Harnessing Big Data for Drug Development – A FasterCures Bloggersation

This post is the first in a series of FasterCures-hosted bloggersations featuring solutions-focused insights from experts and innovators. Posts will build on our Top 10 medical research trends to watch in 2012. 

This week’s question: What is the most important thing that could happen in 2012 to ensure better utilization of big data—housed in EMRs or other platforms—for drug development?

There are massive amounts of data from the mapping of the human genome, expansion of health IT, and the increased use of electronic health records. And yet, “big data” to advance development of new therapies for disease remains largely untapped. We know the potential behind these vast troves of information. Earlier this week, McKinsey Quarterly outlined how various sectors are grappling with the best ways to manage “big data” to guide their decisions.

The Office of the National Coordinator (ONC) at HHS has overseen progress in doctors’ and hospitals’ use of Health IT to enhance care; but how can those of us in the medical research advocacy community make sense of the sea of data and make it useful? With the Medicare and Medicaid incentive programs for the "meaningful use" of certified EHR technology—that is, their use by providers to achieve significant improvements in care—taking off, health information exchange standards continuing to be defined, and patient engagement in their health information starting to increase through initiatives such as Blue Button, the opportunities to leverage big data for medical solutions are plentiful. And with the Healthcare Information and Management Systems Society (HIMSS) conference underway this week, it’s as good a time as any to contemplate the possibilities.

But where do we start? And what matters most in the next year? We asked a few individuals on the frontlines of health technology and research innovation to weigh in:

Leslie Power , Practice Owner, Health IT, and Will FitzHugh, Chief Science Officer and Practice Owner, Research, 5AM Solutions Straightforward Broad Consent Mechanism: The issue of consent is not about big data or technology – it is trust. The most efficient, cost effective way to further a national dialogue on the benefits of research and participation would be to broaden consent beyond a specific study. Many people (perhaps most?) would participate in research - but only with clarity on the benefits and risks, and the control over who can use their data for what purpose. Read more . . .

Rich Elmore, Query Health Initiative Coordinator Standards for Distributed Population Health Queries: Pooled “Big data” in healthcare has its benefits but also several drawbacks. While there are terrific applications of pooled data, including registries and successful use of large research databases, there remain critical issues of policy and strategy. New standards are being defined that allow researchers to send questions to the data while leaving patient level information secure behind the data source’s firewall. Furthering these standards will make support for a learning health system possible. Read more . . .

Kris Joshi, Global Vice President – Healthcare, Oracle Prospective Informed Consent: As technical and security barriers to extracting data from EMRs decreases, and motivation to collaborate grows, there remains one significant hurdle - patient consent. In 2012, institutions that want to participate in clinical R&D collaborations would do well to begin establishing patient consent and privacy guidelines that support R&D processes. Read more . . .

Since our inception, FasterCures has spotlighted the need to leverage health IT for research purposes. We’ve comprehensively analyzed the issue, spotlighted solutions that are in play and yielding outcomes, and provided recommendations. To accelerate the process of turning ideas into therapies that will benefit patients, we the medical research advocacy community, must find tangible ways to make sure our health IT framework is built to improve care and advance cures. For more information, read our recent Still Thinking Research report.

Straightforward Broad Consent Mechanism
By Leslie Power and Will FitzHugh, 5AM Solutions

Electronic medical records (EMRs) can transform our healthcare system by making medical data accessible and available to everyone concerned with a patient’s health. Significant progress, particularly with regard to technology adoption, has been isolated. So, we wave a magic wand creating widespread EMR adoption - what value could we attain, who would benefit, and when?

Researchers who want to improve standards of care and identify predictive markers for treatment efficacy and adverse events would be one set of cross-industry stakeholders who could benefit immediately. Significant barriers to value would still remain. The lack of a consistently-applied, straightforward consent mechanism and the lack of support for molecular data storage and use of genomic data are just two.

The issue of consent is not about big data or technology - its trust. The solution is national dialogue on the social, economic and personal benefits of research and participation. Many people (perhaps most?) would participate in research - but only with clarity on the benefits and risks, and the control over who can use their data for what purpose. With expanded adoption of EMRs, this discussion becomes easier. If an EMR contains clinical and genomic data on a person, trips to a research center may never be required - people can consent to have their clinical and genomic data used for research purposes which would largely be computational efforts in data mining and biomarker discovery. Whether we choose existing or novel technologies to implement, the most efficient, cost effective way to further the dialogue would be to broaden consent beyond a specific study. This will address the current patchwork quilt of consent burdening biomedical research. Efforts like ‘Consent To Research’ (http://www.weconsent.us) target this issue.

The issue of broader consent becomes even more important when molecular data is considered. This data has value across areas of research, such as identifying risk factors for rare and common diseases, and finding predictive markers for efficacy and adverse reactions to pharmaceuticals. Requiring narrow consent for specific uses limits efficiency. In addition, current EMRs are not set up to store such data, and the fact that DNA sequence can be generated from different devices with different quality parameters renders platform comparisons suspect. Modules to securely store and process DNA genotype data need to integrate into EMR systems, and those models should be focused on creating a layer of interpretation above the raw genotype data.

