Short Takes and Updates—February 20, 2025

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1. FDA At Risk on Four Fronts

All the federal public health programs are under siege. While my focus is on FDA, I want readers to know that NIH and CDC face similar (but not identical) threats. 

Reduction In Force (RIF). The new Administration sent lay-off notices to an unknown number of FDA staff over the holiday weekend. The common element is that they were on probationary status and thus have a lower level of job protection. I described and commented on this in my column earlier this week: If Being Mission Critical Is Not Important Enough, What Is? 

The situation is still unclear as of midday on February 20. Over the last 24 hours, I have been responding to reporters with the following statement:  

From watching OMB and FDA interpret these types of orders and directives in 2017, I learned there is a lack of clarity when they are first announced. Whether and how much FDA would be affected requires reading the fine print—in the overall declaration and then how it is to be applied to the massive responsibilities, different personnel systems, multiple funding sources, and wide range of skills needed at FDA. 

In this case, we do not know the numerator (laid off by center/office) or the denominator (total laid off at FDA), so it is impossible to talk about either general or specific impacts. We need to let the dust settle.

For those of us who are watching, it is frustrating not to know. For FDA employees--those who are living it—it is probably closer to excruciating. 

FY 2025 Appropriations/Possible Shutdown. FY 25 has been funded by Continuing Resolutions since October 1 of last year. The current CR expires on March 14 and so far, there is no deal on total spending or specific bills. According to Politico, “Republican appropriators sent a new offer to their Democratic counterparts over the weekend.”

FDA is at risk in two ways. If they reach an agreement, there is potential for FDA funding to be cut. If they do not reach an agreement, then there could be an additional CR or a shutdown. 

Budget Reconciliation. Compared to the other two threats, this one is going to take longer and unwind a bit more slowly but could lead to substantially greater downward pressures on domestic discretionary programs. I did a deep dive on this last week: FDA and Budget-Reconciliation 2025: A Primer and supplemented it here.  The process continues in both the House and Senate. 

Impoundment of Appropriated Funds (new and a wildcard). President Trump and OMB Director Russ Voight have asserted the President’s authority to impound Congressionally-appropriated funds….notwithstanding Constitutional provisions that have always been interpreted to the contrary. It is possible that FDA funds could be impounded, and the agency drawn into this battle. (h/t to Alec Gaffney at Agency IQ. You can sign up for their daily newsletter, FDA Today, at https://www.agencyiq.com/subscribe-fda-today/).

2. FDA and the Upcoming Attempt to Gut Medicaid: Are There Any Overlapping Issues?

The status of Medicaid may seem to be in doubt[1], rather than pre-determined. However, under the pending House Budget Resolution, House Energy and Commerce committee will be expected to deliver $880 billion in savings over the next decade. There is no way to achieve that result without massive reductions in the Medicaid program. All options to do so are painful[2].

As reconciliation efforts advance in the House and Senate, the status of Medicaid is going to be grabbing headlines. On the one hand, the President’s agenda and tax cuts cannot be paid for without the Medicaid cuts. On the other hand, the traditional Republican position on the value of Medicaid is going to clash with the reality that Medicaid (including program expansions of the last dozen years) are popular in many red states. 

My question to FDA Matters’s readers: do FDA and FDA stakeholders have a stake in the Medicaid battle? If so, what are the issues? 

3. What I Am Listening To

Wayne Pines and Shelly Garg have launched FDA Watch (https://fdawatch.libsyn.com/), which offers podcasts about FDA. I recommend signing up. 

This week’s guest is Janet Woodcock, discussing FDA’s future and the evolving regulatory landscape. You can also catch my appearance on January 20, along with Marc Scheineson of Alston & Bird, discussing the “The New-Look FDA in 2025.”     

4. Pass the word: sign-up to receive FDA Matters.

Forward this e-mail to colleagues and friends and urge them to subscribe at www.fdamatters.com. 

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1. Politico has reported this morning that: “Hours after pledging that the safety net program [Medicaid] “is not going to be touched,” Trump surprised some of his own staff Wednesday when he endorsed a House budget that would gut Medicaid.”

2. https://www.medpagetoday.com/publichealthpolicy/medicaid/114286?xid=nl_mpt_morningbreak2025-02-20&mh=29c54ab68180a56fae3d0da7029532a9&zdee=gAAAAABm4xE_8LqQ41dXUZAuGt2ybZFptmDDrkqWtRSJDN7TumcpCbqCgoGHLH3C0yUuUSbSeq0P-58l2HJlKPNjmbPnXTXnf_uS9sNSi6wLif3Sob3Ci7A%3D&utm_source=Sailthru&utm_medium=email&utm_campaign=MorningBreak-B_022025&utm_term=NL_Daily_MB_B

If Being Mission-Critical Is Not Important Enough, What Is?

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FDA employees—public-spirited and committed—are the heart of the agency and can proudly point to a lengthy record of accomplishment. Our country has safe food and safe and effective medical products because of their efforts. We are all affected: every American uses FDA-regulated goods and services at least several times each day. 

It is said that FDA’s best days are quiet ones—everything runs smoothly, nothing of public concern happens. Given the vastness of the agency’s responsibilities that is an incredibly high standard. 

Although the numbers are still uncounted, the agency suffered a brutal and seemingly arbitrary purge of probationary employees over the holiday weekend. Not only were relatively new employees swept away, but also many employees who received competitive promotions in the last two years (a process by which many of them might be considered “the best of the best.”). As extensive as the Reduction in Force (RIF) seems to have been, there are likely to be more attempts to lay off FDA employees. 

It is wholly appropriate for the FDA community to deeply mourn its institutional and individual losses. 

Employees on probation were an arbitrary target—chosen solely because they are an easier target than employees with more job protections. As far as I can tell, there was no triage based on protecting priority FDA functions and services or assuring needed skills. 

For many years past–and presumably for many years to come—a key goal of advocates has been to impress upon Congress, the Executive Branch, and the public that FDA is an extraordinarily  valuable agency providing a core function of government. The explicit message: the agency needs more money so it can better fulfill its growing mission. The implicit message: if cuts need to be made in government spending, FDA’s funding should be among the last to be considered. 

Government-wide, protecting the more valuable functions of the government (agencies, service  and people) formed no apparent part of the employee reductions[1]. 

It may be crazy to do a RIF at FDA when laid-off employees are mission-critical to medical advances and food safety and play a key role in national and global commerce. But that’s no crazier than cutting FAA staff who assure safe air travel after three incidents in the last few weeks (here), or probationary FBI agents (here), or cutting (since rescinded) the jobs of personnel who maintain nuclear safety and security (here). USDA work that relates to Avian Flu also experienced frontline cutbacks (here) that are inexplicable.        

If any of these agencies were told: next year you will have to function with fewer employees….it would be awful, but foreknowledge and flexibility would certainly help the agency lessen the damage by prioritizing mission-critical activities. 

In assessing the current situation, I am left with an obvious question that appears to have no immediate answer: if being mission-critical is not valuable enough to save an employee, service, or program, what is? 

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INFORMATION FOR FDA EMPLOYEES from the FDA Alumni Association: 

New resource page tailored to the needs of FDAers: Today the FDAAlumniAssociation posted a reference page outlining information sources that FDA staff might find helpful during this time of transition. The reference page is a work in progress, so if there are additional links you believe would be helpful that we should consider adding, please contact FDAAA. A huge shout out to the EPAAlumniAssociation that permitted us to poach some of their information sources. If you are planning to leave the agency or choosing to stay and would enjoy seeing familiar faces, please consider joining FDAAA. We appreciate you! https://lnkd.in/eR3bsQSA

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1.  According to one article I read, the White House has claimed that the government-wide RIF followed priorities and chose people whose jobs were less important. The article continued with a quote from a third party to the effect: if that is the case, then the people making the decisions had no understanding of the jobs people were performing.

Short Takes and Updates—February 14, 2025

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1. When Will Congress Engage on the Future of FDA User Fees?

Either the agency is captured by industry (Secretary Kennedy), or it is a hindrance to industry (Vivek Ramaswamy). I understand how it could be neither…but not how it can be both.

The lack of clarity is of concern to nervous corporate executives and many FDA employees. I have pointed out that PDUFA VII and MDUFA V and GDUFA III do not expire until September 30, 2027, with initial discussions only just starting this summer. There is time for this discussion after the new Administration has been in office for a while and taken care of more pressing issues. 

I am starting to feel a bit nervous about this answer. So far, the new Administration has shown little patience for waiting on anything. If they feel strongly, they seem to want to engage immediately….contractual agreements and statutes not necessarily representing barriers. 

This is not a prediction, just a lessening of my certainty that user fees are off the table until later.

2. More on FDA and Reconciliation.

My primer on FDA and reconciliation (here) was widely read and should have an extended shelf life.  The point of my primer is that the deep budget cuts from reconciliation (adopted by authorizing committees) will create an appropriations funding environment that puts FDA (and NIH, etc.) at risk. 

The tie between reconciliation and appropriations was reinforced at yesterday’s  House Budget Committee mark-up, where cost-cutting targets were increased to accommodate the House Freedom Caucus (here). HFC members are eager for deep cuts in appropriated funding.

A couple of readers asked: is FDA also vulnerable to cuts in funding through the reconciliation process itself? Under the House reconciliation bill adopted in the Budget Committee yesterday, House Energy and Commerce Committee was directed to generate a minimum of $880 billion over ten years. At that scale, the primary target will be Medicaid. 

To achieve savings from FDA, House E&C would have to adopt statutory language that had a budget impact on the agency. This seems unlikely but not out of the question.

3. What We Are Reading: Reversing Misinformation by Michael Miller, MD.

Inspired by Teddy Roosevelt’s comment that “complaining about a problem without offering a solution is called whining,” health policy analyst, Michael Miller decided that confronting rampant medical misinformation deserved a practical guide for individual action.

