OBJECTIVES: To understand the role of patient input in Highly Specialized Technology (HST) assessments of ultra-orphan drug treatments by NICE.

METHODS: A systematic review of all NICE HST HTAs was conducted. A database was developed for each ultra-orphan drug assessment based on the type of patient input. Case studies were designed to understand the role, importance, and context of patient input. Insights and lessons were developed to aid future reimbursement and market access for ultra-orphan drugs.

RESULTS: From 2015 to 2023, NICE conducted 27 HST assessments for ultra-orphan products. There were 439 instances in which NICE quoted, cited, or mentioned patient input. The patient stakeholder was most often cited as a “Patient Expert” and occasionally as a “Patient Group.” All HST assessments included some mention of patient input. In some assessments, patient input was mentioned more than 20 times (e.g., Selumetinib, Afamelanotide, Onasemnogene abeparvovec). The majority of comments were related to the effects of the treatment (159 instances), followed by quality of life (58 instances). Patient input also highlighted the burden of the disease—severe or extreme impact of the disease (42 instances), school (21 instances), social life (20 instances), and delay in diagnosis (17 instances). Other less frequently mentioned topics included transplants, disability, and the effect of infusion. In 18 instances, NICE acknowledged the patient input, while in others, patient input was mentioned or cited along with clinician input.

CONCLUSIONS: NICE reports show a significant number of mentions of patient input and some acknowledgements. In some reports, patient feedback highlighted new or additional topics for consideration.