PRO11 Analysis of NICE Assessments for Orphan Treatments and the Role of Managed Access Agreements (MAAs)

Abstract

There are challenges in the development of orphan treatments, such as small patient numbers limiting clinical trial recruitment and uncertainties related to trial outcomes. This can lead to challenges in the health technology assessment (HTA) of orphan treatments. Treatments may receive recommendation with an MAA for further data collection to address uncertainties while enabling patient access. Pricing agreements such as a patient access scheme (PAS) or commercial access agreement (CAA) may also be required. We reviewed NICE assessments of orphan treatments and the role of MAAs. The EMA website was searched to identify orphan treatments approved between January 2010 and December 2020. The NICE website was searched for assessments of these treatments. Decision outcomes and key drivers were evaluated. Between 1st January 2010 and 17th December 2020, the EMA approved 137 orphan treatments. As of 4th January 2021, 65 had undergone complete NICE HTA with 92% recommended. A greater proportion of orphan treatments for oncology indications (80%) had a completed NICE assessment compared with non-oncology indications (31%). HTAs were in progress for 18 treatments (14 non-oncology) and 42 treatments were not assessed (40 non-oncology). Only 10% of recommended treatments did not have a PAS/CAA. MAAs were required for 19% of recommended non-oncology treatments and 33% of oncology treatments were recommended through the Cancer Drugs Fund (CDF) with an MAA. Uncertainties resulting in the requirement for an MAA included the lack of long-term and/or comparative data, and uncertainties in the size of the clinical benefit and cost-effectiveness estimates. Although NICE recommended most orphan treatments assessed, an MAA (including CDF) was required for 27% of treatments as further data collection was required. Furthermore, a CAA/PAS was important for 90% of recommended treatments. Understanding of the data uncertainties identified by HTA bodies may inform the market access strategy for orphan treatments in development.