Research focused on improving healthcare requires large sample sizes; the effects of genetic factors and other methods for personalizing medicine can be modest. Only by creating consented, large, nationwide sample sets can we reach the statistical power to identify these factors and create the predictive models to personalize healthcare and make more efficient use of our current crop of therapeutic, diagnostic and preventative tools. It will take a magic wand to make this happen in 2012, but making progress on these fronts will ignite faster cures and better health the fastest.

Standards for Distributed Population Health Queries
By Rich Elmore, Query Health Initiative Coordinator
“Big data” is typically managed in large pooled data sets, combining data from many settings of care. While there are terrific applications of pooled data, including registries and successful use of large research databases, there are critical issues of policy and strategy. Pooled “Big data” in healthcare has its benefits but also has several drawbacks.

From a policy perspective, pooled data approaches are problematic. Large pools of PHI are targets for attack from bad actors. Also, many PHI-holders have their own consent agreements with their patients. It is difficult to manage these different consent agreements when pooling PHI in one place. Additionally, HIPAA requires covered entities to control the flow of PHI, either directly or through agreements. When data is pooled, the party pooling the data must have a business associate agreement or data use agreement (in the case of research databases) with each covered entity that contributes data to the pool, with the same (or similar terms). This can be impracticable for the third party or undesirable for covered entities, as they often have to agree to non-negotiable terms in the agreement in order to pool their data.

From a strategic standpoint, pooled data is inflexible, stale and inaccurate. Pooled data approaches aren’t generally sustainable: the benefits of pooled approaches are too indirect to support the operational costs and complexity. Furthermore, health care organizations are unwilling to lose control of their information not just for policy reasons, but also due to competitive considerations.

But the absence of a standards-based alternative has given rise to pooled data approaches with all of these substantial drawbacks.

2012 is the defining moment for new standards that will enable big data analytics in a distributed environment. An ONC sponsored open government initiative, Query Health, is defining the standards and specifications for distributed population queries. Researchers will be able to leverage these standards to be “send questions to the data”. Questions can be sent to data sources including EHRs, HIEs, PHRs, payers’ clinical record or any other clinical record. Aggregate responses leave patient level information secure behind the data source’s firewall. Aggregate responses support questions related to disease outbreak, quality, CER, post-market surveillance, performance, utilization, public health, prevention, resource optimization and many others.

The path for these new standards will dramatically cut cycle time for deployment of new questions from years to days – making possible support for a learning health system.

The focus of 2012 should be laying the foundation for success: defining the standards and services for distributed population health queries. This is one extremely impactful way to leverage the potential of big data for research. For more information, visit QueryHealth.org.

Prospective Informed Consent
By Kris Joshi, Global Vice President – Healthcare, Oracle

Utilizing EMR data for secondary use, particularly to accelerate drug R&D, has been an Industry aspiration for a long time. However, lack of incentives for data sharing, technical hurdles, and security concerns all kept that vision out of reach for many years. In the last couple of years some leading institutions have finally made significant progress on multiple fronts to bring that long-held vision to life. The technology to extract data from EMRs has been around for a while, but it is now easier to deploy, cheaper, and more reliable thanks to investments in a new generation of analytic platforms. The motivation to share data has also grown significantly as health systems have become more confident in their ability to deal with security and privacy concerns, and see the potential benefits of better research collaboration. With the technical and security issues addressed, and motivation to collaborate growing, there remains one significant hurdle - patient consent.

Very few institutions today have a good system in place to gather prospective informed consent from patients for research use of clinical data. It takes a while to put a prospective consent mechanism in place with IRB oversight. Hence, in 2012, aspiring institutions that want to participate in clinical R&D collaborations would do well to begin establishing patient consent and privacy guidelines that support R&D processes. Even with de-identified data, full use for research purposes including the ability to re-contact a patient if needed requires proper consent. It would be a shame if after all the hard work to address the technical and business barriers, institutions discover in the end that the lack of patient consent prevents them from moving forward. Now is the time to get moving on it!

On Huffington Post: Top 10 Medical Research Trends to Watch in 2012

Margaret Anderson, Executive Director, FasterCures

1. NCATS: Who moved my cheese?This year, the National Institutes of Health will establish its new National Center for Advancing Translational Sciences (NCATS), created to speed the translation of basic discoveries into therapies that will improve public health. In light of our current fiscal reality (and limitations), we breathed a sigh of relief to see it up and prioritized in the appropriations process. Solutions can be tough to develop, and tougher to implement. How will NIH translate its translational focus to its cadre of basic research-focused scientists? Will NCATS strengthen and streamline existing efforts at the 26 other institutes and centers? Will it help to move discoveries out of the lab and to patients more quickly? NCATS is one way to get moving (and, as we know, "movement in a new direction helps you find new cheese"). The stakes are high with more than 100 million patients waiting for meaningful treatments options. But with Dr. Francis Collins as the visionary behind this effort, the same man who defied expectations by completing the Human Genome Project ahead of schedule and under budget, we like the odds.

