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Cost-Effectiveness Analysis Alongside Clinical Trials II—An ISPOR Good Research Practices Task Force Report
Open accessAbstract
Clinical trials evaluating medicines, medical devices, and procedures now commonly assess the economic value of these interventions. The growing number of prospective clinical/economic trials reflects both widespread interest in economic information for new technologies and the regulatory and reimbursement requirements of many countries that now consider evidence of economic value along with clinical efficacy. As decision makers increasingly demand evidence of economic value for health care interventions, conducting high-quality economic analyses alongside clinical studies is desirable because they broaden the scope of information available on a particular intervention, and can efficiently provide timely information with high internal and, when designed and analyzed properly, reasonable external validity. In 2005, ISPOR published the Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials: The ISPOR RCT-CEA Task Force report. ISPOR initiated an update of the report in 2014 to include the methodological developments over the last 9 years. This report provides updated recommendations reflecting advances in several areas related to trial design, selecting data elements, database design and management, analysis, and reporting of results. Task force members note that trials should be designed to evaluate effectiveness (rather than efficacy) when possible, should include clinical outcome measures, and should obtain health resource use and health state utilities directly from study subjects. Collection of economic data should be fully integrated into the study. An incremental analysis should be conducted with an intention-to-treat approach, complemented by relevant subgroup analyses. Uncertainty should be characterized. Articles should adhere to established standards for reporting results of cost-effectiveness analyses. Economic studies alongside trials are complementary to other evaluations (e.g., modeling studies) as information for decision makers who consider evidence of economic value along with clinical efficacy when making resource allocation decisions.
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“The economic evaluation will be performed from a societal perspective, and all analyses will be conducted according to the intention-to-treat principle, in which we adhere to the related guidelines [67–69].”
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“For example, parameter uncertainty will be covered by, for example, sensitivity analyses in which imputed uncertainty is also taken into account by repeating the economic evaluation on the imputed dataset and sampling uncertainty will be taken into account by providing a cost-effectiveness acceptability curve and frontier [67].”
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“Best practice within-trial economic evaluation methods will be adopted53”
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Self-citationEdwards, K., Jones, N., Newton, J. L., Foster, C. E., Judge, A., Jackson, K., Arden, N., & Pinedo‐Villanueva, R. (2017) · Health Economics Review · doi:10.1186/s13561-017-0173-3 · Cited by 49
The Current Status of EMDR Therapy, Specific Target Areas, and Goals for the Future
Matthijssen, S. J. M. A., Lee, C. W., de Roos, C., Barron, I. G., Jarero, I., Shapiro, E., Hurley, E. C., Schubert, S. J., Baptist, J., Amann, B. L., et al. (2020) · Journal of EMDR Practice and Research · doi:10.1891/emdr-d-20-00039 · Cited by 48
“Future research into the cost-effectiveness of EMDR should be guided by expert guidelines in this area such as CHEERS (Husereau et al., 2013) or the ISPOR Good Research Practices Task Force Report (Ramsey et al., 2015).”
Conducting Trial-Based Economic Evaluations Using R: A Tutorial
Ben, Â. J., van Dongen, J. M., Alili, M. E., Eßer, J., Broulíková, H. M., & Bosmans, J. E. (2023) · PharmacoEconomics · doi:10.1007/s40273-023-01301-7 · Cited by 48
Campins, L., Serra‐Prat, M., Palomera, E., Bolíbar, I., Martínez, M. À., & Gallo, P. (2017) · Gaceta Sanitaria · doi:10.1016/j.gaceta.2017.09.002 · Cited by 47
Mangalore, R. P., Peel, T. N., Udy, A. A., & Peleg, A. Y. (2023) · Journal of Antimicrobial Chemotherapy · doi:10.1093/jac/dkad223 · Cited by 49
Dehmer, S. P., Maciosek, M. V., Trower, N. K., Asche, S. E., Bergdall, A. R., Nyboer, R. A., O’Connor, P. J., Pawloski, P. A., Sperl‐Hillen, J. M., Green, B. B., et al. (2018) · JACCP JOURNAL OF THE AMERICAN COLLEGE OF CLINICAL PHARMACY · doi:10.1002/jac5.1001 · Cited by 47
Hughes, D. A., Charles, J. M., Dawoud, D., Edwards, R. T., Holmes, E. A. F., Jones, C., Parham, P. E., Plumpton, C., Ridyard, C. H., Lloyd‐Williams, H., et al. (2016) · PharmacoEconomics · doi:10.1007/s40273-015-0371-y · Cited by 45
“Approaches to trial-based economic evaluation have consequently evolved, as detailed in recent guidelines and reviews [2-4].”
