Friday, May 27, 2011

TRAIN Group at Center of New NIH Collaborative Effort

Kristin Schneeman, Program Director, FasterCures

aims to expedite cures by stimulating innovative collaborations across all sectors. We recognize that a collective approach is needed to successfully address the barriers to innovation. That is why we were fascinated to note this week the announcement that NIH has just signed a cooperative research and development agreement (CRADA) to move promising therapies for rare blood cancers into clinical proof-of-concept studies. This is an excellent example of collaboration that we’d like to see more of in the future.

While NIH has engaged in CRADAs for many years to make government facilities, intellectual property, and expertise available to further the development of marketable products, this one caught our attention because of the involvement of the Leukemia & Lymphoma Society, a major funder of blood cancer research. The press release announcing the agreement explicitly noted the role that philanthropic and patient organizations have in the drug discovery process. Also engaged in the collaboration is the University of Kansas Medical Center.

The agreement is notable to us also because NIH is viewing it as a “Learning Collaborative,” the goal of which is “to bridge the gap in time and resources that often exists between basic research and human testing of potential new treatments.” “We plan to learn more about and improve the drug development process for all diseases,” says Chris Austin, director of NIH’s Therapeutics for Rare and Neglected Diseases program, which is spearheading this effort.

FasterCures, through our TRAIN program, has long been a proponent of the important role that patient-driven philanthropic organizations can play in funding innovative approaches to medical research and in helping move promising discoveries across the translational “valley of death” toward new treatments. They bring not just risk capital but also the voice and priorities of patients into the research process.

This agreement is part of a heartening trend (no pun intended) at NIH to play a more active role in bridging the gap between the many promising discoveries it funds and the point at which biotechnology or pharmaceutical companies are willing to invest in expensive late-stage clinical research. We are pleased to see that Leukemia & Lymphoma Society has a seat at the table in this effort, and we hope to see more collaborations of this kind in the near future. We look forward to learning along with The Learning Collaborative about how government, academia, nonprofits, and ultimately industry can work more efficiently and effectively to get badly needed new treatments to patients.

Tuesday, May 17, 2011

The Data Deluge

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

Earlier this year FasterCures put together a Top Ten list of Medical Research Trends to keep an eye on in 2011. Coming in at number 5 on the countdown was Where is our GPS: Mapping the pre-competitive space. The trend outlined whether we could create a "marketplace" that will help facilitate the exchange of pre-competitive information and communal data sharing.

Recently, I saw first-hand a plan in action to work on this issue. I was among the participants at the second annual Sage Bionetworks Commons Congress. Sage Bionetworks is a new medical research organization with a focus on understanding biology as a network science and biological research as a community effort. Sage’s approach - shift to data intensive science built upon open access and broad collaborations to design network maps of disease built upon vast databases linking genotpyes, treatments, and medical outcomes.

Genomics is moving biology from an observational science to an informational science. Our way of thinking of disease is shifting from one gene, one disease to multiple genes with numerous phenotypes. In fact, diseases like cancer are now considered to be hundreds, if not thousands, of different diseases with a variety of underlying genotypes. This shift in understanding disease also means we need a shift in researching them as well. Finding cures for complex diseases in the 21st century will require us to fundamentally rethink how we conduct biological research.

Sage Congress participants noted that scientific disciplines such as physics and astronomy have benefited tremendously from data sharing and collaboration to build a fundamental new understanding or the universe. Similarly, the computer industry learned decades ago that open source access to software accelerated innovation, allowing tailoring of new applications to reach broader consumer markets. However, the field of biology has not yet undergone such a cultural shift. Despite technological revolutions in genomics and information technology, its approach to data sharing and knowledge generation still remains largely confined to closed networks and research silos with a focus on journal publications.

Sage is attempting to challenge this traditional paradigm by building a federated, open network commons for researchers to share large data sets, protocols, and research findings (positive or negative) to encourage collaboration and cross discipline interactions. This model supposes that the next biomedical revelations will be data driven and will require diverse talents and a community approach to research. Sage is currently underway building a data repository and the Congress featured a demo of Synapse, a portal that will ultimately include the repository, a cloud compute component, and tools for collaboration and workflows.

This is a decidedly different approach to biology, one that will require support from the research community as well as the patient organizations and the public. However, if this new world of data-driven medical science is to be a success, funding agencies and academia will also need to conform to the new reality. Incentives will need to shift to promote open access to data and collaborative science, both in terms of funding and career promotion.

