OBJECTIVES:

The EMA orphan drug legislation incentivises the development of therapies for rare diseases. Medicines approved with orphan designation have become increasingly common; however regulatory approval does not guarantee favourable reimbursement. This research undertakes an analysis of EMA-approved orphan drugs and discusses regulatory versus access requirements. Haematological malignancies have been selected as an area of focus; an indication containing many therapies with orphan designations.

METHODS:

All publically-available EMA and HTA reports from NICE, SMC and HAS of blood cancer drugs that have been EMA-approved with an orphan designation have been reviewed (from November 2002 to April 2017) and key data extracted.

RESULTS: CONCLUSIONS:

Therapies for haematological malignancies with EMA-orphan designations have obtained marketing authorisation on data packages lacking comparative Phase 3 data or any data on any survival endpoints. However, at an HTA-level some have struggled to attain favourable recommendations. As competition within the therapy area is increasing, the hurdles for reimbursement may further increase, widening the evidentiary divergence between regulators and payers, potentially necessitating greater utilisation of RWE studies and/or innovative contracting for optimal reimbursement.