More about the book and about “Dr. Mike” can be found here.

4. Pass the word: sign-up to receive FDA Matters.

Forward this e-mail to colleagues and friends and urge them to subscribe at www.fdamatters.com. 

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FDA and Budget Reconciliation 2025: A Primer

Permission is granted to reprint or recirculate this column, as long as attribution is made to FDA Matters (www.fdamatters.com) and the author. 

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“A cynic knows the price of everything and the value of nothing.”—Oscar Wilde

Part 1:  Meaningful Deficit Reduction Requires More Than Cuts to Discretionary Spending

In FY 2024, the federal government spent $6.75 trillion and collected $4.92 trillion in revenue, resulting in a deficit. The amount by which spending exceeds revenue, $1.83 trillion in 2024, is referred to as deficit spending[1].

There is near-universal agreement that the annual federal budget deficit must be reduced. If allowed to continue, it would add $18 trillion to the national debt over the next ten years. 

“The iron triangle of deficit reduction” refers to the three ways (and only three ways) in which annual government deficits can be reduced or eliminated[2]: 

• discretionary spending cuts (defense and/or non-defense); 

• reduction in entitlement payments and other mandatory program spending; and

• increases in federal revenue through economic growth or additional taxes. 

All discretionary spending taken together ($1.7 trillion in FY 24) is less than the annual deficit. 

Nonetheless, every year, Congress attempts to achieve meaningful deficit reduction through the appropriations process, while also supporting increases in funding for defense, homeland security, and veterans. This puts overwhelming downward pressure on all other discretionary programs, including FDA. 

Meaningful deficit reduction can only be achieved by substantially increasing federal revenue and/or significantly reducing the costs of entitlements and mandatory programs. 

Addressing revenue and mandatory programs can be done by stand-along bills. However, as a practical matter, major changes in revenue and/or mandatory programs are unlikely except through budget reconciliation. 

Part 2: Budget Reconciliation Explained

As envisioned by the Budget and Impoundment Control Act of 1974, Congress has an obligation to develop and pass a budget resolution that determines spending, revenues, deficit or surplus, and the public debt for a fiscal year. 

The  appropriations committee then takes over and allocates the discretionary programs monies by subcommittee and program so as not to exceed the amount provided in the budget resolution. Most years, Congress does not pass a budget resolution and, instead, total discretionary funding is set as part of a bi-cameral leadership package that includes appropriations bills[3]. 

In the years that Congress has passed a budget resolution, it is usually for the purpose of creating “reconciliation instructions” that direct authorizing committee to find specific amounts of savings and/or find offsets for increases in the programs they oversee. Reconciliation directions do not go to the appropriations committees because they are already constrained by spending ceilings. 

Since 2000, seven out of the eight budget resolutions/reconciliation instructions are associated with a period when the same party controlled the House, the Senate, and the Presidency and six of them increased projected deficits[4]. This reflects the procedural advantages: the budget reconciliation only requires a majority vote in the Senate and cannot be filibustered. That makes it a more desirable way to move major legislative initiatives than the ordinary legislative route. 

There are two notable limitations: reconciliation can only be used to make changes that have budgetary impact; and the reconciliation process can only be used twice in every fiscal cycle.

Part 3: Complex Reconciliation Goals/Politics in 2025

Republicans in Congress are trying to achieve three primary goals in budget reconciliation in 2025:

• Increase resources available to support President Trump’s agenda, notably increased funding for efforts to secure the border, bolster our military, and increase American energy independence ($342 billion over four years according to a proposal under consideration in the Senate Budget Committee. Mark-up starts on February 12, 2025).

• Reduce the annual federal budget deficit from its current unsustainable levels (a 25% reduction in the annual deficit would require $4.5 trillion over 10 years; halving the annual deficit would require about $9 trillion over 10 years). 

• Find savings and revenue enhancers to pay for the cost ($4.7 trillion over 10 years) of extending the individual tax cuts enacted in President Trump’s first year (2017) and expiring on December 31, 2025. 

In the Senate, Budget Chair Graham is trying to do program changes (bullet one) in a bill now…and deficit reduction (bullet two) and taxes (bullet three) in a bill later in the year. Speaker Johnson is trying to achieve agreement in the House for one bill to do all of this—to be moved this Spring.

The politics of this….and how it will play out…are beyond the scope of a blog focused on FDA issues and policies. However, achieving these three goals will probably require $1 trillion per year in additional revenue (tariffs?), mandatory program cuts[5], and savings from continued powerful downward pressure on domestic discretionary spending. 

This is a cynical process (h/t to Oscar Wilde) in which many programs will be cut or eliminated because of their cost (price), regardless of their value. 

In such an environment, FDA’s fate is not sealed, but its prospects for sustaining and increasing its budget are not promising. 

The arguments for the value of FDA to public health, safety, and commerce will need to be voiced loudly by the entire FDA stakeholder community. They will need to do so repeatedly, until the value produced by FDA is seen as so great that paying the agency’s cost (price) becomes seen as a necessity….even in a dramatically smaller federal budget. 

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1.  https://fiscaldata.treasury.gov/americas-finance-guide/national-deficit/ 

2.  The fourth major category of budget expenses is interest payments on the national debt. These are set by market forces and cannot be reduced by law or fiat without jeopardizing the government’s ability to finance the totality of the  US debt, which exceeds $36 trillion. The public holds nearly $30 trillion of that debt. 

3. There are no consequences if Congress fails to adopt a budget resolution.

4. https://www.cbpp.org/research/introduction-to-budget-reconciliation 

5. Social Security and Medicare are likely to be spared for political reasons, meaning  even deeper cuts in Medicaid, SNAP, and other means-tested human services programs. 

6. According to the 2-12-25 issue of POLITICO Inside Congress, some House Freedom Caucus members are pushing for a $2 trillion target. 

Short Takes and Updates — February 6, 2025

Today is the first “Short Takes and Updates” column. For those who have not signed up to receive the FDA Matters newsletter in your email box, go to the home page to register.

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1. US to Leave the World Health Organization—An Alternative Interpretation.

For those who followed Trump 1.0 closely and parsed Project 2025 carefully, it is not a surprise that the World Health Organization (WHO) has been targeted by the new Administration. A summary of items in dispute is here.

However, I did not expect the notice of withdrawal to be a “day one” priority nor did I expect it would be in the form of an executive order (here). Fortunately, there is some leeway–best understood as: our participation ends now, but the required notice of one year means we are, at least for now, still members.

At last week’s wide-ranging Kinexum-sponsored webinar (https://youtu.be/9GMdrZ6fdY4 and https://youtu.be/eK9dUwcLNiY), a deep bench of participants with FDA expertise, spoke eloquently about the value and continuing need for WHO. Clearly, exiting WHO would be a loss for both US and global health.

In response, I offered an alternative interpretation of what might happen next. The new Administration likes to open with an extreme position (e.g., tariffs will be imposed in three days) then use that to gain leverage in subsequent negotiations. I am hopeful—albeit far from certain—that WHO will work out that way. It may require changes at WHO—perhaps significant one—but the US will continue to belong. At least that is my hope.

2. The Time Is Ripe for Improving Food Safety.

My earlier column (Down is Good and Up Is Bad; Or is the Other Way Around?) concluded that:  “There is no better time than now to create a comprehensive plan for improving food safety at the federal, state, and local levels — and push forward together as a community for its funding and implementation.”

As reported by Food Safety News (here), the General Accounting Office has issued a new report on food safety (here), which concludes that: “We previously reported on the need for a national strategy to guide federal efforts to address ongoing fragmentation and improve the federal food safety oversight system. This strategy could address our other previous matters for congressional consideration about a government-wide performance plan and sustained leadership for federal food safety. We maintain that such a strategy could create an opportunity to further strengthen federal oversight of the nation’s food supply and reduce the economic and public health effects of foodborne illness.”

To my many friends in the food stakeholder community, as well as the leaders of FDA, CDC, and FSIS: the time is now, let’s work together to make this happen!

Another voice in agreement: Frank Yiannas, former Deputy Commissioner for Food Policy and Response at FDA weighed in this morning with a column in Food Safety News (here).

3. Retirement-eligible FDA Employees Likely Key to How Many FDA Employees Accept the Proposed Buy-Out

My inaugural column on January 10, (Is a Mass Exodus From FDA Coming Soon?), urged FDA employees not to resign prematurely. Despite the subsequent “fork in the road” buy-out offer from OPM, that still seems to be the predominate advice. See analysis and recommendations:  here, here, here, and here.    

When numbers come out, probably soon, it is important to remember that there are many different jobs, situations, and career goals among FDA employees. As I wrote in the original column, one segment, those who are “retirement-eligible,” may “have a different calculus than those with shorter tenures in the federal government. In many cases, they do not face a significant loss of income if they leave the agency. Presumably, they also skew older and may have less interest in waiting to see how things turn out.” 

“An unknown is how much of the FDA workforce is retirement-eligible. Last Fall, an FDA official told me ‘15%.’  However, it was a casual conversation, and I do not know whether his answer was  actual or speculative. At the 15% level, with perhaps 25% uptake, the impact of resignations by retirement-eligible individuals would be significant, but perhaps not overwhelming.”

As a point of reference: 15% of 18,000 employees is 2700. If the uptake is 25%, as I have speculated, that would be about seven hundred retirement-eligible employees taking buy-outs. The actual percentage might be higher or lower for this group of individuals. Of course, this does not predict any particular outcome for the remaining 85% of FDA employees.

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Is RFK, Jr., Better Suited To Be an Advisor than an Executive?

The Robert F. Kennedy, Jr. confirmation hearings and the non-stop media coverage have focused, among other things, on his views on vaccines, abortion, drug pricing, and the negative aspects of the health and dietary choices of the American people.

