Patients Come First

It is a distracting time for the biopharmaceutical and medical device industries. Everyone is scrambling to gain advantage in the Senate health reform negotiations. Two major pharmaceutical companies disappeared in mega-mergers last week. Reportedly, more medical product companies are working on settlements with the Department of Justice.

All this frenzy makes it a good time to stop, draw a breath and remember why seriously-ill patients care about the success of biopharmaceutical and medical device companies.

Medical products—drugs, biologics, devices—are the hope of millions of Americans who are in ill health, disabled, or face the prospect of chronic disease. Patients want relief from symptoms, reduction in pain, the slowing of chronic disease and fewer restrictions on their ability to think, communicate and manage their own lives.

Who can deliver this? NIH research is essential to the advancement of biomedical science. But it is industry that creates the products for FDA to review for safety and efficacy. Take the companies out of the equation and you have nothing.

For this reason, seriously-ill patients need the biopharmaceutical and medical device industries just as much as industry needs patients. They share a common mission and a pressing need to improve public and individual health. It should be basis for a stronger alliance than exists today.

To unlock this additional potential, industry needs to rise above the many distractions that are unrelated to the needs of patients. Patients don't care which company bought another company, what US Attorney is announcing a lawsuit today, or whether there is a regulatory pathway for follow-on-biologics. Seriously-ill patients want innovations that provide relief and cures and they want to be able to manage their own care.

FDA's two hearings this week--one on risk communications and the other on the use of social media in medical product promotion--provide a timely example. Patients want reliable information in a context they can understand. They would like to be informed without being scared by long lists of unlikely side-effects. Patients want their doctors to be knowledgeable and don't really care how their doctors received truthful, medically-useful information.

In short, patients are not interested in communications that elevate structure over content, coherence or accessibility.

Arguably, this is an area where industry let itself become distracted by its battle with FDA on marketing issues. An earlier resolution might have been possible if a patient-centric approach had been advanced, rather than a commercial one.

Most seriously-ill patients wake each morning wondering how they will surmount the day's challenges. They are concerned about the health of all Americans, but can't help being focused upon their own medical situation. Most of the people I know in the medical products industry wake each morning with the hope that their day's efforts can contribute to improving the health of all Americans, as well as provide specific benefits to seriously-ill patients.

The world-views of patients and industry are not dissimilar. They can share a dream (and an action plan)…if it is built on an unwavering commitment to patients coming first.

Steven

Animal Research: One of FDA’s Core Values

Earlier this year, ABC's Nightline did a story about alleged abuses at the nation's largest primate research center. Fueled by this, the Great Ape Protection Act (HR 1326) now has 95 co-sponsors, compared to 29 sponsors on a similar bill in the last Congress.

The bill would virtually eliminate research using chimpanzees, even where there is no other animal model that could serve to predict safety in humans. This is a threat to animal research and, ultimately, to ourselves. The loss of chimpanzees would be a serious blow to research and would encourage animal rights activists to push for even more restrictions.

The value of animal research in the life sciences is considered an NIH issue. The research advocacy groups opposing the legislation are all part of the network that supports NIH

FDA Matters believes that the FDA and its stakeholders should be equally concerned.

Animal research is the vital first step in the development of new medical products. Before any safety or efficacy testing is permitted in humans, FDA must be satisfied with animal testing data submitted by the product sponsor. Pick any medical breakthrough and you will find animals were tested prior to humans.

For understandable reasons, we tend to focus on the human part of new products. What patients will be helped and by how much? By the time a company files a New Drug Application (NDA) or the equivalent in biologics and devices, the headline is the human data. While the animal data is always relevant, it has largely served its purpose as the gateway for human trials.

We talk about the people part without recognizing that the pipeline of innovative drugs and devices would narrow without chimpanzee research. It would collapse completely if a broader range of research on animals (e.g. monkeys, pigs, sheep, dogs, rats) was heavily restricted.

