A Critical Appraisal and Recommendations for Cost-Effectiveness Studies of Poly(ADP-Ribose) Polymerase Inhibitors in Advanced Ovarian Cancer.
Journal
PharmacoEconomics
ISSN: 1179-2027
Titre abrégé: Pharmacoeconomics
Pays: New Zealand
ID NLM: 9212404
Informations de publication
Date de publication:
11 2020
11 2020
Historique:
pubmed:
15
8
2020
medline:
14
9
2021
entrez:
15
8
2020
Statut:
ppublish
Résumé
Ovarian cancer is the fifth leading cause of cancer death in women in the US. With poly(ADP-ribose) polymerase (PARP) inhibitors having shown promising results in ongoing trials, there is interest in better understanding their economic value. This study aimed to review and evaluate the quality of published cost-effectiveness analyses (CEAs), and provide recommendations for CEAs in this setting. A systematic literature review of the MEDLINE and EMBASE databases was conducted in June 2019 to identify CEAs of PARP inhibitors in treating advanced ovarian cancer from peer-reviewed journals and conferences. Key information from the identified publications were extracted and reviewed. The quality of full-text studies was assessed using the Quality of Health Economic Studies instrument. Recommendations for future CEAs were developed based on the findings from the literature review. Eighteen CEAs (five in full texts) met the inclusion criteria. Most adopted a US healthcare or societal perspective. The majority of the studies did not clearly display the economic model structure. No studies reported the validation of model projections based on internal or external data. Surrogate outcomes such as incremental costs per progression-free life-year gained were the most common outcomes reported. The majority of studies drew their conclusions based on surrogate outcomes, even with no theoretical or empirical threshold for cost effectiveness. All five full-text studies included some type of sensitivity or scenario analyses. The key drivers of the incremental cost-effectiveness ratio were treatment duration, effects, and costs, health utility, and prevalence of BRCA mutations. In the existing CEAs for PARP inhibitors, there were uncertainties and challenges leading to variation in quality. We provided recommendations to improve consistency and quality of CEAs in this setting, which will help to better understand the value of PARP inhibitors, improve decision making, and reduce potential misallocation of resources.
Sections du résumé
BACKGROUND
Ovarian cancer is the fifth leading cause of cancer death in women in the US. With poly(ADP-ribose) polymerase (PARP) inhibitors having shown promising results in ongoing trials, there is interest in better understanding their economic value.
OBJECTIVE
This study aimed to review and evaluate the quality of published cost-effectiveness analyses (CEAs), and provide recommendations for CEAs in this setting.
METHODS
A systematic literature review of the MEDLINE and EMBASE databases was conducted in June 2019 to identify CEAs of PARP inhibitors in treating advanced ovarian cancer from peer-reviewed journals and conferences. Key information from the identified publications were extracted and reviewed. The quality of full-text studies was assessed using the Quality of Health Economic Studies instrument. Recommendations for future CEAs were developed based on the findings from the literature review.
RESULTS
Eighteen CEAs (five in full texts) met the inclusion criteria. Most adopted a US healthcare or societal perspective. The majority of the studies did not clearly display the economic model structure. No studies reported the validation of model projections based on internal or external data. Surrogate outcomes such as incremental costs per progression-free life-year gained were the most common outcomes reported. The majority of studies drew their conclusions based on surrogate outcomes, even with no theoretical or empirical threshold for cost effectiveness. All five full-text studies included some type of sensitivity or scenario analyses. The key drivers of the incremental cost-effectiveness ratio were treatment duration, effects, and costs, health utility, and prevalence of BRCA mutations.
CONCLUSION
In the existing CEAs for PARP inhibitors, there were uncertainties and challenges leading to variation in quality. We provided recommendations to improve consistency and quality of CEAs in this setting, which will help to better understand the value of PARP inhibitors, improve decision making, and reduce potential misallocation of resources.
