MassBio Features Hamburg Keynote at Annual Meeting – Part 2 of 2

monoclonal antibodies.pngOn March 14-15, the Massachusetts Biotechnology Council (“MassBio”) held its Annual Meeting in Cambridge, Massachusetts. The Meeting also featured a Keynote from FDA Commissioner Margaret A. Hamburg, M.D. (see related blog here). Key themes at the Meeting were the importance of the Cambridge/Boston biotechnology community for advancing new therapies and the unique resources available in the area that have made it an industry leader. Some of the Cambridge/Boston advantages discussed were the intellectual research capital (local universities such as Harvard and Massachusetts Institute of Technology), venture capital, and local biotechnology businesses, such as Biogen Idec and Genzyme, as well as other biotechnology companies that now have offices in the Cambridge/Boston area and are seeking partnerships to develop new products, such as AstraZeneca, Pfizer, Merck, Novo Nordisk, and Sanofi.

On the second day, Hamburg described here “special affection” for the Cambridge/Boston region dating back to her days at Harvard, saying that she hopes D.C. “would be as efficient and congenial as here.” Hamburg said that the Cambridge/Boston region is a life sciences enterprise fueled by top notch research and medical care with the top five NIH-funded hospitals and a “biotech supercluster second to none” with “a remarkable 500 biotech and pharma companies here, and some thirty venture capital firms.”

Hamburg described FDA as striving for true collaboration and regulatory flexibility with industry, including MassBio, and has been hearing that industry wants more clarity, certainty, transparency with decisions. Hamburg said that FDA is trying to have creative approaches–not a one size-fits-all approach. To this end, Hamburg described approaches that FDA has taken with four new products from the Massachusetts area: 1) Inclusig® for two rare forms of leukemia, 2) Juxtapid® (an orphan drug), 3) Linzess® for irritable bowl syndrome, and 4) Kalydeco® for cystic fibrosis. In addition, Hamburg highlighted new provisions in the Food and Drug Administration Safety and Innovation Act (“FDASIA”) for expedited approvals, citing 31 breakthrough therapy designation requests, of which 9 have been granted, 10 denied, 11 pending, and 1 withdrawn. To help with more companies taking advantage of this new process, FDA will be publishing a new guidance shortly, Hamburg announced.

Hamburg said that FDA has been working to put together a robust biosimilars program, which has taken “much time and development.” Hamburg confirmed that FDA has still not received a biosimilar application but as of March 14, there had been 51 requests for meetings on 12 different biological products, where FDA had held 38 initial meetings with sponsors and had received 15 INDs for biosimilar development programs.

Hamburg said that FDA is looking to advance regulatory science and announced that FDA is considering developing a new approval mechanism may be created for “special limited use.” Under current FDA requirements, once a product is approved, FDA cannot be assured its use would be limited. As a result, drug development plans need to consider the risks in broader patient populations with larger, lengthier trials. The proposed pathway would limit a drug’s use, e.g., a new antibiotic could be limited for a population of patients with a drug-resistant infection, or an obesity drug could be limited to patients with serious obesity. The new mechanism, therefore, could allow for product approvals with smaller, more targeted clinical trials, which would enable potentially-lifesaving medicines to be available sooner.

Regarding the development of regulatory science, FDA has established three Centers of Excellence in Regulatory Science at the universities in Arkansas, the Georgetown University, and the University of Maryland. These centers are conducting targeted research, strengthening education and training in regulatory science, and enhancing scientific exchanges and collaboration. FDA is also working the Reagan-Udall Foundation, a private and independent nonprofit research organization on several regulatory science projects.

Hamburg reminded the members of MassBio that these initiatives, however, come at a time of new budgetary constraint and continuing budget measures that may limit FDA’s expansion plans or immediate use of user fees. In response to questions on sequestration, Hamburg said it was “very concerning” for both FDA and NIH, and it is unclear what the long-term impact will be for constricting budgets. Hamburg said that the sequestration places an unexpected burden at critical time especially when it now looks like user fees will be part of cutting process, because they “look” like budget dollars. And while Hamburg is “guardedly optimistic” that FDA can come out of the current situation where FDA is limited to the budget from fiscal year 2012, she is worried that without more resources, FDA will not be able to work on critical programs in biomedical space, which may affect industry’s willingness to come to table for user fee discussions in 5 years.