2. FDA: The science of failure.Companies usually turn to the FDA to help them succeed (and with 35 approvals in FY2011, it helped many succeed), but perhaps it can also help them fail, faster. Commissioner Hamburg seems committed to having FDA play a role in improving R&D efficiency for all. FDA is sitting on mountains of data, and she has made it a priority -- through the agency's Strategic Plan for Advancing Regulatory Science and specific efforts like the PACES Initiative -- to figure out how to share more of it, particularly from compounds that were not approved, to help researchers and companies learn from failures. Will the industry see its self-interest in this and allow data to be shared -- and recognize that there is a business case for opening up the file cabinet?

3. Intellectual property: Keep it simple, stupid.Everyone in medical research seems to agree that intellectual property is a significant issue slowing progress, from disagreements over the value of discoveries to the time it takes for agreements to be negotiated, and the aversion to standardized contracting. We say, why does this need to be so complicated? Can't we come up with a simple set of guiding principles or values that we all agree to abide by -- something along the lines of Google's "Don't be evil"? Ideas, anyone?

4. Cures on the stump.Last time we checked, 2012 was a presidential election year. Will life sciences make an appearance on the agenda? The Obama Administration is working on a National Bioeconomy Blueprint to "harness biological research innovations to address national challenges in health, food, energy, and the environment" -- oh, and of course to "grow the jobs of the future." What commitments will the candidates make to support this critical economic driver -- whether NIH funding, tax policies for industry, workforce training, etc.? Can we make innovation in the life sciences a topic du jour in the election season?Venture capital investment in life sciences is on the wane, but venture philanthropy investment in drug development is on the rise. We're hearing discussion in several quarters about the idea of blending venture capital and venture philanthropy in some sort of hybrid investment fund. The wider world of social entrepreneurship has been experimenting with business models and forms of capital all along the spectrum from not-for-profit to for-profit for some time; the field of medical research is slow to catch up. We'll be watching for the launch of one or more of these efforts in 2012. Look for more discussion of financial innovations for translational research from FasterCures in 2012 as well.

5. 2012: Year of the Patient?TIME magazine notably called 2011 the "year of the protester." Could 2012 be the "year of the patient"? We offered up a roadmap to effective activism in 2011, built on the example of the HIV/AIDS advocacy movement. We identified its ingredients for success as attention, knowledge and solutions, community, accountability and leadership. Many of us point to the successes this movement created in HIV/AIDS drug development. Thirty years later, will more groups seize the mantle and make 2012 as much about changing the research and regulatory paradigm as about raising more money and awareness?

6. Reimbursement is the new approval.Even if we are successful at getting new treatments through the R&D and approval process, the question remains whether payers (government and private) will be willing to pick up the tab. Insurance coverage decisions -- and insurers themselves -- are playing a role farther upstream in the R&D and investment process, as companies begin to craft their research priorities and programs to maximize the possibility of reimbursement. Can payers position themselves as part of the solution to getting truly innovative therapies to patients? CMS is currently seeking comments on a revamp of its "coverage with evidence development" program. Could this become a model for other insurers to get patients access to new treatments while continuing to generate data about its effectiveness? Will smaller companies start reading the tea leaves and work with payers to avoid "failure to launch"?

7. HIT: If we build it, will research come?The federal government has succeeded in driving a doubling of providers' adoption of health IT over the last two years through its stimulus funding. But their interest in ensuring all this data is available for research to drive new, more effective treatments for patients is still not evident. "Meaningful use" of electronic health records (EHR) to qualify for federal funding seems to have been an effective carrot/stick combination for driving adoption of EHR systems for patient care. Let's use it in the service of research by requiring that using EHR systems for clinical trial screening and matching be included as a measure for meaningful use in the next round of definitions. Unlike the "Field of Dreams," just because we built the infrastructure, research use will not magically appear.

8. CTSAs: Who you gonna call?The first five years of NIH's Clinical and Translational Sciences Awards (CTSA) program are over, and by many accounts the focus at most of the 60 institutions has been on standing up the program on their own campuses, not on maximizing the power of the network. That may start to change in 2012 with the creation of a new Coordinating Center at Vanderbilt, which is already ramping up development of tools and resources, such as a shared IRBs, contract language, IP database and research data capture. Its director, Gordon Bernard, wants to make it a "virtual institute of drug development," a one-stop shop for companies, investors or nonprofit funders looking for academic partners. At Partnering for Cures 2011, he invited participants to contact him with their needs, saying that until the right partners are in place "we'll own that problem." So give him a call.

9. Putting old wine in new bottles.Enthusiasm continues to grow in academia and among patient groups for the idea of repurposing existing compounds. Companies, if they're interested at all, are looking for some sort of exclusivity mechanism to allow them to make money. NIH sees this as a central opportunity for NCATS (see #1), and NIH's TRND program is piloting a repurposing effort with the Leukemia & Lymphoma Society and the University of Kansas as partners, with the aim of recruiting an industry partner. But pilots won't be enough; there needs to be funding sources identified, IP policies sorted out and regulatory pathways clarified. Could 2012 be the year we create a business model to support repurposing? Tick tock goes the clock. Patients are waiting.

