NCHR Comments on FDA’s Draft Guidance on Post-Market Studies and Clinical Trials

National Center for Health Research, December 20, 2019

National Center for Health Research’s Public Comments on
Postmarketing Studies and Clinical Trials-Implementation of Section 505(o)(3) of the
Federal Food, Drug, and Cosmetic Act; Draft Guidance for Industry; Availability

The National Center for Health Research (NCHR) is a nonprofit think tank that conducts, analyzes, and scrutinizes research, policies, and programs on a range of issues related to health and safety.  We do not accept funding from companies that make products that are the subject of our work.

The FDA has made it a priority to get medical products on the market faster.  Unfortunately, not all product safety concerns will be identified prior to approval, and it is not feasible for sponsors to conduct clinical trials that can evaluate every possible safety concern.  Thus, in some cases post-market clinical trials and other studies are needed to further the understanding of the occurrence of adverse events after products are approved.  Although the guidance indicates that such studies should only be required when surveillance based on adverse event reports are not sufficient, the fact is that in almost all cases the adverse event reporting system is inadequate to draw conclusions about safety.

The guidance should make it clearer that post-market studies can supplement but not replace essential information about safety or efficacy.  To make informed medical decisions, patients need and deserve solid scientific information about the likely benefits and possible risks of all prescription medications, whether treatment alternatives are available or not.  Relying on post-market clinical trials or other studies is problematic for three reasons:

  1. Patients should not pay for treatments that have not been adequately proven to be safe and effective.  Patients and their physicians need and deserve certainty about safety and efficacy in order to make an informed decision.
  2. Post-market studies often are delayed or inadequately implemented, and in many cases it is impossible to control for confounding variables and changes in treatment protocols that occur after a product is on the market.
  3. Despite efforts to improve diversity, post-market studies frequently have inadequate representation of different racial, ethnic, and age groups.

Even when post-market studies and trials are appropriate, there are major limitations regarding the type of information and quality of data that can be obtained.  These depend on the type of study or trial, many of which are described in the draft guidance.  Choosing methods that are simpler or less burdensome for sponsors, but are less likely to provide information about which patients are most likely to benefit or be harmed, can be wasteful for FDA and the sponsor, while at the same time putting patients at risk.  That is why it is incumbent on the FDA to seek the type of study or trial most likely to provide the data necessary to address each safety concern.

Additionally, where FDA requires a clinical trial or other type of study, and the sponsor fails to follow the regulatory requirements for such, FDA should use all available enforcement powers to ensure the needed information is delivered in a timely and complete manner.  Delayed and incomplete studies harm the public’s ability to make informed decisions about their healthcare.  Penalties, including a moratorium on sales, should be used to prevent delays and improper implementation of required research.

Post-market clinical trials and other studies can help inform FDA and the public about the risks and benefits of medical products.  However, when they are relied upon to answer questions better addressed prior to approval, or if less effective studies or trials are used, or if studies are delayed and inadequate, they do not provide the intended data that is needed by FDA, patients, and healthcare providers.  This adds to the already high cost of medical care.

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