Fix Our Broken HealthCure System

by Margaret Anderson, COO, FasterCures
An ever-evolving economic crisis continues to challenge our U.S. and global leaders. The marathon search for solutions is approaching sprint speed and yet, we are surprised at how hardly anyone has turned to leveraging our greatest strength: human capital as part of the answer.

The 20th Century’s greatest achievement was the increase in life expectancy. This fueled the growth and increased the productivity of our economy and allowed nations worldwide to build wealth. The 21st Century presents us with the opportunity to not just leverage the extension of life but to also improve the quality of life. Instead of taking this opportunity head-on, we shy away from investing in human capital, the one resource we have an abundance of, even as we know that doing so will yield immense returns for generations to come.

Along with the moral imperative to conquer disease and relieve suffering comes the economic reality that illness and its costs are debilitating to our economy. According to the Centers for Medicare and Medicaid Services, the U.S. spent $2.1 trillion on healthcare in 2006, more than $7,000 per person. For all the intellectual and financial capital flowing through our healthcare system, there remains a formidable list of diseases for which there are no cures or even meaningful treatment options. Treating disease is not only the right thing to do to save lives; it is the right thing to do to save our economy.

Consider that the elimination of cancer as a cause of death and suffering is worth more than $50 trillion to the US economy. If we can do this, we would greatly add to the growth of our economy, productivity, and quality of life, making up for the financial assets we may have lost in the last 90 days, and preventing further loss in years to come. Yet our national investment in cancer research is going down and is nowhere near commensurate with the costs we bear or the gains we could expect if we made progress in curing cancer.

If President-elect Barack Obama cares about fixing America’s broken healthcare system, he needs to think about fixing the broken healthcure system.

Research!America released a report last week that showed 73 percent of Americans believe research is a solution to rising healthcare costs, and yet, out of every dollar we spend on health, we invest only 5.5 cents in research. It takes 15 years to develop new medical treatments. But we know that the thousands of people diagnosed with a deadly disease today cannot wait 15 years. We created a research enterprise system at the National Institutes of Health (NIH) whose central organizing principle was the study of human biology. This brought enormous breakthroughs in the fundamental knowledge necessary to understanding, preventing, diagnosing, and treating many diseases. But our ability to translate exciting new discoveries into products that can help patients is severely lagging behind the pace of discovery.

If President Obama cares about improving America's standing around the world, he needs to make good health our most important export. Global health is not just for ex-presidents. Our global diplomatic strategy should include directing resources to finding cures for global diseases.

Reorienting our medical research enterprise to cure diseases will pay-off in dividends far greater than health outcomes alone. It is one of the critical pieces needed to rebuild America’s global standing. Diseases transcend national borders and trump domestic policy agendas.

We need to immediately begin creating a medical research system for the 21st Century, one that will focus on curing diseases. To do so would require the right leaders are in the right positions at the right time.

We need strong, capable individuals at the helm of government agencies with bold, nearly-impossible missions. Presidential appointments need to rest on credentials, not connections. With Former Senate Majority Leader Tom Daschle (D-S.D.) holding the top post at the Department of Health and Human Services, other officials with equal gravitas and expertise need to be in place protecting the health of all Americans, providing essential human services, and effectively managing an annual budget of over $700 billion dollars (representing almost a quarter of all federal dollars). We need and expect the right leader to steer the vast NIH enterprise down the path of finding cures, accelerating the pace of over 38,000 research projects nationwide, and managing 27 separate health institutes and centers. We need and expect a formidable FDA Commissioner ensuring the safety of foods and cosmetics, and the safety and efficacy of drugs and devices; products which represent almost 25 cents out of every dollar in U.S. consumer spending.

Among the tasks at hand, these leaders will have the opportunity to:

1. Transform the existing fragmented, bureaucratic research infrastructure into a collaborative network.
2. Ensure that the ultimate goal of all scientific research is to improve health and cure disease.
3. Highlight the critical role patients play in the search for cures and incentivize patient participation in research.
4. Ensure that the nationwide health information system being built can improve patient care and enable medical research.
5. Transform the NIH Intramural Research Program (IRP) to focus on translational research.
6. Support efforts to build capacity and improve effectiveness at the FDA.
7. Develop a responsive system for reviewing and funding research that will identify the most promising areas of scientific exploration in terms of their potential to contribute to improved human health and well-being.
8. Encourage innovative, integrated, and information-based research approaches and new models of research funding.

As President-elect Barack Obama turns his attention to reforming our healthcare system, he, too must nurture our 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.
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A Manhattan Project to Replace the Moon Shot

by Greg Simon, President, FasterCures

Next summer I have my fortieth high school reunion. (Ouch!) I haven’t decided whether I’ll go. To be honest, I’m not sure I want to be trapped for a few days talking about what happened forty years ago. I always cringe when I hear "Glory Days," the Springsteen song about a guy stuck reliving his high school achievements the rest of his life. I’ve had a lot of good fortune in my life since high school and there’s a lot to talk about; the problem is I don’t feel comfortable talking about it around people who only want to talk about the "glory days."

Well, I hate to say it, but America is stuck at its own fortieth reunion, talking about past glory days – I’m talking about landing a man on the moon forty years ago, my senior year in high school.

How often have you heard in the past election campaign, or at meeting after meeting on healthcare, education, technology innovation, whatever, that what this country needs is another moonshot, a commitment to do something hard and inspirational? And for a special bonus, don’t forget to mention the Manhattan Project when you really want to inspire people to do something hard.

Right. What we need to do to cure cancer or fix our broken schools is to lock a group of Hungarian, German, and Russian immigrants in a gym under a football field, put them under the dictatorial command of a General and not let them out until they develop something that can end life as we know it.

Is this the best we can do? Have we accomplished nothing since the Bomb and the Apollo projects? Have we been wandering in a desert of underachievement for the last forty years, or are we just metaphor-deprived?

Let me take a different tack. Have you ever noticed that nearly all the metaphors we use in daily life originated in 18th century sailing terms?

• "We stayed ‘til the bitter end." (The end of the anchor cable attached to the "bit.")
• "He was three sheets to the wind." (Sheets are the ropes that haul the sails, three sheets flying mean you are out of control.)
• "I gave him quite a start." (To "start" someone on a ship is to strike them with a whip, or worse to get them moving and working.)
• "He has lost his bearings."
• "The campaign fired back with a full broadside." (I’ve long advocated for the America’s Cup to require cannon battles during the race. Anyone can learn to sail, but can you sail and fire a broadside at the same time?)

None of these terms retain their original meaning, they are now shorthand for situations or conditions.
When I worked for Vice President Gore, he gave a commencement speech at MIT devoted exclusively to the poverty of modern metaphors to represent our lives. (We did two all-nighters on that speech, but that’s a different story.) His point is just as true today; for all of our innovation in science and technology that has transformed our daily lives, we have not found a way to replace the metaphors we inherited from our grandparents’ grandparents. (Somehow, "My hard drive crashed" still doesn’t trump "It took the wind out of my sails.")
So, how do we …uh…chart a different course? Are we doomed to be linguistically lost at sea, rudderless and unmasted by a lack of imagination? Or can we damn the torpedoes of inertia and go full steam ahead with a wind of new metaphors at our back, flowing with a creative current of concepts to a bright shining shore? Whew.

First, let’s leave high school. We can no longer ride the moonshot to glory, and those scientists we need for another Manhattan Project can’t get work visas. Where can we look for inspiration? Let’s leave the world of rockets and bombs and look at life.