Unfortunately, there has been far more heat than light on these topics. The hearings have left many of us with no clear sense of who he is and what he really thinks.

Most of all, we have no idea how, if confirmed, he would carry out the massive responsibilities of HHS Secretary. This raises the question: is Robert F. Kennedy, Jr’s suitable to be Secretary of Health and Human Services, independent of his views on the issues.

Kennedy is a free-wheeling advocate; HHS has never been a pulpit. Even if there are substantial reductions in the size and scope of the federal government, HHS will still be a major government organization with a broad array of responsibilities that must be tended to. That consumes the bulk of the Secretary’s time.

Because it is not a particularly good pulpit, past Secretaries of HHS have never been chosen for the specific purpose of advocacy leadership. As a rule, they are chosen to run the department. The role of chief spokesperson has been barely more than a secondary consideration. Dr. Louis Sullivan (1989-1993) was probably the last Secretary to legitimately see himself as “hired to be a public health leader.”

Kennedy has no experience managing complex organizations; HHS leaders have always been chosen for their executive experience. HHS’ discretionary budget is more than $130 billion and the agency has more than 80,000 employees.[1] Given the size and complexity of HHS, the Secretary usually has experience running a large organization.

Since 1985, the post has been held by four former governors (Bowen, Thompson, Leavitt, Sebelius), two academic leaders (Sullivan and Shalala), the Director of the Office of Management and Budget (Burwell), a Deputy HHS Secretary and corporate executive (Azar), and the California Attorney General (Becerra).[2]

Kennedy has no experience of being held publicly accountable for his positions and actions; HHS leaders have all had that experience prior to their appointments. Not all managers are publicly visible and accountable. However, if—as is the case with HHS--part of your job is to testify before legislative bodies and face a barrage of reporters about a decision you made, then that is public accountability.

Since 1985, every Secretary of HHS had a prior history of public accountability. I do not think any of them would have been comfortable with Kennedy’s answers that often sounded like: “I prefer my rarely cited and discredited study to your frequently cited definitive study, Senator (Doctor) Cassidy.” They would know such statements come back to trouble you at a later date.

Conclusion: The degree of uncertainty about Robert F. Kennedy, Jr.’s positions may not be  disqualifying, but it is discomforting to have so much unknown after two days of public testimony and thorough scrutiny of his career accomplishments.

More pertinent is whether the Secretary of HHS is the appropriate place for Kenndy to serve President Trump. The profiles of his predecessors (Republican and Democratic) have been similar to each other, but quite different from his.

One interpretation is that he is nominated for a position that ill-suits him. He would be a better counselor to the President or counselor to the Secretary where he would be able to research and expand his ideas:

• without being pulled away by the hundreds of other responsibilities that go with being Secretary of HHS, and  

• without being weighed down by a Senate confirmation process that exposes much of the “in development” aspects of his vision of leading Americans to healthier lives and more nutritious diets.

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[1] The Secretary oversees 13 operating divisions: Administration for Children and Families (ACF); Administration for Community Living (ACL);  Agency for Healthcare Research and Quality (AHRQ);

Advanced Research Projects Agency for Health (ARPA-H); Administration for Strategic Preparedness and Response (ASPR); Agency for Toxic Substances and Disease Registry (ATSDR); Centers for Disease Control and Prevention (CDC); Centers for Medicare & Medicaid Services (CMS); Food and Drug Administration (FDA);

Health Resources and Services Administration (HRSA); Indian Health Service (IHS); National Institutes of Health (NIH); Substance Abuse and Mental Health Services Administration (SAMHSA).

[2] At the time of their appointment: Secretary Otis Bowen was a physician and former governor of Indiana. Secretary Louis Sullivan was physician and Founder/President of Morehouse School of Medicine. Secretary Donna Shalala was Chancellor of the University of Wisconsin-Madison and before that President of Hunter College and Assistant Secretary in the US Department of Housing and Urban Development.

After Shalala, there were three former governors: Tommy Thompson (Wisconsin), Mike Leavitt (Utah), and Katherine Sebelius (Kansas). Secretary Sylvia Matthews Burwell was Director of the Office of Management and Budget. Secretary Alex Azar had previously been General Counsel and then Deputy Secretary of HHS and was President of Eli Lilly USA. Secretary Xavier Becerra was Attorney General of California and prior to that was a Member of the House of Representatives for 24 years.

I have omitted Secretary Tom Price because he served only 7 months (between Burwell and Azar). However, he was also well-credentialed, having been a Member of the House for 12 years and served as Chair of the House Budget Committee.

Planning is a Virtue, But Government Agencies Are Paid to Deliver Results

Planning is a Virtue, But Government Agencies Are Paid to Deliver Results 

The Federal Emergency Management Agency (FEMA) is responsible for helping the American people before, during, and after a disaster (how-fema-works). It has vast responsibilities and the unenviable burden of never knowing when it will have to ramp up or what it will face.

Last week, President  Trump raised the possibility of shutting down FEMA, saying, in effect, that FEMA was not doing its job well and states could do the job better. In just a few words, the President managed to throw FEMA under the bus, along with its more than 20,000 employees.

It is too soon to know if there will be consequences for the agency—but devolving its staff, responsibilities, and budget to the states has a high probability of being less effective than what we have today. Plus, the existing process is already built on federalist principles: FEMA only goes into the field when there is a local request conveyed to the agency through a governor’s request for national disaster designation.

Very few states and localities have a sufficiently trained workforce and available equipment in the case of a disaster. It is a costly solution to have each come up to full readiness year-round, versus having a co-op arrangement where common resources are available on an as-needed basis through FEMA.

Having FEMA on the ready also addresses interstate disasters and is responsive to the need for effective centralized command structure to coordinate in emergencies.

In all my years in Washington, I have dealt with FEMA only once. But it was instructive then….and relevant now to both FEMA and FDA.

At the time, I was at HHS and was asked to be the Acting Assistant Secretary for Health for two weeks while my boss traveled overseas. A Russian communications satellite was about to fall out of orbit and my primary responsibility was to coordinate with FEMA in case they needed Public Health Service involvement at a crash-site that could be anywhere in the world.

I was given a 100+ page FEMA briefing book. It was methodical in its analysis of possible scenarios, and it was comprehensive in scope. If you had the book, you knew how the US government was going to respond in any situation….and what you were responsible for doing.

Much of what I know about contingency planning I learned that week reading their briefing book. I was incredibly impressed by their preparation.

Near the end of my two weeks, the satellite came down in the early morning, crashing harmlessly into the sea. I heard about it first on the radio. I did not get my “all-clear” call for a couple more hours.

Despite all that beautiful planning….it took so long for the “all-clear” because FEMA had never set up a telephone chain[1] for efficiently distributing  information to the task force outside of business hours!

That was a long time ago. Few people remember phone chains. But it reflects an important truth: no matter how thoroughly you plan, you cannot anticipate everything.

In any case--even with perfect plans and best of intentions--we are all judged by the results we achieve, not by the quality of the plan we worked from.

In that regard, FEMA and FDA have it rough. Their missions are different [2] [3], but they have in common that they require the agencies to plan for:

• multiple,

• constantly changing, and

• largely unpredictable

challenges that reflect a rapidly changing and increasingly complex world.

That distinguishes FEMA and FDA from other agencies[4].

Because those challenges have become larger, more numerous, and even less predictable than in the past, Acting FEMA Administrator Cameron Hamilton (here) and FDA Commissioner-designate Marty Makay (here) will have far more difficult jobs than any of their predecessors.

Both agencies need to change to become more effective and efficient. But rather than moving boxes around on an organizational chart and calling it progress, the new Administration and Congress need to provide support—budgetary, advanced IT systems, etc. At the same time, the agencies need to act with more discipline as they: engage in more comprehensive, change-oriented planning; and streamline their operations to be more productive.

Fund appropriately. Plan better. Change sensibly. Achieve more. Those are big goals, but they are also the right ones for FEMA and FDA.

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[1] https://www.dialmycalls.com/glossary/telephone-chain

[2] In FEMA’s case, the scope of their responsibilities includes natural and man-made disasters, mixed in with responsibilities that have no other home, such as pandemic response (here) and sheltering migrant children at the border (here).

[3] In FDA’s case, the scope of their responsibilities encompasses goods and services in the US that total about $2.7 trillion and involve every American multiple times every day.

[4] Since this is a blog about FDA, I should add that the scope of FDA jurisdiction makes it even more vulnerable to unpredictable events than FEMA. In addition to what I enumerated in footnote three, FDA-regulated products—raw ingredient and finished product—come from every nation in the world, even hostile ones.

Keep Calm and Carry On: Lessons from Trump 1.0

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Eight years ago, the first Trump Administration (Trump 1.0), came into office with the stated intent of reducing regulation, shrinking the federal workforce, and cutting agency budgets.

They were quick out of the gates with their plans. For the most part, FDA was not specifically targeted, but many (if not most) of the proposals would have impacted the agency. Understandably, it created a lot of concern among agency employees and stakeholders.

A few days into the new Administration (Trump 2.0), it has become clear that objectives are the same, albeit rolled into the rubric of “less government.” What we are seeing—compared to 2017--is more executive orders and directives that are 1/ conceptualized with greater sophistication and 2/ being expressed with more urgency and determination.

Even still, I am experiencing a strong sense of déjà vu rather than some wholly new sense of peril for FDA. Although the ultimate outcomes in 2025 may be quite different—even quite a bit worse-- the challenges are mostly ones FDA has seen before in 2017 and in the later years of Trump 1.0.

In 2017, President Trump ordered a freeze on new regulations. This morphed into a requirement that agencies “eliminate two regs for every new one.” Eventually, it led to the realization that, in most cases, regulations could only be eliminated through the time-consuming “notice and comment rulemaking” process.

