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Putting Stakeholder Engagement at the Center of Health Economic Modeling for Health Technology Assessment in the United States.

ABSTRACT: While evidence generated from health economic (HE) models is being used more commonly in health technology assessment (HTA) in the US, it is not consistently adopted by different stakeholder groups or across therapeutic areas. We hypothesize that actively engaging with multiple stakeholder groups throughout the model development process may result in models more widely considered by decision makers. To test this hypothesis, the Innovation and Value Initiative has launched a modeling effort to build an open-source HE model focusing on the disease state 'major depressive disorder'. A 20-member advisory group has been formed with representatives from patients, employers, clinicians, innovators, payers, and researchers to guide the model development process. While this effort is still in the early stages, the ongoing stakeholder engagement effort has yielded valuable insights that inform the model design. We have also identified several challenges to implementing this new approach. Our early findings suggest that the stakeholder engagement approach to HE model development has the potential to improve HTA in the US.

SUBMITTER: Xie RZ

PROVIDER: S-EPMC8166701 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature

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Putting Stakeholder Engagement at the Center of Health Economic Modeling for Health Technology Assessment in the United States.

Xie Richard Z RZ Malik Erica deFur ED Linthicum Mark T MT Bright Jennifer L JL

PharmacoEconomics 20210513 6

While evidence generated from health economic (HE) models is being used more commonly in health technology assessment (HTA) in the US, it is not consistently adopted by different stakeholder groups or across therapeutic areas. We hypothesize that actively engaging with multiple stakeholder groups throughout the model development process may result in models more widely considered by decision makers. To test this hypothesis, the Innovation and Value Initiative has launched a modeling effort to bu ...[more]

PMID: 33982198

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Project description:BackgroundThe cost-effectiveness of prophylactic vaccination against human papillomavirus types 16 (HPV-16) and 18 (HPV-18) is an important consideration for guidelines for immunization in the United States.MethodsWe synthesized epidemiologic and demographic data using models of HPV-16 and HPV-18 transmission and cervical carcinogenesis to compare the health and economic outcomes of vaccinating preadolescent girls (at 12 years of age) and vaccinating older girls and women in catch-up programs (to 18, 21, or 26 years of age). We examined the health benefits of averting other HPV-16-related and HPV-18-related cancers, the prevention of HPV-6-related and HPV-11-related genital warts and juvenile-onset recurrent respiratory papillomatosis by means of the quadrivalent vaccine, the duration of immunity, and future screening practices.ResultsOn the assumption that the vaccine provided lifelong immunity, the cost-effectiveness ratio of vaccination of 12-year-old girls was $43,600 per quality-adjusted life-year (QALY) gained, as compared with the current screening practice. Under baseline assumptions, the cost-effectiveness ratio for extending a temporary catch-up program for girls to 18 years of age was $97,300 per QALY; the cost of extending vaccination of girls and women to the age of 21 years was $120,400 per QALY, and the cost for extension to the age of 26 years was $152,700 per QALY. The results were sensitive to the duration of vaccine-induced immunity; if immunity waned after 10 years, the cost of vaccination of preadolescent girls exceeded $140,000 per QALY, and catch-up strategies were less cost-effective than screening alone. The cost-effectiveness ratios for vaccination strategies were more favorable if the benefits of averting other health conditions were included or if screening was delayed and performed at less frequent intervals and with more sensitive tests; they were less favorable if vaccinated girls were preferentially screened more frequently in adulthood.ConclusionsThe cost-effectiveness of HPV vaccination will depend on the duration of vaccine immunity and will be optimized by achieving high coverage in preadolescent girls, targeting initial catch-up efforts to women up to 18 or 21 years of age, and revising screening policies.

Project description:Key Points Median annual direct costs (including medication, diet, emergency room visits, hospitalizations, and clinic visits) were $3464 (interquartile range [IQR] $844–$5865) for adult patients and $1687 (IQR $1035–$4763) for caregivers. The time spent planning/prepping meals was 183 h/yr (IQR 114–331) for adults and 173 h/yr (IQR 84–205) for caregivers. Providers can better understand the burden of living with nephrotic syndrome, consider barriers when treating patients, and develop supportive strategies. Visual Abstract Background Nephrotic syndrome (NS) is a rare kidney syndrome with high morbidity. Although a common contributor to the burden of chronic kidney disease, the direct and indirect costs of NS to patients and family caregivers are unrecognized. The objective was to characterize the direct and indirect costs of NS to patients. Methods Adults with NS and family caregivers of children with NS were eligible to participate if they had a diagnosis of primary NS, had disease for at least 1 year, and had no other severe health conditions. Data-collection surveys were generated with input from the Kidney Research Network Patient Advisory Board, and surveys were mailed to the eligible participants. Participants were provided $50 for the return of completed surveys. Costs were defined as either direct out-of-pocket costs or indirect costs (e.g., time). Descriptive statistics, including percentage and median (interquartile range [IQR]) are reported. Results Respondents included 28 adult patients and 17 caregivers of patients who were minors. Reported health insurance coverage included 35 (78%) with private insurance, 12 (27%) with public insurance, six (13%) with Children’s Special Health Care Services, and one (2%) uninsured. Median annual direct costs were $3464 ($844–$5865) for adult patients and $1687 (IQR $1035–$4763) for caregivers. Of these costs, diet-associated costs contributed $1140 (IQR $600–$2400). The most substantial indirect cost was from the time spent planning/prepping meals (adults: 183 h/yr [IQR 114–331]; caregivers: 173 h/yr [IQR 84–205]). Conclusions Adults and caregivers of children with NS face substantial disease-related direct and indirect costs beyond those covered by insurance. Following replication, the study will help health care providers, systems, and payers gain a better understanding of the financial and time burden incurred by those living with NS, consider barriers when treating patients, and develop supportive strategies.

Dataset Information

Putting Stakeholder Engagement at the Center of Health Economic Modeling for Health Technology Assessment in the United States.

Publications

Putting Stakeholder Engagement at the Center of Health Economic Modeling for Health Technology Assessment in the United States.

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