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.

Summer, Camp, Kids, Cancer

By Margaret Anderson

While we focus on improving the efficiencies of the system that discovers treatments and cures for disease, there are untold numbers of people taking a medical treatment journey right now.

For the kids partaking in the 28th year of Camp Fantastic in Virginia this week, they get to focus more on the fun, and less on the challenges of coping with a cancer diagnosis and with treatment. Camp Fantastic is a program of a nonprofit called Special Love that gives cancer families support.

I learned of this amazing place from Kathy Russell who has been involved since its beginning and who also runs the Children’s Inn at NIH. The NIH Children’s Inn helps families with kids in treatment at the NIH Clinical Center get a bit of normalcy in their lives by providing a warm atmosphere for them to stay in versus an isolating hotel room. The overriding philosophy is that families make a key difference in the lives of their sick children. The work they do represents the full spectrum of NIH investment – from bench to bedside.

By the end of my chat with Kathy, after I dabbed my eyes, I was ready to pack my bags and tell everyone I knew to come with me to help prepare Camp Fantastic and allow kids there to take a break from cancer and be just kids. This year there will be nearly 100 kids at Camp Fantastic between ages 7-17. Usually one-half of them are in active treatment and there are upwards of 60 medical professionals (in addition to countless other folks) who volunteer their time before and during the camp. They literally set up a mini-hospital on-site because it’s in a remote location and far from a hospital with specialized pediatric oncology services. She told me of how kids get their bloodwork done in the am, and a van takes the samples into NIH to do labs and then turns back around with results and medication.

Every year, at least one child is usually transported from the camp in an ambulance or a helicopter to a hospital for further treatment, requiring diligent attention to medical details as well as a carefully thought through psychosocial plan of action to share that news with the other campers. Families are communicated with each day, and as you’d imagine many are nervous and excited about their kids being there. Some children participate while in their final stages of life. Their families make the ultimate sacrifice by being selfless enough to share their children with others, so that their kids can live out their final hopes and dreams – the same dreams we all have. To do the things we dream of doing, especially those seemingly simple things associated with summertime.

Stories like this remind me of the passion and dedication of the medical research community, of the care providing community, and of the volunteer community. It’s a reality check for me, and for those of us who work on policy-related issues. These kids and their families point out the obvious – that time is of the essence in all we are doing to get to faster cures.

It can be hard to make the FasterCures message personal at times as we deal with mostly macro-level issues, but hearing about Camp Fantastic reminded me why we do what we do. Because it’s summer vacation time, and every kid (and for that matter, every grown-up) deserves a shot at creating their own lazy crazy hazy days of summer memories.

Join Us at Partnering for Cures 2010

Be a part of an effort that brings together people with the expertise, experience, and creativity needed to transform the medical research system. Join us at Partnering for Cures to be held December 14 and 15 in New York.

This year’s program will feature cutting-edge discussions about key topics in medical research – from translational research to data frameworks, from regulatory science to new business models to accelerate therapeutic development.

Among the speakers will be medical research decision and policy makers, high-profile philanthropists, leaders of innovative nonprofit foundations, forward-thinking industry executives, and scientists engaged in some of the most ground-breaking research on the globe. Confirmed speakers include:

• Margaret Hamburg
  Commissioner, U.S. Food and Drug Administration
  
• Carolyn M. Clancy
  Director, Agency for Healthcare Research and Quality
  
• Delos (Toby) Cosgrove, M.D.
  Chief Executive Officer and President, Cleveland Clinic
  
• Jesse Dylan
  Founder, Lybba.org; Filmmaker, Creative Director and CEO, FreeForm
  
• Maria C. Freire
  President, The Albert and Mary Lasker Foundation
  
• Stephen H. Friend
  President, CEO, and Co-Founder, Sage Bionetworks
  
• Jeff Hammerbacher
  Vice President, Products, Cloudera
  
• Isaac (Zak) S. Kohane
  Harvard Medical School, Children's Hospital Medical Center
  
• Edison T. Liu, M.D.
  Executive Director, Genome Institute of Singapore
  
• Susan Love
  President, Dr. Susan Love Research Foundation
  

The Partnering for Cures agenda will feature:

• One-on-One Partnering Meetings. Connect with nontraditional allies to explore collaboration and develop new strategies.
  
• Therapeutic Affinity Roundtables. Engage with leaders in your field through focused, informal networking opportunities.
  
• Innovator Presentations. Learn first-hand about multi-sector collaborations, novel research portfolios, and effective partnerships at dozens of sessions featuring innovators on the frontlines.
  
• Hot-Button Panels. Participate in dynamic, candid, cutting-edge discussions about key topics in medical research including translational research, shelved compounds, data networks, and many more.
  
• Expert Consultations (new addition!). Access technical and scientific experts on-site to help you address some of your organization’s mission-critical challenges.