Tuesday, November 8, 2011
9:15 AM - 10:10 AM
Panel - Streamlining Drug Repurposing and Approval
Michael Beckloff , President, Beckloff Associates, Inc.
Regis B. Kelly, Ph.D., Director, The California Institute for Quantitative Biosciences (QB3), University of California
Stephen P. Spielberg, MD, PhD, Deputy Commissioner for Medical Products and Tobacco, U.S. Food and Drug Administration
Scott J. Weir, Pharm.D., Ph.D., Director, Institute for Advancing Medical Innovation, University of Kansas Cancer Center
Lesa Mitchell, Vice President, Ewing Marion Kauffman Foundation
It used to be that finding new uses for existing drugs or abandoned compounds occurred primarily through serendipity, but more and more, the process for identifying potential "hits" is becoming more deliberate, less expensive, and faster. Strategic collaborations between the public, private, and nonprofit sectors are springing up to shepherd research efforts around abandoned compounds that might work in other diseases, and ensure the policies, processes, and resources are in place to permit their appropriate reuse. This panel examined models and ideas for streamlining the regulatory process for repurposed assets to save time in clinical trials and review .
Moderator Lesa Mitchell, Kauffman Foundation, opened the discussion by highlighting the growing trend of taking technologies that have come off market, or in some cases are still on the market, and repurposing them to address unmet medical needs. Particularly embraced by the disease philanthropy community, Mitchell asked panelists to talk about their experiences with this practice and outline how they've overcome challenges like patenting and cost to bring the resulting technologies to market.
Scott Weir, University of Kansas, talked about their focus on bridging the valley of death through collaboration with industry and foundations - "Over the past three years, we've advanced eight therapies into clinical trials." He went on to describe their role in The Learning Collaborative Partnership with NIH and The Leukemia & Lymphoma Society, aimed at accelerating the development of potential clinical therapies for rare blood cancers. Currently the partnership is evaluating a small molecule drug used to treat arthritis as a possible therapy for chronic lymphocytic leukemia.
QB3's Regis Kelly discussed the Institute's work in rare and neglected diseases; and how it has looked at which proteins bind to different targets and attempted to characterize targets by their ligand profiles to make the repurposing process more efficient and effective. He noted that a key challenge to screening existing FDA-approved compounds is that not all of them are still being made.
Michael Beckloff, Beckloff Associates, addressed the importance of applying science to a regulatory path, offering the example of a topical product that was identified by a researcher to have an activity target in acute myeloid leukemia (AML). Through a collaborative partnership, they were able to identify the hit and build a regulatory pathway that allowed for first in man trials within a window of about 13 months.
FDA's Stephen Spielberg brought up impact of different approaches to drug repurposing: "We've been doing this based on clinical outcomes or phenotypes," he said "based on the side effects of drugs." The difference we face now is the genomic revolution he pointed out. "We are realizing that the diseases that we've given names to in the last 200 years are related even though they look very different. Thus, similar genes may play in the role in a cause of cancer and in arthritis or schizophrenia. Five years from now, diseases will not be called the same things as they are today. Hypertension won't just be hypertension. It will be hypertension due to x, y, and z." He then brought up the issue of using data for drug repurposing: "One of the challenges is how good is the old data? Most FDA data used to be submitted on paper. It's hard to look back at old drugs because of this. Much of the old data is in handwriting that no one can read and stored away in archives and the FDA doesn't own the data. The sponsor does."
Mitchell then steered the conversation towards the "how-to" of the repurposed space. Kelly talked about the importance of creating a library of compounds and what they'd been tested for: "If every lab had a library for drugs and a list of diseases, lining up old drugs with new diseases would be much easier." Beckloff then brought up the subject of exclusivity: "People are asking 'how do I find an exclusivity mechanism?' Exclusivity is important to make a drug attractive for companies." Spielberg pointed out that driving this forward is not easy. "It is truly complex. Public/private partnerships are crucial in getting this done."
The conversation concluded with a discussion of what the single most important thing to keep in mind for drug repurposing is. Two points rose to the top - 1) the need for public policy initiatives dealing with exclusivity (to incentivize innovators), and 2) the need for better training practices around genetics and pharmaceuticals.