Solutions not challenges

by Margaret Anderson, Executive Director, FasterCures

The President’s Council of Advisors on Science and Technology (PCAST) released its “Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation” this week.

The punch line of the briefing came toward the end when Steve Galson from Amgen opined that “There are rooms of reports on innovation and the challenges – what will differentiate this one?” It was helpful to have it said out loud, because I felt a sense of déjà vu listening to the presentations and perspectives. Things may look tough now, but they’ll be much tougher if we don’t wake up and smell the coffee.

• We heard about how innovation has played a huge role in advancing the health of our nation, but that the system is under stress.
• We heard about the key role the FDA plays in influencing the ecosystem’s behavior, but even with changes at the agency, more needs to be done.
• We heard about how collaboration is central to future discovery, but the system doesn’t consistently incent it. 
• We heard about unmet need in the form of Alzheimer’s, treatment for psychiatric diseases, cancer, and heart disease and stroke, but that we have been making some inroads with treatments for diseases like HIV/AIDS. 
• We heard about how the entire system is struggling for many reasons – funding, patent cliffs, low-hanging fruit being picked, and overall costs of drug development rising, but that there are byways being built. 
• We heard how patients and venture philanthropy are central to solving the problem, but there aren’t groups in existence capable of taking on all the challenges. 
• We heard there has been progress – at times breathtaking progress – but that there is still a long way to go to reap the benefit of the scientific promise that lies ahead.
• We heard how we need money to do all of this, but we don’t know exactly where it’s going to come from, especially in these tight times.
• And we heard we’d better get moving.

There is a shift underway; during the briefing we heard people like Todd Sherer, who is the CEO of the Michael J. Fox Foundation for Parkinson’s Research, saying he’s optimistic based on things he’s seeing. And Bob Beall, president and CEO of the Cystic Fibrosis Foundation, talking about the game-changing approval of Kalydeco and the need to look at the PCAST report with the express purpose of figuring out what recommendations would speed things up. There was some discussion of consumers and patients needing a seat at the table. Well, we learned doing our report “Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change” that you don’t get handed a seat at the table, you earn it. And you earn it by getting smart about the system and what solutions look like. I’d like to see us moving beyond giving patients lip service to ensuring we’re having a real dialogue with them about what they are already doing, and what is possible if and when they are engaged. As Sherer reminded us, “patients want to be part of a solution.”

We know current resources are threatened by the possibility of sequestration, which would automatically cut the federal budget in January 2013. Future resources to address these pressing needs would also be slashed.

The PCAST report cites two primary needs: 

• better methodologies and tools to translate basic biological insights to validated therapeutic targets and leads, and
• the need to have a more efficient clinical trial system, as it currently constitutes 40 percent of the biopharmaceutical R&D budget.

It posits recommendations in areas of improving drug discovery and development; improving drug evaluation; monitoring and communication about benefits and risks; improving FDA management; and economic incentives. There’s a timeframe for action and a list of steps to be taken. We need to assess how this gets done, which entities can and should do it, in what timeframe, and also look at what agencies like the FDA are already beginning to do under the recently enacted PDUFA V agreement, and perhaps most importantly how to get the resources to do it. We were probably all raised with the phrase “money doesn’t grow on trees,” so we need to figure out creative ideas that will focus attention on the imperative for funding.

By focusing on specific solutions and not just the challenges, this report is different than many before it – but it can’t be put on a shelf, action must be taken. As my husband often reminds me, “How do you eat an elephant? One bite at a time.”

Healthcare’s Digital Pioneers

A distinguished panel of health technology pioneers at the Milken Institute Global Conference gathered to discuss the digital revolution in healthcare and agreed that, with the right drivers, mobile health tools could help close the gap between patients and providers.

Painting a picture of what a physician’s visit might looks like in 10 years, panelists agreed that it will be patient-centric and patient-driven, a partnership between patient and provider, and fueled by data captured in real time.

“What’s missing right now,” said Frank Moss, former director of the MIT media lab, “is that people have to understand the power they have, and be engaged in unleashing the creative potential of technologies that speak to their interests and needs.” He described it as a “cognitive apprenticeship” where the doctor becomes the mentor and the patient the apprentice.

Eric Topol, cardiologist and director of Scripps Translational Science Institute, showed a few mobile technologies that are now available to better improve patient care, including a device that captures heartbeat patterns and emails them to your doctor, and a glucose monitor attachment to your smartphone. “We have to get over the reimbursement-centric, doctor-knows-best paternalism, and introduce some creative destruction into the healthcare system,” he said.

John Dwyer, chairman of Telcare, agreed that broad adoption of these tools will depend greatly on incentives. “It’s all about the data – providing for patients and caregivers in the cloud,” he said. “People should be able to get their data when they want it, not be limited because of the few, or no, reimbursement options for doctors to manage their patients’ health remotely.” Dwyer also pointed out that the financial industry invests twice as fast and twice as much in technology as healthcare does, and that medicine could learn a lot from their model. “Information is powerful, but the wallet plus information is a winner – market takers will become market makers.”

Kate Black, staff counsel at the Center for Democracy and Technology, reminded the group that even with all the potential for good, if we can’t build trust among patients within the digital ecosystem, we may not be able to realize the full range of possibilities for improved health. “We need a policy framework that protects privacy and individuals, while allowing data that needs to flow, to flow to the right places at the right time.”

