Are we there yet? Navigating the Path through the Valley of Death

by Margaret Anderson, Executive Director, FasterCures

Characterizing the abyss separating basic and clinical research with tangible and intangible landmarks, we held our fifth annual blue-sky brainstorming session in partnership with Esquire magazine. FasterCures hosted a provocative and productive discussion last week on “Crossing the Valley of Death” with a group of thinkers and innovators from across the medical research spectrum. Participants represented large pharmaceutical companies, small biotechnology companies, venture capital funds, universities, and nonprofit foundations that fund research. There was broad agreement on the significant challenges we all face – scientific, financial, and cultural – in moving promising research across the valley and that we are at an inflection point where more action and less talk is required.

The objectives of the session were:

• To hear about new models of R&D collaboration and highlight lessons learned from them, and
• •To identify broader applications of existing models and opportunities for new collaboration mechanisms.
  

10 themes that emerged from the discussion:

1. A fundamental restructuring of the system is what's needed if the biopharmaceutical industry is to survive and thrive and patients are to benefit from innovation. The rest of the world is innovating outside the constraints of the U.S. system.
2. We need to redefine the process to make it cheaper, more sustainable.
3. We need to get beyond buzzwords like “open innovation” and “collaborative research,” and dig in to create standards for intellectual property, precompetitive research, collaborative contracting, or we will never be able to scale up models that exist.
4. We need to find ways to increase the output of valuable intellectual property, not just any intellectual property.
5. While we can’t predict the successful business models of the future, we can create the conditions that will allow for disruptive innovation.
6. We need to change the terms of the conversation – we need to talk more about capital efficiency and not stop at building capital, address the issue of patents but also really focus on productivity.
7. Decision-making in the biotechnology and pharmaceutical industries is not entirely rational or evidence-driven -- how can we change that?
8. There is a crying need for better communication to and understanding by the public and policymakers about the process and roles of the players in medical research. We need to elevate success stories of collaboration as a means to thread the needle more and create a fabric of innovation. It’s time these models transcend the patchwork of case studies and best practices and become the overarching approach that’s sorely missing.
9. We need to have the right incentives in place. Universities need to incentivize their faculty towards commercialization and collaboration with industry. They need to change their internal metrics of success. Federal and state policies need to create incentives for the results we want to see.
10. We need to rationalize the allocation of our resources -- money, time, and human capital.
    A variety of actionable suggestions, for FasterCures and other players, also emerged throughout the day. Stay tuned, we’ll summarize those in another blog post.
    

We will also be producing a meeting report on the topics that were discussed throughout the day. We expect the discussion to inform the program at this year’s Partnering for Cures conference in New York on December 14-15. And the action items will feed into FasterCures’ strategic planning for next year.

Most importantly, this was an opportunity to have a candid, honest discussion of where medical research is and where we need to go, among those steeped in the system but with the foresight and will power necessary to infuse life into the valley of death. Onward.

NIH Working Group Recommends Opening the Doors of the Clinical Center to External Investigators

by Gillian Parrish, Manager of Alliance Development and Communications

Last week, an eight member working group of the National Institutes of Health’s (NIH) Scientific Management Review Board (SMRB) issued recommendations for improving the fiscal sustainability and utilization of the NIH Clinical Center, which comprises almost 6,000 scientists and constitutes nearly ten percent of the NIH’s budget.

After over a year of deliberation – and consultation with dozens of internal and external stakeholders from research hospital administrators to potential external users of the Clinical Center to key NIH investigators and advisors – the group, chaired by University of Pennsylvania School of Medicine’s Executive Vice President Arthur Rubenstein, offered three core recommendations:

1. That the NIH Clinical Center expand its vision and role to serve as a state-of-the-art national resource for both internal and external investigator use.
2. That the governance structure of the Clinical Center be modified to facilitate the development and implementation of an overall strategic vision for clinical research, including eliminating oversight by the NIH Steering Committee and establishing a new governing board comprised of Institute and Center directors.
3. That the Clinical Center maintain a stable, responsive budget underpinned by priority setting and funded as a line item in the Office of the Director appropriation.

The working group’s recommendations in many ways echoed those set forth by a FasterCures’ task force chaired by Nobel Laureate Dr. David Baltimore, which in January of 2009 called for the IRP to adopt a new, more outcomes-focused mission that was capable of responding quickly to opportunities and challenges in translational research. At the time, the task force called upon NIH to articulate an overarching mission for the IRP and lay out a strategy for meeting goals over the next five years, focused specifically on advancing translational and clinical research in the interest of public health. It also suggested that the SMRB be tasked with reviewing options for funding the Clinical Center to enhance greater utilization and removing the current disincentives for use.