Identifiants
pubmed: 32794041
doi: 10.1007/s40273-020-00949-9
pii: 10.1007/s40273-020-00949-9
pmc: PMC7547040
doi:
Substances chimiques
Poly(ADP-ribose) Polymerase Inhibitors
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Systematic Review
Langues
eng
Pagination
1201-1218Références
Center for Disease Control and Prevention, US Department of Health and Human Services. Ovarian cancer. Inside knowledge about gynecologic cancer 99-9124. 2019. https://www.cdc.gov/cancer/ovarian/pdf/ovarian_facts.pdf . Accessed June 2019.
Siegel RL, Miller KD, Jemal A. Cancer statistics, 2019. CA Cancer J Clin. 2019;69(1):7–34.
doi: 10.3322/caac.21551
National Cancer Institute. BRCA mutations: cancer risk and genetic testing. 2018. https://www.cancer.gov/about-cancer/causes-prevention/genetics/brca-fact-sheet#how-much-does-having-a-brca1-or-brca2-gene-mutation-increase-a-womans-risk-of-breast-and-ovarian-cancer . Accessed July 2019.
O’Connor MJ. Targeting the DNA damage response in cancer. Mol Cell. 2015;60(4):547–60.
pubmed: 26590714
doi: 10.1016/j.molcel.2015.10.040
US FDA. FDA approves olaparib tablets for maintenance treatment in ovarian cancer. 2017. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-olaparib-tablets-maintenance-treatment-ovarian-cancer . Accessed June 2019.
Moore K, Colombo N, Scambia G, Kim BG, Oaknin A, Friedlander M, et al. Maintenance olaparib in patients with newly diagnosed advanced ovarian cancer. N Engl J Med. 2018;379(26):2495–505.
doi: 10.1056/NEJMoa1810858
Ledermann J, Harter P, Gourley C, Friedlander M, Vergote I, Rustin G, et al. Olaparib maintenance therapy in platinum-sensitive relapsed ovarian cancer. N Engl J Med. 2012;366(15):1382–92.
pubmed: 22452356
pmcid: 22452356
doi: 10.1056/NEJMoa1105535
Pujade-Lauraine E, Ledermann JA, Selle F, Gebski V, Penson RT, Oza AM, et al. Olaparib tablets as maintenance therapy in patients with platinum-sensitive, relapsed ovarian cancer and a BRCA1/2 mutation (SOLO2/ENGOT-Ov21): a double-blind, randomised, placebo-controlled, phase 3 trial. Lancet Oncol. 2017;18(9):1274–84.
pubmed: 28754483
pmcid: 28754483
doi: 10.1016/S1470-2045(17)30469-2
Ray-Coquard I, Pautier P, Pignata S, Perol D, Gonzalez-Martin A, Berger R, et al. Olaparib plus bevacizumab as first-line maintenance in ovarian cancer. N Engl J Med. 2019;381(25):2416–28.
pubmed: 31851799
doi: 10.1056/NEJMoa1911361
National Comprehensive Cancer Network. Ovarian cancer (version 2.2019). https://www.nccn.org/professionals/physician_gls/pdf/ovarian.pdf . Accessed Oct 2019.
Gonzalez-Martin A, Pothuri B, Vergote I, DePont Christensen R, Graybill W, Mirza MR, et al. Niraparib in patients with newly diagnosed advanced ovarian cancer. N Engl J Med. 2019;381(25):2391–402.
pubmed: 31562799
doi: 10.1056/NEJMoa1910962
Institute for Clinical and Economic Review. Poly ADP-ribose polymerase (PARP) inhibitors for ovarian cancer: effectiveness and value. 2017. https://icer-review.org/wp-content/uploads/2017/02/MWCEPAC_OVARIAN_FINAL_EVIDENCE_REPORT_10112017-1.pdf . Accessed June 2019.
