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

New Opportunities for Patients to Engage

Patients are the most underutilized resource in medical research – and they have the most to gain. Two things this past month struck us as truly promising in the effort to build a research culture that encourages and inspires broader public participation.

1) The National Institutes of Health (NIH) invited patient advocacy and disease research organizations to partake in the strategic planning process for the NIH Common Fund. The NIH Common Fund was enacted into law by Congress through the 2006 NIH Reform Act to support cross-cutting, trans-NIH programs that require participation by at least two NIH institutes or centers and that can be achieved over a 5-10 year period. This program supported NIH initiatives in nanomedicine, molecular libraries and imaging, the Clinical and Translational Science Awards, and the newly formed Center for Regenerative Medicine.

With this request for input, NIH is seeking innovative and transformative ideas from the outside community to drive the formation of new programs to be funded in 2013. This provides an opportunity for patients to help NIH clinical researchers prioritize novel research ideas and collaborations likely to drive new solutions to medical challenges. NIH lists a host of concepts on which they are seeking input, from artificial organs to Big Data initiatives to mobile health technologies. With this announcement, NIH is providing the public with an opportunity to peruse its strategic planning site and share its thoughts on the Nation’s biomedical research plans for the future. It is NIH’s hope that public input will help drive innovative new ideas through the Common Fund.

2) The Department of Health and Human Services (HHS) recently slated an update of the requirements for protecting human subjects in research – referred to as the Common Rule. This update, the first major overhaul in the 20 years since the regulations were enacted, is designed to enhance protection for research subjects while and reducing administrative burdens, delays, and ambiguity for researchers. The Common Rule covers a number of areas specific to Institutional Review Boards (IRBs), which review the ethics of research, and the need to obtain informed consent from research subjects.

HHS is requesting input on a number of key provisions. The department is proposing to simplify the informed consent process and allow consent on biospecimen research to extend broadly to future new studies. Stored biospecimens are an important source of information and material for researchers, and the reuse of existing data and materials can be an efficient resource for a new research study. If appropriately modified, this rule would respect patients’ contribution to science, as well as their rights and welfare, while removing unnecessary timely and costly administrative burdens to researchers.

In addition, in terms of oversight, HHS is proposing to restructure the missions of IRBs by prioritizing their workload based on the risks posed to subjects. The department also is evaluating the use of a single IRB of record to oversee studies conducted at multiple sites. Such modifications could greatly improve efficiency in coordinating research studies.

In this new era of transparency, patients and the broader public have an opportunity to redefine their role in research by sharing their ideas for innovative models of research and sharing perspectives on how to improve the informed consent process. These two opportunities are opening the door to more meaningful public input in the medical research enterprise.

At FasterCures, we are strong proponents of working to improve our medical research system to better work for patients. Similarly, we urge the public, and especially patients, to become engaged, proactive participants in the process.

A few months ago, we released the report Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change, which highlights how people affected by HIV/AIDS rallied together and created an advocacy movement that demanded change and got results. We have much to learn from HIV/AIDS activists, how they redefined patient engagement in the medical research process, and changed the drug development paradigm. Thanks in large part to the groundwork they laid, the public is increasingly viewed as an essential ally and partner in advancing medical research.

RELATED EVENT:

REGISTER NOW for a free FasterCures webinar Bits and Bytes: Making Patient Information Available and Meaningful to Researchers
September 16, 2011
1:00 - 2:30 p.m. (Eastern)

Three distinguished leaders will discuss opportunities for accelerating research through improvements in biobanking, the use of electronic medical records, and the release of personal health information for research. Don't miss this important discussion!

Speakers:

• David Blumenthal, M.D., Director, Institute for Health Policy, Massachusetts General Hospital and Partners Health System; former National Coordinator for Health Information Technology


• Carolyn Compton, M.D., Ph.D., Director, Office of Biorepositories and Biospecimen Research at the National Cancer Institute


• Sharon F. Terry, President and CEO, Genetic Alliance


• Moderator: Margaret Anderson, Executive Director, FasterCures

The Science of Innovation

by Gillian Parrish, Manager, Alliance Development and Communications
Now that the holiday dust (and snow!) has settled, the medical research community is buckling down for a busy and productive year. This past week brought forth a flurry of ideas and activity on everything from:

• Evaluating the research implications of HHS’s new rule on what constitutes “meaningful use” of health information technology; to
• Moving the next phase of FDA’s Sentinel Initiative to track product safety forward; to
• Driving more and better collaboration among patient registries and biobanks.

FasterCures participated in a number of these, including a meeting of the President’s Council of Advisors on Science and Technology (PCAST), the 2nd Annual Sentinel Initiative Public Workshop and The NIH’s Office of Rare Disease Research’s (ORDR) Advancing Rare Disease Research Conference. A common thread we heard across all of the discussions was the need for better communication and collaboration, especially around procedural practices and data.
Atul Gawande refers to this systematic exchange of best practices as “process science.” He describes it as applying the same scientific rigor currently placed on the discovery of new medical solutions to delivery of those solutions. But between discovery and delivery, we believe there is another step that is just as critical to this equation – translation.
Translation, the bridge between basic and clinical research, is a seminal process that drives the engine of delivery, but one in which we often lose the most time and resources. Essentially considered phase two of discovery, it is the application of ideas and insights generated through that rigorous science Dr. Gawande talks about to the treatment and prevention of human disease.
Regardless of how exact and scientific the initial discovery process may be, however, if valuable data sets and research models uncovered though that process aren’t shared and systematized, their translation into solutions that providers can deliver will take years longer than necessary.
The NIH is trying to incentivize more translational research through its Roadmap initiative and specific programs such as the Clinical and Translational Science Awards and the new Therapeutics for Rare and Neglected Diseases (TRND) program. But there remains an enormous amount to be done.
This week, we heard some great ideas for getting those research models and data sets flowing in ways that could considerably reduce the time and improve the standard operating procedures between discovery and delivery. They are:

• Supporting the exchange of de-identified patient data through electronic health records (EHRs) for research purposes, and ensuring that the final meaningful use rule includes language to that effect
• Reevaluating the informed consent process to more clearly distinguish between the use of electronic information for public health surveillance versus scientific research
• Building an empirically evaluated methods library for innovative clinical trial models
• Developing standards and guidelines for biospecimen collection and management so those processes can be reproduced
• Identifying and training biospecimen champions at hospitals and health systems where procedures take place -- possibly even establishing a national “concierge resource” for biospecimen collection
• Connecting biorepositories with robust clinical data sets, such as those available through the National Cancer Institute's cancer Human Biobank (caHUB)
• Expanding and improving ORDR’s “registry of registries,” building it out with individual researchers across all rare diseases and making it fully query-able

We’ll closely track the progress of these efforts in the coming months and find ways to work with the drivers to see where and how FasterCures can help. The more information we can get flowing between researchers now, the faster we’ll be able to translate those discoveries into solutions that Dr. Gawande and his peers can deliver.

Hope, Healing, and Heroism Through Science

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

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

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

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

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

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

Let's Make Cancer Research Happen

By Margaret Anderson, COO, FasterCures
I was going through some clips this week and the Houston Chronicle Op-Ed caught my eye Now's time to reinvigorate country's War on Cancer. U.S. Senators Kennedy and Hutchison outlines in the piece what their bipartisan legislation proposes to do to get things moving. Of the many important proposals detailed in the legislation, the third solution the senators present focuses on a more coordinated approach to cancer research. There was also mention of how "Integrated research will help accelerate the progress of lifesaving research. The search for cures should be a collaborative goal." Indeed!

I was particularly pleased when the senators highlighted the need to establishing an interconnected network of biorepositories with broadly accessible sources of tissue collection and storage. Biospecimens are often cited by the research community as the most critical of all issues. Unfortunately, experts contend that "of the 350 million specimens collected in the United States, only 30 percent of them - some say the number is even lower - are of a quality standard high enough for research purposes." That's why we developed and facilitate BioBank Central. We want to link researchers with resources, encourage the donation of specimens, and educate the public about scientific advancements. I am hopeful that this bipartisan legislation will be instrumental in breaking down this obstacle to research progress.

The senators went on to state in this piece that "our national policy should encourage all stakeholders in the War on Cancer to become allies and work in concert toward cures." It's certainly not that scientists and doctors don't want to be allies, if they could have done anything to save the lives of the half a million Americans who died of cancer in 2008, they surely would have. But there is work to be done to create the necessary networks, and then to maximize their potential.

