9-5-2013 - Inside Health Policy - Cancer Breakthrough Designations Could Provide Clues for Other Diseases | Friends of Cancer Research

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9-5-2013 - Inside Health Policy - Cancer Breakthrough Designations Could Provide Clues for Other Diseases

9-5-2013 - Inside Health Policy - Cancer Breakthrough Designations Could Provide Clues for Other Diseases

September 5, 2013

By Nanci Bompey

Strategies that have allowed a substantial number of cancer drugs to qualify for the new breakthrough designation could be used by sponsors developing drugs in other disease areas to speed up their own development programs, investor and cancer research sources said. FDA's newest expedited pathway is driving a new way of thinking about drug development and, along with efforts to accelerate development of companion diagnostics, could help drive down the high cost and time of bringing new drugs to market, the sources said.

The breakthrough therapy designation created by last year's FDA Safety and Innovation Act has been one of the most watched and praised provisions of the new law. FDA received more than 90 requests for the designation intended to speed up development of treatments for serious or life-threatening diseases that show potential in early clinical trials. The agency granted 26 designations, according to the most recent statics, with 20 designations made public so far.

There have been a substantial number of designations awarded to oncology drugs, including therapies for blood cancers, breast cancer, melanoma and lung cancer. About 17 percent of the breakthrough designations granted by FDA as of the beginning of August have been in oncology, and 25 percent of the designations granted in hematology, according to statistics the agency presented to the Alliance for a Stronger FDA, which advocates for agency funding.

The ability to target groups of patients and detect potential activity early in clinical trials helped cancer drugs qualify for the coveted breakthrough designation, said sources monitoring implementation of the new breakthrough pathway.

Oncology has seen an explosion of targeted therapies in recent years. Researchers are able to select patients who are likely to respond dramatically to a therapy, oftentimes through use of a companion diagnostic, which is enabling these drugs to qualify for the breakthrough pathway, sources said. One requirement of the new designation is that a drug must show substantial response rates in early clinical trials.

Further, oncology drugs use response rate as an endpoint in clinical trials, giving sponsors an early indication of potential activity that is also necessary to gain the designation, sources said. Some other therapeutic areas -- like Alzheimer's disease -- don't have the same kind of endpoints to detect clinical effects, making it more difficult for these disease areas to access the breakthrough designation, said Jonathan Leff, partner at Deerfield Management, who also serves on the boards for the National Venture Capital Association and Biotechnology Industry Organization.

However, Leff noted that FDA has awarded several breakthrough designations for rare diseases and other types of therapies. About 29 percent of the designations have been for antiviral treatments, with multiple therapies for Hepatitis C.

"I think FDA is trying to look broadly and use the breakthrough designation outside of oncology," Leff said. "In general, I think over time we will see breakthrough designations applied to more and more therapeutic areas as we start to see sponsors adjust to the program and adjust their development programs in light of breakthrough."

Jeff Allen, executive director of Friends of Cancer Research, said some of the strategies used to expedite development programs for oncology drugs could be extrapolated to other disease settings. He said oncology drug developers have experience in validating surrogate endpoints and there is an opportunity to think about how to do that in other settings. Further, other diseases may be able to follow in oncology's footsteps as the genetic basis of these diseases become better known.

Allen noted that about three-quarters of the breakthrough designations include some method of selecting a population that will benefit from the therapy, either through a new companion diagnostic, an existing diagnostic or, in the case of some blood cancers, the physical characteristics of the cancer cells.

Friends of Cancer Research is holding a briefing Friday (Sept. 6) to unveil a proposal for speeding up the development of companion diagnostics used alongside therapies that have been granted the breakthrough designation. He said the proposal prioritizes data elements for these devices by outlining high priority elements that need to be collected up front and lower priority elements that could be collected post-market.

Allen said the group is not proposing to eliminate the amount of data collected, but attempting to make the diagnostic development program fit with the compressed development program of the drug. He said a lot can be accomplished within the current statutory framework, but noted that the device center does not have the same post-market authority as the drug center, including the ability to require post-market studies or withdraw a product from the market.

"That is just an important thing to keep in mind but it doesn't mean FDA can't implement a lot of things" in the proposal, he said. Allen added that the group is "looking at all sorts of ways to optimize utilization of the breakthrough designation."

Allen said stakeholders will have a better idea of how the process can be improved once FDA approves a few of the drugs that have received the designation. Some of the first approvals could come for a blood cancer drug being developed by Johnson & Johnson and Pharmacyclics that has received three designations and for Genentech's chronic lymphocytic leukemia treatment. Allen said he is also keeping an eye on a Novartis lung cancer drug that could receive accelerated approval.

Stakeholders and FDA officials have noted that many initial designations went to additional indications for approved products or drugs further along in development, which may not be representative of the types of products that will benefit from the designation in the future.

Allen said FOCR, which spearheaded the breakthrough pathway's inclusion in FDASIA, envisions that the pathway will be used earlier and earlier in the development process and eventually could adjust the traditional course of drug development. He said once there is a better understanding of what happens when a drug receives the designation, companies may even want to evaluate early stage research protocols to find strategies to show large effects early in clinical studies.

"We want to see sponsors be bold and apply for it and we want to see FDA be bold and use it," he said.

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