions.
All these events are a prelude to FDA and Congress undertaking a major re-evaluation of the product approval process for medical devices. It would be a relief if FDA could diagnose and treat its own medical device problems, leaving the Congress and the media to watch. Read the rest of this entry »
FDA: An Honest Broker on the Slow Path to Biosimilars
">FDA Matters' enthusiasm for biosimilars is a matter of public record. The market will build slowly, but 10 years from now the new law will be seen as ushering in a new age of biopharmaceutical product development. FDA will present the next glimpse of the future on November 2 and 3, 2010, when it holds hearings on implementing the new approval pathway.The key to the future will be the FDA's strong commitment to expanding prescriber and patient choice among biological products. FDA will be satisfied (and successful) if the new law stimulates biosimilars, bio-betters, and innovative new biological products, along with a dramatic increase in knowledge about the nature and characterization of biologic products.
FDA Matters' enthusiasm for biosimilars is a matter of public record. The market will build slowly, but 10 years from now the new law will be seen as ushering in a new age of biopharmaceutical product development. FDA will present the next glimpse of the future on November 2 and 3, 2010, when it holds hearings on implementing the new approval pathway.
The key to the future will be the FDA's strong commitment to expanding prescriber and patient choice among biological products. FDA will be satisfied (and successful) if the new law stimulates biosimilars, bio-betters, and innovative new biological products, along with a dramatic increase in knowledge about the nature and characterization of biologic products.
Misunderstandings abound about the new law and how it is likely to reshape the biopharmaceutical landscape. Experts keep saying that innovator (reference) products have been granted 12 years of market exclusivity. Reading the sentences carefully, data exclusivity prevents biosimilar products from being approved through the new abbreviated biosimilars pathway for 12 years. It does not prevent approval of a biosimilar through a traditional biological license application (BLA).
There is also the perception that the biosimilars market will be limited to billion-dollar products and a few companies that have the capital to enter the marketplace. Despite this view, there are at least five or six companies ready to advance biosimilar and bio-better products over the next few years. More will come.
Over time, innovation will bring costs down and significantly lower barriers to market entry. Competition will bring prices down to competitive levels. Discounts may not be as low as those in the generic drug market, but significant savings will result from 20% to 30% discounts on drugs costing $50,000 per year and more.
We are told many things about the agency: it is going to lower standards, be extremely cautious, fail to develop adequate guidance for industry or proceed with no ground rules, etc. Passage of Hatch-Waxman 26 years ago elicited similar concerns. A quarter century later, that law has resulted in 70% of US prescriptions being filled with generic drugs.
The path to a more vigorous biopharmaceuticals market will not be easy. The law is not well-written and the patent provisions seem an additional barrier. FDA will be very cautious about the new approval pathway, but it may look favorably on biosimilars submitted as BLA's. Although I don't agree, it has been suggested that even a single serious safety problem for an approved biosimilar will kill the market. Also, at least one reimbursement expert has told me that a biosimilars market may never emerge because doctors lack financial incentives to use these products.
The Federal Register notice for next week's FDA hearings lists a careful series of questions upon which the agency wants comment. At the hearings, expect little, if any, feedback from FDA. They won't ask many questions either. At the risk of leaving everyone guessing, the agency will keep its own counsel, determined to be an honest broker among competing interests.
Guidances and regulations take years to develop and publish. FDA will proceed carefully and consistent with its public responsibilities. The impact will not be measured by how many products come through the new abbreviated pathway (perhaps not many) or how many products are deemed "interchangeable" (maybe none).
FDA's ultimate success will be the broad expansion of biopharmaceutical products. This will happen eventually, but patience will be required.
Steven
Background on the FDA hearing on biosimilars:
http://edocket.access.gpo.gov/2010/pdf/2010-24853.pdf
Information if you want to attend the hearing or watch the webcast:
http://www.fda.gov/Drugs/NewsEvents/ucm221688.htm
Data Exclusivity and Bio-Similars: Both More and Less Than It Seems
May 2nd, 2010
FDA Matters has been very upbeat about the prospects for the bio-similar marketplace. With this in mind, this column explores why there is a persistent belief that the bio-pharmaceutical industry got something better than data exclusivity. I also explore whether data exclusivity will really provide valuable protection for original reference biologic products. Read the rest of this entry »
March 21st, 2010
The long fight is over for follow-on biologic (FOBs). The world of biopharmaceuticals will never be the same, but not in the ways that many players expect. Here is FDA Matters' guide to understanding the next phase. Read the rest of this entry »
The Follow-on Biologics Market
Since the debate began several years ago, the policy and politics of follow-on biologics (FOB) have been driven by assumptions and projections of the anticipated market. FDA Matters believes there has been a lot of fuzzy thinking about what type of companies will be players and how they will position themselves. The Federal Trade Commission report, released last week, is just the latest illustration. Read the rest of this entry »
Deficit Hawks in the New Congress Threaten FDA Funding
FDA Matters doesn't know who the majority party in the House and Senate will be next year. There seem to be a very large number of races where incumbents are vulnerable or are too close to call. The fate of FDA will be driven by the post-election tone of Congress, more than by the fates of individual Members or who holds the majority.
FDA Matters doesn't know who the majority party in the House and Senate will be next year. There seem to be a very large number of races where incumbents are vulnerable or are too close to call. The fate of FDA will be driven by the post-election tone of Congress, more than by the fates of individual Members or who holds the majority.
Looking around the country, nearly every candidate is running "against Washington." The major themes--among Democrats and Republicans alike--are the need to create more jobs and the need to reduce the burgeoning government deficit and the national debt.
Congress may be stymied in the jobs area, since it is unclear how to intentionally create more jobs in our current economic situation. In any case, Congress will have to work through divergent views about how to approach this task. If creating jobs proves difficult, then Members will be working extra hard to show the electorate that the deficit is being tamed.