It’s 2008 and so far, we have defined life in the 21st Century in terms of having everything instantaneously and simultaneously. We want speed (10 seconds to open an attachment?!). We multitask (ITunes, YouTube, instant messaging, Facebook updates, all while watching CNN’s split screens). We live in communities connected by social networks (are the 2,489 friends really your friends?).

And yet, when speed matters most and its consequences are measured in terms of lives saved, we’ve gotten complacent. It takes 15 years to develop new medical treatments. If living longer and having a better quality of life are not enough to spur us into making this a national priority, where can we look for inspiration? What will it take for us to focus on curing diseases?

We need a moonshot equivalent in the life sciences. Until then, any metaphors we should consider?

FasterCures President Gregory C. Simon Joins President-Elect Obama’s Transition Team

FasterCures president Gregory C. Simon, former Chief Domestic Policy Advisor to Vice President Al Gore, has been named by President-elect Barack Obama to his transition team.

Simon is part of the team reviewing the Department of Health and Human Services (HHS). He brings experience and expertise in biomedical research policy, knowledge of the transition process, and close familiarity with the mission of HHS and its 11 operating divisions, including the National Institutes of Health and the U.S. Food and Drug Administration.

“I am honored to join President-elect Obama’s HHS transition team to help ensure that the important work of the agency continues to move forward seamlessly,” said Simon. “HHS touches the lives of millions of Americans every day in its quest to protect the health of all Americans and provide essential human services,”

Greg Simon is president of FasterCures, a center of the Milken Institute formed in 2003. Simon’s leadership has positioned FasterCures into an organization valued and recognized for catalyzing systematic change in the process of discovery and development of new therapies for deadly and debilitating diseases. In October 2008, Nature Medicine named Greg one of 10 influential people to watch in biomedical policy and noted that he is among a “handful of influential people who quietly help keep the wheels of biomedical science turning.”

# # #

About FasterCures
FasterCures / The Center for Accelerating Medical Solutions is committed to saving lives by saving time in the research, discovery and development of new treatments for deadly and debilitating diseases. FasterCures, as a center of the Milken Institute, is nonpartisan, nonprofit and independent of interest groups. For more information, visit www.fastercures.org.

Venture Philanthropy’s Next Chapter

by Margaret Anderson, COO, FasterCures

The story of venture philanthropy in medical research is getting told with even greater frequency lately. Many of its leaders are on the road constantly presenting about why their organizations came to be, what strategies they use to understand the root causes of their diseases, and what they are doing to support translational research. This was the central question of an Institute of Medicine (IOM) Forum on Neuroscience and Nervous System Disorders meeting in early October.

At FasterCures, we work with many of these groups through our TRAIN initiative. We released the “Entrepreneurs for Cures” report that articulates the reason many of these groups were formed: that their only bottom line is curing disease and improving the lives of patients.

At the IOM meeting, some of the problems in reaching cures were restated before the group dove in to the various approaches and models that the venture philanthropy groups use to move the needle. The statistics about drug development are a stark reminder of the enormous challenges facing biomedical research and the quest for faster cures. Despite increasing knowledge and investment in R&D spending, drug registrations are falling and the investment costs per successful drug launch has escalated in recent years. For many diseases, treatments – let alone advances – are few and far between. The ‘valley of death’ in drug development is a story that venture philanthropy groups know all too well.

The tag lines for the meeting could have been “Focus, Focus, Focus” with a healthy dose of “If you don’t know where you are going, any road will get you there.” Two themes that are particularly poignant in today’s economic climate. With each day’s headlines of the stock market tumbling to new lows, there is a greater sense of uncertainty about how this will impact medical research philanthropy. Groups need to be laser-focused, and they need to have an action plan.

Dick Insel of the Juvenile Diabetes Research Foundation pointed out that doing this work requires that you know your product, partners, and yourself to be effective. Expectations between partners need to be aligned. In terms of knowing yourself, he pointed out the importance of managing expectations with donors and the patient community, and also the balance between hope versus hype in doing disease research. Sophia Colamarino of Autism Speaks described how patients and in the case of autism, the parents of patients have to be brought into the research process and need translation services. And when there are accomplishments, they have to be given credit too. Everyone agreed that knowledge needs to be constantly pushed out to many audiences in order to move closer to the goal. The scientific community needs it, industry needs it, and of course patients need information to know how new findings get mapped against the search for cures.

Katie Hood of the Michael J. Fox Foundation spoke of how the value-add they bring is that her group is constantly looking at what worked and didn’t work, and what MJFF needs to do to increase the odds of success of what they are funding. She also noted that the valley of death is not the same for each disease, and that venture philanthropy groups need to create a roadmap. Each group should ask what unique value is of what they bring to the table, and how they can target everything towards that goal.

These groups often talk about milestones, and the requirements they place on grantees when they get their funds. Some groups pointed to examples where they stopped or held payments. This requires active management and forecasting of the research portfolio. Some described how the goal was to bring more partners and players into their fields, and they saw their role as lowering those barriers to entry.

One participant summed it up, “Nobody has the incentive to think about our disease process like we do.” Jonathan Simons of Prostate Cancer Foundation talked about their metrics in plain and simple terms – lives saved and a reduction in the death rate.

Someone pointed out that the role of these groups is to shine a light on what is possible in disease research. Indeed they do. Next challenge, how do you spread this line of thinking and these tactics even farther?

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From Social Entrepreneurship to Cure Entrepreneurship

by Cecilia Arradaza, Communications Director, FasterCures

Social entrepreneurship has attracted scholarship, media attention, and financial resources over the last decade. Social and professional networks have emerged from this discipline. Medical research has been largely missing from the dialogue and the discipline, even as it seeks to address a market failure no less important than other social enterprises – the development of therapies that cure disease and alleviate suffering.

More than 30 senior leaders from organizations that fund medical research and those from the broader sphere of social entrepreneurship convened a week ago at what has become an annual leadership forum on medical research innovation brought together by FasterCures and Esquire magazine. The meeting explored what lessons have been learned by social entrepreneurs that might be applied by those pursuing what FasterCures calls "cure entrepreneurship," or novel approaches to accelerating the process of treating and curing disease.

Much of the discussion during the day centered on the need for better information flows about what work is being done, by whom, and its quality; the need for better communication about why new approaches are needed and the promise they hold; and the need for nonprofits to find creative ways to leverage their relatively small funds into larger investments into new therapies. There was also a common focus on the centrality of patients and the transformative nature of empowering them to play a greater role in research.

The social entrepreneurs and cure entrepreneurs in the room found a lot of common ground. In part because both distinct sectors function in a similar nonprofit space where success is often the result of effectively leveraging the assets that it creates. It was noted however that the social business model is difficult to achieve in medical research because of its cost implications. Throughout the day, participants alluded to the need for unconventional solutions, unusual channels to reach nontraditional audiences, outcomes-focused collaborative efforts, and ideas that could be acted on in real-time by the right people.

Perhaps the meeting’s location, set in a swank, Architectural Digest-ready mansion in Hollywood Hills triggered the right brain to yield more inspirational ideas. Perhaps it was the collective braintrust in the room that stimulated an engaged energy and infused thoughtful intrigue that are often absent in more typical conferences. Greg Simon, FasterCures’ president aptly set the tone when he said in his welcome remarks that “we do not need to go somewhere, we just need to be somewhere…”

Indeed, we were somewhere. It was a place where we tried to create an intersection between social entrepreneurship and medical research philanthropy – an ecosystem to support the work of cure entrepreneurs. It was a place where ideas reigned and the seemingly impossible task of challenging our biomedical research system to produce results was not a far-fetched goal. The meeting reinforced the unique role that FasterCures can play in helping to build a "knowledge commons" to accelerate medical research – whether that is collection of the best organizational practices of TRAIN groups, or continued work on the Philanthropy Advisory Service to make transparent information about the most effective medical research foundations, or perhaps an entirely new project to escrow vetted but unfunded proposals from funders. Additionally, meeting attendees urged FasterCures to focus on the idea that philanthropy is a whole new capital market in need of rules and auditable facts.

