Estimating the Allocation of the Economic Value Generated by Utilization of All-Oral Direct-Acting Antivirals for Hepatitis C in the United States, 2015 to 2019.

OBJECTIVES: Between 2013 to 2019, several all-oral direct-acting antivirals (DAAs) were launched with the potential to cure patients with hepatitis C virus (HCV). They generated economic value in terms of the health gains for patients and cost-savings for the US healthcare system. We estimated the share of this value allocated to 4 manufacturers vs society. METHODS: For 2015 to 2019, we estimated the incremental impact of DAAs on HCV health outcomes and costs. We used the Center for Disease Analysis Foundation Polaris Observatory database to estimate utilization. Per-patient projections of lifetime quality-adjusted life-years (QALYs) gained and medical costs avoided were based on a standard 9-state HCV disease-progression model for DAA treatment vs alternatives. Annual QALY gains were valued at $114 000 per QALY. Outcomes and costs were discounted at 3%. Estimated revenues were based on reported sales. RESULTS: An estimated 1 080 000 patients received DAAs: 81.5% would not have received the pre-DAA standard of care. On average, these patients were projected to gain 4.4 QALYs and save $104 400 in lifetime healthcare costs, generating $531.8 billion in value. Those who would have received treatment gained 1.7 QALYs and saved $41 500 in lifetime costs, generating $47.4 billion in economic value. As treatment costs fell nearly 75%, the 4 manufacturers reported $37.4 billion from DAA sales-an allocation of 6.5% of the total value. CONCLUSIONS: The significant majority (∼90%) of the economic value of curing HCV with DAAs were health benefits to patients and net cost-savings to society. DAA manufacturers received a minority share (6.5%) of the aggregate economic value generated.

The evolving value assessment of cancer therapies: Results from a modified Delphi study.

The move toward early detection and treatment of cancer presents challenges for value assessment using traditional endpoints. Current cancer management rarely considers the full economic and societal benefits of therapies. Our study used a modified Delphi process to develop principles for defining and assessing value of cancer therapies that aligns with the current trajectory of oncology research and reflects broader notions of value. 24 experts participated in consensus-building activities across 5 months (16 took part in structured interactions, including a survey, plenary sessions, interviews, and off-line discussions, while 8 participated in interviews). Discussion focused on: 1) which oncology-relevant endpoints should be used for assessing treatments for early-stage cancer and access decisions for early-stage treatments, and 2) the importance of additional value components and how these can be integrated in value assessments. The expert group reached consensus on 4 principles in relation to the first area (consider oncology-relevant endpoints other than overall survival; build evidence for endpoints that provide earlier indication of efficacy; develop evidence for the next generation of predictive measures; use managed entry agreements supported by ongoing evidence collection to address decision-maker evidence needs) and 3 principles in relation to the second (routinely use patient reported outcomes in value assessments; assess broad economic impact of new medicines; consider other value aspects of relevance to patients and society).

Opportunities to improve the adoption of health-related quality of life evidence as part of the French Health Technology Assessment process.

OBJECTIVES: Patient's health-related quality of life (HRQoL) is an important outcome measure that is considered by many payers and health technology assessment (HTA) bodies in the evaluation of treatments. We aimed to identify opportunities for HRQoL to be further incorporated into the assessment of the French HTA by comparing three health systems. We put forward recommendations that could bring further innovations to French patients. METHODS: We reviewed methodologies by the French, German and British HTA, and conducted a systematic review of all French (n = 312) and German (n = 175) HTA appraisals from 01 January 2019 to 31 December 2021. We also setup an advisory board of 11 ex-HTA leaders, payers, methodologists, healthcare providers and patient advocates, from France, Britain and Germany, to discuss opportunities to improve acceptance and adoption of HRQoL evidence in France. RESULTS: Our systematic review of HTA appraisals showed a higher HRQoL data rejection rate in France: in > 75% of cases the HRQoL evidence submitted was not accepted for the assessment (usually for methodological reasons, for example, data being considered exploratory; 16-75% of the appraisals mentioned HRQoL evidence, varying by therapeutic area). Overall, we found the French HTA to be more restrictive in its approach than IQWiG. CONCLUSIONS: Based on these findings we articulate collaborative proposals for industry and the HAS to improve acceptance of HRQoL evidence and create a positive feedback loop between HAS and industry along four dimensions (1) patient perception, (2) testing hierarchy, (3) trial design and (4) data collection.