This year's first continuing resolution only provides FY 11 funds for the federal government through December 3. It has to be seen for what it is: a placeholder for more severe cutting that might occur later this year or early in the next Congress. Regardless of the outcome of the election, there will be a working majority of Republicans and Democrats in the House and Senate in 2011 that are deeply committed to substantial deficit reduction.
While it may surprise many, President Obama has a strong commitment to deficit reduction and will be working with newly-empowered deficit-cutters in Congress.
The White House clearly sees what the campaigns see: nearly across the political spectrum, voters want the federal government to spend less…or at least dramatically slow the increases that are already built into budget projections. The White House is likely to disagree with Congress on some budget items, but the focus will be on very specific items or on how far and fast to cut. Philsophically, President Obama sees himself as a deficit-cutter.
None of this sounds good for FDA. And it isn't. The Alliance for a Stronger FDA anticipated a year ago that future increases would be harder to get for FDA. Throughout 2010, the Alliance has been building on the theme that FDA needs to be an exception to whatever budget cutting occurs.
The Alliance's focus has been to show how a well-resourced FDA creates jobs in the American economy, which is a particularly potent argument with nearly 10% unemployment. The Alliance has also focused on the extraordinary demands being placed on the agency, be it growing responsibilities for medical product reviews and food imports or unexpected items such as pandemic flu and contaminated eggs.
The case for FDA "exceptionalism" has been effective in at least two recent years when most of the federal government received flat funding and FDA received an increase. No one knows if it will work in 2011 and 2012. FDA Matters does know that the agency will find it hard to function if it is caught in an "across the board" 5% or 10% cut in domestic discretionary spending.
It could be even worse. At least one proposal being discussed would roll the Federal Government's funding levels back to the FY 08 base. For FDA, this would mean reducing funding from $2.345 billion in FY 10 to the FY 08 level of $1.713 billion. A quarter of the agency might disappear.
Can I imagine things going that badly? Frankly, no. But so many things happen that we don't believe possible that I worry for the agency and hope readers will lend their voices to those advocating for increased FDA funding.
Steven
For purposes of disclosure: I am a founder and Deputy Executive Director of the Alliance for a Stronger FDA. It is the only multi-stakeholder group devoted to education and advocacy to increase the appropriated resources available to FDA. Members include patient and consumer groups, professional societies, research advocacy groups, associations, companies, consultants and individuals. For more information about the Alliance, go to www.StrengthenFDA.org or write to me at sgrossman@StrengthenFDA.org.
FDA Funding for FY 11: Back to the Future
October 3rd, 2010
Not so long ago, FDA appropriations barely budged from year-to-year. A good year was a 2% to 3% increase. This changed four years ago, after the formation of the Alliance for a Stronger FDA. The agency's case for more resources—always a good one—finally had an independent, multi-stakeholder voice. Champions on the Hill and in the Executive Branch emerged.
The agency appropriation has grown 50% in the last three fiscal years. So far, FY 11 looks more like the past than it does the last few years. FDA Matters believes that the consequences could be severe. Read the rest of this entry »
Success is Uncertain for FDA’s Regulatory Science Initiative
FDA Matters was an early advocate for regulatory science. It has been exciting to see the concept grow from the Commissioner's first public speech to the President's request for $25 million in FY 2011. And now, FDA has released a White Paper describing the Regulatory Science Initiative (RSI).It is an excellent report and I applaud those who worked hard to create it. Still, I have misgivings about the way the White Paper characterizes regulatory science, leading to concerns about whether RSI will develop the necessary political and public support to be a long-term, permanent part of FDA.
FDA Matters was an early advocate for regulatory science. It has been exciting to see the concept grow from the Commissioner's first public speech to the President's request for $25 million in FY 2011. And now, FDA has released a White Paper describing the Regulatory Science Initiative (RSI).
It is an excellent report and I applaud those who worked hard to create it. Still, I have misgivings about the way the White Paper characterizes regulatory science, leading to concerns about whether RSI will develop the necessary political and public support to be a long-term, permanent part of FDA.
According to the report:
"Regulatory science is the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality and performance of FDA-regulated products." (emphasis added).
I would suggest that:
"Regulatory science is the tools, techniques and knowledge needed by food, medical product and other FDA regulators to carry out their public responsibilities."
FDA's definition makes regulatory science about what FDA develops (new tools, standards, etc.) and about regulated products. Instead, it should be about how regulatory science makes it possible for FDA to carry out its public health mission in an increasingly complex scientific and global environment. It needs to be clear that consumers, patients and regulated industries benefit when regulators have sophisticated, state-of-the-art capabilities and use them transparently, so that no stakeholder has to guess about the agency's approach. These are public purposes for which it is possible to gather broad support.
Most agency funds are spent to carry out applied regulatory science. A smaller, but not insignificant part of the agency budget is already being spent to create and improve regulatory science. For example, much of the budget of the Center for Food Safety and Nutrition that is not spent on inspections and enforcement is focused on better tools and knowledge to create a safer food supply. Most of the work of the National Center for Toxicological Research is devoted to improving regulatory science through the creation of science-based standards.
Regulatory science is not new and is much larger than an agency initiative. Rather, it is a re-conceptualization of who the agency is and what it does. Without this larger and more visionary framework, I fear that RSI is going to be underfunded by Congress and pushed aside when dollars are tight.
What reason is there to think so? The Critical Path Initiative (CPI) was launched with significant fanfare. The original report was one of the most lucid statements I have ever read from a government agency. The projects that Critical Path supported appear to have been well-conceived and responsive to broad, serious regulatory science needs within FDA.
Yet, the Critical Path completely failed to capture Congress' imagination. It received very modest financial support from Congress, even in the last few years when the FDA's budget expanded significantly.
