Propelling the medical research system
This was an exciting and productive year at FasterCures, as we deepened the reach of our program work and collaborated with new partners to propel the medical research system forward.
We highlighted many areas in which we work during our sixth annual Partnering for Cures, held Nov. 16 to 18 in New York. The collective energy and urgency of the participants fed a dynamic exchange of ideas and solutions for solving some of the thorniest issues in medical research — from re-imagining clinical trial infrastructure to creating the tools and resources needed to translate basic science into cures.
From ballrooms to board rooms, FasterCures staff have been featured panelists and presenters at meetings convened by the U.S. House Energy & Commerce Committee, Institute of Medicine, Patient-Centered Outcomes Research Institute, Biotechnology Industry Organization (BIO), the Pharmaceutical Research and Manufacturers of America, Association of American Universities, Association of University Technology Managers, DIA, Brookings Institution, and at dozens of smaller gatherings of key opinion leaders from across the biomedical ecosystem.
Throughout the year, we also engaged with key federal agencies, such as the U.S. Food and Drug Administration (FDA) and National Institutes of Health, on collaborative and patient-focused programs.
Five takeaways from 2014:
1. Patients are becoming an even more powerful voice and partner in R&D. From research to regulatory approval to reimbursement, there is tremendous momentum gathering as industry, government, and academia find new ways to ensure patient perspectives are truly heard and used to inform decision-making.
- The FDA’s Patient-Focused Drug Development initiative and Structured Benefit-Risk Assessment programs continue to seek out patients’ perspectives on their condition, the therapies available to treat it, and the tradeoffs they are willing to make on the regulatory front for human drug and biologic products.
Through our benefit-risk assessment program, we are working to expand opportunities for patient perspectives to shape product development and influence regulatory decisions.
In September, leaders came together for the Benefit-Risk Boot Camp, which focused on providing tools to quantify benefit expectations and risk tolerance for disease communities. Several workshops in this year’s TRAIN (The Research Acceleration and Innovation Network) Webinar series highlighted these issues for nonprofit disease foundations and those interacting with venture philanthropies. TRAIN Webinars, which are designed to spotlight innovative approaches to disease research, had more than 1,300 unique participants this year.
- In 2014, House Energy and Commerce Committee Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) launched the 21st Century Cures initiative with the goal of accelerating the discovery, development, and delivery cycle of new cures and treatments. The committee hosted eight hearings and four roundtables in Washington as well as 15 roundtables across the country.
FasterCures has been actively engaged in this initiative, from serving as a resource to submitting comments, from speaking at roundtables to hosting Chairman Upton and Rep. DeGette at Partnering for Cures. The committee listed “expanding patient input in regulatory decision-making” as the top priority for legislation it will introduce early next year. We submitted a proposal to the committee urging creation of a public-private partnership to advance the science of patient input.
2. The rapid emergence of consortia in biomedical research could change how research gets done. These consortia are centered on collaborative approaches that leverage the expertise and resources of a wide range of partners to create tools and knowledge that advance the research objectives of all stakeholders.
- Nearly 400 research collaborations operate as consortia. In an effort to quantify this trend, FasterCures pioneered the Consortia-pedia project, which defines the parameters of consortia, dissects their characteristics, and categorizes existing efforts.
Our goal is to ensure that research-by-consortium efforts are at their highest performance and achieving the best possible outcomes. Science Translational Medicine highlighted this program by publishing “Consortium Sandbox: Building and Sharing Resources" in its June 25 issue, which garnered more than 15,000 downloads in its first five months.
3. Universities and foundations want to accelerate research and translation together. Members of these sectors are working to create a common language around which to build more productive collaboration and move promising discoveries from lab to patient.
- While both patient foundations and research institutions share the goal of translating research into effective therapies for patients, differing views about how best to achieve these goals have negatively impacted these partnerships.
FasterCures convened a TRAINworkshop, where more than 60 stakeholders representing academic research institutions, nonprofit disease foundations, industry, investors, and the legal community convened in Boston to address university-foundation partnerships. In our report, University-Foundation Relations: From Transactional to Transformative Partnerships, we identify five work streams designed to implement some of the action items proposed at the workshop, in an effort to improve these partnerships.
4. All parties need to work together to align the biomedical discovery and reimbursement systems. In order to sustain both the innovation pipeline and insurance payer budgets, we need transparent ways to evaluate the value of new products.
- "We have made significant [drug development] advances," said Cartier Esham of BIO at Partnering for Cures. “The question is what we do now to make sure we allow that progression to continue but sustain a system that allows for access.” The FasterCures Value and Coverage program was designed to foster a sustainable, patient-centric reimbursement system that incentivizes innovation. This year, we published educational issue briefs that provide fundamental overviews of six important aspects of the current system – from provider networks to alternative payment models.
5. Philanthropists want to know that they are improving medical research through informed, measurable giving.
- In an effort to educate stakeholders through our Philanthropy Advisory Service (PAS) program this year, we published two oncology disease landscape reports – on glioblastoma multiforme (GBM) and colorectal cancer – that highlight promising research efforts and identify critical funding gaps that need to be filled to advance new treatments. These reports are developed with input from expert-led Scientific Advisory Boards who are on the front lines of developing new therapies.
Our year was productive, but we have a long way to go to ensure we bring maximum efficiency and speed to the business of finding new treatments and cures. We know patients are waiting, and that is what fuels us to do our work.
We so look forward to working with you and our partners to foster even more collaboration and innovation in 2015. Please let us know your thoughts and ideas. And, here’s to a happy and healthy new year.
FasterCures in the news: Top stories of 2014
- Jan. 13 — Xconomy: Rock Health’s Top 50 Digital Health Entrepreneurs
Margaret Anderson was one of the 50 people chosen who “led the pack in making the largest impact on digital health.”
- May 30 — BioWorld Today: All together: Consortia boom as scientific complexity grows
"Science has gotten more complex, so it’s hard for people developing medical products to answer all the questions they have," said Mark Lim, associate director of medical innovation.
- Nov. 20 — The Diane Rehm Show: The Cystic Fibrosis Foundation: New Opportunities, New Questions For Nonprofits
"What we have seen in the last decade in terms of how patients and disease foundations, such as the Cystic Fibrosis Foundation, have pioneered success, it is transformational and the entire biomedical ecosystem is watching," said Margaret Anderson.
Most-read tumblr posts of 2014In 2014, FasterCures posted more than 100 blogs to our tumblr feed, covering news on our programs as well as commentary on the medical research system, including: