Monday, March 30, 2009

You Can Take That to the Bank!

By Angelo Bouselli, Communications Manager, FasterCures

It’s one of the most-awaited magazine covers of the year: when Time magazine announces its so-called “Person of the Year.” Every December, the editors of Time devote an issue to the person who “most affected the news of our lives, for good or ill, and most embodied what is important about the year.”

The folks at Time, in keeping with their long-held tradition of list-making, recently compiled a list of “10 Ideas Changing the World Right Now.” Coming in at number 8 – the National Cancer Institute (NCI) and its efforts to establish the United States’ first national biobank.

With the global economy being reshaped before our eyes, Time Magazine highlights “10 Ideas Changing the World Right Now.” At number eight on the Magazine’s list… Biobanks! Time devotes this slot to the scientists at NCI who are heading up an effort to establish the United States first national biobank.

Established in 2005, the mission of NCI’s Office of Biorepositories and Biospecimen Research (OBBR) is to “guide, coordinate, and develop NCI’s biospecimen resources and capabilities and ensure that human biospecimens available for cancer research are of the highest quality.” The NCI biobank will be a safe house for tissue samples, tumor cells, DNA, and blood, which will be used to for research into new treatments for diseases. OBBR hopes to have mapped out a plan for a national biobank by fall 2009, but the recent stimulus could accelerate that timetable.

The OBBR biobank is an organic bank account, in which biomaterial deposits earn interest in the form of knowledge and therapies. There’s no monetary reward -- just the potential that you might benefit from the accumulated data at some later date. This knowledge is the very reason FasterCures launched BioBank Central in 2006. Biobank Central is a Web based portal to connect researchers to resources, encourage the donation of specimens, and educate the public about the benefits of research on banked biospecimens. It also serves as a timely source of news and information about biorepositories and their role in medical research and development.

In 2009, BioBank Central launched a new feature, Spotlight on Innovation highlighting individuals and organizations doing exceptionally innovative work in biobanking. Our first Spotlight focused on the Susan G. Komen for the Cure® Tissue Bank at the Indiana University Simon Cancer Center. This bank’s mission is to collect samples of normal, healthy breast tissue and other biospecimens from healthy women, providing a baseline for breast cancer research. Biobanks are changing the world – one cell at a time.

For more information on biobanks visit BioBank Central.

Tuesday, March 24, 2009

FasterCures is Stopping TB

By Loren Becker, Global Health Program Analyst, FasterCures

Today, as health workers and communities globally observe World TB Day, 25,000 new people will be diagnosed with tuberculosis and nearly 5,000 will die from the disease. Most TB sufferers are adults in their working years, costing the world about $12 billion in economic productivity each year. Many more also are infected with HIV/AIDS, compounding an already massive humanitarian crisis, particularly in sub-Saharan Africa. The slogan for World TB Day is “I am stopping TB.” Unfortunately, TB control efforts are unable to keep up with the epidemic: a new report released by the World Health Organization this week tells us that the number of new cases and deaths continues to grow with each passing year.

Many factors contribute to the severity of the global TB problem, not least of which is that the tools we have to fight the disease are antiquated and inadequate. In a world where we can splice genes and grow new tissue out of stem cells, very little attention is paid to improving the options for patients infected with this disease that has long since ceased to be a concern for most of us in the developed world. As a result, health workers in the poorest countries are stuck with a vaccine that is over 80 years old and only protects children, drugs that are over 40 years old and difficult to stomach, and diagnostics that frequently fail to give clear, actionable results. Harsh treatment regimens frequently lead to noncompliance, which, in turn, lead to drug resistance. Increasingly, health workers are confronting strains of TB bacteria that are able to resist some or even most of the available drugs.

