FasterCures Finds Outcomes, Metrics, and Strategic Leadership Define the Effectiveness of Venture Philanthropy Groups

Two new reports outline the practices and approaches of nonprofit funders of medical research

FasterCures today released two reports that paint a vivid picture of how medical research foundations, also known as venture philanthropy groups, have transformed the medical research enterprise. These entrepreneurial groups are accelerating medical research and development by tackling science where it needs the most effort and resources, and applying innovative business approaches in the pursuit for a cure.

Both resources tell a compelling narrative of how venture philanthropy groups are creating a culture in medical research that is mission-driven, results-oriented, and focused on the true bottom line: preventing, diagnosing, and curing disease.

• The first, Honest Brokers for Cures: How Venture Philanthropy Groups are Changing Biomedical Research, features insights from leaders of 20 venture philanthropy groups. This publication features valuable insights and perspectives from leaders of medical research foundations that are transforming the cure enterprise by virtue of how they find and fund research. In this report, FasterCures dissects the business model that has emerged from the shared sense of urgency and frustration stemming from these patient-driven organizations, and their laser-sharp focus on outcomes.

• The second, Measuring and Improving Impact: A Toolkit for Nonprofit Funders of Medical Research, is a how-to guide for foundations seeking to apply some of the best practices and lessons learned from venture philanthropy groups who’ve demonstrated their effectiveness.  It provides a common framework for assessing and improving organizational effectiveness, and a panoply of ideas, questions, and models to help guide new and emerging nonprofits with strategic and tactical choices.

Venture philanthropy groups play an outsized role in improving the medical research and development system to better meet the needs of patients. Since its inception, FasterCures has continued to shine a light on the successes of these foundations in an effort to amplify their lessons learned and sense of urgency to the rest of the medical research community.

“In the end, the most central characteristic of these groups is their close connection to the disease they are pursuing. For most, it is personal—either they or a family member or close friend is or has been affected by the disease,” said Margaret Anderson, executive director of FasterCures. “It spurs them to find the dollars to meet the challenges and change the trajectory of research. We can all benefit from the lessons they've learned.”


                                                  

To download these publications, visit http://www.fastercures.org/Publications/vp.php

 

Top 10 Medical Research Trends to Watch in 2013

By Margaret Anderson, Executive Director of FasterCures

As seen in The Huffington Post

1. Federal funding: Fasten your seatbelts, it's going to be a bumpy ride.

Congress has pushed the date of the "sequester" off another two months, delaying the prospect of automatic 8.2 percent cuts in the budgets of NIH, FDA, and other federal science programs. But a sequester (or other cuts) could still happen. Consider that current funding for these programs expires in March, the FY14 budgets are still being drafted, and there will likely be a fight over the debt ceiling. Advocates need to keep making noise to make a compelling case for the critical importance of medical research. The health of our citizens and the economic and fiscal health of the nation are at stake. Resources are available on our Sequestration Station website; also check out our Time Equals Lives social media campaign, where you can contribute your story to show our leaders why medical research matters, because we will all be patients eventually.

2. FDA: Recalibrating the benefit-risk equation. 

Patient groups have been saying for years that people living with a disease are often willing to accept more risk than the FDA is generally willing to tolerate in a new product. Well, now the agency can do something about it. In 2012, the latest reauthorization of PDUFA created a new "Patient-Focused Drug Development" initiative at the FDA, which allows the FDA to formally consult with patients -- initially in 20 disease areas -- about what their priorities are and what tradeoffs they are willing to accept. It may not sound revolutionary, but it could represent the beginning of a paradigm shift in the regulatory process if they get the process right, and if we don't get bogged down in fighting over what the 20 disease areas are. This will be an important initiative to track and be active in this year, and an important area for the FDA to focus on across its enters.
By the way, we heard at Partnering for Cures that nobody thanks the FDA -- so here we go! Way to go, FDA, on a second year in a row of 30-plus new drug approvals. If they do it again in 2013, is it a trend?

