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.