PP299 A Framework And Analysis Assessing The Impact Of Patient-Centered Outcome Evidence In HTA Appraisals

IntroductionThe importance of patient-centered outcome (PCO) evidence is increasingly recognized, but its inclusion in Health Technology Assessment (HTA) submissions remains inconsistent. We explored the impact of PCO evidence on HTA decision-making.MethodsA framework was developed to assess the impact of PCO evidence (excluding EQ-5D) on HTA appraisals. An impact rating was determined by reviewing company, committee and Evidence Review Group (ERG) opinion. This was applied to publicly available appraisal documents (National Institute for Health and Care Excellence [NICE]: 8; Scottish Medicines Consortium [SMC]: 2) in a pilot study. The framework was then refined and applied to a larger dataset.ResultsPCO evidence had ‘substantial impact’ in 3/8 NICE and 1/2 SMC appraisals, and ‘some impact’ in those remaining. PCO evidence informed the cost-effectiveness model in 2/8 NICE and 1/2 SMC submissions, and was considered superior to EQ-5D evidence in one NICE and one SMC submission. The ERG considered PCO evidence relevant to decision-making in 5/8 NICE appraisals. PCO evidence was mentioned in guidance for 7/10 appraisals (deemed relevant in 5/10). In one assessment, committee comments were notably more favorable than ERG comments. Larger dataset analysis results provided further insights to the pilot study.ConclusionsThe framework allows a systematic approach to evaluating the impact of PCO evidence on HTA appraisals.BL, AP, DGB and NY are employees of Symmetron Ltd, which received funding from Pfizer UK in connection with the development of this manuscript. KH, HT, SLC and JB are employees of Pfizer UK. This study was sponsored by Pfizer UK.

Brentuximab Vedotin for Treating Relapsed or Refractory CD30-Positive Cutaneous T-Cell Lymphoma: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

As part of the single technology appraisal process, the National Institute for Health and Care Excellence invited Takeda UK Ltd to submit clinical- and cost-effectiveness evidence for brentuximab vedotin (BV) for treating relapsed or refractory CD30-positive (CD30+) cutaneous T-cell lymphoma (CTCL). The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned to act as the evidence review group (ERG). This article summarises the ERG’s review of the company’s submission for BV and the appraisal committee (AC) decision. The principal clinical evidence was derived from a subgroup of patients with advanced-stage CD30+ mycosis fungoides (MF) or primary cutaneous anaplastic large-cell lymphoma (pcALCL) in the phase III ALCANZA randomised controlled trial (RCT). This trial compared BV versus physician’s choice (PC) of methotrexate or bexarotene. Evidence from three observational studies was also presented, which included patients with other CTCL subtypes. The ERG’s main concerns with the clinical evidence were the lack of RCT evidence for CTCL subtypes other than MF or pcALCL, lack of robust overall survival data (data were immature and confounded by subsequent treatment and treatment crossover on disease progression) and lack of conclusive results from analyses of health-related quality-of-life data. The ERG noted that many areas of uncertainty in the cost-effectiveness analysis were related to the clinical data, arising from the rarity of the condition and its subtypes and the complexity of the treatment pathway. The ERG highlighted that the inclusion of allogeneic stem-cell transplant (alloSCT) as an option in the treatment pathway was based on weak evidence and generated more uncertainty in a disease area that, because of its rarity and diversity, was already highly uncertain. The ERG also lacked confidence in the company’s modelling of the post-progression pathway and was concerned that it may not produce reliable results. Results from the company’s base-case comparison (including a simple discount patient access scheme [PAS] for BV) showed that treatment with BV dominated PC. The ERG’s revisions and scenario analyses highlighted the high level of uncertainty around the company base-case cost-effectiveness results, ranging from BV dominating PC to an incremental cost-effectiveness ratio per quality-adjusted life-year gained of £494,981. The AC concluded that it was appropriate to include alloSCT in the treatment pathway even though data were limited. The AC recommended BV as an option for treating CD30+ CTCL after at least one systemic therapy in adults if they have MF, stage IIB or higher pcALCL or Sézary syndrome and if the company provides BV according to the commercial arrangement (i.e. simple discount PAS).