Analyses of quality of life in cancer drug trials - a review of measurements and analytical choices in post-reimbursement studies.

Publisher: BioMed Central Country of Publication: England NLM ID: 100967800 Publication Model: Electronic Cited Medium: Internet ISSN: 1471-2407 (Electronic) Linking ISSN: 14712407 NLM ISO Abbreviation: BMC Cancer Subsets: MEDLINE

Original Publication: London : BioMed Central, [2001-

Quality of Life* ; Neoplasms*/drug therapy; Humans ; Health Facilities ; Motivation ; Sweden

Objectives: For drugs reimbursed with limited evidence of patient benefits, confirmatory evidence of overall survival (OS) and quality of life (QoL) benefits is important. For QoL data to serve as valuable input to patients and decision-makers, it must be measured and analyzed using appropriate methods. We aimed to assess the measurement and analyses of post-reimbursement QoL data for cancer drugs introduced in Swedish healthcare with limited evidence at the time of reimbursement.
Methods: We reviewed any published post-reimbursement trial data on QoL for cancer drugs reimbursed in Sweden between 2010 and 2020 with limited evidence of improvement in QoL and OS benefits at the time of reimbursement. We extracted information on the instruments used, frequency of measurement, extent of missing data, statistical approaches, and the use of pre-registration and study protocols.
Results: Out of 22 drugs satisfying our inclusion criteria, we identified published QoL data for 12 drugs in 22 studies covering multiple cancer types. The most frequently used QoL instruments were EORTC QLQ-C30 and EQ-5D-3/5L. We identified three areas needing improvement in QoL measurement and analysis: (i) motivation for the frequency of measurements, (ii) handling of the substantial missing data problem, and (iii) inclusion and adherence to QoL analyses in clinical trial pre-registration and study protocols.
Conclusions: Our review shows that the measurements and analysis of QoL data in our sample of cancer trials covering drugs initially reimbursed without any confirmed QoL or OS evidence have significant room for improvement. The increasing use of QoL assessments must be accompanied by a stricter adherence to best-practice guidelines to provide valuable input to patients and decision-makers.
(© 2024. The Author(s).)