Despite the dire statistics, TB experts see room for optimism. As awareness of the global epidemic has grown, so has funding and attention from governments, foundations, and other sources. In the last decade, several new initiatives have joined the fight to control TB and to develop better tools to aid future efforts. Researchers within some of these groups are making key contributions in terms of expanding our basic scientific knowledge about TB, as well as translating that knowledge into improved vaccines, drugs, and diagnostics. The entrance of these new players has led to exciting advances and expanded the opportunities for those concerned to get involved in stopping TB.

Many of the new research groups, as well as their more established counterparts, are nonprofit organizations that receive most or all of their funding from governments and foundations. Private philanthropists also can, and do, play an important role in funding research to develop and deliver new tools in the fight against TB. In order to help these donors understand the field of TB research, the major nonprofit players, and targeted areas of philanthropic R&D investment, the FasterCures Philanthropy Advisory Service is including TB as one of four diseases covered during the project’s pilot phase. The TB module of our Philanthropy Advisory Service is expected launch this June. Through this effort, FasterCures is stopping TB.

Monday, March 16, 2009

You Get What You Pay For?

By Melissa Stevens, Director of Special Projects, FasterCures
You wouldn’t choose a restaurant based on how little it pays its chef, or a surgeon based on how cheaply the hospital retains his services. So why would you choose a charity based on how low its overhead is?

Dan Pallotta is the author of the newly released Uncharitable: How Restraints on Nonprofits Undermine Their Potential. Pallotta spoke at a Milken Institute Forum on February 11 about his new work, which is described as “a manifesto that puts a new cause on the map - equal economic rights for charity.” He addressed the compensation disparities between for-profits and nonprofits, and challenged long-held conventional wisdom that overhead is a reliable measure of effectiveness.
Pallotta argues that charities are pressured into playing by different rules than for-profits, and that these pressures can stifle the organization’s productivity.
  • Compensation – Salaries for nonprofit executives are frequently criticized as too high. When nonprofits are compelled by external forces to tamp down executive compensation, recruitment quality is the first casualty.
  • Advertising – Mass marketing is considered wasteful and frivolous in the nonprofit world – a costly and shortsighted judgment in the era of the multimedia-driven, 24-hour news cycle. The refusal to play in the mass marketing arena stifles charities’ ability to capture market- and mind-share, and cedes the stage to campaigns about the latest soft drink or i-product rather than malaria eradication or ending hunger.
  • Vision –Nonprofits are incentivized to spend funds in the near-term to make an immediate impact, leaving them unable to invest in long-term, strategic initiatives, and sacrificing long-term gains for short-term returns.
  • Learning – Nonprofits are given very little room for error in the court of public opinion. The climate of hard judgment creates disincentive for creativity and risk-taking. A risk-averse entity will never reach its full potential.
  • Capital – Nonprofits are locked out of the financial markets because they do not pay financial returns. This leaves them continually dependent on what FasterCures’ calls “passion capital,” which only promises social returns.
Pallotta also articulated his frustration with the habitually asked “What percentage of my donation goes to the cause and how much goes to overhead?” Pallotta asserts that overhead actually is part of the cause. Investment in management and infrastructure are critical aspects for ultimate success, and should not be treated as incidentals. Further, he noted that measuring overhead does not give donors sound information about outcomes and the impact made by the organization.

Pallotta calls for developing a metric for assessing charitable giving. FasterCures agrees with this approach, and is leading the charge in the biomedical research space with the Philanthropy Advisory Service (PAS), an information resource that aims to create a transparent marketplace about nonprofit disease research organizations that fund and facilitate disease research. The PAS organizes and analyzes information about strategy, research portfolio, management, and financials. Even more importantly, the PAS creates an assessment framework to evaluate an organization’s internal and external contributions to the field. These assessments are made based on the organization’s own mission statements, as well the unique nuances of the disease research system.

