OBJECTIVES

An orphan designation from the EMA is for indications which have a prevalence of ≤5 in 10,000 in the EU, are life-threatening or severely disabling, and have no satisfactory treatment available. The NICE Highly Specialised Technology Programme (HSTP) and the SMC consider ultra-orphan to be <1 in 50,000, and meeting other specialised criteria. This research reviewed recent assessments of orphan and ultra-orphan drugs by NICE and the SMC, and disparities in availability for NHS patients between England and Scotland.

METHODS

EMA website searched May 2019 to identify orphan treatments approved between 1^st January 2018 and 30^th April 2019. Treatments approved by the EMA, then subsequently assessed by NICE and SMC were reviewed.

RESULTS

Twenty-one orphan treatments received EMA marketing authorisation in the timeframe specified. Six were assessed by NICE and five received positive recommendations; one via HSTP (burosumab). For SMC, five have been assessed, and four given positive recommendation. There are several differences in recommendations between SMC and NICE. For the four recommended by SMC, two are recommended by NICE via single technology appraisal (STA) process. Tisagenlecleucel for R/R DLBCL was not recommended by SMC via ultra-orphan process, however this is available for NHS patients in England via NICE CDF, as part of an STA. The SMC stated that the high upfront cost is associated with financial risk due to uncertainties in long-term effectiveness. Although NICE had similar concerns regarding uncertainty, the NICE CDF allows for tisagenlecleucel to be available to patients through a managed access scheme while further data is collected.

CONCLUSIONS

There are different NICE and SMC procedures, leading to disparities in availability of orphan treatments for NHS patients. The SMC Patient and Clinician Engagement meetings address evidence gaps when assessing orphan diseases, whereas NICE has policies within their STA process which can promote different results despite rarity of the condition.