Drugs

FDA Working with EMA in 2 New Clusters

Since 2004, staff in FDA and the European Medicines Agency (EMA) have collaborated through groups, known as clusters, to help solve major challenges facing the agencies. An FDA Voice blog post says that clusters have focused on treatments for children, measures for developing and using biosimilars as cost-effective alternatives to branded biologics, evaluating new treatments for cancer patients, setting standards to help develop medicines personalized to a patient’s genetic makeup, and more. “Both agencies have benefited from this joint work,” write CDER Rare Diseases Program associate director Jonathan Goldsmith and FDA Europe Office deputy director Sandra Kweder.

They say that last month the two agencies established a cluster to work to advance treatments for patients with rare diseases. “This cluster’s primary goal is for FDA and EMA scientists to share valuable information about their work and to collaborate on certain review aspects of rare disease drug development programs,” they say. Included will be:

identification and validation of trial end points;
potential trial designs when only small populations of patients are available for testing the safety and effectiveness of prospective new therapies;
ways to apply flexibility in evaluating drug development programs; and
expediting the review and approval of drugs to treat rare diseases to bring new drugs to patients in need as soon as possible.

Goldsmith and Kweder say this work builds on a patient engagement cluster formed in June to incorporate the patient’s involvement and viewpoint in the drug development process. “FDA and EMA are interested in understanding patients’ experiences and gaining input on their tolerance for risk and uncertainty, on current therapy and its benefits or shortcomings, and on the benefits that patients seek,” they say. That cluster will:

help each agency learn how the other involves patients in their work and develop common goals of expanding future engagement activities with patients;
discuss ways of finding patients who can serve as spokespersons for their community;
explore ideas to help train selected patients and advocates to effectively participate in agency activities; and
develop strategies for reporting the significant impact of patient involvement.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome