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Pink Sheet – FDA’s Breakthrough Products: Could Patients Drive The Designations?

Pink Sheet – FDA’s Breakthrough Products: Could Patients Drive The Designations?

With the excitement about FDA’s breakthrough designation continuing to grow, patients now are looking to gain some influence over the process, although it is unclear how the agency would incorporate them.

Sen. Michael Bennet, D-Colo., said he and other members of Congress are receiving letters from constituents and patients asking for a say in what is a breakthrough therapy.

Bennet asked Commissioner Margaret Hamburg during a March 13 Senate Health, Education, Labor and Pensions Committee hearing on FDA priorities, whether the agency would talk to patients about information they would supply for a breakthrough designation.

“As a result of this effort to get drugs to patients more efficiently, we’ve seen a number of people writing to our office asking for more direct dialogue with FDA regarding groundbreaking drugs that should be considered to be breakthrough therapies,” Bennet said.

“While no one wants to unduly influence the science, the data that FDA looks at, I believe there should be an avenue for patients to inform the FDA of their opinions about what might be considered for breakthrough,” he said.

Hamburg did not give a direct answer to the question, but the possibility of including patients more directly in the breakthrough process could dramatically affect the program.

FDA has made a concerted effort over the last couple years to increase patient engagement as part of new mandates in the prescription drug user fee reauthorization and 2012 FDA Safety and Innovation Act.

The agency has considered ways of allowing patient comments during the early stages of drug development, including meetings with sponsors and investigators (“FDA Hunting For Ways To Expand Patient Role In Drug Development” — “The Pink Sheet” DAILY, Aug. 8, 2013).

The breakthrough therapy designation, also a product of FDASIA, allows a sponsor to gain early and more-frequent involvement from senior level FDA officials to streamline development. FDA must determine that the product is a significant advance over existing treatments.

Since the program launched, the Center for Drug Evaluation and Research has granted 40 breakthrough requests and denied 68. The Center for Biologics Evaluation and Research has granted one request and denied 16.

Several also have been approved, including Pharmacyclics Inc.’s Imbruvica (ibrutinib) (“Ibrutinib’s Clean Label For Mantle Cell Leukemia Bodes Well For CLL Claim” — “The Pink Sheet” DAILY, Nov. 13, 2013).

Breakthrough designation decisions are made by the CDER Medical Policy Council with input from the relevant review divisions (“Senior CDER Management Panel Could Facilitate Novel Therapy Approval” — “The Pink Sheet” DAILY, Jan. 11, 2012).

There almost surely is patient input considered as part of that process. While there does not appear to include direct patient involvement, it would seem logical for the agency to consider the needs and wants of patients when determining whether a potential therapy would be such an advance as to merit the increased FDA involvement (“CDER Medical Policy Council Balances “Breakthrough” Requests With Broader Issues” — “The Pink Sheet,” Apr. 15, 2013).

How Would Patients Increase Involvement?

It remains unclear how FDA could further involve patients in the breakthrough process other than through listening to their treatment needs.

The agency has commissioned a number of disease-specific meetings with patients and advocates to learn more about unmet needs and disease burden. They have been well-attended and the agency has said they have provided a lot of information that its staff can use (““Patient Voice” Report Puts Chronic Fatigue Symptoms, Treatments In Benefit/Risk Framework” — “The Pink Sheet,” Oct. 7, 2013).

Some of that information appears to have helped the agency write guidance on the development of drugs treating chronic fatigue syndrome/myalgic encephalomyelitis (“FDA Chronic Fatigue Guidance Takes Flexible Stand On Patient-Reported Outcomes” — “The Pink Sheet” DAILY, Mar. 10, 2014).

CFS/ME was the subject of one of the patient-focused drug development meetings in 2013 (“Chronic Fatigue Meeting Shows Benefits, Hurdles To Patient-Focused Drug Development” — “The Pink Sheet,” May 6, 2013).

But stakeholders also have raised concerns about how the agency should use patient input.

Stakeholders have asked the agency to create a formal system for including patient input into decision-making, but the agency has expressed skepticism (“Systematizing Patient Views Unlikely, FDA’s Temple Says” — “The Pink Sheet,” Feb. 24, 2014).

Could All Of FDA Embrace Breakthrough Concept?

Hamburg reiterated to Bennet during the hearing that the breakthrough program has been much more popular than anticipated.

Bennet said he hoped the idea eventually could spread throughout the agency.

“As we see the FDA exercise its muscles around this one part of the pipeline, my hope is that … we’re going to be able to see it across the FDA,” he said.

Agency officials have said that breakthrough brings a number of increased resource demands, although it said that it would be able to balance the demands of that with its other product review programs (“FDA’s Breakthrough Designations Face Question Of Review Resources” — “The Pink Sheet” DAILY, Jul. 25, 2013).

Office of New Drugs Director John Jenkins said in December 2013 that some items may slip around the edges because of the drain breakthrough has caused (“FDA Strains Under New NDA Review Model; Will Industry Want To Retain It?” — “The Pink Sheet,” Dec. 16, 2013).…;