FDA’s Dr. Peter Marks on Ways to Preserve Ongoing Medical Research During the Pandemic

By Jenn McNary, Patient Advocate, Founder of One Rare


While most Americans are beginning to talk excitedly – albeit cautiously – about getting back to normal life in the months ahead once the acute effects of COVID-19 wane, those of us in the rare disease community see a much longer and uncertain road.

Recently some patients have not been able to see their physicians for care. For the lucky ones who do have treatments available, those have been interrupted. And most disturbingly for those clinging to a possibility of a scientific breakthrough, clinical studies have been impeded or in some cases, halted altogether. As resources have been shifted to the needs created by COVID-19, other clinical trials have been put on hold and it’s a grim certainty that lives will be lost as a result.

For those of us who continually wait for word of a medical research triumph – like the success of a new gene therapy – the idea that we could lose years of rare disease clinical trials because of this pandemic is unthinkable.

I reached out to Peter Marks, M.D., Ph.D., the director of the FDA’s Center for Biologic Evaluation and Research (CBER) to better understand the agency’s thinking regarding the long-term impact of COVID-19 on drug development for rare disease patients and those studies that have been interrupted.

The good news is that Dr. Marks shares our concern and let me know that an internal working group had been established to address the growing questions around clinical trials in the midst of a pandemic. This working group had already released some guidance with more details to come.

Dr. Marks shared several important thoughts with me during the conversation. Among them:

  • Institutional Review Boards should not preclude moving to home infusions if a patient has been receiving those treatments without any complications in a hospital setting. In order to reduce any interruption in therapy, those changes could be made immediately.

  • We don’t know the outer bounds of the timeline for COVID-19’s impact and how long resources will be focused on coronavirus vaccines and treatments, so we need to plan for the worst. In cases, where there is good solid data, even if gaps exist, the FDA will be flexible and will make the best possible use of existing and placebo data to make decisions on drug approvals.

  • There is no one-size-fits-all approach to handling clinical trial issues during a pandemic. The safety and care of the patient needs to be top of mind, as does moving the needle and continuing to innovate. Trial sponsors need to be proactive, rather than reactive, in working with the FDA on a one-on-one basis to develop the best plan for moving forward. To that end, sponsors should identify the possible issues within their clinical trial, create a data generation plan, then contact their project manager at FDA to help resolve those issues. If sponsors do not get a prompt response, they can email The Center for Biologics (industry.biologics@fda.hhs.gov).

Dr. Marks understands that for those of us whose lives have been affected by rare diseases, time is not on our side. We all know the statistics by heart– for 95 percent of rare disease patients, there is no available treatment option. We all have the perseverance to tolerate one failed study after another because there is always hope that the next trial will yield an answer. What we can’t accept is the idea that no clinical trials will take place.

We, as advocates, should make our voices heard now more than ever. We can help spread the word that there is a path for research to continue even during the pandemic. We are counting on every single component of the medical research engine to think creatively, quickly, and in corroboration with us to move the ball forward. If this isn’t what you’re seeing in your community, you should insist that sponsors reach out to the FDA for support. My conversations with Dr. Marks offered hope that support will be provided.

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The Institute for Gene Therapies advocates for a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access. 

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