König, H., Bleibler, F., Friederich, H., Herpertz, S., Lam, T., Mayr, A. J., Schmidt, F., Svaldi, J., Zipfel, S., Brettschneider, C., et al. (2018) · International Journal of Eating Disorders · doi:10.1002/eat.22822 · Cited by 45
“The recommended perspective for economic evaluations is the societal perspective wherein possibly all kinds of resource use are considered (Ramsey et al., 2015).”
BackgroundLung, T. W.-C., Marks, G. B., Nhung, N. V., Nguyen, T. A., Hoa, N. L. P., Anh, L. T. N., Hoa, N. B., Britton, W. J., Bestrashniy, J., Jan, S., et al. (2019) · The Lancet Global Health · doi:10.1016/s2214-109x(18)30520-5 · Cited by 45
van Dongen, J. M., Persoon, S., Jongeneel, G., Bosmans, J. E., Kersten, M. J., Brug, J., Nollet, F., Chinapaw, M. J. M., & Buffart, L. M. (2019) · Journal of Cancer Survivorship · doi:10.1007/s11764-019-00775-9 · Cited by 45
“This is due to differences in the applied statistical methods, including (1) the use of mixed effect models in the effectiveness analyses, which are typically not applied in cost-effectiveness analyses, (2) multiple imputation, which is recommended for imputing missing cost data [26] versus maximum likelihood estimati…”
MethodThe DISCHARGE Trial Group, Napp, A. E., Haase, R., Laule, M., Schuetz, G. M., Rief, M., Dreger, H., Feuchtner, G. M., Friedrich, G. J., Špaček, M., et al. (2016) · European Radiology · doi:10.1007/s00330-016-4620-z · Cited by 44
Snoek, A., Beekman, A. T. F., Dekker, J., Aarts, I., van Grootheest, G., Blankers, M., Vriend, C., van den Heuvel, O., & Thomaes, K. (2020) · BMC Psychiatry · doi:10.1186/s12888-020-02713-x · Cited by 44
“We will take into account the CHEERS statement [92] and the 2015 ISPOR good research practices task force report on cost-effectiveness analysis alongside clinical trials [93].”
MethodSchaub, M. P., Blankers, M., Lehr, D., Boß, L., Riper, H. M., Dekker, J., Goudriaan, A. E., Maier, L. J., Haug, S., Amann, M., et al. (2016) · BMJ Open · doi:10.1136/bmjopen-2016-011457 · Cited by 46
“Cost-effectiveness calculations The economic evaluation will be conducted alongside the randomised trial, taking into account the CHEERS statement(66) and the 2015 ISPOR good research practices task force report on cost-effectiveness analysis alongside clinical trials.(67) From the technical point of view, the economi…”
MethodOoms, P., Blankers, M., Figee, M., Bergfeld, I. O., van den Munckhof, P., Schuurman, P. R., & Denys, D. A. J. P. (2017) · Brain stimulation · doi:10.1016/j.brs.2017.04.120 · Cited by 44
Dakin, H., & Gray, A. M. (2017) · Statistics in Medicine · doi:10.1002/sim.7322 · Cited by 43
“172); given that most two-arm trials are not powered to detect significant differences in mean costs or NMB [4,62] and the variance around the interaction term is four-fold higher than that for main effects [17,23], this finding is likely to be common among economic evaluations of factorial trials.”
