By Jerry Avorn and Aaron S. Kesselheim
As the US National Institutes of Health faces a $300 million budget reduction, it is creating a unit to facilitate the development of new drugs. The National Center for Advancing Translational Sciences (NCATS) envisioned by NIH Director Francis Collins would redeploy resources from the agency’s existing programs to identify discoveries that could be transformed into therapeutic products.
The road that brings treatments from bench to bedside is notoriously bumpy, and recent years have seen a scarcity of transformative medications and novel drug designs. Of the 47 new products approved in 2009–2010, by our count approximately two-thirds were members of an existing therapeutic class or managed their indicated condition about as well as available treatments. We clearly need more and better innovation.
The assumption underlying NCATS is that many potential drug targets or compounds have been identified but are not being adequately exploited, so the new NIH unit would pursue leads that drug companies or investors have overlooked or have chosen not to invest in. NCATS will also facilitate the exchange of ideas about common therapeutic approaches across different conditions by bringing together similarly oriented mechanism-based researchers currently separated in the NIH’s disease-specific institutes.
These plans raise important questions. Are there really many clinically promising compounds or targets that have been discovered but are languishing, neglected, in some laboratory—or that remain unexploited even though their properties are known? This may be true for some antibiotics, since the need for their restrained use makes them potentially unprofitable products for industry to develop. In such cases, the NCATS model could fill an important niche. But for treatments for conditions such as common cancers or Alzheimer’s disease, how likely is it that a new NIH center will be able to develop candidate therapies that armies of smart investigators, drug companies and venture capitalists have all somehow overlooked? It also isn’t clear that a governmental agency will generate more creative decision-making than the time-tested approach of investigator-initiated research.
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