McQueen RB, Whittington MD, Chapman RH, Kumar VM, Campbell JD. Comment on “Cost-effectiveness of niraparib versus routine surveillance, olaparib and rucaparib for the maintenance treatment of patients with ovarian cancer in the United States”. PharmacoEconomics. 2019;37(7):963–4.
pubmed: 31044348
doi: 10.1007/s40273-019-00802-8
Wallace K, Goble S, Isaacson J, Maloney L, Cameron T, Bedel J. Comment on: “Cost-effectiveness of niraparib versus routine surveillance, olaparib and rucaparib for the maintenance treatment of patients with ovarian cancer in the United States”. PharmacoEconomics. 2019;37(8):1065–7.
pubmed: 31172449
pmcid: 6830408
doi: 10.1007/s40273-019-00815-3
Ofman JJ, Sullivan SD, Neumann PJ, Chiou C-F, Henning JM, Wade SW, et al. Examining the value and quality of health economic analyses: implications of utilizing the QHES. J Manag Care Pharm. 2003;9(1):53–61.
pubmed: 14613362
Alvarez-Secord A, Barnett J, Ledermann J, Peterson B, Myers E, Havrilesky LJ. Cost-effectiveness of homologous recombination defect testing to target PARP inhibitor use in platinum-sensitive recurrent ovarian cancer. Gynecol Oncol. 2012;125(Suppl 1):S92–3.
doi: 10.1016/j.ygyno.2011.12.223
Alvarez-Secord A, Barnett JC, Ledermann JA, Peterson BL, Myers ER, Havrilesky LJ. Cost-effectiveness of BRCA1 and BRCA2 mutation testing to target PARP inhibitor use in platinum-sensitive recurrent ovarian cancer. Int J Gynecol Cancer. 2013;23(5):846–52.
doi: 10.1097/IGC.0b013e31829527bd
Chin LM, Carlson JJ. Cost-effectiveness analysis of rucaparib maintenance treatment for recurrent ovarian carcinoma after response to platinum therapy. Value Health. 2018;21(Suppl 1):S30.
doi: 10.1016/j.jval.2018.04.193
Dottino JA, Moss HA, Lu KH, Alvarez-Secord A, Havrilesky LJUS. Food and drug administration-approved poly(ADP-ribose) polymerase inhibitor maintenance therapy for recurrent ovarian cancer: a cost-effectiveness analysis. Obstet Gynecol. 2019;133(4):795–802.
pubmed: 30870286
pmcid: 7181403
doi: 10.1097/AOG.0000000000003171
Dottino JA, Moss HA, Lu KH, Alvarez-Secord A, Havrilesky LJ. Are FDA-approved PARPi cost-effective as maintenance treatment of platinum-sensitive recurrent ovarian cancer? Gynecol Oncol. 2018;149(Suppl 1):11–2.
doi: 10.1016/j.ygyno.2018.04.032
Fernandez-Santos JM, Andreas GS. Relapsed ovarian cancer treatment costs comparison in Spain. Value Health. 2017;20(Suppl 5):A100.
Guy H, Walder L, Fisher M. Cost-effectiveness of niraparib versus routine surveillance, olaparib and rucaparib for the maintenance treatment of patients with ovarian cancer in the United States. PharmacoEconomics. 2019;37(3):391–405.
pubmed: 30478649
doi: 10.1007/s40273-018-0745-z
Liu AY, Cohen JG, Walsh CS, Holschneider CH, Sinno AK. A cost-effectiveness analysis of three PARP inhibitors for maintenance therapy in platinum-sensitive recurrent ovarian cancer. Gynecol Oncol. 2018;149(Suppl 1):9.
doi: 10.1016/j.ygyno.2018.04.027
Liu AY, Cohen JG, Walsh CS, Holschneider CH, Sinno AK. A cost-effectiveness analysis of three PARP inhibitors for maintenance therapy in platinum-sensitive recurrent ovarian cancer. Gynecol Oncol. 2017;147(1):196.
