Pmu77 Cell and Gene Therapies Are Associated With Substantially Larger Quality-Adjusted Life Year Gains Than Conventional Drugs and Biologics

Abstract

Objectives: Combinations of existing therapies are increasingly being developed and marketed across many indications. Whilst they promise substantial clinical benefits, dual-branded therapies may have substantial financial impacts. Obtaining positive HTA approvals and public reimbursement may be a major challenge, which may be amplified if each monotherapy is marketed by a different company. We evaluated whether combination therapies developed by one manufacturer had faster and/or better outcomes than those where each constituent was developed by a separate manufacturer and whether this varied by different HTA archetypes. Methods: ECapproved combination products, comprising at least one on-patent therapy marketed under a separate brand were identified (01/01/2015-31/12/2018) and the number of manufacturers identified. Corresponding HTA reports were identified from G-BA, HAS, SMC, and NCPE websites and relevant data extracted. Results: 114 HTA assessments across 34 combination products:indications were identified (same manufacturer: 58 assessments, two manufacturers: 56 assessments). For clinicaleffectiveness HTA bodies (G-BA and HAS), the rates of positive recommendations (defined as: any additional benefit or ASMR I-III) was similar for combination therapies developed by one vs. two manufacturers (32% vs. 40%). Whereas, for costeffectiveness HTA bodies (NICE, SMC, and NCPE), combination therapies developed by one manufacturer received numerically higher rates of positive recommendations (defined as: "full or optimized/conditional") than those involving two manufacturers (80% vs. 58%). However, there were no corresponding differences in the median time from EC-approval to positive recommendation (5.5 vs. 5.0 months). Conclusions: HTA bodies assessing cost-effectiveness were more likely to issue positive recommendations for dual-branded treatments if each constituent monotherapy was marketed by one company versus two companies. A single company may have more flexibility in its price setting, facilitating higher HTA recommendation rates. Although time to positive HTA decisions were similar, this may not reflect time to launch in some jurisdictions, due to additional time required for pricing negotiations.

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