Value Health Reg Issues. 2018 Sep;16:81-91. (PMID: 30316029)
ESMO Open. 2022 Oct;7(5):100569. (PMID: 36037568)
Cancer Discov. 2021 Apr;11(4):822-837. (PMID: 33811119)
Health Qual Life Outcomes. 2016 Apr 12;14:60. (PMID: 27072351)
Ann Oncol. 2021 Apr;32(4):488-499. (PMID: 33385521)
Value Health. 2019 Feb;22(2):203-209. (PMID: 30711065)
BMJ. 2017 Oct 4;359:j4530. (PMID: 28978555)
Oncologist. 2011;16(5):632-40. (PMID: 21459902)
Int J Cancer. 2019 Apr 1;144(7):1746-1751. (PMID: 30374970)
Int J Technol Assess Health Care. 2021 Jan 25;37:e23. (PMID: 33491617)
JAMA. 2018 Feb 6;319(5):483-494. (PMID: 29411037)
Lancet Oncol. 2013 May;14(6):552-62. (PMID: 23598172)
BMC Cancer. 2021 Nov 18;21(1):1237. (PMID: 34794404)
Qual Life Res. 2011 Jun;20(5):653-64. (PMID: 21110123)
Pharmacoeconomics. 2016 Jul;34(7):645-9. (PMID: 26892973)
Int J Technol Assess Health Care. 2006 Winter;22(1):10-8. (PMID: 16673675)
J Natl Cancer Inst. 2019 Nov 1;111(11):1170-1178. (PMID: 30959516)
Clin Drug Investig. 2023 Aug;43(8):621-633. (PMID: 37505421)
Lancet Oncol. 2018 Oct;19(10):e504. (PMID: 30303117)
J Clin Oncol. 2018 Mar 10;36(8):757-764. (PMID: 29377755)
JAMA Netw Open. 2023 Jan 3;6(1):e2251564. (PMID: 36656587)
Lancet Oncol. 2020 Feb;21(2):e83-e96. (PMID: 32007209)
Lung Cancer. 2018 Jul;121:5-11. (PMID: 29858027)
Ann Oncol. 2014 Jul;25(7):1428-1436. (PMID: 24769640)
BMC Med. 2017 Jul 21;15(1):134. (PMID: 28728605)
J Thorac Oncol. 2016 Nov;11(11):1927-1939. (PMID: 27496650)
Eur J Cancer. 2020 Jan;125:49-57. (PMID: 31838405)
Eur J Cancer. 2017 Sep;83:166-176. (PMID: 28738257)
Ann Oncol. 2015 Jan;26(1):231-237. (PMID: 25355720)
J Clin Oncol. 2022 Jul 1;40(19):2138-2147. (PMID: 35290101)
J Natl Cancer Inst. 1993 Mar 3;85(5):365-76. (PMID: 8433390)
Eur J Cancer. 2018 May;94:206-215. (PMID: 29574365)
Ann Med. 2001 Jul;33(5):337-43. (PMID: 11491192)
Cancer. 2010 Sep 15;116(18):4256-65. (PMID: 20549832)
BMC Cancer. 2016 Feb 18;16:122. (PMID: 26892541)
Lancet Oncol. 2018 Aug;19(8):1117-1125. (PMID: 30026000)
Breast Cancer Res Treat. 2019 Apr;174(3):719-729. (PMID: 30632023)
Lancet Oncol. 2018 Aug;19(8):1126-1134. (PMID: 30026002)
Lung Cancer. 2019 Dec;138:79-87. (PMID: 31654838)
N Engl J Med. 2022 Oct 20;387(16):1439-1442. (PMID: 36129992)
Qual Life Res. 2022 Jul;31(7):2167-2173. (PMID: 35247152)
Am J Transl Res. 2021 Nov 15;13(11):13108-13116. (PMID: 34956530)
Lancet Oncol. 2020 Oct;21(10):e488-e494. (PMID: 33002444)
Eur J Cancer. 2019 Mar;110:1-7. (PMID: 30735832)
EClinicalMedicine. 2023 Apr 06;59:101953. (PMID: 37089618)
Lancet Oncol. 2018 May;19(5):e267-e274. (PMID: 29726391)
N Engl J Med. 2017 May 25;376(21):2001-2004. (PMID: 28538133)
J Clin Oncol. 2020 Apr 10;38(11):1164-1174. (PMID: 32073956)
Am J Hematol. 2021 Sep 1;96(9):1112-1119. (PMID: 34050972)
J Natl Cancer Inst. 2021 May 4;113(5):532-542. (PMID: 33146385)
Breast Cancer Res Treat. 2018 Jun;169(3):469-479. (PMID: 29404806)
Onco Targets Ther. 2015 Jun 08;8:1363-73. (PMID: 26089686)
Eur J Cancer. 2022 Nov;175:236-245. (PMID: 36155117)
Oncologist. 2002;7(2):120-5. (PMID: 11961195)
JAMA. 2013 Feb 27;309(8):814-22. (PMID: 23443445)
J Clin Oncol. 2018 Jun 20;36(18):1853-1860. (PMID: 29733770)
Lancet Oncol. 2017 Jul;18(7):874-886. (PMID: 28602779)
Br J Cancer. 2021 Nov;125(11):1477-1485. (PMID: 34400802)
BMC Res Notes. 2019 Jan 14;12(1):18. (PMID: 30642397)

Keywords: Cancer; Health-related quality of life; Patient-reported outcomes; Quality assessment; Randomized controlled trials

Date Created: 20240306 Date Completed: 20240308 Latest Revision: 20240309

20240309

PMC10916053

10.1186/s12885-024-12045-8

38448848