10. Convergence of venture capital and venture philanthropy.Venture capital investment in life sciences is on the wane, but venture philanthropy investment in drug development is on the rise. We're hearing discussion in several quarters about the idea of blending venture capital and venture philanthropy in some sort of hybrid investment fund. The wider world of social entrepreneurship has been experimenting with business models and forms of capital all along the spectrum from not-for-profit to for-profit for some time; the field of medical research is slow to catch up. We'll be watching for the launch of one or more of these efforts in 2012. Look for more discussion of financial innovations for translational research from FasterCures in 2012 as well.

Read FasterCuresTop 10 lists from previous years:

• Top 10 Medical Research Trends to Watch in 2011

• Top 10 Medical Research Trends To Watch In 2010

For more by Margaret Anderson, click here.

The Data Deluge

Adam M. Clark, Ph.D., Director of Scientific and Federal Affairs, FasterCures

Earlier this year FasterCures put together a Top Ten list of Medical Research Trends to keep an eye on in 2011. Coming in at number 5 on the countdown was Where is our GPS: Mapping the pre-competitive space. The trend outlined whether we could create a "marketplace" that will help facilitate the exchange of pre-competitive information and communal data sharing.

Recently, I saw first-hand a plan in action to work on this issue. I was among the participants at the second annual Sage Bionetworks Commons Congress. Sage Bionetworks is a new medical research organization with a focus on understanding biology as a network science and biological research as a community effort. Sage’s approach - shift to data intensive science built upon open access and broad collaborations to design network maps of disease built upon vast databases linking genotpyes, treatments, and medical outcomes.

Genomics is moving biology from an observational science to an informational science. Our way of thinking of disease is shifting from one gene, one disease to multiple genes with numerous phenotypes. In fact, diseases like cancer are now considered to be hundreds, if not thousands, of different diseases with a variety of underlying genotypes. This shift in understanding disease also means we need a shift in researching them as well. Finding cures for complex diseases in the 21st century will require us to fundamentally rethink how we conduct biological research.

Sage Congress participants noted that scientific disciplines such as physics and astronomy have benefited tremendously from data sharing and collaboration to build a fundamental new understanding or the universe. Similarly, the computer industry learned decades ago that open source access to software accelerated innovation, allowing tailoring of new applications to reach broader consumer markets. However, the field of biology has not yet undergone such a cultural shift. Despite technological revolutions in genomics and information technology, its approach to data sharing and knowledge generation still remains largely confined to closed networks and research silos with a focus on journal publications.

Sage is attempting to challenge this traditional paradigm by building a federated, open network commons for researchers to share large data sets, protocols, and research findings (positive or negative) to encourage collaboration and cross discipline interactions. This model supposes that the next biomedical revelations will be data driven and will require diverse talents and a community approach to research. Sage is currently underway building a data repository and the Congress featured a demo of Synapse, a portal that will ultimately include the repository, a cloud compute component, and tools for collaboration and workflows.

This is a decidedly different approach to biology, one that will require support from the research community as well as the patient organizations and the public. However, if this new world of data-driven medical science is to be a success, funding agencies and academia will also need to conform to the new reality. Incentives will need to shift to promote open access to data and collaborative science, both in terms of funding and career promotion.

It was noted at the conference that biomedical research is not so much limited by technology as it is by its culture. And cultural change can be extremely difficult. But if efforts like the Human Genome Project are any example, outcome-oriented research and collaborative scientific enterprises can transform our understanding of biology and with it, our ability to treat and to cure diseases. And at FasterCures, that is the bottom line.

Related Resource:FasterCures webinar on “Data Sharing Policies and Opportunities for Nonprofit Funders of Research?” View an archive online.

The Business of Personalized Medicine

Angelo Bouselli, Senior Communications Manager, FasterCures

With the sequencing of the human genome, and the scientific and technological advances of the 21st Century, many say we are at the advent of personalized medicine. But realizing the full potential of “personalized medicine” continues to be met with many questions largely left unanswered: Is it applicable across a wide range of diseases or just for a select few? Is it economically sustainable to truly personalize care?

“Everyone has a very different experience with healthcare,” said Greg Simon of Pfizer Inc., setting the stage for an engaging discussion on personalized medicine at the 2011 Milken Institute Global Conference.

Laura Esserman, Director, Carol Franc Buck Breast Care Center, Professor of Surgery and Radiology, University of California San Francisco said, “We need to learn how to treat disease and cancer better. We have learned that some cancers are biologically designed to kill and for those cancers, we need to treat them aggressively and immediately. Survival depends on giving the right drug at the right time, to the right person.

Paul Billings of Life Technologies echoed Esserman’s point and said that it’s about infrastructure and we need on that “will enable us to learn as we go.” “Personalized medicine can be a great thing if we put it in a framework we can learn from,” he said. We are improving care but we are still learning how to best apply the technologies to improve clinical decision making. “We need to educate patients that being involved in research makes a difference and provides better care.”

Panelists agreed that a system that combines data from the clinical care setting should be combined with the system that enables research to move forward.