The President also announced a hiring freeze for 90 days at the beginning of 2017. This was intended to be a bridge to an OMB plan to place “a hiring freeze on all federal employees to reduce the federal workforce through attrition (exempting military, public safety, and public health).” A long-term or permanent hiring freeze would have had a disproportionate impact on FDA, which was trying to fill upwards of a thousand vacancies at the time.

Later into Trump 1.0, his Administration developed the so-called “Schedule F,” a plan to replace a large swath of senior policy positions into political appointees (analysis here). The plan was promulgated in October 2020, then withdrawn by President Biden soon after taking office.

We are only a few days into Trump 2.0, but we can already see many of the parallels.

Reducing the federal workforce—in terms of both size and influence—is clearly a top priority. This is, of course, of particular concern to FDA—with its large, well-educated, and multi-skilled workforce. About 80% of FDA’s budget pays for people-related costs (salary, benefits, rent, IT, training, etc.).

Among other things, a hiring freeze has been put in place. Special attention is being given to the potential to reduce head count by eliminating new and probationary employees and targeting those working on diversity, equity, and inclusion programs.

Return to office (RTO) policies have been put in place for federal workers and the first steps have been taken to reinstall Schedule F (here). On the latter, the National Treasury Employees Union (which represents the FDA workforce) has already filed suit to block its implementation.

Other initiatives announced this week include big picture items (such as withdrawing from the World Health Organizations) and small, presumably temporal ones (within HHS, cancelling public meetings and external communications through February 1).

From watching OMB and FDA interpret these types of executive orders and directives in 2017, I learned there is a lack of clarity when they are first announced. Whether and how much FDA would be affected requires reading the fine print—in the overall declaration and then how it is to be applied to the massive responsibilities, different personnel systems, multiple funding sources, and wide range of skills needed at FDA.

Eight years ago, just before President Trump was sworn in for the first time, I expressed my belief in FDA’s ability to survive change. I wrote:

“Despite the stridency of the rhetoric and the substantial uncertainty about 2017, Republicans and Democrats have, for decades, mutually supported an FDA that is well-functioning and fulfills its incredibly important public health mission.”  

It is reasonable to have doubts whether this remains true in 2025.

In the meantime, I urge everyone to “keep calm and carry on.”

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READ Steven’s last column, “Down is Good and Up is Bad: Or is It the Other Way Around?”

PODCAST ALERT: Available Now at https://lnkd.in/e26EjEkn Shelly Garg and Wayne Pines sit down with Marc Scheineson and Steven Grossman to discuss the FDA in transition.

WEBINAR ALERT: Friday, January 31 · 11am - 12:30pm EST Register Here
“8th Annual Wow! or Yeow!?: A Review of FDA in 2024 and Predictions for 2025.” Steven will be part of a panel discussion along with Janet Woodcock, Alexander Fleming, Frank Sasinowski, Kate Rawson Powell, and others.

RECOMMENDED READING: “What the Trump Administration Shouldn’t Change About the FDA” by Stuart Pape, Wayne Pines, and Mitch Zeller https://www.statnews.com/2025/01/21/fda-trump-nominees-rfk-jr-marty-makary-confirmation-hearings/

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Steven was quoted in AP story this week, which appeared in Washington Post, ABC 7, STAT, and Al Jazerra

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About the author: Steven Grossman is the author of “FDA Matters: The Grossman FDA Report.” For additional commentary about FDA issues sign up to receive Grossman’s weekly columns at www.fdamatters.com. 

Down is Good and Up is Bad; Or is It the Other Way Around?

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“FDA Matters: The Grossman FDA Report” provides short-form analysis and commentary on FDA policy and regulatory issues. My prior column, “Is a Mass Exodus from FDA Coming Soon?” can be found here.

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First appeared in Food Safety News. Reprinted with permission.

Chuck Jolley of Food Safety News recently wrote to me and several others asking (https://www.foodsafetynews.com/2025/01/were-recalls-really-up-in-2024/): With so many high-profile recalls in 2024, have the federal, state, and local institutions responsible for oversight suddenly failed us? Or is something else happening?

I imagined that he would get a range of answers from “yes, the sky is falling” to “it’s just random fluctuation.” 

I do not believe either of these are true, so I provided a middle-of-the-road answer based on my own experiences. 

Years ago, I was involved in a project to develop metrics of success for certain Food and Drug Administration activities. We discovered that sound data and observations can still be ambiguous in interpretation. If outbreaks have increased, that may be evidence of an overtaxed, under-resourced and underperforming system. However, it may also reflect better oversight, increased voluntary disclosure and recall, and greater producer and consumer awareness of food safety requirements.

Much to my surprise, all six of the experts Jolley consulted gave similar answers. We expressed ourselves in different ways reflecting our different perspectives, but no one said 2024 was a disaster and no one said that 2024 was just noise in the system. 

Professor Keith Warriner said: “We are not seeing a rise in recalls but in the number of outbreaks detected. . . . This has been achieved by advances in enabling technologies such as DNA sequencing, artificial intelligence and traceability.”

Thomas Gremillion of Consumer Federation of America said: “Concerns about all the recent recalls in the headlines, not to mention the long-term trends, are understandable. For now, this may be a story of better surveillance rather than increasing illnesses, but the uncertainty around whether food safety is getting better or worse is itself unsettling.” 

Professor Marion Nestle said: “I view recalls as indications that the regulatory part of the system is working, at least to some extent. If anything, there are not enough of them, and they do not come quickly enough.”

David Acheson, former FDA Associate Commissioner for Foods, said:  “My personal view is that, although there are always companies that struggle and don’t do things right, the food supply continues to get incrementally safer relative to many other factors that impact the likelihood of an outbreak being detected.” 

Phyllis Entis of eFoodAlert.com said: “There is nothing like a cluster of high-profile, high-volume recalls to catch the public’s attention. In fact, the number of recalls (FDA and FSIS data combined) due to food pathogens has not altered significantly in the last three years: 115 in 2022, 102 in 2023, and 127 in 2024.”

What do I take away from the six experts who are quoted in Chuck’s article? 

• Trends  in the number of recalls do not tell you whether things have gotten better or worse. 

• There is a remarkable absence of finger-pointing — a sign  that various interests are willing to work together toward the common good. 

• There is no better time than now to create a comprehensive plan for improving food safety at the federal, state, and local levels — and push forward together as a community for its funding and implementation. 

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Steven’s last column, “Is a Mass Exodus from FDA Coming Soon?”, was recently featured in the FoodFix and FDA Today newsletters. You can catch him on the FDA Watch podcast that drops January 20. Find the link here.

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About the author: Steven Grossman is the author of “FDA Matters: The Grossman FDA Report.” For additional commentary about FDA issues sign up to receive Grossman’s weekly columns at www.fdamatters.com. 

Is a Mass Exodus From FDA Coming Soon?

Califf’s Warning, Cavazzoni’s Resignation, Fuel Speculationa

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INAUGURAL EDITION—January 10, 2025

FDA Matters: The Grossman FDA Report provides short-form analysis and commentary on FDA policy and regulatory issues.

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The election spurred fears that a large number of federal scientists and public health staff at FDA might resign from their government posts. The potential for that was elevated by President-elect Trump’s nomination of Robert F. Kennedy, Jr. to be Secretary of HHS. His anti-vaccine stance is widely seen as a threat to science-based decisionmaking at the agency. 

Recently, outgoing FDA Commissioner Robert Califf “echoed the fears of other critics of the Trump administration, saying brain drain in the government was a legitimate concern, including at FDA.” (STAT 1-8-25). The STAT interview just two days ago and Dr. Bumpus’ earlier resignation, followed by this morning’s announcement of Dr. Cavazzoni’s resignation as CDER director, are certain to fuel further speculation. As reported by BioPharma Dive, two other agency veterans, Douglas Throckmorton and Robert Temple, have announced their retirements.” (BioPharma Dive 1-10-25). 

No one knows what will happen next week, later this month, or after the incoming Administration’s leadership team for HHS and FDA take office. The threat of a mass exodus of employees is real enough, but the likelihood in the short-term seems small. 

My experience is that few people actually quit their jobs because new incoming management sounds unfriendly. They wait to see…and even then, few people walk away from a job until they have another one—anticipatory concerns about principle and discomfort be damned! That takes time. 

If there is a short-term impact at FDA, it is far more likely to be in recruitment and onboarding. Questions about management philosophy, workplace conditions, and organizational stability are always issues when individuals decide whether to pursue or accept new employment, especially within a government agency which is unable to compete with the benefits frequently offered by private companies. 

There is one wrinkle in this analysis. FDA staffers who are retirement-eligible have a different calculus than those with shorter tenures in the federal government. In many cases, they do not face a significant loss of income if they leave the agency. Presumably, they also skew older and may have less interest in waiting to see how things turn out. 

An unknown is how much of the FDA workforce is retirement-eligible. Last Fall, an FDA official told me “15%.”  However, it was a casual conversation, and I do not know whether his answer was  actual or speculative. At the 15% level, with perhaps 25% uptake, the impact of resignations by retirement-eligible individuals would be significant, but perhaps not overwhelming. 

For those FDA employees considering what to do, I would echo Dr. Califf’s sentiments: “A lot of people are sticking it out, and I have encouraged them to do so. And you know, sometimes you get surprised by how well things work that you thought might not work so well. So, it is worth it, given the mission.”

I would distill this to: there is time enough later to decide whether principles are really at stake. 

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This is the inaugural issue of FDA Matters: The Grossman FDA Report. Our goal is to provide short-form analysis and commentary on FDA policy and regulatory issues. If you want to receive these columns regularly, please go to www.fdamatters.com to subscribe.

