Tools of Engagement: Building patient-centered research networks

By Kristin Schneeman, Program Director, FasterCures

Engaging patients in the research process is more complex and exciting now than ever before. Patient-driven organizations are moving beyond simply creating patient registries or serving as a conduit to participants for clinical trials and are marshaling their forces in new and more comprehensive ways. In a FasterCures Webinar on March 20, participants heard about two exciting new models of such “patient-centered research networks,” the T1D (Type 1 Diabetes) Exchange and Registries for All Diseases.

Dana Ball, CEO of the T1D Exchange, began by saying that after much due diligence about where the barriers and inefficiencies were in research and care for Type 1 patients, the Helmsley Charitable Trust set out to create an end-to-end solution to connect the R&D and healthcare enterprises with the patient resources necessary for all to succeed. What it has built in the T1D Exchange is a national network of 69 clinics treating 100,000 T1D patients, a 26,000-patient registry, and a 1,000-patient “living biobank” (i.e., samples are collected to answer specific questions). Integral to the network is Glu, a social network through which the Exchange is collecting valuable patient insight via surveys and discussions and which serves as a “real time access point for community-driven research using an innovative mobile platform.” With all these tools, the Exchange can share patient data, recruit patients, and collect samples quickly. Ball noted that absolutely critical to the success of the entire Exchange is a carefully constructed universal consent from patients.

The Exchange is meant to be self-sustaining. Ball noted that it provides products and services to academic and industry researchers and insisted that these are not “dirty words” for a nonprofit. “We think of ourselves as a high-quality CRO [contract research organization],” he said, with a unique trust relationship with the patient community. Another element of the sustainability model will be a new nonprofit company called Unitio, to be launched mid-year, which will license the platform the Exchange has created on reasonable terms to other disease groups. Ball noted that none of this would be possible without philanthropic support – no other single sector has the incentives to do this work. He was passionate about the need to educate philanthropists about how to invest for impact – the need for resources to hire good people, to create infrastructure that facilitates the whole enterprise but that others won’t create.

Sharon Terry followed with a presentation about Registries for All Diseases (Reg4All), which recently won the Sanofi Collaborate/Activate challenge – a cross-disease, crowdsourced registry that promises to break down the silos between diseases and gather information from patients in a standardized way. Terry began by noting that while recruiting appropriate patients for trials is viewed as “finding a needle in a haystack,” in fact “the haystack is made of needles” – we are all potential trial participants and just need the engagement, education, and trust to get on the information highway.

Reg4All is an evolution of work that Terry and Genetic Alliance have been involved with for a number of years that continues to widen in scope and sophistication. It is built on the platform of DiseaseInfoSearch, a database that provides disease-specific information and resources about 13,000 different diseases. Reg4All is not a disease-specific registry but rather a platform for individuals to enter self-reported medical information via a “gamefied survey” – and eventually to import clinical data from electronic health records – fully identified and protected. The survey gathers information on both common data elements across diseases and on disease-specific data elements. Patients use very sophisticated privacy software to authorize access at a very granular level about what information different types of users can see, e.g. researchers recruiting for clinical studies.

Participants had many and excellent questions for the panelists, beginning with, “We’ve gone from having none to too many cost-effective open source registry choices. How do we choose…?” Terry commented that there are valid reasons for multiple registries to flourish, and that our goal should not be one solution but rather lots of interoperable solutions. In her view, the granular privacy settings that Reg4All offers are very important in getting there. She mentioned that Genetic Alliance will be hosting a series of nuts and bolts Webinars to instruct groups on topics such as how to leverage or build on an existing registry/biobank, and will provide Reg4All modules others can pick up and use.

Ball closed with another plea for philanthropists and philanthropies to support this kind of project, calling on them to be like Ben Franklin, building libraries instead of buying books for everyone.

Resources

• View the Webinar slides
• Watch an archive of the Webinar
• Innovator: Sharon Terry's Global Database for Disease Research

http://train.fastercures.org/

TRAIN Workshop Gets ‘in the Weeds’ on Making Deals

Kristin Schneeman, Program Director, FasterCures

Earlier this month, FasterCures hosted a workshop on “The Nuts and Bolts of Cross-Sector Dealmaking,” bringing together a number of the venture philanthropies in TRAIN (The Research Acceleration and Innovation Network) to learn from and share with their peers the actual strategies and tactics nonprofits and biopharmaceutical companies are using to partner, as well as how foundations can better find and fund the most promising, commercializable research within universities. It was a valuable boots-on-the-ground exchange of specific resources and ideas for further exploration among a cross-section of all the stakeholders involved in medical research – foundations, industry, academia, government, and finance.

The day began with a discussion of a number of nonprofit collaboration models, intended to demonstrate the range of relationships and activities that currently exists among foundations and their academic and industry partners, from building and managing academic consortia for precompetitive and clinical research, to providing “concierge” services for academic researchers interested in commercialization, to investing in small companies through a variety of mechanisms and co-funding with large companies, to nonprofit and virtual biotechs.

Subsequent sessions focused on sharing strategies for finding partners (“the dating game”); the nitty-gritty of negotiating relationships, from licensing and royalties to milestones and data-sharing; and finally, what’s needed to sustain and replicate these relationships.