“We need a connected research and care system that continually learns and improves on patient treatment and care,” said Esserman. “All of the information in the world won’t help you if you don’t know how to use it.”

Scott Jenkins of Dell agreed, noting that “electronic health records can assist in collecting and sharing valuable health information to improve treatment and care,” but that records alone aren’t the solution. He said patients play a major role and need to recognize the “value of sharing medical information and how this improves care.”

“We need to educate the general public to participate in medical research. It’s not just the patients that are struggling; the physicians are struggling to provide care. The entire system needs to be retooled.” Ricardo Guggenheim of McKesson Health Solutions said.

Personalized medicine, concurred the panel, starts with personal accountability for health decisions and greater patient participation in the research process.

Watch a video of the session.

Information Liberation: How Data Sharing Drives Medical Innovation

Adam Clark, Ph.D., Director of Scientific and Federal Affairs, FasterCures

Expert panelists shared their thoughts on best ways to encourage pre-competitive data sharing for research from the vantage point of public sector, private sector, and nonprofit organizations. The panel was among the more than 150 panels at the Milken Institute’s Global Conference. They outlined areas of opportunities for collaborations to accelerate research and development timelines for drug and diagnostic development that would result in significant cost reductions for drug development.

“We [drug companies] compete in the marketplace on products. We don’t compete on science. We don’t compete on clinical endpoints,” said Garry Neil, corporate vice president, Corporate Office of Science and Technology, Johnson & Johnson.

With the expansion of genomics, the science of biomedicine itself is transitioning toward biomedical computational informatics and a systems understanding of biology.

Anna Barker, consultant, Transformative Healthcare Initiatives and former deputy director of the National Cancer Institute, noted that “every patient is different. But every medical record doesn’t have to be,” highlighting the need for a standardized approach to collecting and sharing patient information electronically.

However, the current academic science career system and culture does not incentivize or reward scientists for making data available or for engaging in team science.

Eslie Dennis, executive director, Polycystic Kidney Disease Consortium, The Critical Path Institute provided examples of how nonprofits could drive data sharing and spotlighted lessons learned from the Alzheimer’s Disease Neuroimaging Initiative, the largest public-private partnership to date in Alzheimer's disease research. It is led by the National Institute on Aging at NIH, through a grant to the non-profit Northern California Institute for Research and Education, with private sector support provided through the Foundation for the National Institutes of Health..

Funding agencies like the National Institutes of Health and nonprofit disease research organizations carry the ability to fund non-traditional, collaborative projects in academia and drive data sharing and standards in large science initiatives. Similarly, the private sector has increasing incentives to encourage data sharing that would reduce the attrition rate of failed drugs in phase 2 and 3 clinical trials that could be accelerated with a more efficiently designed clinical trials infrastructure. By collaborating with regulatory agencies like the FDA, data sharing can drive better science for risk assessment and support contemporary regulatory science applications for emerging technologies for biomedical innovation.

Panel was moderated by John Dwyer, chairman, Telcare.

Watch a video of the session.

The Impact of an Aging Population

By Angelo Bouselli, Senior Manager of Communications, FasterCures

A couple hundred participants of the Milken Institute Global Conference packed into the Wilshire Ballroom for the session “The Impact of the Aging Population.” Paul Irving, senior managing director of the Milken Institute, moderated the panel and started with some facts for context: the 65 and older group is the fastest growing in the United States and faces numerous health challenges, including obesity, Alzheimer’s Disease, diabetes, and loneliness. Panelists agreed that this growing population offered economic and innovation opportunities, and that access and accountability were essential elements for this generation to thrive.

According to Mark Hodin of the Global Coalition on Aging, there are three components to understanding aging – longevity, low fertility, and the Baby Boomer bulge. “This subject is interesting because life longevity is new. This new middle age presents us with an opportunity to drive wealth creation and productivity around healthcare, research, learning, and creating new institutions that are right for the 20th Century.”

Marc Freedman of Civic Ventures said that social security was brilliant when it was introduced decades ago, and it worked for half a century. However, the traditional model of working for a long period of time and then retiring until the end of life doesn’t fit what many Baby Boomers today want. “Now what we need as a society and as individuals is the freedom to work. We really have to go back to the drawing board. We need a whole new set of social institutions.”

Paul Kusserow of Humana agreed that the infrastructure for the aging population needs to change. He warned about the current and growing problem of doctor shortages and said “The key is going to be access.” He saw the aging population as a “tremendous opportunity” for the emergence of electronic medical records and for innovation in care coordination. Sherry Lansing of The Sherry Lansing Foundation also saw aging as an opportunity for seniors to start something new and emphasized that seniors have an opportunity to “rewire, not retire.”

Redefining this stage of life and getting it right will change the whole picture of life, and all generations have a stake in getting this right. Irving closed the session by emphasizing that “we have a personal responsibility to get engaged in this issue.”

Watch a video of the session.

On Huffington Post: Top 10 Medical Research Trends to Watch in 2011

Technology, social networking, collaborative work, data sharing, crowd sourcing, interdisciplinary science, convergent disciplines are all increasingly second nature to the establishment of tomorrow.

