Tuesday, June 30, 2009

Clinical Research should be Part of EHR "Meaningful Use"

On June 26, 2009, FasterCures submitted comments on the proposed definition of "meaningful use" of electronic health records, urging the Office of the National Coordinator for Health Information Technology to ensure that meaningful use requirements for EHRs address support for clinical research at the earliest possible time. FasterCures' comments include:
  • FasterCures has long advocated that one of the highest and best uses of a nationwide health information infrastructure – and one of its most underappreciated – is in accelerating the search for new and better treatments for disease. As the healthcare system addresses the challenges of widespread adoption of EHR systems, research capacity must be part of the architecture. Given the simultaneous priority the federal government has placed on comparative effectiveness research, we feel that this imperative cannot be overlooked or delayed.

  • A truly effective nationwide health information network will require that a single set of standards be developed and adopted for the collection and exchange of data across all health communities, including the clinical research community. If a common set of standards for key information is not required from the beginning (in this case 2011), it will be much more difficult to require these downstream.

  • One of the widely agreed-upon objectives of meaningful use is clearly to provide clinical decision support at the point of care. Clinical decision support is informed by research, and it is therefore important to include an opportunity to support clinical research through EHRs in 2011 (with standards that will be available before 2010). Similarly, if one of the stated objectives of meaningful use is “aggregating clinical summaries from multiple sources available to authorized users,” standards and controlled terminology are essential for such aggregation if it is to be useful. These should be required in 2011 so that this 2015 goal can be realized.
For more information, download a copy of the FasterCures publication, "Think Research: Using Electronic Health Records to Bridge Patient Care and Research"

Monday, June 22, 2009

Just in time for July 4th, FasterCures signs the Declaration of Health Data Rights

by Kristin Schneeman, Program Director, FasterCures

What is the most important ingredient in the recipe to cure cancer, or Alzheimer’s, or Lou Gehrig’s disease? Battalions of highly-educated doctors and Ph.D.’s? Buckets of cash? An armament of high-tech lab equipment? The answer is none of the above – it’s you. All of us. Patients.

Each of us contains the information necessary to unlock the relationship of genetics, proteomics, behavior, nutrition, and environment to the emergence and, ultimately, the management of diseases. That information is contained in the data in our medical records; biological material such as tissue, blood, and DNA; and our biology as observed in clinical trials. By enrolling in clinical trials to test potential new therapies, providing tissue and blood samples, and allowing researchers access to our medical histories with appropriate safeguards, patients provide the critical information and resources without which the search for cures would grind to a halt.

Patients are also an increasingly important driving force in setting the medical research agenda and providing the capital needed to turn promising ideas into new treatments. What started as fundraising telethons and walks is rapidly expanding to patient-oriented groups using social media to conduct virtual clinical trials of experimental treatments; foundations participating in the design of trials that meet patients’ needs; and patient groups providing seed funding to companies to incent them to develop drugs for their particular disease.

In the words of a recent Forbes magazine article, “Patients are the new power brokers.”

A strong democracy needs informed, thoughtful, and active citizens. A strong healthcare and “health cure” system requires informed, thoughtful, and active patients – especially now, as the health system undergoes major reform, and as the use of information technology and electronic health records is being ramped up to improve the system.

FasterCures is pleased to join with other organizations signing on to the Declaration of Health Data Rights, which state that individuals have the right to their own health data, to know where data in their record has come from, to take possession of it in paper or electronic form, and to share it with others as they see fit. We hope this is an important part of a movement to empower all of us to become more engaged with our health, our care, and our cures. It can’t happen without us.

Wednesday, June 17, 2009

Lives and Livelihoods are on the Line

by Margaret Anderson, COO, FasterCures
For the past three weeks, the nation's policymakers have turned their attention to healthcare. It seems like each day featured a seminal study, a pivotal finding, a fresh perspective. National statistics and budget forecasts are strengthened by stories from the frontlines. Patient-centered care. Comparative effectiveness. Cost. Private vs. public plans. Government option. Access. Quality. Affordability. Incentives. Health reform's steady drumbeat is at its crescendo.

