One of the high-profile provisions of the 2012 Food and Drug Administration Safety and Innovation Act was the Patient-Focused Drug Development (PFDD) initiative. It required FDA to hold 20 meetings with the patient community over five years to hear directly from those with lived experience about the symptoms that most impact their lives and the variety of measures they take to manage their condition.
This series of meetings, which numbered 24 by the end of FY2017, has now been expanded to include externally-led meetings, for a total of 34 sessions to date. There are more externally-led meetings on the calendar, plus a robust pipeline of requests before the agency.
Our panelists will share their experiences in working with patient communities to plan for these often-intense listening sessions, as well as what they’ve learned from them and how they are applying this information. Please join us for an update and a forecast for what’s next in patient-focused drug development.
• Meghana Chalasani, Analyst, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
• Annie Kennedy, Senior Vice President for Legislation & Public Policy, Parent Project Muscular Dystrophy
• Colleen Rye, Director, Research and Regulatory Policy, FasterCures, a center of the Milken Institute
• Cynthia Grossman, Director, Science of Patient Input, FasterCures, a center of the Milken Institute (moderator)
When it comes to medical product discovery, development, and delivery, patients need to be front and center – but how close (or far away) are we from truly having a system that routinely measures what matters to patients? A panel of industry, regulatory, and patient leaders will discuss what’s next for patient-centered measurement.
Easing the pain of partnerships: How can we make cross-sector collaborations in R&D worth the effort?
Everyone knows that to get a discovery from bench to bedside, no one can go it alone. In today’s biomedical R&D, all stakeholders have to work with other sectors to achieve their goals. Even if they don’t want to. And even if it’s hard (and it’s likely hard).
Many of the behaviors slowing faster progress in the discovery and development of new medical products are things that organizations everywhere have been confronting for decades, if not centuries – lack of effective communication and collaboration, silo-ization of information and expertise, incentives not aligned with the collective behaviors we want to see. What can the biomedical R&D ecosystem learn from the...
Peering into the crystal ball, the trajectory for medical R&D over the next year may seem a bit hazy. But never fear – FasterCures has analyzed trends and determined some of the issues critical to the future of medical innovation that we’ll be tracking over the coming year and that we think you should as well.
Biomedical innovation is vital to America’s health and economic well-being. President-elect Donald Trump has the opportunity to lead in this area and construct policies to maintain the system’s strength and productivity. FasterCures engaged our unique network of stakeholders across the R&D ecosystem to interview more than 150 thought leaders across eight sectors.
The Cancer Moonshot Summit, held June 29 in Washington, brought together hundreds of leaders across the health, academic, private industry, philanthropic and patient advocacy sectors under the national charge of doubling the rate of progress toward ending cancer as we know it. Join FasterCures for a free Webinar, Tuesday, July 19, from 11 a.m.
We’ve managed to get patients more integrated into many areas of medical research, but a final frontier is putting patients into the discussion on value. FasterCures has long been engaged in work that strives to bring the patient perspective to the medical R&D process and, in partnership with Avalere, is committed to developing the first value framework that truly includes patient perspectives...
Since day one, FasterCures’ work has been aimed at promoting effective models of collaboration to advance and accelerate biomedical research. Getting new medical products from discovery to patients requires all sectors—academia, industry, government, clinical care, nonprofits and philanthropy— to work together throughout the research and development process.
The relationship between nonprofit patient foundations and academic institutions has evolved in significant ways in recent years. While this evolution has the potential to create more productive partnerships, it has also created new challenges. Shifting priorities and a move toward more outcomes-driven philanthropy have prompted a reexamination of the traditional partnership model.
The role of patients has evolved dramatically, from recipient of care to active partner in defining and prioritizing research strategy. This heightened focus on patient-centricity has brought increased demand from scientists and sponsors for “research-ready” communities to accelerate patient-centered studies. While patient registries are often cited as the solution to overcoming research barriers, the platforms, objectives, and data collected through...
Since the Patient-Focused Drug Development (PFDD) initiative was launched in 2012, the Food and Drug Administration (FDA) has held or scheduled 14 disease-specific meetings to better understand patients’ experience with symptoms, impacts on daily living, and available therapies. With more meetings on the docket for 2015, and growing interest among patient groups in applying the PFDD model to developing their...
The Cystic Fibrosis Foundation’s recent return on investment in Kalydeco has placed a magnifying glass on the venture philanthropy model and its business-minded approach to funding for-profit biotech and pharma companies. Driven by the urgency to find treatments and cures for their own diseases, these patient-powered foundations are often the players with the greatest incentive to make strategic, early-stage investments...
