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

Change Together – 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.…