We at FasterCures stand at the ready to support efforts that hold the potential to fixing what we all know is a broken, unsustainable healthcare system. Our medical research enterprise and the life sciences industry - the forces behind medical innovation and progress - are at stake. Today, we join other health advocates in an effort to ensure that the health reform conversation continues to focus on improved health and an opportunity to gain access to care for individuals, families, and communities. Forty-five million Americans without health insurance is not just a factoid, it's the reality that face Americans whose lives and livelihoods are on the line.

Fixing America’s broken healthcare system is a crucial step toward fixing America's broken healthcure system. Only if we translate promising scientific research into new treatments will we have any hope of reducing healthcare costs, productivity losses, and human suffering.

Tuesday, June 16, 2009

A Cure for What Ails FDA

by Margaret Anderson, COO, FasterCures

No agency is asked to do more, with less, than the Food and Drug Administration. It regulates 80 percent of U.S. food supply, every drug, vaccine, and medical device, consumer cosmetics, and could potentially now have a hand at regulating tobacco products. One entity is charged with ensuring the safety of everything from spinach to statins, produce to pacemakers, totaling over $1 trillion in consumer products; 25 cents of every dollar consumers spend in the U.S. every year. No government entity is more important than FDA to the daily lives – and safety – of Americans.

Unfortunately, the importance of the FDA is often lost in the din of political arguments over health reform. But no one is well-served by omitting FDA reform from the health reform debate; indeed, it could be argued that both the health and welfare of every American is guarded within FDA’s walls. The healthcare debate should begin with FDA. Yet, we rarely hear about FDA unless there’s a recall, or a salmonella outbreak, a political skirmish over a “controversial” drug, or when its already stretched scope is further expanded. We hope that the newly formed Transparency Task Force, in its effort to make more information available and foster better understanding of decision-making within the FDA, will also shed light on the breadth and depth of the agency's scope.

Every day, far too many patients receive diagnoses that are tantamount to death sentences, and every day, researchers uncover promising new treatments. But before any treatment can reach a patient’s local pharmacy, it must first go through the approval process. If you have a system based on rigorous standards that doesn’t have enough resources to meet those standards, either science or safety suffers.

Last February, former FDA chief counsel Peter Barton Hutt warned a Congressional panel of an agency that was “barely hanging on by its fingertips.” His comments were followed by testimony of several other experts, all of whom agreed that the agency has neither the financial nor human resources to do its job. Indeed, the FDA’s own science board arrived at the same conclusion.

We hope that the Senate’s swift voice vote approval of Dr. Margaret Hamburg as Commissioner indicates a renewed commitment to strengthening the FDA. It’s a start, and not an insignificant one. But for the FDA to maximize its effectiveness, further steps must be taken.

Once regarded as the regulatory gold standard of the world, the FDA has a way to go to come back. A New England Journal of Medicine piece co-authored by Hamburg and Deputy Dr. Joshua Sharfstein outlines the call to action. We agree with their assertions, and add these as our own:

FDA needs consistent, strong leadership. We have every confidence that Drs. Hamburg and Sharfstein will meet that overarching need. Just as important, FDA needs political and financial support from both the White House and Capitol Hill. FDA also needs to redefine its mission, streamline its institutional portfolio, and bring its IT systems into the 21st century. An updated mission, and the infrastructure to achieve it.

For too long, medical research has not been a political priority, and the tide of progress has been stemmed because of it. The FDA, charged with protecting 300 million people, has a budget that mirrors that of the school budget in Montgomery County, Maryland. Good news for Montgomery County; less so for the rest of us. FDA needs increased appropriations from Congress, especially for initiatives such as the Reagan-Udall Foundation and the Critical Path Initiative.

  • The Reagan-Udall Foundation was established to address weaknesses within the FDA’s scientific infrastructure. Though Reagan-Udall was aimed at establishing a public-private partnership to modernize medical product development, accelerate innovation and enhance product safety, Congress failed to appropriate seed funding.

