Abstract
BACKGROUND: This study aimed to summarize the current practices and mechanisms of market access, pricing and reimbursements for gene therapy products (GTPs) in major jurisdictions, and to identify the key barriers and facilitators affecting the translation of GTPs from regulatory approval to market access across the countries. METHODS: An integrated scoping review was conducted to identify publicly available literature and documents on the marketing access, pricing and reimbursement of GTPs under the PRISMA-ScR guidelines. Key barriers and potential enablers were identified and thematically analyzed using the Consolidated Framework for Implementation Research (CFIR). RESULTS: Thirty-four studies published between 2020 and May 2025 were included in this study. A total of 21 GTPs had received market authorization from the FDA, EMA, PMDA, or NMPA, targeting four major disease areas: genetic diseases (n = 8), hematologic disorders (n = 7), cancer-related indications (n = 5), and vascular diseases (n = 1). Despite growing approvals, substantial variation exists across jurisdictions in terms of access, pricing, and reimbursement pathways. Commonly reported challenges include concerns over budget impact and affordability (n = 25), uncertainty in clinical evidence (n = 24), limited value assessment frameworks (n = 17), lack of clearly defined reimbursement pathways (n = 17), misalignment between regulatory requirements and real-world implementation capacity (n = 15), and insufficient delivery infrastructure (n = 12). To address these barriers, 15 studies proposed potential solutions involving five areas: cross-country regulatory alignment and processes streaming (n = 3), pricing and reimbursement reform including budget caps, annuity payments and patent buyouts (n = 12), enhanced evidence generation through RWE and adaptive trial designs (n = 5), institutional infrastructure and workforce capacity building (n = 4), and early multi-stakeholder engagement among regulators, HTA bodies, payers, healthcare providers, and patients to align expectations and accelerate access (n = 7). CONCLUSION: By adopting the CFIR framework, this study has systematically identified the key challenges and potential solutions in translating GTPs from regulatory approval to patient access. To ensure effective access, it is essential to adopt context-adapted value assessment models, diversified payment mechanisms, and coordinated policy strategies to guide the implementation processes. Building on international experiences, advancing localized implementation strategies encompassing tailored value frameworks, innovative payment models, regional pilots and institutional readiness offer actionable pathways for developing forward-looking access and reimbursement systems for GTPs.