News & Insights

As evidence teams plan for 2026, expectations in rare diseases are shifting towards evidence that is carefully contextualised and clearly linked to decision-making needs. Recent guidance and industry commentary highlight the growing role of patient experience data, integrated real-world evidence, and earlier cross-functional planning, reinforcing that credibility comes from clarity and relevance rather than scale or complexity alone.

Inherited bleeding disorders (iBDs), such as haemophilia A and B, von Willebrand disease (VWD), and Glanzmann thrombasthenia, present unique challenges for health technology assessment (HTA). Treatments are often lifelong, costly, and evolving rapidly, especially with the advent of gene therapies and non-factor replacement options like emicizumab. Economic evaluations (EEs) are essential to guide reimbursement and policy decisions, and modelling plays a central role in these