New cross-sector deal-making tools and resources on TRAIN Central Station

by Kristin Schneeman, Program Director, FasterCures

Nonprofit foundations in our TRAIN network are perhaps more concerned than most folks about not “reinventing the wheel,” not spending time and resources learning things and making mistakes that others have already made. Financial and human capital is especially precious at these organizations, and inefficiency has significant consequences for the patients they serve. We have always featured on TRAIN Central Station resources that groups have shared with us that they feel others can benefit from having as a starting point for their own work.

We’re pleased to announce that we now have available on the site a treasure trove of new tools and resources, shared by participants in a workshop we held in June on “The Nuts and Bolts of Cross-Sector Dealmaking.” This cache includes template agreements of all sorts; due diligence guides; publications on collaborative models, venture philanthropy royalties, and charitable investing; and much more. 

Among the documents and resources available are:

• The master agreement between the Addario Lung Cancer Medical Institute and the 15 academic institutions that are part of its consortium;

• The Chordoma Foundation’s guidelines and agreement for $10,000 prizes for the creation, validation, and deposit of new chordoma cell lines;

• “Everything You Ever Wanted to Know about Royalties and Their Monetization but Were Afraid to Ask,” a new publication by the law firm Schaner & Lubitz;

• A set of model legal documents for venture capital investments put together by the National Venture Capital Association, a window into the priorities of companies and investors with whom foundations may be interested in partnering.

This is just a sampling of the more than 50 new tools and resources available. We hope you’ll find these and the others available on TRAIN Central Station useful models for your own efforts. If you have a resource, internal or external, that you would like to contribute or recommend to the community, we hope you will send it to us at train@fastercures.org!

Interested in engaging with a medical venture philanthropy but not sure where to start? Try here

Forward-thinking philanthropic funders of disease research can play an absolutely critical role in stimulating medical progress, particularly in under-resourced areas, and helping to bridge the Valley of Death. Free of external pressures, nonprofit foundations are ideally positioned to make relatively high-risk investments that could significantly move a field of research forward and increase the likelihood that other parties also will invest.

Today FasterCures launched a first-of-its-kind resource designed to help potential collaborators better understand the landscape of nonprofit disease research foundations and engage in meaningful partnerships with them. The Research Acceleration and Innovation Network (TRAIN) Inventory is a free, Web-based index that catalogues the operational and partnering practices of over 50 leading nonprofits that find and fund cutting-edge medical research.

As the medical research community continues to explore new business models, we can all learn from the approaches these organizations have taken to speed innovation. Our goal is to amplify what they’ve done, share what they’ve learned, and help those interested in engaging them determine how best to connect. The Inventory succinctly provides information crucial to understanding and working with over 50 of the most impactful foundations in this space.

With downloadable profiles for every organization that participates in FasterCures’ TRAIN initiative, the Inventory uses a common set of metrics to outline and compare each organization’s research portfolio, collaboration efforts, financials, and more. It also highlights the great medical advances these organizations have fostered over the years, often in collaboration with partners from other sectors.

For example, did you know . . .

• Multiple Myeloma Research Foundation (MMRF), through a partnership with Onyx Pharmaceuticals, provided the clinical trial support and resources necessary to develop the first new treatment for multiple myeloma in five years, Kyprolis. Trials conducted through MMRF’s Research Consortium are opened an average of 60 percent faster than the industry average.
  
• The Alzheimer’s Drug Discovery Foundation (ADDF) has granted more than $51 million to fund more than 370 Alzheimer’s drug discovery programs and clinical trials in academic centers and biotechs in 18 countries.
  
• A collaboration between the Cystic Fibrosis Foundation (CFF) and Vertex Pharmaceuticals brought about the cystic fibrosis drug Kalydeco, which received FDA approval in January 2012. CFF provided funding, information about the patient population, and helped recruit patients for clinical trials.
  
• Through a partnership between the Translational Genomics Research Institute and the Virginia G. Piper Cancer Center, the Center’s clinical trials program is currently running more than 50 active clinical trials for advanced and rare cancers.
  
• The Michael J. Fox Foundation (MJFF) for Parkinson’s Research engaged 3,000 volunteers across 20 countries in studies to speed development of LRRK-2 related therapies for Parkinson’s disease.
  
• Thanks to Autism Speaks’ advocacy efforts, 29 states have enacted autism insurance reform laws.

These are just a few examples of model practices among our TRAIN groups. To learn more about them and the other organizations in the network, check out our inventory – and pass it on!

'Translation is a Team Sport'

The Learning Collaborative, a partnership between the Leukemia & Lymphoma Society (LLS), University of Kansas Cancer Center, and the National Center for Advancing Translational Sciences (NCATS) at NIH, is attempting to break the traditional drug discovery and development paradigm by bringing together blood cancer, drug discovery, and development expertise across several organizations. The Collaborative was formed with the goal of targeting repurposed drugs as well as novel, new drugs for the treatment of rare blood cancers.

