I hope you received my last email regarding PMWC 2022 Silicon Valley’s June 28-30 dates (Program).
Real-world data (RWD) and real-world evidence (RWE) have been receiving increasing amounts of attention from both medical researchers and regulators, playing an increasing role in health care decision-making. Real-world data, as defined by the FDA, relates to patient health status and the delivery of healthcare and includes electronic health records (EHRs) data, claims and billing data, product and disease registries, patient-generated data, and data gathered from other sources, such as our mobile devices.
While to date, RWE has been primarily used for postmarketing drug safety surveillance, there is a growing interest in the usage of RWE by regulatory agencies to evaluate safety and efficacy of medical treatments. This is further substantiated by the FDA increasing their focus on RWE for regulatory decision making for both new approvals as well for evaluating additional indications for approved therapies. Specifically, the FDA has taken action in an attempt to reduce the burden in both time and cost of bringing a new therapy to market.
Due to the fact that clinical end points (i.e., outcomes that show direct clinical benefits, such as increased overall survival) may take a long time to develop, many drug trials with breakthrough therapy designation use surrogate end points, which are measurements or signs predictive of clinical outcomes but do not directly indicate clinical benefits. RWE is the approach to study these surrogate and intermediate end points when a therapy addresses a serious condition without existing options. Examples of surrogate end points include blood pressure for hypertension drugs and serum low-density lipoprotein cholesterol for hypercholesterolemia treatments. Shorter trials and the use of surrogate end points present a strong need for postapproval surveillance for both safety and effectiveness, especially in the context of traditional clinical end points.
The advancements and the clinical importance are further exemplified by the recent launch of several initiatives via a UCSF-Stanford partnership to improve the development and approval of effective medical products:
• The UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (CERSI): This joint undertaking with the FDA and in collaboration with the pharma, biotech, and high-tech industries in the San Francisco Bay Area (and on the West Coast in general) provides modern technology tools that will help evaluate medical products for safety, efficacy, quality, and performance. It addresses key relevant points in the area of drug discovery, drug development, and regulation, cypersecurity, and innovations in regulatory science
• The Pediatric Device Consortium to expand access to pediatric devices by using RWE in new and innovative ways.
To support and expedite these important industry efforts PMWC 2022 Silicon Valley has an entire track with several timely sessions dedicated to this hot topic of RWE use:
• The track starts out with a panel chaired by Atul Butte (UCSF) diving into a discussion focusing on “Realizing the Promise of Precision Medicine using Real-World Evidence”
o Contributors: John Concato (FDA), Mark Laabs (Rare Cancer Research Foundation), Rhonda Cooper-DeHoff (University of Florida), and Nancy Dreyer (IQVIA)
• Followed by a panel chaired by Vivek Rudrapatna (UCSF) that tackles the various “Opportunities and Challenges associated with Real-World Data”
o Contributors: Ida Sim (UCSF) and Joseph Wu (Stanford University)
• The third panel chaired by Clara Ljonchere (UCLA) will focus on “How are Patient Data Revolutionizing Precision Medicine?”
o Contributors: Sharon Terry (Genetic Alliance), Farid Vij (Invitae), and Latha Palaniappan (Stanford University)
• The fourth panel, which is chaired by Kathryn Phillips (UCSF), focuses on “Leveraging RWE to Create Value”
o Contributors: Phil Febbo (Illumina), Suzanne Belinson (Tempus), and Stacey DaCosta Byfield (OptumLabs)
• And last, but certainly not least, Keith Yamamoto (UCSF) will chair a panel focusing on “The Past, Present, and Future of RWE”
o Contributor: Matthew Porteus (Stanford University)
• Additional sessions in this track are as follows:
o “Regulatory Requirements and Challenges for Using RWE” chaired by Sheila Walcoff (Goldbug Strategies) and with Kathy Hibbs (23andMe)
o “Government Partnerships: California Initiative to Advance Precision Medicine“, chaired by Julianne McCall (CA Governor’s Office), with William Kim (UCSD) and George Slavich (UCLA)
Don’t miss this opportunity to join a world-class group of medical researchers and regulators debating where we need to go next and how we get there, in order to expedite the applications of RWE and RWD to create safer and more efficacious medical treatments.
Co-founder & President, PMWC
Precision Medicine World Conference – June 28-30, 2022 Silicon Valley