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Institute of Medicine (US) Forum on Neuroscience and Nervous System Disorders. Venture Philanthropy Strategies to Support Translational Research: Workshop Summary. Washington (DC): National Academies Press (US); 2009.

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Venture Philanthropy Strategies to Support Translational Research: Workshop Summary.

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4Partnerships, Data Sharing, and Intellectual Property

In session four, the hypothetical voluntary health organization, the Colten Foundation, wants to expand its portfolio of contributors and partners in order to develop an effective research program. This has led to many questions around data sharing, partnerships, and intellectual property. What should the Colten Foundation consider as it builds its program? Workshop participants discussed the answers to this question in the context of best practices and lessons learned.

A fundamental task for voluntary health organizations supporting a translational research program is creating and managing networks and partnerships. The importance of collaboration and coordination between academics, the National Institutes of Health (NIH), voluntary health organizations, and the private sector in the work to find cures and treatments could not have been underscored more during the meeting. Everyone in the medical and philanthropic community relies on formal and informal partnerships. There are significant challenges to forming these partnerships, and understanding how to navigate these challenges is just as important as having resources to invest.


Academic Partnerships

Often there is a gap between the investigation of disease mechanisms in academia and drug discovery in a corporate pharmaceutical world, said Linda Van Eldik, professor in the Department of Cell and Molecular Biology at Northwestern University Feinberg School of Medicine. She believes that there needs to be open communication around the fundamentals of what an industry partner is looking for in the drug discovery process from the research done in an academic setting. When the final recipient of the research is a company that will ultimately need to mass produce a drug, it is essential to get academics on board with the drug discovery mission and process, Van Eldik explained.

A challenge in partnering with academia is cultural, particularly surrounding the tenure process. If modern science requires unfettered collaboration and information flow, this creates a real barrier to investigator and institutional buy-in. Van Eldik believes that the issue can begin to be addressed by getting the institutions behind the translational research process, which is not always valued in the tenure track culture of academia. A major question that arises is how to do “team science” and how individual investigators get credit for team science. What is required is creativity and compromise from all parties. “Academics do want to do this,” she said. “They want to translate their basic science discoveries. It’s just [a matter of] figuring out the best way to partner with the appropriate people.”

Partnership with the National Institutes of Health

Any discussion about funding for medical science inevitably turns to the NIH. Partnering with the NIH gives a voluntary health organization’s efforts credibility in the research process that might be missing if the agenda were driven solely by the organization, according to the workshop participants. Further, voluntary health organizations and the NIH are not competing entities but are instead tremendously complementary.

Many expressed that the NIH is essential to biomedical research, and while not traditionally focused on funding applied science, this is changing with relation to new drug development. Consequently, there exists an opportunity for voluntary health organizations to address this gap and fund more applied science, according to Katie Hood, president and chief executive officer of The Michael J. Fox Foundation for Parkinson’s Research.

A natural way for a new voluntary health organization to enter the research process is to participate in collaborative projects with the NIH on early-stage research. The interaction has the chance of being mutually advantageous, as the NIH may also benefit from the work of organizations. When collaborating, these entities support each other: The NIH provides critical research funding in areas such as biomarkers, while voluntary health organizations can help guide the NIH agenda by sharing the results of translational research.

Building Credibility Through Partnerships

A voluntary health organization is often the most obvious, most easily accessible expert in a disease space. In this role of disease expert, an organization such as the Colten Foundation can bring tremendous value to a partnership with a for-profit company. Peter Heinecke, chief business officer of Experimed Bioscience, Inc., suggested that if an organization does its due diligence and decides to invest in a particular area or company, venture capitalists take note of that and are more likely to also invest. A voluntary health organization probably already knows far more about a disease area than anybody in the venture capital firm does or is going to be able to learn.

