A791between 2010 and 2015 were conducted. For each CAV appraisal, information regarding the level of evidence and clinical effectiveness was collected. Results: Twenty antiviral drugs (47 indications) were assessed. All obtained a favourable opinion for reimbursement with 11 a major to moderate CAV, 14 a minor and 22 no CAV. No one obtained a major CAV. The majority of studies [46% (22/47)] were non-active comparator studies based on virological response and supported an important to moderate CAV in 32% (7/22), as sofosbuvir that granted the highest CAV level (important CAV). Among the 19 comparative trials, 68% (13/19) were of superiority design supporting a major to moderate CAV in 31% (4/13) of the cases. A higher proportion of important to moderate CAV were observed in trials using a relevant primary endpoint (26%) versus no one for a study using an exploratory endpoint such as pharmacokinetic endpoint. ConClusions: A high proportion (53%) of CAV is recognized in virology compared to the other therapeutic areas (15%) while non-active comparator studies are made. This report shows that HAS appraisals remain multi-factorial. HAS' expectations in terms of level of evidence take into account the medical need, therapeutic area specificity and a rapid evolution of the therapeutic strategy. The emergence of anti-viral drug resistance can also influence the CAV appraisal.

diabetes. Diabetic patients treatment acceptance is primarily driven by perceived effectiveness. Long-term treatment is their major concern. These findings give indications about T1D and T2D patients' priorities and unmet needs.

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We investigated how utility values (UVs) were selected by manufacturers and the evidence review groups (ERGs) and what were discussed on UVs during the appraisal process in NICE. Methods: We reviewed NICE Technology Appraisal (TA) guidance and committee papers available on the NICE website, including the types of instrument of UVs; source of utility data and utilization (i.e., mapping) in the submission by manufacturers and the ERGs; the final decision by NICE. Our review included individual TAs for pharmaceuticals published between April 2018 and March 2019. Results: After exclusion of 13 TAs (3 multiple TAs; 2 cost-comparison TA; 1 non-pharmaceutical TA; 7 terminated TAs) from 56 TAs (TA517-TA572), 43 TAs which contain 150 unique health state UVs were identified. Sixty-four UVs (43%) and 13 UVs (9%) were obtained by using EQ-5D (3L) and EQ-5D-5L in the manufacturers' submissions, respectively. Seventy-three UVs (49%) were mainly taken from pivotal clinical trials, followed by 62 (41%) from published studies including previous NICE TAs. Mapping was performed for 33 UVs (22%) in the manufacturers' submissions. The ERGs agreed to 74 UVs (49%) selected by manufacturers, while 67 UVs (45%) were criticized and 9 UVs (6%) were not clear from TAs and committee papers. Of the 67 UVs criticized by the ERGs, 47 UVs (70%) were finally replaced to the values selected by the ERGs, while 13 UVs (19%) remained in the final appraisals. Conclusions: EQ-5D, which was recommended in the NICE 2013 reference case, was a major measure of the health-related quality of life in the manufactures' submissions, but there were substantial differences in the selection of UVs between manufactures and the ERGs during the appraisal process. Nearly half of the UVs used in NICE TAs were criticized by the ERGs, and only around half of the original UVs submitted by manufacturers were adopted in the final guidance.