The National Institutes of Health, 10 major pharmaceutical firms, and several nonprofits are joining forces to reduce the time and costs of developing new drugs for complex diseases.
The $230 million, five-year partnership will initially focus on identifying drug targets for Alzheimer’s disease, type 2 diabetes, rheumatoid arthritis, and lupus. NIH hopes eventually to expand the effort to other diseases. NIH will provide about half the funding, with industry contributing the rest.
Bringing a new drug to market takes an average of $1 billion and 14 years, and only about 1% of drug candidates make it through the process, NIH Director Francis S. Collins noted at a Feb. 4 news conference announcing the partnership. Drugs that target the wrong molecule are ineffective and costly, “both in terms of research dollars and patient lives,” Collins said.
Government, companies, and nonprofits will collaborate
◾ Biogen Idec
◾ Bristol-Myers Squibb
◾ Johnson & Johnson
◾ Eli Lilly & Co.
◾ Merck & Co.
◾ Alzheimer’s Association
◾ American Diabetes Association
◾ Lupus Foundation of America
◾ Foundation for the NIH
◾ Geoffrey Beene Foundation
◾ Rheumatology Research Foundation
Under the new collaboration, called the Accelerating Medicines Partnership, researchers from academia and the private sector will sift through a deluge of genomics, proteomics, imaging, and other biomedical data. The goal is to collectively identify which biological targets are most likely to respond to new therapies.
All data and analyses generated by the partnership will be considered precompetitive and will be made freely and swiftly available, Collins said. After the initial targets are identified, the pharmaceutical industry can then compete to develop therapeutic molecules.
The announcement culminated two years of negotiations between NIH, pharma firms, and other stakeholders. The rationale was the same from day one—lower the rate of failure in drug development by gathering better information about drug targets in humans, said Lon R. Cardon, senior vice president of alternative discovery and development at GlaxoSmithKline. But the challenge was finding the right focus for the program, he said.
The initial therapeutic areas were picked on the basis of patient need. Each of the projects will take a different approach. For Alzheimer’s disease, researchers will look for biomarkers, such as molecules in blood or spinal fluid, which can be used to assess disease progression. The type 2 diabetes effort will focus primarily on building a Web-based portal that brings together genetic and clinical data. And for rheumatoid arthritis and lupus, researchers will examine single cells from joint tissues to better understand the molecular basis of the diseases.
Industry observers agree that the willingness of pharma firms to join forces with a third party like NIH at the helm is a positive development. Multiple pharma companies often work in parallel and in secrecy on the same drug target, a fractured effort that can result in all failing.
That redundancy manifests most acutely for Alzheimer’s disease, which will receive the biggest chunk of the partnership’s funding. The pharma industry has sunk billions of dollars into testing more than 400 compounds with “nothing to show for it,” said Bernard H. Munos, founder of the InnoThink Center for Research in Biomedical Innovation.
“If we knew what causes the disease, we could probably design an effective treatment within months and get it to patients within a few years,” Munos said. “Everyone would benefit. It’s a case of succeeding collectively rather than failing separately.”
GSK abandoned Alzheimer’s research in 2010, but it was drawn into the partnership by the possibility of capitalizing on companies’ various strengths to finally make progress in understanding the disease, Cardon said. “That’s what it’s going to take for some of these diseases—it’s going to have to be a team sport.”
Although encouraged by the pharma industry’s step toward more open, early collaboration, observers remain cautious about the prospects for the new partnership. Concerns include how to guarantee that data are shared in a timely and complete way and whether the grant review process will be nimble enough to shift with market forces, said David A. Steinberg, partner at the life sciences technology development firm PureTech Ventures.
“There are a lot of ways this could stray,” he said, “but overall I am a huge fan of the idea.”