  • In an effort to explain the reasons for declining productivity in the field of medical research, FDA launched the Critical Path initiative. More and more money is being spent on medical research, with fewer and fewer results. Critical Path was created to find out why. Critical Path can go a long way toward answering the question that has stymied researchers and activists: why aren’t we getting more bang for our research buck, and what can be done to fix it?

Our mission here at FasterCures is exactly what our name implies. We work to help foster a climate conducive to smart, productive medical research that doesn’t waste time or money. FDA is a critical element in the fight to save lives by saving time. We hope that Congress and the White House will work with Dr. Hamburg to provide her with the resources she needs to govern the FDA, and depoliticize the drug research process. Millions of lives are at stake, and patients shouldn’t be held hostage by politics. When elephants fight, it’s the grass that suffers.

FasterCures joins healthcare advocates in convening "flash mob" meeting

The impetus to pass comprehensive health reform legislation continues to grow as an energetic Congress and new leadership in our federal health agencies provide unprecedented support for expanding access to quality healthcare. The road ahead is both exciting and full of the unknown. Congress is promising passage of healthcare reform this summer. Now is the time for us to come together in urgency and openness to articulate our shared principles that focus reform on what truly matters: improved health for individuals, families, and communities. Fixing America’s broken healthcare system is a crucial step toward fixing America's broken healthcure system. Only if we translate promising scientific research into new treatments will we have any hope of reducing healthcare costs, productivity losses, and human suffering.

What: The Future of Advocacy and Improved Health During Healthcare Reform
When: Wednesday, June 17, noon to 4 p.m. EDT
Where: Biotechnology Industry Organization 1201 Maryland Ave SW, Suite 900, Washington, D.C. 20024

This meeting is convened by the Biotechnology Industry Organization, Coalition for 21st Century Medicine, Coalition for Affordable Health Coverage, FasterCures, Inspire, Research!America and Genetic Alliance. We welcome other umbrella/networks to co-convene this meeting with us.

Please RSVP to Julie Baumberger at BIO, outbind://25-000000006B968182C502C54C9BA36E0AE48FFDEB070058DAEF7DB9ED77439F719E1ECD4423210000027B8F21000058DAEF7DB9ED77439F719E1ECD442321000003A766740000/jbaumberger@bio.org, by June 15. Questions? Please contact Andria Cornell, Genetic Alliance's Advocacy and Health Policy Coordinator, at acornell@geneticalliance.org, (202) 966-5557 x207.

Thursday, June 11, 2009

FasterCures Comments on the Federal Coordinating Council for Comparative Effectiveness Research Draft Prioritization Criteria and Strategic Framework

FasterCures submitted the below comments on the prioritization criteria and strategic framework developed by the Federal Coordinating Council for Comparative Effectiveness Research.
June 10, 2009

To: Federal Coordinating Council for Comparative Effectiveness Research

From: Margaret Anderson, Chief Operating Officer, FasterCures

Re: Draft Prioritization Criteria and Strategic Framework

Submitted Electronically: http://www.hhs.gov/recovery/programs/cer/draftdefinition.html