Everybody should be for protecting the welfare of animals. Any means to lessen our dependence on research animals should be welcome. Animals should always be treated ethically and pain reduced or eliminated. The fewest number of animals should be used to reach a conclusion that can be relied upon. Laboratories should be accredited and subject to inspection. Problems should be addressed within a facility under the watchful eye of government, accrediting and licensing agencies.

While purportedly about the welfare of animals, the House bill is really designed to grant rights to animals, starting with chimpanzees. There are elaborate arguments about whether animals should have rights or just have their welfare protected. For me, the choice is easy. I want a product or procedure tested in animals before it is given to me or my loved ones. I believe in protecting animals, but human rights come first.

Chimpanzees are crucial to animal research. If the House bill were to become law, important animal research might be halted in vaccines, hepatitis A, B and C, HIV/AIDS, malaria and some types of cancer. I am told that in these diseases areas, research on chimpanzees often provides essential information that cannot be obtained in any other way.

The importance of animal research needs to be a core value for FDA. The stakeholder community needs to "seize the day" and make clear where its stands. Those who benefit from animal research (including patients) need to provide the manpower and financial resources to counter the animal rights movement in America and its threat to medical progress for humans.

Steven

The ABC Nightline story is at: http://i.abcnews.com/Nightline/story?id=6997869&page=1.

A good review of appropriate animal research activities and processes is at: http://www.arvo.org/EWEB/dynamicpage.aspx?site=arvo2&webcode=AnimalsResearch

There are many good organizations that work to counter the People for the Ethical Treatment of Animals (PETA) and other animal rights organizations. One of the most effective is Americans for Medical Progress (AMP), an organization that educates media and the public about the importance of animal testing in advancing human health. Among other projects, they are organizing scientists to be more pro-active and articulate about the importance of testing on animals. AMP can always use more support for their work, www.amprogress.org.

The Beatings Will Continue…

….until the biopharmaceutical and medical device industries clean up their act.

It has been an expensive year for pharmaceutical companies. Billions of dollars have been paid to federal and state governments and whistleblowers in settlement of allegations and lawsuits. The complaints include off-label marketing and overcharging Medicaid, but there are many others.

While I am sure there are some "innocent" companies that are the targets of allegations, there are far too many expensive settlements and verdicts for industry to claim that they are victims of overzealous prosecutors and "get rich" whistleblowers. Earlier, FDA Matters called on the biopharmaceutical and medical device industries to recognize that "sales are not more important than laws." ("Off-Label Promotion and Whistleblowing" at www.fdamatters.com/?p=479)

I worry that companies figure they are ahead financially as long as profits exceed penalties. Not for long! The civil and criminal complaints are going to increase and settlements and verdicts are going to get larger. Legislation will get even more restrictive….and a wave of bad publicity will haunt the medical products industries and may forever destroy the public appreciation of the human benefits these companies provide.

US attorneys, state attorneys general, and inspectors general will keep probing until there is nothing left to investigate in the biopharmaceutical and medical device area. This won't happen anytime soon.

Over 180 pharmaceutical fraud cases involving more than 500 drug products are being investigated by the Department of Justice under the False Claims Act. Reportedly, most involve allegations of false and misleading statements made with intent to defraud or mislead. An additional back-log of 1000 whistleblower cases is waiting for DOJ to decide whether to participate. (From comments made by Jennifer Bragg, a partner in the law firm, Skadden, Arps, as reported in Dickenson's FDA Webview).

CEO's need to see corporate accountability as their personal responsibility. The handwriting is on the wall, based on the recent successful criminal prosecution of a small biotech CEO. Sometime soon, a US attorney is going to decide that monetary settlements should not be allowed to wipe out criminal charges. As a result, some senior executives in multi-billion dollar pharma companies may go to jail.