Saying thank you to FDA's Janet Woodcock

3. Financial innovation: Attack of the megafund. 

In October 2012, MIT finance professor Andrew Lo and colleagues proposed creation of a $30 billion "megafund" to bridge the translational research-finance gap by using financial engineering to securitize a highly diversified portfolio of therapies in development, an idea FasterCures got a preview of at its Financial Innovations Lab in July 2011. The model securitizes a portfolio of research assets as collateral to raise funds in the capital markets using a mix of debt and equity that appeal to a variety of investors' risk-reward ratios. Is this the kind of large-scale solution required for the big problem of financing risky research, particularly at the early stages? Lo plans a conference in 2013 to further develop the idea. Stay tuned for more work in this area by FasterCures and the Milken Institute in 2013, because this is one of the issues we are hearing the most consternation about.

4. Reimbursement: Can value drive innovation? 

Concerns in the pharma industry about the impact of reimbursement on their upstream R&D strategies are escalating, as efforts to rein in health-care costs are gaining steam. But do medical progress and the quality of care have to be sacrificed on the altar of cost-cutting? We heard some interesting discussion in 2012 from former pharma exec Frank Douglas and his new Austen BioInnovation Institute in Akron about the concept of "value-driven engineering" in medical devices, based on the principles of clinical utility, reduced complexity, and cost savings and efficiency. Douglas believes this framework can be applied to drug development as well. Can we shorten the time from molecule to hu(man) to ultimately drive down costs for payers? Will a focus on value be an innovation killer, as some fear, or can it be a disruptive driver for companies to tackle risky areas of high unmet need and first-in-class therapies? What is the methodology for deciding value? Where do patients fit into the equation? We look forward to more broad-based discussions of -- and hopefully more answers to -- these critical issues in 2013, as reimbursement could be our next cliff, if we survive the fiscal one.

5. Venture philanthropy: Playing in the major leagues.

FasterCures has been tracking and supporting the rise of venture philanthropy in medical research for almost a decade, and these strategic, patient-driven research funders have hit the big leagues and gotten the attention of the large pharma companies. Small companies have found them valuable sources of seed capital and other non-financial assets for some time, but large pharma has been slower to understand what they bring to the table. 2012 saw the Cystic Fibrosis Foundation (fresh off a big success investing in Vertex's newly-approved drug Kalydeco) invest $58 million in Pfizer's research into therapies targeting the most common CF subtype (yes, you read that right -- the foundation is funding Pfizer), and Sanofi awarded $300,000 to a team of patient groups to create a cross-disease registry to accelerate translational research. Companies like Merck Serono and Celgene are working with nonprofits like the National MS Society and the Leukemia and Lymphoma Society as "talent scouts" of sorts to identify promising early-stage research. As foundation head Bonnie Addario advised a company CEO at Partnering for Cures in November, "Put us on your boards -- we put you on ours!" We call that speaking truth to power.

6. Academic research: Let's see you do that again. 

Industry and investors are becoming increasingly frustrated with the lack of reproducibility of positive results from academic science published in peer-reviewed journals. In 2012, Amgen could not reproduce the results of 47 out of 53 so-called "landmark" oncology studies, and in 2011 Bayer discontinued 65 percent of its target validation projects when its results did not match published experiments. Some patient groups, like the ALS Therapy Development Institute and the Chordoma Foundation, have made replication of published results and validation of research products a core piece of their missions. Some investors are funding their own "killer experiments" before making investment decisions, and efforts like Science Exchange's Reproducibility Initiative are cropping up to find ways to systematically address the disincentives for academic researchers to validate their findings. Expect this debate to get louder in 2013 -- and hopefully coalesce around some productive solutions.

7. Data standards: Coming soon to a therapeutic area near you. 

We hear over and over again that medical research desperately needs standards for collecting and analyzing data, but it's always seemed like a bite too big to chew. But the Critical Path Institute and CDISC are stepping up to the plate with CFAST, the Coalition for the Advancement of Standards and Therapies, in partnership with the FDA and the new industry consortium TransCelerate. We first heard about it at Partnering for Cures 2011, and it formally launched in October 2012, but it already has standards in seven therapeutic areas (out of 55 targeted) in various states of development. Watch for accelerated progress in 2013.