RSI risks the same failure if Congress and other policymakers can neither explain "regulatory science" nor understand why it is fundamental to FDA's mission success. As judged by CPI funding and the limited enthusiasm that it generated, Congress saw it as a nice add-on, but not a necessity.
If RSI can't do better than this, it will not grow and prosper once the initial excitement fades. This would be a great shame because improving regulatory science is essential to making FDA into a 21st century regulatory agency.
That's why it matters what FDA says now about the RSI. Undoubtedly, the agency wants regulatory science to be seen as a better, more holistic way to understand and improve the agency. At the moment, it still looks more like a collection of worthwhile agency improvements that have been bundled into a new initiative.
Steven
The FDA White Paper, Advancing Regulatory Science for Public Health, can be found here, along with the Commissioner's speech on the topic at the National Press Club: http://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/default.htm
One of several earlier FDA Matters' columns on regulatory science:
CARS: The Vehicle for FDA's Future October 25th, 2009
Since Labor Day, Commissioner Hamburg has spoken a number of times about the importance of regulatory science. She is right. FDA must have the scientific tools and methodologies to be a 21st century regulatory agency. FDA needs to define regulatory science, develop programs to support it, and package them in a way that will quickly bring recognition and funding. Read the rest of this entry »
FDA Funding for FY 11: Back to the Future
Not so long ago, FDA's appropriation barely budged from year-to-year. A good year was a 2% to 3% increase. This changed four years ago, after the formation of the Alliance for a Stronger FDA. The agency's case for more resources—always a good one—finally had an independent, multi-stakeholder voice. Champions on the Hill and in the Executive Branch emerged.The agency appropriation has grown 50% in the last three fiscal years. So far, FY 11 looks more like the past than it does the last few years. FDA Matters believes that the consequences could be severe.
Not so long ago, FDA's appropriation barely budged from year-to-year. A good year was a 2% to 3% increase. This changed four years ago, after the formation of the Alliance for a Stronger FDA. The agency's case for more resources—always a good one—finally had an independent, multi-stakeholder voice. Champions on the Hill and in the Executive Branch emerged.
The agency appropriation has grown 50% in the last three fiscal years. So far, FY 11 looks more like the past than it does the last few years. FDA Matters believes that the consequences could be severe.
For the new fiscal year (FY 11) starting on October 1, Congress passed a Continuing Resolution (CR) that funds the government until December 3. As a result, the government is open, but no agency received new funding. For the next 2 months, government agencies (including FDA) will only be able to spend the amount they were appropriated in fiscal year 2010.
One of this year's Alliance themes has been the need for FDA to be an exception to the overall dismal federal budget situation. The agency must receive funding increases to do its job, which grows larger every day. There is strong support for this in Congress. The Alliance expects FDA to receive serious attention anytime Congress debates funding priorities.
This didn't happen for this first round of the CR for a fairly simple reason: Congress put this CR together without acknowledging any funding priorities. The Alliance believes FDA can win the debate to get increased funding, but only if that debate occurs.
When Congress returns in mid-November and looks at FY 11 funding again, there will be an opportunity to make the case that FDA should receive more funds. However, it is only a guess that this debate will occur before December 3. Congress may decide to pass the same CR to cover the period from December 3 to February 4. If so, the pain of level funding will only become more intense for FDA.
Funding FDA at last year's levels is a particularly acute problem because over 80% of the agency's budget is people-related costs: salary, benefits, rent, IT, travel, support services. FDA cannot delay spending, as NIH might, by waiting a few extra months to disburse grant funds.
FDA management is reviewing contingency plans to preserve its priorities and maximize output. Retaining the fiscal 2010 spending levels will impact FDA's ability to hire medical product reviewers and food inspectors and personnel for other areas of growing need. The agency must use its CR funding to pay for October 1 raises and rent increases, which obviously are not reflected in the FY 10 appropriations level.
The overall situation, as well as these new costs, restricts FDA's ability to make commitments in hiring, new programming, better IT, and contracts. They will also not want to create obligations now that they may not be able to fund later in the year.
Congress will eventually be ready to address the nation's funding priorities, hopefully in November. Regardless of when this debate occurs, all of FDA's supporters will need to rise up as one to convince Congress that more monies are needed.
Steven
For purposes of disclosure: I am a founder and Deputy Executive Director of the Alliance for a Stronger FDA. It is the only multi-stakeholder group devoted to education and advocacy to increase the appropriated resources available to FDA. Members include patient and consumer groups, professional societies, research advocacy groups, associations, companies, consultants and individuals. For more information about the Alliance, go to www.StrengthenFDA.org or write to me at sgrossman@StrengthenFDA.org.
FDA's Yearly Appropriations Growth Since FY 2003
FY 03 (base year): $1.390 billion
FY 04 ($11M increase): $1.401 billion
FY 05 ($51M increase): $1.452 billion
FY 06 ($41M increase): $1.493 billion
FY 07 ($77M increase): $1.569 billion (most domestic programs received no increase)
FY 08 ($145M increase): $1.714 billion (plus $150M one-time supplemental, non-add)
FY 09 ($325M increase): $2.039 billion
FY 10 ($306M increase): $2.345 billion
FDA: A Hit and A Miss
">FDA Matters has watched FDA handle the Avandia decision differently from any prior controversy. I like the new approach. In the same week, FDA provided a status report on its long-overdue social media and Internet communications policy. Because the agency's efforts have been glacial, the prospect of useful guidance is dim. I think this is a serious problem.
FDA Matters has watched FDA handle the Avandia decision differently from any prior controversy. I like the new approach. In the same week, FDA provided a status report on its long-overdue social media and Internet communications policy. Because the agency's efforts have been glacial, the prospect of useful guidance is dim. I think this is a serious problem.
