HTA decision-making for drugs for rare diseases: comparison of processes across countries.

ABSTRACT:

Introduction

Drugs for rare diseases (DRDs) offer important health benefits, but challenge traditional health technology assessment, reimbursement, and pricing processes due to limited effectiveness evidence. Recently, modified processes to address these challenges while improving patient access have been proposed in Canada. This review examined processes in 12 jurisdictions to develop recommendations for consideration during formal government-led multi-sectoral discussions currently taking place in Canada.

Methods

(i) A scoping review of DRD reimbursement processes, (ii) key informant interviews, (iii) a case study of evaluations for and the reimbursement status of a set of 7 DRDs, and (iv) a virtual, multi-stakeholder consultation retreat were conducted.

Results

Onl

SUBMITTER: Stafinski T 

PROVIDER: S-EPMC9264608 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature