Monday, August 30, 2010

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.

Thursday, August 19, 2010

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.

Monday, July 26, 2010

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.

Tuesday, July 6, 2010

A Critical Need for Cures Acceleration

How appropriations can make a crucial difference in advancing medical progress

The Cures Acceleration Network (CAN) – authorized through the Patient Protection and Affordable Care Act or health reform law – will allow the National Institutes of Health to seek out and support biotech companies, universities, and patient advocacy groups that are pursuing promising, innovative research. CAN promises to establish a new funding mechanism that promotes outcomes-driven research. As authorized, CAN would have a budget of $500 million to accelerate the development of “high need cures,” defined as drugs, biological products or devices:

  • that are a priority to diagnose, mitigate, prevent, or treat harm from any disease or condition, and
  • for which the incentives of the commercial market are unlikely to result in adequate or timely development.

But without appropriations, CAN will remain an unrealized promise.

We urge all in the patient advocacy community to support establishment of CAN within the NIH Office of the Director, as called for by the health reform law, with appropriated budget of $50-$150 million. Opportunities to change the trajectory of medical research across sectors and for all diseases do not come often. Consider reaching out to Members of House and Senate Appropriations Committees and their staff to voice your support for the Cures Acceleration Network

Thursday, June 3, 2010

Building a 21st Century FDA: Getting new treatments out of the pipeline and into the hands of patients requires more than just good research

By Gillian Parrish, Manager, Alliance Development and Communications

Every day, hundreds of thousands of lives are indefinitely on hold – patients waiting for effective treatment options to become available, families waiting for their loved ones to start having a better quality of life, the list goes on. At a standstill – and that’s if they’re lucky – because research that promises to curb their disease has yet to translate into new drugs or devices they can utilize.

Often, when a new molecular entity or biomarker is discovered that has promising implications for disease treatment, it gets waylaid for years in a needless web of outdated regulatory processes that are under-resourced and ill-equipped for modern scientific review. According to a report by Nature Reviews Drug Discovery, the number of new molecular entities approved by the FDA has dropped steeply in the last 15 years – from 53 in 1996 to 19 in 2009, over a period when the budget for National Institutes of Health (NIH) nearly doubled. And most patients living with serious illness can’t wait the 10-15 years it takes to move a new discovery through the current development, testing, and regulatory review process.

While increasing funding for NIH research is a critical step in finding cures, it’s only part of the equation. The remaining part depends on improving the regulatory process, building capacity—both financial and scientific—at the Food and Drug Administration (FDA) so it can carry the baton of innovation from the research community across the regulatory “finish line” to patients. One vehicle for speeding review and access is the Prescription Drug User Fee Act (PDUFA), which is due for its fifth Congressional reauthorization in 2012.

The advocacy community’s role in reauthorizing an effective PDUFA was the focus of a panel hosted by BIO and PhRMA last week. Panelists Marc Boutin (National Health Council), Marcie Bough (American Pharmacists Association) and Jeff Allen (Friends of Cancer Research) called upon advocates to step up and be disruptive, working collaboratively with each other and with industry to speed product reviews and ensure the right balance between safety and access. Margaret Anderson (FasterCures) who moderated the discussion summarized the key themes at the end of the briefing:

  • Making sure the patient perspective is heard throughout the reauthorization process
  • Applauding FDA’s efforts to be more transparent, and taking them up on their willingness to have a two-way dialogue
  • Working together across sectors and diseases to find and leverage commonalities like:
    - Improving REMS – Creating a standard Risk Evaluation and Mitigation Strategies (REMS) template, involving patients in the evaluation process, and establishing clearer metrics
    - Incorporating Regulatory Science – Though user fees aren’t typically applied to scientific programs, reviewing drugs is a complex process that requires a complex way of thinking, and process science should be incorporated into PDUFA to increase efficiencies
    - Advancing Public Education – Helping patients and consumers understand what FDA can do for them, and mobilizing them to act
    - Balancing Fees and Appropriations – The appropriated base of human drug review has not kept pace with FDA’s workload (currently ~65% is paid for by user fees); a consistent, multi-year funding approach is necessary to operate a modern-scientifically based regulatory program