My father, Don Anderson, was one of the thousands who died prematurely of cancer in 2005, and the research team that led his leukemia clinical trial at Johns Hopkins University did everything they could to find treatments to extend and maybe even save his life. On the day that someone you love dies of cancer, you cannot help but think about all of the other families that are going through that experience at the same time. It is not a comforting thought. The barriers that stood in the way of progress in the research he participated in went well beyond the scientific questions the team faced about how to shut down the mechanism of his cancer, and surely included the barriers this legislation aims to address.

Let's be sure to have a robust discussion about these barriers and potential solutions in the context of the Kennedy-Hutchison cancer bill. There will be an anticipated 1.4 million Americans diagnosed with cancer this year that will get added to the group of people whose lives have been changed forever by cancer. They and their families and friends are all anxious for rapid progress in cancer research and care. Let's make it happen.

You Can Take That to the Bank!

By Angelo Bouselli, Communications Manager, FasterCures

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

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

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

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

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

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

For more information on biobanks visit BioBank Central.

Biobanks Going Mainstream

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

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

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

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

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

Big Changes at Biobank Central

by Kate Blenner, Program Analyst, FasterCures

We at FasterCures are very pleased to announce some big changes to BiobankCentral.org, the Web site we have established to highlight the importance of biobanks to medical research. This site links researchers to resources, encourages the donation of specimens, and educates the public about the benefits of research on banked biospecimens. After interviewing key stakeholders, including patient advocates, biobank operators, and leaders in the field of biospecimen research, we will begin staging some new features and functions that will make Biobankcentral.org even more useful to visitors hoping to learn more about these critical resources.

The first of these new features is the Spotlight on Innovation which will highlight individuals and organizations doing exceptionally innovative work in biobanking. Our first Spotlight focuses on the Susan G. Komen for the Cure® Tissue Bank at the Indiana University Simon Cancer Center, or Komen Tissue Bank (KTB) for short. This bank’s mission is to collect samples of normal, healthy breast tissue and other biospecimens from healthy women for breast cancer research. Yes, you read that correctly—normal tissue. Healthy women.

In its 1998 priorities for cancer research, the National Cancer Institute identified the lack of knowledge about the normal biology and development of the mammary gland as a significant barrier to finding a breast cancer cure. Most research to date has focused on characterizing diseased tissue, but without the frame of reference of how healthy tissue develops and functions opportunities for a cure could be missed. Complicating the issue was a shortage of normal tissue available for study. The NCI’s recommendations to address the ‘tissue issue’ languished for a few years, until some motivated advocates and clinicians at IU Simon Cancer Center decided to form the KTB.

Despite initial skepticism that healthy women would want to go through an invasive collection procedure, KTB put its faith in the motivation of the breast cancer advocacy community—and it paid off. They have collected thousands of samples to date, and communities across the country have asked KTB to set up its collection tent at their local Race for the Cure events. As bank co-founder and patient advocate Connie Rufenbarger told me: “These women have walked, they’ve written checks, they’ve lit candles—they’ve done everything they can to demonstrate they want to help. [The response] really speaks to the fact that there isn’t a whole lot you could ask that women wouldn’t give you to cure this disease.”

I hope you enjoy this first Spotlight of the Komen Tissue Bank as much as I enjoyed speaking with its remarkable founders and staff. If you have a moment, stop by the KTB Web site to find out how you can get involved in their work to find a cure. And, of course, keep an eye on BiobankCentral.org—we have many more exciting new changes to come.

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' 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.

Exploring the Progress and Potential of Cancer Care

I had the privilege of hearing some terrific speakers at a recent forum in Washington, DC called “Outlook on Oncology: Exploring the Progress and Potential of Cancer Care” sponsored by Pfizer Oncology. After an overview of the Pfizer oncology portfolio, Dr. Kavita Patel, Deputy Staff Director of the Senate Health, Education, Labor & Pensions committee, spoke about her work on Senator Kennedy’s much anticipated cancer legislation. The legislation will look at the highest priorities for federal action and will take a much needed comprehensive approach so as not to pit one cancer against another. She highlighted many of the unfortunate realities in cancer research that continue to need attention: a biospecimen network that is not entirely networked and not universally accessible to researchers, a clinical trials system that mystifies even the most savvy patients and practitioners, and a need to do more…much more.