As the context we all are a part of changes dramatically by the day, last week’s meeting was a place to start, an opportunity to put some markers on important work that needs to continue if we, as a nation, are serious not just about reforming our broken healthcare system, but also about fixing our broken health cures system.

Related Blogs:
Common Knowledge's The Unknowns by John Wilbanks
http://scienceblogs.com/commonknowledge/2008/11/the_unknowns.php

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Personalized Healthcare Needs to Get Personal

by Greg Simon, President, FasterCures

A personalized approach to healthcare starts with patients being involved in medical research. We are all walking medical record repositories for ourselves and our loved ones. And yet, patients are still the healthcare system’s largest untapped resource.

Embedded within each patient is the information – medical records, biological material such as tissue, blood, and DNA, and our biology as observed in clinical trials – that is crucial to understanding and managing disease. The ability to effectively preempt, accurately diagnose, and successfully treat disease relies on a patient’s willingness to provide personal genetic and medical information and to become an involved participant throughout the medical research continuum.

Each of us has experienced how patients are not at the center of healthcare. It's no surprise why patients are not chomping at the bit to participate in research – information about clinical trials is scarce and confusing, there are few incentives in exchange for your effort and time, and healthcare providers rarely provide you with the guidance and the care you need and deserve to navigate through a complex process.

Everything has to change to put patients at the center. To make personalized medicine a reality for all, we need to transform our current healthcure and healthcare systems from the outdated model of the last century to an integrated, information-based, high-quality, health-sustaining model. We need a healthcure system that engages patients from the very beginning of the research process instead of turning to patients as an afterthought. We need a healthcare system that will improve treatment options for patients, prevent the onset of disease in the first place, and improve the quality of life for generations to come.

When it comes to your health -- take it personally.

For more information, download a copy of the just released FasterCures white paper "Patients' and Consumers' Interests and Perspectives in Personalized
Healthcare." This paper looks into the cutting-edge
possibilities of personalized healthcare and the need for patient involvement
and provides a glimpse into how "personal" personalized healthcare is and what
consumers think about the advent of this era. FasterCures was
commissioned to develop this paper by the Office of the Assistant Secretary for
Planning and Evaluation at the U.S. Department of Health and Human Services, as
part of Secretary Michael O. Leavitt’s Initiative on Personalized Health
Care.

Related Blogs:
11.18.08
Science Progress Blog:
The Revolution Will Be Personalized
A Cautious HHS Report on the Latest Buzz In Medicine

Long Live Mr. Health's Legacy

by Greg Simon, President, FasterCures


"By your deeds you shall be known."

That is why Paul Rogers – so well known from a lifetime of good deeds – will be missed by millions of people. I say millions because if you or a loved one has ever been sick or poor or hungry, Paul Rogers did a good deed for you. With a stentorian voice that seemed made in heaven, Paul Rogers advocated for this nation to make protecting the health and well-being of all of us a national imperative. The organization he loved and that loved him – Research! America – is the fountain from which sprang so many organizations (including FasterCures) trying to improve the health and quality of life of all people. His work through Research!America to double the NIH budget will lead to cures and therapies that will affect millions. The Paul G. Rogers Society for Global Health Research Ambassadors program funded by the Gates Foundation to promote awareness of global health issues will affect hundreds of millions.

We all could learn a thing or two from Congressman Rogers' 24-year tenure. He kept his eye on the nation's top priorities and made it his own. He helped ensure that generations of Americans have access to the best that medical science has to offer -- life-saving cancer therapies, medical devices, emergency medical care, public health programs -- and made sure that the water we drink is clean and safe and the air we breathe is free of pollutants.

Congressman Rogers saw the potential for improving our nation's healthcare system and worked tirelessly to turn that potential into tangible benefit for millions of Americans. He helped rally Congress to wage a war on cancer, supported increased investment in the National Institutes of Health, worked to maintain the integrity of the Medicare and Medicaid programs. More significantly, he paved the way for the biomedical research enterprise to become the most scientifically and technologically advanced system in the world.

The FasterCures team extends our sincere condolences to the family of the late Congressman Rogers and to Research!America for the loss of this great and caring man. There are few absolutes in life, but here’s one: Paul Rogers devoted his life to helping others and never asked a thing for himself. The greatest gift we can give his family and friends is to try and do that ourselves everyday.

Greg Simon named one of 10 Most Influential in Biomedical Policy

This month's Nature Medicine named Greg Simon, FasterCures President, one of the ten most influential people to watch in biomedical policy. The article presented "a handful of influential people who quietly help keep the wheels of biomedical science turning."

According to the article --

...influence can take many forms. When it comes to biomedical science policy in the US, the names of some people with clout are obvious. But beyond the Nobel laureates and university presidents there is another set of people who wield power. Some of these influential players, such as patient advocates or experts who churn out reports for think tanks, are well known within their own communities. Others, such as Senate staffers or policy makers at the US National Institutes of Health (NIH), operate under the radar.

Not enough Alzheimer’s patients?!

by Jeongyeon Shim, Program Analyst, FasterCures

As part of the FasterCures’ Philanthropy Advisory Service program, we invited four eminent scientists in Alzheimer’s disease research to learn more about their perspectives on key opportunities and challenges that the field is facing. We discussed many of the exciting development occurring in the field. In addition to progress in basic research, many potential treatments are showing promise in animal studies and various stages of clinical trials. Many of the efforts targeting what scientists believe to be the causes of the disease are moving along the pipeline, and novel mechanisms of interventions are also discovered.

The meeting also addressed challenges in AD research. Among others, low enrollment in clinical trials was highlighted as a key challenge in transforming exciting scientific discoveries into treatments that benefit the patients. Alzheimer’s is not exactly an orphan disease—it impacts 4.5 million patients in the US today, with a drastic increase of disease burden expected. How is it possible that there are not enough clinical trial participants?

Many clinical trials in Alzheimer’s target the patients in early stage of the disease, since that is when the debilitating effects of dementia, caused by the death of neurons, can be averted. However, a diagnosis is less straightforward at early stage, given that clear biomarkers of the disease are yet to be established. Even in cases where diagnosis is made early, additional challenges to clinical trial participation exist. For example, under the current system of care where primary care physicians are in charge of most Alzheimer’s patients, it is difficult to have patients enroll in clinical trials, which are conducted in more specialized healthcare centers. Trial participation may also increase the burden to the caregiver due to the need for additional visits, especially when the trial site is at a distant location.

While scientists and trial administrators focus on finding solutions to this challenge by developing biomarkers for early diagnosis, reaching out to primary care physicians, educating participants on the importance of trials and developing technological solution to reduce the need for physical visits, the most important lever would be that patients and caregivers learn more about trial options, and when possible, enroll in trials. Participating in trials requires substantial effort from the patients and caregivers as well as courage to commit to a therapeutic regimen that is yet to be proved. No other research tool can substitute for clinical trials in developing novel therapeutics. Trial participants make an invaluable contribution to medicine by making themselves available—we cannot overemphasize the fact that none of the medical breakthroughs we have today would have been realized without their contribution.