There were far too many valuable bits of information to relay in a brief blog post, and we will be working on a longer summary of the discussion, but I’ll highlight a few key takeaways:

• Lawyers and tech transfer officers shouldn’t run the show, but should absolutely be part of the team to craft terms that support the goals of a collaboration and help trouble-shoot any problems that might arise. It is critical to understand the legitimate needs of all partners.
• Communication is key, and there are so many ways we can communicate informally that we don’t – e.g., sharing non-confidential information with potential partners, holding non-binding legal roundtables to talk through issues such as the distinction between commercial and non-commercial research, etc.
• Industry-quality project management is fundamental to keeping programs on track and developing an asset that will be attractive to industry partners.
• Collaborations need to develop legs of their own and not depend on individuals within companies and foundations to sustain them. Building in surrogate markers of success that show progress over time (not just that “research is happening”) can help expand the circle of champions.

There were also many ideas for actions that would be helpful in smoothing the creation and sustenance of these collaborations, including:

• More transparent information about points of contact within companies, nonprofits, and academia;
• Education of tech transfer offices about what criteria potential partners are looking for (other than the Association of University Technology Managers data points they have historically been judged against);
• An effort to require more sharing of data about “failed” research efforts (and perhaps rebranding them as something other than “failures”); and
• Working with the IRS to better define “program-related investments” in the medical research space (outside of global health) to increase foundations’ comfort level with making such investments.

One of the most important outcomes of the day was a collection of resources that participants brought to share with one another and with the broader research community. These included template agreements of all sorts; due diligence guides; publications on collaborative models, venture philanthropy royalties, and charitable investing; and much more. This treasure trove of new tools will be available very soon on TRAIN Central Station.

Medical Philanthropy: Investing in the Cure Enterprise

by Gillian Parrish, Manager of Alliance Development and Communications, FasterCures

Though philanthropic investment in medical research accounts for only three percent of overall spending, a pittance compared to government and industry funding, it plays a vital role in catalyzing progress. Unburdened by the demands of shareholders, and the legacy mandates of institutional funders like the NIH, nonprofit disease foundations have the flexibility to fund high risk, high reward research that disproportionately accelerates the pace of innovation.

The “Investing in the Cure Enterprise” panel Monday afternoon at the Milken Institute Global Conference explored what’s working in medical philanthropy and how to encourage more philanthropists to invest effectively in the development of new and better treatments for disease.

Melissa Stevens of FasterCures moderated the discussion, noting the important role that medical philanthropy plays in stimulating research in under-resourced disease areas, and bridging the so-called “valley of death” in funding, between basic discovery and later-stage, clinical research.

Panelists from across the philanthropy spectrum agreed that a mix of accountability and measurement in research, together with investments in process infrastructure, would speed the discovery and development of new treatments.

Though perhaps not as “sexy” as disease-specific investments, funding for the development of medical philanthropy process infrastructure—including the sharing of data and best practices across sectors and diseases—is critical to replicating success and learning from failure, noted Matthew Bishop of The Economist. Melanie Schnoll Begun, Managing Director, Morgan Stanley Smith Barney Philanthropic Services, echoed the importance of understanding failures, saying that often in medical R&D, “failures” are actually successes, in that they eliminate ineffective research options and point the way to better cures.

Jane Wales of the Global Philanthropy Forum talked about the growing convergence between the social sector and private sector. “Philanthropists are bringing the rigor of the private sector to the work they do in medical research,” she said, “and the most important thing they can do moving forward is to pass their values along to the generations that follow to drive sustainability towards long-term goals.”

Susan Axelrod of 23andMe, Inc., said: “Philanthropists like us have a high tolerance for risk. We’d rather push the envelope in dramatic ways, even if we might fail, than play it safe.”

Jeff Kindler, Anthony Fauci, Kathy Giusti, Michael Milken among featured speakers at Partnering for Cures

Philanthropists, Medical Research Foundations, and Industry Leaders Gather in New York for Inaugural Partnering for Cures Meeting
First-of-its-kind meeting designed to forge collaborations needed to accelerate the pace of therapeutic development

(November 5, 2009) WASHINGTON, DC – Hundreds of medical research leaders, investors, and decision-makers are coming together at the inaugural Partnering for Cures meeting to explore innovative approaches to pursuing high-risk, long-term investments that could lead to therapeutic breakthroughs.

This first-of-its-kind meeting, to be held December 1-3 at the Millennium Broadway Hotel in New York City, brings together innovators from philanthropy, medical research foundations, and the biotechnology and pharmaceutical industries with the goal of forging collaborations necessary to develop new medical solutions.

