The heart of Ellen’s innovation was to foster collaboration among parties that had once held one another at arm’s length, and the innovation that resulted is a turning point for modern FDA reform on behalf of patients. Ellen and her collaborators helped develop policy for the FDA’s Breakthrough Therapy Designation (BT Designation), a provision first authorized by Congress in 2012 as part of the FDA Safety and Innovation Act (FDASIA).
A BT Designation 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 BT Designation applications, has granted over 100 designations, and has approved more than 30 BT Designation treatments. Cancer therapies make up the largest share of BT Designation applications and ultimate approvals.
Collaborating for Patients
So far, Bristol-Myers Squibb has successfully collaborated on five BT Designation applications for Immuno-Oncology agents to treat life-threatening cancers like renal cell carcinoma, non-squamous non-small cell lung cancer, melanoma, Hodgkin lymphoma and multiple myeloma.
We have also brought important new treatments to patients with hepatitis C using this mechanism and we were able to accelerate the Phase 3 development of one of our HIV medicines using BT Designation. In fact, Bristol-Myers Squibb has been one of the most innovative companies in the industry in using BT Designation to bring treatment advances to patients with speed and efficiency.
Today, I had the opportunity to represent Bristol-Myers Squibb at a Senate briefing sponsored by FOCR to provide an update to Congress on the successes and impact of BTD. I reported 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.