The President’s National Bioeconomy Blueprint outlines how Washington intends to apply biological innovations toward national challenges that include health, food, energy, and the environment. [© Vacclav - Fotolia.com]
President Barack Obama’s administration rolled out its National Bioeconomy Blueprint last week, on April 26. It details measures by which Washington intends to apply biological innovations toward national challenges that include health, food, energy, and the environment.
At the top of the Blueprint’s five priorities is supporting “R&D investments that will provide the foundation for the future U.S. bioeconomy.” Also on the list: increasing the focus on translational and regulatory sciences, reforming regulations, updating training programs, and identifying and supporting opportunities for public-private partnerships.
While those are all helpful components, the report should have also identified one or more “grand challenges”—a question asked in solicitation of public comment on the draft Blueprint last year. For example, developing tools to study the human immune system was the challenge suggested by Aprile L. Pilon, Ph.D., president and CEO of Clarassance. The firm is developing a recombinant human CC10 protein for prevention of neonatal bronchopulmonary dysplasia.
The Blueprint should have also outlined initiatives to assist small- and medium-sized businesses in maximizing their own contribution to the nation’s bioeconomy. In her comment to OSTP and an interview with GEN, for example, Dr. Pilon suggested increased funding for FDA’s Orphan Products Grant Program, which has stayed flat at $14 million for five years despite a growing number of funding applications. She also suggested more money for SBIR. The program’s recent reauthorization phases in 1% more funding over six years, but it also lets agencies use 3% toward administration, which takes away from grant money.
Bruce W. Stillman, Ph.D., president of Cold Spring Harbor Laboratory, offered grand-challenge suggestions that included launching a national Cancer Therapeutics Initiative, making a major commitment to fundamental neuroscience, investing in basic plant science to address energy and food needs, and continuing to invest in the training of scientists.
Nevertheless, Dr. Stillman told GEN, the report offered several valuable recommendations for building biotech-based economic activity. “A key question is how this report will be implemented,” Dr. Stillman pointed out. “It will take a continued and concerted national commitment to keep the U.S. at the forefront of bioscience. Only with such a commitment can we achieve the recommendations described in the report.”
Augmenting Basic Research
The report envisions cross-agency and cross-disciplinary R&D efforts that expand and develop “essential” technologies, mentioning by name “synthetic biology, biology-related information technologies, proteomics, and others.” Also envisioned are new funding “mechanisms” or programs to “support creative, high-risk/high-reward research” that is too risky for existing government programs, let alone private investors.
One such “mechanism” is conducting prize competitions. The Blueprint also highlighted NSF’s Creative Research Awards for Transformative Interdisciplinary Ventures (CREATIV) pilot grant program. It was designed to fund “bold” interdisciplinary projects in all science, engineering, and education research areas supported by the agency.
CREATIV requires only an internal merit review, which as the report said lowers the risk of external panels favoring tried-and-true projects for funding. But that system raises some risk of an insular bureaucracy tilted toward too-risky research unable to withstand scrutiny from outside experts.
Moving Findings out of the Lab
In calling for increased focus on translational and regulatory sciences, the Blueprint largely promotes existing administration-created programs. Last year, for example, NIH established the National Center for Advancing Translational Sciences (NCATS), whose priorities for FY 2013 include developing an improved chip for drug safety assessments, rescuing unapproved drugs and repurposing approved drugs, promoting rare disease R&D, and launching the second phase of the Clinical and Translational Science Awards.
Translating those priorities into policy will take money. NCATS is the brainchild of NIH director Francis S. Collins, M.D., Ph.D., who has proposed a $64.32 million increase for the center in FY ’13 to $639.033 million, paced by a fivefold increase (to $49.624 million) in the Cures Acceleration Network (CAN). While Congressional support for NIH should result in more funding, how much remains uncertain.
The Bioeconomy Blueprint also recommends regulatory reforms that reduce barriers, increase speed and predictability of regulatory processes, reduce costs, and encourage technology development. The report noted that NIH and FDA are spending $9 million over three years on projects that could reshape biopharma regulations. Projects include advancing nanotechnology, developing microdevices for testing safety and efficacy, and adaptively designing clinical trials. The effort is one of many launched by FDA following criticism by industry that the administration’s approach to biopharma regulation has scared investors away with slow, heavy-handed, and inconsistent rulemaking.
Partnering for Solutions
The Blueprint also identified ongoing federal public-private partnerships toward solving key bioeconomy challenges. NIH is organizing a joint NIH-Industry Target Validation Consortium comprising experts from the agency, industry, and academia. FDA and NIST are joining biopharma to develop measurements and standards to support development, manufacturing, and regulatory approval of new protein therapeutics.
Other public-private efforts highlighted by the report include whole-genome sequencing, drug rescue/repurposing, and Alzheimer disease research. NIH, FDA, and other federal agencies joined more than 20 pharma companies and others from industry to create the Alzheimer’s Disease Neuroimaging Initiative. President Obama proposed $130 million for Alzheimer research this fiscal year and next, but the outlook for next year’s $80 million is uncertain.
Public-private efforts will also be needed beyond advancing science, toward nurturing companies as well. “It’s just not possible in the current economic climate to see a start-up company go all the way to become a self-sustaining company without either strategic partnerships with established companies or by being acquired by them,” Stephen S. Tang, Ph.D., president and CEO of the University City Science Center in Philadelphia, told GEN.
In written comments, Dr. Tang proposed a public-private effort to commercialize early-stage research at academic laboratories, federal laboratories, and private-sector companies. Such an effort, he said, could be modeled on the Science Center’s successful QED Proof-of-Concept Program, which has awarded or offered funding to 12 projects by academic researchers throughout Greater Philadelphia who are developing early-stage life sci technologies with high commercial potential. Five of those projects have resulted in private-sector licensing or optioning.
Another Science Center concept, Phase 1 Ventures, joins SBIR/STTR-eligible businesses with one or more nonprofit sources of high-potential federal laboratory or university technologies. Joining them would be strategic partners that include economic development organizations, investors, entrepreneurs, and corporate giants. The Science Center board has approved launching Phase 1 and is seeking operating partners.
Providing a Starting Point
Overall, Dr. Tang concluded, the Blueprint was a good start. “It’s always good when the White House sets policy that recognizes the impact of industries like the life sciences. We’re not going to legislate our way into healthcare reform, we’re going to innovate our way.”
Other Blueprint recommendations fall in the category of commonsense ideas that, according to the report, the administration has begun to implement: more technology transfer, more entrepreneurship by students and their researcher-professors, and new regulations that balance safety with economic growth.
While the report addresses job training, it largely trumpeted present initiatives while leaving readers hanging about a potentially valuable effort. Sometime this year, the NIH’s Biomedical Workforce Task Force will recommend actions “to support a sustainable biomedical training infrastructure that provides incentives for updated training programs to prepare trainees for academic and industry careers.” That’s a key component of a true Bioeconomy Blueprint and should have been included in the report.
That said, the Blueprint still offers many other solid recommendations for how Washington can best catalyze additional biopharma activity. Its challenges, however, are two-fold. As Dr. Stillman said, implementation is one. And as NCATS shows, implementation cannot take place without funding and policy approvals from Congress. Those approvals will be difficult to come by this election year and what is still shaping up to be a period of tight budgets afterward.