FDA's Handling of the Avandia Situation. A HIT
FDA Matters was heartened by the agency's deft handling of the Avandia situation. Previous columns have explored how hard it is to achieve cultural change at FDA and why it requires the Commissioner and the agency's senior leadership to be role models.
With the Avandia decision, the agency has demonstrated progress toward two related changes: improved transparency and allowing internal dissent to become part of the public dialog.
Transparency is not part of the FDA's DNA. Despite this, the agency has responded to the President's government-wide transparency initiative with a serious effort. In doing so, it has struggled--mostly against itself--in making its processes and decisions more readily available within and outside FDA.
The Avandia advisory committee meeting and the FDA website were remarkably transparent about the controversy and the details. To clarify the ultimate decision, Drs. Woodcock, Sharfstein and Hamburg authored a 1500-word explanation, which appeared in the New England Journal of Medicine.
Embracing dissent has always been hard for FDA. Its customary position of "speaking with one voice" is not an accurate reflection of what happens when well-trained, analytically-oriented people gather to make a decision. With Avandia, Commissioner Hamburg has conspicuously incorporated dissent into the process.
Cultural change at FDA is difficult and always takes time. The Avandia decision is a significant step forward, demonstrating that agency leadership is committed to changes and understands that it must serve as role models.
FDA's Handling of Policy Development for New Media: A MISS
As a rule, businesses do not like additional regulation. There are two exceptions: where it levels the playing field with competition and when it creates certainty on how companies should conduct their activities. These are the exact reasons why FDA-regulated medical products companies have repeatedly asked FDA to issue policy guidance on Internet activities.
As best I can tell, FDA's first hearing, "FDA and the Internet: Advertising and Promotion of Medical Products" occurred on October 16 and 17, 1996. http://www.fda.gov/AboutFDA/CentersOffices/CDER/ucm175775.htm. That's 14 years ago!
While FDA has studiously ignored the opportunity to provide policy guidance and regulation, the Internet has become a primary means of written and visual communication. Search engines control an ever expanding numbers of online interactions. Social media has flourished.
Apart from agency action against some companies for activities that many consider ethical and reasonable and a two-day hearing in November 2009…not much progress seems to have occurred toward providing more direction to industry. At a Food and Drug Law Institute meeting last week, the agency suggested a draft guidance--the first of many--may be issued later this year. Even assuming this occurs on schedule, it will take at least a year to finalize.
I suggested last year that FDA was mistaken to see Internet policy development as overwhelming. Rather, it is a number of smaller issues that can be addressed separately without a broad Internet policy. An additional advantage of this approach: it recognizes that any Internet policy guidance will be outdated before FDA can ever issue it.
After 14 years, this calls for the intervention of agency leadership, not continued delays.
Steven
Gardner Harris of the New York Times also observed the changes at FDA that have occurred as part of the Avandia decision. His column is at: http://www.nytimes.com/2010/09/25/health/policy/25avandia.html?_r=1&emc=eta1.
For those interested in more coverage of FDA and social media, I recommend Mark Senak's Eye on FDA column at www.eyeoffda.com.
Is the New FDA Different than the Old FDA?
June 13th, 2010
Two weeks ago, FDA Matters explored Dr. Hamburg's legacy, focusing on advocacy for resources, prioritizing regulatory science and upgrading enforcement. These will be accomplished before she leaves office. But is she making similar progress in creating "a new FDA, including changes in agency culture?" Read the rest of this entry »
Dissent and Efficiency: Difficult Trade-offs for FDA
May 9th, 2010
FDA has a reputation for being tough on dissent, whether it comes from employees or regulated companies. Whatever the truth has been in the past, FDA is trying to develop an institutional cultural that welcomes and accepts dissent from employees, industry and other stakeholders. It is difficult, even messy, to do this. Read the rest of this entry »
Internet Communications: FDA Needs to Divide the Issues to Conquer the Problem
December 2nd, 2009
Creating an Internet communications policy for regulated medical product companies is so daunting that FDA has largely ignored the responsibility. New policy will not be announced anytime soon. FDA needs a different approach. This is not a matter of a large, complicated problem with many facets. Rather, it is a number of smaller problems that can be addressed separately. Read the rest of this entry »
ACRA: Little Attention to an Important Appointment
For more than a year, FDA Matters has talked about the position of Associate FDA Commissioner for Regulatory Affairs (ACRA), who is the agency's chief officer for inspections, enforcement and compliance. Of FDA's appropriated (non-user fee) budget, the ACRA oversees one-third of the agency's monies and more than 40% of the staff. It is FDA Matters that dubbed the ACRA the "uncrowned prince" of FDA. Because of the importance of ACRA and the level of resources it receives, we have been awaiting a new appointee to this long-vacant position. This has now occurred, but the announcement was so unassuming as to raise concerns.
For more than a year, FDA Matters has talked about the position of Associate FDA Commissioner for Regulatory Affairs (ACRA), who is the agency's chief officer for inspections, enforcement and compliance. Of FDA's appropriated (non-user fee) budget, the ACRA oversees one-third of the agency's monies and more than 40% of the staff. It is FDA Matters that dubbed the ACRA the "uncrowned prince" of FDA.
Because of the importance of ACRA and the level of resources it receives, we have been awaiting a new appointee to this long-vacant position. This has now occurred, but the announcement was so unassuming as to raise concerns.
Dara Corrigan, JD, will be the new ACRA and run the Office of Regulatory Affairs (ORA). She is a veteran of the HHS Inspector General's office and has also held policy positions at HHS. We wish her well. We also hope that she and the Commissioner can return this position to the visibility it needs.
Congressional and media attention are increasingly focused on FDA's capacity to perform effective inspections and rigorously enforce the law. The agency's good name and public credibility are tied to success in these areas.
