Improving access to orphan medicines – policy proposals January 2020
MAP BioPharma’s (MAP) report, Access to Orphan Medicines: A Case for Change, was published in February 2019. It highlighted significant challenges in the appraisal process for orphan medicines referred for review via the National Institute for Health and Care Excellence (NICE) Single Technology Appraisal (STA) compared to those assessed via the Highly Specialised Technology (HST) programme, which was developed specifically for orphan medicines.
As outlined in Access to Orphan Medicines: A Case for Change, NICE has a history of adapting its processes to ensure they are fit for purpose: end of life-criteria, Cancer Drugs Fund (CDF) arrangements and the fast-track appraisal all fit under the STA umbrella.
This suite of policy proposals sets out workable solutions that should be adopted in order to ensure a fair assessment of orphan medicines and timely access for patients to innovative treatments for conditions where there is often very high unmet need. There are three focus areas:
- Embedding revised evidence requirements for orphan medicines and rare disease expertise in the Single Technology Appraisal workstream
- Introducing interim recommendations for orphan medicines
- Adjusting incremental cost-effectiveness ratio (ICER) requirements for orphan medicines
Find the executive summary here.