On February 20, FasterCures, a center of the Milken Institute, held a webinar exploring innovative financial and business models that could speed therapies to patients and catalyze future investments in biomedical R&D.
Speakers include:
Tanisha Carino, Executive Director, FasterCures, a center of the Milken Institute
Freda Lewis-Hall, Chief Patient Officer and Executive Vice President, Pfizer
Dan Liljenquist, Senior Vice President and Chief Strategy Officer at Intermountain Healthcare, Chair of Civica Rx
Colleen Rye, Director of the Aligning Incentives Program, FasterCures, a center of the Milken Institute
The U.S. Food and Drug Administration (FDA) and patient organizations use patient-focused drug development (PFDD) meetings as a tool to listen to patients and gather their perspectives. During these meetings, patients give their views on living with their conditions and current and future therapies. In 2013, FDA launched the PFDD initiative with plans to host 20 condition-specific meetings, a goal they have since surpassed. In 2015, after two years of demonstrated success, FDA shared the mantle and gave patient organizations the chance to host PFDD meetings (often called “externally led PFDD meetings”). Since then, patient organizations have successfully amplified the voices of patients through nearly 20 additional meetings.
Perhaps you’ve heard about the value of these meetings, or you’re considering or currently planning one. The success of previous meetings may make it look easy, but don’t be mistaken: PFDD meetings are not cut-and-paste. Each event is unique and requires months of planning, but there have been enough meetings that common practices and lessons learned can be shared, and organizations don’t have to start from scratch. For this reason, FasterCuresis excited to announce that we are unveiling three new resources based on lessons from the field, specifically designed for those who are interested in hosting externally led PFDD meetings: the PFDD Readiness Assessment, the Smart Practices from Patient Community Leaders planning guide, and the PFDD Community Toolbox.
Dec. 13, 2018, marked two years since the signing of the 21st Century Cures Act (P.L. 114-255). In its first two years, the Cures Act has contributed to significant advances in research, changed how stakeholders share health data, and helped modernize the biomedical ecosystem through far-reaching studies such as the All of Us Research Program and the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative. These advances have reduced obstacles to data sharing and encouraged more patient engagement and the use of real-world data in product development and approval. In addition, the Cures Act provided research funding, prioritized increased support for early-career researchers and high-risk, high-reward research, and provided new abilities for federal agencies to more competitively hire the skilled workforce needed in a 21st Century health-care system.
Through FasterCures’ 21st Century Cures tracker, we continue to monitor the implementation of Cures Act provisions and applaud the U.S. Department of Health and Human Services (HHS) for completing requirements that are improving the biomedical innovation system to the benefit of patients. New for 2019, FasterCures will issue a series of blog posts digging deeper into the impact the Cures Act has had on specific issues of interest. Stay tuned throughout 2019!
In Progress: Some requirements of the section have yet to be completed
Complete: All requirements of the section have all been met
Monitoring: FasterCures is monitoring the use of selected (completed) sections that establish new procedures or processes
by Brenda Huneycutt, Director, and Julien Rashid, Associate
One of the ways FasterCures helps bring patient perspectives into product research, development, and regulatory review is by convening groups of stakeholders to voice their perspectives on various topics. We recently brought together a small group of patients and patient representatives to explore their thoughts on the U.S. Food and Drug Administration’s (FDA) Digital Health Software Precertification (Pre-Cert) Pilot Program to help the agency shape the program.
This innovative pilot program shakes up the old paradigm of how FDA regulates certain medical devices. With this new approach, patient perspectives, needs, and opinions should be considered. Below we outline the Pre-Cert pilot program and describe some of what we heard from participants at our convening. For those wanting to offer their opinions on this pilot program to FDA, the agency is accepting public comments through its open docket until March 8, 2019. FasterCures will continue to provide updates as this program develops.
When is philanthropy no longer philanthropy? This and other existential questions—as well as more practical ones—were asked in a small-group dialogue that took place at the Milken Institute’s Future of Health Summit on “Venture Philanthropy 2.0.” More than 40 leaders from public charities and private foundations, investors of various stripes, biomedical product developers, and other interested parties heard about established and emerging models of philanthropic investment to accelerate R&D in the interest of patients. They considered issues such as:
What makes philanthropy unique as a source of capital?
What are the financing gaps where philanthropy can make the greatest difference?
What are the most significant challenges for venture philanthropy right now?
How can these financing approaches be scaled? What’s needed to build capacity and partnerships?
This dialogue will inform FasterCures’ continuing work to provide thought leadership and capacity-building to support the growth of venture philanthropy in biomedical R&D. We share some of the insights gleaned from the discussion in this blog.
On December 10, FasterCures, a center of the Milken Institute, hosted a webinar titled “So You Want to Start a Master Protocol Trial.” Master protocols are an innovative model for conducting clinical trials, and they are gaining traction with regulators, product developers, and patients. These trials have the benefit of being patient-focused, time-efficient, cost-efficient, and highly collaborative. However, this model also poses challenges in the initial stages, requiring thoughtful planning and up-front collaboration and investment. The discussion focused on the advantages of conducting master protocol trials, current trials successfully using this framework, and lessons learned through implementing this trial design.
On Oct. 3, FasterCures, a center of the Milken Institute,partnered with the Kraft Precision Medicine Accelerator at the Harvard Business School to present a webinar entitled “Exploring the Frontiers of Data and Analytics for Precision Medicine.”
The goal of the webinar was not only to introduce attendees to the Accelerator but also to highlight its landscape of organizations working in real-world data and advanced analytics to support precision medicine, including artificial intelligence and machine learning.
With so many changes and innovations occurring in the space in recent years, the webinar provided an opportunity for reflection on where the field of data for precision medicine has been and where it needs to go in order to meet the needs of patients across disease areas.
In today’s health-care environment, you would be hard-pressed to find someone who would disagree that patients should be at the center of everything we do. Despite this widely accepted belief, there is still much work to be done to make this sentiment a reality. When it comes to patient engagement, we are still learning to walk the walk instead of simply talk the talk.
The good news is that there has been an expansion over the past few years in efforts to directly engage patients across the continuum of medical product development and delivery. Now we must build upon this foundation by encouraging more organizations and individuals to add their voices and action to further accelerate patient engagement in medical R&D.
This is why FasterCures is excited to partner with Patient Focused Medicines Development (PFMD), a Brussels-based non-profit coalition focused on lifting up the patient voice across the lifecycle of medicine, to expand the suite of patient engagement initiatives contained in its SYNaPsE platform as well as to share its Pledge to Patients.
FasterCures, a center of the Milken Institute, the People-Centered Research Foundation (PCRF), and the National Evaluation System for Health Technology Coordinating Center (NESTcc) have embarked on a collaboration to better integrate patient perspective and participation, as well as patient-generated health data, into the growing landscape of shared data networks. This collaboration—the first of FasterCures’ new series of Acceleration Labs—will identify and describe models of partnership between patient organizations and research networks that are supporting the creation, use, and diffusion of evidence generated in disease-specific data platforms and in large, disease-agnostic data networks. The effort will also clarify the value of such partnership models in bringing patient perspective, experience, and data into the research process.
Five years ago, FasterCures turned our attention toward the emergence of consortia in medical research. With some collaborations just starting and others having worked together for years, there were several fundamental questions on our mind: How many consortia are there in the medical research space? Who is involved in them? What are they working on? Are these collaborations really spurring medical research to happen more quickly and efficiently?
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