The Breakthrough Therapy Designation: How a best practice campaign can shift the lines in patient access to medicines

Dec 18, 2019
The Breakthrough Therapy Designation: How a best practice campaign can shift the lines in patient access to medicines

In little over seven years, FDA Breakthrough Therapy Designation has been given to almost 200 drugs, resulting in more than 70 treatments being approved approximately two years earlier than would otherwise have been expected, according to Dr. Jeff Allen, CEO at Friends of Cancer Research (FoCR).

First passed by Congress in 2012, the designation was the brainchild of the Friends of Cancer Research (FoCR), according to Jeff, who credits effective collaboration with FDA with the program’s success.

“About 15 years ago, we recognized the critical role the FDA plays in the advancement of new drugs and medical technologies,” he observes. “At that time, very little attention was focused on the agency, despite it being essentially the cross-section between the discovery and the delivery of new therapies.”

At the beginning, Jeff reveals, “we brought together various stakeholders to analyze cases where a new drug, very early on in development, showed particularly remarkable effects; for example, a phase one clinical trial that had an unprecedented response rate in a previously untreatable cancer.

Expediting transformative therapies

“What we intended to do, through that publication, was to try and expedite the development of these truly transformative products. It was about moving away from the traditional phases of drug development.”

Breakthrough status, which a drug company can apply for if they have preliminary clinical evidence showing a clear advantage over existing therapies, allows for help from the FDA to fast track the development and licensing process, leading to greater access to cancer medicines, Jeff explains.

The report’s findings informed a policy program that the FDA, with support from FoCR and other stakeholders, developed and passed into law. Since then, says Jeff, “we’ve seen the program be implemented extremely robustly by the agency – probably more than we originally thought.”

“It’s helped to identify new targets and we are encountering treatments that offer benefits to patients with previously untreatable diseases.”

 21st Century Cures Act

The group also worked with the FDA on the 2016 21st Century Cures Act, designed to help accelerate funding for biomedical research. As part of this, the Oncology Center of Excellence at the FDA was created to streamline regulatory activities for different medical technologies.

“We were interested in looking at this because there is an increased frequency of new drugs requiring a companion diagnostic, but these technologies are regulated by a different part of the FDA,” says Jeff. “This results in a cumbersome process for patients, regulators and drug makers.”

“There’s a growing interest from pharmaceutical organizations and the FDA itself to think about how they engage patients in the process,” he adds. Optimizing the regulatory pathway for complex treatments will not only promote the science of precision medicine and the development of innovative cancer therapies but could also benefit patients in need of life saving therapies.

The Breakthrough Therapy Designation: How a best practice campaign can shift the lines in patient access to medicines

Jeff Allen is President and CEO of Friends of Cancer Research (Friends). Friends is an advocacy organization based in Washington, DC that drives collaboration among partners from every healthcare sector to power advances in science, policy, and regulation that speed life-saving treatments to patients. For more than two decades, Friends has been instrumental in the creation and implementation of policies ensuring patients receive the best treatments in the fastest and safest way possible.
For over 10 years, Jeff has been a driving force in the growth and success of the organization. Under his leadership, Friends has evolved into a nimble, forward-thinking policy, public affairs, and research organization. As President and CEO, he leads the strategic development and implementation of Friends’ scientific, policy, research, and legislative initiatives, as well as overseeing Board governance and organizational operations.
As a thought leader on many issues related to the Food and Drug Administration, regulatory strategy, and healthcare policy, he is regularly published in prestigious medical journals and policy publications. In addition to participating in major scientific and policy symposiums around the country each year, Jeff has had the honor to be called to testify before Congress on multiple occasions and regularly contributes his expertise to the legislative process. Recent Friends initiatives include the establishment of the Breakthrough Therapies designation and the development of the Lung Cancer Master Protocol (Lung-MAP), a unique partnership that will accelerate and optimize clinical trial conduct for new drugs.
Jeff has the privilege to also serve on a variety of influential committees, boards, and advisory councils including the Alliance for a Stronger FDA (Board Member, Past President), the Medical Evidence Development Consortium (MED-C; Board Chair), Lung-MAP Senior Leadership Team Member, and a participant on working groups convened by the National Academies of Medicine and President’s Council of Advisors on Science and Technology (PCAST).
Prior to joining Friends, Jeff was an endocrinology researcher at the National Institutes of Health. His background in cancer research focused upon molecular changes associated with cancer formation as well as treatments to prevent cancer progression. Jeff received his Ph.D. in cell and molecular biology from Georgetown University, and holds a Bachelor’s of Science in Biology from Bowling Green State University.

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