RARE Daily

Doing What They Can

June 17, 2020

The U.S. Food and Drug Administration want to assure rare disease patients that rare disease drug development and access are still top priorities despite the COVID-19 pandemic.

Last week, Patrizia Cavazzoni, acting director of the agency’s Center for Drug Evaluation and Research, and Peter Marks, director of the Center for Biologics Evaluation and Research posted a piece to the FDA Voices blog.

The blog comes as concerns reverberate in the rare disease community over the disruptive effects of the pandemic on different aspects of rare disease life. Cavazzoni and Marks note that a recent national COVID-19 Community Survey Report from the National Organization for Rare Disorders found among patients suffering from rare diseases that 74 percent of respondents had a medical appointment cancelled due to COVID-19. The survey also showed that nearly a third of respondents (29 percent) had temporarily or permanently lost a job due to COVID-19, with 11 percent of those job losses resulting in lost health insurance.

The disruption to clinical trials has been documented in numerous press releases from drug developers—last month we reported on the work of a researcher who found by analyzing clinicaltrials.gov that of 2,522 clinical trial clinical trials that had been suspended between December 1, 2019 and May 5, 2020, 1,099 (44 percent) provided an explanation that mentioned COVID-19.

“FDA staff work every day to do everything they can to prevent the COVID-19 pandemic from impeding progress in finding treatments for rare diseases through support to sponsors and investigators conducting clinical trials, facilitating access to products while they are in development, and working to help ensure continued availability of approved products critical for patients with rare diseases,” they wrote. “As we continue to prioritize development of new therapies for rare diseases, we are also working to minimize COVID-19 impact on the progress we’re making.”

The FDA has also reached out to manufacturers to evaluate their entire supply chain to identify medications that may be impacted by disruptions caused by the outbreak. For example, as a result of the increase in demand for hydroxychloroquine as a potential treatment for COVID-19, the agency established priority review for all generic hydroxychloroquine drug applications and expedited their review and approval to help mitigate shortages for the approved use of the drug as a treatment for the rare disease lupus.

One way some trial sponsors have sought to avoid delays to their studies has been to bring the trial to patient. Some companies have used digital health technologies, such as telehealth and remote monitoring, and sent health workers to patients’ homes to gather blood. Our recent RARECast with Kathy Goin, vice president of development operations for Palvella Therapeutics, highlights how one rare disease drug developer has navigated the pandemic to avoid clinical trial disruptions.

This may be one positive long-term effect on the pandemic as drug developers adapt to the pandemic and find better ways of conducting trials, particularly for rare disease therapies where trial participants may be geographically diverse and not easily able to travel to a trial site in normal times.

While the FDA has shown flexibility and their efforts to advance therapies are welcome, the reality is that the pandemic will remain a threat to clinical studies until its spread is controlled.  The FDA’s words are encouraging, but the pandemic, by and large, is not a regulatory problem.

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