In May of this year – together with 86 other patient organizations – FasterCures again urged the SMRB to open up clinical center facilities to other researchers through a joint letter to the board.

As our nation’s crown research jewel, the Clinical Center features some of the greatest scientific minds using the most advanced medical technologies in the world. It ignites hope and has a distinguished history of discovery, yet remains underutilized due to fiscal and governance constraints. The workgroup’s recommendations chart a path forward for advancing the cause of clinical research, both within and beyond the agency.

We hope to see the NIH leadership act upon these recommendations and continue the focus on advancing translational and clinical research in the interest of public health. They have already demonstrated a commitment to integrating efforts, collaborating across sectors, and working together to meet the goal of getting therapies to patients faster.

Relevant FasterCures Resources:

• Patient Advocacy Letter to NIH Director Calling for Greater Access to the Clinical Center
• Recommendations from the FasterCures Task Force on the Intramural Research Program

A Connected, Collaborative Approach to Streamlining Clinical Trials in Children

Melissa Stevens, Director, Strategic Initiatives

According to the Centers for Disease Control and Prevention, at least 14 million children in this country have a brain disorder for which there is no treatment or cure. This figure represents 17 percent of children between birth and 19 years of age.

The Children’s Neurobiological Solutions Foundation (CNS) recently convened a meeting that brought together policy makers, researchers, nonprofit organizations, and industry to address the barriers to clinical trials and treatments for children affected by neurological conditions. We participated at that meeting.

According to CNS, when research into a potential new treatment advances to the clinical stage, obstacles arise. Among the reasons are that few clinicians are experienced in devising pediatric clinical trials for brain disorders, the potential risks of pediatric trials discourage institutional review boards and scare drug companies, and biomarkers or imaging technologies readily used for adult trials have not been adapted to pediatric populations.

CNS is hoping to create the Centers for Excellence for Pediatric Neurological Disorders, a network of locations across the U.S. focused on conducting pediatric brain clinical trials. Because of its specialty focus, testing of treatments for pediatric neurological disorders would be accelerated, significantly enhancing the possibility that laboratory discoveries are translated into safe and effective treatments. The proposed network would be organized and managed under several Centers of Excellence – a hub and spokes system that would be populated by academic child neurologists across the United States. The network would also be charged with:

1. Providing training for child neurologists and their physician and nurse
   colleagues in clinical trial design management, and interpretation;
2. Exploiting existing infrastructure for clinical trials design, execution, and,
   evaluation, as well as creating infrastructure where needed;
3. Evaluating emerging basic science discoveries as potential treatments;
4. With colleagues at the FDA and industry, assessing the feasibility of developing
   discoveries into drugs or other therapeutic modalities;
5. Enlisting international partnerships whenever necessary to advance the mission; and
6. Communicating the results of studies to colleagues and to patients and their
   advocates, including assembling a searchable accessible database.

At FasterCures, we are firm believers that expediting cures requires collaboration. It was affirming to hear the enthusiasm of participants at this meeting all eager to see this network come to fruition and deliver on the promise of a more connected clinical trial system for children with neurological conditions.

At the upcoming Partnering for Cures meeting, we are specifically spotlighting innovative, cross-sector collaborations that are advancing medical progress. A call for applications for innovator presentations is now underway. Our goal is to feature up to 30 of the most forward-thinking, transformative efforts. To learn more, visit http://www.partneringforcures.org/.

Why it’s Time for Congress to Write New Stem Cell Legislation

We need policy that allows the research to proceed, with federal dollars and with appropriate oversight.

By Margaret Anderson
Executive Director, FasterCures

On August 23, a federal judge blocked NIH from funding human embryonic stem cell research, ruling that the support violates the rider (the Dickey Wicker amendment) of the Health and Human Services appropriations bill written by Congress in 1996. The rider prohibits the use of taxpayer money for research “in which a human embryo or embryos are destroyed, discarded, or knowingly subjected to risk of injury or death greater than that allowed for research on fetuses in utero.”

In 1999, the Department of Health and Human Services General Counsel determined that this law does not prevent NIH from supporting research that uses embryonic stem cells derived—using private funds—from embryos destined to be destroyed by those no longer needing them for infertility treatment. That ruling was expectedly controversial but it set the wheels in motion for U.S. scientists to explore how these most versatile of human cells could be used to safely test new drugs, restore worn and torn tissue, and replace diseased cells with healthy cells.

In 2001, President Bush halted research on all but a few embryonic stem cell lines. Last year, President Obama lifted some restrictions, but only after a clear and publicly vetted set of guidelines was in place for proceeding with scientific work.

