Details
| Original language | English |
|---|---|
| Pages (from-to) | 483-497 |
| Number of pages | 15 |
| Journal | Expert Review of Pharmacoeconomics and Outcomes Research |
| Volume | 23 |
| Issue number | 5 |
| Early online date | 19 Apr 2023 |
| Publication status | Published - 2023 |
Abstract
Objective: No consensus exists on the ideal methodology to evaluate the economic impact and value of new, potentially curative gene therapies. We aimed to identify and describe published methodologic recommendations for the economic evaluation of gene therapies and assess whether these recommendations have been applied in published evaluations. Methods: This study was conducted in three stages: a systematic literature review of methodologic recommendations for economic evaluation of gene therapies; an assessment of the appropriateness of recommendations; and a review to assess the degree to which the recommendations were applied in published evaluations. Results: A total of 2,888 references were screened, 83 articles were reviewed to assess eligibility, and 20 papers were included. Fifty recommendations were identified, and 21 reached consensus thresholds. Most evaluations were based on naive treatment comparisons and did not apply consensus recommendations. Innovative payment mechanisms for gene therapies were rarely considered. The only widely applied recommendations related to modeling choices and methods. Conclusions: Methodological recommendations for economic evaluations of gene therapies are generally not being followed. Assessing the applicability and impact of the recommendations from this study may facilitate the implementation of consensus recommendations in future evaluations.
Keywords
- Cost-effectiveness analysis, economic evaluation, gene therapy, health technology assessments, health-related quality of life, onasemnogene abeparvovec, spinal muscular atrophy, systematic literature review
ASJC Scopus subject areas
- Medicine(all)
- Health Policy
- Medicine(all)
- Pharmacology (medical)
Sustainable Development Goals
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In: Expert Review of Pharmacoeconomics and Outcomes Research, Vol. 23, No. 5, 2023, p. 483-497.
Research output: Contribution to journal › Review article › Research › peer review
}
TY - JOUR
T1 - Recommendations for economic evaluations of cell and gene therapies
T2 - a systematic literature review with critical appraisal
AU - Toumi, Mondher
AU - Dabbous, Omar
AU - Aballéa, Samuel
AU - Drummond, Michael F.
AU - von der Schulenburg, Johann Matthias Graf
AU - Malone, Daniel C.
AU - Neumann, Peter J.
AU - Sullivan, Sean D.
AU - Tunis, Sean
N1 - Funding Information: Editorial support was provided by Wynne Dillon, MS, of Kay Square Scientific. This support was funded by Novartis Gene Therapies, Inc.
PY - 2023
Y1 - 2023
N2 - Objective: No consensus exists on the ideal methodology to evaluate the economic impact and value of new, potentially curative gene therapies. We aimed to identify and describe published methodologic recommendations for the economic evaluation of gene therapies and assess whether these recommendations have been applied in published evaluations. Methods: This study was conducted in three stages: a systematic literature review of methodologic recommendations for economic evaluation of gene therapies; an assessment of the appropriateness of recommendations; and a review to assess the degree to which the recommendations were applied in published evaluations. Results: A total of 2,888 references were screened, 83 articles were reviewed to assess eligibility, and 20 papers were included. Fifty recommendations were identified, and 21 reached consensus thresholds. Most evaluations were based on naive treatment comparisons and did not apply consensus recommendations. Innovative payment mechanisms for gene therapies were rarely considered. The only widely applied recommendations related to modeling choices and methods. Conclusions: Methodological recommendations for economic evaluations of gene therapies are generally not being followed. Assessing the applicability and impact of the recommendations from this study may facilitate the implementation of consensus recommendations in future evaluations.
AB - Objective: No consensus exists on the ideal methodology to evaluate the economic impact and value of new, potentially curative gene therapies. We aimed to identify and describe published methodologic recommendations for the economic evaluation of gene therapies and assess whether these recommendations have been applied in published evaluations. Methods: This study was conducted in three stages: a systematic literature review of methodologic recommendations for economic evaluation of gene therapies; an assessment of the appropriateness of recommendations; and a review to assess the degree to which the recommendations were applied in published evaluations. Results: A total of 2,888 references were screened, 83 articles were reviewed to assess eligibility, and 20 papers were included. Fifty recommendations were identified, and 21 reached consensus thresholds. Most evaluations were based on naive treatment comparisons and did not apply consensus recommendations. Innovative payment mechanisms for gene therapies were rarely considered. The only widely applied recommendations related to modeling choices and methods. Conclusions: Methodological recommendations for economic evaluations of gene therapies are generally not being followed. Assessing the applicability and impact of the recommendations from this study may facilitate the implementation of consensus recommendations in future evaluations.
KW - Cost-effectiveness analysis
KW - economic evaluation
KW - gene therapy
KW - health technology assessments
KW - health-related quality of life
KW - onasemnogene abeparvovec
KW - spinal muscular atrophy
KW - systematic literature review
UR - http://www.scopus.com/inward/record.url?scp=85153330591&partnerID=8YFLogxK
U2 - 10.1080/14737167.2023.2197214
DO - 10.1080/14737167.2023.2197214
M3 - Review article
AN - SCOPUS:85153330591
VL - 23
SP - 483
EP - 497
JO - Expert Review of Pharmacoeconomics and Outcomes Research
JF - Expert Review of Pharmacoeconomics and Outcomes Research
SN - 1473-7167
IS - 5
ER -