Sequestration Has Less Impact on FDA? Just Not True

“The Hill” newspaper recently reported that: “a survey of federal budgets devoted to developing and enforcing regulations found that many agencies will spend more in 2013 and 2014 than in previous years, indicating that the writing and enforcing of new regulations is largely unimpeded by the massive cuts, known as sequestration.”

That certainly sounds authoritative…until you look at the analysis. In fact, the report’s authors appear to know nothing about the federal budget and have used inherently unreliable data in calculating FY 13 and FY 14 spending levels.  One can only hope that the authors—allegedly academic experts--know more about regulatory policy than they do about federal budgets.

When I first saw the “regulatory spending analysis” from George Washington University in DC and Washington University in St. Louis, I expected to write about how FDA’s budget is mostly not regulatory spending. After all, what fractional part of the FDA’s budget is actually devoted to “writing and enforcing regulations?” And the claim that FDA is growing is highly suspect, as addressed below.

Equally troubling to the “innovation economy” is that funding for the Patent and Trademark Office is counted as regulatory, placing PTO on par with the Securities and Exchange Commission (see page 6 of the report).  It is not a subtle nuance to say they are fundamentally different.

FDA and PTO are the “federal regulatory agencies” projected to have the largest growth and are the linchpins of the GW/Washington University study. If most of their budgets are not regulatory spending, then it is impossible to draw conclusions about how regulatory agencies are faring under sequester.  

But I can’t leave it there because the analysis is riddled with serious methodological issues.

The first of many errors is the use of the proposed spending levels in the FY 14 President’s Budget Request as the measure of what agencies will have to spend in fiscal year 2014. One example of the variation this creates: FDA’s “regulatory growth” is calculated by including the President’s request for more than $200 million in food user fees, a proposal that has drawn no Congressional interest.

More broadly, no one really knows what the actual FY 14 spending levels will be. They are dependent on the resolution of the difference between the House and Senate budget bills (about $90 billion in FY 14 discretionary spending), the actual spending levels adopted by Congress in appropriations bills, the vagaries of funding under (likely) continuing resolutions, and the very real threat of yet another sequester in FY 14. Most federal agency heads would be exceedingly grateful to wind up with as much money as the President requested for them.

The study’s assertions about the FY 13 spending levels are equally unfounded. The GW/Washington University report uses estimated “outlay” numbers contained in the appendix to the President’s FY 14 budget. Since these tables were compiled before the passage of the FY 13 Ag/FDA appropriations or the final FY 13 continuing resolution, the GW study is using estimates based on the President’s FY 13 request (as ungrounded in reality as the FY 14 request), perhaps modified by part-year CR’s passed in late 2012.

In short, the actual FY 13 spending levels were not used in the analysis (indeed, weren’t even determined at the time of the President’s FY 14 budget request). So, the already-unreliable numbers in the report are unadjusted for the subsequent rescission and sequester. How can the authors conclude that “agency spending levels for regulation have increased modestly despite sequester,” without having reliable numbers that reflect the sequester?

Mark Twain once observed: “There is something fascinating about science. One gets such wholesale returns of conjecture out of such a trifling investment of fact.” Thinking of the GW/Washington University study, I can only say “amen.

More Columns Coming; The Alliance for a Stronger FDA

FDA Matters appreciates your patience. New columns will be coming in June, with fresh insights into FDA and the FDA-regulated world. 

Meantime, I write a weekly column in the Friday Update, published by the Alliance for a Stronger FDA. If you want to receive the Friday Update when it's published each week, you can sign up at http://www.strengthenfda.org, where it  says "subscribe to Alliance Updates."  You can also follow the Alliance on Twitter: @strengthenfda. Past columns are also available on the website. 

I urge readers of FDA Matters to consider becoming members of the Alliance for a Stronger FDA. It is a multi-stakeholder coalition working for increased appropriated resources for FDA. Members include patient and consumer organizations, research advocacy groups, health professions societies, companies, trade organizations, consulting firms and interested individuals. For membership information, contact me at: sgrossman@strengthenfda.org. 

Steven

FDA’s Indifferent Attitude Towards the First Amendment

The FDA doesn’t care about the First Amendment rights of the companies it regulates. It cares even less about the “free speech” rights of those companies’ sales and marketing representatives.

And why should the agency care? One of FDA’s primary missions is to protect the public health and safety of the American people from illegal, adulterated and misbranded products. Doing so involves restraining food, drug, device and cosmetics companies from committing fraudulent and deceptive acts that are not protected by companies’ commercial free speech rights.  

Nonetheless, FDA Matters envisions opportunities for FDA and industry to broaden permissible product communications. The key is understanding history, not constitutional law.

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I recently participated in a forum at American University Washington College of Law on “Evolving First Amendment Protection of Commercial Speech” and offered up my mantra that constitutional analysis is largely irrelevant from FDA’s perspective. I said “largely” because FDA is still part of the federal government and can’t act arbitrarily. On the other hand, the agency mostly operates within the zone in which government is given the most leeway: where public health and safety is at stake and the threat is from commercial (as opposed to individual) speech.

History, not constitutional law, provides the best explanation. The sale of bad food and drugs—often accompanied by slick, deceptive pitches—goes back millennia and was even addressed as a problem in most ancient legal codes.

FDA’s own birth comes from a time when state regulation and inspection of food and drugs was minimal, inconsistent and often corrupt. It is hard for any of us to imagine what an unregulated market in food and drugs is like.  Yet, it is not so long ago.

The 1938 Amendments to the Food, Drug and Cosmetics Act gained popular support in part because of a traveling exhibit that portrayed the death and disability that resulted from patent medicines, counterfeit products, false medical and scientific claims, and adulterated and misbranded products. Lax to non-existent cosmetics standards were particularly singled out for their role in causing burns and blindness, as well as some deaths.

It is nice to imagine that this world is behind us, just an interesting piece of history. But it isn’t.

No industry regulated by FDA is immune from shoddy products, false claims, unscrupulous behavior and greed-induced threats to public health and safety.  I am sure that none of my readers count themselves among these “bad guys,” but they exist in the U.S., as well as globally.

Further, even the great and innovative companies—household names that we view with great trust—have often proven to be quite fallible. A certain amount of informal off-label promotion of drugs and devices is rightfully ignored--when good studies have been published, when the off-label indication is very close to an approved use, when assertions are made with great care about the extent of proven scientific knowledge.

In contrast, most of the off-label promotions that have resulted in billion dollar settlements with big-name drug companies have not been based on such close questions. Nor have they been the result of an individual salesperson crossing the line in some excess of enthusiasm.

Rather, the off-label promotions have been the product of marketing departments and sales managers who encouraged, empowered, or authorized the off-label promotion. There never seems to be a good answer as to who was supervising marketing and sales, which is why so many drug companies are now operating under government-negotiated corporate integrity agreements.

From FDA’s perspective, there are legitimate, well-documented reasons to scrutinize all companies: none are immune from the impulse to over-hype products to expand markets and sales beyond what FDA has approved. This is not a matter of FDA being over-fussy. It is the inevitable conclusion from about 4000 years of human commerce in food and drugs.

In the face of this, the First Amendment really has very little place. Commercial free speech does not extend to misleading statements, blatant fraud, or deception. FDA sees too much of this to ignore. 

FDA Matters believes the agency still has an obligation—but not a constitutional one--to clarify its standards, provide published guidance, and demonstrate acceptance that the Internet has fundamentally changed the nature of product promotion. The lack of FDA guidance on social media (first raised at an agency hearing 15 years ago) is particularly outrageous and the agency’s tendency to create de facto policy with enforcement letters is an abdication of responsibility.

The drug and device industry can also improve the situation. They need to stop looking at the current controversies in product promotion as noble causes involving sacred constitutional rights. If these industries have a claim to better treatment and clearer policies, it needs to be grounded in the contributions they make to improve public health and evidence of serious efforts to rid their companies of unscrupulous promotional practices.

Steven

I have written previously on some of the issues in drug and device promotion, as well as about opportunities for FDA and industry to reach accommodation:

Off-Label Promotion: Best Resolved by Congress, Not Courts     December 2012

On December 3, a federal appeals court ruled against one of the FDA’s untouchable restrictions on industry—thou shalt not promote the off-label use of pharmaceutical products. An industry that is little interested in constitutional law suddenly finds itself talking about the First Amendment. At stake: permitting off-label promotion undercuts the incentive for companies to thoroughly investigate the safety and efficacy of a drug for a second or third use.

Off-Label Uses Need to Become On-Label Indications  December 19, 2009

A friend asked: what advice would you give a pharmaceutical company in the late stages of developing a new product that will be widely used off-label? The company’s concern was that FDA might hold the first use to a very high, perhaps unrealistic standard to protect patients that might receive the drug off-label after approval.

Internet Communications: FDA Needs to Divide the Issues to Conquer the Problem  Dec. 2nd, 2009.

Creating an Internet communications policy for regulated medical product companies is so daunting that FDA has largely ignored the responsibility. November’s FDA hearing on social media was an important step, but offered no sign that new policy will 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. 

Off-Label Promotion and Whistleblowing    September 9th, 2009

Whistleblowing and off-label promotion of drugs and devices have become hot topics because of the September 2 Pfizer settlement with the federal government. While none of my views are specific to Pfizer, the company’s settlement provides an opportunity to comment on off-label promotion….and to encourage bio-pharma and medical device companies to engage in deeper soul-searching. 

A Salmon on Every Plate/The Hard Road of Innovation

Chicken was once an expensive delicacy. In 1928, America’s quest for a better diet and a better standard of living was summarized by the campaign promise of “a chicken in every pot.” Today, chicken is a ubiquitous, low-cost source of protein, which we largely take for granted. Despite depletion of ocean-based stocks, fish hold similar potential.

To begin this transformation, FDA must approve a scientifically-based innovative product—a faster growing genetically-engineered (GE) Atlantic salmon. When FDA Matters wrote about this subject 18 months ago, I believed the agency was near to approval of this first-ever food product from a GE animal. It is still not resolved and there are implications for all innovations that require FDA approval.

