Rare Disease Access in the Middle East: Key Challenges

Rare diseases present unique challenges for healthcare systems across the Middle East. While advances in diagnostics and innovative therapies are creating new possibilities, patients can still face significant barriers from diagnosis through treatment access.

The evidence challenge

Small patient populations and limited local epidemiological data can make it difficult to understand the true burden of rare diseases. Recent regional research highlights gaps in national registries, local data and diagnostic infrastructure, limiting evidence available to clinicians, policymakers and payers.

Registries and real-world evidence can help address these gaps by improving understanding of patient populations, disease pathways, outcomes and unmet needs.

Diagnosis remains a major barrier

Rare diseases can be difficult to recognize, and access to specialized expertise and genetic testing varies across the region. Recent Middle East research identifies limited diagnostic expertise, testing costs and fragmented care among important barriers to timely diagnosis.

From approval to patient access

Regulatory approval does not necessarily translate into immediate patient access. Rare-disease therapies often come with high costs and limited clinical evidence, creating challenges for health technology assessment, reimbursement and funding decisions. Research from Saudi Arabia has similarly identified evidence uncertainty, economic constraints and reimbursement complexity as key challenges.

These characteristics increase the importance of health economics, real-world evidence, value assessment and innovative access approaches when developing the evidence case for rare-disease therapies.

A multidisciplinary challenge

Rare-disease access rarely has a single solution. Progress can require collaboration between patients, clinicians, researchers, pharmaceutical companies, payers, policymakers and patient organizations.

For life sciences companies, this means thinking beyond regulatory approval and considering the complete evidence pathway—from understanding disease burden and patient experience to demonstrating clinical, economic and societal value.

How VI Research can support

VI Research supports rare-disease evidence and access strategies through epidemiological research, evidence synthesis, real-world evidence, HEOR, patient research, value assessment and market access analysis.

By bringing these perspectives together, we help organizations develop and strengthen the evidence case for access and support evidence-informed healthcare and reimbursement discussions.