Filling the Pharma Pipeline - FDA, NIH, and industry seek new strategies to spur drug development and promote access to therapies. - BioPharm International


Filling the Pharma Pipeline
FDA, NIH, and industry seek new strategies to spur drug development and promote access to therapies.

BioPharm International
Volume 24, Issue 5, pp. 14-18


The changing biomedical research landscape and cutbacks in industry R&D programs are encouraging more public support for pharmaceutical research around the world. The Innovative Medicines Initiative in Europe is building a schizophrenia database of industry-sponsored clinical trials to better identify signals of patient response to test drugs. The United Kingdom's Medical Research Council has established the Developmental Pathway Funding Scheme to support basic research on drugs and medical devices, and the Wellcome Trust's Seeding Drug Discovery initiative is funding efforts to take drug candidates through early clinical trials. Both US and EU scientists are wary of being left behind by soaring Chinese investment in R&D.

At home, NIH Director Francis Collins launched a high-profile campaign to promote translational medicine as a way to spur development of new medical treatments that can benefit patients. In December, an NIH advisory committee recommended establishing an NIH National Center for Advancing Translational Sciences (NCATS), a move engineered by Collins to bring together several NIH programs that provide resources for translating basic discoveries into new medicines and diagnostics. These include a program that supports development of therapies for rare and neglected diseases, along with NIH's national network of research sites at academic medical centers supported by Clinical and Translational Science Awards.

As the former director of NIH's Human Genome Project, Collins is optimistic that new genetic discoveries can chart pathways for discovering new medical treatments, and that the emergence of more well-validated genes will be useful in "identifying drug targets in unprecedented numbers," he said in an interview. The scientific enterprise is yielding up a lot of new ideas about therapeutics, he observed, yet "traditional private sector efforts to capitalize on that are taking a hammering." NCATS aims to bolster the funding of research projects at a time when biotech and pharma companies face serious financial challenges. Along these lines, the initiative also will encourage more collaboration between academic researchers and biopharmaceutical companies and to strengthen ties with FDA to ensure that NIH-sponsored studies provide the data needed to support registration of new products.

The project envisioned by Collins will help re-engineer the drug-development pipeline by investing in new assays that can screen thousands of molecules to find ones that will hit defined disease targets. Another objective is to improve assessment of toxicity, which may involve shifting from the use of animal testing to identify potential problems.

As part of the process, Collins' translational science campaign aims to convince Congress and the American public that the federal investment in biomedical research can pay off in terms of new, life-saving therapies. The Obama administration has proposed a very slight increase in the NIH budget for fiscal year 2012, which would just barely maintain current funding levels. Even during the Republican budget cutting campaign of the mid-1990s, NIH retained several strong GOP advocates on Capitol Hill and largely escaped the chopping block; that kind of support seems to be lacking among current Republican leaders.

Collins believes that today there is greater private-sector interest in NIH-funded preclinical and clinical testing, as well as in compound rescuing or "repurposing." Pharmaceutical companies have long lists of compounds that have been abandoned along the way, maybe because a business plan changed or the money ran out or clinical trials failed to show efficacy, Collins notes. "We have been talking with leaders in the pharmaceutical industry about an opportunity to open the freezers and make such compounds available, with appropriate intellectual property protection for them," Collins explains. "This isn't a giveaway, but could be a win-win if such a compound were found to be active for a different application than originally considered." NIH will not move into drug development per se, Collins emphasizes, but will hand promising compounds off to private sector sponsors.

Jill Wechsler is BioPharm International's Washington editor, Chevy Chase, MD, 301.656.4634,

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