Challenges and Opportunities in Interdisciplinary Research and Real-World Data for Treatment Sequences in Health Technology Assessments

NICE. Position statement: consideration of products recommended for use in the Cancer Drugs Fund as comparators, or in a treatment sequence, in the appraisal of a new cancer product. Available at: https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-technology-appraisal-guidance/cancer-drugs-fund/CDF-comparator-position-statement.pdf. Accessed 23 Mar 2020.

Claxton K. Exploring uncertainty in cost-effectiveness analysis. Pharmacoeconomics. 2008;26:781–98.

Article  PubMed  Google Scholar 

Tosh J, Stevenson M, Akehurst R. Health economic modelling of treatment sequences for rheumatoid arthritis: a systematic review. Curr Rheumatol Rep. 2014;16:447.

Article  PubMed  Google Scholar 

Benedict A, Stevenson M, Sorensen S. ISPOR Europe 2017: Modeling separate lines of treatment versus treatment sequences in cancer (Workshop 8). Available at: https://www.ispor.org/docs/default-source/presentations/1221.pdf?sfvrsn=6f1f29a1_1. Accessed 20 June 2020.

Al M, Jin H, Robins J, et al. Virtual ISPOR Europe 2020: novel methods for modeling treatment sequencing (spotlight session 3). Available at: https://www.ispor.org/conferences-education/conferences/past-conferences/ispor-europe-2020/program/spotlight-sessions. Accessed 19 Nov 2020.

Kaufman HL, Atkins MB, Subedi P, et al. The promise of immuno-oncology: implications for defining the value of cancer treatment. J Immunother Cancer. 2019;7:1–11.

Article  Google Scholar 

Chehayeb RJ, Hood A, Wang X, et al. Treatment sequencing patterns and associated direct medical costs of metastatic breast cancer care in the United States, 2011 to 2021. JAMA Netw Open. 2022;5:e2244204–304.

Article  Google Scholar 

Roux J, Grimaud O, Leray E. Use of state sequence analysis for care pathway analysis: the example of multiple sclerosis. Stat Methods Med Res. 2019;28:1651–63.

Article  PubMed  Google Scholar 

Kim AH, Jang JE, Han J. Current status on the therapeutic strategies for heart failure and diabetic cardiomyopathy. Biomed Pharmacother. 2022;145: 112463.

Article  CAS  PubMed  Google Scholar 

Kern DM, Cepeda MS, Defalco F, et al. Treatment patterns and sequences of pharmacotherapy for patients diagnosed with depression in the United States: 2014 through 2019. BMC Psychiatry. 2020;20:1–10.

Article  Google Scholar 

Zheng Y, Pan F, Sorensen S. Modeling treatment sequences in pharmacoeconomic models. Pharmacoeconomics. 2017;35:15–24.

Article  PubMed  Google Scholar 

Viola MG, Diamantopoulos A. Determining the baseline strategy in a cost-effectiveness analysis with treatment sequences. Appl Health Econ Health Policy. 2020;18:17–29.

Article  PubMed  Google Scholar 

NICE. NICE Process and methods: guide to the methods of technology appraisal 2013 (PMG 9). Available at: https://www.nice.org.uk/process/pmg9/chapter/foreword. Accessed 2 June 2020.

NICE. NICE health technology evaluations: the manual (PMG 36). Available at: https://www.nice.org.uk/process/pmg36/chapter/introduction-to-health-technology-evaluation. Accessed 17 Aug 2023.

Canadian Agency for Drugs and Technologies in Health (CADTH). Guidelines for the economic evaluation of health technologies: Canada — 4th edition. Available at: https://www.cadth.ca/sites/default/files/pdf/guidelines_for_the_economic_evaluation_of_health_technologies_canada_4th_ed.pdf. Accessed 11 Mar 2023.

Faria R. Problem structuring in economic evaluation. In: Oxford Research Encyclopedia of Economics and Finance. Oxford University Press, 2021.

NICE. Briefing paper for methods review working party on choosing comparators. Available at: https://www.sheffield.ac.uk/media/34080/download?attachment. Accessed 26 Jan 2023.

NICE. Briefing paper for methods review working party on treatment sequences and downstream costs. Available at: https://www.sheffield.ac.uk/media/34080/download?attachment. Accessed 26 Jan 2023.

NICE. Taking a proportionate approach to technology appraisals. Available at: https://www.nice.org.uk/about/what-we-do/proportionate-approach-to-technology-appraisals. Accessed 26 Jan 2024.

