Editor’s note: This post is published in conjunction with the April issue of Health Affairs, which explores the many subjects raised by Alzheimer’s disease including a new public-private research collaboration designed to produce improved treatments.
Earlier this year, the National Institutes of Health joined forces with ten major pharmaceutical companies and several nonprofit disease interest groups to create the Accelerating Medicines Partnership (AMP). With an integrated governance structure consisting of representatives from all partners, the AMP venture aims to combine public-private expertise and pooled resources to reduce the time and cost of developing biomarkers for therapeutic targets.
The initial capitalization is reported to be $230 million. The AMP is the first national cross-sector partnership of its size and scale, and is the latest initiative in the drug development market to embrace open data exchange, encouraging collaboration over competition as pathways for promoting innovation.
The AMP management chose to focus on four diseases—Alzheimer’s disease, type 2 diabetes mellitus, rheumatoid arthritis, and systemic lupus erythematosus—in which there was solid knowledge about the underlying pathophysiology, a sufficient level of potential therapeutic targets open to pursuit, and a lack of substantial individual manufacturer commitment. The latter criterion explains why more prevalent diseases such as cancer did not make the list.
Although tremendous progress has been made in basic research in the past two decades—with advancements in genomics, proteomics, and image processing—some have been disappointed with the number of breakthroughs in disease treatment that have followed. Various theories have been proposed to account for this disappointing output from the pharmaceutical and biotechnology industries.
Some commentators blame an intellectual property environment in which excessive reliance on patents leads to competing ownership rights and reduced collaboration. Others point to financial incentives that encourage investment in incremental improvements within known therapeutic approaches over transformative discoveries that may open up new avenues to fight disease.
The AMP seeks to improve innovative therapeutic output by reforming the industry’s inefficient and fragmented approach in sorting through massive genomic and molecular data to find new drug targets. Rather than having companies working in-silo, which can lead to wasteful repetitive testing of failed compounds, AMP hopes to create a more systematic and collaborative approach towards pinpointing the right biological targets early in the process, sifting through potential biomarkers, and identifying specific genes or proteins that can be altered by a drug to effectively curb disease.
The appeal of such public-private partnerships is that historically such partnerships have led to transformative discoveries. The cancer drug paclitaxel (Taxol) emerged from decades of testing at NIH, and was then scaled up and marketed based on work done at Bristol-Myers Squibb after clinical proof of principle was established. The first treatment for HIV (zidovudine) was discovered based on testing done at NIH laboratories on a compound sitting on the shelf at Burroughs Wellcome.
Government-funded investigators in academic medical settings, working collaboratively with colleagues in pharmaceutical companies, discovered the use of imatinib (Gleevec) for chronic myelogenous leukemia, vascular endothelial growth factors to treat ophthalmologic disease, and TNF blockers in rheumatologic and inflammatory bowel diseases. The list goes on and on.
Perhaps as a result of greater recognition of these historical success stories, public-private efforts in the pharmaceutical sector are becoming increasingly popular. In recent years, Eli Lilly, Pfizer, and GlaxoSmithKline are just a few of the companies that have founded collaborations with academic researchers that range from joint ownership of laboratories (e.g., Pfizer’s Centers for Therapeutic Innovation) to open access to industry resources and facilities (e.g., GSK’s Tres Cantos Open Lab Foundation).
Another major partnership, the WIPO Re:Search, shares intellectual property and resources among its participants in 19 neglected tropical diseases, malaria and tuberculosis, to overcome the lack of economic incentive for companies to pursue these areas separately.
The AMP differs from such existing partnerships in both scale and openness of collaboration. With a nod to the “open-source” movement within computer science, in which software code is made free and accessible to the community to encourage multiple contributions towards its development, the AMP requires that all data, methods, and analyses from early-stage trials be made publicly accessible to the broad biomedical community. However, the current plan is for companies to resume proprietary drug development once early clinical trial data from the partnership have been published.
This open-source/competitive entrepreneurship hybrid strategy also presents challenges. For example, the time point at which companies will be allowed to resume competition is a crucial one, but how will this time point be decided? For participating manufacturers, there may be potential gain in resuming competition at a point in time when their progress in a target disease area is ahead of other collaborators. Furthermore, the opportunity to overtake collaborators down the line may pose a disincentive for companies to participate fully in the sharing initiatives in the first place.
Other key obstacles that may stand in the way of successful partnership between AMP’s public and private entities include the variable attitudes towards intellectual property and misaligned reward systems. Academic researchers and industry employees may approach the partnership with different expectations or goals, and may differently prioritize such outcomes as regulatory filings or pipeline success. For example, while NIH and other public entities are driven towards reaching public objectives that are dependent on political will and funding, private industry actions tend to be dictated by market-share and growth potential.
It will also be interesting to consider how downstream products arising from the AMP will be priced, or how proceeds will be distributed. Pharmaceutical manufacturers justify high drug prices by citing large investments in the discovery, evaluation, and development of the products. Pricing of AMP products at similar levels would place an unfair double burden on taxpayers, who in this case would have directly financed the early-stage drug development. Promising compounds that emerge through the AMP process should come with responsibilities on the manufacturer regarding pricing or reinvestment of revenues in the AMP budget to appropriately reflect the reduced risk that these products present to companies taking them through clinical testing.
The AMP has designated three to five years as its initial pilot period. With such an ambitious timeline, setting milestones and tracking progress towards meeting those goals will be essential. The decline in R&D productivity among drug and biotechnology firms has created a sense of urgency and necessity for public and private entities to emphasize collaboration over competition. The AMP fulfills this role with unprecedented size, scale, and scope. Although many challenges lie in its future, it could prove to be an important milestone in the path towards building successful drug innovation systems.