Toward Best Practices for Economic Evaluations of Tumor-Agnostic Therapies : A Review of Current Barriers and Solutions
Copyright © 2023. Published by Elsevier Inc..
OBJECTIVES: Cancer therapies targeting tumor-agnostic biomarkers are challenging traditional health technology assessment (HTA) frameworks. The high prevalence of nonrandomized single-arm trials, heterogeneity, and small benefiting populations are driving outcomes uncertainty, challenging healthcare decision making. We conducted a structured literature review to identify barriers and prioritize solutions to generating economic evidence for tumor-agnostic therapies.
METHODS: We searched MEDLINE and Embase for English-language studies conducting economic evaluations of tumor-agnostic treatments or exploring related challenges and solutions. We included studies published by December 2022 and supplemented our review with Canadian Agency for Drugs and Technologies in Health and National Institute for Health and Care Excellence technical reports for approved tumor-agnostic therapies. Three reviewers abstracted and summarized key methodological and empirical study characteristics. Challenges and solutions were identified through authors' statements and categorized using directed content analysis.
RESULTS: Twenty-six studies met our inclusion criteria. Studies spanned economic evaluations (n = 5), reimbursement reviews (n = 4), qualitative research (n = 1), methods validations (n = 3), and commentaries or literature reviews (n = 13). Challenges encountered related to (1) the treatment setting and clinical trial designs, (2) a lack of data or low-quality data on clinical and cost parameters, and (3) an inability to produce evidence that meets HTA guidelines. Although attempted solutions centered on analytic approaches for managing missing data, proposed solutions highlighted the need for real-world evidence combined with life-cycle HTA to reduce future evidentiary uncertainty.
CONCLUSIONS: Therapeutic innovation outpaces HTA evidence generation and the methods that support it. Existing HTA frameworks must be adapted for tumor-agnostic treatments to support future economic evaluations enabling timely patient access.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2023 |
|---|---|
| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:26 |
|---|---|
| Enthalten in: |
Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research - 26(2023), 11 vom: 03. Nov., Seite 1608-1617 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Weymann, Deirdre [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
Economic evaluation |
|---|
| Anmerkungen: |
Date Completed 30.10.2023 Date Revised 03.11.2023 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1016/j.jval.2023.07.004 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM360428940 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM360428940 | ||
| 003 | DE-627 | ||
| 005 | 20231226083008.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2023 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1016/j.jval.2023.07.004 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1201.xml |
| 035 | |a (DE-627)NLM360428940 | ||
| 035 | |a (NLM)37543205 | ||
| 035 | |a (PII)S1098-3015(23)03070-X | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Weymann, Deirdre |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Toward Best Practices for Economic Evaluations of Tumor-Agnostic Therapies |b A Review of Current Barriers and Solutions |
| 264 | 1 | |c 2023 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 30.10.2023 | ||
| 500 | |a Date Revised 03.11.2023 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2023. Published by Elsevier Inc. | ||
| 520 | |a OBJECTIVES: Cancer therapies targeting tumor-agnostic biomarkers are challenging traditional health technology assessment (HTA) frameworks. The high prevalence of nonrandomized single-arm trials, heterogeneity, and small benefiting populations are driving outcomes uncertainty, challenging healthcare decision making. We conducted a structured literature review to identify barriers and prioritize solutions to generating economic evidence for tumor-agnostic therapies | ||
| 520 | |a METHODS: We searched MEDLINE and Embase for English-language studies conducting economic evaluations of tumor-agnostic treatments or exploring related challenges and solutions. We included studies published by December 2022 and supplemented our review with Canadian Agency for Drugs and Technologies in Health and National Institute for Health and Care Excellence technical reports for approved tumor-agnostic therapies. Three reviewers abstracted and summarized key methodological and empirical study characteristics. Challenges and solutions were identified through authors' statements and categorized using directed content analysis | ||
| 520 | |a RESULTS: Twenty-six studies met our inclusion criteria. Studies spanned economic evaluations (n = 5), reimbursement reviews (n = 4), qualitative research (n = 1), methods validations (n = 3), and commentaries or literature reviews (n = 13). Challenges encountered related to (1) the treatment setting and clinical trial designs, (2) a lack of data or low-quality data on clinical and cost parameters, and (3) an inability to produce evidence that meets HTA guidelines. Although attempted solutions centered on analytic approaches for managing missing data, proposed solutions highlighted the need for real-world evidence combined with life-cycle HTA to reduce future evidentiary uncertainty | ||
| 520 | |a CONCLUSIONS: Therapeutic innovation outpaces HTA evidence generation and the methods that support it. Existing HTA frameworks must be adapted for tumor-agnostic treatments to support future economic evaluations enabling timely patient access | ||
| 650 | 4 | |a Review | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a economic evaluation | |
| 650 | 4 | |a health technology assessment | |
| 650 | 4 | |a histology-independent | |
| 650 | 4 | |a review | |
| 650 | 4 | |a tumor-agnostic | |
| 700 | 1 | |a Pollard, Samantha |e verfasserin |4 aut | |
| 700 | 1 | |a Lam, Halina |e verfasserin |4 aut | |
| 700 | 1 | |a Krebs, Emanuel |e verfasserin |4 aut | |
| 700 | 1 | |a Regier, Dean A |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research |d 1998 |g 26(2023), 11 vom: 03. Nov., Seite 1608-1617 |w (DE-627)NLM115934545 |x 1524-4733 |7 nnns |
| 773 | 1 | 8 | |g volume:26 |g year:2023 |g number:11 |g day:03 |g month:11 |g pages:1608-1617 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1016/j.jval.2023.07.004 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 26 |j 2023 |e 11 |b 03 |c 11 |h 1608-1617 | ||