It was noted at the conference that biomedical research is not so much limited by technology as it is by its culture. And cultural change can be extremely difficult. But if efforts like the Human Genome Project are any example, outcome-oriented research and collaborative scientific enterprises can transform our understanding of biology and with it, our ability to treat and to cure diseases. And at FasterCures, that is the bottom line.

Related Resource:FasterCures webinar on “Data Sharing Policies and Opportunities for Nonprofit Funders of Research?” View an archive online.

Tuesday, May 10, 2011

Biomedical Innovation: Can the U.S. Keep Its Edge?

by Adam Clark, Ph.D., Director of Scientific and Federal Affairs, FasterCures
Over the past decade, international investments in life sciences research and development (R&D) in the in the private-sector have shifted away from the U.S. An expert panel at the 2011 Milken Institute Global Conference examined this shift and explored policies that influence biomedical innovation in the U.S.

Ross DeVol of the Milken Institute framed the issue by noting that it was not until the late 1990s that the U.S. began to dominate global pharmaceutical R&D. U.S. leadership can be attributed to a number of factors, he said, including a more predictable regulatory framework and adoption of intellectual property policies such as the Bayh-Dole Act, which encouraged entrepreneurship. However, DeVol said that “our regulatory approval process has gotten more cumbersome,” which has resulted in a risk-averse environment that inhibits medical innovation.

Edward Holmes of Singapore’s Agency for Science, Technology, and Research (A*STAR) said that Singapore has made the biomedical industry a top priority and developed policies that have resulted in a four-fold growth in the biomedical industry in the past decade. Such policies created an environment with sound intellectual property strategies, efficient regulatory programs, effective ethical frameworks for clinical trials, and an educational infrastructure that strongly supports science and math education to secure a strong scientific workforce. Singapore continues to focus on biomedical manufacturing and a strong effort to build up efforts in R&D.

Michael Leavitt, former Secretary of the U.S. Department of Health and Human Services, emphasized that the next decade of U.S. biomedical innovation will be built upon the “Era of the Value Proposition” in which “the future innovation will have to involve a value proposition that will demonstrate to a payor that the innovation will result in a change in the cost curve.”

Bijon Dorri of General Electric, explained GE’s interest in the healthcare sector and highlighted GE’s Healthymaginations program as an area in which GE is increasing its global competitiveness in healthcare. For projects supported through the Healthymaginations, it must address at least one of three pillars of healthcare: reduce cost, improve quality, or increase access. These guiding principles, he said, are “driving a change in the value proposition of healthcare innovation.”

The panel discussed the growing costs of clinical trials as one of the primary drivers to move R&D out of the U.S. to countries where such costs are significantly lower.

Collaboration was another key theme the panel deliberated on. Partnerships between the private and public sectors are key to moving therapies forward. There needs to be open communication channels between industry and the National Institutes of Health, the FDA, and the Centers for Medicare and Medicaid to ensure a seamless continuum of R&D innovation from discovery to development to dissemination and access.

Lesa Mitchell of the Kauffman Foundation moderated this panel. Watch a video of this session.

Monday, May 9, 2011

The Business of Personalized Medicine

Angelo Bouselli, Senior Communications Manager, FasterCures

With the sequencing of the human genome, and the scientific and technological advances of the 21st Century, many say we are at the advent of personalized medicine. But realizing the full potential of “personalized medicine” continues to be met with many questions largely left unanswered: Is it applicable across a wide range of diseases or just for a select few? Is it economically sustainable to truly personalize care?

“Everyone has a very different experience with healthcare,” said Greg Simon of Pfizer Inc., setting the stage for an engaging discussion on personalized medicine at the 2011 Milken Institute Global Conference.

Laura Esserman, Director, Carol Franc Buck Breast Care Center, Professor of Surgery and Radiology, University of California San Francisco said, “We need to learn how to treat disease and cancer better. We have learned that some cancers are biologically designed to kill and for those cancers, we need to treat them aggressively and immediately. Survival depends on giving the right drug at the right time, to the right person.

Paul Billings of Life Technologies echoed Esserman’s point and said that it’s about infrastructure and we need on that “will enable us to learn as we go.” “Personalized medicine can be a great thing if we put it in a framework we can learn from,” he said. We are improving care but we are still learning how to best apply the technologies to improve clinical decision making. “We need to educate patients that being involved in research makes a difference and provides better care.”

Panelists agreed that a system that combines data from the clinical care setting should be combined with the system that enables research to move forward.