To highlight one trial, the Alzheimer’s Disease Cooperative Study, a clinical trial network funded by the NIH, is currently recruiting participants for a trial aiming to curb the toxic effects of beta-amyloid .* This is not the only trial targeting Alzheimer’s disease—ClinicalTrials.gov lists 121 trials in the US and 216 when counted across the globe, actively recruiting participants, as of September 24, 2008. FasterCures has been highlighting clinical trial participation as a key element to accelerate medical research. I look forward to a day when an informed conversation about clinical trial participation becomes part of routine medical care.

*FasterCures does not endorse any specific clinical trial. Decisions on clinical trial participation should be made based on consultation with healthcare professionals.

Calling All Ideators, Gearheads, and Entrepreneurs

by Kristin Schneeman, Program Director, FasterCures

As many of you who follow what we do at FasterCures know, we’ve been interested for a while in the approach to innovation taken by InnoCentive, a Web site on which "seekers" can post problems to be tackled by a universe of "solvers," many of them from entirely different disciplines. Born out of a need to open up innovation within the pharmaceutical industry, the site is now beginning to be used by groups like the "Prize for Life" challenge to find a biomarker for ALS, and the Global Alliance for TB Drug Development.

FasterCures and the folks at InnoCentive are now embarking on an experiment together by posting an Ideation Challenge on the site, one of the company’s first "Public Policy and Citizens in Action" challenges seeking solutions to important issues facing society. We’re hoping to benefit from the "wisdom of the crowd" by asking for novel approaches and/or incentives that might induce companies to invest more in biomarkers research and qualification.

Within a day of posting the challenge we were pleasantly surprised to see that there were dozens of "project rooms" opened by individuals interested in possibly submitting a solution; by the end of the week there were more than a hundred. The proof will be in the pudding, of course. This sort of challenge is quite different from the ones that have become InnoCentive’s bread and butter (with apologies for mixing my food metaphors).

InnoCentive’s successes can be attention-grabbing. One example is a challenge posted by the Ocean Spill Recovery Institute to solve a thorny problem that has long hampered cleanup efforts from the Exxon Valdez oil spill, among others: the difficulty of separating frozen oil from water in collection barges. A chemist from the concrete industry proposed an approach based on a technique that has been used for years to move liquid concrete; he won $20,000, and the oil industry solved a decades-old problem within two months after posting it on the Web.

Nonprofits are beginning to use InnoCentive with success to help solve problems of poverty and disease in the developing world, as well. A Rockefeller Foundation-sponsored challenge produced a design for a compact milling machine that would allow rural farmers in India to increase their income to as much as $30 a day. The solver is a self-described "ideator, gearhead, and entrepreneur" – probably not someone in Rockefeller’s network of usual suspects.

Our challenge is a good deal less concrete than these, but no less important or in need of innovative thinking. We’re eager to see what kinds of responses we get back after the November 15th deadline. We could have tapped likely academics or consultants to write us a paper on overcoming the challenges of biomarker research and qualification. But for now we say, bring on the ideators, the gearheads, and the entrepreneurs!

Find Drugs that Work for Cancer

by Cecilia O. Arradaza, Communications Director, FasterCures
To win the war on cancer, our nation’s decision-makers need to enact policies that support cutting-edge developments in cancer research and facilitate patient-centered approaches throughout the research continuum. This message permeated through discussions at Friday’s Clinical Research Conference convened to address pressing scientific and regulatory challenges and opportunities for progress in innovations in cancer therapy. The conference was organized by Brookings Institute, Friends of Cancer Research, American Society of Clinical Oncology, and American Association of Cancer Research.

More than a hundred leaders in cancer research and advocacy representing academia, government, industry, and the nonprofit sectors convened for a full day discussion of scientific and policy issues impacting clinical cancer research. The diversity of viewpoints and the depth of commitment to furthering cancer research spurred an engaged discussion among participants anxious to address our nation’s cancer challenges.

NCI Director John Niederhuber said in his opening remarks, that the 21st century paradigm will be anchored on translational science and based on multiple, highly-targeted agents matched to molecularly selected patients. Important issues were raised by panels that followed and some key recommendations rose to the top. Highlights include:

• The first panel looked into data standards and evidence requirements and noted that “it’s easy to get data…the challenge is to ensure the data we have is any good.” To make sure new drugs are safe and effective with the optimal amount of information, the panel recommended the development of qualitative and quantitative standards for data collection to streamline the path to more efficient clinical trials.
• Measuring treatment efficacy is a complex and subjective process and the oncology community has long sought endpoints other than overall survival. Looking for a better way of measuring the effectiveness of new drugs and accelerating safe and effective cancer drug development, the second panel proposed three different scenarios that look into progression-free survival as an indicator of clinical benefit.
• The promise of personalized cancer therapy remains largely unfulfilled and there have been few successful efforts to develop therapies and their targets simultaneously. The third panel recommended the following:
  - External stakeholders should be engaged in the design of a pathway for the development of diagnostics for tumor markers.
  - Tumor-marker clearance and approval should be based on demonstrated clinical benefit.
  - An advisory committee similar to the FDA’s Oncologic Drugs Advisory Committee should be developed for tumor marker clearance and approval in order to improve consistency and coordination with other oncology programs in the agency.
• The final panel painted a vision for the FDA’s future. FOCR’s Ellen Sigal noted that complex science is at the base of all the discussions and that the process for defining a clear path forward is always evolving, making it critical to garner the input of diverse stakeholders every step of the way.

At day’s end, having mined the combined braintrust of panelists and participants, former FDA Commissioner David Kessler articulated a key action step and perhaps a challenge for the next person to lead the FDA. Kessler said we “need an FDA Commissioner who will say: find drugs that work for cancer.” Someone who is willing to take risks, make mistakes but who has a clear idea of what they want and be able to articulate this amidst competing priorities. On this note, be on the lookout for FasterCures’ upcoming recommendations for the new administration regarding the FDA.
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Magical Thinking

Margaret Anderson, COO, FasterCures
I appreciated the message of Carol Diamond and Clay Shirky’s recent piece in the August 2008 Health Affairs titled “Health Information Technology: A Few Years of Magical Thinking?” In it they say that “proponents of health IT must resist “magical thinking,” such as the notion that isolated work on technology will transform our broken system.” It’s interesting to think about systems change at the front end, and how easy it is to get stars in our eyes about how things like health IT or personalized medicine will transform the world as we know it, and how all of our problems will then magically go away.

I was speaking recently to a colleague here Kristin Schneeman about her life working in Vice President Gore’s office in the early days of the Clinton Administration and how new and novel email was at that time. I recall getting my first work email account and not having anyone to really email anything to, except my colleagues on the same floor. Greg Simon, President of FasterCures has talked about how he had a cell phone in the early days and was embarrassed to be talking on the street with it so he’d take it into a pay phone booth to look more “normal.”

Cell phones, email, and the Internet have certainly transformed things in ways we couldn’t have imagined, but they’ve introduced problems we couldn’t have imagined. Technologies such as FAX machines have been leapfrogged over. Problems such as the overabundance of information, and the speed of information flow are here to stay it seems. In the case of health IT, FasterCures sees it as a vital bridge to the future of more rapid information collection, characterization, and analysis which could speed our time to cures.