8. The other translation gap: Turning outputs into outcomes. 

FasterCures has spent a lot of time focusing on the "first translation gap," between basic scientific discoveries and research in humans subjects; somewhat outside our scope has been "T2," the gap between approval of a treatment and its adoption into widespread use to improve patient outcomes, which can take as long as the R&D process. Many others have analyzed this problem, but the ImproveCareNow network has gone from analysis to action, creating a rapid learning system that is speeding the adoption of evidence-based care practices for kids with Crohn's disease and ulcerative colitis at almost 50 care centers across the country and improving their health dramatically. The Kauffman Foundation is working to scale and replicate their success through its Health Network Trust initiative. We hope 2013 will see this model launched in other disease areas.

9. Collaboration: Writing the playbook. 

There now seems to be broad acceptance of the idea that collaboration among R&D stakeholders (government, academia, industry, nonprofits) is required if we are going to make progress in an increasingly complex scientific and business environment. There are many experiments in collaboration going on, and many successful outcomes that can be highlighted. It is time for more systematic analysis of what's working and what's not. While these efforts vary widely depending on the goals and the collaborators, can we draw some common lessons about, for example, what are fertile areas for collaboration, what one should look for in partners, what are the pitfalls to avoid? After all, as Luke Timmerman of Xconomy wrote recently, what we're after isn't collaboration for its own sake, it's a "creative rethinking of the biomedical R&D continuum," and "while those [collaborative] efforts are encouraging, this really should be a national conversation that involves a whole lot more players." Will 2013 be the year we start trying to know what we know, and perhaps even start redrawing that linear R&D pipeline we all know so well?

10. Innovation: Who's outside the box (you tell us). 

While there's no shortage of brilliant scientists and discoveries in medical research, it can be notoriously hard to move them forward in the expensive environments of academia and industry, where risk is not necessarily rewarded. We are seeing more efforts to try to bust out of this box in ways that are more common in other fields. Philanthropist Peter Thiel, who started out by giving young people $100,000 to skip college and work on their ideas, has established Breakout Labs to fund early-stage companies with radical ideas. The National Institutes of Health is getting into the action with its "skip the post-doc" Early Independence Awards to help free exceptional young minds from the academic treadmill. Jimmy Lin's Rare Genomics Institute is crowdfunding genomic sequencing for patients with rare diseases -- and there's much discussion about crowdfunding's utility in life sciences. Crowdsourcing is gaining traction in life sciences, from FoldIt's online protein folding games to Transparency Life Sciences' crowdsourced clinical study design. Who will be redefining "the box" in 2013? You tell us -- send us your ideas at info@fastercures.org.

Using IP Strategies to Break Down Barriers to Progress

Intellectual property (IP) protection is critical to driving investment in new products that can help patients as well as create economic value. Some maintain that IP protection can make scientific collaboration, technology transfer, and commercialization more challenging. At Partnering for Cures, five leaders in biotechnology, science, and intellectual property joined together to discuss how IP could aid the pace of innovation and technology to get to patients who most need it.

Moderator Maria Freire of the Foundation for the NIH began by pointing out that some people “go into panic attacks when having to deal with intellectual property … it seems like a big hurdle between basic science research done in universities or companies and getting it to the market.” However, this is not a new issue. The biggest change is that, within the last 10 years, new players have emerged: venture philanthropies. Freire referenced FasterCures’ newest publication, Unlocking Intellectual Property: Principles for Responsible Negotiation, which serves as a useful tool for all parties in biomedical research, in particular these new nonprofit disease groups and philanthropists.

