David Altman is manager of marketing and communications in Boston Children’s Hospital’s Technology and Innovation Development Office.
Successful therapeutic development requires multiple stakeholders along the path from discovery to translation to clinical trials to FDA approval to market availability. At various points along this path, academia, industry, government, hospitals, nonprofits and philanthropists may work together. Would bringing these stakeholders together from start to finish lead to greater success?
A growing number of private-public consortia are launching in defined “pre-competitive” spaces where potential rivals collaborate to generate tools and data to accelerate biomedical research. In 1995, consortia were rare in health care: Only one was created. In 2012, 51 new consortia were launched, according to the organization Faster Cures.
Why? you may ask. Banding together in consortia can reduce costs, minimize failures and shorten the timeline to approval for new drugs. Currently, only one of 12 drugs entering clinical trials meets its endpoints. A successful drug can cost more than $1 billion to develop and take more than 10 years to clear clinical trials. For some conditions, such as Alzheimer’s disease and autism, the need for new treatments is enormous, yet the lack of disease understanding has delayed clinical development.
After two years of discussions and negotiations, the National Institutes of Health (NIH) recently announced a unique, unprecedented consortium. The $230 million partnership brings together the NIH, academic medical centers and institutions, foundations and 10 drug companies, including GlaxoSmithKline, Merck, Eli Lilly and Sanofi, to jointly identify and validate promising biological targets of disease. (A full list of industry and foundation partners is found here.) That is a welcome effort, since choosing a suboptimal target can result in costly drug failures late in the development process.
Called the Accelerating Medicines Partnership (AMP), the program is funded half by private companies and half by the NIH. It will focus on three disease areas: Alzheimer’s, diabetes and autoimmune disorders (rheumatoid arthritis and lupus). The selection of projects differs by disease area: Projects related to Alzheimer’s will be selected from existing National Institute on Aging grants, rheumatoid arthritis and lupus projects from existing NIH grants and type 2 diabetes projects both from NIH grants and through an open call for proposals.
Project management of the selected pilots will be through the NIH’s own foundation (FNIH), whose mission is to improve health by forming and facilitating public-private partnerships for biomedical research. Scientists from NIH and industry will develop research plans and contribute relevant data and samples from clinical trials. The studies themselves will be conducted by the NIH, the companies and the academic research centers receiving AMP grants, with companies providing the “deep computing” for analyses. Disease foundations will participate by backing projects and recruiting patients for trials. Finally, NIH scientists will review the studies’ progress and help make scientific decisions.
Here’s what really makes this consortium special: Within five years, all data and analyses will be shared publicly with the biomedical community beyond the participants in the consortia. No company may use any discoveries for its own research until specific project data become public.
Another unique aspect of this model is its governance structure. The steering committees will be made up of representatives from NIH, FNIH, participating companies and patient advocacy organizations.
Interestingly, researchers will be added to disease steering committees after AMP research grants are awarded, but according to David Wholley, the FNIH’s director of research partnerships, their participation will be only to provide progress updates. Grantees will not participate in any votes or decisions about whether that research continues. Those decisions will be managed through the usual NIH processes.
The U.S. government’s increased funding and commitment to understanding cancer biology in the 1970s has led to a large increase in targeted drug therapies for cancer. In 1971, the year the “War on Cancer” was declared, there were 3 million cancer survivors in the U.S. As of 2007, that number has increased to more than 12 million—a startling result. Thirty years from now, we may see similarly impressive results from this new government-backed consortium model—and more treatment options for the patients who most need them.