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.

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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/.