FDA and Patient Groups Take Center Stage as Part of Effort to Accelerate #Path2Cures

July 11, 2014

WASHINGTON, DC – The Energy and Commerce Subcommittee on Health, chaired by Rep. Joe Pitts (R-PA), today held a hearing in the 21st Century Cures initiative to incorporate the patient perspective in the #Path2Cures process. Witnesses shared their suggestions and recommendations to bring more cures and treatments to patients more quickly. As Chairman Pitts explained, “Keeping our work centered on the patient and understanding the patient perspective will bring much needed focus on results for patients who may lack adequate treatment options.”

Dr. Janet Woodcock, Director of FDA’s Center for Drug Evaluation and Research, explained, “In recent years, there have been important advances to help ensure that therapies for serious conditions are approved and available to patients as soon as there are sufficient data to show that the therapies’ benefits outweigh their risks. Despite this progress, there is much more work to be done. Many scientific discoveries still need to be translated into treatments for patients in need of new lifesaving therapies. FDA is committed to doing our part to help bridge this gap.”

Dr. Leonard Lichtenfeld with American Cancer Society testified, “Every disease is unique in terms of the unmet need for therapies and the risks that patients are willing to accept in the quest for new treatments. If researchers, pharmaceutical companies and the FDA engage widely and meaningfully with patients, the result will be better treatments delivered to patients faster.”

“I would encourage greater flexibility in clinical trials, particularly for fatal rare diseases like Duchenne that have smaller populations. Business-as-usual trial design simply will not hit the mark when working with such populations,” testified Founding President and CEO of the Parent Project Muscular Dystrophy, Pat Furlong. “We need treatments and therapies, right now, so we can end Duchenne and address the thousands of other rare diseases in need of treatments and cures.”

Dr. Robert Beall with the Cystic Fibrosis Foundation said, “As policy options are considered, it is important to find a balance between accelerating drug discovery and innovation, while also ensuring that patients have safe, effective treatments. … (T)here must be clear communication about the benefits of current therapies so that patients can make informed decisions… as new therapies are developed.”

Members and stakeholders are committed to working together and continuing efforts on the Cures initiative. Dr. Marshall Summar with the National Organization for Rare Disorders emphasized the importance of these efforts, stating, “We view the 21st Century Cures Initiative as a great opportunity to move the process of therapeutic development and approval forward. This is particularly important for Rare Diseases where need is often acute and classic approval models can delay life-saving treatment.”

Alkermes CEO Richard Pops summed up the importance of patient perspectives, testifying, “The simple and powerful concept of incorporating insights of patients, individually and collectively, throughout the continuum that begins with the discovery of new drugs and extends all the way to ensuring patients have access to them – is centrally important to the future of the life sciences industry – and one of the great opportunities for us to have a transformative impact on our nation’s healthcare system.”

Learn more about the 21st Century Cures initiative here and join the conversation on Facebook and Twitter using #Path2Cures.

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