doi: 10.1016/j.ygyno.2017.07.025
Mylonas C, Aravantinos G, Bamias A, Kourlaba G, Maniadakis N. Economic evaluation of olaparib in patients with BRCA-mutated Psroc in Greece. Value Health. 2016;19(Suppl 7):A732.
doi: 10.1016/j.jval.2016.09.2208
Smith HJ, Walters Haygood CL, Arend RC, Leath CA 3rd, Straughn JM Jr. PARP inhibitor maintenance therapy for patients with platinum-sensitive recurrent ovarian cancer: a cost-effectiveness analysis. Gynecol Oncol. 2015;139(1):59–62.
pubmed: 26303225
doi: 10.1016/j.ygyno.2015.08.013
Smith HJ, Walters Haygood CL, Arend RC, Leath CA III, Straughn JM Jr. PARP inhibitors as maintenance therapy for patients with platinum-sensitive recurrent ovarian cancer: can we afford it? Gynecol Oncol. 2015;137(Suppl 1):9.
doi: 10.1016/j.ygyno.2015.01.017
Wolford JE, Bai J, Eskander RH, Keller R, Minion L, Chan JK, et al. A Markov model to evaluate cost-effectiveness of the PARP inhibitor, olaparib, for fourth-line treatment of recurrent ovarian cancer. J Clin Oncol. 2016;34(15 Suppl):5563.
doi: 10.1200/JCO.2016.34.15_suppl.5563
Wolford JE, Bai J, Eskander RH, Keller R, Minion LE, Chan JK, et al. Evaluating the cost-effectiveness of current FDA-approved PARP inhibitors for the treatment of recurrent ovarian cancer. J Clin Oncol. 2017;35(15 Suppl):5516.
doi: 10.1200/JCO.2017.35.15_suppl.5516
Wolford JE, Bai J, Minion LE, Keller R, Eskander RN, Chan JK, et al. Cost-effectiveness of maintenance therapy in advanced ovarian cancer: paclitaxel, bevacizumab, niraparib, rucaparib, olaparib, and pembrolizumab. J Clin Oncol. 2018;36(15 Suppl):5508.
doi: 10.1200/JCO.2018.36.15_suppl.5508
Wolford JE, Liang SY, Tewari KS, Eskander RN, Kapp DS, Mann AK, et al. An economic analysis: examining the cost-effectiveness of bevacizumab and olaparib as upfront maintenance treatment of advanced ovarian cancer. Gynecol Oncol. 2019;154(Suppl 1):25.
doi: 10.1016/j.ygyno.2019.04.060
Wolford JE, Tewari KS, Liang SY, Bai J, Mann AK, Moore KN, et al. SOLO1 versus SOLO2: cost-effectiveness of olaparib as maintenance therapy for newly diagnosed and platinum-sensitive recurrent ovarian carcinoma among women with germline BRCA mutations (gBRCAmut). J Clin Oncol. 2019;37(15 Suppl):5545.
doi: 10.1200/JCO.2019.37.15_suppl.5545
Zhong L, Tran AT, Tomasino T, Nugent E, Smith JA. Cost-effectiveness of niraparib and olaparib as maintenance therapy for patients with platinum-sensitive recurrent ovarian cancer. J Manag Care Spec Pharm. 2018;24(12):1219–28.
pubmed: 30479195
Eddy DM, Hollingworth W, Caro JJ, Tsevat J, McDonald KM, Wong JB. Model transparency and validation: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force-7. Med Decis Mak. 2012;32(5):733–43.
doi: 10.1177/0272989X12454579
Gutman SI, Piper M, Grant MD, Basch E, Oliansky DM, Aronson N. AHRQ methods for effective health care. Progression-free survival: what does it mean for psychological well-being or quality of life?. Rockville: Agency for Healthcare Research and Quality; 2013.