The meeting is designed to facilitate informed investments and cultivate relationships by featuring panels that spotlight solutions to decades-long challenges facing medical research, adapting the outcomes-oriented approach of investor conferences, and building on the networking opportunities at industry "partnering" meetings. Speakers include:

Decision-makers from the biotechnology and pharmaceutical industries:

• Jeffrey Kindler, Chief Executive Officer and Chairman of the Board, Pfizer
• The Hon. Billy Tauzin, President and Chief Executive Officer, Pharmaceutical Research and Manufacturers of America (PhRMA)
• The Hon. James Greenwood, President and CEO, Biotechnology Industry Organization
• Garry Neil, M.D., Corporate Vice President, Corporate Office of Science and Technology, Johnson & Johnson

Venture philanthropy leaders:

• Robert J. Beall, Ph.D., President and Chief Executive Officer, Cystic Fibrosis Foundation
• Debi Brooks, Co-Founder, Michael J. Fox Foundation
• Kathy Giusti, Founder and Chief Executive Officer, Multiple Myeloma Research Foundation
• Hala Moddelmog, President and Chief Executive Officer, Susan G. Komen for the Cure

Philanthropists who are transforming the medical research enterprise

• Michael Milken, Chairman, FasterCures/The Center for Accelerating Medical Solutions; Chairman, The Milken Institute
• Debra Black, Founder and Chair, Melanoma Research Alliance

And, some of the most forward-thinking leaders in medical research:

• Anthony S. Fauci, M.D., Director, National Institute of Allergy and Infectious Diseases
• Douglas A. Melton, Ph.D., Co-Director, Harvard Stem Cell Institute
• Seth Berkley, M.D., President and Chief Executive Officer, International AIDS Vaccine Initiative
• Anne Wojcicki, Co-Founder and President, 23andMe

Philanthropic investment in medical research, though it accounts for only three percent of overall research spending – relatively small compared to government and industry funding –plays an essential role in catalyzing high-risk research, far beyond its proportion of funding. But, it requires a significant investment in effort, resources, and time to find the right research partner. Partnering for Cures streamlines this process by connecting similar-minded organizations and sharing best practices, further amplifying the transformative impact of medical research philanthropy.

Partnering for Cures is convened by FasterCures, the Washington, D.C.-based center of the Milken Institute, that's committed to breaking down the barriers that exist across the research continuum – from basic research to drug development – to clear the path to faster medical progress.

NOTE TO MEDIA:
Complete information about Partnering for Cures, including a full program and an up-to-date list of all speakers, is available at www.partneringforcures.org. Credentialed press wishing to cover Partnering for Cures, please email Cecilia Arradaza, FasterCures Communications Director, at carradaza@fastercures.org.

Call for Presentations: Apply for One of 48 Slots Dedicated to Innovative Approaches to Medical Research

Apply for One of 48 Presentation Slots Dedicated to Innovative Approaches to Medical Research Submit your application today. Space is limited; decisions are made on a rolling basis.
By now we hope you're aware of Partnering for Cures, a first-of-its-kind conference being hosted by FasterCures December 1-3 in New York. Partnering for Cures will bring together a unique audience of philanthropists, medical research foundations, and biopharmaceutical companies interested in building cross-sector collaborations to accelerate the search for cures.

Partnering for Cures is inviting nonprofit foundations that fund medical research to apply to present their cutting-edge work and innovative strategies to potential investors and partners. This is one of the unique features of the event, which distinguishes it from other conferences on medical research or philanthropy, and is based on the proven model of industry partnering meetings. We are planning to feature 48 medical research foundations, each with the opportunity to provide a 25-minute overview of its strategy, operations, assets, capabilities, and funding needs to an audience of potential investors and partners.

There is an open application process for these slots, and we want to encourage your organization to apply for one. An external advisory committee will help review applications and provide input to the selection process. Your completed application will be reviewed by our committee and organizations will be notified of the decision, the first round in late-October and on a rolling basis after that. Space is limited, however, so organizations are encouraged to apply early. FasterCures will provide successful applicants with a template for their presentations and Web-based training to help foundations maximize this opportunity.

You must register for the conference in order to apply for a slot. Click here for information on the registration and application process.

We very much hope you avail of this unique opportunity!

Join Medical Research Innovators at the Inaugural Partnering for Cures Meeting

"When it comes to medical research, never has it been more important to
think about the end game: getting therapies to people. To realize this, industry
and medical research foundations must work together early in the research and
development process, with philanthropy helping to pave the way."
- David Panzirer, Trustee, The Leona M. and Harry B. Helmsley Charitable Trust

FasterCures is committed to accelerating the process of turning exciting new medical discoveries into therapies that can treat patients. But, as you know, no one can do this work alone. Expediting cures requires collaboration. Join medical research innovators at the inaugural Partnering for Cures meeting. Uniting the passion of medical research foundations, the power of philanthropy, and the focus of biopharmaceutical companies, this meeting will catalyze a more effective and efficient medical research enterprise.
This first-of-its-kind meeting is designed to forge strategic cross-sector collaborations that will:

• empower philanthropists to measure the impact and return on their philanthropy;
• enable medical research foundations to find the right partners and investors needed to discover and commercialize new therapies; and
• facilitate biopharmaceutical industry partnerships that could yield cutting-edge medical solutions.

Register today and take part in this timely and crucial effort. Make one-on-one connections, share information, and meet potential collaborators through a customized partnering system designed to build relationships that could accelerate the pace of life-saving research and development. http://www.partneringforcures.org/
Space is limited, please register early. Significant discounts apply for early registration, special rates available for nonprofit medical research and patient advocacy groups.