BackgroundMethodInfluentialBatchelor, J., Millington, A., Thomas, K. S., Akram, P., Azad, J., Bewley, A., Chalmers, J. R., Cheung, S. T., Duley, L., Eleftheriadou, V., et al. (2020) · Health Technology Assessment · doi:10.3310/hta24640 · Cited by 44
“Number unblinded, n (%) 45 (39) 66 (55) 55 (44)”
Feliu‐Soler, A., Borràs, X., Peñarrubia‐María, M. T., Rozadilla‐Sacanell, A., D’Amico, F., Moss-Morris, R. E., Howard, M. A., Fayed, N. M., Soriano‐Mas, C., Puebla‐Guedea, M., et al. (2016) · BMC Complementary and Alternative Medicine · doi:10.1186/s12906-016-1068-2 · Cited by 46
“Gotink RA, Chu P, Busschbach JJ, Benson H, Fricchione GL, Hunink MG. Standardised mindfulness-based interventions in healthcare: an overview of systematic reviews and meta-analyses of RCTs. PLoS ONE.”
MethodInfluentialThomas, K. S., Bradshaw, L. E., Sach, T., Cowdell, F., Batchelor, J., Lawton, S., Harrison, E., Haines, R., Ahmed, A. I., Dean, T., et al. (2017) · Health Technology Assessment · doi:10.3310/hta21160 · Cited by 43
“ICERs will be calculated using accepted methodology (Ramsey et al 2015, Drummond et al 2005).”
MethodFor the EVINCI Investigators, Lorenzoni, V., Bellelli, S., Caselli, C., Knuuti, J. M., Underwood, S. R., Neglia, D., & Turchetti, G. (2019) · The European Journal of Health Economics · doi:10.1007/s10198-019-01096-5 · Cited by 42
Alili, M. E., van Dongen, J. M., Eßer, J., Heymans, M. W., van Tulder, M. W., & Bosmans, J. E. (2022) · Health Economics · doi:10.1002/hec.4603 · Cited by 41
“Over the last decades, various recommendations have been made on how to analyze trial-based economic evaluations (Petrou & Gray, 2011; Ramsey et al., 2015; van Dongen et al., 2014).”
BackgroundMethodCost-effectiveness of home-based cardiac rehabilitation: a systematic review
Shields, G., Rowlandson, A., Dalal, G., Nickerson, S., Cranmer, H., Capobianco, L., & Doherty, P. (2023) · Heart · doi:10.1136/heartjnl-2021-320459 · Cited by 41
Luciano, J. V., D'Amico, F., Feliu-Soler, A., McCracken, L. M., Aguado, J., Peñarrubia-María, M. T., Knapp, M., Serrano-Blanco, A., & García-Campayo, J. (2017) · The Journal of Pain · doi:10.1016/j.jpain.2017.03.001 · Cited by 42
Kunst, N., Siu, A., Drummond, M., Grimm, S. E., Grutters, J. P. C., Husereau, D., Koffijberg, H., Rothery, C., Wilson, E., & Heath, A. (2023) · Value in Health · doi:10.1016/j.jval.2023.06.014 · Cited by 40
Berdahl, J. P., Khatana, A. K., Katz, L. J., Herndon, L. W., Layton, A. J., Yu, T. M., Bauer, M. J., & Cantor, L. B. (2017) · Journal of Medical Economics · doi:10.1080/13696998.2017.1327439 · Cited by 40
Gyamfi‐Bannerman, C., Zupancic, J. A. F., Sandoval, G. J., Grobman, W. A., Blackwell, S. C., Tita, A., Reddy, U. M., Jain, L., Saade, G. R., Rouse, D. J., et al. (2019) · Archives of Pediatrics and Adolescent Medicine · doi:10.1001/jamapediatrics.2019.0032 · Cited by 38
Cost-effectiveness of adherence-enhancing interventions: a systematic review
Simon‐Tuval, T., Neumann, P. J., & Greenberg, D. (2016) · Expert Review of Pharmacoeconomics & Outcomes Research · doi:10.1586/14737167.2016.1138858 · Cited by 38
“An ideal comparator is one that represents the best current or most common practice (ideally both).[66] The most common practice does not include any educational communication, thus it was reasonable to choose usual care as a comparator and conclude that these counseling strategies are cost-effective; however, it may…”
BackgroundHernández-Tejedor, A., Peñuelas, Ó., Sirgo, G., Llompart-Pou, J. A., Herrejon, E. P., Estella, Á., Cabanes, M. P. F., Alcalá-Llorente, M. A., Galleymore, P. R., Azuara, B. O., et al. (2017) · Medicina Intensiva (English Edition) · doi:10.1016/j.medine.2017.03.008 · Cited by 37
Paniagua-Ávila, A., Fort, M. P., Glasgow, R. E., Gulayin, P., Hernández-Galdamez, D. R., Mansilla, K., Palacios, E., Peralta-García, A., Roche, D., Rubinstein, A., et al. (2020) · Trials · doi:10.1186/s13063-020-04345-8 · Cited by 39
“We will analyze differences in costs following a similar analytical approach as that used for estimating health outcomes [32].”