Last week at a TRAIN (The Research Acceleration and Innovation Network) Webinar, the leaders of this unique effort shared what they’ve learned since they signed the Cooperative Research and Development Agreement (CRADA) in 2010. CRADA defined the resources and expertise each collaborator brought to the effort.

The three partner organizations bring complementary strengths to this collaborative. LLS has ample experience working with industry partners, an established network of experts, about 400 active research projects to contribute, and the sense of urgency that patient-driven foundations uniquely bring to the table. The University of Kansas Cancer Center excels in “bench to bedside” translation in drug repurposing, leadership in chemistry, and pharmaceutical experience. NIH’s NCATS brings a focus on rare diseases, industrial scale capabilities, and pharmaceutical experience.

“While some progress has been made in combating these cancers – half of the FDA-approved drugs of the last decade were approved for use in blood cancers – 50 percent of those newly diagnosed will die from their disease within five years,” said Louis DeGennaro, PhD, chief mission officer of the Leukemia & Lymphoma Society.

Scott J. Weir, PharmD, PhD, director, Institute for Advancing Medical Innovation, University of Kansas Cancer Center, gave a snapshot of the first Learning Collaborative effort, the auranofin project, and shared key insights:

• Keeping the patient in mind will accelerate the process and ensure their needs are front and center in the research agenda.
• Defining the collaboration, setting collective objectives, and managing expectations are fundamental.
• Project management is critical, both to manage across organizations and to maximize appeal to industry.
• Tech transfer must be integrated into teams to optimize the leverage of the data.
• Regulatory science issues that will impact their chances of success need to be addressed in the course of the research.
• Defining exclusivity paths and reimbursement strategies from the earliest stages is central to the effort. The group is working on what they call a “ValueMaP” (Value Maximization Path) to help define the value proposition for drug repurposing.

Currently, there are four active projects, two of them involving repurposing rheumatoid arthritis drugs for use in rare blood cancers (the other two are in earlier stages). The goal is to advance projects to clinical proof of concept within 14 months and to engage industry partners along the way.

This model, said Christopher P. Austin, MD, director, Division of Preclinical Innovation, National Center for Advancing Translational Sciences, can be applied to other organizations, and other diseases. “Translation is a team sport. It needs to be approached that way, and very consciously and deliberately managed that way.”

Documents critical to the Collaborative formation will be made available on FasterCures’ TRAIN Central Station soon.

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

by 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?

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.

enture 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.

NCATS: 'Disruptive Innovation' at NIH

More than 650 participants tuned in to a Webinar on March 16 hosted by FasterCures with National Institutes of Health Director Francis Collins, who gave the latest updates on the progress on the proposed National Center for Advancing Translational Sciences (NCATS), answered questions, and sought to dispel myths about this effort. Participants were from 38 states and represented all medical research sectors – more than 200 patient advocates from 100 different organizations, nearly 200 academics from 78 research institutions, 62 from biotechnology and pharmaceutical companies, 82 from various government agencies, and dozens of other stakeholders from the venture capital community to policy influencers.

Calling NCATS “a disruptive innovation, in a good way,” Collins said the motivation for the new center is “the need to view the drug development pipeline as a scientific problem, ripe for experimentation and process engineering.” He noted that now is the time to act as opportunities for new drug targets to be tackled successfully have been growing well beyond capacity of the private sector to take on by itself and that the appetite for public-private partnerships is high. Engaging in translational research is not new for NIH, he said. A 2010 survey showed 550 translational research projects going on at NIH (65 percent preclinical and 35 percent clinical), and a recent New England Journal of Medicine article reported that 153 new therapies have been discovered through research carried out in public-sector research institutes, most of which have been supported by NIH.

But he also acknowledged that the productivity of the medical research system overall is declining, and stated his belief that NIH – by facilitating and supporting partnerships, providing resources, and enhancing training – has an increasingly important role to play in moving the results of promising research it funds down the pathway toward new treatments. “The opportunity for new targets is growing well beyond industry’s ability to address them all. … In addition to neglected diseases, for many common diseases there are ‘neglected targets,’” which NCATS can help de-risk and make attractive for investment. He also believes that NCATS could be “quite catalytic” in helping repurpose abandoned compounds, and mentioned that NIH will be holding a meeting with biotech and pharmaceutical executives in April to discuss such ideas.

Collins was emphatic that NCATS is intended to complement, not compete with, the private sector. “We are not turning NIH into a drug development company,” he said, contrary to some headlines that the initiative has generated. He also took care to say that there is no intent to take focus and resources from NIH’s basic science mission and programs. As a matter of fact, he believes NIH’s investment in translation can empower basic research.