Thank you for the opportunity to comment on the Council's Draft Prioritization Criteria and Strategic Framework. They are both very concise and thoughtful documents with which we substantially concur. We did, however, want to highlight some issues which we don't feel are directly addressed that may inform your thinking going forward.
  • In addition to informing better point-of-care decisions by patients and providers, building the evidence base through comparative effectiveness research can elucidate critical clinical research questions deserving investigation, which will accelerate the development of new and improved diagnostics and therapeutics. If that can be reflected in the prioritization criteria in some way (perhaps under #5, "potential for multiplicative effect"), we believe that would be of great value.
  • We are pleased that the Strategic Framework addresses not only the research studies themselves, but also the human and scientific capital necessary to execute the research -- including, very importantly, developing methodologies needed to conduct the research efficiently and effectively. We urge you to give this issue the attention it requires. The scientific underpinnings of comparative effectiveness research are still being developed, and it will be important to monitor the progress of the field as early studies funded through ARRA yield results.
  • Also addressed in the Strategic Framework is the data infrastructure supporting CER, another area we hope will be given careful attention. In particular, we hope the Council will make an effort to ensure that investments in health information technology being advanced separately with ARRA funds are supportive of the requirements for conducting CER to the greatest extent possible.
  • We are also pleased to see recognition of the fact that translation, dissemination, and adoption of the results of CER are as important as the studies themselves and hope that funding will be devoted to pursuing this critical goal.
  • We hope and expect that the vision driving federal spending on CER will continue to be enhancing and customizing care for patients, and that it will not be used to limit access to or availability of effective treatments on an individualized basis.
FasterCures' mission is to identify ways to accelerate the discovery and development of new therapies for the treatment of deadly and debilitating diseases both in the United States and around the globe. The organization was founded in 2003 under the auspices of the Milken Institute to aggressively catalyze systemic change in cure research and to make the complex machinery that drives breakthroughs in medicine work for all of us faster and more efficiently. FasterCures is independent and non-partisan. We do not accept funding from companies that develop pharmaceuticals, biotechnology drugs, or therapeutic medical devices. Our primary mission is to improve the lives of patients by improving the research environment, research resources, and research organizations.

Thursday, June 4, 2009

How many business plans did Mother Theresa have? Probably none, but just imagine what she could have done if she had one!

by Melissa L. Stevens, Director of Special Projects, FasterCures

Almost 200 participants from over 20 countries convened a couple of weeks ago in London to talk about individual efforts to evaluate nonprofits and a possible mechanism to unify these efforts under a professional association. New Philanthropy Capital, a London-based charity research organization, and Bertelsmann Foundation, one of the largest German Foundations, hosted this conference Valuing Impact: Building an Association of Nonprofit Analysts. Inspiring discussions were interspersed with challenging questions about the community’ preparedness for such an organization.

Matthew Bishop, Chief Business Writer/US Business Editor of The Economist spoke about creating an impact in his keynote address. He said that in this time of economic crisis it is important for "philanthrocapitalism" to emerge, and that a critical role of “virtuous intermediaries” is to rigorously analyze what does and does not work. He underscored that this evaluation work needs to be funded and be democratized for all philanthropists, not just the super-rich.

Other highlights from the meeting include learning about other evaluation frameworks and ways for improving FasterCures' Philanthropy Advisory Service metrics and hearing first-hand accounts of how best to build models and establish evaluation organizations.

Appropriately, participants checked their rose-colored glasses at the door. There was challenging commentary and acknowledgement of significant obstacles in building the field of nonprofit analysts. One of which being the lack of data produced by nonprofits. Ken Berger, CEO of Charity Navigator, noted that in his pilot effort to measure outcomes (in addition to information gleaned from 990 financial data) he found that 85 percent of 4-Star charities could not produce the necessary data for an expanded evaluation. Scary. We also heard there are at least 150 different frameworks in existence, so already, there is significant divergence. Yikes. Finally, there were the weighty pauses after the rhetorical questions like “Who should pay for transparency?”, “Who is the audience – donors or organizations?”, and “Won’t this be done at the expense of programming costs?” Oh my.

But at the end of the day the delegates regrouped and voted on their interest in establishing an association and at least 75 percent indicated they would be. Perhaps it will be a soft launch with at least a bulletin board, listserv, and sector-specific working groups to think through common frameworks. I would be very pleased to join an effort that can quickly connect me to others who have pioneered evaluation frameworks, business models, and donor education efforts. PAS would greatly benefit from the collective thoughts of such a network of relative experts in the field. We look forward to seeing where the journey takes us from here and applaud New Philanthropy Capital and Bertelsmann Foundation for getting us out of the starting blocks.

Wednesday, June 3, 2009

Hope, Healing, and Heroism Through Science

by Susan Semeleer, Associate Director of Communications, FasterCures
There is some exceptionally innovative work in biobanking. BioBank Central, a web portal that provides information and resources to the biobanking community recently launched its "Spotlight on Innovation series" to highlight the individuals and organizations involved in some of biobanking's best practices. We recently trained our spotlight on Autism Speaks and two of their research programs, the Autism Tissue Program (ATP), a post-mortem brain tissue bank for autism research, and the Autism Genetic Resource Exchange (AGRE).

