ArticleJournal of pharmaceutical policy and practice2025
Challenges in evaluation and reimbursement of drugs for rare diseases in Saudi Arabia: a Delphi expert consensus.
Article in Journal of pharmaceutical policy and practice, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
1 citing paper in PubMed.
- Research Gaps and Priorities for aHUS in the Gulf Cooperation Countries (GCC): Findings from a Payer-Focused Expert Meeting.ClinicoEconomics and outcomes research : CEOR · 2026Article
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Authors and funding
19 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.