It would seem like there’s never been a better time for drug development. Recent advances in genomics, proteomics, imaging, and other technologies have led to the discovery of more than a thousand risk factors for common diseases—biological changes that ought to hold promise as targets for drugs.
But this deluge of new opportunities has to be put in context: drug development is a terribly difficult business. To the dismay of researchers, drug companies, and patients alike, the vast majority of drugs entering the development pipeline fall by the wayside. The most distressing failures occur when a drug is found to be ineffective in the later stages of development—in Phase II or Phase III clinical studies—after years of work and millions of dollars have already been spent . Why is this happening? One major reason is that we’re not selecting the right biological changes to target from the start.
To put it more bluntly: if a drug is aimed at the wrong target, it probably won’t work against the disease it was intended to treat. Such misplaced bets waste untold time and precious resources. All who care about therapeutics agree, we need new strategies to avoid such failures. Patients and their loved ones can’t wait.
So, at a news conference today at the National Press Club in Washington, D.C., I was thrilled to help launch the Accelerating Medicines Partnership (AMP). This unprecedented public-private effort will strive to identify better targets so we can develop better drugs at a faster pace. As you can see, the list of AMP participants is most impressive. Besides NIH and Foundation for the NIH, the partners include 10 biopharmaceutical firms and a number of non-profits, including patient advocacy groups.
AMP has been more than two years in the making, with hundreds of hours of intense discussions among creative minds who truly rolled up their sleeves and left their affiliations at the door. And here’s one important lesson we learned: you can’t change the world—or in this case, drug development—overnight.
Consequently, AMP will begin by investing $230 million in pilot projects in three disease areas: Alzheimer’s disease, type 2 diabetes, and the autoimmune disorders, lupus and rheumatoid arthritis. These three-to-five year pilot projects will set the stage for broadening AMP to other diseases and conditions.
Let me share an example of how this approach can lead to success: the recent identification of a drug target called PCSK9. Researchers were tuned in to this target by the discovery that people with a rare gene variant that greatly reduces production of the PCSK9 protein have very low levels of cholesterol, along with a dramatically decreased risk of heart disease. After some additional studies to validate that PCSK9 is a solid target, a half-dozen pharmaceutical firms are now racing to develop drugs that lower cholesterol by blocking this protein .
I think there are a whole lot more PCSK9’s out there waiting to be discovered . We just need to assemble the right teams to help us find the right targets—and AMP has the right stuff to do that.
 Trial watch: phase II and phase III attrition rates 2011-2012. Arrowsmith J, Miller P. Nat Rev Drug Discov. 2013 August.
 Genetics: a gene of rare effect. Hall SS. Nature. 2013 Apr 11.
 Validating therapeutic targets through human genetics. Plenge RM, Scolnick EM, Altshuler D. Nat Rev Drug Discov. 2013 August.
AMP News Release (NIH)
President’s Statement on AMP (The White House)