This month's Spotlight is the first in a two-part series on the Autism Speaks banks and focuses on the ATP. Through a dedicated and innovative public education campaign, patient advocacy groups like Autism Speaks have made tremendous strides toward dispelling the stigma and misinformation that surrounds autism. With the ATP, they are taking that education campaign one critical step forward: providing researchers with precious tissue that could hold the key to unlocking the mysteries of the autistic brain.

To learn about the ATP from both a clinical and personal perspective, we interviewed Autism Speaks' Vice President of Clinical Programs, Dr. Clara Lajonchere; the ATP Director Dr. Daniel Lightfoot; and the family of Reid Thompson, an 11-year old tissue donor. Dr. Lajonchere and Dr. Lightfoot told us that tissue samples – particularly pediatric tissue samples – are absolutely critical for gaining better understanding of autism and are necessary for the kinds of molecular, genetic, and biochemical research needed to keep the pace of discovery moving.

The ATP wouldn’t be possible without the generosity of families like Reid Thompson's. The day after Reid passed away unexpectedly in his sleep, his parents and step-parents learned about the ATP tissue donation program. At a time when many among us would be immobilized by loss, Reid's family didn't hesitate; Reid's tissue was collected for the ATP bank and is now being used in studies that will help doctors learn more about the brain's development in autism. Reid's family told us that he was a teacher during his life, and that they take great comfort in knowing the ATP enables him to continue teaching.

What Dr. Lightfoot and his colleagues are doing, and what Reid Thompson and donors like him have done, is nothing short of heroic. What we found most impressive about them is that they didn’t seem to see the heroism in their actions. But we here at Biobank Central do, and we’ve no doubt you will as well. Please stop by ATP Spotlight, and visit the Autism Speaks and Autism Tissue Program Web sites to learn more about their innovative programs.

Stay tuned--BioBank Central's Spotlight will focus on the Autism Genetic Resource Exchange.

Waiting for a Tipping Point in Science

by Margaret Anderson, COO, FasterCures

Where are we on the path to treatments and cures for brain and nervous system illnesses? Had a chance to learn more about new models of collaboration, funding, and partnerships at the recent Collaborating for Cures summit hosted by the Neurotech Industry Organization. Too often we focus on the overflowing bins of problems and forget to focus on where change has taken hold and started to move the needle. John Wilbanks of Science Commons gave participants food for thought when he asked how many nonprofit funders put dollars into collaboration, and cited the need to put our knowledge into a network instead of relying on tin cans connected by a string.

If, as Wilbanks said, resistance to collaboration in science is enormous, what are some ways to change behavior? What is possible when you take John’s advice and “resist the resistors?” What are some ways to get to collaboration, in a purposeful way instead of hoping it will happen? Some tried and true tools such as AlzForum, a 13-year-old online destination for the scientific community to learn and share information and research developments about Alzheimer's disease, is adding new features. AlzForum's June Kinoshita cited upcoming features of the SWAN (Semantic Web Application in Neuromedicine) database, a community-driven knowledge base of Alzheimer disease that relies on a moderated community process to capture the collective insights of the AD field. Michael T. Rogan from the Michael J. Fox Foundation cited the need to “move away from huddled hallway chats” in science to better our chances to find treatments and cures. Rogan leads the development of “PD Online Research” which is a collaborative scientific community which will conduct ongoing critical analysis of PD research and help connect high-priority research with the necessary financial resources.
I moderated a panel on “New Models of Funding” featuring:
With the imperative for them to have “more shots on goal” for their disease areas, these organizations are forging ahead into the future using new models via prizes, venture funds, open science tools, and managing the science. The conference ultimately posed the question: if we have seen massive efficiency for things that might be deemed frivolous (internet shopping, learning about celebrities every move via Twitter), when can we expect a tipping point in science? Many of the models discussed there will create a path for others to follow, and hopefully end up with faster cures.