1. NIH: Action on Translation.
Last year on the "Top 10" list, we highlighted NIH's new Therapeutics for Rare and Neglected Diseases (TRND) program as a potential model for the role NIH could play more broadly in helping promising basic discoveries bridge the "valley of death" between preclinical development and clinical research. In late 2010, the agency's Scientific Management and Review Board proposed the creation of a Center for Advancing Translational Sciences to do just that. The new Center, which NIH Director Francis Collins has committed to have up and running by the end of 2011, "would generate innovative tools, technologies, and ideas that would transform translational efforts throughout the scientific community." There's sure to be debate about what this new Center will and should do, and whether it will detract from NIH's basic research activities or deliver greater value to already important work.
2. Health IT: Will "meaningful use" be meaningful for research?
Since 2009, the federal government has put the pedal to the metal to accelerate widespread adoption of electronic health records by health providers and hospitals, and get them to "meaningfully use" these systems for data capture and sharing of the most elementary kind. What has not yet happened is a necessary focus on the secondary uses (an unfortunate term) of that data, including, very importantly, clinical research. 2011 will be the year to tell the feds what needs to happen to ensure that all that data being collected by docs is accessible to researchers trying to cure the diseases they're treating. Researchers and patients must drive that conversation. Have you asked your provider how their EHR system is faring? Stay tuned for FasterCures' paper on this topic in early 2011.
3. FDA: Stand and deliver.
2010 ended with industry commenting about the low number of products approved by FDA over the year, and FDA commenting about the low number of New Drug Applications submitted by industry. 2011 will almost certainly be consumed by the wrangling over PDUFA reauthorization. Getting more done with less will most certainly be a theme. We'll be focusing our attention on longer-term concerns, such as: Has the agency made progress on its regulatory science agenda (debuted last year)? What is the new Joint Leadership Council with NIH doing? Could this be the year the Reagan-Udall Foundation takes off? The heat is on.
4. Pharma: Can you learn something from the oil and gas industry?
Industries like microprocessors, film, and oil and gas have all faced market-driven inflection points and have reinvented themselves to respond. For example, the oil and gas industry has consortia that engage in pre-competitive research and exploration; companies take a more holistic view, and understand the need for sharing data to reduce risk and price volatility. Similarly, the pharmaceutical industry needs data-sharing and trusted third parties to help it tackle common challenges. What can be learned from these models, and who can create the framework for applying those lessons in this sphere?
5. Where's our GPS: Mapping the pre-competitive space.
Over the last year we've seen increasing interest within the biotech and pharmaceutical industries in collaborating more across companies and sectors on pre-competitive areas of research. But the concept remains ill-defined. Can you pre-compete and remain competitive? How should intellectual property be treated, and can it be handled in a standardized way? Will this be done in a one-off manner, or can it be managed/organized? Can we create a "marketplace" that will help facilitate the exchange of pre-competitive information? Keep an eye on new efforts likeSage Bionetworks' Archipelago initiative, which aims to create just such a marketplace.
6. Follow the leaders across the Valley of Death (but remember to pack some provisions).
We have been very interested to see over the last year a wide variety of new experiments aimed at addressing barriers to financing, skills, and incentives that will move promising ideas through the pipeline. At the end of 2010, we released a new white paper, Crossing Over the Valley of Death, which highlighted the importance of translational research and the challenges it presents. At Partnering for Cures 2010, we heard presentations from almost half a dozen different efforts with similar goals - from various initiatives to develop university intellectual property further before licensing it, to creation of portfolios of projects rather than companies for investment, to "social business enterprises" to fund development of promising discoveries. We'll be interested to see which models rise to the top but more importantly, who can demonstrate tangible results, and then how do we grow them?
7. Grab your partner: Is venture philanthropy the new venture capital?
More and more biotech and pharma companies are forming alliances with nonprofit disease foundations - not just as advocates for their products and sources of clinical trials participants, but, at a time of increasing costs and declining success rates for new therapies, as potential research funding partners. Is this a phenomenon of significant scale and impact? How do these deals work? What does each party bring to the table? What results have been achieved? On the subject of venture capital, we're watching folks like Third Rock Ventures who are still willing to invest in early-stage development, and Enlight Biosciences, which is investing pharma funds in enabling technologies.
8. Tear down this wall: The academia/industry business interface.
The business interface between universities and companies is an area in crying need of more standardization. One-off deals have high transactional costs, and yet the parties are reluctant to adopt the best practices of others. Successful models of university-industry collaboration in places like University of California San Francisco - where the focus is on high-value research moving from universities into development at companies, rather than on royalty revenue for either party - need to be spotlighted, studied, and replicated. And universities need to significantly rethink their internal reward structures to value industry collaboration and patents, not just government grants and publications. Who best to lead the charge here?
9. The future of research: Don't trust anyone under 40?
By now the statistics are familiar: The average age of an investigator receiving his or her first NIH grant is 42 years old. NIH is funding significantly more investigators over the age of 60 than under the age of 40. Plenty of people are worrying about how to attract and keep young people in the system as it currently exists. Not many are trying to see the world through their eyes and build a system that works for them. Technology, social networking, collaborative work, data sharing, crowdsourcing, interdisciplinary science, convergent disciplines are all increasingly second nature to the establishment of tomorrow. Maybe we need to learn a lesson from them today - and get out of their way, or risk losing an entire generation, and more.
10. See the world: Disease and the search for cures know no borders.
To help think about what research and business models of the future can and should look like, see what's happening in places that don't operate within the system that has grown up over 50 years in the U.S. Not only is intellectual capital locating elsewhere, but places like India, China, Singapore, and Africa are innovating out of necessity and producing some remarkable results with great speed and in some cases, few resources. And what about countries with wealth like Norway and Qatar - how can we engage them to invest significantly in medical research; can we initiate a Global Giving Pledge for sovereign wealth funds to dedicate resources to finding cures? The Rolling Stones told me that "Time waits for no one."