But that role as disease expert is just one bargaining chip in what is always a negotiation. There might be many restrictions that an organization is going to want to put on the company, said Heinecke, adding that the challenge is to discover the common ground in any given deal. “In the end you have to come up with a package that is balanced, that overall is an attractive option for the company.”

Liaison to the Patient Community

Tricia Brooks, managing director for alliance development at BIO, believes that some voluntary health organizations are particularly good at what is often a missing link in the process: working with the medical community and patients once a treatment is commercialized. As we move toward personalized medicine and look at these better-targeted therapies, we are going to need someone to play that role in education, Brooks said.

An example is Herceptin, a treatment for breast cancer that is used in highly personalized medicine. Breast cancer patients with the HER2 gene can take Herceptin to reduce the odds of recurrent cancer. Many patients with breast cancer do not have the HER2 gene, yet they demand Herceptin from their physicians because they believe it to be a blanket therapy. It is a classic case where better education is needed, and a case where breast cancer foundations are working to deliver the simple message. This type of patient education will become more common as more targeted treatments are discovered for various diseases, especially as treatments cross boundaries between pharmaceutical manufacturers or involve cocktail treatments.

The direct connection that voluntary health organizations have to patients is perhaps the most valuable contribution an organization can make and enables awareness of the disease from a patient perspective. “You bring expertise that no company can walk in with,” Brooks advised. A patient organization knows from its patient community that often the incremental outcome measurements are far different to a patient than they may be to a researcher, and it is important that the foundation bring this message out.

Limiting Risk in Partnerships

One major role in a partnership for a voluntary health organization is to remove risk from relationships. Venture philanthropists step into the translational research gap, funding research that makes future industry investment more attractive and, in fact, possible. The voluntary health organization’s role in connecting the dots between academia and the clinic can go beyond simply awarding grants. Margaret Anderson, chief operating officer of FasterCures, listed just a few of the ways voluntary health organizations “de-risk” translational research (Box 4-1). Each of these activities removes a level of risk from the process, making it easier for industry to be involved sooner in the process and helping ensure that funding flows into academic research as efficiently as possible. In many instances, it makes sense to simply approach industry and ask what the organization can do to de-risk their involvement in the process.

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BOX 4-1

Limiting Risk in Translational Research Opportunities. Develop preclinical tools that can be used by other partners. Target research to support translation from basic science to clinical trials.


Sharing data successes or failures runs against a common cultural problem, the silo effect. “Some data and resources may as well not exist because you cannot access them,” said Sharon Hesterlee, vice president of translational research at the Muscular Dystrophy Association. Workshop participants discussed the silo phenomenon throughout the day in many guises: animal models behind laboratory doors, valuable data from failed research endeavors, narrowed funding sources, even varied research methodology. Even in things such as end point development, what you quickly find out is that you have academic groups doing it different ways, said Hesterlee. Sometimes this difference makes data incomparable from one trial to the next; thus, aggregate data can be very hard to examine.

Data sharing is critical to advancing toward cures by avoiding repeat efforts between voluntary health organizations focused on the same disease space, said Anderson. A common theme at the workshop was the need for the hypothetical Journal of Failure or Journal of Negative Results, a place where data from failed research could be collected, shared, and discussed. Understanding why something failed can sometimes yield important information that helps researchers move an idea forward or suggest entirely new directions for research.

Data and products from studies should be centralized and shared, advocated Anderson. There are many different tools to do this, and with the advent of new information technologies, data sharing can be substantially improved. But Anderson was also quick to point out that these technologies are not free, nor is the labor required to document and disseminate information. Keeping that efficiency in mind, a few workshop participants shared how they disseminate information.

Most organizations use meetings as a basic way to disseminate information and share data, whether at formal annual meetings or more frequent brown-bag meetings. FasterCures, for example, has a loose affiliation of groups called TRAIN (The Redstone Acceleration and Innovation Network) groups where disparate groups come together throughout the year to share information and innovations. The Prostate Cancer Foundation’s annual meeting involves all of the foundation’s funded scientists meeting with alumni and outsiders. For two-and-a-half days, they discuss the current state of prostate cancer research, brainstorming and sharing ideas, successes, and failures. Other groups are working to create shared databases.

