The UK Biobank: Showing the World How to Bank on Trust

By Margaret Anderson, Executive Director, FasterCures

If you have ever had blood drawn, been biopsied, or had surgery, chances are good that some piece of you, even if microscopic, is stored somewhere in a healthcare facility or laboratory. Most states have laws requiring that laboratories retain such materials for a period of time for clinical, diagnostic, and legal reasons. Current regulations allow those facilities to use those materials for research, so long as they are no longer needed for your care and all identifying material has been stripped from them.

Human biological material—whether cells, tissues, organs, or subcellular structures such as DNA—have become one of the most valuable research resources available to science. Although scientists have long studied human cells to understand, diagnose, and treat disease, technological breakthroughs and powerful new molecular techniques allow cellular information to be mined in ways never before possible.

However, their use raises ethical issues because such material also contains information that is unique to the individual from whom it came —for example, the existence of tiny mutations that affect health and predict future disease.

The increasing demand in the biomedical research community for access to large collections of human biological materials has led to the creation of “biobanks.” Sometimes, technical and scientific needs dictate that material be collected prospectively for current and future studies. Other times, existing collections, of which there are thousands, can provide material of sufficient quantity and quality to explore some fundamental research questions.

Yet, the debate about biobanks continues, mostly centered on whether donors could be harmed in some way by the information contained in the material or by a database getting into the hands of someone who does not have the best interests of the donor in mind or who does not know how to interpret the information. While the scientific value of biobanks is not at issue, what remains contested is how much individuals need to be told, when they need to be told, and what they can be allowed to decide for themselves about how materials will be used in research.

In response to these concerns, numerous regulatory and statutory requirements have made our current biobanking system nearly unworkable. The growing systems of protections of privacy and the tendency to allow individual autonomy to trump generosity have created obstacles that are onerous, expensive, and excessive when compared to the level and likelihood of risk. Privacy rules make it difficult, if not impossible, for researchers to share data across institutions. Current policy proposals could make research even more difficult, if enacted, by insisting that consent be obtained for research use of all materials, even those that have been stored for years, even decades, and for which the identity of the donor has been entirely removed.

If we want to be able grow biobanks, and reap a research return on what we, as healthy volunteers and patients, invest in them, we need to build a system of trust, not distrust. The UK Biobank is a large-scale model of just how to do that.

The UK Biobank is a massive national effort, with nearly 500,000 participants between the ages of 40 and 69 registered to participate. It is a registered charity, funded by public and private interests. To gain public trust and encourage participation, the UK Biobank has undergone rigorous review and consultation at all levels. The biobank sought approval in England and Wales from the Patient Information Advisory Group—representatives of patient groups, healthcare professionals, and regulatory bodies—for gaining access to information that would allow the project to invite people to participate. In part, the project’s success is due to the availability of national healthcare in the UK, which eliminates fears about loss of insurance and access to healthcare based on pre-existing or emerging medical conditions. The British public has been eager to participate.

Recently, the UK biobank announced another move that will enhance the return on investment. For a nominal fee, it will provide more than 1,000 bits of data on each of its volunteers to any scientist, regardless of nationality, aiming to understand the development and treatment of human disease. Prime Minister David Cameron has embraced the goal of turning every user of the UK’s National Health Service into a “research patient,” in which biobank materials will be anonymously linked to data in the participants’ medical records.

Russian writer Anton Chekhov once said “You must trust and believe in people or life becomes impossible.” Like the UK, we need to lay the groundwork for a biobanking system in which trust prevails. The key to achieving success lies in building the foundations of trust among patients, advocacy groups, healthcare providers, and researchers. Those who are collecting and using samples for research must earn the trust of those who donate their samples.

There are a number of ways to earn that trust. Sophisticated encryption systems and public policies that outlaw medical discrimination seem preferable to slowing or restricting critical research efforts through unnecessarily restrictive interpretations of what constitutes informed consent or sufficient privacy protection. Medical progress is dependent on wide participation in research, including investing ourselves, literally, in biobanks. The benefits of donating biobank materials for research accrue to all individuals and future generations.

