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Health News Digest – The FDA’s Breakthrough Therapy Designation – Innovation Through Collaboration to Speed Patient Care

Health News Digest – The FDA’s Breakthrough Therapy Designation – Innovation Through Collaboration to Speed Patient Care

Today is a new day for cancer research, with the introduction of transformational immune-based therapies that have the potential to change patients’ lives. Such potential means that everyone must innovatively collaborate on ways to speed the drug development and regulatory process so that potentially life-saving therapies reach patients with the greatest urgency, many of whom do not have time to wait.

Like so many true innovations in patient care, some of the delays in drug development and approval have been addressed most effectively by people with a personal experience of loss. Ellen Sigal watched her sister wait for new treatments to fight her breast cancer, but they did not come in time. In response, Ellen founded Friends of Cancer Research (FOCR) and began to bring the cancer community together with congressional leaders, members of the FDA and the pharmaceutical industry to collectively develop ways to expedite promising drugs for patients with serious or life-threatening conditions that are inadequately treated with existing therapies.

What resulted was policy for the FDA’s Breakthrough Therapy Designation (BTD), a provision first authorized by Congress in 2012 as part of the FDA Safety and Innovation Act (FDASIA). BTD grants the FDA authority to review drug candidates that show early promise in fighting serious and under-treated conditions.

BTD is intended to expedite the development and review of drugs for serious or life-threatening conditions and provides for frequent meetings between the drug sponsor and the FDA team in a collaborative, cross-disciplinary process. Research is evaluated sooner, issues that may result in potential delays in development are anticipated, and deserving therapies are approved faster.

In its first three years, the FDA has received almost 300 BTD applications, has granted over 100 designations, and has approved more than 30 BTD treatments. Cancer therapies make up the largest share of BTD applications and ultimate approvals.

Bristol-Myers Squibb has worked closely with the FDA on five successful BTD applications for Immuno-Oncology agents to treat life-threatening cancers, as well as others for HIV and hepatitis C. At a Senate briefing sponsored by FOCR last week, I had the opportunity to report on the efficiencies that this review process has brought to our therapies that are extending the lives of patients. There are many ways to measure the success of the FDA’s BTD review that Ellen Sigal helped bring about in memory of her sister. Patients like these are the most important measure. They are a testament to how we are collectively working for patients.

We hope that Congress and the FDA will continue to make this important regulatory mechanism available for the future needs of patients. It is innovation born through collaboration and it helps save lives. As an R&D organization, we are committed to prioritizing, together with FDA and other Regulatory Agencies, the development of transformational therapies.…;