She cited the need to look closely at public/private partnerships and to fortify what the public and private sectors can do together to advance cancer research and care. When I asked her if the legislation was going to incent opportunities to fund and target innovative research at the National Cancer Institute she replied that they’d looked closely at existing models that allow this flexibility. Will this cost money? You bet. But Dr. Patel pointed out that if we don’t put money into this now, we’ll lose tremendous opportunities for innovation, not to mention the prospect of losing talent to other nations that are not intimidated by risk and are recruiting scientific talent faster than the American Idol winners are getting scooped up. Finally, she said they want to do health care policy forecasting so they can pre-anticipate what the implications of the legislation will be on all the systems in six months, one year, five years, etc. How refreshing to think about some advance planning in biomedical research! It is inspiring to hear her talk about Senator Kennedy’s renewed commitment to this legislation, and comforting to know that the Lion of the Senate is roaring.

Linda Ellerbee, a noted journalist and breast cancer survivor gave a captivating luncheon speech about the power of each patient and described her personal journey after her diagnosis 16 years ago. It was remarkable to think back to a time when speaking of breast cancer or any cancer for that matter was a taboo subject. I have worked in the HIV/AIDS movement and have seen the same transformation in that arena, where HIV invoked fear and silence. Ms. Ellerbee talked about how when the public demands something, things can change. I reflected on how AIDS and cancer activists have changed the landscape of biomedical policy. We still need much more change, so her words were an important reminder that there is always more to do. At FasterCures, we are embarking on several exciting new initiatives in our new strategic plan. Stay tuned to hear about ways we want to impact many of these issues and foster change, in areas such as biobanking, the conduct of clinical trials, and in fostering innovation.

- Margaret Anderson, COO, FasterCures

Improving your Risk-Reward Ratio Could Depend on Who Your Partners Are

Breaking away from the traditional model of financing medical research, biotechnology companies have started turning to nonprofit disease research organizations not only for their leadership, patient network and advocacy clout, but also for their financial capital. A panel of nonprofit leaders and biotechnology executives at this year’s BIO convention, organized by FasterCures and moderated by Margaret Anderson, FasterCures Chief Operating Officer, unanimously concurred that new paradigms are needed and that therapeutic outcomes are not likely to come out of academic research centers.

At FasterCures, we believe that one of the most significant impediments to accelerating progress toward cures is the difficulty of transforming research with therapeutic promise into marketable, profitable products that help patients. To address this major roadblock, there are growing numbers of biotech companies beginning to forge new and innovative alliances with nonprofit research foundations to reach new product development milestones. And many of the nonprofit research foundations understand that the only way their work will ultimately help the patients they serve is if they can "hand off the baton" at some point to an entity that makes products and can get them to patients.

Free of the imperatives of publication and career advancement in academia and the bottom-line imperatives of the private sector, disease research foundations are ideally positioned to make relatively high-risk investments that could significantly move a field of disease research forward and increase the likelihood that other parties will also invest.

Panel highlights:

• Katie Hood, CEO at the Michael J. Fox Foundation (MJFF), talked about how they are proactive about project management with their grantees and believe in collaboration. Their work with Elan shows a recognition of the leadership that MJFF has provided in the Parkinson’s research arena. (Read Katie Hood's blog on the Huffington Post: Breaking out of the 'academic comfort zone' for higher returns on medical research philanthropy)

• Ted Yednock, Executive Vice President and Head of Global Research at Elan Corporation, said its relationship with the Michael J. Fox Foundation has "increased the company’s exposure to cutting-edge science."
• Russell "Rusty" Bromley, Chief Operating Officer of the Myelin Repair Foundation said that this new model for medical research allows the foundation to "recognize, overcome hurdles, and remove friction" in the processes of drug discovery.
• Vertex Pharmaceuticals President and CEO Joshua Boger points to its partnership with the Cystic Fibrosis Foundation as a model for closing the gap in treatment for diseases with small or no markets. Additionally, Boger said this partnership energized employees, stimulating renewed commitment to finding a cure for cystic fibrosis.
• Another example of a successful partnership is between the Multiple Myeloma Research Foundation (MMRF) and Proteolix, Inc. Louise Perkins, Director of Research at the MMRF, talked about the many initiatives MMRF is engaged in to aggressively find a cure for multiple myeloma.
• Lori Kunkle, Chief Medical Officer at Proteolix, discussed how this partnership yielded greater enrollment in clinical trials, allowing them to accelerate their schedule, broadened exposure to multiple uses of a particular drug and allowed Proteolix to make early decisions about whether to move a compound forward in he development process.

