The Republican-controlled House of Representatives is slashing funding for federal programs, and on the chopping block may be a new initiative at the National Institutes of Health (NIH) that is aimed at speeding up the time it takes to develop new drugs for diseases.
The initiative, called the
But Congressional approval for that funding is in jeopardy. Although the budget and appropriations process for 2012 is just getting underway, the House is in no mood to increase funding for NIH. This week the House considered eliminating $1.5 billion for NIH’s funding for this fiscal year but restored funding by voice vote at the last minute.
It is in this severe cost-cutting environment that NIH seeks a $1 billion increase to build a new translational science center. The prospect of losing or delaying a new center is disappointing to many scientists, patient advocates and legislators who have pushed NIH to put more emphasis on translating basic research into drug development. Patients and their families who search for treatments for such maladies as autism and rare cancers are frustrated by the pharmaceutical industry’s slow pace of drug development. Pharmaceutical companies have been introducing fewer new drugs to the market as the costs for development and regulatory approval have soared in recent years. They also rarely, if ever, invest in research and development for the treatment of rare diseases and disorders, because the market for such drugs would be too small to recover costs and generate profits.
NIH has historically focused on upstream, basic research to understand the underlying genetic and biological mechanisms of diseases, while private pharmaceutical companies have researched and developed drugs that can be used to treat diseases. In recent years NIH has directed more of its funding to translational research, particularly in its larger institutes such as the National Cancer Institute. After the mapping of the human genome, it became apparent that there was a greater need to translate the basic research of genetics into treatments, but institutional and structural constraints within NIH inhibited change. NIH has 27 different institutes and centers, and fourth- fifths of its budget goes to funding research grants to more than 3,000 research institutions. In addition, ideas for research are mostly generated by scientists who apply for competitive grants, which are then peer-reviewed. This model works well for directing basic scientific research, but often more direction is needed to steer the findings of that research into the development of new drugs and treatments.
During the administration of George W. Bush, former NIH Director Elias Zerhouni began working with Congress to expand the Office of the Director to include what is known as The Common Fund. The fund gave the Director of NIH more authority to identify research priorities based on public health needs and to consolidate some resources for translational research. For example, the Common Fund was used in part to establish a molecular libraries program to give researchers access to large-scale drug screening capabilities similar to those found at pharmaceutical companies. Since its formation, researchers have identified more than 100 new drug compounds.
One of the champions of translational research was former Senator Arlen Specter (D-PA), and he pushed NIH to make further changes. A cancer survivor, he used his powerful position as chairman of the Senate subcommittee that funded NIH to include in the 2010 heath care reform bill a new entity at NIH, the Cures Acceleration Network (CAN). Although no funding was provided for CAN in the bill, his message to NIH to focus on translational research fell on receptive ears. NIH’s new director, Dr. Francis Collins was the former director of the Human Genome Project, and he understood the importance of accelerating drug development. “I think we’re at a critical juncture,” he said in December, 2010. “It is clear that scientific advances, many supported by NIH, are providing new insights into the molecular causes of disease at a dizzying rate.” However, he agreed that further structural and organizational changes were needed at NIH “to ensure that we capitalize on these new opportunities for science.”
Collins wasted no time assembling the scientific panels necessary to consider establishing a new translational science center, and he got the go-ahead last December. He plans to bring together three existing programs at NIH, including the National Human Genome Research Institute, to create a core structure for the center, and the center’s budget initially will be imported from those programs with the hope of eventually getting additional funding from Congress.
The proposed center is not without its critics. Some have characterized it as a government-run drug company that will have an unfair competitive advantage over pharmaceutical companies. Collins emphatically states that the center will not be a drug company. He deftly describes the center as a “facilitator of translational research” that will concentrate on areas that attract little commercial interest. “It would develop and offer innovative services and expertise in moving promising products through the development pipeline, as well as develop novel approaches to therapeutics development, stimulate new avenues for basic scientific discovery and complement the strengths of existing NIH research activities.” He likened the center to the way in which the Genome Institute has provided a hub for genomics at NIH, even though all of the institutes are invested in gene research.
From a research scientist’s point of view, this is exactly the right time to put more money into translational research. It would be short-sighted, they say, to cut off funding now when scientists are at the threshold of discovering new drugs that can transform modern medicine and lead to economic growth. Most members of Congress, republicans included, see the important role that NIH plays in fostering scientific discoveries. Until this year, NIH enjoyed strong bipartisan support. In fact, NIH was one of the biggest beneficiaries of funding in last year’s stimulus bill.
But the political climate has shifted. The new Republican-controlled House has succeeded in changing the debate and the agenda, and now every federal agency, including NIH, is on the defensive. The question is not if NIH funding will increase, but whether it can hold on to the funding level it had last year.