RWD/RWE for Rare Disease Drugs Webinar 

September 22, 2021
Webinar Video: https://youtu.be/gpxi1LHzLf0
Webinar Slides: https://bit.ly/2XKyoGz

Panelists
Tara Cowling, Medlior
Laurie Lambert, CADTH
Craig Campbell, London Health Sciences
Sandra Anderson, Innomar Strategies
Brad Alyward, Canadian Organization for Rare Disorders
Durhane Wong-Rieger, Canadian Organization for Rare Disorders

Moderator
Bill Dempster, 3Sixty Public Affairs

Challenge: Rare disease drugs and other advanced therapeutic products (ATPs) pose challenges for regulatory and reimbursement decision makers, in part, because the evidence of potential benefits and risks available at the time of submission may not come from traditional clinical trials. Trials may include small numbers of patients (dozens rather than hundreds or thousands), carried out over a short period of time (months rather than years) with a homogenous set of patients (infants, early stage disease, severely affected, no previous treatment), non-random assignment to treatment, quasi or no control /placebo group comparison, and predominantly surrogate outcome measures (biomarkers, delayed or less severe symptoms, survival), any of which can create a degree of “uncertainty” as to how the therapy may work with real patients in the real world. may be clinical trials and evidence. 

Opportunity: Healthcare decision makers are increasingly looking to Real World Evidence (RWE) as important source of information for reviewing rare disease drugs. In 2016, Health Canada and CADTH announced the development of an action plan for “systematic use and integration of RWE for both regulatory and reimbursement decision-making.” In 2019, the federal government committed to creating a national Rare Disease Drug Strategy to improve patient access to new therapies and in 2021, Health Canada reported back strong stakeholder consensus on use of managed access programs to allow timely access to therapies with on-going monitoring and collection of “real-world” data.  

RWD are “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.” RWE is the “clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD.”

In October 2021, CIHR, CADTH, and CORD will begin a collaborative process toward defining and using RWE to optimize patient access to rare disease drugs. To promote informed patient participation in the process, CORD will host webinars and workshops on RWD and RWE for patients and public. An introductory Real-World Data and Real-World Evidence Webinar will be offered on Wednesday, September 22, 2021 at 12-1:30 pm. A number of patients attending the Webinar will be invited to participate in the initial CIHR/CADTH/CORD workshop in early October.

Registration: https://bit.ly/3EgC7wA