The FDA’s Breakthrough Therapy Designation Helps Speed Patient Care

By Mathias Hukkelhoven, Ph.D, Sr. VP, Global Regulatory, Safety and Biometrics, Bristol-Myers Squibb

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oday 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.

Today is a new day for cancer research, with the introduction of transformational immune-based therapies that have the potential to change patients’ lives.
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An Act of Innovation

Like so many true innovations in patient care, some of the delays in 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, she founded Friends of Cancer Research (FOCR) and began to bring the cancer community together with congressional leaders, members of the Food and Drug Administration (FDA) the pharmaceutical industry to collectively develop ways to expedite promising drugs to patients with serious or life-threatening conditions that could not be adequately treated with existing therapies.

Like so many true innovations in patient care, some of the delays in development and approval have been addressed most effectively by people with a personal experience of loss.

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.

Bristol-Myers Squibb has been one of the most innovative companies in the industry in using Breakthrough Therapy 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.