Since the Commissioner has so many roles, she needs someone to be the highly-visible, public face of tough enforcement at FDA. Two decades ago, when I worked at HHS, the Inspector General was a former professor who had become the supervisor of the organized crime units in the FBI's Chicago Office. He was a good, smart man and a friend…but you knew immediately that you didn't want to be a target of one of his investigations.
FDA needs Ms. Corrigan to perform this function on behalf of ORA and FDA.
Well run, conscientious companies have little to fear. If you run a solid plant operation, import ingredients with care, use multiple system controls, and renew your commitment to pedigree and chain of custody, you are unlikely to be affected by a stronger ORA. If you have an inspections or enforcement problem: cooperate with FDA and correct it quickly.
On the other hand, if you are cutting corners, heedless of consumer and patient risk, or stonewalling the agency, you deserve what you get from FDA.
Commissioner Hamburg is working to make the agency more scientifically knowledgeable, more innovation-oriented and a more reliable partner in its interactions with industry and other stakeholders. She doesn't have the leeway to accomplish these goals if she doesn't continue to strengthen inspections and enforcement.
It makes sense to take ORA out of the FDA shadows and make it a more visible force. The initial announcement did not live up to this.
Steven
Commissioner Hamburg's Most Important Personnel Decision
February 21st, 2010
With due respect to the many fine individuals that Commissioner Hamburg has recruited, FDA Matters thinks the most important appointment needs to be made soon: choosing the right person to be Associate Commissioner for Regulatory Affairs. Read the rest of this entry »
The Uncrowned Prince of FDA
September 15th, 2009
Which FDA line manager has the most appropriated resources to work with in FY 09? Is it Janet Woodcock, head of the drug center or Stephen Sundlof, [then]head of the food center? The correct answer: neither. Read the rest of this entry »
FDA and Quran Burning: Trouble Can Start With a Tweet
Bravo! Florida Pastor Terry Jones has decided not to burn a copy of the Quran. The next danger is that the Pastor's "success" will be seen narrowly as the unique confluence of 9/11, the Ground Zero mosque, and the readiness of millions to take to the streets at signs of American intolerance toward Muslims.FDA Matters thinks the lessons are larger and urges FDA to pay attention to how this reflects changes in the way crises develop and decisions are made.
Bravo! Florida Pastor Terry Jones has decided not to burn a copy of the Quran. The next danger is that the Pastor's "success" will be seen narrowly as the unique confluence of 9/11, the Ground Zero mosque, and the readiness of millions to take to the streets at signs of American intolerance toward Muslims.
FDA Matters thinks the lessons are larger and urges FDA to pay attention to how this reflects changes in the way crises develop and decisions are made.
According to the Washington Post, on July 12 the Pastor sent a simple message on Twitter: "9/11/2010 Int Burn a Koran Day." From that beginning, he became the focus of international concern and one of the most watched people on the planet. All news sources—print, television and internet—were filled with stories about his intended actions. The situation most resembled a dramatic, high-stakes hostage negotiation. In a sense, it was, with Pastor Jones'copy of the Quran as hostage.
If we are imaginative, there is much more to concern us than Pastor Jones. We now know that one man's temper tantrum (or cause?)—distributed via Internet, Twitter, YouTube, 24/7 news outlets, etc.—can leave the world's leaders pleading for a favorable outcome. The power to capture the public eye, move public sentiment and galvanize the world...now belongs to all us. As Pastor Jones demonstrated, not everyone has the restraint to use that power responsibly.
In the late 1980's, AIDS activists staged a large demonstration that caused a multi-day lock-down situation at FDA. Patients and their friends were expressing their desperate need for access to drugs in development. It was a scary time at the FDA. No one wanted to create the precedent that FDA "would bend to a mob," but there also was a lot of sympathy for the plight of HIV patients.
Soon thereafter and over the next few years, FDA made a number of significant changes to the drug approval process. For example, the demonstrations eventually led to an accelerated approval process and "fast-tracking" of certain new drug applications. FDA began to treat surrogate endpoints seriously (e.g. CD4 viral load count rather than HIV disease progression). Most of these changes have been judged "good" with the passage of time, but there was a lot of uncertainty at the time.
With all the new avenues for communication, this scenario could easily repeat itself today on behalf of any number of diseases that are life-threatening or crippling. Could a group of parents with dying children try to force FDA to grant compassionate use access to a drug in pre-clinical (animal) testing? To make their point, the parents could create a vigil for the children with countdown clocks, while posting daily video showing their deterioration.
Yet another scenario might blend the skills of the Tylenol poisoner with the long-term persistence of the Unabomber. Every three months, anonymous videos might be posted that show gloved hands adulterating some additional batches of a food, drug or device. This would be a corporate nightmare that would also bring all FDA-regulated products under suspicion. FDA would be at the center of an enormous public health crisis.
I know I have not reflected the depth or breadth of challenges FDA could face. Hopefully, these few make the point that FDA is increasingly vulnerable as an agency. It must prepare for risks well beyond those faced by most federal agencies.
Commissioner Hamburg ought to meet soon with senior staff to review, extend, and refresh FDA's crisis plans. Even if they met two weeks ago, they should do it again because Pastor Jones has proven that the world has changed.
And while senior staff is having this discussion, they should also plan some simulations and practice drills. A crisis plan is only as good as the ability to implement it.
Steven
The Washington Post story describing the escalation after the tweet is at: http://www.washingtonpost.com/wp-dyn/content/article/2010/09/10/AR2010091007033.html?hpid=topnews
FDA’s “Summer of Safety Concerns”
One of industry's great fears is that FDA will become obsessed by theoretical or miniscule safety concerns and ignore the difficult realities of providing consumers with a varied and plentiful food supply and providing patients with effective medical therapies.It is even possible to think this has occurred. It has been a long summer of media and Congressional attention to safety: whether drug manufacturing, medical products already on the market or Salmonella contaminated eggs. A closer look suggests to FDA Matters that theoretical safety risks and inappropriate FDA concerns about safety are not the issue.
