Cost Effectiveness of Nivolumab in Patients with Advanced, Previously Treated Squamous and Non-squamous Non-small-cell Lung Cancer in England.

OBJECTIVE: The aim of this study was to investigate the cost effectiveness of nivolumab versus docetaxel in previously treated, advanced non-small-cell lung cancer (NSCLC) in England and assess how conditional reimbursement within the Cancer Drugs Fund (CDF) can be used to ensure timely patient access to effective treatments. METHODS: Cost-effectiveness models developed for the National Institute for Health and Care Excellence (NICE) TA483 (squamous) and TA484 (non-squamous) technology appraisals were supplemented with updated overall survival (OS), progression-free survival (PFS), and time-to-treatment discontinuation data collected as part of the CDF data collection agreement. Both models were developed by using a partitioned-survival approach based on PFS and OS predictions from CheckMate 017 and CheckMate 057 to estimate the projected proportion of patients in each health state (progression free, progression, death) throughout the model's time horizon. The primary outcomes were estimated costs, quality-adjusted life-years (QALYs), and the resulting incremental cost-effectiveness ratio (ICER) expressed as cost/QALY gained. RESULTS: Base-case ICERs for treating patients with nivolumab versus docetaxel were £35,657/QALY and £38,703/QALY for squamous and non-squamous NSCLC patients, respectively, which are substantially lower than those obtained from what were deemed to be the most appropriate analyses for decision making in the original submissions when run with the same patient access scheme discount: £68,576/QALY and £73,189/QALY gained for squamous and non-squamous NSCLC, respectively. CONCLUSIONS: Nivolumab versus docetaxel is cost effective for treating locally advanced/metastatic NSCLC after prior chemotherapy in adults, regardless of tumour histology or programmed death-ligand 1 expression status.

Cost-effectiveness of cardiovascular imaging for stable coronary heart disease.

OBJECTIVE: To assess the cost-effectiveness of management strategies for patients presenting with chest pain and suspected coronary heart disease (CHD): (1) cardiovascular magnetic resonance (CMR); (2) myocardial perfusion scintigraphy (MPS); and (3) UK National Institute for Health and Care Excellence (NICE) guideline-guided care. METHODS: Using UK data for 1202 patients from the Clinical Evaluation of Magnetic Resonance Imaging in Coronary Heart Disease 2 trial, we conducted an economic evaluation to assess the cost-effectiveness of CMR, MPS and NICE guidelines. Health outcomes were expressed as quality-adjusted life-years (QALYs), and costs reflected UK pound sterling in 2016-2017. Cost-effectiveness results were presented as incremental cost-effectiveness ratios and incremental net health benefits overall and for low, medium and high pretest likelihood of CHD subgroups. RESULTS: CMR had the highest estimated QALY gain overall (2.21 (95% credible interval 2.15, 2.26) compared with 2.07 (1.92, 2.20) for NICE and 2.11 (2.01, 2.22) for MPS) and incurred comparable costs (overall £1625 (£1431, £1824) compared with £1753 (£1473, £2032) for NICE and £1768 (£1572, £1989) for MPS). Overall, CMR was the cost-effective strategy, being the dominant strategy (more effective, less costly) with incremental net health benefits per patient of 0.146 QALYs (-0.18, 0.406) compared with NICE guidelines at a cost-effectiveness threshold of £15 000 per QALY (93% probability of cost-effectiveness). Results were similar in the pretest likelihood subgroups. CONCLUSIONS: CMR-guided care is cost-effective overall and across all pretest likelihood subgroups, compared with MPS and NICE guidelines.

Economic Modeling Considerations for Rare Diseases.

OBJECTIVES: To identify challenges that affect the feasibility and rigor of economic models in rare diseases and strategies that manufacturers have employed in health technology assessment submissions to demonstrate the value of new orphan products that have limited study data. METHODS: Targeted reviews of PubMed, the National Institute for Health and Care Excellence's (NICE's) Highly Specialised Technologies (HST), and the Scottish Medicines Consortium's (SMC's) ultra-orphan submissions were performed. RESULTS: A total of 19 PubMed studies, 3 published NICE HSTs, and 11 ultra-orphan SMC submissions were eligible for inclusion. In rare diseases, a number of different factors may affect the model's ability to comply with good practice recommendations. Many products for the treatment of rare diseases have an incomplete efficacy and safety profile at product launch. In addition, there is often limited available natural history and epidemiology data. Information on the direct and indirect cost burden of an orphan disease also may be limited, making it difficult to estimate the potential economic benefit of treatment. These challenges can prevent accurate estimation of a new product's benefits in relation to costs. Approaches that can address such challenges include using patient and/or clinician feedback to inform model assumptions; data from disease analogues; epidemiological techniques, such as matching-adjusted indirect comparison; and long-term data collection. CONCLUSIONS: Modeling in rare diseases is often challenging; however, a number of approaches are available to support the development of model structures and the collation of input parameters and to manage uncertainty.