Moderator Cecilia Arradaza asked the group if they thought increased access to health information would create a “generation of hypo – or ‘cyber-chondriacs,’” but Topol and others agreed that while we’ll never collectively get over some of the fears associated with our health, having access to your own personal health information will be far more empowering than distracting.

View the video to see some of the latest tools discussed.

Read Margaret Anderson's Blog on The Huffington Post

Top 10 Medical Research Trends to Watch in 2010
by Margaret Anderson, Executive Director, FasterCures

This Top-10 list, assembled by Margaret Anderson, Executive Director, FasterCures, reveals some likely medical and scientific trends that will affect research in 2010, including FDA regulation, healthcare, information technology, and new methods in clinical trials and research. Read more.

'Tis the season

'Tis the season for digging out from under and clearing off the piles and the clutter. 'Tis the season for looking back at the year that flew and planning ahead for a year that's new.

At FasterCures, we will launch our new strategic plan in the coming months, an effort to help us "stay in our lane" and focus on much needed outcomes towards accelerating medical solutions. As FasterCures board member Larry Flax has said, "That which gets measured gets done."

As we further define our strategic priorities and implement outcomes-driven programs, here’s a preview of what’s on our agenda for 2010:

• Fostering innovation and accountability in medical research.
• Creating a more informed, transparent, and results-oriented marketplace for medical philanthropy.
• Empowering patients to play an active role in clinical research.
• Exploring and expanding opportunities at the Food and Drug Administration for acceleration of cures.
• Strengthening medical research tools (e.g., electronic health records, biobanks) to better support a more effective and efficient health cures system.

We look forward to hearing from you in the new year, and working with you to advance our mission of saving lives by saving time. Wishing everyone health and a multitude of outcomes that advance cures in the new year.

Protecting Patients, Protecting Privacy?

Susan Semeleer, Associate Director of Communications, FasterCures
I consider myself a pretty savvy patient. Since being diagnosed in my teens with a rare heart condition, I have seen at least a dozen specialists, undergone still more tests and procedures, and openly provided my most sensitive information to medical personnel. I remember when I first started getting with every procedure goldenrod yellow authorization forms on which my caregivers pledged to guard the privacy of my health information. My first reaction was, “What had you been doing with it?”

Last week, I attended my first FasterCures briefing – a discussion of a new Institute of Medicine report entitled Beyond the HIPAA Privacy Rule: Enhancing Privacy, Improving Health through Research. In 1996, Congress enacted the Health Insurance Portability and Accountability Act, which called for a set of federal standards for protecting the privacy of all personally identifiable health information. The statute attempted to walk a regulatory tightrope: preserving a patient’s reasonable expectation of privacy, while maintaining the free-flow of information necessary for high-quality health care.

The FasterCures panel was the second in a new series of discussions on issues of importance to facilitating medical research. The fact is, though we at FasterCures have been hearing HIPAA bemoaned as an impediment to research since we launched in 2003, the IOM study is the first attempt to gauge its full impact.

Biomedical researchers use health information culled from sources like patient registries, medical records, biobanks and government-supported statistics databases; the HIPAA Privacy Rule was enacted to ensure that patient privacy was not compromised by the compilation of these data. But, according to the findings of the IOM report, HIPAA has compromised both patient privacy and medical discovery, and needs to be revamped if it is to serve the interests of patients as well as researchers.

Panelist Marc Boutin is Vice President of Policy, Development and Advocacy for the National Health Council, and a member of the IOM committee. He says that on the question of medical privacy, a vast difference exists between public perception and legal reality; the average patient assumes a level of protection under the Privacy Rule that simply doesn’t exist, and entities covered under the Rule inconsistently interpret the definition of “privacy.” IOM calls for a revamping of HIPAA, which would include an entirely new framework for health research that would enhance protection of medical data through improved security, transparency and accountability – not just a one-size-fits-all provision that, in reality, fits no one.

What it seems HIPAA has managed to do is create a labyrinthine tangle of regulations, codes and procedures that have thrown roadblocks on the path to medical discovery. The heaviest unintended consequences of HIPAA are felt in research that is abandoned, and opportunities that are lost. In its efforts to protect privacy, HIPAA has hamstrung basic components of medical research, increased research costs, and rendered useless a lot of biomedical data that could help cure disease. What’s more, according to an IOM-commissioned survey, only 13 percent of respondents said they did not want their medical data used “under any circumstances.” What IOM calls for is a mechanism that respects the wishes of those patients, while making it possible to gain safe, transparent access to the potentially-critical medical data of patients who are willing to share it. The key here is accountability, and sensible, practical distinctions between interventional research, and research that is exclusively information-based.

I want my privacy protected. I don’t want my family, my friends, the government, my employer, or my nosy next door neighbor to have the ability to troll my medical data. That said, I came to work for FasterCures for a reason: to dedicate my professional life to finding cures for diseases that kill and cripple. If my medical information is shared on a “need to know” basis – and if that hurdle is sufficiently high – then I have absolutely zero objection to clinicians and researchers seeing my blood work or echocardiograms. Have at it. Find a cure. Save a life.

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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