The Role of Gene and Cell Therapy in the Era of Health Care Reform

Ettinger, Walter H.; Flotte, Terence R.

doi:10.1089/hum.2011.2526

Cited by 3 publications

(5 citation statements)

References 3 publications

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“…Payment and billing in healthcare systems is generally organized to occur at the same time treatment is provided. 11,13 For chronic treatments this results in a longitudinal and predictable spending pattern, which allows payers to plan budgets and spending. 37 Additionally, if a treatment is deemed ineffective, a treatment can be stopped, and payment is discontinued.…”

Section: Payment Modelsmentioning

confidence: 99%

“…6 Authors reason that GTs address highburden disease, which also affects relatives and caregivers. 6,13,37 Treatment with GTs may result in (re)introduction of these patients and their caregivers to the workforce, which could generate tax income and (informal) care savings. Drummond et al and Ettinger et al propose structural inclusion of these costs to reflect this greater economic impact.…”

Section: (Re)definition Of Perspectivesmentioning

confidence: 99%

“…Drummond et al and Ettinger et al propose structural inclusion of these costs to reflect this greater economic impact. 6,13 Carr et al advocates to, besides cost of treatment, also include personal, social, and economic benefits but does not specify what exactly this entails. 37 Raymakers et al takes it a step further and proposes inclusion of infrastructure and capital cost in evaluations.…”

Section: (Re)definition Of Perspectivesmentioning

confidence: 99%

“…Since publication of the NICE mock appraisal, more methodological elements for incorporation in the economical evaluations of GTs were discussed in literature. 6,[8][9][10][11][12][13] The importance of EEs in healthcare decision making is well recognized. 14 Although individual jurisdictions might weigh the results of the evaluations differently, the requirement by authorities to include EEs in assessments is increasing.…”

Section: Introductionmentioning

confidence: 99%

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A Review of Methodological Considerations for Economic Evaluations of Gene Therapies and Their Application in Literature

et al. 2020

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Objectives: To identify methodological considerations discussed in literature addressing economic evaluations (EEs) of gene therapies (GTs). Additionally, we assessed if these considerations are applied in published GT EEs to increase understanding and explore impact.Methods: First a peer-reviewed literature review was performed to identify research addressing methodological considerations of GT EEs until August 2019. Identified considerations were grouped in themes using thematic content analysis. A second literature search was conducted in which we identified published evaluations. The EE quality of reporting was assessed using Consolidated Health Economic Evaluation Reporting Standards.Results: The first literature search yielded 13 articles discussing methodological considerations. The second search provided 12 EEs. Considerations identified were payment models, definition of perspectives, addressing uncertainty, data extrapolation, discount rates, novel value elements, and use of indirect and surrogate endpoints. All EEs scored satisfactory to good according to Consolidated Health Economic Evaluation Reporting Standards. Regarding methodological application, we found 1 methodological element (payment models) was applied in 2 base cases. Scenarios explored alternative perspectives, survival assumptions, and extrapolation methods in 10 EEs.Conclusions: Although EE quality of reporting was considered good, their informativeness for health technology assessment and decision makers seemed limited owing to many uncertainties. We suggest accepted EE methods can broadly be applied to GTs, but few elements may need adjustment. Further research and multi-stakeholder consensus is needed to determine appropriateness and application of individual methodological considerations. For now, we recommend including scenario analyses to explore impact of methodological choices and (clinical) uncertainties. This study contributes to better understanding of perceived appropriate evaluation of GTs and informs best modeling practices.

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Section: Payment Modelsmentioning

confidence: 99%

Section: (Re)definition Of Perspectivesmentioning

confidence: 99%

Section: (Re)definition Of Perspectivesmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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A Review of Methodological Considerations for Economic Evaluations of Gene Therapies and Their Application in Literature

et al. 2020

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“…This is new, uncharted territory, and it will take a multifaceted force of scientists, clinicians, insurers, business people, and patient advocacy groups to generate an operational map forward. [1][2][3] As President of the ASGCT, I will place a major focus on fostering the delivery of gene and cell therapy to the patient, including education, advocacy, and public awareness of the issues. I will start by organizing a commercialization workshop to be scheduled for 2013.…”

mentioning

confidence: 99%

The ASGCT in 2012: Charting a Path to Bring Novel Biologic Therapies to the Clinic

Breakefield¹

2012

Molecular Therapy

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Characterization and comparative performance of lentiviral vector preparations concentrated by either one-step ultrafiltration or ultracentrifugation

et al. 2013

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The Role of Gene and Cell Therapy in the Era of Health Care Reform

Cited by 3 publications

References 3 publications

A Review of Methodological Considerations for Economic Evaluations of Gene Therapies and Their Application in Literature

A Review of Methodological Considerations for Economic Evaluations of Gene Therapies and Their Application in Literature

The ASGCT in 2012: Charting a Path to Bring Novel Biologic Therapies to the Clinic

Characterization and comparative performance of lentiviral vector preparations concentrated by either one-step ultrafiltration or ultracentrifugation

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