Breakthrough Therapy Designation: Exceeding Expectations Three Years Later

Program highlights a commitment to getting new options to patients quicker.


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As the U.S. Senate discusses the 21st Century Cures Act, which promises to accelerate the discovery and development of cures in the country, we look at the success of another program with the same goal: breakthrough therapy designation. Three years after its introduction by the U.S. Food and Drug Administration (FDA), this program is exceeding expectations.

“We never envisioned the breakthrough designation to be so robustly implemented as it has been,” said Jeff Allen, executive director at Friends of Cancer Research, a Washington, D.C. advocacy organization that helped to develop the program. “It’s wonderful to see how widely the FDA has been using it because it means patients are getting access to more treatment options sooner.”

In 2012, the FDA introduced the designation to speed the development of new therapies that could significantly improve the treatment of serious medical conditions. It does so by providing developers with regular interactions with FDA staff at all levels, a rolling review and a shortened review timeline.

Since then, the FDA has received 330 requests for a breakthrough designation and has granted it to 99 of those requests, including a chimeric antigen receptor T-cell therapy that is the focus of a collaboration between Celgene and Juno Therapeutics.

Breakthrough Therapy Designation: More Designations Granted Than Expected

“I don’t think anyone would have expected close to 100 designations in a little more than three years,” Allen said. “We’re already starting to see a number of approvals coming from products that have received the designation.”

Already, the program has provided patients with access to 26 new treatment options up to three years sooner than the standard approval process. And 16 of those were first-time approvals for novel therapies, which represent a new or different approach to attacking a particular disease. Others were approvals for existing medicines to treat different diseases.

Breakthrough Therapy Designation: More Approvals Than Expected

The diversity of therapeutics areas that the program has involved has also been a pleasant surprise. While cancer therapies received many of the designations, as expected, rare inherited disorders, infectious diseases and cardiovascular diseases also benefited.

Breakthrough Therapy Deisgnation: More Diversity Than Expected

The program’s success comes down to the all-hands-on-deck approach by the FDA and its industry partners, according to Allen. “While many focus on how the designation has changed the culture at the FDA, it’s a very intensive process for the sponsors as well,” Allen said. “They have to meet the deadlines as rapidly as the FDA is advancing their internal processes.”

Some have voiced worries about safety and efficacy data from accelerated clinical trials and the strain on resources for the FDA and sponsors, but Allen does not share those concerns.

Thankfully for the patients that will benefit from these therapies, a lot of hard work is going into this program.

“Having had a hand in writing the law, we explicitly did not alter the standards that have been in place for decades,” Allen said. “From a legal standpoint, there’s nothing adjusted in terms of the levels of evidence needed to demonstrate efficacy and safety.”

The program is only applied to therapeutic areas of unmet medical needs for serious and life-threatening diseases. The alternatives for these patients are limited, so making new treatment options available as quickly as possible while still maintaining confidence in the therapy is important.

While the program is resource-intensive, the FDA and sponsors have both shown committed efforts to prioritizing these much-needed therapies and expediting their development. “It requires a lot of careful planning and a lot of collaboration,” said Allen. “Thankfully for the patients that will benefit from these therapies, a lot of hard work is going into this program.”

Additional resources may be coming to more broadly implement programs such as the breakthrough designation. For instance, the 21st Century Cures Act would continue to modernize the clinical trials process and increase FDA funding by $550 million over 5 years.  Following months of legislative and advocacy work, the U.S. House of Representatives passed their version of the bill in July. Now it’s the Senate’s turn to show they are equally committed to providing new treatment options for patients quickly and safely by passing this legislation.