FasterCures Executive Director Margaret Anderson moderated this virtual meeting, noting that NIH’s effort to make translational research a priority is significant and meaningful for the thousands of patients with a deadly and debilitating disease for which there are no viable treatments or cures -- especially when you consider industry’s estimate that for every 5,000 compounds tested, only five make it to clinical trials, and only one is ever approved by the Food and Drug Administration. Patients are anxious for – and deserve – better returns on our nation’s research investment.

Collins appealed for all research stakeholders to be reasonable about expectations for this new effort, saying that NCATS should not be judged by the number of drugs ultimately approved. He ended by saying, “This is science, so there will be surprises along the way. But there is an opportunity to do something historic here.”

NIH is actively seeking feedback on the proposed structure and functions of NCATS at a new Web site. An archive of the FasterCures Webinar is available for viewing.

Welcome to TRAIN Central Station 2.0

by Kristin Schneeman, program director, FasterCures

Welcome to TRAIN Central Station 2.0, a new and improved version of FasterCures’ online platform for venture philanthropy in medical research. We’re excited to be bringing you lots of frequent updates and fresh content in the coming months, including:

• Monthly Webinars, beginning on March 16, 4:00-5:00 pm ET with a special appearance by NIH Director Francis Collins, engaging in a Q&A on the proposed new National Center for Advancing Translational Sciences. April’s Webinar will feature legal experts Ken Schaner and David Lubitz on “Unlocking IP at Academic Institutions.”
• New interviews with trailblazers in our Innovator Spotlight. Read the latest with Alpha-1 Foundation’s John Walsh.
• The good old E-News, back on a monthly schedule and chock-full of interesting stuff. This month, we highlight Institutional Investor magazine’s piece on venture philanthropy; an open letter to the pharma industry asking them to contribute discontinued compounds to a central repository; and the launch of HealthResearchFunding.org, which aims to connect funders with peer-reviewed, meritorious research proposals.
• Links to case studies and best practices. Check out the smorgasbord of fascinating presentations on cross-sector collaborations at December’s Partnering for Cures conference.
• Occasional guest blogs from people we find fascinating.

We want this to be not just a megaphone for FasterCures, but a community-building tool, and for that to happen, we need you.

• Use the discussion board as a place to solicit others’ ideas on how to handle problems you’re facing, or share information about issues of concern to you. While you’re there, offer up your questions for our Webinar with Francis Collins on March 16!
• Share with us any tools or resources you’ve developed at your organization (e.g. legal documents, project management tools, etc.) that you think might to useful to others in doing their work and we’ll post them in our Tools and Resources section.
• Send us your calendar items and job postings that might be relevant to the TRAIN community and we’ll share them on the site.

As always, we look forward to your feedback on how we can continue to improve the site, as well as your contributions of material for it. Enjoy the ride!

Innovation Nation: State of the Union Emphasizes Science and Technology as Engines of Growth

by Gillian Parrish, Manager, Alliance Development & Communications

“Innovation doesn’t just change our lives; it is how we make our living.” – President Obama

During the State of the Union address, President Obama extolled the virtues of science and technology in advancing America’s strength and ensuring our competitiveness in the global economy. He spoke of his administration’s commitment to making new investments in biomedical research and IT, and training an additional 100,000 science, technology, engineering, and math teachers by the end of the decade.

This focus on scientific innovation goes a long way in making the search for cures a national priority. As Dick Gephardt and Mike Leavitt, co-chairs of the Council for American Medical Innovation (CAMI), said in their editorial in The Hill, “nowhere is there a better value proposition for America than the biosciences.” This echoes a sentiment expressed in the New Biology for the 21st Century report, released through the National Academy of Sciences, which calls for greater integration across the sciences – biology, physical, computational, and earth, as well as mathematics and engineering – to find solutions to four key societal needs: sustainable food production, ecosystem restoration, optimized biofuel production, and improvement in human health. The report outlined the need for a coordinated effort to leverage resources across the federal, private, and academic sectors to help meet challenges and improve the return on life science research in general.

For more than 100 years, the United States has been synonymous with medical discovery. In fact, the Milken Institute estimates that 50 percent of all economic growth is the result of lives improved or extended by medical science. But the system that made all that progress possible in the last century needs to now work for this century. Medical outcomes could be greatly improved if we spend the same kind of manpower and resources we spend on consumer products on discovering and developing new cures for disease.

To regain and maintain our global scientific leadership, and to speed the path to cures for patients who need life-saving treatments and therapies, FasterCures stands at the ready to work with the government and all sectors to advance the following:

• Increasing resources and scientific capacity at the U.S. Food and Drug Administration to support more efficient and effective approval of new medical solutions
• Advancing policies that foster innovation and translation at the National Institutes of Health
• Encouraging innovative, cross-sector collaboration among all players in the medical research enterprise

All stakeholders, public and private, have a critical role to play in this process. What are we waiting for?