Final comments on the “environmental assessment” of GE salmon are due to FDA in April. Hopefully, this is the final procedural step before a decision. Approval could come mid-year or may take months or may not happen at all. The agency is still dealing with the political fall-out of questionable safety claims from environmental groups…and politicians and companies trying to protect the market for Pacific salmon.

The health benefits of fish are well-known. They are also a valuable source of dietary protein. However, our oceans are over-fished and aquaculture is now the source of almost 50% of the fish consumed worldwide. Expanding the availability of fish products meets a growing demand and is an important component of improved nutrition for Americans.

The proposal before FDA is for a genetically-engineered salmon that is biologically and chemically identical to the Atlantic salmon that is served in restaurants and at our own tables. The only difference is the inclusion of a Chinook salmon gene that provides the potential to grow Atlantic salmon to market size in about half the time.

Opponents have labeled the product as “Franken-fish.” It’s a catchy slogan that tries to devalue over a decade of scientific research and undercut many years of FDA review. Ultimately, the appeal is to emotion—that something dramatically new and different must automatically be dangerous. At some point, scientific review and product safeguards should be sufficient for FDA to make a decision that is based on facts and not fears.

Decisions about new and different products are hard for FDA, as I wrote in a column entitled: “FDA and Things that Might Go Bump in the Night.”  Among other things, I reminded readers that in the late 1970’s and early 1980’s, biotechnology was subject to the same types of concerns and evaluation as FDA is currently giving nanotechnology, GE food and synthetic biology. While there are risks to saying “yes” to innovation, there are also potentially large consequences to saying “no.” Imagine today’s world without biotechnology.

Approval of genetically-engineered animals will always require serious consideration of safety, environmental and ethical issues.  In this case, no one questions the legitimate demands for plentiful, high quality supplies of salmon. Further, the sponsor has agreed upon multiple redundant safeguards. For example, the GE salmon will be sterile females and grown in inland fisheries without access to either wild or farmed salmon stocks.

And yet, the years have piled up, waiting for FDA to be ready to say “yes” or “no” to GE salmon. This isn’t intended as a complaint about FDA…it’s really more of a reminder for the rest of us that a pro-innovation culture at FDA requires hard work from stakeholders.

Scientifically-based, well developed applications are a must. Those of us who support innovation must also make common cause. This is both policy-driven (e.g. by supporting development of regulatory science) and procedural (e.g. not complaining too loudly when FDA takes a somewhat longer deliberative path than we might like).

Further, brave thoughtful decisions by FDA are easier if they are met by public and stakeholder support. GE salmon may be a food, but FDA’s decision affects the environment in which drug, vaccine, and device innovation are also being judged.

Thus, the current fight is not just about “a salmon on every plate.” It is also about whether FDA has the resources and support to sort through the many “alternative futures” represented by the products submitted for its approval.

Steven

More information about salmon, aquaculture, regulation of genetically-engineered foods and the current controversy can be found at: http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/VeterinaryMedicineAdvisoryCommittee/ucm222635.htm and http://www.aquabounty.com/PressRoom/#l7

A final note: I almost never write about or even mention individual products because FDA Matters’ aim is to analyze and comment on policy, regulation, and FDA trends.

This column is an exception because I believe that FDA’s consideration of GE salmon is an important part of understanding the agency’s role in innovation and how such decisions can become far more difficult that they should be. I have no clients or financial interests in development of GE food products.  On the other hand, a salmon dinner is a favorite.

Funding Cutbacks at FDA: A Sequester Primer

At a time when FDA’s responsibilities continue to grow rapidly, the agency has been caught in an across-the-board reduction (sequester) in federal discretionary spending, effective March 2, 2013. Although Congress may yet reverse course and restore money to affected federal agencies, this is not considered a high probability.

Altogether, FDA will lose about $209 million between now and September 30, 2013. This will reduce inspections, slow drug and device approvals, and restrict implementation of the Food Safety Modernization Act and other recent legislation. Because of the many questions about the process and outcome, this is FDA Matters’ primer on the sequester of FDA funds.

How much of FDA’s FY 13 (current year) money is involved and which funds are cut? 

Sequestration	FY 13 CR (to 3/27/13)	Less sequester	Net amount
Budget appropriations	$2.521 billion	$ 126 million	$2.395 billion
User fees (inc. tobacco & generics UF)	$1.647 billion	$   82  million	$1.565 billion
Totals	$4.168 billion	$ 209 million (rounded)	$3.960 billion (rounded)

Will all programs within FDA be cut by precisely the same amount? Generally, yes.  Unless Congress or OMB change the sequester rules (and they might), each Center and the Commissioner’s office will be cut by about 5.0% to achieve the $209 million reduction. However, fixed items (rent, utilities, etc.) will still cost the agency the same amount, creating an internal deficit. This will require additional cuts (above the 5.0%) from the agency’s program activities.

Beyond the specific dollars, why will the impact be greater on FDA than on most federal agencies?  FDA is a staff-intensive organization. More than 80% of its funding is spent on personnel-related costs. Much of the remainder is fixed costs, rather than grants or contracts that could be cancelled or cut back. Many federal agencies are hoping to meet their sequester obligations without personnel cutbacks. FDA will do its best to follow this model.

FDA has said that no employees will be furloughed as a result of the sequester. How is that consistent with the agency’s staff-intensive nature and the depth of the cuts? To absorb the $209 million without furloughs, most of the savings will have to come from unfilled and/or delayed filling of vacancies—a process that probably started months ago. Many offices are probably already short-staffed and most will become so as we get deeper into the sequester. Hence, the predicted impact of the sequester is slowdowns in food (and other) inspections, drug and device reviews, and implementation of new FDA legislation and its global responsibilities.

In offices where staff reductions are needed as part of the sequester but no vacancies exist, some appropriately qualified employees may be saved through internal transfers into slots that can be paid for by the Generic Drug User Fee program (FDA’s one big growth area this year and for which it will definitely need to be hiring).

How would you characterize FDA’s “story” about the impact of the sequestration?  While $209M is a large cut with severe consequences, it may well be achieved by a thousand little bits and pieces, a vacancy here, a training contract there, a bunch of cancelled plane flights, some underspending early in the year, etc. It doesn’t make for a very dramatic story, which may account for why FDA has been relatively quiet while other departments and agencies have been talking about the severe impact of the sequester cuts.

Conclusion

If food is less safe…if drug and devices reviews go more slowly….if problems with imports increase....and if regulatory science and agency modernization stall….then there is a huge loss to the American people.

This loss is made larger by the knowledge that the agency’s mission is not static.  Congress has passed 6 new laws since 2009 giving FDA substantially more responsibility. Globalization and complex science makes the agency’s job larger and more difficult each year.

FDA staff wasn’t large enough to get the whole job done last year. Problems will  multiply quickly over the coming months as existing employees represent an every smaller proportion of the numbers needed to carry out this year’s and next year’s responsibilities.

Steven

*  For purposes of disclosure: I am one of the founders and serve as Deputy Executive Director of the Alliance for a Stronger FDA, www.strengthenfda.org, a multi-stakeholder group that advocates for increased FDA funding.  For more information on joining, contact me at sgrossman@strengthenfda.org.

Note that my duties with the Alliance are in addition to, and apart from, the work of my policy and regulatory consulting firm, HPS Group, LLC, which is the publisher of FDA Matters.  The views expressed in FDA Matters are my own, and those of HPS Group, and are not necessarily the views or positions of the Alliance.

FDA Funding: Agency Mission "At Risk", Says Alliance President

FDA’s mission is “at risk” because of inadequate funding. So says Alliance for a Stronger FDA President Diane Dorman, testifying before the FDA Science Board. Her remarks come 5 years after the Science Board made a similar declaration, concluding that decades of underfunding had left FDA without the resources to fulfill its mandate and make science-based decisions.

Congress responded with more monies for the agency, but since then the FDA’s workload has increased even faster. The current threat to FDA comes from two sources: four major new laws to implement since 2009; and changes in the environment in which FDA operates, notably acceleration of globalization and increasing scientific complexity.

Ms. Dorman’s remarks are reprinted below. If you care about FDA, FDA Matters urges you to read her testimony, go to the Alliance’s site (www.StrengthenFDA.org) and join.

Testimony of Diane E. Dorman

President, Alliance for a Stronger FDA

Before the

Science Board of the U.S. Food and Drug Administration

February 27, 2013

Good afternoon and thank you for the opportunity to address the FDA Science Board.

My name is Diane Dorman and I am President of the Alliance for a Stronger FDA, as well as Vice President for Policy at the National Organization for Rare Disorders. The Alliance is a 200-member coalition of all FDA’s stakeholders—consumers, patients, health professionals, trade groups and industry. Our sole purpose is to advocate for increased appropriated resources for the FDA.

When we started in 2006, FDA appropriations stood at slightly less than $1.5 billion for an agency tasked with overseeing 100% of drugs, vaccines, medical devices, and personal care products and 80% of our nation’s food supply. Altogether, the products and industries regulated by FDA account for nearly 25% of all consumer spending in the United States.

In short, FDA was the victim of decades of underfunding. It was quite small, despite its vital, complex world-wide responsibilities. Presidents weren’t asking for nearly enough money for FDA and Members of Congress were responding by giving the bare appropriations that had been asked for.

The Alliance’s goal was to change this situation by galvanizing the FDA’s broad stakeholder community to focus attention on the consequences of underfunding. We never doubted the accuracy of our analysis or the importance of our cause.

Nonetheless, it was immensely helpful when--18 months after our founding--the FDA Science Board released its own report in November 2007. As the media described it---the FDA’s own Science Board evaluated the agency’s capacities and responsibilities and declared that the agency’s mission was “at risk.”  The word “crisis” was often used and was an appropriate description of the situation.