Although the private sector is always free to conduct this type of research, most agree that we need the rigor, stature, and transparency of federal funding for the field to move forward.

At the bench, there are many ways to read the intent of Congress. Judge Lamberth read the intent of Congress in a way that would prohibit federal funding for such research.

If you visit the NIH’s Stem Cell information page today you will read that:

“Pursuant to a court order issued August 23, 2010, NIH is not accepting submissions of information about human embryonic stem cell lines for NIH review. All review of human embryonic stem cell lines under the NIH Guidelines is suspended. The February 23, 2010, proposal to revise the Guidelines is also suspended.”

The impact of this decision cannot be understated. Millions of research dollars and the grant review process have been frozen. Policymakers on both sides of the aisle have spoken out in support of this area of research, given that specific safeguards are in place.

The public opinion on pursuing this research is clear. The scientific need to pursue these avenues is clear. If, as the judge in this case asserts, the 1996 amendment is in the way, then it needs to be reexamined.

A June 2008 Time magazine poll conducted by the SBRI research group found that 73 percent of Americans support embryonic stem cell research using cells derived from embryos about to be discarded by couples after infertility treatment. A majority of Americans support embryonic stem cell research as long as there are strict guidelines and systems of oversight in place, which there are.

In addition to extensive ethics review at NIH, every institution receiving federal funding for this research—that could mean the academic medical center down the street from you—has several committees in place to review this area of research to ensure it is conducted ethically and with only the highest scientific justification. Citizens sit on these committees, as well as scientists, physicians, lawyers, and ethicists.

The solution now is for Congress to craft a new policy that allows the research to proceed, with federal dollars and with appropriate oversight. In the absence of a legislative change, the lawyers will continue to battle, which will take valuable time, while patients and their families sit by helplessly, waiting for a political miracle.

What Metrics Matter Most in Nonprofit Medical Research? Options

The FasterCures Philanthropy Advisory Service wants your input on what matters most when evaluating nonprofit disease research foundations and their impact on the discovery and development of new cures!

Last year, we launched the PAS Web site as a resource for philanthropists looking to better understand and evaluate the R&D landscape, and strategically guide their investment dollars in high-impact areas. In our first phase, we evaluated 23 organizations using a set of metrics developed in consultation with experts in nonprofit management, organizational effectiveness, and medical research. This evaluation tool is designed to help measure the performance of nonprofit research organizations, looking at everything from accountability to collaboration to overall contributions to the field. Many of the metrics are based on effective practices identified
through our work with TRAIN organizations like yours.

Now that our first year is complete, we’re taking another look at the framework to learn how we can make it an even more effective tool—not just for use by philanthropists looking to evaluate investment opportunities, but also for organizations like yours that are seeking ways enhance your effectiveness. From operational processes to research effectiveness, tell us what measures you think matter most in evaluating nonprofit disease research foundations and their impact on the discovery and development of new cures.

Review our metrics and tell us how we can make them better and stronger. You can share your thoughts on this thread, by email to philanthropy@fastercures.org, or in the comments section on the Web link provided above. We appreciate your input and believe that, together, we can inform and increase the flow of philanthropic dollars to this critical field of research.

Welcome to the Station

For the last five years, FasterCures has been tracking and learning from an ever-widening circle of nonprofit foundations that fund medical research, an informal affinity group we dubbed TRAIN – The Research Acceleration and Innovation Network.

These groups have taught us a lot about the elements of innovation that could accelerate progress in curing disease. We have provided them with a unique venue for cross-disease and cross-sector networking and collaboration – most recently at Partnering for Cures, a first-of-its-kind event last December that brought together 600 individuals from the nonprofit, philanthropic, and industry sectors for two days of dialogue and formal partnering opportunities.

We’re now offering up this online hub, TRAIN Central Station, as the gathering place for a virtual community where individuals interested in maximizing the impact of philanthropic disease research funding can connect with one another to network, learn, inspire and share resources.

We’re calling the site “a platform for venture philanthropy in medical research.” We at FasterCures have generally subscribed to the broader definition of venture philanthropy that's represented in Wikipedia, that it "takes concepts and techniques from venture capital finance and high technology business management and applies them to achieving philanthropic goals." Others limit the term to mean only foundations that actually invest their funds in for-profit companies in one way or another.

What will you find here?

• Information about the strategies and best practices of innovative nonprofit funders of medical research;
• Useful tools and resources that can help you in your daily work;
• A venue for communicating with your peers to address practical challenges or debate important issues.

We’re launching the site in beta form and look forward to your feedback about what’s useful, what’s not, and ways to make this a more valuable resource for you.

Look forward to seeing you aboard!
Margaret Anderson