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CLSA Wire

A Conversation Around Patients

For the last several years, BayBio Board Member Steve Engle has urged us to increase the involvement of patients in our activities and programming. We agree with him and have themed   our upcoming annual conference around patients. CALBIO2012: Driven By Patients will connect you with disease foundations and patient groups when we convene our statewide meeting on March 8-9. Steve returned from Partnering for Cures in early November and we asked for his insights from the conference.

BayBio: How was the Partnering for Cures conference? What were the key insights and takeaways for you?

Steve Engle: With over 800 participants, the meeting was well attended by patients, researchers, philanthropists, companies, and regulators. There were several great sessions with industry leaders. KPCB’s Partner Brook Byers spoke to the critical need for a personalized medicine approach and the rapid adoption of diagnostic tools. Onyx Pharmaceutical’s CEO Tony Coles, like others in our industry, shared his passion for helping suffering patients.

Everyone agrees that medical science and research tools are more powerful today and there remain numerous major unmet medical needs. Unfortunately, the approval process is longer and more expensive if not prohibitive in some diseases. In response, industry is focusing more on translating innovative science into treating orphan diseases using support from NIH, venture capital, and disease foundations. BIO President and CEO James Greenwood emphasized the need to support FDA funding in order to assure the availability of innovative medicines that are safe and effective.

BB: You attended and spoke at the 2010 conference. Have you seen changes in the relationship between companies and patients since then?

SE: Based on the patient advocacy presentations, it would appear that industry is working closer with many patient groups, and increasingly in the orphan disease area. Some patient advocacy groups like Michael J. FoxSusan B. Komen for the Cure and the Cystic Fibrosis Foundation manage the R&D process very well. Other groups are still learning.

Compared to last year, patient advocacy groups are more involved in driving the research agenda through multisite research collaborations, early and clinical stage funding, FDA collaborations and other means. There was concern that potential near-term reductions in government spending could affect basic research efforts at the NIH and regulatory capacity and capabilities at the FDA. There were calls to maintain and even dramatically increase the FDA and NIH budgets. The FDA mentioned several new efforts to improve the drug development and approval process that include finding a way to share results to avoid duplicating the failure of previous studies. Research Foundations really are becoming a key source of funding and a resource for companies, especially if government funding is cut.

Venture investors are investing larger amounts in fewer new companies to better assure success. FasterCuresfounder Michael Milken brought some very knowledgeable investors that confirmed the concerns of many – it is difficult to finance medical innovation in the near-term. Of course, everyone is concerned about this impact on long-term medical costs and patients’ quality of life.

BB: What’s your current evaluation of industry/patient relationships?

SE: Both sides are interested in improving the relationships as they have a common goal: medical innovation. Despite the 35 FDA drug approvals last year, large unmet medical needs like neurodegenerative diseases are not being well addressed. Reduced research on Alzheimer and Parkinson’s is stymied primarily because disease progression is slow and irregular – studies require 18 months and 3,000 patients – and researchers don’t yet have the tools to see changes in the brain. Research spending by industry on Type II diabetes disease, a worldwide epidemic, has been greatly curtailed in the last two years and some would say primarily due to the increase in regulatory burden. There really is a great opportunity here for industry/patient collaboration.

BB: How can companies work better with patients and disease foundations?

SE: The meeting was a starting point for finding ways to improve the medical innovation and approval process. As Susan Desmond-Hellmann, M.D.,  Chancellor, University of California, San Francisco, said, we need to conduct five-year studies in less than five years. In a brainstorming moment, John Mendlein, Chairman, Fate Therapeutics, suggested our generation’s “moon shot” might be a national genome registry with patient follow up. Of course, these conversations must continue. The next chance will be at the CALBIO2012 conference in San Francisco on March 8-9.