Monday, June 1, 2009

FasterCures: It’s not just our name, it’s our mission

By Margaret Anderson, COO, FasterCures
Six years ago, FasterCures started working on creating a medical research system for the 21st Century that saves lives by saving time. It’s a lofty goal. But one that must be met without compromise. Millions of people who have been diagnosed with a deadly or debilitating disease have compromised enough. There is not a moment – or a life – to lose.

FasterCures is an “action tank” for a reason – we focus on clearing the path between an idea for treatment and getting a treatment to the patient. That path is no straight line. Akin to being in the middle of a maze, we know there is an opening in the end. This maze has multiple paths leading to the ultimate goal. Some of the dead-ends we hit are actually facades – enough to slow you down, stop you at times, but with the right tools, and more importantly, the right perspectives, these artificial, arbitrary barriers can be overcome to clear the path to faster progress.

Barriers to progress in accelerating cures exist all along the research continuum – from basic research to development, from medical education to medical practice, from investment capital to human capital. We’ve spent our first six years identifying and analyzing these barriers and developing innovative and relevant programs to overcome these barriers.

Our commitment is steadfast as we continue to implement tangible programs and develop pragmatic solutions. Our eye is on the prize as we nimbly respond to shifting markets, an ever-evolving policy landscape, and medical breakthroughs that disrupt tried and tested approaches. We work hard to transform medical research so it could work faster and more efficiently. Here’s a glimpse of some of our programmatic efforts that allow us to do this:
(please click on image for printer-friendly version of the FasterCures Blueprint for Change Update)

We all share an interest in finding improved treatments and cures. Most of us know someone – a father, sister, child, friend or colleague – who has suffered from or died from a terrible disease. No one is immune. Much remains to be done. Get involved with FasterCures today.

Passion Capital at BIO

by Margaret Anderson, COO, FasterCures
Is venture philanthropy the new venture capital? The FasterCures-organized session at BIO focused on this question and highlighted the similarities and differences in the two. The panel featured a terrific lineup of:
First up, the topic of “how bad is it out there with respect to funding?” Mark Simon has been involved in raising capital for life science companies for years and sees venture philanthropy as a great option for companies , especially when you see the statistics of how few biotechs currently have enough cash for long-term stability. According to BIO, 99 companies are operating with less than six months' worth of cash, which accounts for 25 percent of all public U.S. biotech companies. The vast majority of America's biotechnology companies are small, research and development focused companies with fewer than 100 employees. Simon was concerned that we stand to lose valuable scientific opportunities in biotech as funding continues to be elusive.

As for how the venture philanthropy groups view their mission, it turns out failure is an option. “There will be failures, this funding from organizations like ours is not a panacea,” said Bob Beall of the CF Foundation. Eric Olson of Vertex spoke of the pass-off from CFF where they take the funding baton after CFF funds discovery. The synergy with Vertex and the other companies CFF partners with wasn’t always so easily found. Turns out Bob had to pass through the phase where there were a lot of calls unanswered from biotech in his early days at CFF. Expectations must be realistic on both sides for these relationships to work, reflected Beall.

Katie Hood of the Fox Foundation talked about how one size doesn’t fit all. They are driven by different imperatives in terms of their disease state as well as their pipeline , which has resulted in different types of relationships with companies; what they have in common with foundations like CFF is that they are trying to remove risk for companies potentially interested in investing in their disease. Katie also discussed PD Online, which they are launching to provide a collaborative space for discussion and problem-solving in PD research.
Even though philanthropic research funding accounts for only two percent of total health research funding, it's a small but substantial source for novel, high-risk research that might not be able to compete successfully for public funds. It fills gaps in funding research that is high risk but also with potential of high return.

So, is venture philanthropy the new venture capital? Not exactly, but it is an exciting new kind of collaboration that can bring benefits -- funding, but also intelligence, relationships, and access to patients -- to companies and to foundations.