Gottlieb Facing 3 Complex Challenges: Blog Post

A Health Affairs blog post outlines three complex challenges facing FDA commissioner nominee Scott Gottlieb and ways in which he could address them. The post says that critics and supporters who seek to judge what Gottlieb would do as commissioner are trying to discern the answer to a question that is “shockingly simplistic. It masks the far more complex challenges that, if confirmed, Dr. Gottlieb will face. For today’s debate is more about the limits of science to deal with the inevitable uncertainty about the safety and effectiveness of new medicines and the proper role of FDA in encouraging innovation as it protects public health at a time when FDA can no longer control information on drugs in the way it once did. It is also occurring as the discussion of drug pricing turns louder and more bellicose.”  The challenges include:

•          The Limits of Science. The post notes that few drug applications represent sure things and resources and time limit the size and length of clinical trials testing new drugs. It notes that the FDA career staff has done a good job under administrations led by both political parties in balancing the need for prompt drug approvals with the need for assurances of safety and effectiveness. “Dr. Gottlieb can encourage the difficult work that these career staff do in examining their own procedures and in working with industry and academic scientists to bring the latest tools to the task of resolving uncertainty,” it says. “The bipartisan 21^st Century Cures Act, passed by Congress last year, suggests but does not force these tools on FDA. These include new methods of trial design and statistical analysis. They also include the use of evidence from real world use post-approval that can provide important information on the benefits and risks of new medicines. To the extent that these tools can reduce uncertainty in drug approvals, we all win.”
•          Medical Information. The author notes that in addition to regulating approval of new drugs and their manufacturing, FDA also regulates speech in terms of what manufacturers may say or must say about their products. “In a world when medical information was closely controlled by physicians and when information on drugs was largely limited to medical journal advertisements, drug company sales pieces, and direct-to-consumer ads, such a role made much sense,” the article says. “Today, however, information on drugs respects few boundaries, including national borders. Medical information is accessible freely through any search engine…. Dr. Gottlieb has the opportunity to challenge FDA staff to navigate this new world where it is less able to control information and where, arguably, there is less need to exert such control as sophisticated audiences have increased abilities to analyze information….Dr. Gottlieb can challenge the agency to adapt their rules in a manner that continues to prevent companies from making unsubstantiated marketing claims, but allows them to participate fully in a lively and truthful dialog with doctors and patients.”
•          Pricing. “In Dr. Gottlieb,” the article says, “FDA would have a leader who has worked at CMS (the HHS Centers for Medicare and Medicaid Services) and has thought deeply about drug-pricing issues. It would be surprising if Dr. Gottlieb disturbed the agency staff’s traditional abhorrence of drug importation. However, FDA can play a role in encouraging the competition that health plans and other major purchasers can leverage to control costs.” One way, it says, would be to focus on making the new biosimilar pathway work. Another area of emphasis it recommends is to speed the approval of generic drugs in cases where there is little or no current competition.