The PAS framework organizes our assessment around the following metric categories:
  • Accountability: The degree to which an organization engages in planning, demonstrates transparency, and upholds responsibility to stakeholders.
  • Collaboration: The degree to which an organization can engage and nurture relationships that accelerate the overall funding and research cycle.
  • Research Effectiveness: The degree to which the organization’s research portfolio yields sufficient data and deliverable returns to achieve its stated mission.
  • Resource Building: The degree to which the organization contributes critical resources and infrastructure to scientific advancement.
FasterCures has engaged scientific and organizational experts to provide input to the organizational assessments, ensuring an informed, credible, and objective review. The PAS is currently being piloted across four disease areas – Alzheimer’s disease, multiple sclerosis, malaria, and tuberculosis – and is targeted to launch later this spring. FasterCures is excited about the PAS and its contribution to a new paradigm of nonprofit evaluation – the overhaul on overhead is long overdue.

Friday, March 13, 2009

Is the HIPAA Privacy Rule Impeding Research?

FasterCures invites you to a special briefing and discussion
Wednesday, March 25, 1:30 pm – 3:00 pm

  • Marc Boutin, Executive Vice President & Chief Operating Officer, National Health Council; Institute of Medicine (IOM) Committee Member
  • Sharyl Nass, Study Director and Senior Program Officer, IOM
  • Greg Simon, President, FasterCures
Biomedical researchers use health information from multiple sources including patient registries, medical records, biobanks, and government-supported databases of statistics. Ten years ago, Congress called for a set of standards known as the HIPAA Privacy Rule to protect the privacy of patient medical records held by covered entities, which are often used in such research.

But has the HIPAA Rule compromised both patient privacy and medical discovery? A recently released IOM report, Beyond the HIPAA Privacy Rule: Enhancing Privacy, Improving Health Through Research, examines HIPAA one decade later, and finds that these federal standards may be due for an overhaul, to serve the interests of progress as well as patients. Come join us to hear about the report's findings, and to discuss what happens next.

Please join FasterCures for this timely discussion on: Wednesday, March 25, 2009
1:30 pm – 3:00 pm
1101 New York Avenue, NW, Suite 620 Washington, DC 20005
Space is limited, please RSVP to Patricia Wolf at or 202-336-8917, by COB Monday, March 23, 2009.

Monday, March 9, 2009

FasterCures Applauds Stem Cell Ban Reversal

FasterCures applauds today’s executive order by President Barack Obama to lift the eight-year old ban on federal funding for research on new stem cell lines. President Obama’s order marks the reversal of the August 2001 decision by the Bush Administration banning federal funding for all stem cell lines created after that date.

Today’s order marks an important step toward exhaustively exploring every possible avenue in the search for new treatments for deadly diseases. Polls demonstrate that more than 70 percent of the U.S. public supports federal funding for stem cell research. This reversal is a long overdue measure that moves toward the ultimate goal of all medical research: saving lives.

FasterCures president Greg Simon weighed in on the reversal: “President Obama has taken politics out of science and put science where it belongs – in the forefront of the fight to defeat the diseases that are taking our loved ones before their time. We cannot afford to let ideology trump ideas rhetoric defeat research. FasterCures applauds the President’s decision, and urges the NIH to move with deliberate speed and care to develop and finalize the new guidelines.”

Biobanks Going Mainstream

By Kate Blenner, Program Analyst, FasterCures
At FasterCures, we believe biobanks are critical resources for biomedical research and personalized medicine, which is why we were so pleased to see significant advances in the field at the end of 2008. In December, two major regional biobanks were launched with significant investment of resources and big goals for donor recruitment, and a national biobank has been mentioned in legislation and is being explored by the NIH. But even as biobanking is embraced by health systems, researchers, legislators, and funders, will the field be ready to meet the expectations of patients and donors?

The two new banks announced in December will be housed at Mayo Clinic and at Kaiser Permanente (Kaiser’s bank is funded by the Robert Wood Johnson Foundation.) Both banks will attempt to link samples to information stored in the donors’ medical records, and both aim to use the DNA samples to study how genetics, environment, and lifestyle contribute to the development of disease and the effectiveness of treatment and preventive measures. They will also both rely on the voluntary participation of tens of thousands of donors—Mayo plans on building a bank of 20,000 or more donor samples, and Kaiser Permanente hopes to collect as many as half a million.

