Cutting-Edge Science, Collaboration, and Sustained Funding Needed to Get New Medicines from Lab to Patient

FasterCures and Friends of Cancer Research Host Capitol Hill Briefing

“While I can tell you there’s never been a more exciting time for science, I can also tell you there’s never been a more stressful time,” said Francis Collins, director of the National Institutes of Health (NIH), addressing the issue at hand – tightening fiscal resources that threaten the research and development ecosystem to fully deliver science’s full potential to improve health and well-being. This was the main topic of a Capitol Hill briefing co-hosted by FasterCures and Friends of Cancer Research.

Moderated by FasterCures Executive Director Margaret Anderson, Collins and other medical research leaders representing distinct sectors provided a glimpse into what it takes to turn a scientific discovery into a safe and effective therapy that will improve, and maybe even save, patients’ lives. More than 300 policy decision makers, advocates, and key legislative staffers participated.

“There is no one in this room who hasn’t been touched by disease, whether it’s yourself or a loved one,” said Ellen Sigal, chair and founder of Friends of Cancer Research, opening the discussion. “We cannot let the FDA or NIH become a victim of political polarization.” The discussion kicked off with video highlights from a viral campaign, Time=Lives, that featured images and stories of patients, families, researchers, and executives, reminding everyone in the room about what’s at stake.

Onyx CEO Anthony Coles explains impact
of public investment on drug development 

Underscoring the role of public investment in basic research by the NIH, N. Anthony Coles, president and CEO of Onyx Pharmaceuticals, noted that pharmaceutical companies depend solely on this research to understand biology and disease origins. “Companies like Onyx then take these findings, advance the research, and move it toward delivery to patients,” he said.

Improving and accelerating research and development requires all stakeholders in the medical research ecosystem to work together – including federal agencies, industry, academia, and patient groups. Collaboration – existing collaborative efforts, and the need for even more partnerships – was a resounding theme throughout the discussion.

Disease research organizations feel the excitement for an improved system. “This is the most robust pipeline we’ve seen. Now is not the time to step back from working together, but to do more,” said Deborah Brooks, co-founder of the Michael J. Fox Foundation for Parkinson’s Research.

“The FDA is the final common pathway to translating science to patients,” said Margaret Hamburg, commissioner of the U.S. Food and Drug Administration (FDA). “If the FDA is not fully funded and supported, the ecosystem will not function optimally. When we engage early with the scientific community, we’re able to cut five years off of the drug development process.”

Roy Jensen, director of the University of Kansas Cancer Center, is already seeing how limited resources are impacting research progress. “We’re not even funding a fraction of the best science. We’re starting to cut to the bone – scientists are having to close labs, they aren’t able to train the next generation… It’s fundamentally altering our infrastructure,” said Jensen.

The importance of supporting the next generation of brilliance and innovation weighs heavily on the minds at the NIH, and researchers are hopeful for the next group of young investigators. “It is very tough right now to be a grad student, or a post-doc. As they look at the landscape of this country, they wonder if there’s room for them,” Collins said.

Coles echoed Collins’ concern with a sobering concept about the future of scientific innovation: “What answers won’t we have in 10 years if we don’t fund this research?”

“The decisions we make today will have implications long into the future. Unlike delaying construction of a bridge that can be resumed in a few years, if we lose a generation of scientists, there’s no way to rebuild that human capital quickly,” said Mike Milken, founder of FasterCures and chairman of the Milken Institute. “We fully understand the need for overall budget restraint in Washington; but short-term cuts in bioscience only assure a less-healthy America down the road. Modest investments in prevention and cures today will help avoid the catastrophic costs of care tomorrow.”

* A video of this briefing is now available.

From L to R: Margaret Anderson, Roy Jensen, Deborah Brooks, Tony Coles, Ellen Sigal, Margaret Hamburg, Mike Milken, Francis Collins

Releasing Unlocking IP: Principles for Responsible Negotiation

By Elizabeth West, Program Manager, FasterCures

Readers of the FasterCures blog are well aware of the importance of reducing cost, time, and inefficiency in the biomedical research system. One area consistently raised to us as having unnecessarily high transaction costs - both human and financial - is intellectual property (IP) negotiation.

We've long heard how IP can be a roadblock to innovation, but since it's a necessary and critical "cost" of doing business in drug development, we began to wonder if it might be possible to create an agreed-upon culture of responsibility in the practice of negotiating IP for medical research collaborations.

Last week we released Unlocking IP: Principles for Responsible Negotiation to serve as a set of guiding principles and points to consider when engaging in intellectual property negotiations involving biomedical R&D. The principles are a useful tool for all parties in biomedical research, in particular disease foundations, nonprofit disease groups, and philanthropists negotiating IP with academic, industrial, and nonprofit partners. The document includes principles for before, during, and after negotiations, for both new and seasoned organizations engaging in IP negotiations.