Are we optimizing our health IT system to improve patient outcomes?

Dozens of public and private stakeholders in medical research joined FasterCures last week at a forum about health information technology (health IT) policies – what these mean, what these represent, and how these will impact patient outcomes. The discussion focused on our national health IT infrastructure and how it is designed (or not) for clinical research. FasterCures Executive Director, Margaret Anderson, drove the discussion with Adam Clark, Ph.D., Director of Scientific and Federal Affairs at FasterCures. Adam sits on the policy committee of the Office of the National Coordinator for Health Information Technology (ONC) as a consumer advocate.

Key points raised during the forum include:

• The lack of communication between different sectors of the medical field despite the ever-growing state of health information technology.


• The current efforts by the ONC to establish standardized electronic health record (EHR) systems as a means of enhancing the quality of clinical care and


• Utilizing patient data to advance research and development.


• Addressing the challenges of patient privacy and security in implementing standardized EHR systems.


• The need for patients to have access to information in EHR systems, not only clinicians and providers.


• The importance of health information technology encompassing clinical research moving forward, creating effective avenues towards scientific innovation.

This effort is part of FasterCures’ Think Research program that supports the utilization of medical records and clinical datasets towards research on the progression of diseases and the development of treatment methods for them. An updated version of the FasterCures’ white paper entitled “Think Research: Using Electronic Medical Records to Bridge Patient Care and Research” will be released in early 2011.

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.

Building the Platform for the 'Health Internet'

Kristin Schneeman, Program Director, FasterCures

For two days this week, I joined a group of leading thinkers and entrepreneurs from a variety of sectors gathered to discuss an idea that originated with Harvard’s Isaac Kohane and Ken Mandl – the development of an “iPhone-like platform” for health information technology (HIT). Their basic premise, expressed in a paper published in The New England Journal of Medicine last March, is that “a vibrant and evolving health system requires a healthcare information technology infrastructure based less on monolithic, pre-defined products and more on a general-purpose platform that would support a collection of simple applications each doing a single task consistently and reliably.”

Internet pioneer Mitch Kapor started the meeting with this proposal: that the federal government expand its efforts to enable a Nationwide Health Information Network (NHIN) by engaging consumer health information platform providers and consumer health organizations as participants in the effort; instead of the status quo that focuses on standards for health information exchange among providers and government agencies. Kapor said the agenda for such an initiative would be to identify changes and extensions to NHIN specifications needed to support consumer requirements; to implement these changes in the government’s open-source CONNECT software gateway and elsewhere; with the goal of getting live patient data flowing by the second quarter of 2010.

Harvard Business School professor Clay Christensen, author of The Innovator’s Prescription on disruptive innovation in healthcare opened up day two with his analysis of what it will take to drive down costs in the healthcare system. In his view, the general hospital – which seeks to solve every problem for everybody – is no longer a sustainable business model. Care must become increasingly decentralized, and technology must be driven outward: to outpatient clinics, doctors’ offices, and even patients’ homes. In this environment, the locus of care coordination is a patient’s electronic health record. The patient/consumer must be at the center.

Lo and behold, by the end of day two, the federal government – in the form of President Obama’s Chief Technology Officer, Aneesh Chopra, and the Department of Health and Human Services’ CTO Todd Park – arrived and committed to doing exactly what Kapor had proposed. They even proposed renaming the NHIN the “Health Internet” (bless you, gentlemen). Many details remain to be worked out, of course, but the energy and enthusiasm of the dynamic duo of Chopra and Park was infectious, and many in the room committed their energy and resources to the project. The commitment of the new Administration to realizing the benefits of health IT is evident and welcome.

FasterCures’ interest in health IT is in research uses of electronic health information. We know the hosts of this meeting share the view that the value of HIT is as much in its potential to accelerate the development of new and better treatment options as it is in its ability to improve the quality of care and reduce costs. We are also interested in how patients can become drivers of disruption in the search for cures; those who suffer from disease are highly motivated to share their health information through vehicles like patient registries and social networking sites, and they are beginning to understand how they might leverage their data resources to influence the research agenda.

We hope the organizers of this new federal effort will make research uses of the network a priority, and that patient groups will be among the new stakeholders invited to the table. Then the Health Internet can revolutionize cures as well as care.