A recent study conducted by the Johns Hopkins University Genetics and Public Policy Center (GPPC) suggests recruiting these numbers of participants may not be as daunting as it sounds. GPPC conducted a public engagement study on behalf of the National Institutes of Health to assess attitudes and concerns about a proposed national biobank. Researchers found that most (84%) of survey respondents reacted favorably to the proposed bank, and 60% said they would be willing to donate. Interestingly, when participants were offered different incentives to participate, it was the feedback of research results to the donor that motivated participants most—even more so than monetary compensation. What’s more, 75% of survey respondents said they would be less likely to participate if they were unable to receive research results generated from their samples. (The findings of this study are summarized in the November issues of The American Journal of Bioethics and Genetics in Medicine. )

Returning research results to genetic study participants is a topic of significant debate; while there is much literature regarding the return of medically relevant results from clinical trials to participants, research data from genetics studies is difficult to assess for clinical relevance because of the often tenuous connection between genotype and clinical outcomes. The pioneering studies providing feedback to participants often do so on a case-by-case basis, with laborious review and deliberation by the researchers and IRB (the Genetics and Pharmacogenetics of Airways Diseases project at Partners Healthcare is one example of a study providing limited, case-by-case feedback to participants.) Clarity and guidelines on this issue are sorely needed.

But the good news is, where there is consumer demand, there is a vacuum waiting to be filled—if participation in studies is contingent on information feedback, researchers and study sponsors of large and ambitious banks will have to find a way to provide it. As consumer awareness and engagement grow, we could see not only increased participation and funding to large-scale genetic studies, but new channels of feedback, more sophisticated models of consent, and appropriate integration into care processes to meet the demands of a more informed patient population—if we do it right. The GPPC study and the initiation of two ambitious new banks show that funders are willing to invest resources and patients are willing to ‘invest’ samples, but of course a return is expected on any investment for support to be long-lived. The will is there—it is up to the biobanking community to find a way.

Thursday, March 5, 2009

Do Not Spend the Lion’s Share of the Stimulus Money for NIH on Traditional “RO1” Grants

By Greg Simon, President, FasterCures

There I said it.

You would think we learned that lesson during the doubling of the NIH budget when NIH went farther down the payline without necessarily a commensurate increase in positive outcomes for patients. But apparently not.

In the transition planning for NIH (where I was a part of the Agency Review team) we met with all sorts of groups about NIH’s future. And while there was considerable support from the research community and universities for more RO1 grants, since the NIH budget has declined in real terms for several years, there also was considerable support from patient groups for new approaches to research and training that focused on translational research and new public-private partnerships to help move discoveries toward new and better treatments. So, how to decide?

FasterCures Task Force on the NIH Intramural Research Program (led by Nobel Laureate and FasterCures Board member David Baltimore) noted that the NIH’s world-class research hospital in Bethesda, the Clinical Center, is held back by a dysfunctional funding approach. We proposed a dedicated budget for the Clinical Center, so groups and consortia with the best ideas for clinical trials could perform them there. Meanwhile, other world-class programs such as the Chemical Genomics Center, the Cancer Genome Atlas and caBIG (cancer Bio-Informatics Grid) have demonstrated their potential to radically transform our approach to – and our success in treating – deadly diseases.

