The 17th World Orphan Drug Congress Europe (WODC) convened 26-28 October 2026 at the RAI Amsterdam Convention Centre, bringing together industry leaders, regulators, payers, clinicians, researchers and patient organisations from across the rare disease community.

A throughline across the congress was the gap between scientific progress and the patients it is meant to reach. Posters and sessions returned repeatedly to delays at both ends of the journey: diagnosis that arrives decades after first symptoms, and approved medicines that are not available to everyone who needs them. As one session slide put it, a rare disease is not a rare policy challenge.

As the new EU pharmaceutical legislation, joint clinical assessment and the proposed Biotech Act take shape, and gene, cell and RNA-based therapies move into ever rarer indications, a recurring concern was that evidence standards, reimbursement models and national access routes are not yet settled. Centralised approval does not guarantee availability in every member state, and the speed of innovation risks outpacing the systems needed to deliver it.

This report covers posters and sessions across three key themes.

1.Diagnosis presents data on diagnostic delay in HHT, the modelled impact of earlier diagnosis in ATTRv-PN, misdiagnosis of rare gastrointestinal diseases, and emerging AI and data tools.

2.Evidence and Access reviews the EU pharma package, JCA and the Biotech Act alongside German benefit assessment, comparator choice, representative evidence, early HTA, ATMP availability and the transition from paediatric to adult care.

3.Innovation captures the next frontier in rare disease, from antisense, siRNA and CAR-T therapies to early-stage assets, funding levers and complement therapeutics.