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Inside Health Policy – Senate 'Breakthrough' Bill May Complement FDA Accelerated Approval Reform

Inside Health Policy – Senate 'Breakthrough' Bill May Complement FDA Accelerated Approval Reform


A recently introduced bipartisan Senate bill proposing a breakthrough drug designation to expedite development for drugs that show promise early on could work in tandem with another pending proposal

targeting accelerated approval process, according to stakeholders backing both proposals, which are eyed as policies to be attached to FDA user fees. FDA drug officials voiced support for the breakthrough designation idea, which comes as the Senate health committee gears up to release a user fee draft and hold a hearing on the issues.

A bipartisan group of senators introduced a bill Monday (March 26) that calls for the breakthrough drug designation to expedite the drug development process for products that show dramatic responses early, with the proposal calling on FDA to hold meetings, interact with sponsors and consider smaller or alternative designs for clinical trials for drugs with the designation.

The bill, backed by venture capitalists and a cancer research group, was unveiled after FDA and industry hammered out a separate accelerated approval proposal with Sen. Kay Hagan (D-NC). Sens. Michael Bennet (D-CO), Orrin Hatch (R-UT) and Richard Burr (R-NC) introduced the Advancing Breakthrough Therapies for Patients Act aimed at giving FDA more flexibility when drugs show an early response.

“We are very supportive of both of these pieces of legislation and feel that they each would provide the FDA with unique and enhanced tools to get new treatments to patients,” said Ellen Sigal, chair and founder of Friends of Cancer Research. “Through the bipartisan ‘Breakthrough’ legislation, Senators Bennet, Hatch and Burr are showing great leadership to address the real needs of patients suffering from diseases like cancer.”

Hagan’s accelerated approval proposal is included in a bill that outlines other FDA reforms, the Transforming the Regulatory Environment to Accelerate Access to Treatment Act (TREAT). It would codify FDA’s current accelerated approval regulations. There is also a pending bipartisan House bill that is similar to the accelerated approval section of TREAT.

Stakeholders said TREAT and the breakthrough designation would complement each other. “I would say that these two (bills) have the potential to work very well in tandem together,” said Jeff Allen, executive director of Friends of Cancer Research.

While TREAT’s accelerated approval provision targets approval standards, the breakthrough designation creates a process where FDA would work closely with drug sponsors throughout the development of certain products, he said. The breakthrough designation would apply to serious or life-threatening diseases where clinical evidence shows an improvement over current treatments, the lawmakers said. Breakthrough therapies could still seek a fast-track product designation, accelerated approval or priority review.

FDA officials have spoke in favor of the proposal, saying they would like to focus on the data that shows early effectiveness and work with sponsors on clinical trial designs to bring the product to market more quickly (see related story).

Allen said preliminary estimates show there could be a couple such designations per year, therefore it would not be overly burdensome to the agency. “I wouldn’t imagine this is going to be a resource intensive activity for the agency,” he said.

An industry source agreed that the breakthrough designation proposal could complement TREAT, but said the designation creates a process for the agency that could require more resources, while the accelerated approval proposal codifies existing practices.

“The problem is that this is coming in way late in the (Prescription Drug User Fee Act) process,” the source said. But while it won’t likely make it into the forthcoming Senate draft, it could make it into a later version, especially given that it has bipartisan support, the source said.

“We’re seeing major breakthroughs in drugs and other treatments for debilitating and terminal diseases, but we’re not always getting them to patients though the most efficient and safe pathways,” Bennet said in a statement. “I have heard from countless Coloradans that the FDA needs to modernize its regulatory science to keep up with 21st century science and technology. Our bill provides the flexibility in approval mechanisms so home-run treatments that show great promise early on reach patients more quickly. It also strikes a careful balance between providing regulatory certainty for developers of these breakthrough treatments and maintaining the level of drug safety and efficacy patients expect and deserve.”

In addition to Friends of Cancer Research, the proposal has garnered support from the National Venture Capital Association. Jonathan Leff, member of the NVCA’s board of directors and managing director of the healthcare investing practice at Warburg Pincus, said the two legislative proposals are separate tools that could sometimes work together depending on the therapy.

“I think they’re both important and they do different things,” he said. “The main importance of both of them is the fact that we’re having this discussion.” The breakthrough designation will foster learning experiences and feedback, with the hope that stakeholders will become more comfortable with tools used to rapidly develop drugs, he said. Therefore, those lessons could be applied more broadly to the drug development process, he said.