We are working on a white paper for the U.S. Department of Health and Human Services about educating and building awareness among consumers about personalized healthcare. This is another area where we must resist “magical thinking” and get down to brass tacks. Too often, the discussion about personalized medicine has been at a 30,000 foot level. For this paper, we’ve talked to many patient advocacy and disease research groups and everyone holds their breath about the potential power that these technologies may hold for their disease areas. They all want more targeted therapies with fewer side effects, which is ultimately the promise of personalized medicine. But they also recognize its complexities. It needs to take into account the world of co-morbidities we all live in; even if baby boomers are out running marathons and eating their greens and blueberries, the reality is that many of us are living with many conditions and diseases, not just one. It will probably raise costs before it can lower them. It's unlikely many diseases will yield to the relatively easy HER2-Herceptin gene-to-drug relationship. Patients are likely to get much more information about their genetic makeup than they can act on in the near-term. So, with all these complexities, is it magical thinking to ponder how scientific advancements in genomics and proteomics may change things for the better? Is it magical thinking to imagine a health IT system that can propel these advancements forward even faster? Or can we make it real? You tell me.

Is Philanthropy Going to the Dogs? Perhaps it’s Still the Cat’s Meow…

by Melissa Stevens, Program Director, FasterCures
The Bradley Center for Philanthropy and Civic Renewal and the Chronicle of Philanthropy hosted a discussion, “Is Philanthropy Going to the Dogs?,” to debate the issues of donor intent, social justices, and public interest within the context of Leona Helmsley’s $8 billion bequest made in August 2007. The session, moderated by The Chronicle’s Stacy Palmer, brought together Professor Ray Madoff, Professor Leslie Lenkowsky, Pablo Eisenberg, and Judge Robert Burk from the Hudson Institute.

Among the discussion’s highlights:

• Professor Ray Madoff from Boston College argued this bequest is actually subsidized by the taxpayers because it is stipulated to be held in perpetuity and thus avoids the payment of $3.6 billion in estate taxes to the government. So, essentially this averting of taxes is equal to the government making a matching charitable gift to causes supported by the Leona M. and Harry B. Helmsley Charitable Trust.
• Professor Leslie Lenkowsky from Indiana University countered that the philanthropists like Ms, Helmsley leverage their charitable gifts to express their values and their beliefs in what is important for the public good.
• Pablo Eisenberg from Georgetown University projected that with the mass transfer of wealth ($41 trillion dollars between 2002 and 2052) we will see the rise of mega-foundations with assets of $50-$60 billion that will operate under the direction of only a few family members. He argued that limited input into the guidance of vast volumes of resources is contrary to democracy and perhaps the installation of antitrust laws similar to those enacted in the early 1900’s to protect consumers from big business would help to protect our society from philanthropic monopolies. This concentration of resource oversight would most likely also create a funding stream for the organizations (specifically higher education, healthcare, and cultural) that are of interest to the leadership. Many of these recipient organizations are selected because they have large names and thus are presumed to be “accountable” whereas lesser-known start-up nonprofits are assumed to be less fiscally responsible. Therefore, the aggregation of philanthropic dollars under a few mega-foundations would allow the “rich to get richer and the poor to get poorer.”
  

Such a provocative discussion begs you to ask whether philanthropy is indeed going to the dogs.

Amidst all the talk, what struck me most is the apparent need for increased transparency in the philanthropy marketplace to better inform giving and more effectively channel funds to where they are most needed and can make most impact. The FasterCures Philanthropy Advisory Service intends to do this for biomedical research nonprofits such that large organizations like Alzheimer’s Association and smaller organizations like Cure Alzheimer’s Fund are evaluated equally on their approach and execution in accelerating research in the Alzheimer’s disease area.

Eisenberg closed the session noting that despite the potential threats to philanthropy, we should remember that foundations should remain in a “special place” in our economy because they fund innovation, new ideas, and high-risk opportunities that others are unwilling to do. FasterCures has echoed this unique ability and responsibility of nonprofit disease research to tackle the high-risk yet high-reward translational research that the government and industry are not inclined to fund. Although philanthropic dollars only account for about 2% of the R&D investment in the life sciences, it is an invaluable piece of the funding pie. We hope our efforts to inform charitable investment, build collaborations, and promote knowledge-sharing across sectors keep philanthropy from going to the dogs for its true value is the cat’s meow.

InnoCentive and FasterCures Align to Seek Help in Finding Innovative Treatments and Cures for Major Diseases

The first Challenge posted on InnoCentive will seek ideas for encouraging more investment and collaboration on biomarker research within the biopharmaceutical industry to accelerate medical solutions

Waltham, Mass. and Washington, DC — InnoCentive, Inc., the Global Innovation Marketplace, and FasterCures / The Center for Accelerating Medical Solutions, today announced that they have partnered to seek solutions from the public that advance medical research, encourage faster development of new diagnostics, and enable personalized medical treatments. The just-released Challenge seeks innovative ideas for spurring biopharmaceutical industry investment and collaboration in biomarker research.

News Highlights

• The first Challenge will seek ideas that encourage companies to invest and collaborate more in biomarker research and qualification.
• According to the Institute of Medicine, a biomarker is “any characteristic that can be objectively measured and evaluated as an indicator of normal biological or pathogenic processes, or of pharmacological response to a therapeutic intervention.”
• Biomarkers are used by medical professionals to determine proper diagnosis, prognosis and the optimal course of treatment for a patient. Furthermore, biomarkers are widely viewed as a critical technology to personalizing treatment choices and maximizing the impact of medical treatments.
• Biomarkers are also a pivotal part of biomedical research process and can shave years and millions of dollars from the research and development process.
  FasterCures is a nonprofit organization whose mission is to identify and implement global solutions to accelerate the process of discovery and clinical development of new therapies for the treatment of deadly and debilitating diseases.
• This Challenge will be posted in InnoCentive’s Global Health Pavilion, a section of the website that focuses on Challenges that contribute to solving some of the world’s biggest Health challenges.
• In support of its mission to change the world and influence the lives of people everywhere, InnoCentive is providing shared funding for this Challenge as its initial philanthropic initiative in open innovation.
• The partnership will allow FasterCures to reach InnoCentive’s network of more than 160,000 Solvers based in 175 countries worldwide.
• This is one of many partnerships InnoCentive has had with nonprofit organizations to encourage solutions to world problems. Other partners include: GlobalGiving, Prize4Life, the Rockefeller Foundation, the ASSET India Foundation, and the Rural Innovations Network.

Executive Quotes
Gregory C. Simon, President, FasterCures

“Accurate and reliable biomarkers can shave years and millions of dollars from the biomedical research process. But the healthcare industry has little incentive to invest in biomarker research and development and keep the results of their investment in the public domain, where it can do the most good. Reducing the ‘first-mover disadvantage’ in biomarker research and validation can significantly accelerate the search for cures and improved treatments for deadly and debilitating diseases.”

Tom Venable, Executive Vice President, InnoCentive, Inc.

“We are excited to announce this partnership as our first philanthropic challenge where InnoCentive is providing award funding as part of our mission to invest in causes that our employees and shareholders are passionate about. We believe FasterCures is a great organization to partner with as any ideas generated will be shared with research organizations across the disease spectrum. Over the years we’ve found that some of the best solutions have come from the most unexpected sources and by using our solver community, FasterCures will increase its likelihood of finding great ideas to change the economics and speed of biomarker development.”

