US and Europe Collaborate on Patient Treatments

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FDA is working with EMA to treat rare diseases and keep patients involved in healthcare.

In an Oct. 18, 2016 FDAVoice blog post, Jonathan C. Goldsmith, MD, associate director of FDA’s Rare Diseases Program in the Center for Drug Evaluation and Research’s (CDER) Office of New Drugs, and Sandra Kweder, MD, deputy director of FDA’s Europe office and liaison to the European Medicines Agency (EMA), expressed their excitement over FDA’s collaboration with EMA to advance treatments for rare diseases and engage with patients.

The two agencies are involved in ongoing collaborations, called clusters, to address a number of healthcare issues and share information. Experts from CDER’s Rare Diseases Program, the Office of Pediatric Therapeutics, the Center for Biologics Evaluation and Research’s director’s office, and the Office of Orphan Products Development are participating in the current rare disease cluster. EMA and FDA members are collaborating to identify trial end points, discuss potential trial designs, apply flexible evaluation of drug development programs, and expedite the review and approval of drugs to treat rare diseases.

A patient engagement cluster, formed in July 2016, was developed to understand patient experiences and gain input on a patient’s tolerance for risk. The cluster will help the two agencies learn how each other involves patients in their work and explore ways that patients can help be their own advocates.


“Given the focus of both of these new clusters, we expect they will address new areas of interest and also draw on expertise from all of the other clusters, such as oncology, pediatrics, and orphan diseases, contributing to more advanced and robust collaborations across both of our organizations,” Goldsmith and Kweder said in the blog post.

Source: FDA