1. NIH: Action on Translation.
Last year on the FasterCures "Top 10" list, we highlighted NIH's new Therapeutics for Rare and Neglected Diseases (TRND) program as a potential model for the role NIH could play more broadly in helping promising basic discoveries bridge the "valley of death" between preclinical development and clinical research. In late 2010, the agency's Scientific Management and Review Board proposed the creation of a Center for Advancing Translational Sciences to do just that. The new Center, which NIH Director Francis Collins has committed to have up and running by the end of 2011, "would generate innovative tools, technologies, and ideas that would transform translational efforts throughout the scientific community." There's sure to be debate about what this new Center will and should do, and whether it will detract from NIH's basic research activities or deliver greater value to already important work.
2. Health IT: Will "meaningful use" be meaningful for research?
Since 2009, the federal government has put the pedal to the metal to accelerate widespread adoption of electronic health records by health providers and hospitals, and get them to "meaningfully use" these systems for data capture and sharing of the most elementary kind. What has not yet happened is a necessary focus on the secondary uses (an unfortunate term) of that data, including, very importantly, clinical research. 2011 will be the year to tell the feds what needs to happen to ensure that all that data being collected by docs is accessible to researchers trying to cure the diseases they're treating. Researchers and patients must drive that conversation. Have you asked your provider how their EHR system is faring? Stay tuned for FasterCures' paper on this topic in early 2011.
3. FDA: Stand and deliver.
2010 ended with industry commenting about the low number of products approved by FDA over the year, and FDA commenting about the low number of New Drug Applications submitted by industry. 2011 will almost certainly be consumed by the wrangling over PDUFA reauthorization. Getting more done with less will most certainly be a theme. We'll be focusing our attention on longer-term concerns, such as: Has the agency made progress on its regulatory science agenda (debuted last year)? What is the new Joint Leadership Council with NIH doing? Could this be the year the Reagan-Udall Foundation takes off? The heat is on.
4. Pharma: Can you learn something from the oil and gas industry?
Industries like microprocessors, film, and oil and gas have all faced market-driven inflection points and have reinvented themselves to respond. For example, the oil and gas industry has consortia that engage in pre-competitive research and exploration; companies take a more holistic view, and understand the need for sharing data to reduce risk and price volatility. Similarly, the pharmaceutical industry needs data-sharing and trusted third parties to help it tackle common challenges. What can be learned from these models, and who can create the framework for applying those lessons in this sphere?
5. Where is our GPS: Mapping the pre-competitive space.
Over the last year we've seen increasing interest within the biotech and pharmaceutical industries in collaborating more across companies and sectors on pre-competitive areas of research. But the concept remains ill-defined. Can you pre-compete and remain competitive? How should intellectual property be treated, and can it be handled in a standardized way? Will this be done in a one-off manner, or can it be managed/organized? Can we create a "marketplace" that will help facilitate the exchange of pre-competitive information? Keep an eye on new efforts like Sage Bionetworks' Archipelago initiative, which aims to create just such a marketplace.
6. Follow the leaders across the Valley of Death (but remember to pack some provisions).
We have been very interested to see over the last year a wide variety of new experiments aimed at addressing barriers to financing, skills, and incentives that will move promising ideas through the pipeline. At the end of 2010, we released a new white paper, Crossing Over the Valley of Death, which highlighted the importance of translational research and the challenges it presents. At Partnering for Cures 2010, we heard presentations from almost half a dozen different efforts with similar goals -- from various initiatives to develop university intellectual property further before licensing it, to creation of portfolios of projects rather than companies for investment, to "social business enterprises" to fund development of promising discoveries. We'll be interested to see which models rise to the top but more importantly, who can demonstrate tangible results, and then how do we grow them?
7. Grab your partner: Is venture philanthropy the new venture capital?
More and more biotech and pharma companies are forming alliances with nonprofit disease foundations -- not just as advocates for their products and sources of clinical trials participants, but, at a time of increasing costs and declining success rates for new therapies, as potential research funding partners. Is this a phenomenon of significant scale and impact? How do these deals work? What does each party bring to the table? What results have been achieved? On the subject of venture capital, we're watching folks like Third Rock Ventures who are still willing to invest in early-stage development, and Enlight Biosciences, which is investing pharma funds in enabling technologies.
8. Tear down this wall: The academia/industry business interface.
The business interface between universities and companies is an area in crying need of more standardization. One-off deals have high transactional costs, and yet the parties are reluctant to adopt the best practices of others. Successful models of university-industry collaboration in places like University of California San Francisco -- where the focus is on high-value research moving from universities into development at companies, rather than on royalty revenue for either party -- need to be spotlighted, studied and replicated. And universities need to significantly rethink their internal reward structures to value industry collaboration and patents, not just government grants and publications. Who best to lead the charge here?
9. The future of research: Don't trust anyone under 40?
By now the statistics are familiar: The average age of an investigator receiving his or her first NIH grant is 42-years-old. NIH is funding significantly more investigators over the age of 60 than under the age of 40. Plenty of people are worrying about how to attract and keep young people in the system as it currently exists. Not many are trying to see the world through their eyes and build a system that works for them. Technology, social networking, collaborative work, data sharing, crowd sourcing, interdisciplinary science, convergent disciplines are all increasingly second nature to the establishment of tomorrow. Maybe we need to learn a lesson from them today -- and get out of their way, or risk losing an entire generation, and more.
10. See the world: Disease and the search for cures know no borders.
To help think about what research and business models of the future can and should look like, see what's happening in places that don't operate within the system that has grown up over 50 years in the U.S. Not only is intellectual capital locating elsewhere, but places like India, China, Singapore, and Africa are innovating out of necessity and producing some remarkable results with great speed and in some cases, few resources. And what about countries with wealth like Norway and Qatar -- how can we engage them to invest significantly in medical research; can we initiate a Global Giving Pledge for sovereign wealth funds to dedicate resources to finding cures? The Rolling Stones told me that "Time waits for no one."