July 22, 2014: How the Research-by-Consortia Model Will Lead to Faster Cures
Leaders from across sectors in medical research and development delivered a strong message to Capitol Hill last week: to accelerate medical progress, we need to encourage and incentivize public-private partnerships that advance shared goals.
Consider that in 1997, there were 6 consortia in medical research and development; today there are 393 active cross-sectors efforts focused on advancing shared research goals. This emerging model was the focus of a FasterCures briefing held July 22, which attracted more than 130 stakeholders, including dozens of legislative staff. A robust panel discussion ensued about the unique value proposition of these partnerships, the complex scientific challenges it can address, and the power of engaging various sectors, including industry, academia, patients, and government.
In sum, four themes rose to the top:
1. Government plays a leading role in coordinating big science.
According to Eric Green, director of the National Human Genome Research Institute at National Institutes of Health, “collaborations give the ability to exercise strategic vision—we help craft the vision, but we don’t do it alone.” According to FasterCures’ research, government agencies were responsible for 44% of the 393 consortia surveyed.
ShaAvhrée Buckman-Garner, who leads partnership efforts at the U.S. Food and Drug Administration, said that the FDA has a “unique perspective to share with other stakeholders to translate common challenges into solutions.” FDA’s leadership and participation in consortia help ensure these efforts are focused on its impact on patients.
“A consortium is not just good to have, it’s absolutely necessary if we are to realize big, bold audacious goals in science,” added Green.
Panelists overwhelmingly agreed that federal agencies need the resources to be able to actively participate in these efforts.
2. The complex challenge of understanding disease requires multiple sectors to come together to share resources and advance common goals.
Marc Bonnefoi, head of Sanofi’s North America R&D Hub, said that the scientific issues are too big for industry to fix on its own. Consortia can help pave the path for industry to work with other sectors in order to provide better solutions for patients. “Consortia allow industry to push, but not break, the line between pre-competitive and competitive research,” he said. “For too many years we worked in siloes. There is now the ability to for multiple companies with the common goal of tackling the complex biology of disease to come together, where the degree of understanding is still lacking.
Angela DeMichele of the University of Pennsylvania and co-director of ISPY-2 highlighted the research done through the I-SPY trial which is “interrogating multiple drugs from different companies simultaneously using the same master protocol, cutting the time and cost of development by two thirds.” DeMichele further explained that this adaptive clinical trial design allowed the FDA, industry and academia to come together to “build the infrastructure to test multiple drugs at the same time, matching patients and their tumors to the right drugs.” The biggest challenge, she says, is to sustain these efforts and keep it running.
3. Collaboration in research benefits patients.
Patients are also steering consortia and are critical to its success. ”Patients are stepping up, enrolling in clinical trials, making their voices heard,” said Richard Insel who manages the scientific priorities and portfolio of JDRF. Insel emphasized that “the current tools to manage diabetes are not enough, we need research by consortia to advance disease research and gather data and insights needed.
In its analysis, FasterCures found that almost 50% of the 393 consortia are focused on scientific challenges specific to a disease or condition.
- 44 consortia focused on cancer – 52% of which were started by a government agency;
- 21 consortia focused on diabetes – 52% of which were started by a government agency (same as cancer), 5% of which were started by a non-profit foundation such as JDRF.
Margaret Anderson, executive director of FasterCures, moderated the panel and underscored the sense of urgency and need for these models to work – “patients are waiting for cures,” she says, “and these partnerships help advance or create efficiencies needed to accelerate the process.”
4. Consortia provide tools for regulatory science to accelerate the medical R&D process.
Tackling the biggest problems in the drug development pathway – from creating new clinical trial designs to incorporating patient reported outcomes – is at the core of regulatory science and often the fundamental drivers of consortia efforts.
Consortia already are working in a number of areas, such as developing new animal models and biomarkers that could be used in clinical trials. In fact, approximately one in five consortia is designed to enhance regulatory science, often with the participation of a regulatory scientist who can learn in a neutral-forum about scientific challenges faced by researchers.
“Collaboration is not easy,” said FDA’s Buckman-Garner. “But the more we can focus on developing the consortia model, the better.” “It’s important for consortia not to take on more than it can accomplish,” she added as she urged for greater coordination amongst consortia.
However, DeMichele cautioned that “leading and running a consortia is not a skill set that most go into research knowing. We have to train and educate.” A theme that resonated with all the panelists and Buckman-Garner added to by noting that FDA is “considering a proposal for regulatory science consortium training.”
Members of Congress, especially the U.S. House of Representatives Energy and Commerce committee focused on the 21st Century Cures initiative, may find it highly valuable to turn to some of these best practices that are streamlining drug development and delivering better solutions to patients. The consortia model is the “wave of the future that allows the science to be lifted up,” Anderson said.
To learn more about Consortia in Medical Research and Development, visit consortiapedia.fastercures.org.
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