Hesterlee discussed the Muscular Dystrophy Association’s TREAT-NMD patient registry. With a €10 million grant from the European Union, TREAT-NMD put together a massive database of registries from 20 countries in Europe that have agreed to file mandatory data items. The project brings together different stakeholders including patients, scientists, health care professionals, the private sector, and patient groups from many countries to collaborate and discuss what information is most important in the battle against muscular dystrophy. This international registry is going to be hugely valuable to industry and to academic research, claimed Hesterlee. The Muscular Dystrophy Association recognized that this was a valuable system for data sharing and networking and made the strategic decision that any U.S. registry they support needs to have the same core data elements that can be contributed to the European registry. The key to successful data sharing, according to Hesterlee, is to make sure that the voluntary health organization has defined its goals, that all the partners are aligned, and that a good collaborative structure is in place.

Ethics in Data Sharing

The construction of biobanks and patient registries raises not only policy and process issues, but ethical questions. Susan Wolfe, the McKnight Presidential Professor of Law, Medicine, & Public Policy at the University of Minnesota School of Law, agreed that there is a major opportunity to affect the process and the results. Wolfe, a bioethicist, presented the workshop with some issues already in play at many foundations.

One unresolved issue in data sharing is that of consent. What kind of consent is legally and ethically needed for patient registries and bio-banks? For example, if a young child’s data are included in a registry, must new consent be sought to keep the child’s information in the registry when he or she turns 18? What about patients who were unable to make their own medical decisions when their data were collected and deposited? Do they need to reconsent when they regain that ability?

Once data are in a registry, confidentiality is one of the primary concerns. Wolfe mentioned a study that showed confidentiality to be a bigger problem than first imagined. Even batched genomic data can be reidentified, explained Wolfe, as the study tested the assumption that it is not possible to identify individuals using pooled data (Homer et al., 2008). The findings of the study caused a scramble to improve the registry system from the smallest contributor all the way to the NIH, she said. These kinds of privacy issues feed back into how the Colten Foundation and other organizations manage consent.

Ethically, voluntary health organizations must consider whether data sharing should always be a two-way street. For example, Wolfe posited, what should data banks and biobanks do about return of individual research results and individual findings? She noted that studies asking about this very issue point out that the primary concern patients have is the return of individual research results and individual findings, yet there was no agreement or established practice for doing this. Some voluntary health organizations struggle with what it means to do right by the people who are generously participating.

At the time of the workshop, none of the participants had implemented a data-sharing program that returns data to patients. Lucie Bruijn of the Amyotrophic Lateral Sclerosis Association explained that communication was the key for that organization. When samples are collected for a genome-wide scan, said Bruijn, the consent form indicates that data will not be returned. Because of this clear communication, and the understanding in the patient community about the research goals, there has been little concern that data sharing has been inequitable.

The issue of genetic markers is itself an issue of ethics, not just in the research field, and one that the Colten Foundation may need to contend with as its research progresses from basic science toward therapy. Maria Carrillo, director of medical and scientific relations for the Alzheimer’s Association, discussed how this issue has been evolving in the Alzheimer’s disease community. Recent research has suggested that a specific gene is linked to a greater susceptibility of developing Alzheimer’s disease after the age of 55. Consequently, patient studies now routinely collect and analyze data on the gene. But the gene is never disclosed, and there is a reason for that she said: it is an imperfect indicator. The risk of overdisclosure of imperfect science is, in and of itself, an ethical dilemma, said Carrillo.

Maintaining awareness of important ethical issues will be a key factor. Wolfe advocates pulling in bioethicists as a start to help lay out the issues.


