OBJECTIVES: To understand trends in assessments of HSTs and develop any lessons learned.

METHODS: A systematic review of all UK’s NICE HSTs reports was conducted. Reports were reviewed for all types of evidence. For identified reports, we analyzed the trends in assessment decision, products, cost and evidence.

RESULTS: Since the inception of the HST program, six products have been assessed by the committee. The indications for these 6 products covered severe diseases, with very poor prognosis, multiple organ damage and high mortality rates. All six products were recommended for coverage. However, 3 of 6 products were recommended with a conditional managed access agreement. Three products were required to have patient access schemes. One product was recommended with a conditional program for monitoring, national protocol and research. The median annual drug cost was $410,189 (Range: $291,900-$548,605). The median overall prevalence of number of patients in England was ~212 (Range: 1 to 855). The total number of patient for all six products combined is less than 1500 per year. For some products, the prevalence estimates had a wide range, hence there is some uncertainty in determining accurate population estimates. Interestingly, 4 of the 6 products submitted clinical evidence with data from randomized clinical trials, including 2 with double blind studies. While RCTs studies were small with 15-20 patients per arm, some studies had more than 150 patients (e.g. ataluren). Two of the 6 products’ clinical data was based on open label studies with extension arms. For two products, natural history RWE studies were cited for burden of disease evidence. For economic evidence, the cost-effectiveness ratios were not reported in public domain. Interestingly, only Quality-Adjusted Life Years (QALYs) were reported; with median incremental QALY being 11 (Range:1-22).

CONCLUSIONS: Review of HST reports provides valuable insights for new rare disease products for their evidence generation.