Friday, January 28, 2011

Innovation Nation: State of the Union Emphasizes Science and Technology as Engines of Growth

“Innovation doesn’t just change our lives; it is how we make our living.” – President Obama
During the State of the Union address, President Obama extolled the virtues of science and technology in advancing America’s strength and ensuring our competitiveness in the global economy. He spoke of his administration’s commitment to making new investments in biomedical research and IT, and training an additional 100,000 science, technology, engineering, and math teachers by the end of the decade.

This focus on scientific innovation goes a long way in making the search for cures a national priority. As Dick Gephardt and Mike Leavitt, co-chairs of the Council for American Medical Innovation, said in their editorial in The Hill, “nowhere is there a better value proposition for America than the biosciences.” This echoes a sentiment expressed in the New Biology for the 21st Century report, released through the National Academy of Sciences, which calls for greater integration across the sciences – biology, physical, computational, and earth, as well as mathematics and engineering – to find solutions to four key societal needs: sustainable food production, ecosystem restoration, optimized biofuel production, and improvement in human health. The report outlined the need for a coordinated effort to leverage resources across the federal, private, and academic sectors to help meet challenges and improve the return on life science research in general.

For more than a hundred years, the United States has been synonymous with medical discovery. In fact, the Milken Institute estimates that 50% of all economic growth is the result of lives improved or extended by medical science. But the system that made all that progress possible in the last century needs to now work for this century. Medical outcomes could be greatly improved if we spend the same kind of manpower and resources we spend on consumer products on discovering and developing new cures for disease.

To regain and maintain our global scientific leadership, and to speed the path to cures for patients who need life-saving treatments and therapies, FasterCures stands at the ready to work with the government and all sectors to advance the following:
  • Increasing resources and scientific capacity at the U.S. Food and Drug Administration to support more efficient and effective approval of new medical solutions
  • Advancing policies that foster innovation and translation at the National Institutes of Health
  • Encouraging innovative, cross-sector collaboration among all players in the medical research enterprise
All stakeholders, public and private, have a critical role to play in this process. What are we waiting for?

Monday, January 24, 2011

On Huffington Post: Top 10 Medical Research Trends to Watch in 2011

Technology, social networking, collaborative work, data sharing, crowd sourcing, interdisciplinary science, convergent disciplines are all increasingly second nature to the establishment of tomorrow.

1. NIH: Action on Translation.
Last year on the "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's 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 likeSage 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, crowdsourcing, 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."

Wednesday, January 12, 2011

UK Proposes a New Clinical Trials Agency to Improve its Global Standing in Clinical Research

by Adam Clark, Ph.D.,Director of Scientific and Federal Affairs at FasterCures

Fewer patients in clinical trials, increasing costs and time for drug development, and a “one-size fits all approach” to drug approval – are among the standard descriptors of the challenges facing clinical research in the U.S. However a new report by the Academy of Medical Sciences, A New Pathway for the Regulation and Governance of Health Research, points to these as barriers to medical progress in the U.K. as well.

The report details that in just four years the U.K.’s global share of patients involved in clinical trials fell by over half to under 3 percent. And, it will be even lower as pharmaceutical companies cited expectations to continue to reduce the number of trials due to the complex regulatory environment. With disparate agencies and bodies needed to approve numerous steps in the clinical trials process, it takes well over a year and a half to move from applying for a trial to enrolling the first patient – a process that does not weigh in favor of benefiting individuals, the public health, or the economy.

To revamp the system and enhance U.K.’s prominence in clinical research, the report calls the British government to establish a new Health Research Agency (HRA) to streamline the governance and approval across all aspects of a clinical trial and to have a single system for ethical approvals. The proposed HRA would embed research as a core activity within the National Health Service and increase public involvement and patient participation in clinical trials. They propose to establish “safe havens” to identify patients who match for clinical trials and offer the option of an investigational drug. Additionally, the HRA would implement appropriate measures that balance the benefit:risk ratio based on patients needs.

From across the pond, members of the 112th Congress and policy-makers should pay close attention to these recommendations and steps the U.K. may take to streamline and improve its clinical trials regulatory environment. For the U.S. to maintain its global leadership in medical innovation, we must step up to the task of ensuring our clinical research system is able to deliver the best possible outcomes for every patient. Maximizing the return on our nation’s investment in both basic scientific discovery and improved health care delivery hinges on a modernized approach to clinical research.

As the challenges are shared, the solutions may be even more so. When nations compete to improve its global standing in clinical research, patients win.

Monday, January 10, 2011

“Convergence Creole” – Is Interdisciplinary Research the Future of Medicine?

by Gillian Parrish, Manager of Alliance Development and Communications, FasterCures
Innovation in biomedicine and other fields is dependent on the merging of life, physical, and engineering sciences into a unified whole, according to a group of leading Massachusetts Institute of Technology (MIT) scientists. Called Convergence, this new model emphasizes integration between disciplines that were originally viewed as separate and distinct.
In a white paper released January 4 at the American Association for the Advancement of Science (AAAS) in Washington, D.C., the 12 scientists – based in the David H. Koch Institute, a center for integrative cancer research at MIT – explain how the practice of Convergence will herald a “Third Revolution” in biomedical research, and speed the path to personalized, accessible, and affordable health care. (They point to the discovery of the DNA structure in 1953 as the first revolution, and the sequencing of the human genome in the late 1990s as the second one.)
As FDA Commissioner Dr. Margaret Hamburg explained, this is not about creating yet another academic track or siloed pipeline of “convergent scientists,” but rather building fundamental comfort with cross-disciplinary language and practice early on so that when faced with a research challenge, collaborative problem solving becomes the norm.
Dr. Alan Leshner, CEO of AAAS and Executive Publisher of Science, likened this approach to taking an immersion language class. But instead of studying Spanish or Mandarin, students would be required to become proficient in what he coined “convergence creole” – derived from multiple “parent languages” across the scientific and engineering fields. I loved this analogy, not only because increased collaboration at all stages of biomedical research is one of FasterCures’ key priorities, but also because as a native Louisianan, I happen to know that all things creole are both striking and delicious.
Convergence in action:
Dr. Paula Hammond, Bayer Professor of Chemical Engineering at MIT and one of the paper’s authors, offered a few examples of the recent fruits of convergence in the biomedical space, such as:
  • The engineering of nanoparticles to specifically target cancer cells and improve chemotherapy delivery
  • The development of brain grafts to treat victims of traumatic brain injury
  • The creation of a CTC Chip for cancer metastases detection, which uses a blood test to detect a single cancer cell from among a billion healthy cells before it shows up on scan (recent media coverage is available here)
In order to capitalize and expand on outcomes like these, the scientists called on NIH and other affected agencies to take up Convergence as the wave of the future – by enhancing the pipeline of convergence-oriented researchers, rethinking academic incentives for collaboration, and expanding funding sources. More about their specific recommendations.