How many business plans did Mother Theresa have? Probably none, but just imagine what she could have done if she had one!

by Melissa L. Stevens, Director of Special Projects, FasterCures

Almost 200 participants from over 20 countries convened a couple of weeks ago in London to talk about individual efforts to evaluate nonprofits and a possible mechanism to unify these efforts under a professional association. New Philanthropy Capital, a London-based charity research organization, and Bertelsmann Foundation, one of the largest German Foundations, hosted this conference Valuing Impact: Building an Association of Nonprofit Analysts. Inspiring discussions were interspersed with challenging questions about the community’ preparedness for such an organization.

Matthew Bishop, Chief Business Writer/US Business Editor of The Economist spoke about creating an impact in his keynote address. He said that in this time of economic crisis it is important for "philanthrocapitalism" to emerge, and that a critical role of “virtuous intermediaries” is to rigorously analyze what does and does not work. He underscored that this evaluation work needs to be funded and be democratized for all philanthropists, not just the super-rich.

Other highlights from the meeting include learning about other evaluation frameworks and ways for improving FasterCures' Philanthropy Advisory Service metrics and hearing first-hand accounts of how best to build models and establish evaluation organizations.

Appropriately, participants checked their rose-colored glasses at the door. There was challenging commentary and acknowledgement of significant obstacles in building the field of nonprofit analysts. One of which being the lack of data produced by nonprofits. Ken Berger, CEO of Charity Navigator, noted that in his pilot effort to measure outcomes (in addition to information gleaned from 990 financial data) he found that 85 percent of 4-Star charities could not produce the necessary data for an expanded evaluation. Scary. We also heard there are at least 150 different frameworks in existence, so already, there is significant divergence. Yikes. Finally, there were the weighty pauses after the rhetorical questions like “Who should pay for transparency?”, “Who is the audience – donors or organizations?”, and “Won’t this be done at the expense of programming costs?” Oh my.

But at the end of the day the delegates regrouped and voted on their interest in establishing an association and at least 75 percent indicated they would be. Perhaps it will be a soft launch with at least a bulletin board, listserv, and sector-specific working groups to think through common frameworks. I would be very pleased to join an effort that can quickly connect me to others who have pioneered evaluation frameworks, business models, and donor education efforts. PAS would greatly benefit from the collective thoughts of such a network of relative experts in the field. We look forward to seeing where the journey takes us from here and applaud New Philanthropy Capital and Bertelsmann Foundation for getting us out of the starting blocks.

FasterCures Report Applies Lessons of Social Entrepreneurship to Search for Cures

by Kristin Schneeman, Program Director, FasterCures
FasterCures has just released “From Social Entrepreneurship to ‘Cure Entrepreneurship,’” the latest in a series of reports aimed at injecting new, more strategic approaches into the conduct of medical research that hold the promise of accelerating results. The last report in the series, “Entrepreneurs for Cures,” laid out the critical need for innovative approaches to disease research and the important role nonprofit foundations can play in bringing creative thinking and new models to the enterprise.

Stemming from a novel gathering in November 2008 of individuals prominent in the fields of social entrepreneurship and of medical research, the new report poses the question, “Can we create an intersection between social entrepreneurship and medical research philanthropy – an ecosystem to support the work of cure entrepreneurs?”

The report features interviews with key opinion leaders, including:

• Lucy Bernholz, founder and president of Blueprint Research & Design, on the possibilities and limits of social entrepreneurship;


• Victoria Hale, founder and chair emeritus of the Institute for OneWorld Health, on the benefits of being part of a community of social entrepreneurs;


• Jason Hwang, director for health care at InnoSight Institute, on how to bring disruptive innovation to drug development;


• David Green, vice president at Ashoka, on innovative financing models for nonprofits;


• Brad Presner, metrics manager at Acumen Fund, on building a new portfolio data management system for use by all social entrepreneurs; and


• Elias Zerhouni, a senior fellow at the Bill & Melinda Gates Foundation and immediate past director of the National Institutes of Health, on the imperative for engaging patients as decisionmakers in – not merely subject of – the medical research process.

The field of social entrepreneurship has flourished over the last decade, attracting scholarship, media attention, and financial resources and building social and professional networks – as is evidenced by the success of organizations like Ashoka and the Skoll Foundation and the creation of curricula at business schools from Stanford to Harvard.

Medical research, however, is one area of nonprofit social endeavor that has been largely excluded from the dialogue and the discipline (with the exception of global health, where the focus has been largely on challenges related to the delivery of treatments). Yet medical research philanthropy is seeking to address a market failure no less important than other social enterprises: the development of therapies that cure disease and alleviate suffering. Today’s “cure entrepreneurs” are trying to leverage their relatively small dollars to help move promising research through the pipeline from basic discovery to commercial development of products that can help the patients they care about.

Social entrepreneurs aim not merely to serve people in need but to transform the dysfunctional systems that cause social problems. This is true of cure entrepreneurs as well.