For more information contact:
Cecilia Arradaza, Communications Director, FasterCurescarradaza@fastercures.org or
202-336-8912

FasterCures in Esquire’s 75th Anniversary Issue

by Greg Simon, President, FasterCures

Esquire’s 75th Anniversary Issue Features FasterCures Public Service Announcement, FasterCures Chairman Mike Milken as 75 Most Influential People of the 21st Century, and FasterCures Esquire House Event

Esquire's 75th anniversary issue released Sunday debuts the FasterCures public service announcement, Fifteen Years: That’s how long it takes to develop new medical treatments. The ad highlights FasterCures’ core principle of saving lives by saving time. Also in this issue, Esquire names Mike Milken, Chairman of the Milken Institute and Chairman of FasterCures, as one of the 75 Most Influential People of the 21st Century citing his decades-long commitment to improving the medical research infrastructure to accelerate the development of cures.

"FasterCures is honored to be featured in this special Esquire edition and grateful for Esquire’s steadfast support of our mission," said Gregory C. Simon, FasterCures president. "FasterCures is committed to reducing suffering and death from disease in the 21st Century and the 21st Century begins now."

Since 2003, Esquire has hosted a series of Esquire House events on behalf of nonprofit organizations. On November 12, Esquire House Hollywood Hills will host FasterCures, one of six organizations to hold events at this remarkable venue this year.

FastTrack Summer Issue Just Released

Be sure to check out the just released summer issue of FastTrack, FasterCures' quarterly electronic newsletter. We welcome your comments and suggestions for future issues of FastTrack.

Candidates: Can we talk about your medical research platforms?

by Margaret Anderson, COO, FasterCures

Over the next two and a half months, the two presidential candidates will have endless opportunities to give the American public specifics on their platforms. With several competing priorities vying for their attention, it seems hard to imagine that there will be much airplay for the importance of and challenges for medical research. Why is this?

One reason might be America’s low level of scientific literacy. A 2007 Harris Interactive survey of 1,304 adults found that only 26 percent believe that they themselves have a good understanding of science, and 44 percent couldn’t name a single scientist, living or dead, that they’d consider a role model for our young people. But this does not mean Americans do not care about science. In fact, another Harris poll found that 85 percent of Americans want a presidential debate on science. Maybe Americans are leaving the medical research discussions to “those” people who are in the know.

Another reason could be that medical research doesn’t rise up your priority list until your health is personally affected. We know that we’ll all be patients, some day, but if you are not yet a patient, why worry, right? People also probably don’t realize the continuum of how we get from basic research to clinical research to therapies. But as the baby boomers age, the issue of how close we are to cures continues to take on new and more important meaning.

No matter what the reason, the fact remains that Americans need to hear more than just the specifics of the health care reform platforms that focus mainly on economic issues. They need to hear about a care– to–cure platform, and our nation’s leaders need to answer the question Michael J. Fox posed, “Who is in charge of finding cures?” The list of challenges is long: flat funding for NIH, concerns about retaining the next generation of researchers, questions about the appropriate balance between basic and translational research at the, low levels of funding for FDA, inability to get the Reagan-Udall Foundation off the ground because of political games, need for research to be included in discussions of electronic health records...the list goes on.

We are part of ongoing discussions with many organizations about these issues and have several initiatives to address these problems. We are one of the partners supporting Research!America's voter education initiative Your Candidates. Your Health, which highlights presidential and congressional candidates' positions on health and research.

We welcome your thoughts on how to shift the debate from one focused on health care to one encompassing care and cures.

FasterCures' Ten to Watch Mid-Year Review

by Greg Simon, President, FasterCures

FasterCures started the year with its Ten to Watch in 2008. Before summer's end, we wanted to share some of our insights on the ten things we've been watching (other than the Olympics) and how they are faring.

1. Return on philanthropy. This fall, FasterCures will preview its Philanthropy Advisory Service, which will help philanthropists decide where their contributions will have the greatest Return on Philanthropy – the greatest impact in finding new cures. The Gates and Robert Wood Johnson Foundations are supporting this effort to create an information marketplace to improve the efficiency and productivity of both the philanthropic investor and the nonprofit disease research organizations that depend on such support. Watch this space for updates.

2. The FDA. It hasn’t been a great year for FDA. In the first half of 2008 alone, the agency had to deal with contaminated heparin from China, salmonella-tainted jalapenos from Mexico, and a Supreme Court decision that has made it a shield protecting medical device manufacturers from lawsuits by patients. The silver lining is that all this bad news is focusing attention on the agency’s critical importance and woeful lack of resources. FDA chief Andy von Eschenbach even broke ranks with the Administration and confessed that the agency needs a larger budget than requested to do its job. But it needs more than that, starting with Congressional leadership that supports it constructively rather than attacking and denigrating it. A key step would be to fund the Reagan-Udall Foundation, and realize FDA’s Critical Path initiative.

3. Research: Lost in translation. The call for more translational research is now coming from a wider range of stakeholders, including interesting messengers such as Katie Couric. In a recent interview about the September 5, 2008 Stand Up To Cancer fundraising effort on “Larry King Live,” Couric said that its focus will be supporting research that has practical applications and patient-relevant outcomes. She noted that the funding will be"...focusing on cutting edge cancer research, which will hopefully inform people about how close we are to new therapies and new approaches and how…scientists need additional funding so these can go from the lab to the clinic and hopefully, eventually, save a lot of lives.”

4. Science 2.0. On June 3rd, members of FasterCures’ TRAIN network of disease research foundations spent some time with two innovators in the use of online platforms for scientific collaboration. June Kinoshita, Executive Editor of Alzheimer Research Forum, a pioneering e-community for Alzheimer’s researchers, is currently working on an ambitious project called Semantic Web Applications in Neuromedicine (SWAN), which will create a common semantic framework to allow researchers to more quickly and seamlessly integrate and combine data from diverse sources rather than just exchange documents. Jeff Shrager, Chief Technology Officer of CollabRx, demonstrated its product – a “virtual biotech,” a Web-based collaborative research platform that would enable funding organizations and research teams to manage, track, and prioritize their operations as well as to share data, knowledge, resources, and services. CollabRx is also working with Science Commons on its Health Commons initiative, which envisions a virtual marketplace or ecosystem where participants share data, knowledge, materials and services to accelerate research.

5. Crowd-sourcing innovation. FasterCures is seeking to benefit from the “wisdom of the crowd” by posting an “ideation challenge” on InnoCentive’s Web site this fall, as one of the company’s first “Public Policy and Citizens in Action” challenges seeking solutions to important issues facing society. The New York Times reported in July how this approach is “catching on…as would-be innovators can sign up online to compete for prizes for feats as diverse as landing on the Moon and inventing artificial meat....” Keep an eye out for more details – maybe you’ll have some ideas to offer and potentially win the prize!

6. Prize philanthropy. Top-down prizes are getting a lot of attention this year, not only by the press but by politicians. A panel appointed by the National Research Council recommended that the National Science Foundation offer prizes of $200,000 to $2 million in various areas of science to encourage innovation. Two weeks ago, The Wall Street Journal challenged political and business leaders to outline how they’d spend $10 billion in four years to address global problems. Newt Gingrich proposed the development and diffusion of new technologies by using large, tax-free prizes, arguing that prizes would be a useful experiment in large-scale breakthroughs.

7. Biomarkers Consortium. The Biomarkers Consortium public-private partnership has taken the interesting step of identifying “High Impact Biomarkers Opportunities.” Rather than be, essentially, investigator-driven and wait for participants (industry, academia, foundations) to walk in the door with good ideas, the Foundation for the NIH has chosen to develop this more strategic and prescriptive path, laying out projects that it believes “will have the greatest and most proximal impact on future diagnosis and treatment of patients and drug development.” We will continue to watch with interest to see if this effort proves to be an effective way of addressing the “first-mover disadvantage” in biomarker research and validation – i.e., the first mover spends all money and its competitors get the advantage of the biomarker.

