Tarry Ahuja's research while affiliated with Canadian Agency For Drugs And Technologies In Health and other places

The Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration developed an MCDA rating tool to assess and prioritize potential post-market real-world evidence (RWE) questions/uncertainties emerging from public drug funding decisions in Canada. In collaboration with a group of multidisciplinary stakeholders from across Canada, the rating tool was developed following a three-step process: (1) selection of criteria to assess the importance and feasibility of an RWE question; (2) development of rating scales, application of weights and calculating aggregate scores; and (3) validation testing. An initial MCDA rating tool was developed, composed of seven criteria, divided into two groups. Group A criteria assess the importance of an RWE question by examining the (1) drug’s perceived clinical benefit, (2) magnitude of uncertainty identified, and (3) relevance of the uncertainty to decision-makers. Group B criteria assess the feasibility of conducting an RWE analysis including the (1) feasibility of identifying a comparator, (2) ability to identify cases, (3) availability of comprehensive data, and (4) availability of necessary expertise and methodology. Future directions include partnering with the Canadian Agency for Drugs and Technology in Health’s Provincial Advisory Group for further tool refinement and to gain insight into incorporating the tool into drug funding deliberations.

e20006 Background: As new agents are approved for multiple myeloma (MM) and existing drugs become generic, treatments that represent optimal use of healthcare funds need to be reassessed. A therapeutic review was initiated to compare the clinical and cost-effectiveness of treatments for newly diagnosed (NDMM) patients who are ineligible for stem cell transplantation and those who are relapsed and/or are refractory (RRMM). This abstract reports on the clinical efficacy of treatments for both populations along with patient experiences, expectations, and perspectives of treatment for MM. Methods: A systematic literature search of randomized controlled phase III trials (RCTs), published between January 1996 and April 2021, that met the eligibility criteria in either population were included. Two network meta-analyses (NMA), one per population, were conducted to compare the efficacy across treatments for the primary endpoint of progression-free survival (PFS). PFS was defined as the time from randomization to either disease progression or death. A random-effects model was used in both populations to estimate the hazard ratio (HR) and 95% credible intervals (CrIs) for PFS. Myeloma Canada conducted seven surveys between January 2016 and May 2021 that addressed expectations when selecting a treatment option in MM. A literature search of qualitative studies published between January 2016 and May 2021 was conducted on patients’ perspectives and experiences on treatment decisions in MM. Results: There were 29 RCTs in each NMA with an overall low risk of bias. The NDMM NMA identified 11 regimens that had numerically lower hazards for PFS compared to lenalidomide + dexamethasone (Rd), with HRs ranging from 0.38 to 0.99. The RRMM network showed 15 regimens that had numerically lower hazards for PFS compared to Rd, with HRs ranging from 0.44 to 0.99. Due to the wide 95% CrIs, conclusions on differences in PFS for each population are limited. Myeloma Canada collated data from 2,297 survey respondents. Ten qualitative studies of low to moderate quality were included. The survey data and qualitative studies found that choosing a treatment must involve a holistic approach beyond the efficacy of a therapy. Conclusions: The uncertainty in both NMAs limit firm conclusions on differences in PFS across treatment options in each population. Among the survey respondents, socioeconomic status was unclear which may influence patients’ expectations and preferences for treatment decisions in MM. Insufficient reporting in the qualitative studies makes it unclear how a patient’s treatment decisions changes over time. For Canadian payers to optimize the funding of treatment options available in MM, the results from the NMAs combined with Canadian real-world evidence will inform the development of an economic model to assess the cost-effectiveness of treatment sequences in NDMM.

Policy makers face challenges with the number of drugs for rare indications and rapidly rising costs. In facing these challenges, decision-makers see real-world evidence (RWE) as an opportunity. Health Canada and the Canadian Agency for Drugs and Technologies in Health (CADTH) recently announced their intent to co-develop an action plan to optimize the process for the systematic use and integration of RWE into both regulatory and reimbursement decision-making in Canada. When implemented, this will have a significant impact on how drugs are approved and paid for in Canada. We highlight the key opportunities, barriers and future directions related to the use of RWE throughout the life cycle of drugs in Canada.

Background Canada has a long history of the use of clinical evidence to support healthcare decision making. Given improvements in data holdings and analytic capacity in Canada and stakeholder interest, the purpose of this study is to reflect on perceptions of the value of real-world evidence in pricing and reimbursement decisions, barriers to its optimal use in pricing and reimbursement, current initiatives that may lead to its increased use, and what role the pharmaceutical industry may play in this. Methods/Results To capture stakeholder perceptions, ninety-one participants identified as key stakeholders were identified according to background roles and geography and invited to participate in four round table discussions conducted under Chatham House rule. Important themes emerging from these discussions included: (i) the need to understand what “real world” evidence means; (ii) barriers to using real world evidence from differences in access, governance, inter-operability, system structures, expertise, and quality across Canadian health systems; (iii) differing views on industry's role. Conclusions The use of real-world data in Canada to inform pricing and reimbursement decisions is far from routine but nascent and slowly increasing. Barriers, including interoperability concerns, may also apply to other federated health systems that need to focus on the networking of healthcare administrative data across provincial jurisdictional boundaries. There also appears to be a desire to see better use of pragmatic trials linked to these administrative data sets. Emerging initiatives are under way to use real world evidence more broadly, and include identification of common data elements and approaches to networking data.