The legislative and regulatory calendars for 2015 are already filling up with opportunities to impact the biomedical research and development landscape in the United States – from 21st Century Cures to PDUFA VI negotiations to the impact of the omnibus on appropriations. In this Webinar, experts in federal budgetary, legislative, and regulatory matters talked about what to expect from Washington,...
How does a society, inundated with a great number of incurable diseases, ensure that those who would dedicate their time to finding cures have the resources necessary to do so? Declines in investment for innovative medical research and early-stage biotech companies have spurred patients and their supporters to be more engaged than ever in raising both funds and awareness for...
Would you take a drug that could kill you? Understanding benefit-risk assessment in pharmaceutical products
“Would you take a drug that could kill you? We ask questions like this all the time,” noted Bennett Levitan of Janssen Research & Development during a July 29 FasterCures Webinar about understanding the benefit-risk assessment in new pharmaceutical products. “Do I let the wind blow through my hair or do I wear a helmet for safety? Do I text...
We all know that that our clinical trials system is not working as it is. A strong body of evidence suggests that the one test, one drug model is inefficient and expensive; and that a very low percentage of patients are even being made aware of, let alone participating in, clinical trials.
Getting new medical products from discovery to patients requires all sectors—academia, industry, government, clinical care, nonprofits, and philanthropy— to work together throughout the research and development process. But collaboration is a complex endeavor, and integrating the right partners is far from easy.The concept of collaboration is not new to biomedical research and it comes in all forms. Researchers are continuously...
The healthcare system in the United States is ever-evolving and complex. There are tremendous pressures now to reduce the nearly $3 trillion we spend on healthcare each year without compromising the quality of care and outcomes for patients. The notion of value (that is, receiving high quality care at lower costs) is fundamental to reforming the healthcare system away from...
“The way biomedical research is carried out is changing fundamentally,” Sage Bionetworks President Stephen Friend declared at the beginning of a webinar about the crowdsourced computational challenges Sage is facilitating in partnership with the DREAM (Dialogue for Reverse Engineering Assessment and Methods) project that originated at IBM.
Evaluating the balance between the benefits and risks of new products under review is among the most important elements regulators juggle across the lifecycle of a product. And the question of how to bring more structure, rigor, consistency, and predictability to benefit-risk evaluation is receiving intense focus at the moment.
"This platform could change the way research is done in the future … We all need to work together to make this a success,” concluded COPD Foundation Founder and President John Walsh near the end of FasterCures’ Jan. 29 Webinar about the new $100 million PCORnet (National Patient-Centered Clinical Research Network) initiative of the Patient-Centered Outcomes Research Institute (PCORI).
FasterCures has long believed that philanthropy can be a significant force for progress in medical research, when applied strategically. In an extremely informative Webinar on June 19, participants heard about a terrific example of the strategic application of philanthropy at the Leukemia & Lymphoma Society (LLS) in its Therapy Acceleration Program (TAP).
Engaging patients in the research process is more complex and exciting now than ever before. Patient-driven organizations are moving beyond simply creating patient registries or serving as a conduit to participants for clinical trials and are marshaling their forces in new and more comprehensive ways. In a FasterCures Webinar on March 20, participants heard about two exciting new models of...
We've long heard how intellectual property (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
Science Exchange and The Reproducibility Initiative: Improving the efficiency and impact of academic research
Hear about the Science Exchange's Reproducibility Initiative, which aims to reward high-quality, reproducible research and provide a mechanism for industry to identify robust new drug targets for the development of effective new therapies.
Mary Dwight of the Cystic Fibrosis Foundation (CFF) and Cynthia Rice of JDRF shared their views about why it’s important for groups like theirs to engage with regulators, as well as how to get started and how to be effective, during a recent FasterCures Webinar, “Engaging with FDA: A Guide for Foundation Funders of Research.”The panelists recommended that foundations ask...
The Learning Collaborative, a partnership between the Leukemia & Lymphoma Society (LLS), University of Kansas Cancer Center, and the National Center for Advancing Translational Sciences (NCATS) at NIH, is attempting to break the traditional drug discovery and development paradigm by bringing together blood cancer, drug discovery, and development expertise across several organizations.
Last week, over 1,000 participants from all sectors of medical research participated in a virtual town hall with the interim leadership of the new National Center for Advancing Translational Science (NCATS) at NIH to discuss the status of the effort. NCATS is the first new Center at NIH in a dozen years.Convened by FasterCures as part of its Webinar series, it was a follow-up to a...
Three distinguished leaders discuss opportunities for accelerating research through improvements in biobanking, the use of electronic medical records, and the release of personal health information for research.
This Webinar features attorneys Ken Schaner and David Lubitz and Linda Johnson of JDRF on how to craft the most thoughtful, responsible approaches to intellectual property.