- The FDA and the European Medicines Agency (EMA) are working together to advance precision medicine treatments for patients with rare diseases, officials announced last week, marking more than a decade of cross-Atlantic collaboration on drug development, patient safety, and medical research.
The newest FDA-EMA projects will focus on expanding access to clinical trials, improving patient engagement, and continuing to bring precision therapies to market as quickly and safely as possible, wrote Jonathan Goldsmith, MD, FACP, and Sandy Kweder, MD, RADM (Ret.) US Public Health Service, in an FDA blog post.
“Drug development and approval happens across the globe and we at FDA strive to collaborate with other countries and international regulatory agencies to ensure public health,” Goldsmith and Kweder said.
“One of our most valuable collaborators is the European Medicines Agency (EMA) — our counterpart agency for drug regulation in Europe that coordinates a network of 4,500 scientists and evaluates and supervises medicines for more than 500 million people in 31 countries.”
Since 2004, the FDA and EMA have created collaborative “clusters,” or workgroups that each tackle a specific issue related to the complex task of bringing new therapies into the clinical setting.
Previous clusters have addressed pediatric medicine, the development and costs of biosimilar medications, the challenges of treating cancer, and the creation of data standards to aid precision medicine targeted to a patient’s individual genetic makeup.
Last month, the two organizations established a new cluster focused on improving treatment and outcomes for patients with rare diseases, the authors said.
“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.”
Researchers will take a collaborative approach to identifying and validating clinical trial end points, designing meaningful trials for conditions that only affect a very small number of patients, creating flexibility in the drug evaluation process, and expediting the approval of drugs that treat rare diseases to help patients more quickly.
Patient engagement is also on the agenda, Kweder and Goldsmith said. In June, the two agencies formed a workgroup to raise the level of patient involvement in the drug development process.
“FDA and EMA are interested in understanding patient’s 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 wrote.
The patient engagement team will help American and European experts learn more about how patients choose to provide feedback related to clinical trials and other aspects of drug development, and will also brainstorm new strategies for reporting on the impact of patient engagement.
The FDA and EMA are also looking for ways to encourage patients to become community advocates and active participants in agency activities.
“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,” said Goldsmith and Kweder.
“Focusing on patients with rare diseases and working to advance patient input enhances the value of our cluster activities. With our colleagues at the EMA we look forward to accomplishing more than what we can individually.”