For-profit biotechnology companies look to nonprofit disease research organizations for strategic and intellectual guidance as well, allowing access to a broad and deep arsenal of disease-specific research, patient networks, and advocacy tools.

Panelists acknowledge that the bottom-line drivers differ for each partner. On the for-profit side, profit drives the quest for innovation, while for nonprofit foundations the ultimate goal is finding a cure for the disease. The need to accurately understand the drivers and pay-offs for each partner is vital in ensuring a successful relationship; a model that’s beginning to change the paradigm of our medical research enterprise.

Many analysts believe that a "paradigm shift" is occurring at many pharmaceutical companies, which are realizing that relying on the blockbuster drug model for profits is no longer a viable business model and that – particularly as personalized medicine becomes a reality – there is a role for therapies for niche markets in their pipelines. These new business models require new partners, and hopefully the BIO meeting continued to nurture some of those existing relationships and the ones that are beginning to grow.

More discussion of these issues can also be found in our newly released report, Entrepreneurs for Cures, available to download now.

--Cecilia O. Arradaza, Communications Director, FasterCures

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The Future of Biobanking Relies on the Quality and Viability of Biospecimens

The theme of collaboration carried over into day three and four presentations at the ISBER Annual Meeting, from the role of industry in biobanking and the importance of collaboration between academia and the corporate sector to the importance of global biobank networking. Throughout the conference, in asides by presenters and conversations among attendees, another theme began to emerge: the lack of information about quality and viability of biospecimens.

• A roundtable led by Dr. Mark Cosentino of NCI Frederick was tasked with addressing the topic "Repository Science: Establishing a New Scientific Discipline." A packed table engaged in a discussion that covered degree types, funding, and oversight of educational programs to train repository scientists, but talk quickly turned to curriculum: how can biobanking scientists be trained without knowledge of appropriate biospecimen processing, handling, and storage for a variety of analyses? Participants were unanimous that much more information on biospecimen science is absolutely essential to train effective biobanking scientists.
• At poster sessions, there was a similar sentiment – not only is more information needed about biospecimen science, it is needed through a consolidated, open access resource. After walking me through her poster on varying RNA levels in breast tumor tissues collected by different protocols, Rebecca Barnes of the British Columbia Cancer Agency’s Tumor Tissue Repository noted that studies like hers were being performed by other researchers around the world, but it is difficult to know who is studying what and what the results mean without a clearinghouse or forum for information sharing.
• Carolyn Compton of OBBR at NCI and David Horsfall of the Australian Prostate Cancer Bioresource presided over a panel of speakers whose talks addressed the impacts of packaging, fixation, specimen type, and length of storage on biomarkers. One of the key panel presentations was given by Barbara O’Brien of Westat; she presented a study that focuses on stability testing of biospecimens in conjunction with the National Children’s Study (NCS). NCS will examine the effects of environmental influences on the health and development of 100,000 children in a 21-year longitudinal cohort, beginning pre-conception with sample collection and analysis from parents. The collection schedule and length of study mean that NCS will collect literally millions of samples in the first years of the study. To ensure the validity of data generated from these biospecimens, Westat will undertake long-term stability testing of samples collected from parents of study subjects. Sample specimens to be assessed include urine, saliva, vaginal secretions, hair, blood and blood derivatives, toenails, and breast milk, and Westat will evaluate the stability of dozens of analytes in each of these matrices over the course of the 21-year study. The results of this assessment will add a considerable body of evidence to the field of biobanking and biospecimen research.

The success of biobanking as a tool in fighting disease requires more insight into the properties of different types of biospecimens and how these should be collected, processed, and stored to preserve key biomarkers. Piece-meal studies alone would not address this need; instead, a coordinated effort, with data sharing and public access to findings, is necessary to ensure that this knowledge is appropriately applied. We look forward to ISBER’s leadership in paving the way to bridging this critical gap in scientific knowledge.

-- Kate Blenner, FasterCures, Program Analyst

Global Biobanking Collaborations: Challenges and Opportunities

Blogging from the ISBER 2008 Annual Meeting

The ISBER annual meeting theme of collaboration rang true at the opening plenary session and echoed throughout Monday’s program. Cooperation and harmonization among biorepository resources could bolster the scientific toolkit for understanding disease cause and treatment response.