“We need a connected research and care system that continually learns and improves on patient treatment and care,” said Esserman. “All of the information in the world won’t help you if you don’t know how to use it.”

Scott Jenkins of Dell agreed, noting that “electronic health records can assist in collecting and sharing valuable health information to improve treatment and care,” but that records alone aren’t the solution. He said patients play a major role and need to recognize the “value of sharing medical information and how this improves care.”

“We need to educate the general public to participate in medical research. It’s not just the patients that are struggling; the physicians are struggling to provide care. The entire system needs to be retooled.” Ricardo Guggenheim of McKesson Health Solutions said.

Personalized medicine, concurred the panel, starts with personal accountability for health decisions and greater patient participation in the research process.

Watch a video of the session.

Wednesday, May 4, 2011

Consumers Taking Control of Their Health

Cecilia Arradaza, Communications Director, FasterCures

More and more patients are managing their own health, making decisions based on the advice of their healthcare provider and on information garnered from a wide variety of sources – news articles, social networks, and blogs, said an expert panel at the Milken Institute Global Conference. The panel examined what influences these decisions – personal responsibility, workplace programs, and cost implications.

Heyward Donigan of ValueOptions said the “gig is up.” There’s a cultural journey toward health that makes it “hard to hide from the need to be accountable for your own decisions.” She noted that employers need to partner with consumers and facilitate decisions that will ultimately pay in dividends in terms of better health and greater productivity.

Richard Merkin of the Heritage Provider Network emphasized that “more care is not always better care,” and that access to technology and tools alone is not the answer to improving health outcomes. He said that in fact, “technology can sometimes exacerbate the problem if not matched with the comprehensive cognitive skills of an experienced healthcare provider, and accountability from the healthcare organization.”

The panel overwhelmingly agreed on the need to curb the sky-rocketing costs of healthcare delivery, noting that hospitalization is the largest slice of the healthcare spending pie and the system, as we have it, and should have more incentives in place to be able to provide the right care to the right patient at the right cost.

Paul Kusserow of Humana said that “when healthcare becomes episodic, it gets very expensive and unsustainable, but when it’s a dialogue, a continuum, we can start managing costs.”

Donigan noted successful returns in workplace programs that encourage wellness and healthy behavior in the workplace and early management of chronic diseases and that “healthcare costs can be cut without shifting it to consumers.”

Given the right options and information, American consumers will make the right decisions, the panel concurred. This includes sorting through massive amounts of information shared in social networking sites and real-time news coverage and analysis.

Jim Glasheen of Technology Partners moderated the discussion.

Watch a video of the session.

Global Health: Sustaining Momentum

By Kristin Schneeman, Program Director, FasterCures

In a forward-looking discussion on “Global Health: Sustaining Momentum” at the Milken Institute’s Global Conference in Los Angeles, Robert Sebbag – Vice President for Access to Medicines at sanofi-aventis – when asked by moderator Frank Sesno for evidence of momentum in global health, kicked off the panel by noting the diversity of the panelists’ backgrounds and remarking, “Was it even possible to have this debate 20 years ago?” Millions of lives have been saved, and global health, he said, has become a widely accepted tool of global diplomacy.

Panelists also identified risks to progress, including:

1) the global financial crisis, which threatens both financial capital and the political will to continue to invest it;
2) a new focus on chronic diseases in the developing world which threatens to distract from infectious and neglected tropical diseases;
3) the need for human capital to manage and execute programs in developing countries;
4) a brain drain of well-educated health workers; bottlenecks in the regulatory process; and
5) the need to more effectively communicate the progress that’s been made and the great returns on these investments to policymakers and the public.

Regina Rabinovitch of the Gates Foundation issued a call to action, saying “This is not the time to let up,” and “The world of global health has a history of failed five-year initiatives.”

Zeke Emanuel of the National Institutes of Health noted that global health funding has benefited from bipartisan support and that the 2011 budget increased more than $66 million, an achievement in an extremely difficult budget environment. He stressed the need to do a better job of showing Americans the improvements this is buying, and how our investment in health helps improve economies in the developed world and our own security. He also raised several times the need to package proven interventions, scale them up, and push them out to large numbers of people.

Ambassador Amina Ali of the African Union emphasized that the health needs of developing countries are too great to entirely rely on philanthropy to address them. She said that creative approaches to private sector funding and investment are needed for the long term. She also stressed the need for donor coordination and a more integrated approach by NGOs.

Sebbag argued that developed countries have a strong self-interest in these issues as globalization and climate change bring some of these diseases to the developed world. He also claimed that many pharmaceutical companies are realizing that significant growth in the future will come from the developing world, that a different economic approach is required, and that they are gaining valuable knowledge and experience by working in these countries.

Watch a video of the session.

Tuesday, May 3, 2011

Fulfilling the Promise of Science

Cecilia Arradaza, Communications Director, FasterCures

Over the past two centuries, medical science has contributed to half of the world’s economic growth. The Promise of Medical Science panel at the 2011 Milken Institute Global Conference agreed that if we are to improve the economy, we must invest in medical research.

In fact, Michael Milken, Chairman of the Milken Institute and FasterCures, said “medical science is the #1 stimulus to the economy.” Milken moderated the panel that featured Nobel laureates James Watson and Elizabeth Blackburn, University of California San Francisco chancellor Susan Desmond-Hellmann, and Amgen’s Corporate Chief Medical Officer Sean Harper.

Speeding Up Research
Hellman highlighted the vital nature of pursuing translational research, the kind of research that pursues clinical application of promising scientific discoveries.

“The bedside is increasingly becoming the bench and we are learning more from every patient interaction and outcomes,” she said. “Scientists should be more concerned to finish the race for patients rather than the race to be published.”

Blackburn noted that science is now emerging to intercept disease, leading to more effective prevention methods and earlier detection and diagnosis.

Panelists shared concern about how the biotechnology and pharmaceutical companies charged with developing life-saving therapies are valued much lower than companies producing consumer products like potato chips, cosmetics, and soft drinks.

“And yet, everyday, we see around us the need to invest in life sciences,” said Harper. “At Amgen, recent technology advancements allow for the ability to collect biomarkers that screen patients to test their responsiveness to therapeutics. Part of a larger movement towards personalized medicine that is expected to yield the most effective results.

Human CapitalMilken noted that the world’s assets are driven by the productivity of individuals, and economic growth greatly depends on medical research that will extend the average lifespan and improve the quality of life.

Hellmann noted that the pipeline of human capital hinges on education in the sciences. She emphasized that we need to introduce students to the thrill and promise of discovery at a young age. Students should be exposed to global health, encouraging them to use science as a way to solve the world’s problems.

Watson said that society needs to better acknowledge the “marvel of genius” and inspire it across generations. He added the need to give “real power to people under 30.” Watson was 25 when he was part of the team that discovered the structure of the DNA

Blackburn added the need to have a culture where “freedom of ideas is valued and explored.”
The panel acknowledged the critical role of government in science, noting the need to support efforts that encourage greater return for our federal investment in basic research.

Watch a video of the session.

Information Liberation: How Data Sharing Drives Medical Innovation

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

Expert panelists shared their thoughts on best ways to encourage pre-competitive data sharing for research from the vantage point of public sector, private sector, and nonprofit organizations. The panel was among the more than 150 panels at the Milken Institute’s Global Conference. They outlined areas of opportunities for collaborations to accelerate research and development timelines for drug and diagnostic development that would result in significant cost reductions for drug development.

“We [drug companies] compete in the marketplace on products. We don’t compete on science. We don’t compete on clinical endpoints,” said Garry Neil, corporate vice president, Corporate Office of Science and Technology, Johnson & Johnson.

With the expansion of genomics, the science of biomedicine itself is transitioning toward biomedical computational informatics and a systems understanding of biology.

Anna Barker, consultant, Transformative Healthcare Initiatives and former deputy director of the National Cancer Institute, noted that “every patient is different. But every medical record doesn’t have to be,” highlighting the need for a standardized approach to collecting and sharing patient information electronically.

However, the current academic science career system and culture does not incentivize or reward scientists for making data available or for engaging in team science.

Eslie Dennis, executive director, Polycystic Kidney Disease Consortium, The Critical Path Institute provided examples of how nonprofits could drive data sharing and spotlighted lessons learned from the Alzheimer’s Disease Neuroimaging Initiative, the largest public-private partnership to date in Alzheimer's disease research. It is led by the National Institute on Aging at NIH, through a grant to the non-profit Northern California Institute for Research and Education, with private sector support provided through the Foundation for the National Institutes of Health..

