by Susan Semeleer, Associate Director of Communications, FasterCures
FasterCures recently held a briefing in collaboration with the Parkinson’s Action Network (PAN) discussing the translation gap between basic and clinical research, known colloquially as the “valley of death” – where promising ideas for treatments for deadly disease are often slowed and sometimes dropped before they can be fully explored. After reviewing the drug-development pipeline – from the most basic research to large-scale Phase III clinical trials conducted by major pharmaceutical companies – PAN concluded that a critical step toward bridging the valley of death is a rethinking of NIH 's approach to supporting translational research. PAN CEO Amy Comstock Rick told forum attendees that under the current system, too many promising ideas become mired in institutional inertia or lost in information silos. They don’t move or, if they do move, researchers lack the skills and infrastructure to follow through.
“More money is not necessarily the solution,” said Rick. Instead, what is needed is a fundamental soup-to-nuts change in the way NIH conducts its business. It is change that Rick insists must be implemented from within; Congress must mandate change, then leave it to NIH to comply. “If Congress tries to fix NIH,” said Rick, “it’s not going to be done right.” But it must be done; the chasm between concept and cure is growing ever wider.
In a paper "Entrepreneurs For Cures: The Critical Need for Innovative Approaches to Disease Research" released last Fall, FasterCures noted that "for all the money flowing through the [health] system, there is remarkably little emphasis on specific goals or milestones to cure disease or achieve specific clinical results. As a result, emphasis on speed or direct responsiveness to health needs is spotty, and the time from initial discovery to dissemination and commercialization can sometimes be measured in decades—an outcome that is simply unacceptable to the citizens who fund this research and expect to benefit from it."
Rick said that there is no treatment for Parkinson’s -- and nothing in the pipeline -- that slows progression of the disease. Instead, the best that can be done for Parkinson’s patients are treatments that manage or temporarily mask symptoms. It’s a common story that so many patients hear, every day, and it is no less unbearable in its repeated telling: This is all we can do for you.
That’s not good enough for a desperate patient whose shoulder has been clamped by the cruel hand of illness. What PAN is seeking to do is make it not good enough for our medical research community, either.
PAN’s proposal calls for patient advocacy groups, in collaboration with the drug development and research communities, to promote legislative change that would encourage NIH to fully support translational research. PAN Deputy CEO Mary McGuire Richards echoed Rick’s contention that it should fall to Congress to establish goals for reforming NIH, and mandate its reform, but that the implementation of those reforms must originate within the halls of NIH.
Panel moderator and FasterCures COO Margaret Anderson raised the question of possible obstacles on the road to NIH reform. Rick noted a resistance to change within the research community, particularly among basic researchers.
While Rick was careful to emphasize the importance of basic research, she pointed out that a vast difference exists between basic research and drug development. The two must be managed differently and, under the current NIH business model, no culture exists for managing translational research. She used as an example the difference between an elementary school and high school principal: their jobs are equally important, equally challenging, but require wholly different skill sets. The same, Rick said, holds true for basic and translational research. When your central organizing principle is studying biology, as is the aim with basic research, the institutional mechanisms that govern the process are completely different than with research that is organized around the principle of curing disease. Both are vital pieces of the cure puzzle, but translational research is a square peg that can’t be pounded into a round hole; what PAN is calling for is a long-overdue creation of a square -shaped niche within the research community.
As FasterCures noted in its report "Entrepreneurs For Cures: The Critical Need for Innovative Approaches to Disease Research", "the true measure of success should be in lives saved and suffering diminished – not the number of grants awarded, publications presented, or laboratory space acquired."