OBJECTIVES: To evaluate the success of Technology Appraisals (TAs) for Orphan Drugs (ODs) for Rare Oncology (RO) and Rare Disease (RD).

METHODS: Review of NICE and SMC for authorised ODs on the community register of orphan medicinal products up-to June 1st 2018.

RESULTS: Of 102 ODs with 121 orphan indications, 127 NICE (50 RO; 77 RD) and 136 SMC (53 RO; 83 RD) TAs were identified. NICE Highly Specialised Technology (HST) process was applied for 11 RD. SMC Patient and Clinician Engagement (PACE) process was applied for 40 (21 RO; 19 RD). Lack of TAs or none submissions were identified for 48% (16% RO; 69% RD) for NICE and 36% (28% RO, 41% RD) for SMC. Of 43 completed NICE TAs, 39 were recommended [27 Patient Access Schemes (PAS); 9 Managed Access Agreements (MAA) with PAS]. All 7 completed HSTs were recommended (3 PAS, 2 MAA+PAS). 54 of 71 completed SMC TAs were recommended/restricted [14 PAS, 5 PACE and 24 PAS+PACE]. NICE and SMC agreed on 24/25 RO and 8/11 RD.

CONCLUSIONS: OD TA rate is low with absence of TAs in half of indications by NICE and a third by SMC. Published SMC TAs steadily increased to plateau in 2016-17, versus an exponential increase with 40% published in 2017 by NICE. NICE is less likely to appraise RD than SMC and, as more selective, the likelihood of recommendation is higher (a quarter of SMC TAs are not recommended). Variation exists on undertaking HTAs, leading to absence of guidance in England and a higher proportion not recommended in Scotland despite PACE introduced in May 2014. For common TAs the level of consensus is high, but with some disparity for RD. Increased consistency on topic selection with more TAs and quicker, particularly for RD, is needed to assess the success of HTA processes for ODs.