More than 650 participants tuned in to a Webinar on March 16 hosted by FasterCures with National Institutes of Health Director Francis Collins, who gave the latest updates on the progress on the proposed National Center for Advancing Translational Sciences (NCATS), answered questions, and sought to dispel myths about this effort. Participants were from 38 states and represented all medical research sectors – more than 200 patient advocates from 100 different organizations, nearly 200 academics from 78 research institutions, 62 from biotechnology and pharmaceutical companies, 82 from various government agencies, and dozens of other stakeholders from the venture capital community to policy influencers.
Calling NCATS “a disruptive innovation, in a good way,” Collins said the motivation for the new center is “the need to view the drug development pipeline as a scientific problem, ripe for experimentation and process engineering.” He noted that now is the time to act as opportunities for new drug targets to be tackled successfully have been growing well beyond capacity of the private sector to take on by itself and that the appetite for public-private partnerships is high. Engaging in translational research is not new for NIH, he said. A 2010 survey showed 550 translational research projects going on at NIH (65 percent preclinical and 35 percent clinical), and a recent New England Journal of Medicine article reported that 153 new therapies have been discovered through research carried out in public-sector research institutes, most of which have been supported by NIH.
But he also acknowledged that the productivity of the medical research system overall is declining, and stated his belief that NIH – by facilitating and supporting partnerships, providing resources, and enhancing training – has an increasingly important role to play in moving the results of promising research it funds down the pathway toward new treatments. “The opportunity for new targets is growing well beyond industry’s ability to address them all. … In addition to neglected diseases, for many common diseases there are ‘neglected targets,’” which NCATS can help de-risk and make attractive for investment. He also believes that NCATS could be “quite catalytic” in helping repurpose abandoned compounds, and mentioned that NIH will be holding a meeting with biotech and pharmaceutical executives in April to discuss such ideas.
Collins was emphatic that NCATS is intended to complement, not compete with, the private sector. “We are not turning NIH into a drug development company,” he said, contrary to some headlines that the initiative has generated. He also took care to say that there is no intent to take focus and resources from NIH’s basic science mission and programs. As a matter of fact, he believes NIH’s investment in translation can empower basic research.
FasterCures Executive Director Margaret Anderson moderated this virtual meeting, noting that NIH’s effort to make translational research a priority is significant and meaningful for the thousands of patients with a deadly and debilitating disease for which there are no viable treatments or cures -- especially when you consider industry’s estimate that for every 5,000 compounds tested, only five make it to clinical trials, and only one is ever approved by the Food and Drug Administration. Patients are anxious for – and deserve – better returns on our nation’s research investment.
Collins appealed for all research stakeholders to be reasonable about expectations for this new effort, saying that NCATS should not be judged by the number of drugs ultimately approved. He ended by saying, “This is science, so there will be surprises along the way. But there is an opportunity to do something historic here.”
NIH is actively seeking feedback on the proposed structure and functions of NCATS at a new Web site. An archive of the FasterCures Webinar is available for viewing.
Friday, March 25, 2011
Friday, March 11, 2011
Welcome to TRAIN Central Station 2.0
by Kristin Schneeman, program director, FasterCures
Welcome to TRAIN Central Station 2.0, a new and improved version of FasterCures’ online platform for venture philanthropy in medical research. We’re excited to be bringing you lots of frequent updates and fresh content in the coming months, including:
Welcome to TRAIN Central Station 2.0, a new and improved version of FasterCures’ online platform for venture philanthropy in medical research. We’re excited to be bringing you lots of frequent updates and fresh content in the coming months, including:
- Monthly Webinars, beginning on March 16, 4:00-5:00 pm ET with a special appearance by NIH Director Francis Collins, engaging in a Q&A on the proposed new National Center for Advancing Translational Sciences. April’s Webinar will feature legal experts Ken Schaner and David Lubitz on “Unlocking IP at Academic Institutions.”
- New interviews with trailblazers in our Innovator Spotlight. Read the latest with Alpha-1 Foundation’s John Walsh.
- The good old E-News, back on a monthly schedule and chock-full of interesting stuff. This month, we highlight Institutional Investor magazine’s piece on venture philanthropy; an open letter to the pharma industry asking them to contribute discontinued compounds to a central repository; and the launch of HealthResearchFunding.org, which aims to connect funders with peer-reviewed, meritorious research proposals.
- Links to case studies and best practices. Check out the smorgasbord of fascinating presentations on cross-sector collaborations at December’s Partnering for Cures conference.
- Occasional guest blogs from people we find fascinating.
We want this to be not just a megaphone for FasterCures, but a community-building tool, and for that to happen, we need you.
- Use the discussion board as a place to solicit others’ ideas on how to handle problems you’re facing, or share information about issues of concern to you. While you’re there, offer up your questions for our Webinar with Francis Collins on March 16!
- Share with us any tools or resources you’ve developed at your organization (e.g. legal documents, project management tools, etc.) that you think might to useful to others in doing their work and we’ll post them in our Tools and Resources section.
- Send us your calendar items and job postings that might be relevant to the TRAIN community and we’ll share them on the site.
As always, we look forward to your feedback on how we can continue to improve the site, as well as your contributions of material for it. Enjoy the ride!
Friday, January 28, 2011
Innovation Nation: State of the Union Emphasizes Science and Technology as Engines of Growth
by Gillian Parrish, Manager, Alliance Development & Communications
“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 (CAMI), 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 100 years, the United States has been synonymous with medical discovery. In fact, the Milken Institute estimates that 50 percent 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:
“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 (CAMI), 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 100 years, the United States has been synonymous with medical discovery. In fact, the Milken Institute estimates that 50 percent 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?
Top 10 Medical Research Trends to Watch in 2011
by Margaret Anderson, Executive Director, FasterCures
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 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, 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."
Published on The Huffington Post
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 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, 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."
Published on The Huffington Post
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