Relevant FasterCures Resources:
• Publication: Banking on Trust: The Future of Research with Human Biological Materials
• Webinar Archive: Bits and Bytes: Making Patient Information Available and Meaningful to Researchers

FDA Commissioner Focuses on Return on Investment in Challenging Environment

US Food and Drug Administration Commissioner Dr. Margaret Hamburg reflected on her challenging job at a May 8 meeting of the Alliance for a Stronger FDA. “We have to earn the confidence and trust of the public every day,” she said, citing the agency’s need to improve processes – streamline and modernize – while maintaining standards of safety and efficacy for the products FDA regulates. She said the FDA wants to be viewed as an “honest broker” and a responsible steward of the resources entrusted to it.

FDA is, and must continue to be, a science-based, data-driven agency. “The quality of our work and our ability to navigate the complex landscape we are responsible for depends on this,” Hamburg said. Responding to Alliance President (and FasterCures Executive Director) Margaret Anderson, who asked about common misconceptions about FDA, Hamburg expressed surprise that other entities, including government agencies, may not see FDA as a science-based agency. FDA is conducting cutting-edge research in-house that is helping people, according to Hamburg.

Hamburg said that the FDA will continue to focus on its regulatory science initiatives as part of its efforts to streamline and modernize the product approval process. Regulatory science is the science of developing new tools, standards, and approaches to assess safety, efficacy, quality, and performance of regulated products. She called it a highly collaborative effort and said that initiatives will be embedded in program elements throughout the agency – “in the texture of the work we do.”

The “Big Data” phenomenon is becoming part of the conversation at the FDA, according to Hamburg. FDA owns huge stores of data and has identified a need to develop the science of how to do meta-analysis to mine this information. The next frontier is data sharing, she said. Anderson pointed out that currently FDA cannot disclose specifics around new drug applications, so information that might provide valuable insight is unavailable. “We need to be able to find a way to open data about failed trials, subpopulations of responders, side-effects, and so on, but it has to be done thoughtfully,” Hamburg said.

FDA also has to be a global agency, she said. The world has changed since FDA’s original authorizations were drafted, and this requires some changes in the way FDA conducts business. The ability to partner with sister regulatory agencies globally is critical, particularly in the area of legal and regulatory harmonization. “We have an opportunity to provide leadership worldwide,” she said. Raising standards in the international community – particularly in developing countries – benefits everyone. 

Anderson asked Hamburg to describe how FDA is aided by interacting with the patient advocacy community. “Patients bring focus on the real world in which products [regulated by FDA] are used,” Hamburg said, “and we get critical input from patients and stakeholders on the risk-benefit calculus.” The value of benefit balanced by risk is not an absolute. It has a societal valuation, and the same judgments about risk-benefit cannot be applied across all situations.

FDA has also learned valuable lessons from the HIV/AIDS story, Hamburg said. For example, FDA is thinking about how to expand the accelerated approval mechanism to broader domains, and make it a stronger tool. FDA also recognizes the importance of patient involvement in clinical studies and is looking for ways to push clinical research closer to patients in their own communities.

With FDA’s vast and diverse set of responsibilities, one of the most challenging aspects of Hamburg’s job is coping with an increasingly tense budget situation. Identifying core responsibilities and looking at them in the context of the current budgetary reality is key. “We have to think about positioning and building for the next five to ten years – not just for the next fiscal year,” Hamburg said.

Asked about the specter of sequestration, Hamburg said she hopes for a better solution, but this is exactly why it is important to identify priorities. “We may have to step away from certain activities that we value in favor of programs that may have more impact,” she said, while noting that there is a need to protect non-user fee programs (for example, the “food side,” which does not collect user fees). Hamburg left the group with a final thought: “It is important to remember that in terms of return on investment, FDA is extraordinary.”

Engineering Value into Medical Innovation

by Kristin Schneeman, Program Director, FasterCures

FasterCures is increasingly interested in the impact that reimbursement decisions, by the federal government and private payers, are having on biomedical innovation upstream.

Developers of new drugs and devices are getting the message that it is no longer sufficient to have a novel product that’s safe and effective and gets through FDA; if the payers won’t pay, your product will flop. For better or for worse, more companies are consulting with payers earlier and earlier in the research process to ensure that they are developing the kind of evidence that will maximize the likelihood that their products will be covered by insurance.

These themes were at the top of the agenda at a recent conference on “value-driven engineering” (VdE), hosted by the Austen BioInnovation Institute in Akron (ABIA) April 23-24. As described by ABIA Founder Dr. Frank Douglas, this is “a way to simultaneously reduce health-care costs while catalyzing innovation in medical devices,” though he considers the concept to have application in the realm of pharmaceuticals as well. The key principles of VdE are “clinical utility driven by patient-centricity in demand, design, use, and function; reduced complexity in product design, and cost savings and efficiency across the health-care system.”[1]

I found one of the most thought-provoking sessions to be the one on “Clinical Utility and Patient Centricity.” Debra Lappin of FaegreBD Consulting delivered an eye-opening view of “the patient as design engineer,” highlighting the experiences of famous patients, such as the painter Pierre-Auguste Renoir, along with her own battle grappling with the debilitating effects of rheumatoid arthritis. She made a forceful case for the importance of putting the patient at the center – in fact and not only in words – of policy discussions about how to create more innovative approval pathways at FDA, and balancing risks and benefits of new therapies.

She was followed by Reade Harpham from Battelle, who provided fascinating insight into their product design process, which consults patients about their needs and preferences at every step along the way – including, for example, research on YouTube that has turned up videos of patients showing others how to disable the safety features of injectors in order to make them more useable. Developers and regulators who do not understand patients’ needs first-hand are working in the dark, no matter how much scientific expertise they may have.

Other speakers during this session discussed how to improve utility to clinicians, another key factor in achieving adoption, the critical step beyond even reimbursement. For instance, improving clinical workflow, or being able to move complex procedures out of hospitals and into doctors’ offices, are important contributors to value for clinicians.

Among the highlights of the event were the graduate students who had competed to present their value-engineered devices – an ultraportable and inexpensive fetal heart rate monitor, a lightweight and easy-to-assemble surgical table (built with parts like car jacks and yoga mats and flat-packed à la Ikea), and a cooling device for newborns suffering from oxygen deprivation made essentially of clay pots and sand. These were designed to be used in low-resource parts of the world but in all cases were nearly as effective, if not more effective, than their state-of-the-art counterparts in the developed world.

The students were astoundingly smart, sophisticated, and inspiring. And their “disruptive innovations” point to a future in which medical progress and the quality of care don’t have to be sacrificed on the altar of cost-cutting, in the U.S. or anywhere else on the globe.

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[1] Douglas, Frank L. “Innovation and value-driven engineering.” Nature Reviews Drug Discovery, vol. 11, May 2012, p. 335. 

The Race for FasterCures

Going faster, pushing the boundaries requires teamwork, collaboration, innovation, and resources. Everyone needs to be focused on the mission: whether it's accelerating the development of an important new medical treatment or making a race car go faster.

This is why FasterCures partnered with 17-year-old race car driver Andrew Palmer to launch its first Dream Cars raffle. Andrew is one of the top kart racing drivers in the U.S. and the 2010 Rotax National Champion.

At its simplest, this raffle sells for $200/ticket, each with the potential to win two extraordinary Porsches – a white 2012 911 Turbo S and a white 2012 Cayenne Turbo – and $75,000 in cash (note of clarification: ONE winner gets the two cars and cash).

At its core, this is a partnership between two nontraditional allies – a Washington DC nonprofit determined to improve the medical research system, and a senior in high school determined to make a difference.

Andrew and his team of avid motorsports enthusiasts (including his father, David Palmer and Bruce Deifik) are pledging to raise $1 million through the Dream Cars raffle. They’re building excitement for the cause by racing the Dream Cars in the Tire Rack One Lap of America race, an eight-day event consisting of 19 on-track race stages conducted at nine different venues separated by 3,350 miles. Today is day 7.

Why are we doing this? As Andrew so aptly put it, “we all know someone who could use a faster cure.”

Enter today for your chance to win the Dream Cars (plus $75,000) and support a very important and essential cause. Visit http://www.fastercures.org/raffle or call 1.855.624.4392.

The Human Genome: A User's Guide

Nine years after the successful decoding of the human genome, much of the public are still unsure about what to do with genetic information when those data become available, noted Margaret Anderson, executive director of FasterCures and moderator of a panel at last week's Milken Institute Global Conference. Anderson kicked off the discussion by asking panelists to outline key chapters, as if writing a user's guide to understanding the human genome.

George Fisher, an oncologist and director of cancer clinical trials at Stanford University, outlined the first chapter, calling it: User Beware. He said that the technology before us could be a powerful tool if it's well understood. He said that before patients and consumers get into the data, they need to understand why they bought the genetic screening test and what kind of information they wish to know and acknowledge that genetic information can be quite complicated.

Agreeing about the complexity and ubiquitous nature of genetic information, Caroline Lieber, genetics counselor at Sarah Lawrence College, said that the next chapter should focus on how information is disseminated from consumer to provider. "Consumers need a clear understanding of this information and should seek out genetic counselors to help translate the data, and offer their insights in a meaningful way," she said. She also noted that one of the best tools readily available to help understand genetic information is family history. Sometimes, she said, you can save more lives and prevent more diseases by asking for a detailed family history.

Gwen Darien, a patient advocate and member of the board of the Education Network to Advance Cancer Clinical Trials reminded the audience that it's critical to take a step back and assess the baseline level of a consumer's understanding. She proposed that the third chapter focus on educating consumers about the science of genetics. "Most people's knowledge about science stops the moment they stopped studying it in school," said Darien.

Chris Varma, president and CEO of Blueprint Medicines, said the last chapter of the User's Guide should focus on opportunities and challenges, noting that the cost of sequencing a genome is a "huge paradigm shift." In 2001, it cost $100 million to sequence a human genome, and today the Beijing Genome Institute can apply the same methodology for $1,000. He said that the challenge in front of us is to simplify this information and help clinicians understand the possibilities. And all this information presents us with tremendous opportunity to improve our understanding of disease to better manage it.

Fisher added that with the healthcare system we have, today's physician is neither positioned nor equipped to provide the counsel necessary to make genetic information meaningful. Panelists agreed that there's a lot of ambiguity. But as these tests become more available, providers and genetic counselors must step up to the task of managing this information to improve health outcomes and better predict disease probability.

Will the Supreme Court Affect Investment in Healthcare?

What will the Supreme Court decide about the Patient Protection and Affordable Care Act (PPACA)? A panel of experts at the 2012 Milken Institute Global Conference agreed that no matter what the court’s decision, opportunities for investment in healthcare will continue to abound.

Kneeland Youngblood, founding partner of Pharos Capital Group, predicted with some hesitance that the law will be upheld. But even without the law, he said, many of its provisions for cost cutting and data management will continue to go forward. Bob Kocher, partner at Venrock, agreed, noting that Rep. Paul Ryan’s recently released budget plan included the cost control mechanisms from PPACA.

Nandini Tandon, board member of C21 BioVenture, also predicted that the law will stand but either way the decision would not have a major impact on investment in healthcare. Paul Kusserow, senior vice president and chief strategy and corporate development officer at Humana, Inc., added that with change and tumult come investment opportunities. 

The panel explored a number of promising investment areas, including alternative and holistic medicine, virtual medicine, and capitalizing on the growing understanding of genetics. Panelists agreed that one of the hottest investment areas right now is healthcare information technology. “Data is going to be king,” said Kusserow. Kocher said that he sees investment potential in ideas that make healthcare more affordable for the consumer. He is particularly excited about devices that replace drugs, for example vagal nerve stimulation as a treatment strategy to control certain neurological disorders. Tandon sees a new investment focus on blending the U.S. penchant for innovation with emerging markets like India, which has younger-trending age demographics, a large talent pool, and an interest in considering the cost-effectiveness of new products early in the development process. 