In addition to the arrival of new players, Stephen Johnson of Kirkland & Ellis LLP and One Mind for Research emphasized that the IP landscape has changed due to the arrival of new technology. In the past, IP focus was on patents, but the “focus has moved away from patents and toward data,” he said. He used to see resistance to sharing data among pharmaceutical and biotechnology companies. Now, “there is acceptance of the pre-competitive space,” and companies increasingly embrace the opportunity to work together. “The future of creative collaborations will be balancing openness with incentives,” he suggested. Freire summarized that companies are more willing to share all data in the beginning, but once there is true innovation, they will then put protection around it. Johnson agreed with her assessment and advocated for processes that promote transparency. He said that one of the reasons that companies would be reluctant to share data is that they are worried that someone else who has access to the data is smarter than them.

Stephen Friend of Sage Bionetworks gave an example of another hindrance, stating that “many companies don’t feel like they can share what they’re talking about … and it’s hard to get things financed without a clear IP strategy. Many worry that someone else could come in, grab the idea, and take advantage of them.” He supported extending the pre-competitive space: “Too many post-docs think they are working on the next billion-dollar drug long before it is.” He cited the successful collaboration of Merck, Pfizer, and Lilly, who generated data in China and agreed that they would share the data among themselves for one year and then make the data publicly available.

Teresa Stanek Rea said that the U.S. Patent and Trademark Office also has a cooperative approach: “We are trying to collaborate with companies to find out what they need to do their job.” Like industry, the Patent and Trademark Office is trying to be more precise and more efficient in what they do and sees itself as the innovation agency in the U.S. government. “We are an agency in the throes of change, just as you are,” she said. Rea noted that the America Invents Act, puts forth “great provisions that help the user community because it takes a second look at issued patents, and whether the patent should have been granted.” Rea believes that the act should minimize litigation and not inhibit research.

Steven Tregay of FORMA Therapeutics brought the conversation back to venture philanthropies and emphasized the importance of being “cognizant of whether the patent that covers the product can actually be translated into treatment.” The key is helping people understand the value for society versus owning one possible combination that may be pertinent or may never be turned into a drug. He cited the success of the CoMMpass project of the Multiple Myeloma Research Foundation, a collaborative study that brought together a network to decide how IP will be shared and who has access to it. Tregay has also worked with the Leukemia & Lymphoma Society (LLS), which aggregates data from many organizations at once. LLS is concerned with creating value to patients and creating a path forward, and it shouldn’t be burdened with the “nightmare of bringing universities together,” he said. “That is the power of these disease foundations – that they are really laser sharp in their approach,” said Freire. “They don’t want to fix the world; they want to fix something for that indication. The traditional paradigm may not necessarily apply.”

Friend agreed that traditional ways of doing business may stymie dialogue and interaction. “We are at a spectacular time where we have tools, new approaches in order to innovate, and yet the way we have structured our incentives and our rewards around sharing, around who is getting credit, et cetera, is basically independent to that.” He cited the success of CommonMind, a public-private pre-competitive consortium that generates and analyzes large-scale genomic data from human subjects with neuropsychiatric disease and makes the data and results broadly available to the public. Friend said that university tech-transfer offices had a hard time at first agreeing to share data being generated, but that “the data required to build the models needed to develop the drugs had to be accessible in order to innovate.” The parties created collaboration agreements that allowed investigator data to be shared with others and not kept to themselves. “We must come up with incentives and rewards that allow the data to get out there,” he said.

Joseph DeSimone of the Frank Hawkins Kenan Institute of Private Enterprise at the University of North Carolina agreed that “partnerships are what work the best, and new connections should always be made,” but cautioned that “without really clear IP, it’s getting increasingly hard to get things financed. Having a really clear IP strategy and path to market is going to be increasingly important.” His university has a conflict of interest committee that meets with him and his students who start companies. He believes that transparency of partnerships makes it more successful: “If you are open to that kind of openness, it can be powerful to enable these kinds of connections.”

In closing, the panelists discussed the future of intellectual property protection with regards to innovation. There was a consensus that patenting had gotten more difficult, and Freire ended by saying “Let’s not rediscover wheels. If you can put something in a box, it’s a lot easier. If you want to think outside the box, just make sure that what you have already doesn’t fit in an existing box.”