Funding agencies like the National Institutes of Health and nonprofit disease research organizations carry the ability to fund non-traditional, collaborative projects in academia and drive data sharing and standards in large science initiatives. Similarly, the private sector has increasing incentives to encourage data sharing that would reduce the attrition rate of failed drugs in phase 2 and 3 clinical trials that could be accelerated with a more efficiently designed clinical trials infrastructure. By collaborating with regulatory agencies like the FDA, data sharing can drive better science for risk assessment and support contemporary regulatory science applications for emerging technologies for biomedical innovation.

Panel was moderated by John Dwyer, chairman, Telcare.

Watch a video of the session

The Impact of an Aging Population

By Angelo Bouselli, Senior Manager of Communications, FasterCures

A couple hundred participants of the Milken Institute Global Conference packed into the Wilshire Ballroom for the session “The Impact of the Aging Population.” Paul Irving, senior managing director of the Milken Institute, moderated the panel and started with some facts for context: the 65 and older group is the fastest growing in the United States and faces numerous health challenges, including obesity, Alzheimer’s Disease, diabetes, and loneliness. Panelists agreed that this growing population offered economic and innovation opportunities, and that access and accountability were essential elements for this generation to thrive.

According to Mark Hodin of the Global Coalition on Aging, there are three components to understanding aging – longevity, low fertility, and the Baby Boomer bulge. “This subject is interesting because life longevity is new. This new middle age presents us with an opportunity to drive wealth creation and productivity around healthcare, research, learning, and creating new institutions that are right for the 20th Century.”

Marc Freedman of Civic Ventures said that social security was brilliant when it was introduced decades ago, and it worked for half a century. However, the traditional model of working for a long period of time and then retiring until the end of life doesn’t fit what many Baby Boomers today want. “Now what we need as a society and as individuals is the freedom to work. We really have to go back to the drawing board. We need a whole new set of social institutions.”

Paul Kusserow of Humana agreed that the infrastructure for the aging population needs to change. He warned about the current and growing problem of doctor shortages and said “The key is going to be access.” He saw the aging population as a “tremendous opportunity” for the emergence of electronic medical records and for innovation in care coordination. Sherry Lansing of The Sherry Lansing Foundation also saw aging as an opportunity for seniors to start something new and emphasized that seniors have an opportunity to “rewire, not retire.”

Redefining this stage of life and getting it right will change the whole picture of life, and all generations have a stake in getting this right. Irving closed the session by emphasizing that “we have a personal responsibility to get engaged in this issue.”

Watch a video of the session.

Monday, May 2, 2011

Purpose and Partners: Yielding Return on Medical Research Investments

by Kristin Schneeman, Program Director, FasterCures

A Global Conference panel of experienced funders, innovators, and analysts described first-hand the growing “Valley of Death” in funding the early stages of research and development of new treatments, explained the novel approaches they are taking to bridge the gap, and called for a complete rethinking of the financial models for funding medical research.

Ali Andalibi of the Small Business Innovation and Research grant program at the National Cancer Institute, Stephen Seiler, CEO of biotech company AesRx (which has benefited from a novel translational research funding program at NIH), and Lt. Col. Daniel Wattendorf of the Defense Advanced Research Projects Agency (DARPA) all represented efforts by the federal government to move beyond its historical role of funding basic discovery, to help push discoveries down the field toward becoming products in the marketplace. Catherine Arnold of Credit Suisse provided the viewpoint of the investment community, which is increasingly risk-averse and unwilling to invest in life sciences companies before the later stages of research.

DARPA is often cited as a role model for how government should fund research. Wattendorf ascribed this to DARPA’s “goal-orientation” rather than interest in contributing to scientific knowledge, its flat management structure and the complete authority of its program directors to control funding. Other panelists commented on the need for NIH to “take some DNA from DARPA.” DARPA is also teaming with FDA to look at regulatory science and whether there are ways to fundamentally change the development pathway that will accelerate progress.

Chris Elias of PATH, a public-private partnership to develop global health technologies, talked about what the global health field is learning about the Valley of Death: philanthropy and government capital can leverage company interest, particularly if it advances their platforms; and there is a significant role for nonprofits to invest in enabling platforms and to create standards to remove stumbling blocks for all parties.

Seiler pronounced the traditional biotechnology development model dead, noting that by 2009-2010 almost no preclinical research was being funded by the public markets. “We aren’t going to go back to the good old days, so what does the new model look like?” He also noted that what’s needed now is not just more sources of funding but “other management modalities – not just is this good science, but does the management know how to get there? This hasn’t been required before, we need to build those skills.”

Watch a video of the session.