OBJECTIVES: Pathways for drug regulation, HTA, and funding in most health systems rarely account for the unique needs of children, resulting in impediments to access. The indications and costs of innovative therapies for paediatric diseases are rapidly expanding, exacerbating existing access barriers along the therapeutic development pipeline. We sought to understand policy development and implementation challenges related to the evaluation and reimbursement of innovative therapies for children.

METHODS: Study design was informed by interpretive, qualitative methodology. We conducted in-depth interviews with a stratified sample of experts representing regulatory, HTA, and clinical settings, and policy document analysis. We mapped governance and regulatory environments across Canada, the European Union (EU) and the United Kingdom (UK), comparing policy approaches, and focusing on paediatric oncology and rare disease drugs. We drew on theories of the policy process and HTA scholarship to critically analyse themes within the data.

RESULTS: High costs and uncertain benefits associated with precision therapies present allocative challenges for health system decision-makers. Evidentiary and ethical considerations important to the paediatric space (e.g., ubiquity of rare disease, trial enrolment complexities) compound these challenges. Policies that attend to the unique socio-biological, economic, and ethical dimensions of child health are lacking. Jurisdictional distinctions in policy framing and attention, and in governance structures condition differences in regulatory approval and public reimbursement of emerging child health technologies. Respondents suggest that barriers in access to paediatric therapies can be surmounted by implementing carrot and stick drug development incentives, harmonizing evidence review pathways, and engaging stakeholders upstream.

CONCLUSIONS: While Canadian, EU and UK stakeholders grapple with overlapping challenges related to child health technologies, key differences in policy environments have resulted in divergent responses and opportunities for policy evolution. Canada can learn from the international policy landscape where mechanisms to integrate paediatric and precision medicine into drug regulation and HTA processes are being implemented.