8. Comparative effectiveness. The healthcare reform drumbeat is getting louder -- and much of the call for change is focused on the need for more cost effective and efficient approaches to our nation’s healthcare woes. Despite the buzz around comparative effectiveness, it’s important to note that both presidential nominees recognize that this issue is more complex than it may appear. In response to a question on the issue posed by the “Your Candidates - Your Health” program (of which FasterCures is a sponsor), Sen. McCain said we need “to ensure that [this] does not stifle the spirit of innovation,” and Sen. Obama cautions that the “decision on which treatment to use should be made by patients and doctors, not government or insurance companies.”

9. Electronic health records (EHRs). As the federal government muddles through its continuing effort to figure out what its role in the development of the “healthcare information superhighway” should be, there are at least small signs that it might be waking up to the importance of factoring research uses of EHRs into their plans. FasterCures recently joined a panel of research leaders that called on the group charged with creating the successor to the American Health Information Community to expand its scope and mesh data standards for medical research with its work on standards for e-health records. The group said it would consider how best to do that. Meanwhile, the FDA has launched its Sentinel initiative to better monitor medical product safety, a massive effort that will involve using existing electronic health records for post-marketing surveillance of approved drugs.

10. Clinical trials: Something’s gotta give. The discovery enterprise is calling for revolutionary change in how we conduct clinical trials. The NIH is studying how to accelerate and improve the conduct of clinical trials at its own Clinical Center, which could provide useful information and models to other trial sponsors. The Pioneer Portfolio of the Robert Wood Johnson Foundation (which supports FasterCures’ Philanthropy Advisory Service project) has made its largest grant to build ARCHeS, a Web-based interface and delivery system that will allow far more health and policy decision-makers to use the Archimedes simulation model – a sophisticated database that some believe could be used to radically streamline the design and simulation of clinical trials. This is definitely an area worthy of focused attention by the next President.

August Days

by Greg Simon, President, FasterCures

This August my days are focused on preparing to send my first born to college. Seven years ago this week, I was focused on a very different journey -- my father’s passage from this life at the age of 91 after a short struggle with small cell lung cancer. He died on August 27, 2001, a date notable because it spared him in his last days the trauma of seeing the calling card of 21st Century terrorism on September 11 that year.

I have thought often of my father’s death in the context of my work at FasterCures. He was a model of “compressed morbidity” – living long and dying quickly, rather than fading out over many years. He changed his behavior after a small heart attack in his fifties, brought on no doubt by smoking which he promptly quit. He walked home from his grocery store on Main Street every day for lunch and a nap. He ate healthy food, didn’t drink, and – in addition to the walking commute to the store – exercised by shadow boxing in front of the bathroom mirror every morning.

Despite his personal good habits, he became a victim of the healthcare system of small town America. When he complained about “tingling” in his legs, his doctor gave him Valium to which he promptly became addicted to the point of needing sleeping pills at night since he was medicated all day. When my sister and I discovered this we intervened to switch him to Motrin gradually until finally he didn’t need the Valium. A few months later on another doctor’s visit, the same doctor put him back on Valium.

The few times in his later years he was admitted to the hospital for minor operations, he was given Demerol which causes you to go psychotic if you are on Valium. But, of course, in those days of paper records (these days too, I should point out), the hospital didn’t know or negligently failed to notice or care that he was on Valium. They found out when orderlies had to chase him down the hall, trailing his feeding tubes, to get him back to bed. I once had to block him from trying to walk through a wall in his hospital room to what he thought was his dining room at home.

His cancer diagnosis brought a completely different set of challenges. First, it was amazing he was diagnosed at all. There were no oncologists in Blytheville, Arkansas then. Two doctors came over from Memphis twice a week on Tuesdays and Thursdays. As I testified at Sen. Kennedy’s hearing on cancer several months ago (before his own diagnosis), in Blytheville, you could only get cancer on Tuesday or Thursday. But with the diagnosis came the Scylla and Charybdis – chemo or radiation, since that was what the two doctors offered.

How was a patient 91 years old living in a small town supposed to make that decision? Fortunately my father knew people who knew people – that would be me. I called Al Rabson at the National Cancer Institute who was well-known in Washington for offering helpful advice on such matters. He reviewed my father’s records and X-Rays, reviewed the two doctors and their training and gave me the news. My father would die from this cancer but he did not need to be in pain, so skip the chemo, focus on radiation to keep the lungs clear enough to avoid pneumonia and enjoy the last months as much as possible.

Of course, giving effective pain medications to someone who has been addicted to Valium for years is not easy. We went through “pain patches,” Oxycontin and finally morphine. After six months, my father slipped into a coma which gave him release from the pain. After a few days, the hospice nurse – a most miraculous find in my small home town – told us “Your father is going to die in the next thirty minutes.” And as my mother and sister and I sat there, we experienced that ineffable but palpable difference when the quick becomes the dead.

I have often thought how different his death would have been if he and my mother had been on their own. How confused they would have been in dealing with the cancer; how painful the end could have become. I have thought too how strong he must have been to survive the bad medical care he received all his life in the Arkansas Delta and to still live to be 91.

It is simply not acceptable or tolerable that we still are killing people with medication errors in hospitals (both by giving them the wrong drug or by giving the “right” drug to the wrong person). It is inexcusable that the best cancer care is still available primarily only to people who know people – no matter how many people those people, including me, try to help.

My father didn’t live to see me launch FasterCures with Mike Milken five years ago. But his life and the circumstances of his leaving it launched me on this path and live with me every day. Through our work I hope we can improve the lives of people who don’t know people, of people who live in places where you can get cancer only on Tuesdays, and of people whose desires to live a full life and avoid a long, painful decline into death depends on the kindness of strangers.

Electronic Health Records: Linking the Care and Cure Communities

by Cecilia Arradaza, Communications Director, FasterCures

"When you go visit your doctor, only three people know what happens during
that visit: you, your doctor, and your insurance agent. None of these people are
responsible for finding a cure for your disease…and none of them are necessarily
connected to the people working to find a cure for your disease… How do we
integrate the care community with the cure community?" -- Greg Simon,
FasterCures President, in remarks to the American Health Information Community, 7/29/2008

Greg joined an expert panel of medical research leaders and advocates recently to call on the American Health Information Community (AHIC) to ensure that the nationwide health information system being built can improve patient care and enable medical research.

In the ongoing discussion surrounding the creation of a national health information technology (IT) system, medical research lurks in the shadows of privacy and interoperability. At the AHIC meeting at the end of July, the medical research community made a resounding call for AHIC or its successor organization to put in place a supplemental process to ensure that the nation’s health IT system is built to improve both healthcare delivery and medical research – and to do so quickly. See coverage of the AHIC meeting.

Ensuring utmost privacy protections and efficient interoperability capabilities are critical to the success and sustainability of a health IT system. As we build the system’s infrastructure, it is just as critical to consider how best to utilize patient data to improve healthcare delivery, enable medical research, and accelerate cures.

There will be new opportunities for health information exchange to support clinical research activities as electronic health record (EHR) adoption increases and a national standardized system evolves. Incorporating medical research into a national health IT system is inevitable. As AHIC transitions into its next entity, it has an important choice to make: to implement a health IT system that protects privacy and ensures operability now, only to rebuild that system to better support medical research OR; to implement a comprehensive, interoperable health IT system with the highest privacy standards and a strong research capacity already in place.