One of industry's great fears is that FDA will become obsessed by theoretical or miniscule safety concerns and ignore the difficult realities of providing consumers with a varied and plentiful food supply and providing patients with effective medical therapies.
It is even possible to think this has occurred. It has been a long summer of media and Congressional attention to safety: whether drug manufacturing, medical products already on the market or Salmonella contaminated eggs. A closer look suggests to FDA Matters that theoretical safety risks and inappropriate FDA concerns about safety are not the issue.
As laid out in more detail in "Safe": Many Meanings Complicate FDA Policymaking, there is a strong tendency to think of FDA's safety mission as if it were one type of activity. There are at least six different meanings of "safe" that are relevant to FDA.
For example, we want our food to be "inherently safe," "safe from intentional and negligent contamination" and "safe from unintentional contamination." Much of FDA's attention to food safety this summer has been about unintentional contamination (seafood in the Gulf and eggs with Salmonella).
The agency needs more resources for food inspections and, in the case of eggs, it should benefit from implementation of new standards that were already being phased in. New food safety legislation should also help, but only if Congress appropriates the funds for it to work properly. Whatever substantive or political barriers exist to passage of this legislation, there does not appear to be widespread disagreement about FDA's role or the standards it applies in assuring food safety.
We want medical products (drugs, biologics and devices) to be "safe as tested pre-approval,""safe as used post-approval" and "safe as manufactured and distributed." None of this summer's medical product safety issues appear to be about nuances of safety or shifts in FDA's approach to balancing risk and benefits in these three areas of safety concern.
Concern over potential cardiovascular risks from the diabetes drug Avandia existed before its approval. I do not see this as an issue of FDA standards or focus—but rather conflict over the proper interpretation of studies and data. Policymakers and industry should be wary of drawing any larger meaning from this about FDA's policies on safety.
Much of the rest of the summer's medical product safety issues were about "safe as manufactured and distributed." There seems to be consensus—even among those with problems-- that real lapses occurred and were violations of reasonable safety policies. As described in prior columns, this requires CEO's to take manufacturing and distribution seriously, rather than for FDA to re-think its existing safety policies.
While this may have been FDA's "summer of safety concerns," there doesn't seem to have been any change in how the agency balances competing demands involving safety. As the FY 11 appropriations process comes to a conclusion this Fall, there is good reason to put more funding into safety analysis and enforcement. Drawing any larger conclusions is, at best, premature and probably unwarranted.
Steven
Since before Memorial Day, FDA Matters has blogged extensively about FDA and safety issues, although this was not planned. Here is a sampling of columns:
"Safe": Many Meanings Complicate FDA Policymaking May 23rd, 2010
FDA Matters is in favor of safe foods and safe medical products. Who isn't? If you are a consumer, maybe that's all that matters. However, being in favor of safe foods and safe medical products is not enough if you are FDA, the media, Congressional authorizers and appropriators, OMB, and industry. It sounds good, but what does it really mean? In the FDA context, "safe" means many things, some of which are barely related to each other.
Read the rest of this entry »
Not Too Soon to Consider the Hamburg Legacy May 27th, 2010
May 18 marked one year since Dr. Margaret Hamburg was sworn in as Commissioner of the US Food and Drug Administration. The challenges are great, the torrent of issues is never-ending and most days you can smile but you can't win. Nonetheless, I think it has been a very good first year for her and for Principal Deputy Commissioner, Dr. Joshua Sharfstein. It may seem premature to be discussing "the Hamburg legacy." But you know that she is thinking about it (all commissioners do), so why can't FDA Matters talk about it? Read the rest of this entry »
Quality Control Woes: What's a CEO to Do? June 2nd, 2010
Medical products companies are struggling to assure FDA and the American people that their products are "safe as manufactured and distributed." We don't know whether quality control has become lax, FDA is discovering more problems or industry has just had a run of bad luck.
We do know that quality control relies on a lot of people maintaining tough standards…and that manufacturing is rarely a priority of a drug and device company CEO. Earlier this year, in the wake of Toyota's problems, FDA Matters asked: "what's a CEO to do?" Read the rest of this entry »
FDA to Industry: Contractors R U June 17th, 2010
It seems a rather uncontroversial proposition: FDA-regulated companies are responsible for their vendors, including every contracted piece of work that is done on the company's behalf. If problems develop, it makes no difference whether a company did it…or a contractor did it for them. Two seemingly unrelated items this week suggest that FDA is becoming concerned about whether FDA-regulated companies are overseeing their vendors. Read the rest of this entry »
Hot Town, Summer in the City—2010 July 11th, 2010
For the news media, the only FDA story this coming week will be the two-day advisory committee meeting reviewing the diabetes drug, Avandia. Missing from public dialogue is the extraordinary (perhaps unprecedented) number of large, consequential projects that FDA will be working on this summer. Every part of FDA is involved in some initiative that could become a "game-changer" for the agency. Read the rest of this entry »
Benefit, Risk and the Coming Age of REMS August 8th, 2010
FDA supposedly swings back and forth between emphasizing "expedited approvals of promising therapies" and "extended pre-approval examination of every safety issue." Current thinking is that FDA is now leaning more toward the safety end of this spectrum.