Greg Simon, President of FasterCures, says, “The term ‘nonprofit research organization’ cannot capture the emerging culture these new entrepreneurs have created. They work in small organizations that are fighting against the comfortable habits and familiar traditions that dominate medical research in order to create disruptive change in the interest of saving lives. There are breakpoints in the medical research system that can only be resolved by a strategic rethinking of the fundamental relationship between the patients, researchers, academia, government, and industry. The cure entrepreneurs can play a key role in bringing about such a revolution.”

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

Life After 80: Always Looking Forward

To keep moving is to be vital, engaged, and engaging. A panel of four octogenarians agreed that the secret to a sweet good life, to longevity itself, is sharing, community, reciprocity, friendship and a sense of robustness. Being isolated and/or alienated reduces one’s receptivity to engagement and thwarts one’s focus. Not surprisingly, each of the panelists have made diet and exercise a priority.

It may have been more appropriate to say 80 is the new 30 judging from the stamina and recent accomplishments of the panelists.

A typical day in the life of these spirited 80+ clan would be ambitious for most and not for the faint of heart. Continuing to work was a common theme. John Sperling of Apollo Group, a Sierra Club member, is engaged in taking a revolutionary renewable energy product to market and founded a new solar energy company. Jim Pattison of The Jim Pattison Group captured it best when he said, "Retirement is not in the cards." Producer Norman Lear believes in planning for life changes. In his words, he is always "on to next." Deborah Szekely of Rancho La Puerta and Golden Door faces risk head on and believes these are the "vitamins and minerals of life."

Panelists share their commitment to philanthropy, each has a cause to champion and all believe in the power of giving. Lear is a benefactor of People for the American Way, Pattison has always carved out 10 percent of his earnings for his church, Sperling contributes to an array of scientific endeavors and Szekely helps new immigrants.

University of Southern California’s Marty Kaplan who moderated the panel, appropriately ended the discussion when he said, "In my family, in the Jewish tradition, on a birthday, one was wished a life of 120 years, regardless of his/her real chronological age."

View the panel online.

Read the session summary.

You Get What You Pay For?

By Melissa Stevens, Director of Special Projects, FasterCures
You wouldn’t choose a restaurant based on how little it pays its chef, or a surgeon based on how cheaply the hospital retains his services. So why would you choose a charity based on how low its overhead is?

Dan Pallotta is the author of the newly released Uncharitable: How Restraints on Nonprofits Undermine Their Potential. Pallotta spoke at a Milken Institute Forum on February 11 about his new work, which is described as “a manifesto that puts a new cause on the map - equal economic rights for charity.” He addressed the compensation disparities between for-profits and nonprofits, and challenged long-held conventional wisdom that overhead is a reliable measure of effectiveness.
Pallotta argues that charities are pressured into playing by different rules than for-profits, and that these pressures can stifle the organization’s productivity.
Consider:

• Compensation – Salaries for nonprofit executives are frequently criticized as too high. When nonprofits are compelled by external forces to tamp down executive compensation, recruitment quality is the first casualty.
• Advertising – Mass marketing is considered wasteful and frivolous in the nonprofit world – a costly and shortsighted judgment in the era of the multimedia-driven, 24-hour news cycle. The refusal to play in the mass marketing arena stifles charities’ ability to capture market- and mind-share, and cedes the stage to campaigns about the latest soft drink or i-product rather than malaria eradication or ending hunger.
• Vision –Nonprofits are incentivized to spend funds in the near-term to make an immediate impact, leaving them unable to invest in long-term, strategic initiatives, and sacrificing long-term gains for short-term returns.
• Learning – Nonprofits are given very little room for error in the court of public opinion. The climate of hard judgment creates disincentive for creativity and risk-taking. A risk-averse entity will never reach its full potential.
• Capital – Nonprofits are locked out of the financial markets because they do not pay financial returns. This leaves them continually dependent on what FasterCures’ calls “passion capital,” which only promises social returns.

Pallotta also articulated his frustration with the habitually asked “What percentage of my donation goes to the cause and how much goes to overhead?” Pallotta asserts that overhead actually is part of the cause. Investment in management and infrastructure are critical aspects for ultimate success, and should not be treated as incidentals. Further, he noted that measuring overhead does not give donors sound information about outcomes and the impact made by the organization.

Pallotta calls for developing a metric for assessing charitable giving. FasterCures agrees with this approach, and is leading the charge in the biomedical research space with the Philanthropy Advisory Service (PAS), an information resource that aims to create a transparent marketplace about nonprofit disease research organizations that fund and facilitate disease research. The PAS organizes and analyzes information about strategy, research portfolio, management, and financials. Even more importantly, the PAS creates an assessment framework to evaluate an organization’s internal and external contributions to the field. These assessments are made based on the organization’s own mission statements, as well the unique nuances of the disease research system.

The PAS framework organizes our assessment around the following metric categories:

• Accountability: The degree to which an organization engages in planning, demonstrates transparency, and upholds responsibility to stakeholders.
• Collaboration: The degree to which an organization can engage and nurture relationships that accelerate the overall funding and research cycle.
• Research Effectiveness: The degree to which the organization’s research portfolio yields sufficient data and deliverable returns to achieve its stated mission.
• Resource Building: The degree to which the organization contributes critical resources and infrastructure to scientific advancement.

