Editor’s Note: The nomination of Dr. Scott Gottlieb—who has now been confirmed to lead the Food and Drug Administration (FDA)—drove another Goldilocks debate. Like the fairy tale heroine who tested three bowls of porridge to find one too hot, one too cold and one just right, participants debated whether today’s FDA is too lenient, too tough or just right in reviewing prescription drugs. In a recent post for the Health Affairs Blog, written prior to Dr. Gottlieb’s confirmation and summarized below, Manatt Health examines the views of both the critics and supporters of Dr. Gottlieb, as well as the challenges that he will face as the new commissioner. Click here to read the full post.
Dr. Gottlieb is clearly qualified to assume the role of FDA commissioner. What critics take issue with is whether his extensive experience ties him too closely to the entities he will be regulating and whether the views he’s expressed show a disregard for the FDA’s public health role. Supporters counter that his detailed industry knowledge will make him a more effective regulator and his writings demonstrate that he’s thought about the toughest issues.
Those who seek to judge what Dr. Gottlieb will do as leader of the FDA try to discern where he will come down on the simplistic porridge question. This question masks the far more complex challenges he’ll face—the limits of science in dealing with the uncertainty about the safety and effectiveness of new medicines, the proper role of the FDA when it can no longer control information on drugs, and the issues around drug pricing.
The Limits of Science
In its role of deciding whether to license new prescription drugs and describe their benefits and risks, the FDA wrestles with the limits of science to resolve uncertainty. Resources, time and the recognition that patients are waiting limit the size and length of clinical trials. At the same time, rigorous trials are necessary for discovering whether drugs are effective and discerning potential side effects. The FDA staff has brought great skill and judgment to balancing these challenges throughout both Democratic and Republican administrations. While the veneer of the FDA is political, those making the decisions are career staff whose esprit and perseverance are a strong rebuke to critics of federal employees.
Dr. Gottlieb can encourage the career staff in examining their procedures and putting the latest tools to work in resolving uncertainty, including new methods of trial design and statistical analysis, as well as the use of real-world evidence post-approval to assess benefits and risks. To the extent these new tools reduce uncertainty in drug approvals, we all win.
The FDA not only approves a new drug but also delineates what manufacturers may (or must) say about its benefits and risks. When drug information was largely limited to medical journal advertisements, drug company sales pieces and direct-to-consumer ads, this made sense. Today, however, medical information is freely accessible through any search engine.
Dr. Gottlieb has the opportunity to help the FDA navigate this new world where it is less able to control information. He can challenge the agency to adapt its rule to prevent companies from making unsubstantiated claims but allow them to participate in a truthful dialogue with doctors and patients.
In December, the FDA announced some commonsense rules to open up discussions between drug companies and health plans, such as a draft guidance on how drug companies can communicate with payers about healthcare-related economic information. Dr. Gottlieb can build on these kinds of initiatives.
Though the FDA has maintained that it does not have a direct role in drug pricing, the current volume of the debate will make it harder to stay on the sidelines. In Dr. Gottlieb, the FDA has a leader who has worked at the Centers for Medicare and Medicaid Services (CMS) and thought deeply about drug pricing. While it’s unlikely Dr. Gottlieb would disturb the FDA’s traditional abhorrence of drug importation, he can play a role in encouraging the competition that health plans and other purchasers could leverage to control costs.
One tool would be to focus on making the new biosimilar pathway work, including publishing final guidance. Another would be to speed the approval of generic drugs where there is little to no current competition.
The challenges that the FDA faces are great and will be even greater if its resources are cut as proposed in the president’s budget. Yet the agency and its leaders have consistently tuned out the noise of those arguing that the regulatory porridge is too hot or too cold in order to advance their mission.