MethodSathish, T., Oldenburg, B. F., Thankappan, K. R., Absetz, P., Shaw, J. E., Tapp, R. J., Zimmet, P., Balachandran, S., Shetty, S. S., Aziz, Z., et al. (2020) · BMC Medicine · doi:10.1186/s12916-020-01704-9 · Cited by 40
“We estimated costs from the health system and societal perspectives, in accordance with the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement [23] and cost-effectiveness analysis guidelines [24, 25].”
BackgroundMethodInfluentialAlshamsi, S., Hamidi, S., & Narcı, H. Ö. (2022) · BMC Health Services Research · doi:10.1186/s12913-022-07663-6 · Cited by 38
A Review of Clinical Trials With an Adaptive Design and Health Economic Analysis
Flight, L., Arshad, F., Barnsley, R., Patel, K., Julious, S. A., Brennan, A., & Todd, S. (2019) · Value in Health · doi:10.1016/j.jval.2018.11.008 · Cited by 36
Gomez-Rossi, J., Feldberg, B., Krois, J., & Schwendicke, F. (2022) · JMIR Medical Informatics · doi:10.2196/33703 · Cited by 39
Hurley, D. A., Hall, A. M., Murphy, L., Pincus, T., Kamper, S. J., Maher, C. G., McDonough, S., Lonsdale, C., Walsh, N. E., Guérin, S., et al. (2016) · BMJ Open · doi:10.1136/bmjopen-2015-010728 · Cited by 37
“Finally, the cost of the SOLAS intervention compared with usual PT will be calculated from the health service and societal perspectives using data from resource utilisation measures, alongside exploratory analysis of quality-adjusted life years (EQ-5D) and disease-specific measures.(85)”
Method
Metadata
- Authors
- Ramsey, S. D., Willke, R. J., Glick, H., Reed, S. D., Augustovski, F., Jonsson, B., Briggs, A., & Sullivan, S. D.
- Affiliations
- University of Washington (US)
- Pfizer (United States) (US)
- University of Pennsylvania (US)
- Duke University (US)
- Universidad de Buenos Aires (AR)
- University of Glasgow (GB)
- Corresponding author
- Ramsey, Scott D.
- ORCID
- Venue
- Value in Health
- Publisher
- Elsevier BV
- Published
- 2015-03
- Volume
- 18
- Issue
- 2
- Pages
- 161-172
- ISSN
- 1098-3015 (ISSN-L)
- OpenAlex
- W2139814250
- Cited by
- 811
- References
- 184
- Citations by year
- 2026: 58 · 2025: 75 · 2024: 90 · 2023: 80 · 2022: 100 · 2021: 103 · 2020: 88 · 2019: 65 · 2018: 50 · 2017: 57 · 2016: 37 · 2015: 6
- FWCI
- 17.59
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Value in Health (publishedVersion)
- PubMed (publishedVersion)
- ENLIGHTEN (Jurnal Bimbingan dan Konseling Islam) (submittedVersion)
- Topics
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Health Systems, Economic Evaluations, Quality of Life
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Economic and Environmental Valuation
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
- Keywords
- cost-effectiveness analysis, clinical trials, economic evaluation, trial design, health care utilization, health utility, intention-to-treat analysis, subgroup analysis, uncertainty characterization, reporting guidelines, reimbursement decisions, healthcare resource allocation
- MeSH terms
- Cost-Benefit Analysis, Health Policy, Health Services Research, Humans, Randomized Controlled Trials as Topic, Advisory Committees, Research Report
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