FasterCures has engaged scientific and organizational experts to provide input to the organizational assessments, ensuring an informed, credible, and objective review. The PAS is currently being piloted across four disease areas – Alzheimer’s disease, multiple sclerosis, malaria, and tuberculosis – and is targeted to launch later this spring. FasterCures is excited about the PAS and its contribution to a new paradigm of nonprofit evaluation – the overhaul on overhead is long overdue.

FasterCures’ Ten to Watch in 2009: Bigger Bang for Our Research Bucks

1. Philanthropic capital. In this economic environment, investors are going to be more careful with their for-profit and their philanthropic investments. Nonprofit organizations – and particularly those that fund medical research, which requires significant dollars – need to embrace this moment to demonstrate their value. Is your organization ready to provide transparent and relevant information about your practices and results to help donors evaluate their investments?
2. Big pharma business model. Goldman Sachs is putting money into creating a “research pool” of early-stage products from a number of pharmaceutical companies. The discussion on how to build a business model around the targeted therapies of personalized medicine is getting more focused and serious. Is the veil lifting from big pharma’s eyes?
3. Stem cells. Forget about the politics, it’s time to talk about the science and the process. Now the urgent questions are how prepared are we to review, approve, and use stem cell therapies?
4. Cure entrepreneurship. Social entrepreneurship has attracted copious scholarship, media attention, and financial resources to support innovative approaches to solving social problems over the last decade. Now cure entrepreneurs, those pursuing novel approaches to accelerating the process of treating and curing disease, want to get in on the action. Can we create an ecosystem of human, intellectual, and financial capital to support the work of these innovators?
5. NIH U. The National Institutes of Health spends $3 billion every year supporting the work of about 6000 scientists in its Intramural Research Program (IRP), largely on its campus in Bethesda, MD. The IRP is an underutilized weapon in the arsenal of the U.S. biomedical research effort. NIH should articulate an overarching mission for the IRP and strategies for meeting goals over the next five years, focused specifically on advancing translational and clinical research in the interest of public health.
6. Patient power. The Internet empowered patients with information, and now social networking is taking it to the next level. What’s the power of informed patients gathering online to share their information and experiences? Can it help drive the agenda and speed up the pace of medical research?
7. Effective comparisons. There will be a lot of talk during the coming healthcare reform debate about comparative effectiveness. But we’re more interested in effective comparisons. A more efficient way of managing diabetes is a separate debate from a more effective option to treat cancer. Comparative effectiveness research should be about patient access to optimal care and continued medical innovation as solutions to healthcare challenges. Track developments of the Partnership to Improve Patient Care, a new group focused on addressing this.
8. Global health as foreign policy. We think that the President-elect gets that global health is not simply charity or aid and cannot be relegated to goodwill ambassadors. It is an unwavering commitment to medical research and access to cures that will end death and suffering. Global health done right demonstrates our leadership and diplomacy and is key to how we move forward on issues like national security, climate change, and trade.
9. Health IT: Stop talking, start typing. It’s long since time to face the reality that information technology will permeate healthcare with or without the right policies in place. So we might as well manage it from day one to make sure it’s accessible, integrated, and interoperable. We are after all building an infrastructure for 21st Century healthcare. We are all aching for a nationwide health information system that can improve patient care AND enable medical research.
10. Citizen action. This is the year of civic involvement. Americans are answering President-elect Obama’s call to be involved in a public way. How do we channel this activism to improve health? Can we inspire people to not only walk for cancer but to participate in a clinical trial or contribute in other ways to science?

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

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

Among the discussion’s highlights:

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

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

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

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

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.

Nonprofits Find and Fund Big Ideas in Small Spaces

Katie Hood, Michael J. Fox Foundation (MJFF) CEO in a recent blog post discusses getting higher returns on medical research philanthropy, and asserts that “private philanthropists need to break out of the collective comfort zone of a traditional, 'academics-only' approach.” Her organization and several others were profiled at the recent Biotechnology Industry Organization convention in two panels and via the partnering database. Although the models pioneered by groups such as MJFF, Cystic Fibrosis Foundation (CFF) and Multiple Myeloma Research Foundation continue to garner attention and are looked to as best practices, Ms. Hood also raises the important question of why there are not more examples being proliferated.
Our recently released report, Entrepreneurs for Cures, focuses on the need for and importance of more examples that will lead to medical breakthroughs, and notes that, “The measure of our success is lives saved and suffering diminished – not the number of grants awarded, publications presented, or laboratory space acquired.” This report highlights how nonprofit disease research organizations are finding and funding big ideas, effectively paving the way for developing new business models to accelerate research.
At an all-day session at the Institute of Medicine (IOM) on June 23, 2008, I spent the day hearing speakers on the topic of “Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies,” which spotlighted nonprofits as innovators in translating research from basic science to therapies for patients. A wide variety of topics were raised:

• for academic researchers: How can we get beyond the need to only share results via peer-reviewed journals?
• for diseases of the developing world: how can we raise the incentives to address them?

