MAP BioPharma Report on access to OMPs in the UK

Our member MAP BioPharma recently put forward a series of recommendations focused on UK’s appraisals and reimbursement processes, in their latest publication titled Access to Orphan Medicines: A Case for Change.

The report shows that:

• Very small differences between treatments in terms of patient numbers or clinical management can have a significant impact on the criteria against which they are assessed, which severely disadvantages those that are routed for a Single Technology Appraisal (STA) as opposed to the Highly Specialised Technology (HST) programme;
• Of the 24 completed STA reviews of orphan treatments between 2013 and 2017, 50% were recommended with restrictions compared to 21% of non-orphan treatments. Very few (13%) of orphan medicines were recommended within their full marketing authorisation, compared to two thirds of non-orphan medicines;
• The number of non- cancer orphans reviewed by STA is very small (6) and of four completed appraisals, none of these have been recommended within their full marketing authorisation.

In the report’s conclusions, MAP BioPharma and members recommend that:

• it is time for NICE to revisit its arrangements for the very small number of non-cancer orphan medicines assessed via the STA programme,
• The recent commitment in the Voluntary Scheme for Branded Medicines Pricing for NICE to review all new active substances (unless under certain circumstances) could place even greater pressure on the system and
• In this context, it is essential that arrangements for orphan medicines are reviewed without delay.