Davis S, Tappenden P, Cantrell A. NICE decision support unit methods development. A review of studies examining the relationship between progression-free survival and overall survival in advanced or metastatic cancer. London: National Institute for Health and Care Excellence; 2012.
Ledermann JA, Harter P, Gourley C, Friedlander M, Vergote I, Rustin G, et al. Overall survival in patients with platinum-sensitive recurrent serous ovarian cancer receiving olaparib maintenance monotherapy: an updated analysis from a randomised, placebo-controlled, double-blind, phase 2 trial. Lancet Oncol. 2016;17(11):1579–89.
pubmed: 27617661
doi: 10.1016/S1470-2045(16)30376-X
National Institute for Health and Care Excellence. Multivariate meta-analysis of summary data for combining treatment effects on correlated outcomes and evaluating surrogate endpoints (NICE DSU Technical Support Document 20). 2019. http://nicedsu.org.uk/wp-content/uploads/2019/10/TSD-20-mvmeta-final.pdf . Accessed Oct 2019.
Lakdawalla DN, Doshi JA, Garrison LP Jr, Phelps CE, Basu A, Danzon PM. Defining elements of value in health care-a health economics approach: an ISPOR special task force report [3]. Value Health. 2018;21(2):131–9.
pubmed: 29477390
doi: 10.1016/j.jval.2017.12.007
Institute for Clinical and Economic Review. Value assessment framework for 2020–2023. 2019. https://icer-review.org/wp-content/uploads/2019/05/ICER_2020_2023_VAF_013120-4.pdf . Accessed July 2019.
Wolford JE, Bai J, Moore KN, Kristeleit R, Monk BJ, Tewari KS. Cost-effectiveness of niraparib, rucaparib, and olaparib for treatment of platinum-resistant, recurrent ovarian carcinoma. Gynecol Oncol. 2020;157(2):500–7.
pubmed: 32173049
pmcid: 7410501
doi: 10.1016/j.ygyno.2020.02.030
Hettle R, Posnett J, Borrill J. Challenges in economic modeling of anticancer therapies: an example of modeling the survival benefit of olaparib maintenance therapy for patients with BRCA-mutated platinum-sensitive relapsed ovarian cancer. J Med Econ. 2015;18(7):516–24.
pubmed: 25735651
doi: 10.3111/13696998.2015.1024682
Carlson JJ, Walton SM, Basu A, Chapman RH, Campbell JD, McQueen RB, et al. Achieving appropriate model transparency: challenges and potential solutions for making value-based decisions in the United States. PharmacoEconomics. 2019;37(11):1321–7.
pubmed: 31485925
pmcid: 6860462
doi: 10.1007/s40273-019-00832-2
Sampson CJ, Arnold R, Bryan S, Clarke P, Ekins S, Hatswell A, et al. Transparency in decision modelling: what, why, who and how? PharmacoEconomics. 2019;37(11):1355–69.
pubmed: 31240636
doi: 10.1007/s40273-019-00819-z
Wu EQ, Zhou ZY, Xie J, Metallo C, Thokala P. Transparency in health economic modeling: options, issues and potential solutions. PharmacoEconomics. 2019;37(11):1349–54.
pubmed: 31591672
doi: 10.1007/s40273-019-00842-0
Mattingly TJ, Levy JF, Slejko JF, Onwudiwe NC, Perfetto EM. Estimating drug costs: how do manufacturer net prices compare with other common US price references? PharmacoEconomics. 2018;36(9):1093–9.
pubmed: 29752675
pmcid: 6061401
doi: 10.1007/s40273-018-0667-9
Weinstein MC, O’Brien B, Hornberger J, Jackson J, Johannesson M, McCabe C, et al. Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR Task Force on Good Research Practices—Modeling Studies. Value Health. 2003;6(1):9–17.
pubmed: 12535234
doi: 10.1046/j.1524-4733.2003.00234.x