Challenges in evaluation and reimbursement of drugs for rare diseases in Saudi Arabia: a Delphi expert consensus
Year: 2025
Type: article
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.
Authors: Laila Carolina Abu Esba, Consuela Cheriece Yousef, Mansoor Ahmed Khan, Sakra Balhareth, Hussain A. Al-Omar, Amal Al-Najjar, Aljawharah Alkoraishi, Abdullah M. Alhammad, Ziyad Saeed Almalki, Esraa S. Altawil, Roaa Al Gain, Mohamed Ahmed, Hend Metwali, Layla Al Anizy, Fatma Maraiki, Abdulrazaq Al Jazairi, Abdulaziz Alhossan, Mohammed Al Harbi, Hind Almodaimegh
Institutions: King Saud bin Abdulaziz University for Health Sciences, King Abdullah International Medical Research Center, National Guard Health Affairs, Imam Abdulrahman Al Faisal Hospital, King Abdulaziz Medical City, King Faisal Specialist Hospital & Research Centre, King Saud Medical City, King Saud University, Ministry of Health, Prince Sattam Bin Abdulaziz University, King Fahd Medical City
Language: English
Subfield: Economics and Econometrics
Domain: Social Sciences
Open Access status: gold
Estimated APC paid: $2,390
OpenAlex ID: W4415620151