FDA Matters thinks a lot of this is perceptual. Approval decisions reflect FDA's honest and relatively clear judgment on medical need, quality of the clinical data, and the risks and benefits of a specific product. Mostly, I can understand FDA's decisions, even when I don't agree. Still, there is a lot of tension within FDA and with various stakeholders about approvals versus safety risk. Read the rest of this entry »
FDA’s Pivotal Role Fighting Bioterrorism and Emerging Infectious Diseases
With Congress out of session until September 13, the Executive Branch has the opportunity to gain extra column inches and media bandwidth. Thus, last week's report on medical countermeasures (MCM), released by HHS Secretary Sebelius, drew a lot of interest and a minimum of Congressional comment. The Secretary released the findings and recommendations from a top-to-bottom review of the Department's efforts with regard to the development of MCM. In the view of FDA Matters, the report thrusts FDA back into its rightful place as a key agency deserving more resources and respect for its national security responsibilities.
With Congress out of session until September 13, the Executive Branch has the opportunity to gain extra column inches and media bandwidth. Thus, last week's report on medical countermeasures (MCM), released by HHS Secretary Sebelius, drew a lot of interest and a minimum of Congressional comment.
The Secretary released the findings and recommendations from a top-to-bottom review of the Department's efforts with regard to the development of MCM. In the view of FDA Matters, the report thrusts FDA back into its rightful place as a key agency deserving more resources and respect for its national security responsibilities.
MCM are products that will decrease morbidity and mortality from a bioterror attack or from naturally occurring emerging infectious diseases. Think anthrax or radioactivity from an improvised nuclear device for the first, think H1N1 influenza for the second.
Scientifically and medically, these are difficult products to discover and develop. Financially, they won't ever be developed without:
- federal assistance to promising research; and
- a strategic national stockpile and government contracts that will buy proven MCM's.
As with the larger promise of moving medical therapies from "bench to bedside," there is no progress without FDA. The agency encourages companies by helping them to define appropriate safety and efficacy endpoints for their particular MCM and works with them to resolve questions of animal models, lab standards, statistical plans, quality manufacturing, etc.
Then, the agency evaluates the testing results and determines whether to approve the product. This work has an additional wrinkle. With most MCM's (maybe all) it is unethical to do human efficacy trials (e.g. intentionally expose a human being to anthrax to see if the MCM works). Instead, the agency (and the company) must make the difficult evaluation as to whether efficacy in animals is a sufficient surrogate for efficacy in humans.
The Secretary (and the underlying report) found that FDA's efforts in this area are insufficiently funded. Perhaps for the first time, there was a more global recognition of FDA's central role in making us safer from bioterrorism and naturally occurring emerging infectious diseases. The Secretary also recognized that FDA needs resources above its current level to do this job well.
The Administration placed a price tag--$170 million in funds to be available until expended--on the size and scope of the monies needed to upgrade FDA's efforts in this area. The monies will come from dollars previously appropriated to HHS to combat pandemic flu. HHS and OMB have agreed that the monies can be transferred administratively as long as they retain their original purpose of helping to deal with pandemic flu.
However, the Secretary's recommendation is for the transferred funds to also be used for non-pandemic medical countermeasures. This requires a budget amendment to be sent to Congress to broaden the permitted uses of these funds. How likely is Congress to approve this? We probably won't know until after September 13.
What we do know for sure is that FDA can use the additional resources and America will ultimately be safer as a result.
Steven
FDA Matters' most recent (and still accurate) assessment of FDA and the FY 2011 appropriations process:
Update on FDA's Appropriation for FY 11 July 18th, 2010
On July 1, the House Appropriations Agriculture/FDA Subcommittee marked up it FY 11 bill. The bottomline was good for FDA: $2.571 billion, a $214 million increase over FY 10, about 9%. No further details will be released until the full committee marks up.
On July 15, the Senate Appropriations Committee marked up its version of the Agriculture/FDA funding bill. The good news is that the Senate agreed that FDA needs better funding….and provided $2.516 billion, a $158 million in new monies. This is a bit more than 6%. Here is FDA Matters' analysis of this critical budget battle. Read the rest of this entry »
For Those Who Don’t Work With Patients: A Reality Check
Guest Column: Summer, Camp, Kids, CancerBy Margaret Anderson, Executive Director, FasterCuresWhile we focus on improving the efficiencies of the system that discovers treatments and cures for disease, there are untold numbers of people taking a medical treatment journey right now. For the kids partaking in the 28th year of Camp Fantastic in Virginia this week, they get to focus more on the fun, and less on the challenges of coping with a cancer diagnosis and with treatment.
Guest Column: Summer, Camp, Kids, Cancer
By Margaret Anderson, Executive Director, FasterCures
While we focus on improving the efficiencies of the system that discovers treatments and cures for disease, there are untold numbers of people taking a medical treatment journey right now. For the kids partaking in the 28th year of Camp Fantastic in Virginia this week, they get to focus more on the fun, and less on the challenges of coping with a cancer diagnosis and with treatment. Camp Fantastic is a program of a nonprofit called Special Love that gives cancer families support.
I learned of this amazing place from Kathy Russell, who has been involved since its beginning and who also runs the Children's Inn at NIH. The NIH Children's Inn helps families with kids in treatment at the NIH Clinical Center to get a bit of normalcy in their lives by providing a warm atmosphere for them to stay in versus an isolating hotel room. The overriding philosophy is that families make a key difference in the lives of their sick children. The work they do represents the full spectrum of NIH investment – from bench to bedside.
By the end of my chat with Kathy, after I dabbed my eyes, I was ready to pack my bags and tell everyone I knew to come with me to help prepare Camp Fantastic and allow kids there to take a break from cancer and be just kids. This year there will be nearly 100 kids at Camp Fantastic between ages 7-17. Usually one-half of them are in active treatment and there are upwards of 60 medical professionals (in addition to countless other folks) who volunteer their time before and during the camp. They literally set up a mini-hospital on-site because it's in a remote location and far from a hospital with specialized pediatric oncology services. She told me of how kids get their bloodwork done in the am, and a van takes the samples into NIH to do labs and then turns back around with results and medication.
Every year, at least one child is usually transported from the camp in an ambulance or a helicopter to a hospital for further treatment, requiring diligent attention to medical details as well as a carefully thought through psychosocial plan of action to share that news with the other campers. Families are communicated with each day, and as you'd imagine many are nervous and excited about their kids being there. Some children participate while in their final stages of life.
Their families make the ultimate sacrifice by being selfless enough to share their children with others, so that their kids can live out their final hopes and dreams – the same dreams we all have. To do the things we dream of doing, especially those seemingly simple things associated with summertime.
Stories like this remind me of the passion and dedication of the medical research community, of the care providing community, and of the volunteer community. It's a reality check for me, and for those of us who work on policy-related issues. These kids and their families point out the obvious – that time is of the essence in all we are doing to get to faster cures.
It can be hard to make the FasterCures message personal at times as we deal with mostly macro-level issues, but hearing about Camp Fantastic reminded me why we do what we do. Because it's summer vacation time, and every kid (and for that matter, every grown-up) deserves a shot at creating their own lazy crazy hazy days of summer memories.
www.fastercures.org
With thanks to Margaret and Faster Cures for allowing me to reprint this from their blog.
Steven
One of my earlier columns for FDA Matters:
Patients Come First
November 11th, 2009
Most seriously-ill patients wake each morning wondering how they will surmount the day's challenges. They are concerned about the health of all Americans, but can't help being focused upon their own medical situation. Most of the people I know in the medical products industry wake each morning with the hope that their day's efforts can contribute to improving the health of all Americans, as well as provide specific benefits to seriously-ill patients.
The world-views of patients and industry are not dissimilar. They can share a dream (and an action plan)…if it is built on an unwavering commitment to patients coming first. Read the rest of this entry »
Late Friday Afternoon: FDA, Politics, and Scientific Integrity
When my Smartphone delivered an e-mail at 5:12 p.m. on Friday: "FDA approv…," I knew that FDA had just announced something controversial. All public relations people (including those at FDA) have been taught that late Friday is the time to release stories you don't want to receive much attention. Indeed, it was the 5-day emergency contraceptive pill, Ella, that was approved. So far, FDA seems to have achieved its goal of less coverage. But I was left wondering if the announcement required that treatment and why it led one advocate to describe the decision as "further evidence that the FDA is committed to restoring scientific integrity in its decisions."
When my Smartphone delivered an e-mail at 5:12 p.m. on Friday: "FDA approv…," I knew that FDA had just announced something controversial. All public relations people (including those at FDA) have been taught that late Friday is the time to release stories you don't want to receive much attention.
Indeed, it was the 5-day emergency contraceptive pill, Ella, that was approved. So far, FDA seems to have achieved its goal of less coverage. But I was left wondering if the announcement required that treatment and why it led one advocate to describe the decision as "further evidence that the FDA is committed to restoring scientific integrity in its decisions."
Abortion is, of course, one of the so-called "third rails" in American politics. For politicians, this means: if you touch it, you will get burned politically. It has never been easy for FDA either. For many years, they were caught in the middle of Congressional and societal fights over the abortifacient, RU-486.
As I have written before (link below), FDA has been a master of little-noticed decisions that create or re-position a disease category. With the new approval, they seem on relatively safe ground in deciding it is an emergency contraceptive. Apparently, there is a nuance as to whether the drug's mechanism of actions solely delays ovulation or also makes the womb less receptive to implantation. There may be a theological difference, but I don't see any practical difference.
Ella's approval doesn't seem to have been a hard decision and I can't imagine FDA found it particularly difficult. By announcing the decision late on Friday afternoon, they probably made it seem more consequential and controversial than if they had sent out a more complete press release at a time more conducive to news coverage.
That brings me back to whether this approval is really about "restoring scientific integrity" in FDA decisions. The comparison is being made to the controversy over Plan B, an emergency contraceptive with a much shorter window of efficacy than Ella.
The main issues for FDA regarding Plan B were: should the drug be available over-the-counter (OTC) without the input of a prescribing physician and whether it should be available OTC for women under 18. In my opinion, these were not primarily scientific issues, but social and societal ones. It is completely unlike the current approval of Ella, which will be dispensed only by prescription and doesn't raise the same societal issues or maybe doesn't raise any at all.
I am not defending the Bush administration's handling of Plan B. They could not have done a worse job and they deserve the criticisms they received. But the administration was probably right that the issues were not primarily scientific ones and required input from politically-accountable levels of the government and elected officials—people who have responsibility for public policy that impacts social and societal interests. The Bush Administration should have been more honest about that, rather than pushing the decision down to FDA officials.
Apart from a couple of decisions like Plan B, which are rare, controversial and were badly mishandled, I don't see that the agency made scientific decisions during those years that lacked integrity. There are always controversies about application of "safe" and "effective" standards….and accusations that science is being ignored. Ella is science-based decisionmaking and business as usual for FDA, not some restoration of agency integrity.
Steven
The FDA press release and the WP and NYT coverage:
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm222428.htm
http://www.washingtonpost.com/wp-dyn/content/article/2010/08/13/AR2010081305098.html
http://www.nytimes.com/2010/08/14/health/policy/14pill.html?scp=1&sq=ella%20contraceptive&st=cse
An earlier column that is relevant. It includes a brief comment on FDA and pregnancy.
FDA: Invisible Arbiter of What Constitutes Disease
November 19th, 2009
According to Wikipedia, "disease" refers to any condition that causes pain, dysfunction, distress and social problems. What constitutes disease is more varied and changeable than this definition might suggest.
The nature of disease and its constant changes are pertinent to FDA, which often makes decisions on behalf of society that reshape our understanding of disease. Read the rest of this entry »