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Multiple initiatives are moving forward to maintain US leadership in biopharm R&D.
Policymakers are devising "pro-innovative" approaches for testing and regulating medical products amidst the clamor to curb the cost and time of drug development and thus speed more new products to patients. A lead strategy for the White House, Congress, and the biopharmaceutical industry is to portray the development of new biotech therapies as key to driving US economic growth and creating high-wage, high-value jobs, desired by every state and nation.
In pressing for fast approval of legislation to reauthorize user fees for FDA, House Republicans highlighted how improvements in the biopharmaceutical regulatory process will promote American innovation and jobs growth. Legislators on all sides agreed that more predictability, consistency, and transparency in drug oversight help stem the shift in jobs overseas and ensure patient access to innovative therapies.
Biotech manufacturers also stand to gain from an effort in Congress to revive tax credits and grants for emerging companies. A small provision in the 2010 healthcare reform legislation provided $1 billion to support biotech firms investing in R&D, and the Biotechnology Industry Organization is pressing for a second round. Sen. Robert Menendez (D-NJ) recently introduced a bill (S.3232) to provide $1 billion to extend the "therapeutic discovery project program" for two years, but tight federal budgets may make it much harder to find the money this time around.
Legislative efforts to promote biopharmaceutical innovation should gain support from the national "Bioeconomy Blueprint," issued in April by the White House Office of Science and Technology Policy (OSTP) to promote economic growth while improving public health. The report proposes multiple strategies for encouraging innovation in the biological sciences, from genetically engineered crops and bio-based fuels to next-generation biomanufacturing methods and regulatory reform. The report states that these strategies would foster a "robust" biomedical research enterprise able to speed drug development and harness biology as a manufacturing platform for rapid production of new high-value materials, medicines, devices, and fuels. The administration says it's serious about reducing regulatory barriers to increase the speed and predictability of processes for bringing new biotech products to market.
The blueprint specifically backs research to advance pluripotent cell technology, to validate promising drug targets, and to establish standards that can improve protein-manufacturing processes. Multiple federal agencies are involved, such as the National Institute for Standards and Technology (NIST), which is working with FDA on measurements that can more accurately characterize key safety and efficacy attributes of proteins. The Department of Defense is developing more flexible and rapid biotech manufacturing platforms, and the National Science Foundation is investing in biomanufacturing approaches for advanced tissue engineering and flexible bioelectronics.
In unveiling the blueprint, the White House highlighted potential gains from expanded use of FDA's vast repository of clinical data, including safety, efficacy, and performance information from clinical trials and a growing volume of post-market safety data. Analyzing information could "revolutionize the development of new patient treatments" and address fundamental questions relevant to development of personalized medicine. Biomanufacturing will be enhanced by FDA efforts to rebuild its information technology and data analytic capabilities and to establish "science enclaves" where multi-disciplinary teams can analyze large sets of data extracted from FDA resources to speed the development of new therapies at reduced cost. Although many of the "new" initiatives described in the report are well underway, they stand to gain from the administration's explicit statement in support of the long-term economic importance of biotech research.
The blueprint calls for more public-private collaboration to advance biotech discovery, particularly in the pre-competitive arena that may involve sharing of information and resources. Such collaboration, the blueprint notes, already is advancing clinical trial design and biomarker discovery. A prime example is the National Institutes of Health (NIH) initiative to translate biomedical discoveries into effective therapies more efficiently, specifically NIH Director Francis Collins' campaign to find new uses for existing drug compounds.
Collins rolled out the first project in this area in May 2012, announcing that the NIH National Center for Advancing Translational Sciences (NCATS) is linking up with Pfizer, AstraZeneca, and Eli Lilly to support independent studies on approximately 20 drugs that the companies previously tested but never brought to market. NIH will provide $20 million in grants in 2013 to fund promising research proposals and provide templates for agreements on dealing with intellectual property and other rights.
Under the program, which was expanded in June to include Abbott, Bristol-Myers Squibb, GlaxoSmithKline, Janssen, and Sanofi, manufacturers will give researchers the compounds and relevant data, while retaining rights to their products. Independent researchers will be able to publish study results and negotiate licenses on new discoveries. The research agreement templates are key to speeding up negotiations among all parties and making the program work. NIH plans to publish a list of compounds available for research in a few months, with an eye on awarding initial grants next spring. Manufacturers see the NIH program as a way to extend the kinds of arrangements they already have with individual research institutes, including AstraZeneca's partnership with the UK's Medical Research Program.
Despite these positive developments, manufacturers believe that more US support for biopharmaceutical research and product development is needed to prevent innovative firms from looking overseas for financial and regulatory support. An analysis of R&D investments and policies at 18 industrial and emerging countries by the Battelle Technology Partnership Practice, released in May, for the Pharmaceutical Research and Manufacturers of American (PhRMA) warns that the US may lose its pre-eminent position in biotech discovery and product development without more pro-innovation policies. Although pharmaceutical output continues to expand in the US and the EU, the fastest growth now is in China, Russia, Israel, and Singapore, which have established innovation strategies targeted to the biopharmaceutical sector as part of national agendas to establish knowledge-based economies.
The US biopharmaceutical sector indirectly supports four million US jobs and pays hefty state and federal taxes, according to Battelle. Yet, challenges in managing clinical trials, dealing with complex regulatory processes, and obtaining adequate coverage and payment for new treatments makes it hard for US firms to resist foreign overtures accompanied by tax benefits, research support, and favorable regulatory policies. Even the White House blueprint acknowledges that new initiatives are necessary to counter a 30-year decline in pharmaceutical industry productivity, as measured by new molecular entities per dollar spent on R&D (adjusted to reflect inflation). While R&D costs have increased 50-fold during this time period, drug candidates and new drug applications have been flat.
The PhRMA report points out that the R&D tax credit has never become permanent in the US, and corporate taxes remain high, while other countries are offering a variety of tax relief for start-ups and their investors. Federal funding of biomedical research has been static for several years and faces cutbacks, compared to other nations that are funding more biotech research, nurturing young scientists and engineers, and offering incentives to attract world-class researchers to their institutions. The US still leads the world in many areas that are crucial to R&D, but the challenges facing biotech manufacturers require a more sustained response.
Jill Wechsler is BioPharm International's Washington editor, Chevy Chase, MD, 301.656.4634, firstname.lastname@example.org.