But as usual, the battle cry is show me the money – spend it in traditional ways at traditional places for traditional outcomes, which tend to be cautious and incremental. Well, I say, Sow me the money. Let’s sow the future of medicine with the stimulus money, let’s not sprinkle it over the depleted fields of traditional approaches that are no longer the most fertile ground for innovation.
According to Acting NIH Director Ray Kington at a recent briefing at the American Association for the Advancement of Science (AAAS), 8.2 billion of the 10.4 billion dollars will go to the institutes and centers at NIH to spend. They will spend it in three ways:
  • Through R01 and related mechanisms for already approved but unfunded grants. These will be for two years of funding rather than the four that are typical, and projects will need to demonstrate potential for significant scientific advances in two years.
  • Through supplementing existing grants
  • Through new NIH Challenge Grant Programs with areas or "themes" to be identified in concert with the Institutes and Centers. These grants will be $500K/yr for two years, with an expected total yet to be determined but expected to be around $100M-200M.
A modest proposal: of the 8.2 billion dollars allocated to the centers and institutes at NIH, spend only the original 3.2 billion requested in the House bill for RO1 grants and spend the rest on translational awards and challenge grants that support translational and clinical research and researchers. Why? When you fund an RO1 grant, things happen. People hire lab assistants, they buy equipment etc. But when you fund translational programs, more things happen because you can leverage other people’s money. Instead of aiming only to create jobs now, you sow jobs and industries of the future that grow from commercialization of new treatments for disease.

Now, NIH is quick to mention that the money has to be spent in two years and for that, clinical research may suffer. But no one is talking about funding a Framingham study. I’m talking about sequencing more tumors, screening more compounds and doing clinical trials that are ready to go and can be done in two years. If we don’t think anew we can’t renew – and President Obama has made it clear that he is talking about renewing America, not “revisiting” it by doing what we have always done the same way we have always done it.

NIH has to prepare a plan for the stimulus money to submit this month. Those of you who support translational research and have always been told there is no money, listen up. There’s money. Is there a vision? If not, we’ll have no one to blame but ourselves.

Monday, March 2, 2009

Living From One Discovery to the Next

By Kristin Schneeman, Program Director, FasterCures
“Comparative effectiveness” It sounds like a no-brainer: We should study the effectiveness of medicines for treating a condition and use the ones that are proven to work best. But as the drumbeat around this buzz-phrase gets louder, we need to make sure we understand its implications for patients’ access to treatments that work best for them, and for incentives to pursue new and even better and potentially more personalized therapies.

It’s a pretty tall order to make this issue accessible to the average American, but the Partnership to Improve Patient Care – a coalition aimed at educating consumers and policymakers about comparative effectiveness – may have found the perfect spokesperson in former NFL linebacker Eli Alexander. Alexander spoke at a Partnership event in Washington last month about what happened after he was diagnosed in his thirties with multiple myeloma, an incurable blood cancer.

The only available drug for treating Alexander at that time was Thalidomide. While some patients tolerate it well, its side effects in his case were extreme – excessive fatigue, loss of sensation in his hands and feet, muscle atrophy, extreme weight loss. “I played football for 28 years and walked away from the game healthy. After six weeks on thalidomide, I lost a quarter of my body mass. At 37 I was facing being a burden to my wife and children. I would no longer be able to work, run my business. I would have had to become disabled, become a tax burden. Thalidomide was killing me – it was taking care of the cancer, but it was killing me. I’m not really concerned about being alive, I’m concerned about living. It’s hard to put a cost on that.”

The only other alternative treatment, a stem cell transplant, went well, but ten months later his myeloma reappeared. Fortunately, 45 days before his recurrence, the FDA had approved the drug Revlimid for myeloma. It has worked extraordinarily well for him, without the debilitating side effects. It is significantly more expensive than thalidomide, but he has been able to lead a productive life. “I’m here today because Revlimid worked for me. It doesn’t work for everybody, but it worked for me. I’m able to do the things I retired to do – be there for my wife, coach my kids’ football. I can write, I can walk.

“There’s no cure for the disease I have. I don’t know how long Revlimid will work for me. We live from one discovery to the next.”

At the beginning of the event, which came a few weeks before the Super Bowl, Alexander jokingly reminded the audience that football is just a game. Battling cancer is clearly not a game, and patients need to have every available weapon in their arsenals.