CEO's can no longer afford to trust themselves or their corporate management in judging whether they have followed the law. They need to verify. This requires tougher questions, independent audits of sales and marketing efforts, and alerting physicians to report to the company any meeting where a company representative made false or misleading claims. These companies should be hiring and empowering former prosecutors and investigators to review both policy and performance.

I believe in the transformative power of the biopharmaceutical and medical device industries to lengthen lives, deliver cures, control symptoms, and relieve pain. My advice to CEO's is clichéd but true. When the subpoenas arrive: "Do not ask for whom the bell tolls, it tolls for thee." Is anyone listening in the corporate suites?

Steven

The article cited in Dickenson's FDA Webview is available at:

'Whistleblower' Cases Dominate FD&C Act Prosecutions [639 Words] [ Price : $14.95]

With more than 180 pharmaceutical fraud cases now under investigation by the Department of Justice, FD&C Act prosecutions are now largely driven by "whistleblower" lawsuits, according to Eugene Thirolf, director of DoJ's Office of Consumer Litigation. 10/14/2009

Off-Label Promotion and Whistleblowing

Whistleblowing and off-label promotion of drugs and devices have become hot topics because of the September 2 Pfizer settlement with the federal government. For those who were on vacation: Pfizer is paying $2.3 billion to settle criminal and civil complaints dealing with its marketing and sales practices on four drugs. They have also had to accept a stringent corporate integrity agreement with regard to pre-review and audit of its future marketing and sales activities.

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.

Marketing departments should be able to do better. Even if the clinical trial data is ambiguous, FDA is usually precise in the indications it approves. That limits the scope of claims that can be made by a company. The FDA's promotion rules are far from perfect, but they are clear on most points. For virtually all medical products, it should not be hard for the sponsor to identify promotional claims and activities that are permitted.

Product marketing is a centralized function that requires employees to work together to develop programs and oversee initiatives. It is not an ad-hoc activity. I assume there are multiple sign-offs before any action is taken. Why are these safeguards failing? When things go wrong--which happens remarkably often--is it because everyone in the marketing department is drinking the same fatal Kool-Aid that sales are more important than laws and profits larger than penalties?

Sales practices are harder to control and will always leave companies vulnerable. I have often observed that there are three types of people: those who are shy, those who get over being shy, and salesmen. Medical products companies train sales staffs rigorously, re-train them often, and make them sign documents that attest to their knowledge of the marketing and sales restrictions on bio-pharmaceuticals and medical devices.

However, tens of thousands of sales reps have millions of interactions with prescribers. No matter how well trained and cautioned they are…there will be days when some reps cannot restrain their "inner salesman." How do you guard against that?

Whistleblowers serve a societal function that might not be served any other way. This is painful for me to admit, since I believe so strongly in working within an organization to make things right. I worry that "running to Uncle Sam" might be perceived as a better option than having a candid and concerned conversation with your boss.

However, in many situations, there may be no other way to document certain types of corporate wrong-doing. For that reason, whistleblowing and the resulting qui tam lawsuits are encouraged as a matter of public policy.

My conclusion: it is difficult for bio-pharma and medical device companies to conduct business in a way that makes whistleblowing unnecessary. Nonetheless, FDA-regulated industry can do better than they are presently. We should expect medical product companies to serve patients, prescribers, and shareholders without breaking the law. For those who think the promotion rules are illogical or counterproductive, the remedy is to work for policy changes, not to ignore FDA rules.

I believe that leaders and staff of medical products companies are well-meaning and committed to improving the health of patients. It is detrimental to see off-label promotional activities that can be interpreted as blatant bad faith.

Am I missing factors that might lead to a different conclusion? I encourage readers to post constructive comments to set me straight.

Steven

Notes on the news:

• My September 6^th comments on President Obama's options leading into tonight's speech to Congress on health care reform and how that might affect FDA: http://www.fdamatters.com/?p=471
  
• Senator Dodd has decided not to take the HELP chairmanship. Here are my August 30^th comments on the other contenders and the trade-offs involved: http://www.fdamatters.com/?p=459