Led by moderator Aaron Task, host of Daily Ticker on Yahoo Finance, the panel wrapped up with a view of personalized medicine research from the venture capitalist (VC) perspective. Panelists agreed that with the VC tendency to look at an investment with an eye toward an exit (e.g., an initial public offering or a merger/acquisition opportunity) within three to five years, most VCs would decline opportunities to invest in personalized medicine projects. “Personalized medicine is showing that things are more complex than we thought,” said Kocher, and the science is not sufficiently developed to attract VC interest. However, panelists noted that VCs can be appealing partners for large pharmaceutical companies looking to introduce a more entrepreneurial character into their activities. Tandon noted that VCs can provide increased “deal flow,” and assume some of the risk for the partnership, and Kusserow added that VCs are more knowledgeable about how to “migrate companies.”

In response to questions from the audience, the panelists provided additional insight into the idea development process from the investor perspective. Kocher said that VC organizations are looking for big ideas. “They will pass on the $30 million ideas in favor of the $300 million ideas – the test is could it be big enough to change the world, create markets,” he said. The panel agreed that early stage companies are most likely to find traction with angel investors like friends and family members. As Kusserow put it, “the process is like an evolution – you have to embrace the journey.”

Harnessing the Market Opportunity of an Aging Population

As baby boomers age, advances in medicine and technology have dramatically increased average life expectancy, resulting in 98 million Americans who are more than 50 years old. While many people tend to focus on the problems that this demographic transition will cause, a Milken Institute Global Conference panel focused on the opportunities. Because of the huge purchasing power of this age group, why aren’t new products, services, and markets emerging more rapidly to address the needs and aspirations of this population?

Ken Dychtwald, president and CEO of Age Wave, started the conversation by pointing out that this demographic shift is unique in human history: “People in the past did not live long enough to worry about the effects of an aging body.” Osteoporosis, arthritis, lost vision, and other maladies that come with age offer a “wonderland of opportunity” for innovators and entrepreneurs who are willing to pour time and capital into helping elevate these problems.

Other sectors that present huge opportunities for growth by targeting this age group are:

• Financial services
• Entertainment (the top six highest-grossing musical tours last year were bands that were popular in the 60s and 70s)
• Premium services (first-class flights, high-end cars, vacation packages, etc.)

Jody Holtzman, senior vice president of thought leadership at AARP, pointed out that advertisers have been particularly slow to pick up on this opportunity. The overall purchasing power for the 50+ age group is much higher than that for Generation Y, but only 10 percent of advertising resources are focused on this age group while a disproportionate amount of resources go to advertising to younger adults. Holtzman pointed out that ultimately “Demographics are destiny,” and advertisers and wealth managers that have not adopted an aging strategy are missing out on a massive and seemingly obvious opportunity. Moderator Paul Irving, senior managing director and chief operating officer of the Milken Institute, asked the panelists why advertisers still focus on the younger demographic. Dychtwald pointed out that this is based on a myth that people make brand decisions early on in life and stick with them permanently.

Doug Busch, senior vice president and chief operating officer of Intel-GE Care Innovations, pointed out that there is a huge “competitive advantage of simplicity.” He discussed the example of the iPad, which is simple to use whether you are 5 years old or 75 years old. Many products cut themselves off from the older demographic by making products that are too complicated. He said that most members of this age group don’t want to “muddle through or fuss with low-quality or unwieldy products and services.”  

All panelists agreed that the 50+ age group is an incredibly underutilized resource. The combination they present of purchasing power and leisure time offer huge opportunities for those businesses and entrepreneurs who thoughtfully target them with products and services that are designed specifically to their unique needs and are marketed in a way that keeps these needs in mind.