July 31, 2019

Media Inquiries

  Jeremy Kahn
  301-796-8671

“In recent years, companies and the FDA have taken great strides to make the drug development process more efficient, without compromising FDA’s gold standard for evaluating drug safety, efficacy and quality. The FDA’s breakthrough therapy designation program, and its more recent regenerative medicine advanced therapy (RMAT) program, are designed to help speed development of innovative products to address unmet patient needs. In fact, since the breakthrough therapy designation was first introduced in 2012, the FDA has approved more than 150 drug and biologics for patients with unmet medical needs. These treatments have included the first-ever approved gene therapy, the first tissue-agnostic treatment in cancer, and other treatments that have made significant impacts on diseases like cystic fibrosis, depression and cancer. For products in expedited development programs, applicants are still expected to provide timely clinical, non-clinical, and manufacturing information that meets FDA approval standards. The FDA understands that gathering this data on more compressed timelines could present challenges for companies, which is why the agency has been engaged in open dialogue to support companies in meeting approval requirements without lowering the high standards patients have come to expect,” said FDA Acting Commissioner Ned Sharpless, M.D. “We’ve taken important steps to advance how development and approval of these drugs is approached through collaboration with our European counterparts and industry. Last year, we discussed the challenges companies may face in developing breakthrough or RMAT therapies and the possible scientific and regulatory approaches companies could use to facilitate development and the submission of necessary quality information for timely review and approval. Today, we’re offering our report on the meeting. We will continue to work with our EMA colleagues and industry to discuss ways to address quality challenges associated with expedited development programs to help ensure patients receive safe, effective drugs.”

The FDA and the European Medicines Agency (EMA) are publishing the discussion and main conclusions from a workshop held on November 26, 2018, at the EMA headquarters in London, supporting quality development for the FDA’s Breakthrough Therapy Designation and EMA’s Priority Medicines (PRIME) programs for patients with unmet medical needs. The workshop between regulators and industry discussed quality challenges and scientific and regulatory approaches for facilitating development and preparation of robust quality data packages, to enable timely access to medicines for patients while keeping in mind the importance of drug safety and quality and maintaining current standards of approval.

Multiple topics were discussed, including process validation, Current Good Manufacturing Practices, control strategies, obtaining required stability data, and the exploration of additional regulatory tools. Industry was able to take the opportunity to share their successes and concerns in this area.

Following the passage of the Food and Drug Administration Safety and Innovation Act in 2012, the FDA implemented the breakthrough therapy designation, which was intended to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy. In December 2016, Congress amended the Food Drug and Cosmetic Act, adding a new section which specifically addresses the expedited development and review of certain regenerative medicine therapies designated as RMATs. In March 2016, EMA launched the PRIME scheme to strengthen its support in the development of medicines that address unmet medical needs with the aim to help patients to benefit from these therapies as early as possible.

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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.