Subsequently—and with the Alliance’s broad stakeholder advocacy—the prospects for FDA improved. Policymakers acknowledged the underfunding and acted aggressively to reverse it.

Today, in FY 13, the FDA receives slightly more than $2.5 billion in appropriated funding. This amount, might have met the FDA’s funding needs in late 2007 when the Science Board report was issued…BUT NOT NOW.

Today, $2.5 billion is dramatically less than the amount the FDA needs. For reasons I will describe in my testimony, the agency’s mission is again “at risk.” Even without the possibility of funding cutbacks, the American people will lose if FDA does not receive increased funding.

FDA Responsibilities Grow Each Year Because Congress Enacts New Laws

Two months before the Science Board declared FDA to be “an agency at risk,” the FDA Amendments Act of 2007 was signed into law, renewing the prescription drug and medical device user fee programs. It added a slew of new responsibilities, notably in food and drug safety, regulatory science, clinical trial registries, and establishment of a program for risk evaluation and mitigation strategies for new drugs.

The new responsibilities--combined with delays in funding of existing and new programming--had severe consequences. For example, FDA’s efforts in the critical area of drug reviews and approvals were slowed substantially for nearly two years, as demonstrated by this CDER chart.

The message from this experience is clear, albeit not surprising: new laws take enormous resources to implement. Once implemented, they permanently increase agency responsibilities.

Since 2007, Congress has identified a number of new needs that fall within FDA’s jurisdiction. At least six new laws have been passed in the intervening five years:

• Family Smoking Prevention and Tobacco Control Act (2009)
• Biologics Price Competition and Innovation Act (2010)
• Secure and Responsible Drug Disposal Act (2010)
• Combat Methamphetamine Enhancement Act (2010)
• Food Safety Modernization Act (2011), and
• FDA Safety and Innovation Act (2012), including re-authorization of the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act

This is hardly the end of it. Congress is already looking at a number of legislative initiatives for 2013, covering topics such as:

• Bio-security
• Track and trace/counterfeit products,
• Drug compounding, and
• Drug shortages.

 In addition, this year’s legislative requirement--renewal of two Animal Drug User Fees--is widely seen as a vehicle for other legislative mandates that FDA will need to implement.

The problem is not solely Congress’ urge to legislate. While some of our Alliance members may quibble with some of the new programs and requirements, overall I believe there is strong public and stakeholder support for Congress addressing unmet needs and emerging challenges. We all want safe foods and safe and effective medical products.

Ultimately, the real problem is Congress’ failure to acknowledge FDA as a funding priority despite the austere budget environment. Transforming FDA’s mission and responsibilities needs to be met by the necessary resources to do the job well. The current appropriations level is totally inadequate to make up for decades of underfunding AND all of the new laws enacted since 2007.

FDA Responsibilities Grow Each Year Because of Globalization and Scientific Complexity

Even were Congress not active in legislating new mandates for FDA, the agency’s mission and responsibilities would grow enormously each year for reasons unrelated to new laws. While the list is long, my remarks will concentrate on two: globalization and increasing scientific complexity.

One of FDA’s highest priorities since the Science Board report has been to re-align to adjust for the accelerating globalization in all product categories overseen by the agency. While there is no one way to fully convey the enormity of this shift and the resources required, I offer the following sample of key facts:

•      Food Imports are growing 10% annually. Altogether, 10-15% of all food consumed in the U.S. is imported. This includes nearly 2/3 of fruits and vegetables and 80% of seafood.

•      Device Imports are also growing about 10% annually.  Currently, about 50% of all medical devices used in the US are imported.

•      Drug Imports are growing even more quickly, about 13% annually.  Approximately 80% of active pharmaceutical ingredients (API) are manufactured abroad, as are 40% of finished drugs.

Inspections at U.S. ports-of-entry are critical, but ultimately less than 2% of shipments can be inspected. The better alternative--the one encouraged by Congress and chosen by FDA--is to increase foreign inspections and to establish foreign offices to work globally to improve the standards and quality of products entering the U.S.

The value of this approach cannot really be quantified. We know that the cost of illness, death and lost markets--from just a single bad actor in a single food category--can cost as much or more than the entire investment we put into FDA’s food safety activities. Drugs and devices are harder to track for a variety of reasons, but there is no reason to doubt a similar effect.

In contrast to globalization, greater scientific complexity is diffused into every part of the agency and its mission. That makes dealing with it less visible, but doesn’t make it any less costly.

FDA has adopted several approaches, many from the FDA Science Board Report. These include creation of a commissioner-level science office, investment in regulatory science, expanded and more intensive training, changes in time and manpower allotments for complex assignments, and significant reworking of the drug and medical device approval pathways.

Specifically, we have identified five areas that FDA is working on to improve the review process and respond to more complex science. Each comes at a cost in additional dollars and manpower:

•      Sponsors Need More Meeting Time and Other Feedback from FDA

•      Product Applications Require More Patients, Study Sites and Analysis

•      Enhanced Timeliness and Consistency of Product Review is Paramount

•      Expansion of Pre-and Post-Market Safety is Essential

•      Sustain and Increase Core Programs That Enhance Innovation, Speed Approvals

Further, safety inspections have also become more complex—requiring more scientific training, more preparation and, often, more time during the inspection itself.

FDA: An Agency Still Very Much “At-Risk” for Lack of Adequate Funding

It is important to recollect that FDA is a staff-intensive organization. More than 80% of its budget is devoted to staff-related costs. Of the remainder, rent and utilities are fixed costs that must be paid first. There is little grant and contracting to cut.

Sequestration is the most immediate threat to the FDA’s already-inadequate funding. Just a few days from now, the agency faces a loss of 5.1% of its FY 13 (current year) budget. This is the nominal rate. The Alliance’s analysis, confirmed by OMB testimony, is that the actual impact will be close to 9%.

Even if sequestration is avoided, FDA faces challenging funding battles in FY 14 and beyond.

If cuts occur now or in FY 14—or even if the agency budget stalls and fails to grow over the next few years:

•      food will be less safe and consumers put at risk,

•      drug and device reviews will be slower, conflicting with promises made to consumers and companies,

•      problems with imports and globalization will become more numerous, and  

•      critical efforts to modernize the agency and improve its support for innovation will stall.

Is FDA’s mission again at risk? Absolutely, yes.

And those who have the most to lose are the American people.

FOR MORE INFORMATION: www.StrengthenFDA.org or contact Steven Grossman of the Alliance staff at (301) 539-9660, sgrossman@strengthenfda.org.

The State of the FDA---February 2013

FDA is the only federal agency that touches the lives of every American several times every day. Despite this, FDA will probably not be mentioned when President Obama delivers his State of the Union (SOTU) address to Congress on February 12.

Instead, FDA Matters provides its third annual “State of the FDA.” As reflected in last week’s column, I think that FDA did well in 2012. And 2013 is very promising. Potential funding cutbacks are the primary impediment to future successes.

Strengths. Once again, FDA’s most important strength is the dedication of the agency’s staff.   Last year, I viewed staff’s efforts as invisible and largely unappreciated. I believe that more recognition is being given to the staff—driven by a banner year for drug approvals, progress on implementation of the Food Safety Modernization Act (FSMA), and the narrowing of the FDA-industry chasm on medical devices.

Another key strength is a growing self-confidence within the agency that it can solve problems and not just tread water to survive. Over the years, FDA has often spent extended periods in a bunker posture—harassed, defensive, waiting to be forced to act, speaking too softly for fear of unleashing criticism.

Over the last year or so, there appear to be many more instances where the agency has taken the initiative. To be sure, they have talked to stakeholders and checked in with experts first, but then they have acted by making an announcement, releasing guidelines, creating new policies or intervening to solve a problem. 

Both of these strengths have been heightened by continuity of leadership. In May, Commissioner Hamburg will have held the job for four years, the longest tenure of a commissioner since the mid-1990’s. It also feels like there have been fewer top-level personnel changes. In the past, constant changes have undercut achievement and sapped morale and self-confidence.

Weaknesses. Congress has given FDA an ever larger role without providing the funds to do the job. As a result, and despite the agency’s best efforts, important initiatives and activities are not getting the resources they need. Inadequate funding is the most pressing weakness of FDA.

There are really three parts to the problem:

• three new laws over the last three years need to be implemented: food safety (FSMA), biosimilars (BPCIA) and user fee amendments (FDASIA).
• Congressional pressure to do more in complex, expensive areas, such as medical innovation, safety, medical countermeasures, track and trace/counterfeit products, drug shortages and compounding.
• FDA’s job is getting bigger, tougher and more resource-needy each year independent of whether Congress gives them new responsibilities. This emanates from greater scientific complexity, industry globalization, and increased workload (meetings, NDA’s, etc).  

 As will be discussed further under “threats,” there is a strong potential for cuts in funding that would take FDA far below even the current inadequate level.

Opportunities. Dr. Hamburg has made it a priority to improve the agency’s scientific bench strength—better credentials, better training and better tools. The next step—still very much a work in progress—is to integrate patients and human concerns into FDA decisionmaking.  

The agency understands the importance of this opportunity, but underestimates the tension between patient viewpoints and the scientific process. The task is more nuanced than current efforts suggest….and the risk is that patient-input becomes a box that gets checked, rather than a meaningful improvement to the agency’s science-based decisionmaking. 

In a different vein, FSMA is a well-conceived solution to achieving a safe food supply in the 21^st century. The opportunity is enormous, the blueprint largely drawn, and only the inadequacy of funding a substantial barrier to success. I don’t know if the American people will ever properly appreciate the effort required by FSMA or the value its implementation adds to protecting the food supply. Without that public feedback and support, the challenge for FDA will be to continue to see FSMA as the transformative opportunity it is.

