Interest is intensifying across the globe for utilizing real-world evidence (RWE) to inform decision-making by reimbursement organizations. For instance, the Canadian Agency for Drugs and Technologies in Health (CADTH) have noted:
- All evidence is considered for health technology assessment (HTA) submissions
- Data derived outside the confines of a randomized controlled trial (RCT) can be particularly useful when RCTs are considered impractical or unethical, and when limitations of RCTs present uncertainties, such as long-term efficacy and safety, or in external validity (generalizability) to real-world patient populations.
CADTH recently published an environmental scan
on the use of RWE among international HTA and regulatory organizations. Unfortunately, the report did not provide any insights into CADTH’s consideration of RWE within HTA submissions beyond a review by Griffiths et al. (2017) that described the use of non-RCT evidence, in the form of single-arm trials.
Medlior recently reviewed the use of RWE reported in HTA decisions from CADTH’s Common Drug Review (CDR) and pan-Canadian Oncology Drug Review (pCODR) programs. We examined submissions published between January 1, 2017 and May 22, 2018 to identify appraisals where RWE was used or could have been used to address evidentiary uncertainty. The use of RWE ranged from informing costs in economic models to creating historical cohorts for indirect treatment comparisons.
The frequency of RWE utilized by CADTH HTA submissions is summarized below:
- CDR: 43 completed appraisals
- 18 contained RWE – 2 approved, 16 conditionally approved
- pCODR: 33 completed appraisals
- 8 contained RWE – 1 approved, 7 conditionally approved
While RWE has rarely been included in CADTH HTA submissions, RWE can inform a number of uncertainties that arise in HTA submissions across the globe, including:
- Magnitude of clinical benefit: It can mitigate the limitations of RCTs, such as small sample sizes, trial designs employed, and analytical methodologies.
- Lack of comparative data: It can allow for comparisons to alternative therapies in cases where clinical trials are single-arm and comparisons are not available.
- Cost-effectiveness: It provides the opportunity to examine broader patient populations than those represented in clinical trials with respect to healthcare resource utilization and/or clinical effectiveness.
- Lack of long-term follow-up: Long-term efficacy and safety can be examined using RWE to support the limited duration of RCTs and relieve concern for longitudinal results not being available.
- Generalizability of results: Results from RCTs conducted in other countries, or RCTs that enrolled strictly-defined patient groups, can be generalized to broader Canadian or other jurisdictional populations.
- Duration/dosage of treatment: It can help clinical trial findings be generalized to real-world treatment patterns and additional treatments can be compared to those utilized in RCTs.
- Sequencing of available treatments: Incorporating RWE would allow assessment of multiple lines of treatment in cancer therapy.
HTA agencies such as CADTH are recognizing the need for RWE in funding decisions. As the use of RWE becomes more routine in HTA submissions, a collaborative approach between industry, government, payors, providers and patients is necessary to ensure that RWE is used appropriately. To that end, a multi-year initiative has been funded by the Canadian Institutes of Health Research (CIHR) to develop a framework for incorporating RWE into drug-funding decisions in oncology. Establishing standards and performing RWE studies with appropriate and rigorous methodology, statistical analysis and reporting requirements can help support the future use of RWE in HTA submissions.
To help support patient understanding of RWE, please see our attached white paper: RWE for Patients
Real World Radar
Medlior recently launched a brand-new website: Real World Radar, an online tool that provides researches with a comprehensive directory of RWE datasets in Canada. Currently over 200 unique datasets are listed with information on jurisdiction, disease area and available data elements for your research needs. Medlior will continue to expand the list of datasets and include national and international datasets throughout the year.
For a sneak peek or to subscribe to Real World Radar, please visit https://www.medlior.com/real-world-radar/
NICE Conference (June 26, Manchester)
At the NICE Conference, Medlior will be hosting a luncheon session titled: Canadian Real World Evidence & Research Opportunities. Medlior will demonstrate how we can support a variety of ethics-approved, patient-centric research projects utilizing health system data from Canada. The luncheon will be free to conference attendees and an email will be sent prior to the meeting to register. The confirmed agenda, speakers and exhibitors, can be found on the NICE Annual Conference website (http://www.niceconference.org.uk/)
If you would like to set-up a meeting to discuss how Medlior can support your research needs, please feel free to reach out to Tara Cowling at email@example.com
The Medlior Team