OBJECTIVES: NICE evaluates all new active substances; since 2016 the Cancer Drugs Fund (CDF) has supported earlier access to promising cancer treatments, whilst further data is collected to address evidential uncertainties. The Innovative Medicines Fund (IMF) extended this managed access (MA) approach to any condition. We present a comparative analysis of recommendations by rarity of disease to provide insight about rare diseases.

METHODS: Recommendations from NICE’s technology appraisal (TA) and highly specialised technology (HST) programmes were reviewed from January 2021-April 2022. Rare cancers were defined as ≤6 cases per 100,000/year and rare non-cancer conditions as ≤ 1 per 2000. Recommendations and publication dates were extracted.

RESULTS: There were 102 appraisals producing 109 recommendations: 47.7% for cancers. Overall, 29.4% of all conditions appraised by both programmes were ‘rare’: 25.0% of cancers and 33.3% of other conditions. Overall, 90.6% of all rare conditions were recommended for reimbursement (i.e., routine commissioning or with MA) compared with 88.3% for non-rare.

92.3% of rare cancers received reimbursement recommendations compared with 89.7% of non-rare cancers. 89.5% of other rare conditions received reimbursement recommendations and 86.8% for other non-rare conditions. The HST programme reviews treatments for very rare conditions, six of these ‘other’ conditions were reviewed by HST and all received reimbursement recommendations.

Of those cancer topics recommended for reimbursement, 16.7% of rare cancers were recommended with MA compared with 22.9% for non-rare.

CONCLUSIONS: This review has found that rare conditions (cancer and others) are more likely to be recommended for routine use. Recommendations with managed access provide an invaluable secondary route to reimbursed patient access where there is significant remaining uncertainty. The IMF has established a clearer route to MA for rare conditions where evidential uncertainties could be addressed with further evidence generation for a limited period time, as seen in the CDF.