Related resource:
Unlocking Intellectual Property: Principles for Responsible Negotiation

Turning patients into partners

Bonnie Addario is a lung cancer survivor with an extensive family history of the disease. When she was diagnosed, she wanted to do something to help further the search for a cure. She thought she might hold some fundraisers and turn the money over to her oncologist. Instead, she formed two foundations that advocate, fundraise, and perform research.

That should be the modern model of a patient advocacy group, according to the Partnering for Cures panel discussion, “Powered by Patients.” The panel looked at the new breed of patient groups, weighed their importance in the process, and shared best practices of bringing patients into the R&D arena.

Patient advocacy groups are powered by the people who have the diseases or their family members, making them “wonderfully, positively motivating,” said National Institutes of Health Director Francis Collins. Families often get involved to expedite slow or nonexistent movement on disease research. Addario noted that when she was diagnosed, the survival rate for lung cancer hadn’t changed in 40 years. When Scott Johnson of the Myelin Repair Foundation was diagnosed with multiple sclerosis, he was told there would probably be a treatment within the next 25 years. But, 25 years later, there wasn’t.

Johnson looked into the medical research ecosystem and realized there was no cohesive manager of the research and translation process. For him, it made sense for a nonprofit to step into that role. “A nonprofit has more freedom to operate, fewer constraints,” he explained. Nonprofits can be the convener and manager of the process. His organization began by convening partners in academia, developing a research plan, and working closely with their own scientific team on research. The next step was to bring in pharmaceutical companies. Johnson said his organization does the work to validate and de-risk discoveries made in academia so they will be up to industry standards and will be able to be replicated. “We know we have to give them compelling enough data to get their interest,” he said of pharma companies.

Other stakeholders say the involvement of patient groups brings value to the research and translation process. Dennis Urbaniak of Sanofi reported that his company is changing the focus of how it runs its business to better serve communities in different disease areas. He said they want to listen to people who live with the diseases they treat and bring them into R&D by crowdsourcing questions on care to learn how patients actually want to be cared for.

“Most scientists welcome the opportunity to have that connection with what they are working on,” explained Collins. “They may have never been in the same room with someone who has the condition. It is enormously inspiring.”

N. Anthony Coles of Onyx Pharmaceuticals works with the Multiple Myeloma Research Foundation (MMRF) on a variety of projects, including leveraging MMRF’s clinical trials network to collaborate with patients and get information to them about trials, speeding up the enrollment process.

Onyx also invites patients to visit the company two or three times a year, “so we don’t ever lose sight of who we work for,” Coles said. “The most successful patient engagement groups are led by someone with personal experience,” he said, adding the hope that everyone who touches healthcare decisions has the kind of “transformational experience” through meeting patients he had in dealing with his son’s illness.

Patients want to contribute, stated Addario, but they don’t feel valued. “Until we invite them to our tables, to our board rooms, to the places where we are making decisions about their lives, they won’t feel valued,” she said.

She encouraged stakeholders to view patients in a new way. “We have to start to look at patients as someone who has the solution within their body. The biomarkers are there. All the keys to open the door are there in the patient,” she said.

The Patient-Centered Outcomes Research Institute was created as a provision of the Affordable Care Act to look further downstream in the translational pipeline, said Collins, and to figure out what works in the real world and in the minds of patients. “We want to collect information in a way that captures the patient experience and compares it across studies,” he said.

Lastly, Collins’ wish list included open access of information. “We can all go faster if we have the information in front of us, if we aren’t wasting our time doing experiments that already failed,” he said. He is encouraged by the strong movement to ensure that research publications are available to those who need them, and he is optimistic about the amount of clinical trial information available on clinicaltrials.gov.

Patient groups bring value to the ecosystem that can’t be ignored. To conclude the panel, moderator Susan Dentzer of Health Affairs summed up the panelists’ final sentiments: “If you are not bringing patients into your organization, you’re not just a dinosaur, you’re extinct.”