With the nomination of Dr. Scott Gottlieb to head the U.S. Food and Drug Administration (FDA) headed to the Senate floor, we can expect another Goldilocks debate. Like the heroine of the classic tale who famously tested the three bowls of porridge to find one too hot, one too cold, and one "just right," participants will debate whether today's FDA is too lenient, too tough, or just right in reviewing new prescription drugs.
That Dr. Gottlieb is qualified for the role he has been nominated for appears beyond question. What critics take issue with is whether his extensive experience ties him too closely to the entities he would be charged with regulating and whether his previously expressed views show a disregard for the FDA's essential public health role. His supporters will counter that his detailed knowledge of the industry will make him a more effective regulator of bad behavior and that his writings demonstrate that he's given thought to the toughest issues.
Those who seek to judge what he would do as leader of the FDA try to discern where he will come down on the porridge question. This question, as simple as a fairy tale, is also 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 the FDA in encouraging innovation as it protects public health at a time when the 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 Limits of Science
At its core, the FDA's role in deciding whether to license new prescription drugs and how to describe their benefits and risks comes down to wrestling with the limits of science to resolve uncertainty. Few new drug applications represent sure things. Resources and time limit the size and length of the clinical trials that test potential new drugs. Another limit comes from the recognition that patients are waiting.
At the same time, trials of sufficient rigor and length are needed to discover whether drugs are truly effective and to discern potentially rare but important side effects. Balancing these conflicting factors is what the staff of the FDA do each day. It is an unenviable task. Their reward for getting it right is a shoulder shrug that they've done what they are supposed to do. Their punishment for getting it wrong is often a day in front of one or more hostile Congressional committees.
To this situation, the FDA staff has brought great skill and judgement throughout Democratic and Republican Administrations. While a veneer of FDA is political, those making these decisions are the career staff whose esprit and perseverance are a strong rebuke to those who criticize federal employees.
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. The bipartisan 21st Century Cures Act, passed by Congress last year, suggests but does not force these tools on the 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
As much as the FDA regulates the approval of new drugs and their manufacturing, they are an agency that regulates speech. The FDA not only approves a drug for market but also delineates, in precise detail, what manufacturers may say (or must say) about the benefits and risks of the drug. In a world where 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.
Today, however, information on drugs respects few boundaries, including national borders. Medical information is accessible freely through any search engine. Patients and describers can easily gain information and misinformation on medicines.
In addition, much use of drugs by physicians is "off-label" especially for cancer. While the FDA may limit what drug companies may say about their products to the uses that appear on the drug's official label (also known as prescribing information), physicians may prescribe their drugs for any condition they choose. Physicians want to understand what drug companies know about their drugs. And health plans want to be able to contract with drug companies based on outcomes such as hospitalizations avoided that likely were not reviewed by the FDA.
Dr. Gottlieb has the opportunity to challenge the 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. It is also a world in which the courts are now challenging FDA attempts to restrict information. In cases such as Amarin Pharma Inc. v. U.S. Food and Drug Administration, courts have found that the FDA cannot sanction drug company promotion that is "based on truthful promotional speech alone."
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 dialogue with doctors and patients.
In December, perhaps anticipating this need and prodded by the enactment of the 21st Century Cures Act, the FDA announced some common sense rules to open up the discussion between drug companies and health plans. One draft guidance provides helpful detail on how drug companies can communicate with payers related to health care economic information. Another addresses a category of communications that FDA describes as off-label but, nevertheless, permissible because it is consistent with the approved labeling.
These are initiatives that a Gottlieb FDA can build on.
Pricing
The FDA has long maintained that is does not have a direct role to play in drug pricing. Given the current volume of the debate, the agency is going to find it harder to stay on the sidelines. And, in Dr. Gottlieb, the FDA would have a leader who has worked at CMS 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, the FDA can play a role in encouraging the competition that health plans and other major purchasers can leverage to control costs.
One tool would be to focus on making the new biosimilar pathway work. The agency has already devoted substantial resources to this effort and the new Commissioner can ensure that it stays the course including publishing final guidances that will light the pathway to approving interchangeable products. Another area of emphasis can be to speed the approval of generic drugs in cases where there is little or no current competition.
The challenges that face a new Commissioner are very much the same as those faced by his predecessors. The possibility of a radical change in direction is constrained by both the legislative framework under which FDA operates and a strong internal culture of commitment to its core mission of protecting public health and facilitating the creation of new therapies. The challenges that the FDA faces are great and would be made 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.
Author's Note
The author represents a number of stakeholders with interests before the FDA including pharmaceutical manufacturers.
from Health Affairs BlogHealth Affairs Blog http://ift.tt/2qwe7if