Some of the highlights include:

• Do not let fear paralyze you…If the desired result [of a deal] is based on research impact as well as monetary returns, not doing the deal or slowing progress is a far bigger loss to the patient and the public than lower potential economic return.
• Make sure you have the right people at the table at the right time…Think through which stakeholders are needed to  foster innovation, and only exclude a stakeholder group—especially nonprofits—after careful thought.
• Build in “use it or lose it” requirements (interruption licenses)….If you have invested in the IP and the owner is not exercising the rights to use the invention and make it widely available, or if they are using it in a way that impedes progress, ensure you have a way to take it to a party where its value can be maximized.

The principles emerged from a small, expert-led working group convened by FasterCures in July 2012. In addition to developing the principles, the group came to some broader conclusions regarding the intellectual property ecosystem. In particular, it found that there is a fundamentally new landscape in biomedical research resulting from the emergence of venture philanthropy and patient advocacy groups. These groups tend to want to enhance the freedom to operate, versus the limiting model more prevalent in the current environment.  A key conclusion of the working group is that an IP management system is needed that addresses this emerging ecosystem, not reflects the current one. We welcome all of your thoughts on the fundamentals of a forward-looking IP system.

The Webinar – directly from the experts

On Nov. 16, we continued the conversation on IP and the principles through the TRAIN (The Research Acceleration and Innovation Network) Webinar series. The Webinar (available to view here), moderated by Margaret Anderson, was led by an expert panel of individuals with in-depth experience in intellectual property negotiation from a variety of viewpoints.

Maria Freire, of the Foundation for the NIH, laid out the main findings of the working group and gave a detailed overview of the principles themselves, as well as spoke of the increased sense of urgency and interest of many groups in deals that “move towards the goal.” Robert Cook-Deegan of Duke University’s Institute for Genome Sciences and Policy provided the audience with the broader landscape of intellectual property negotiation successes and controversies, as well as analyzed the elements of success through an in-depth case study of Cystic Fibrosis Foundation and the CFTR gene patent. David Lubitz, of Schaner&Lubitz, PLLC, which provides council for many disease foundations, gave a thorough overview of the viewpoint of disease foundations and venture philanthropy groups, including funding goals, trends, and useful IP tools, including  interruption licenses. Many of the questions during the Q&A period focused on the replicability of the Cystic Fibrosis Foundation model, which the panelists agreed would require a foundation with financial resources, technical expertise, and potential commercial partners.

More on IP at Partnering for Cures

We will have more on this topic at our annual conference, Partnering for Cures, in New York City, Nov. 28-30. The IP-focused panel, License to drive (innovation): IP strategies to support, not slow, progress, will explore the evolving IP law and procedures, the IP implications of the movement toward more precompetitive collaboration in biology, examples of responsible IP management practices, and how IP decisions can impact follow-on innovation and patient access. We invite you to join us there!

 

Outside In: NIH Opens Clinical Center to Extramural Researchers

Researchers from academia, nonprofits, and other institutions not within the National Institutes of Health can now access the world-class facilities at the NIH Clinical Center.

Scientists interested in pursuing access to specific Clinical Center resources – including an impressive metabolic unit, pharmaceutical development capabilities, and advanced research-related radiology imaging services – can now explore opportunities and pathways for collaboration with the Center.

Until now, the Clinical Center —NIH’s crown jewel – has been exclusively available to the NIH Intramural Research Program (IRP).  But with a new grant mechanism, a website that provides information on opportunities and pathways for collaboration with the Center, and a toolkit for researchers interested in pursuing access to specific hospital resources, the doors (and medical possibilities) are opening up.

In 2008, FasterCures convened a task force chaired by Nobel Laureate Dr. David Baltimore, which called for the IRP to adopt a new mission that was outcomes-focused, capable of responding quickly to new opportunities, and fully utilized its world-class research hospital and other infrastructure (which at the time rarely reached 60% occupancy). Among the task force’s key recommendations  was that the NIH create streamlined mechanisms by which extramural researchers and industry could more fully use the Clinical Center for projects in collaboration with the IRP.

A few years later, in 2010, FasterCures and 86 other patient organizations sent a letter urging the NIH’s Scientific Management Review Board to open up the Clinical Center facilities to external researchers.

With some of the most advanced medical technologies in the world, the Clinical Center has a distinguished history of discovery. We are delighted to see NIH continue with this important initiative and we encourage researchers to take them up on their offer and make use of the Center’s incredible resources. After all – no one can do this alone.