Although the issues surrounding ethics often converge on agreed principles such as protect privacy, advance the science, and do no harm, different organizations often have diametrically opposed opinions about intellectual property (IP). Anderson laid out some models that various organizations adopt when approaching IP issues. For some organizations, IP rights are explicitly off limits. These types of voluntary health organizations do not attempt to own IP, because they feel that to do so does not support their stated mission of serving a patient population. At the other end of the spectrum is the voluntary health organization that focuses tremendous energy on acquiring and defending IP because its business model hinges on industry partnership for the development of treatments and cures. Most organizations live in between these extremes, including organizations that do not retain ownership of IP for funded research but that retain some rights if IP owners do not commercialize the property.

How an institution values IP will affect any partnerships it makes. As Heinecke said, a company could say, “As much as I’d like your money and your support, I don’t want my IP tied up.” A voluntary health organization should look closely at the opportunity and determine what sort of deal can be worked out, he said. Celia Dominguez, vice president of chemistry at CHDI Management, Inc., said CHDI does not maintain IP rights in order to have as much of a collaborative and noncompetitive environment as possible. The organization’s philosophy is that to enable downstream development by biotech and pharmaceutical partners, it needs to be able to give them something that actually is of value so they can go ahead and do phase 3 trials and registration if need be.

Case Study: University of California, Berkeley

The Intellectual Property Management Office at the University of California, Berkeley, provides a good model for innovative IP and technology transfer. “Over and over, people are saying the goal should be to expedite translational research, to shorten translational research gaps, to traverse the ‘valley of death,’” said Carol Mimura, assistant vice chancellor of Intellectual Property & Industry Research Alliances at the University of California, Berkeley. In 2004, the university set about doing just that, restructuring to align itself explicitly with the goal of accelerating translational research. Viewing itself as an innovation accelerator, the university worked with academia, industry, government, and voluntary health organizations in order to catalyze innovation. “Unless we make use of our network, we can be stranded in many ways, academically and with respect to funding, whereas if we’re open-minded to collaboration, we can leverage what we have and what the other entities have many times over,” Mimura said.

The university came to view technology transfer not just as a process for obtaining IP rights and licensing them to the commercial sector, but also as a system where IP inflow and outflow are based on individual relationships in a process that can span decades. Mimura feels that the university’s goal is social impact, with revenue generation from licensing being secondary. Success, in her opinion, is having the best outcome of research and public–private partnerships, and product development partnerships are the way forward.

Mimura also highlighted the Socially Responsible Licensing Program at the university, where the university is partnering with industry to maximize the humanitarian impact of its research in the developing world. The university is proactively giving away IP rights to companies to commercialize products, explained Mimura; companies are then required to give away or sell those products at cost. The program has had surprising success. The Bill and Melinda Gates Foundation funded $42 million for a low-cost malaria drug, whose price in the developing world will be reduced 10-fold under the agreement. Because the university was able to structure the arrangement from basic science all the way through commercialization, the entire deal was signed in a single day, with a single three-way collaboration agreement and two IP licenses. In a way, that agreement and acceleration is the poster child for accelerated translational research because it dramatically decreased the time it takes to get from bench to bedside and, subsequently, the drug is expected to be on the market 6 years after signing.

Brooks summarized the message to the Colten Foundation this way:

We are all part of the same spectrum. If NIH funding doesn’t continue … then the pipeline doesn’t continue strong. If the FDA doesn’t have the expertise … we’re not going to get products through. If industry does not remain incentivized, it’s only going to slow things down, because industry can go make money someplace else. The people that lose are the patients.

The major points discussed during session four are summarized below (Box 4-2).

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BOX 4-2

Key Points: Partnerships, Data Sharing, and Intellectual Property. Coordinated teamwork, whether formal or informal, and organization are critical. Philanthropic organizations and the NIH are not competing entities; rather, they can be tremendously complementary. (more...)

Copyright © 2009, National Academy of Sciences.
Bookshelf ID: NBK45139


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