Friday, January 18, 2008

FasterCures’ Ten to Watch in 2008

1. Return on philanthropy. When we invest our money, we look for the best ROI; when we donate it, shouldn’t we look for the best ROP? But how can we evaluate the effectiveness of nonprofits funding medical research? So far we’ve been looking in all the wrong places. Charity Navigator isn’t it – not enough information. New efforts like GiveWell definitely aren’t it – GiveWell’s approach of “cost per life saved” is the equivalent of what investors call “chasing performance.” So how are well-meaning philanthropists supposed to decide where their money will have the greatest impact in finding new cures?

2. The FDA. Yes, you heard me right, the FDA. If we want to get new therapies to patients faster, the federal agency whose budget we should be doubling right now is not the NIH’s but the FDA’s. After years of an expanding mission and a shrinking budget, in 2007 the FDA was finally given new authorities and some new resources to try to keep up with the tide of new technologies, methodologies, and products aspiring to or already in the marketplace. The question is, will Congress and the agency itself follow through on the promise? The first step was not promising, as Congress withheld funding for the Reagan-Udall Foundation, newly created to support FDA’s important Critical Path initiative. Everyone who cares about cures needs to be watching.

3. Research: Lost in translation. We all know we need translational research. Let 2008 be the year we define it, promote it, create more resources to support it, train people to do it, reward the ones who are doing it well.

4. Science 2.0. Collaborative science is the name of the game these days, as science gets bigger and more multi-disciplinary and the data available for research grows explosively. The technological opportunity presented by the Semantic Web for networking data and researchers will be transformative. Watch the Neurocommons Project, a demonstration of the power of the Semantic Web approach based on open access information.

5. Crowd-sourcing innovation. We’re all familiar with outsourcing – but how about opening up your R&D challenges to the “wisdom of the crowd?” That’s the approach taken by Innocentive, a Web site on which “seekers” can post problems to be tackled by a universe of “solvers,” many of them from entirely different disciplines. Born out of a need to open up innovation within the pharmaceutical industry, the site is now beginning to be used by groups like the “Prize for Life” challenge to find a biomarker for ALS, and the Global Alliance for TB Drug Development.

6. Prize philanthropy. A more top-down approach to such prizes is that of the X PRIZE Foundation. The Archon Genomics X PRIZE is engaging self-organized teams of scientists all over the world in the quest to make gene sequencing more widely available and affordable. What other challenges in health might lend themselves to this kind of high-profile effort?

7. Biomarkers Consortium. This initiative of the Foundation for the National Institutes of Health is a rare and promising partnership of federal agencies (research funders as well as regulators), the biopharma industry, and nonprofit research foundations, established in 2006 to discover, develop, and qualify biomarkers to support more predictive, preventive, and personalized medicine. With several projects off the ground and many more in the pipeline, this “pre-competitive knowledge commons” is a model which should be replicated again and again in the balkanized world of disease research.

8. Comparative effectiveness. Sounds like a no-brainer, right? We should study the effectiveness of medicines for treating a condition and use the ones that are proven to work best. But as the drumbeat around this buzz-phrase (and its companion, evidence-based medicine) gets louder in the coming debate about healthcare reform in this country, we need to make sure we understand its implications for patients’ access to treatments that work best for them, and for incentives to pursue new and even better therapies.

9. Electronic health records (EHRs). With “only” 17 months left in office, the Bush Administration last year decided to run out the clock instead of trying to win the game, shifting its focus from planning the structure of the “Internet for healthcare” to planning the structure of the government’s EHR bureaucracy two years from now. Sound weird? It is. In 2008 millions of people will be able to Google-ize their health records or deposit them in Microsoft’s Healthvault – leapfrogging not only the feds but their doctors’ offices, which are still photocopying their paper records. But who’s thinking about whether and how all this electronic health information might be accessible (or not) to the researchers trying to treat and cure what ails us? Watch this space.

10. Clinical trials: Something’s gotta give. The cost in time and dollars of clinical trials is crushing the discovery enterprise. Will 2008 be the year the FDA is convinced to break the mold and streamline of the clinical trials behemoth, allowing sponsors to adapt their course based on new information generated by the trial? What is the role of sophisticated computer models like The Archimedes Model in designing and simulating clinical trials? Do we just, in the words of a prominent clinical research leader, need to stop doing randomized controlled trials stupidly? One thing’s for sure – this is an issue in need of revolutionary, not evolutionary, change.

Greg Simon, President, FasterCures


Anonymous said...

I think a project "new uses for old drugs" at Johns Hopkins is relevant to almost all of your points (Chong and Sullivan Nature 2007). May I ask you what is your opinion? Thanks a lot! Boris

Kristin Schneeman said...

“We definitely believe that pursuing new uses for existing compounds is a very fruitful avenue for research. We work with a number of nonprofit disease research foundations that are screening existing compounds for efficacy in their disease areas, such as the ALS Therapy Development Institute and the Foundation Fighting Blindness, and their efforts have produced a number of potential candidates working their way through preclinical testing.

The Hopkins Clinical Compound Library is a great resource. There are certainly significant barriers to achieving the goal of expanding it to include the almost 10,000 known drugs, but it’s a worthy one that FasterCures supports.”