FasterCures believes accelerating cures must be a goal of the emerging Nationwide Health Information Network in addition to improving patient care. While the focus of most efforts to do so has been on improving care by limiting costs and medical errors, the real savings, in terms of both reducing healthcare costs and, more importantly, in eliminating human suffering, will come from curing disease and from limiting its damage.

Related Blogs: Health IT's Real Savings
More information on FasterCures recommendations for using EHRs to bridge patient care and research

The AD Challenge: Hitting the Right Mark

by Jeongyeon Shim, FasterCures, Domestic Program Analyst

Last week, I attended "Early Detection of Alzheimer’s Disease: A Virtual Town Hall*,"organized by the Alzheimer Research Forum and the Alzheimer’s Study Group, sponsored by the Geoffrey Beene Foundation Alzheimer’s Initiative. Moderated by Dr. Harold Varmus, the meeting featured presentations from seven eminent Alzheimer’s researchers, highlighting the importance of an antecedent biomarker and providing an update of ongoing efforts, using media such as imaging, blood, or cerebrospinal fluid.

While the importance of biomarkers is well known, the importance of reliable and low-cost antecedent biomarkers for Alzheimer’s disease (AD) is paramount and should be appreciated in the context of the disease. First, AD is an irreversible neurodegenerative disease—once clinical symptoms appear, the neurons that govern the affected functions are permanently lost. This means that even if we had a cure that addresses the cause of the disease, it will not necessarily revive the lost functions for later-stage patients—unless we also find a way of regenerating lost neurons. Early treatment through early detection is not only ideal but critical in reducing the burden of the disease—preventing the clinical form of the disease.

In addition to its limitation in detecting clinical symptoms, the current AD diagnostics are not always accurate nor available. AD diagnosis based on clinical symptoms has an accuracy of over 90 percent when conducted by experienced clinicians. Unfortunately, this is often not the case for mild AD, when distinction with mild cognitive impairment (which does not progress to dementia) may not be as obvious. And, "experienced clinicians" are a scarce resource. Instead, primary care physicians, thrust on the frontlines of AD care, may not necessarily have the tools and training to recognize AD. With these factors, it is more important than ever to develop a reliable and widely-available diagnostic tool

We discussed Dr. Lee Goldstein’s effort to develop an eye exam to diagnose AD a few weeks ago. And last week, I was pleased to see that scientists are pushing forward multiple other efforts. Identification of multiple markers will allow us to choose a marker that best fits the purpose—whether it is for diagnosis, management of risk factors, or measurement treatment effectiveness. Such efforts exactly match FasterCures’ goal—to accelerate the process of discovery and clinical development of new therapies. We look forward to see many more such efforts, not only in AD but also in other diseases.
*The proceedings from the Town Hall will be made available at www.AlzStudyGroup.org in the near future.



Read and Comment

When It Comes to Nonprofits, Does Location Really Matter?

by Jeongyeon Shim, Domestic Program Analyst, FasterCures

Recently, Charity Navigator published its sixth annual Metro Market Study. The study ranks 30 different metropolitan areas based on the financial performance of nonprofit organizations in each area. Based on the aggregate performance of nonprofits for each metropolitan area, the areas are ranked according to CEO compensation, level of financial reserves, volume of funds raised, and other Charity Navigator financial metrics.

Some nonprofit experts and concerned individuals have highlighted, in various venues, the limitations of Charity Navigator’s focus on financial performance in evaluating nonprofit organizations. Financial metrics based on data from tax forms have the advantage of being available for most organizations, but they provide limited insight into an organization’s actual impact or potential. For this reason, FasterCures’ Philanthropy Advisory Service (PAS) program focuses on the qualitative aspects of nonprofit disease research organizations, including their strategies, management policies, governance structures, and results-to-date.

I am concerned that the Metro Market Study’s focus on financial metrics may provide an unbalanced view of the performance of nonprofit organizations and wonder about the consequences of prioritizing geographic location above all other considerations. What insight does this singular focus on location convey?

Some nonprofit organizations, especially those serving local communities, operate on a local level, and for those organizations, location does affect both their missions and financial performance. However, there are also a sizeable number of organizations whose missions are to serve on a national or even global level. Therefore, the importance of location is different across the types and missions of the organizations—which does not seem to be taken into account in the Metro Market Study.

Fundamentally, I question the value of using location as the first criteria to analyze the financial performance or concluding that charities operate according to the general practices of their metropolitan areas. For example, the focus of FasterCures’ PAS program is on nonprofit organizations engaged in medical research and none of the organizations to be studied aim to develop a treatment only for the residents of the community where they are located, rather they aim to find cures for all. This is true not just for the mission of the organizations, but also for its financial aspects. For example, all of the five organizations ranked as raising the most funds through special events, as reported by Charity Navigator’s Special Event Study, conduct their fundraising events across the country, rather than just in the community where they are headquartered.

Another concern I’d like to raise is the ultimate value of the information produced through this study. Should a nonprofit organization based in Detroit, the metropolitan area with the lowest overall score (Charity Navigator’s measure for overall financial health), move to Miami, which has the highest overall score, and would its financial situation improve (or can it even move given its mission)? Probably not. The same goes for donors. Should the donors make their giving decision based on the location of the headquarters or take the location as a sign of financial health? And how will this help donors interested in giving to nonprofit organizations based in their own community?

A comprehensive analysis of other factors, such as the organization’s area of focus, strategy, process, and governance structure, are probably needed and more important than headquarter location in guiding giving decisions and determining the drivers of financial health.

Charity Navigator is one of the largest databases on nonprofit organizations with a wide scope of coverage. These advantages can be used to understand the key drivers of growth and performance in nonprofit organizations and conduct meta-analyses to understand larger trends. Its analysis of the nonprofit marketplace should focus on variables that really matter—I felt the Metro Market Study fell short of living up to its potential.

NIH Asks How to Create a More Effective Clinical Trials System

The National Institutes of Health recently held a town hall meeting to brief patient advocacy groups and other external constituencies about its initiative to remove barriers to more efficient conduct of clinical trials at the Clinical Center on the NIH campus. The Clinical Center is really a national jewel, the largest hospital in the world dedicated exclusively to research (and therefore a major sponsor of clinical trials). It has almost 2000 active protocols in which tens of thousands of patients participate with the goal of advancing our understanding of disease and, one hopes, developing more effective treatments.

But even though the Clinical Center has spectacular resources compared to many clinical trials sites around the country -- starting with its beautiful new building and state-of-the-art lab space in Bethesda -- it experiences all the same frustrations with the process as everyone else. Regulatory burdens increasing. Inconsistent policies and practices among Institutional Review Boards -- even within NIH there is no uniformity, and different Institutes and Centers handle things differently! Flat research funding. Declining numbers of clinical researchers. It hopes through its study of the problems on its own campus to inform the debate outside its walls, in the extramural research community. The goal of the NIH's review is to share best practices in conducting trials across the institutes and centers within NIH, and to identify over-interpretation of applicable regulations. Officials who spoke at the event seemed confident that many of the barriers were not externally imposed but were rather due to differing interpretations of regulations and the sheer volume of changing requirements, with which everyone finds it hard to keep up.

The NIH's Intramural Research Program -- the research conducted on its campus by NIH-employed scientists -- of which the Clinical Center is a part, is a little recognized critical resource in our taxpayer-funded research portfolio. At its best it provides a model of cutting-edge biomedical research for the extramural, mostly academic research world, taking on the highest-impact science and suggesting solutions to systemic challenges. We hope that this effort to plow through the thicket of complaints and paperwork that surround clinical research and clinical trials will be such a model for the rest of us.

-- Kristin Schneeman, Program Director, FasterCures