Dr. Francis Collins, Director of the National Human Genome Research Institute (NHGRI), an esteemed scientist and luminary, provided the keynote address, outlining several pioneering projects that, with the use of biospecimens and collaboration, would inform our understanding of disease to unlock clues for developing cures. These projects include an international cancer genomics consortium that would collectively sequence 50 different tumor types, yielding molecular data to drive development of more targeted diagnostics, therapies, and prevention mechanisms. It also includes the 1000 Genome Project, an effort between the Wellcome Trust Sanger Institute, Beijing Genomics Institute, and the NHGRI that will sequence genomes from 1000 people. With this detailed information, researchers can better understand at a molecular level why some people contract certain diseases and others don’t, and why some treatments cure disease and others won’t.


Monday’s program highlighted global collaborative efforts, including:

• iBOL (International Barcode of Life) project involving 25 countries that aims to develop DNA barcodes that will allow for automated species identification and thus more active management of biodiversity;
• Asian Network of Biological Resource Centers that serves as a collection of microbial cultures across China, Japan, Thailand, and Korea; and
• Biobanking and Biomolecular Resources Research Infrastructure (BBMRI) that intends to facilitate transnational collaboration, reduce fragmentation in the biobanking industry, and provide access of resources across academia and industry across the European continent.

Perhaps even more uplifting were discussions of not only collaboration, but also current and future activities to promote harmonization across biobanks. The lack of standards and practices across biorepositories is probably the most significant hindrance to realizing these resources' full contribution to the scientific process. An organization called P3G (Public Population Project in Genomics) is an international consortium whose members are public organizations undertaking large-scale genetic epidemiological studies and biobanks across North America, Europe, Asia, and Australia. P3G serves as a repository of information and tools and is developing tools such as their DataSHaPER (Data Schema and Harmonization Platform for Epidemiological Research). P3G notes that DataSHaPER is a comprehensive set of variables that should be collected by large epidemiological studies and biobanks for general-purpose biomedical research. The aim of the DataSHaPER is to provide a template to facilitate harmonization between biobanks and support the design of emerging ones. A future DataSHaPER will be developed for cancer as well.

The first two days of the meeting resonated with it's intended theme and demonstrated some impressive and ambitious collaborative efforts. The introduction of even early harmonization tools shows promise in unifying resources across institutions, countries, and continents. I look forward to tracking the progress of these efforts closely.

- Melissa Stevens, Director of Special Projects, FasterCures

Pre-ISBER Thoughts

In preparing for next week’s International Society for Biological and Environmental Repositories (ISBER) Annual Meeting, I recall some of the key takeaways from March’s Biospecimen Research Network Symposium "Advancing Cancer Research Through Biospecimen Science". The most startling came from Dr. Anna Barker’s opening remarks in which she noted that "of the 350 million specimens collected in the United States, only 30 percent of them were of a quality standard high enough for research purposes." And, we’ve actually heard other experts say that the number of viable samples could be even lower.

What if only 30 percent of the emails we sent everyday actually left our outboxes? What if our metro trains came only 30 percent of the time they were supposed to? That would make for a very unproductive world.

NCI’s Office of Biorepositories and Biospecimen Research (OBBR) brought together leaders in the field to discuss the challenges and recommend solutions. A number of the symposium speakers highlighted the variance in quality and study results across institutions and handling scenarios all underscoring the need for standard operating procedures. Carolyn Compton, Director, OBBR, outlined NCI’s role in elevating quality and Elizabeth Hammond, Professor of Pathology, University of Utah, presented a strong call to action for job aides, standard training, sharing of research, developing financial incentives, and implementing a biorepository “Goodhousekeeping Seal of Approval. ”

I hope the ISBER meeting could bring more focus to these calls to action in a global context. I am looking forward to discussing how international harmonization strategies can contribute in raising specimen quality. I am also very much looking forward to discussing communication strategies for engaging patients who are critical variables in the research equation. Most importantly I am looking forward to understanding how the global community can advance biorepository efforts to in turn accelerate medical solutions.

Check back next week as my colleague Kate Blenner and I bring you highlights from the ISBER meeting.

Melissa Stevens, FasterCures Director of Special Projects