The National Institutes of Health (NIH) today announced a new plan for boosting drug development: It has reached a deal with three major pharmaceutical companies to share abandoned experimental drugs with academic researchers so they can look for new uses. NIH is putting up $20 million for grants to study the drugs.
"The goal is simple: to see whether we can teach old drugs new tricks," said Health and Human Services Secretary Kathleen Sebelius at a press conference today that included officials from Pfizer, AstraZeneca, and Eli Lilly. These companies will give researchers access to two dozen compounds that passed through safety studies but didn't make it beyond mid-stage clinical trials. They shelved the drugs either because they didn't work well enough on the disease for which they were developed or because a business decision sidelined them.
Often tens of millions of dollars and years of research have gone into these compounds, so they are already well along the drug development pipeline. The government program will allow academics to "crowdsource" ways to use them, said NIH Director Francis Collins. The idea of refurbishing compounds is not new, he and others noted: the AIDS drug AZT, for example, started out as an unsuccessful cancer treatment.
NIH first began talking with companies about a drug rescuing effort at a workshop in April 2011 . The resulting Discovering New Therapeutic Uses for Existing Molecules program  is "the first signature initiative by" NIH's 4-month-old National Center for Advancing Translational Sciences (NCATS), Collins said. NCATS plans to put $20 million of its $575 million budget request for 2013 into the new program.
Researchers will be able to browse basic information on the drugs online. If they see one that interests them and successfully apply for a grant, they will receive access to the compounds and detailed data on safety, pharmacokinetics, and dosing. If the drug meets milestones in animal model tests, they may receive funding to take it through early clinical trials. NCATS Acting Deputy Director Kathy Hudson said NIH anticipates making perhaps eight to 10 cooperative agreements that will run for up to 3 years.
The pilot program also includes a "template" legal agreement with companies. The company retains ownership of the compound, but researchers will have rights to new intellectual property that they discover. The researchers are free to publish their results, although the company gets to review the manuscript to protect confidential information.
If a compound shows promise, NIH hopes that the original company (which has the first option to license it) or another company will take it through late stage clinical trials. Collins said NIH hopes the program will benefit areas of research like neurological diseases, which companies have stepped back from because of long development timelines and uncertainty of success.
NIH's new program is similar to an agreement AstraZeneca made in December with the U.K. Medical Research Council to share 22 compounds; it has already attracted more than 100 proposals, said Donald Frail, vice president of the company's Science and New Opportunities Innovative Medicine Unit.
Pfizer is interested in the NIH program because it already has a drug repurposing collaboration with Washington University in Saint Louis and realized it needed a broader effort, said Rod MacKenzie, group senior vice president, head of pharmatheraupetics research and development. "It gives us a chance to access on a grand scale ... the wonderful minds that we have out in the academic community," McKenzie said.
Although 24 compounds add up to only a small fraction of the drugs on companies' shelves—MacKenzie said that Pfizer has "dozens"—NIH is hoping that more companies will participate. "I think people were waiting to see what this [program] looked like," said Sebelius.
*Correction, 4 May: An earlier version of this article incorrectly stated that the companies cannot impose restrictions on publishing results. The research agreements state (i.e. Sec. 11 in Pfizer agreement ) that the company has the right to review and revise manuscripts to protect confidential information. The company can also ask that submission be delayed 30 days so it can apply for a patent.