IRDiRC shares a new publication developed by its Regulatory Scientific Committee (RSC): “Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval”.
With over 300 million people affected by rare diseases, timely access to effective therapies is critical. Despite strong alignment in regulatory expectations, patients in many regions face years of delays highlighting the need for improved international coordination. By examining 53 orphan medicines approved between 2021–2022 across six key regulatory regions, the study underscores persistent delays in worldwide access to innovative rare disease therapies.
Key Insights
- Significant approval delays — Average 3-year gap between first and subsequent approvals.
- Limited global submissions — 99% approved in two or more regions, but few filed broadly across all major jurisdictions.
- Aligned evidence requirements — 69% of approvals relied on identical or highly similar evidence packages.
- Streamlined data needs — Most products were authorized based on a single adequate and well-controlled study with supporting evidence.
- Clear opportunity for acceleration — Findings reinforce the value of collaborative regulatory models to reduce global timelines.
