Challenges in evaluation and reimbursement of drugs for rare diseases in Saudi Arabia: a Delphi expert consensus

Background: Drugs for rare diseases (DRDs) present unique challenges in evaluation and reimbursement due to high costs, limited clinical evidence, and complex healthcare decision-making. While international models exist for DRD reimbursement, Saudi Arabia faces distinct obstacles driven by high incidence of genetic diseases, healthcare system fragmentation, and evolving national policies. This study aims to identify key challenges in evaluating and reimbursing DRDs in Saudi Arabia. Methods: A modified Delphi method was conducted between January and February 2025, involving healthcare policymakers, clinicians, health economists, and formulary decision-makers across multiple institutions. The process included three rounds: (1) open-ended questionnaires to identify challenges, (2) voting on structured statements using a Likert scale, and (3) consensus refinement through a roundtable discussion Results: Nineteen experts participated, with final consensus reached on 88 statements across eight themes: evaluation complexities, evidence limitations, economic and budgetary constraints, supply chain management, data generation and infrastructure, stakeholder concerns, patient and family barriers, and policy and collaboration gaps. The highest concern was economic constraints. Other challenges included inconsistencies in reimbursement decisions, and difficulties in managed entry agreements. Stakeholders emphasised the need for structured decision-making frameworks and national coordination to improve access and equity. Conclusion: This study provides the first consensus on challenges in DRD evaluation and reimbursement in Saudi Arabia. Addressing these issues through policy reforms, stakeholder collaboration, and data infrastructure enhancement is crucial to optimising resource allocation and patient access. Further research should focus on implementing value-based agreement to ensure sustainable reimbursement strategies for DRDs.