As part of this IOM meeting, I moderated a panel on “Strategies for Facilitating Sharing of Research Materials and Data” where Michael Mowatt, Director of the Office of Technology Development of NIH’s NIAID, talked about the imperative to share via repositories and other means. He highlighted the example of the Bill & Melinda Gate’s Foundations AIDS vaccine collaborations as an example to look to. Sharon Terry, President & CEO of the Genetic Alliance urged the audience to “Learn the rules so you can break them properly” and recounted the experience at the Alliance in “herding cats” to get research not just from bench to bedside but to get it into practice. She reflected that one way they herded cats was by moving the food. That would certainly get my cat’s attention. The Genetic Alliance repository was described as an “ebay” for samples. Laurie Ryan, Program Director of Alzheimer's Disease Clinical Trials at the National Institute on Aging, NIH, discussed the Alzheimer’s Disease Neuroimaging Initiative, an important public-private partnership which is gaining increased international attention.

In a panel on intellectual property, Craig Sorensen, Senior Director of Strategic Research Alliances at Vertex Pharmaceuticals outlined their work and collaborations in cystic fibrosis with CFF, Huntington’s disease with the Cure Huntington’s Disease Initiative, and tuberculosis and said that “industry is hiding behind cliches about failure and is doing a lot of running and staying in place.”

As Queta Bond, President of the Burroughs Wellcome Fund said at the end of the day, “We need to let more flowers bloom.” At FasterCures we’ll keep figuring out if we can help the process along and provide as much fertilizer as we can…
- Margaret Anderson, COO, FasterCures
Read and Comment

The Alzheimer's Fight

"I'm not going down without a fight," said Jackson, who often pleads for more money for research and treatment. "It's time to find, if not a cure, at least better medicine so people will live through this better." - Washington Post, Man With Alzheimer's Fights 'Family Disease': 5th-Generation Patient Copes With Early Onset, May 14, 2008.

Yesterday’s Washington Post highlighted the strife of Chuck Jackson, a man diagnosed with Alzheimer’s disease at age 50 and dedicating his life to advocating for research and treatment for the disease. The article notes that 5 million Americans live with this disease, but through our own research of this disease for the FasterCures’ Philanthropy Advisory Service program, we know that the Alzheimer’s Association projects this number to alarmingly double (and even triple) by 2050. This disease was the 7th leading cause of death in 2004 and results in $100 - $180 billion of economic costs for our nation each year.

Because there are so many unanswered questions about Alzheimer’s disease, there are even more areas of research to explore for answers and solutions, including:

• Etiology or cause of the disease, specifically with respect to the protein plaques and tangles in the brains of Alzheimer’s patients;
• Risk factors associated with patient populations, such as the genetic predisposition that Chuck and his family have towards this disease; and
• Biomarkers to better diagnose and monitor the progression of the disease.

Right now the only accurate diagnosis method is via brain autopsy.

Going beyond the current drugs available, there is research ongoing about treatement that target the protein plaques and tangles that are believed to be the fundamental cause of the disease, as well as how to best deliver care to patients suffering from this disease. Through the Philanthropy Advisory Service FasterCures hopes to help philanthropists identify high impact areas for Alzheimer’s disease research and channel nonprofit research funding to those areas. We also know that research and treatment of this disease could be tremendously helped through the development of a centralized map of research activities across sectors in order to identify priority areas for development. Other strategies include educating patients to build awareness of the importance of participating in clinical trials and engaging regulators about how to accelerate the approval of Alzheimer’s disease therapies.


We're glad that U.S. Senate Special Committee on Aging convened yesterday's hearing on Alzheimer's disease. We join the rest of the nation in anxiously awaiting the report of the Alzheimer's Study Group. We need a national strategy to accelerate research into new Alzheimer's treatments. We owe it to Mr. Jackson and to the more than 5 million people with Alzheimer's disease and their caregivers.
Melissa Stevens, FasterCures Director of Special Projects

FasterCures’ Ten to Watch in 2008

1. Return on philanthropy. When we invest our money, we look for the best ROI; when we donate it, shouldn’t we look for the best ROP? But how can we evaluate the effectiveness of nonprofits funding medical research? So far we’ve been looking in all the wrong places. Charity Navigator isn’t it – not enough information. New efforts like GiveWell definitely aren’t it – GiveWell’s approach of “cost per life saved” is the equivalent of what investors call “chasing performance.” So how are well-meaning philanthropists supposed to decide where their money will have the greatest impact in finding new cures?

2. The FDA. Yes, you heard me right, the FDA. If we want to get new therapies to patients faster, the federal agency whose budget we should be doubling right now is not the NIH’s but the FDA’s. After years of an expanding mission and a shrinking budget, in 2007 the FDA was finally given new authorities and some new resources to try to keep up with the tide of new technologies, methodologies, and products aspiring to or already in the marketplace. The question is, will Congress and the agency itself follow through on the promise? The first step was not promising, as Congress withheld funding for the Reagan-Udall Foundation, newly created to support FDA’s important Critical Path initiative. Everyone who cares about cures needs to be watching.

3. Research: Lost in translation. We all know we need translational research. Let 2008 be the year we define it, promote it, create more resources to support it, train people to do it, reward the ones who are doing it well.

4. Science 2.0. Collaborative science is the name of the game these days, as science gets bigger and more multi-disciplinary and the data available for research grows explosively. The technological opportunity presented by the Semantic Web for networking data and researchers will be transformative. Watch the Neurocommons Project, a demonstration of the power of the Semantic Web approach based on open access information.

5. Crowd-sourcing innovation. We’re all familiar with outsourcing – but how about opening up your R&D challenges to the “wisdom of the crowd?” That’s the approach taken by Innocentive, a Web site on which “seekers” can post problems to be tackled by a universe of “solvers,” many of them from entirely different disciplines. Born out of a need to open up innovation within the pharmaceutical industry, the site is now beginning to be used by groups like the “Prize for Life” challenge to find a biomarker for ALS, and the Global Alliance for TB Drug Development.

6. Prize philanthropy. A more top-down approach to such prizes is that of the X PRIZE Foundation. The Archon Genomics X PRIZE is engaging self-organized teams of scientists all over the world in the quest to make gene sequencing more widely available and affordable. What other challenges in health might lend themselves to this kind of high-profile effort?

7. Biomarkers Consortium. This initiative of the Foundation for the National Institutes of Health is a rare and promising partnership of federal agencies (research funders as well as regulators), the biopharma industry, and nonprofit research foundations, established in 2006 to discover, develop, and qualify biomarkers to support more predictive, preventive, and personalized medicine. With several projects off the ground and many more in the pipeline, this “pre-competitive knowledge commons” is a model which should be replicated again and again in the balkanized world of disease research.

8. Comparative effectiveness. Sounds like a no-brainer, right? We should study the effectiveness of medicines for treating a condition and use the ones that are proven to work best. But as the drumbeat around this buzz-phrase (and its companion, evidence-based medicine) gets louder in the coming debate about healthcare reform in this country, we need to make sure we understand its implications for patients’ access to treatments that work best for them, and for incentives to pursue new and even better therapies.

9. Electronic health records (EHRs). With “only” 17 months left in office, the Bush Administration last year decided to run out the clock instead of trying to win the game, shifting its focus from planning the structure of the “Internet for healthcare” to planning the structure of the government’s EHR bureaucracy two years from now. Sound weird? It is. In 2008 millions of people will be able to Google-ize their health records or deposit them in Microsoft’s Healthvault – leapfrogging not only the feds but their doctors’ offices, which are still photocopying their paper records. But who’s thinking about whether and how all this electronic health information might be accessible (or not) to the researchers trying to treat and cure what ails us? Watch this space.

10. Clinical trials: Something’s gotta give. The cost in time and dollars of clinical trials is crushing the discovery enterprise. Will 2008 be the year the FDA is convinced to break the mold and streamline of the clinical trials behemoth, allowing sponsors to adapt their course based on new information generated by the trial? What is the role of sophisticated computer models like The Archimedes Model in designing and simulating clinical trials? Do we just, in the words of a prominent clinical research leader, need to stop doing randomized controlled trials stupidly? One thing’s for sure – this is an issue in need of revolutionary, not evolutionary, change.

Greg Simon, President, FasterCures

GiveWell’s Not Getting It Well — Medical Research Is Not Charity

FasterCures President Greg Simon responds to The New York Times piece by Stephanie Strom “2 Young Hedge-Fund Veterans Stir Up the World of Philanthropy,”

The article states, “Mr. Karnofsky and Mr. Hassenfeld, both 26, founders and sole employees of GiveWell, which studies charities in particular fields and ranks them on their effectiveness. GiveWell is supported by a charity they created, the Clear Fund, which makes grants to charities they recommend in their research.”

After reading this, the third article I’ve seen about these refugees from Wall Street, I feel moved to point out a few things. First, it’s unfortunate that what has drawn attention to their work is using blogs to flame the admittedly flawed ratings groups like Charity Navigator. Second is this obsession of equating philanthropy with “charity.” I do not know any medical research group that considers itself a charity. They are nonprofit disease research organizations developing cures for deadly and debilitating diseases. Charity has nothing to do with it – these organizations represent investments – and yes I feel silly pointing that out to people who used to do research for hedge funds.

Once you realize organizations that are trying to save lives through research are an investment, you evaluate these organizations differently. You look at their strategies, their resources, their connections, board members, partners, and risk taking, and then you can create a diversified portfolio of disease cure investments going forward. That is what FasterCures is doing with our Philanthropy Advisory Service with grants from Gates and Robert Wood Johnson – and it takes time and more than asking a hundred questions and seeing who sends you their annual report faster than others – that really only measures receptionists.

Unfortunately, GiveWell’s approach of “cost per life saved” is the equivalent of what investors call “chasing performance.” Some groups may be efficient in handing out condoms but to discuss “charities” saving lives and not mention groups like IAVI that are developing vaccines for AIDS is like focusing on iron lung distributors instead of a polio vaccine.

We are all trying to get more out of our philanthropy, but saving lives requires strategies that try to create the future – and measuring that is harder than counting dollars and cents – or condoms.

Read the "Philanthropy 2173" blog for more information at: http://philanthropy.blogspot.com/2007/04/products-id-like-to-see.html