Accelerating Access to Promising New Pediatric Medicines in Canada: Comparing International Approaches to Regulation and Reimbursement
Author(s)
Cressman C, Denburg A
Hospital for Sick Children, Toronto, ON, Canada
OBJECTIVES:
Existing policies on drug regulation, HTA methods, and funding mechanisms in most health systems rarely account for the unique needs of children, resulting in significant access constraints. The relevance of innovative, precision therapeutics is rapidly expanding, as are rising costs of the same, exacerbating pre-existing challenges. Our study sought to understand the policy and regulatory challenges related to the evaluation and reimbursement of innovative therapies for children.METHODS:
Using in-depth interviews with experts and comparative analysis of policy documents, we identified the policy, legislative and regulatory environments across select Canadian and international jurisdictions. Specific attention is paid to therapeutic safety and efficacy, market authorization, and funding allocations for children. Focusing on rare disease and pediatric oncology further illuminates where and how policy dynamics impact access. Drawing on HTA scholarship and policy theory, a critical interpretive approach guided analysis.RESULTS:
Health systems globally are grappling with challenges presented by precision technologies and pediatric therapies, namely uncertain benefit and high costs. Policies and regulations that address the unique socio-biological, economic, and ethical considerations inherent in precision child health are lacking but many efforts are underway to innovate clinical research and HTA methods. We describe distinct approaches to a similar set of challenges and identify how policy contexts (governance structures, processes, connections between regulatory and HTA activities) as well as stakeholders and societal values impact access. We highlight differences in policy priorities and attention, in the included sets of values and stakeholder voices, and in the HTA and regulatory pathways created to meet the needs of novel paediatric therapies.CONCLUSIONS:
This work illuminates a shared set of challenges ripe for collaborative efforts at policy reform. We hope to provide provincial/territorial and federal policymakers with evidence-informed considerations for the design and implementation of policies to govern equitable and sustainable access to innovative therapies for children.Conference/Value in Health Info
2023-05, ISPOR 2023, Boston, MA, USA
Value in Health, Volume 26, Issue 6, S2 (June 2023)
Code
HPR8
Topic
Health Policy & Regulatory
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Health Disparities & Equity, Public Spending & National Health Expenditures, Reimbursement & Access Policy
Disease
Oncology, Rare & Orphan Diseases