FDA Commissioner Margaret Hamburg cited stagnated drug development as the reason for FDA's new biomedical innovation initiative, but seasoned FDA watchers may find many of its elements familiar. "The number of new products in the development pipeline is not where we would want it to be, and is not commensurate with medical and public health needs," Hamburg said in a conference call with reporters Oct. 5. "Last year saw the lowest number of new drug applications in several decades. We saw the highest level of investment ever, $100 billion, but the return on investment is not what's expected. Timelines are long, costs are high, and the rates of failure are distressingly high." The report, titled “Driving Biomedical Innovation,” notes that “there is no single cause or party responsible for this stagnation in the development of novel medical products,” and cites a number of factors that affect multiple stages of the development process, “such as an uncertain and challenging economic and global marketplace, increasingly complex science and products, and the imperative to ensure the safety and efficacy of innovative medical products.” Hamburg discussed FDA's responsibility to help deliver new, groundbreaking medical products during a speech in April, where she linked two key initiatives that have been in play during her time at the helm: strengthening regulatory science and increasing transparency of agency actions (Also see "Disclosing Drug Development Failures Would Aid Regulatory Science, FDA’s Hamburg Says" - Pink Sheet, 11 April, 2011.). At that time, she said FDA was looking at how it could mine its databases of submitted applications and approved products to help address scientific uncertainties in the development process. In announcing the Biomedical Innovation Initiative, Hamburg said that in addition to this internal review, she has been meeting with stakeholders to identify common themes where FDA can take action. The initiative will get external support; the Reagan-Udall Foundation, a public-private partnership established in 2008 under former FDA Commissioner and CMS Administrator Mark McClellan, will collaborate on several of the projects. With the biomedical innovation drive, Hamburg appears to be adding another high-profile initiative, but many of the elements derive from or were foreshadowed in previous agency projects – including the similarly ambitious Critical Path Initiative, the Advancing Regulatory Science program introduced in a white paper in 2010, and this year's iteration of FDA's five-year plan. The chart below details the planned steps to improve innovation and puts them in the context of previous agency activity.
FDA biomedical innovation initiative	Details
Expedited drug development pathway	The innovation initiative is introducing a new pathway to speed development for drugs to treat serious diseases with unmet need where dramatic responses are seen in early trials, often because of molecular targeting. FDA will host a series of meetings with academic investigators, patient groups, drug developers, statistical and methodological experts and ethicists, with an eye to developing a draft guidance on the expedited drug development pathway. Two additional draft guidances will address strategies to enrich patient recruitment in clinical trials for targeted therapies and how to use pathogenic complete response (where no disease remains) as a surrogate endpoint for accelerated approval in studies of drugs for primary high-risk breast cancer. "We need to deepen our understanding of subpopulations in terms of effectiveness and adverse events," Hamburg said. FDA's Vicki Seifert-Margolis said the focus on patient selection strategies is key, and will help "usher in an age of using diagnostic tools to guide selection of patients in clinical trials."
Small business outreach	Small Business Liaisons will assist in interactions between FDA and small businesses and will train and advise agency staff on how to work with such companies. The SBL program will run "informational seminars and outreach" for small businesses and the agency will offer fellowships to young entrepreneurs. The agency will partner on these efforts with the Small Business Administration, which will provide in-kind aid, and will obtain some funding from the Reagan-Udall Foundation. FDA has a history of working to help small businesses, especially in the area of user fees (Also see "PDUFA V: FDA Relaxes Pre-Submission Meeting Requirement" - Pink Sheet, 28 March, 2011.). New steps, including a program to match entrepreneurs with small companies, were unveiled during a speech by the commissioner at the City Club of Cleveland in July, where Hamburg hinted at the larger innovation program to come (Also see "FDA's "Entrepreneurs-In-Residence" Program Aims To Help Small Companies" - Pink Sheet, 1 August, 2011.). The agency also intends to establish an exchange program for SBA and FDA employees, for cross-educational purposes.
Supporting personalized medicine	Hamburg's concern with improving personalized medicine goes back to the beginning of her tenure at FDA (Also see "Genomics Revolution Meets Regulatory Evolution: Why Personalized Medicine Is Taking So Long" - Pink Sheet, 26 October, 2009.). Regulatory science is tagged in the report as having “an important part in addressing the challenges presented by personalized medicine." That entails not only guidance efforts, but also organizational and infrastructure elements to address the challenges created by the need for multiple centers to be involved in a product review. Part of the leadership plan comes through the creation of the new Deputy Commissioner for Medical Products; the recently appointed Stephen Spielberg – an expert on personalized medicine – is expected to help with this part of the initiative (Also see "Spielberg To Begin Work At FDA In Late September" - Pink Sheet, 18 August, 2011.). The report refers to the drug-diagnostic co-development guidance, released at long last in July, and outlines continuing work on that topic (Also see "FDA Releases Companion Dx Guidance Broad In Scope, Limited In Detail" - Pink Sheet, 18 July, 2011.). As a follow-on, the agency is working on a guidance on strategies for clinical trial design and regulatory considerations for co-development with a companion diagnostic, including recommendations on strategic use of biomarkers for patient selection and screening, as well as trial designs that allow for ethical patient selection. “FDA is also producing an internal plan for how it will review applications using co-development strategies … to accompany both guidances and ensure the agency meets the special needs of these types of products in a timely way,” the report states.
Helping industry with data mining	FDA will help make de-identified clinical data from its own databases available to industry for data mining through "the FDA scientific computing model, [which] provides an environment where communities of scientists known as enclaves can come together to analyze large, integrated data sets and address important questions confronting clinical medicine," the report says. To make this possible, FDA will continue to overhaul its aging IT infrastructure, an effort that has been under way for years (Also see "FDA Needs Information Technology Strategic Plan, GAO Recommends" - Pink Sheet, 15 June, 2009.). Infrastructure updates will include a clinical data repository and tools to help with enabling analysis of data across multiple studies. FDA has also launched the Partnerships in Comparative Effectiveness Science (PACES) program to support patient-centered outcome research. For device development, the agency is developing a virtual patient.
Training innovators	A new Future Innovators Program will combine regulatory science and policy training, in partnership with the Reagan-Udall Foundation. This will build on the existing FDA Commissioner's Fellowship program, a two-year course of on-the-job training for early- and mid-career scientists and health care professionals that began in October 2008 ("FDA Fellowship Program," "The Gray Sheet," June 21, 2008). The new program will see the agency hiring candidates "who show outstanding promise in their fields" for short-term positions within FDA.
Improving FDA regulations	FDA is working to identify and remove burdensome, unclear, obsolete, ineffective or inefficient regulations and revise its rules to spur innovation and improve access to care, in response to President Obama's Executive Order 13563 on overhauling government regulations. This dovetails nicely with a theme Hamburg has stressed since taking over the FDA commissioner's post in 2009. "Regulatory science is that final bridge that enables us to move from that good idea that might work and even final demonstration in patients to actually making sure it’s a real product, a stable product, and a product that’s safe and efficacious in the marketplace," she said during the Oct. 5 media call. The Advancing Regulatory Science plans were unveiled in 2010 (Also see "FDA Outlines Priorities For Next Five Years In Strategic Report" - Pink Sheet, 4 October, 2010.).

FDA Biomedical Innovation Initiative Draws On Familiar Themes

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