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  1. FDA In Brief

FDA in Brief: New policy to encourage development of treatments for rare diseases

January 16, 2019

Media Inquiries

  Sarah Peddicord
  301-796-2805

“The FDA is committed to supporting the development of treatments for patients with rare diseases and has been focused on advancing policies that will help enable these opportunities. We know that developing a drug or biologic for a rare disease can be especially challenging, which is why it’s important that the FDA continues to provide clear information to drug developers so that they can plan modern, efficient drug development programs that will be successful,” said FDA Commissioner Scott Gottlieb, M.D. “The revised draft guidance issued today discusses select issues commonly encountered in rare disease drug development. While similar issues often also come up for common diseases, they’re frequently more difficult to address in the context of a rare disease for which there’s often limited medical and scientific knowledge. Early consideration of these issues gives sponsors the opportunity to address them and to have productive meetings with the FDA. We’ve revised this guidance to provide additional advice to innovators on how they can pursue more efficient, effective development programs. Many of the updates are in direct response to feedback that we received on our prior draft. For example, we’ve updated the section describing the use of information about natural history as clinical comparators and added more information on the use of historical controls and early randomization. These are approaches that can help make it more feasible to study certain very rare conditions, where finding and recruiting patients and conducting trials can be especially challenging. By continuing to provide clarity and direction to drug developers, we hope to help facilitate the development and approval of more safe and effective treatments for patients with rare diseases.”

Today the U.S. Food and Drug Administration issued a revised draft guidance, “Rare Diseases: Common Issues in Drug Development Guidance for Industry,” that aims to assist sponsors of drug and biological products for the treatment or prevention of rare diseases in conducting more efficient and successful drug development programs. The draft guidance has been updated with information gleaned since the original draft guidance was published in 2015. Among the revisions are updates to the natural history studies section, inclusion of issues for evaluation and validation of biomarkers as surrogate endpoints, discussion of additional flexibility on the non-clinical aspects of the evaluation of novel drug compounds, the addition of a section describing the evaluation of safety questions and added information on changes to drug substance or drug product manufacturing processes with clarification on areas of additional flexibility.

The FDA also plans to hold a public meeting to obtain patients’ and caregivers’ perspectives on the impacts of rare diseases on daily life and to assess their common experiences that may help the agency and medical product developers further understand and advance the development of treatments for rare diseases, potentially through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare diseases.

During this period without a FY19 appropriation for the FDA, the agency has been focused on making sure it continues critical aspects of its work, to the extent permitted by law. At this time, for products covered by a user fee program, such as those for treating rare diseases, the FDA’s review of existing medical product applications and associated policy development regarding FDA review is funded by limited carryover user fee balances. The FDA will continue to update the public on how the agency is approaching its work.

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