Clinical Research should be Part of EHR "Meaningful Use"

On June 26, 2009, FasterCures submitted comments on the proposed definition of "meaningful use" of electronic health records, urging the Office of the National Coordinator for Health Information Technology to ensure that meaningful use requirements for EHRs address support for clinical research at the earliest possible time. FasterCures' comments include:

• FasterCures has long advocated that one of the highest and best uses of a nationwide health information infrastructure – and one of its most underappreciated – is in accelerating the search for new and better treatments for disease. As the healthcare system addresses the challenges of widespread adoption of EHR systems, research capacity must be part of the architecture. Given the simultaneous priority the federal government has placed on comparative effectiveness research, we feel that this imperative cannot be overlooked or delayed.


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


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

For more information, download a copy of the FasterCures publication, "Think Research: Using Electronic Health Records to Bridge Patient Care and Research"

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

by Kristin Schneeman, Program Director, FasterCures

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

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

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

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

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

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

What Happened to My Medical File?

By Angelo Bouselli, Communications Manager, FasterCures
Images of my mother taking me to the doctor in a small town just outside of Scranton, Pennsylvania as a kid in the 1970s flicker in my mind like an old science fiction movie – the women behind the sliding glass window, the ugly chairs in the waiting room, the bright lights, and playing with Matchbox cars. We would wait to be called, walk through that heavy door with the little window, and down a long hallway lined with endless shelves of paper files. As a kid it was all very foreign to me but I knew those folders were somehow important.

I recently had a full physical with my doctor of 11 years. Until this visit, I hardly paid attention to what my doctor was writing in my file. As with every routine exam, he started asking questions. I knew the answers to some, but others he had to help me figure it out. He opened the folder and leafed through pages that should contain my life’s medical history – but he only had the chapters from the last 11 years. The rest of my story is probably stored in a box somewhere in northeast Pennsylvania.

What will it take for me to have a folder with my entire medical history? And, who should bear the responsibility of managing my record? Should I take it upon myself to keep this folder updated? Should I count on my doctors to keep each other appraised? Should I expect some government agency to bear this responsibility? And if I or some other entity were to go through the process of putting all this information in one place, then shouldn't this information be made available to clinical research as well?

In this day and age, we need a system that houses our complete medical record, the same way my bank managers my financial history. And, this system should be accessible by those we turn to deliver medical care as well as those we count on to develop medical cures. My current paper file is useless to me should I need medical care outside of my doctor’s office. Despite years and millions of dollars invested by government agencies and private IT companies to spur adoption, less than 20 percent of physicians currently use electronic health records. A recent survey from the American Academy of Family Physicians identified start-up cost to be a major barrier for providers to purchase an electronic system.

The Congressional Budget Office estimates that of the $787 billion stimulus package Congress approved in February, more than $20 billion will be spent on health-information technology between 2011 and 2015. A comprehensive health IT system is a giant step to making healthcare more efficient. I hope these stimulus dollars stimulate health providers nationwide to go paperless, save some trees, and most importantly deliver more effective and efficient care.While I’m still interested in finding out where the first 20 plus years of my medical history is, I’m more interested in knowing how the $20 billion will actually stimulate health IT adoption. Will my doctor in Washington get his share of the $20 billion pie? What about my doctor in Pennsylvania? And, what of those working in the labs to develop new medical treatments – do they get to benefit from the health IT movement as well?

For more information on EHRs download a copy of the FasterCures report "Think Research: Using Electronic Health Records to Bridge Patient Care and Research"

National eHealth Collaborative Launched

by Kate Blenner, Program Analyst, FasterCures

The national entity charged with prioritizing standards for health IT launched in January, and FasterCures is serving on a parallel effort to ensure that clinical research applications of health IT will remain a top priority to it and other health IT regulators.

On January 8, the successor to the American Health Information Community (AHIC) was relaunched as the National eHealth Collaborative (NeHC). AHIC originated as a 2005 federal advisory board to the Office of the National Coordinator; in its new iteration as the not-for-profit NeHC, it will operate as a public-private partnership to set priorities for IT standardization through its diverse stakeholder membership.

A key initiative of the new NeHC will be setting national priorities for HIT via analysis of ‘value cases’, distinct from ‘use cases’ in that they present cost-benefit analysis of adoption in the context of monetary, business, societal, and technological risk. NeHC is expected to open a call for value case submissions this month.

The concept of a value case should be familiar if you have been following the world of patient safety and care quality improvement, where “making the business case” for any improvements in healthcare delivery systems is nearly the norm. An ample body of literature and reports exists as templates for health IT value cases of established care processes and delivery channels. But how does one anticipate the value and risks of an integrated system that connects healthcare, clinical research, and translational research information-a system that is currently just a gleam in the eye of biomedical research advocates?

FasterCures is part of a working group hoping to do just that. The American National Standards Institute has formed the EHR Clinical Research Value Case Workgroup to develop value cases for interoperability between electronic health records and clinical research applications. We look forward to working with the Workgroup, NeHC, and other leading entities to ensure that an interoperable health IT system interfaces with the clinical and translational research world and is one less barrier to expedient delivery of new discoveries to patients.