Threats. The largest threat to FDA is the potential for immediate and long-term cuts to the resources available to the agency. On March 1, FDA may lose more than 5% of its current-year funding. Even if that cut is averted, funding for domestic discretionary programs is going to be under pressure for the next decade.  

The increasing reliance of user fees for agency funding is also a threat. Including tobacco and the new generic drug user fees, the FDA is now 40% funded by industry. Those fees are put to good use and are not, by themselves, a problem. Rather, American taxpayers need to preserve--if not actually increase--their stake in funding FDA. There are philosophic reasons for this (the integrity of the agency) and practical ones (a large part of FDA’s mission cannot appropriately be funded by industry).

Conclusion. FDA’s strengths and opportunities are immediate and powerful...perhaps more so than in many years. The challenge is to preserve and expand the funding, particularly taxpayer funding, to support the agency. Trying to “do FDA on the cheap” is both a weakness and a threat to the agency and the American people.

Steven

FDA Post-Election: Continuity and Progress Likely to Mark 2013

Looking back over the last 40 years at FDA (as I have), there are three characteristics that create a more progressive environment at the agency: continuity of leadership, presidential support, and increased funding. For FDA in 2013 (as the saying goes): 2 out of 3 ain’t bad.

In particular, medical innovation seems poised to flourish in an FDA environment where there is continuity of policy and leadership, instead of a new team learning the ropes. I explore this and other themes in the latest issue of Pharmaphorum.com. You can read my thoughts at: http://www.pharmaphorum.com/2013/01/29/fda-post-election-continuity-and-progress-likely-to-mark-2013/.

Steven

Orphan Drugs at 30: Will Success Become Too Expensive?

The Orphan Drug Act (ODA) turned 30 this month, demonstrating that good laws really can have an enduring impact. Amidst the celebrations, a reporter asked me a provocative question: can we afford more orphan drugs costing hundreds of thousands of dollars per year? FDA Matters answered “yes.”

However, I added a caveat that should worry everyone eager for orphan drugs to succeed. When genomics and personalized medicine become successful, this will multiply the number of rare diseases and the overall cost of orphan drugs, perhaps beyond what the system can bear.

Orphan Drugs/1983. At the time of the 15^th anniversary of the ODA, I wrote an article entitled: A Good Law is Always at Risk. I tried to convey that the original Act had been no “slam-dunk.”

We were focused on diseases with almost no constituencies—ones that were unknown or neglected and had little chance of attracting human and capital investment. I still think of these as “traditional rare diseases,” such as Parkinson’s, Tourette’s, ALS and inborn errors of metabolism. This was the problem the ODA was designed to address.

Cancer was the counter-case—well-organized, lots of public awareness and concern, a well-funded NIH institute and the attention of researchers and drug companies. Only later did we realize that all but four types of cancers are “rare diseases.” One positive and unexpected outcome is that oncology patients have been among those who have received the most clinical benefit from the ODA.

From the beginning, there were worries that the law would richly incentivize drug development that would have occurred anyway. A rewrite of the ODA in the current environment would undoubtedly add restrictions to prevent “windfall profits,” “excessive pricing,” and “market exclusivity for blockbusters.” I have argued—and will continue to do so-- that the overwhelming benefits of the Act (including its remarkable stimulative effect on the growth of biotechnology) have been worth whatever it has cost society by not restricting these outlier situations.

Orphan Drugs/2013. The pace  of designations, approvals, and orphan drug investment has accelerated over the last few years. Along with continued strong Congressional support for orphan drugs, some very large drug companies have suddenly discovered the orphan market.

At the same time, orphans have seen more than their share of controversy and public questioning. At least one company took an inexpensive compounded drug and set a market price for their approved orphan version at $1500, more than 50 times its prior cost. Recently, there have been projections that orphan drugs in development may cost more than $400,000 per patient per year and that “million dollar orphans” are on the horizon.

It is this latter situation that prompted the reporter to ask me if we can afford orphan drugs and “doesn’t something need to be done?” I think not.

FDA approves orphans, but it is up to insurers, health plans and government programs to decide if they will pay for them. A prior column, Drug Pricing 101: A Fundamental Issue Revisited, explains how most companies come up with prices and how they justify them to payers. Apart from the occasional price made up in the boardroom (e.g. the $1500 drug mentioned above), orphans will continue to be reimbursed because some significant portion of their price is justified by the value they add to patient outcomes, the existing treatment costs they replace and/or the amount and length of the discovery and approval process.

I expect lots of future controversy because the per patient prices ARE shocking, but I also expect that the aggregate costs of traditional orphan drugs—including the more expensive ones that are coming—will still not be a major factor in health care cost containment. Downward payer pressure on orphan drug prices will definitely occur. Wholesale failure to pay for orphan drugs by insurers will definitely not occur.

Orphan Drugs/2023. The future of orphan drugs is not, however, as bright as this suggests because we are moving beyond traditional rare diseases and orphan drugs.

 I’ve argued it will take many years before genomics and personalized medicine finally emerge as cornerstones of FDA-approved medications. Slowly, however, over the next decade or so, large, seemingly homogenous diseases (e.g. breast cancer) will start to become subdivided into many diseases based on patient characteristics that are responsive to selective treatment (e.g. the BRAC mutation in breast cancer).

Any such treatment that works for only one group of patients may represent a “medically plausible subset” of less than 200,000 patients (FDA’s criteria for an orphan drug). Once we have reached the point where orphan drugs based on genomics and personalized medicine start to become numerous, then the societal and economic dynamics are different than today’s focus on orphan drugs for traditional rare diseases.  

Conclusion. I have confidence we can find the money for traditional orphan drugs in 2013 and the coming years. I do not know if the societal resources will be there when the scope of rare diseases is expanded by genomics and personalized medicine and the number of orphan drugs skyrockets.

Steven

Off-Label Promotion: Best Resolved by Congress, Not Courts

On December 3, a federal appeals court ruled against one of the FDA’s untouchable restrictions on industry—thou shalt not promote the off-label use of pharmaceutical products. An industry that is little interested in constitutional law suddenly finds itself talking about the First Amendment and whether, and on what grounds, the case will be appealed. 

Meantime, the court’s decision left FDA Matters torn between cheering and booing. Patients are poorly served if their doctor is prescribing drugs without being able to tap into all sources of relevant knowledge. However, permitting off-label promotion undercuts the incentive for companies to thoroughly investigate the safety and efficacy of a drug for a second or third use.

Benefits and abuses of off-label use. Off-label use is a medical necessity--an acknowledgement that the current inventory of approved drug indications is woefully inadequate to deal with the breadth, complexity and individual idiosyncrasies of human disease. All patients—but particularly those with rare diseases—would suffer grievously if physicians did not have flexibility with regard to the off-label use of pharmaceutical products.

Some off-label uses are supported by extensive published scientific studies, but no corporate entity or individual has the financial incentive to underwrite the FDA approval process. Many other off-label uses are in the evidence-thin realm of “we tried it for lack of alternatives and the patient seemed to be better.” The benefits of specific off-label uses are evaluated by published collections of expert opinions (known as compendia). The practice of prescribing off-label is monitored and approved (inconsistently) by payers.

If doctors can legally prescribe a particular drug for a specific use (albeit off-label), then companies ought to be able to provide “truthful and not misleading” information that they possess. Arguably, they can do so now (via reprints of scientific articles), but only in response to a physician’s request. This is a very limited means of disseminating information.*

The Struggle to Incentivize Approval of Second and Third Uses of Approved Drugs.  As I have written previously, it is in the public interest for off-label uses to become on-label indications. The agency is remarkably positive about deferring to the professional judgment of physicians, but would like to see every off-label use get the scrutiny necessary to assure it is safe and effective.

One of FDA’s great fears is that off-label prescribing will become dominant in clinical medicine (as I am told it has in certain areas of oncology). FDA is concerned that companies will receive approval for a first use, then (directly or subtly) encourage doctors to prescribe off-label. If this strategy is profitable, FDA worries that fewer and fewer companies will commit the time and money to gain approval for additional indications. If a company can’t promote off-label, then it is more likely to invest in clinical trials to gain approval of the additional indications.

Unrestricted promotion of off-label use would definitely undercut FDA. In such an environment, I believe that many companies will “game” the system by finding a comparatively easier first use for approval, then let sales for other uses build off-label. Nor do I think companies are universally concerned about “litigation commenced under states’ product liability laws for ineffective products and the resulting reputational harm from such lawsuits.”**

Congress, Not the Courts, Should Set the Ground Rules. By their nature, courts pick winners in these situations; they don’t create rules that maximize two competing public goods. I have been taught (and believe) that litigation is almost always the worst and most inefficient way to solve a problem. I consider this an example.

To achieve appropriate policy, Congressional action is needed.***  For many reasons, this may never happen. However, it is the only way to reconcile the competing and valid positions held by industry and FDA. I encourage Congress to try to bring peace to this area of contention.

Steven

*   Even without permissive policies, company sales and marketing practices are a source of constant problems, as discussed in Off-Label Promotion and Whistleblowing.

**  For those seeking a more legal and enforcement-oriented perspective on the court’s decision, including a number of critical nuances not covered here, I recommend  the FDA Law blog’s current analysis. The paragraph and the quote are based on that article’s description of why some commentators argue that off-label promotion is not a threat to FDA’s drug approval process.

***  A thorough and forthright FDA administrative proceeding (followed by guidelines or regulations) could also clear the air. This is unlikely because the agency generally lacks objectivity on a topic it feels so strongly about, plus the agency tends to respond to court directives, not act to break judicial deadlocks.  On a similarly thorny issue, the agency’s failure, after more than 15 years, to articulate sound policy (or any policy) on social media is indicative of why I don’t think the agency can resolve the “off-label promotion” issue by itself.