An Alternative Measure of Health for Value Assessment: The Equal Value Life-Year

Campbell, Jonathan D.; Whittington, Melanie D; Pearson, Steven D.

doi:10.1007/s40273-023-01302-6

Cited by 5 publications

(3 citation statements)

References 13 publications

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“…Additional health outcomes included cumulative lifetime VOCs (the most frequent of the events in the HGB-206 VOE protocol definition) and development of chronic complications. We also estimated the equal value life-year (evLY) and health years in total (HYT) outcomes as alternatives to the QALY outcome [ 66 , 67 ].…”

Section: Methodsmentioning

confidence: 99%

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

Herring,

Gallagher,

Shah

et al. 2024

PharmacoEconomics

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Background and Objective Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥ 12 years with ≥ 4 vaso-occlusive events (VOEs) in the past 24 months. Methods We developed a patient-level simulation model accounting for lovo-cel and SCD-related events, complications, and mortality over a lifetime time horizon. The pivotal phase 1/2 HGB-206 clinical trial (NCT02140554) served as the basis for lovo-cel efficacy and safety. Cost, quality-of-life, and other clinical data were sourced from HGB-206 data and the literature. Analyses were conducted from US societal and third-party payer perspectives. Uncertainty was assessed through probabilistic sensitivity analysis and extensive scenario analyses. Results Patients treated with lovo-cel were predicted to survive 23.84 years longer on average (standard deviation [SD], 12.80) versus common care (life expectancy, 62.24 versus 38.40 years), with associated discounted patient quality-adjusted life-year (QALY) gains of 10.20 (SD, 4.10) and direct costs avoided of $1,329,201 (SD, $1,346,446) per patient. Predicted societal benefits included discounted caregiver QALY losses avoided of 1.19 (SD, 1.38) and indirect costs avoided of $540,416 (SD, $262,353) per patient. Including lovo-cel costs ($3,282,009 [SD, $29,690] per patient) resulted in incremental cost-effectiveness ratios of $191,519 and $124,051 per QALY gained from third-party payer and societal perspectives, respectively. In scenario analyses, the predicted cost-effectiveness of lovo-cel also was sensitive to baseline age and VOE frequency and to the proportion of patients achieving and maintaining complete resolution of VOEs. Conclusions Our analysis of lovo-cel gene therapy compared with common care for patients in the US with SCD with recurrent VOEs estimated meaningful improvements in survival, quality of life, and other clinical outcomes accompanied by increased overall costs for the health care system and for broader society. The predicted economic value of lovo-cel gene therapy was influenced by uncertainty in long-term clinical effects and by positive spillover effects on patient productivity and caregiver burden. Supplementary Information The online version contains supplementary material available at 10.1007/s40273-024-01385-9.

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Section: Methodsmentioning

confidence: 99%

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

Herring,

Gallagher,

Shah

et al. 2024

PharmacoEconomics

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“…Different governments may use different strategies in deciding how to approach high drug costs, and various "value calculators" have been devised [76,77]. Each of the current methods of calculating value has limitations [76,[78][79][80][81].…”

Section: Controlling Drug Pricesmentioning

confidence: 99%

New Anticancer Drugs: Reliably Assessing “Value” While Addressing High Prices

Stewart,

Bradford,

Sehdev

et al. 2024

Current Oncology

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Countries face challenges in paying for new drugs. High prices are driven in part by exploding drug development costs, which, in turn, are driven by essential but excessive regulation. Burdensome regulation also delays drug development, and this can translate into thousands of life-years lost. We need system-wide reform that will enable less expensive, faster drug development. The speed with which COVID-19 vaccines and AIDS therapies were developed indicates this is possible if governments prioritize it. Countries also differ in how they value drugs, and generally, those willing to pay more have better, faster access. Canada is used as an example to illustrate how “incremental cost-effectiveness ratios” (ICERs) based on measures such as gains in “quality-adjusted life-years” (QALYs) may be used to determine a drug’s value but are often problematic, imprecise assessments. Generally, ICER/QALY estimates inadequately consider the impact of patient crossover or long post-progression survival, therapy benefits in distinct subpopulations, positive impacts of the therapy on other healthcare or societal costs, how much governments willingly might pay for other things, etc. Furthermore, a QALY value should be higher for a lethal or uncommon disease than for a common, nonlethal disease. Compared to international comparators, Canada is particularly ineffective in initiating public funding for essential new medications. Addressing these disparities demands urgent reform.

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“…To inform these deliberations, analysts should present a range of scenario analyses (with and without carer QOL, and varying any relevant assumptions), and it may be useful if disaggregated results are also presented. Furthermore, alternative measures of value developed to address the classic QALY trap, such as equal value of life-years [17,18] or healthy years in total [19], may be a useful supplement for illustrating the value of life extension whilst also allowing independent consideration of QOL impacts. Deliberations could also be supplemented with carer testimonials and data on the carer burden collected using rigorous qualitative and/ or quantitative scientific methods.…”

Section: If the Carer Qaly Trap Is A Problem Whatmentioning

confidence: 99%

Modelling Spillover Effects on Informal Carers: The Carer QALY Trap

Mott,

Schirrmacher,

Al-Janabi

et al. 2023

PharmacoEconomics

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The provision of informal (unpaid) care can impose significant 'spillover effects' on carers, and accounting for these effects is consistent with the efficiency and equity objectives of health technology assessment (HTA). Inclusion of these effects in health economic models, particularly carer health-related quality of life (QOL), can have a substantial impact on net quality-adjusted life year (QALY) gains and the relative cost effectiveness of new technologies. Typically, consideration of spillover effects improves the value of a technology, but in some circumstances, consideration of spillover effects can lead to situations whereby life-extending treatments for patients may be considered cost ineffective due to their impact on carer QOL. In this piece we revisit the classic 'QALY trap' and introduce an analogous 'carer QALY trap' which may have practical implications for economic evaluations where the inclusion of carer QOL reduces incremental QALY gains. Such results may align with a strict QALY-maximisation rule, however we consider the extent to which this principle may be at odds with the preferences of carers themselves (and possibly society more broadly), potentially leading decision makers into the carer QALY trap as a result. We subsequently reflect on potential solutions, highlighting the important (albeit limited) role that deliberation has to play in HTA.

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An Alternative Measure of Health for Value Assessment: The Equal Value Life-Year

Cited by 5 publications

References 13 publications

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

New Anticancer Drugs: Reliably Assessing “Value” While Addressing High Prices

Modelling Spillover Effects on Informal Carers: The Carer QALY Trap

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