National Institute for Health and Care Excellence. NICE real-world evidence framework [ECD 9]. Available at: https://www.nice.org.uk/corporate/ecd9/chapter/overview. Accessed 30 June 2023.

Greenhalgh T, Peacock R. Effectiveness and efficiency of search methods in systematic reviews of complex evidence: audit of primary sources. Br Med J. 2005;331:1064–5.

Article  Google Scholar 

Schlosser RW, Wendt O, Bhavnani S, et al. Use of information-seeking strategies for developing systematic reviews and engaging in evidence-based practice: the application of traditional and comprehensive Pearl Growing. A review. Int J Lang Commun Disord. 2006;41:567–82.

Article  PubMed  Google Scholar 

Hernandez L, O’Donnell M, Postma M. Modeling approaches in cost-effectiveness analysis of disease-modifying therapies for relapsing-remitting multiple sclerosis: an updated systematic review and recommendations for future economic evaluations. Pharmacoeconomics. 2018;36:1223–52.

Article  PubMed  Google Scholar 

Jansen JP, Incerti D, Mutebi A, et al. Cost-effectiveness of sequenced treatment of rheumatoid arthritis with targeted immune modulators. J Med Econ. 2017;20:703–14.

Article  PubMed  Google Scholar 

Badampudi D, Wohlin C, Petersen K. Experiences from using snowballing and database searches in systematic literature studies. In: Proceedings of the 19th International Conference on evaluation and assessment in software engineering (EASE). Nanjing, China: ACM Digital Library, 2015.

Zwakman M, Verberne LM, Kars MC, et al. Introducing PALETTE: an iterative method for conducting a literature search for a review in palliative care. BMC Palliat Care. 2018;17:82.

Article  PubMed  PubMed Central  Google Scholar 

Haywood P. Increasing complexity of cancer care: How displaced treatments impact efficiency, cost-effectiveness and equity [Doctoral Thesis]. University of Technology Sydney, 2018.

Lewis R. Quantitative evidence synthesis methods for the assessment of the effectiveness of treatment sequences for clinical and economic decision-making [Doctoral Thesis]. Bangor University, 2019.

Tosh J. Simulation optimisation to inform economic evaluations of sequential therapies for chronic conditions: a case study in rheumatoid arthritis [Doctoral Thesis]. University of Sheffield, 2015.

Kim E. Sequential drug decision problems in long-term medical conditions: a case study of primary hypertension [Doctoral Thesis]. University of Sheffield, 2015.

Hawkins N, Sculpher M, Epstein D. Cost-effectiveness analysis of treatments for chronic disease: using R to incorporate time dependency of treatment response. Med Decis Making. 2005;25:511–9.

Article  PubMed  Google Scholar 

Saramago P, Manca A, Sutton AJ. Deriving input parameters for cost-effectiveness modeling: taxonomy of data types and approaches to their statistical synthesis. Value Health. 2012;15:639–49.

Article  PubMed  Google Scholar 

Tordrup D, Chouaid C, Cuijpers P, et al. Priorities for health economic methodological research: results of an expert consultation. Int J Technol Assess Health Care. 2017;33:609–19.

Article  PubMed  Google Scholar 

Heeg BMS. Developing health economic models of chronic diseases for reimbursement purposes [Doctoral Thesis]. University of Groningen: Groningen, the Netherlands. 2015.

Brixner DI, Watkins JB. Can CER be an effective tool for change in the development and assessment of new drugs and technologies? J Manag Care Pharm. 2012;18:S06-S11.

PubMed  Google Scholar 

Tappenden P, Chilcott J, Brennan A, et al. Whole disease modeling to inform resource allocation decisions in cancer: a methodological framework. Value Health. 2012;15:1127–36.

Article  PubMed  Google Scholar 

Lord J, Willis S, Eatock J, et al. Economic modelling of diagnostic and treatment pathways in National Institute for Health and Care Excellence clinical guidelines: the Modelling Algorithm Pathways in Guidelines (MAPGuide) project. Health Technol Assess. 2013;17(58):1–192.

Brennan A, Chick SE, Davies R. A taxonomy of model structures for economic evaluation of health technologies. Health Econ. 2006;15:1295–310.

Article  PubMed  Google Scholar 

Rauner MS, Gutjahr WJ, Heidenberger K, et al. Dynamic policy modeling for chronic diseases: metaheuristic-based identification of pareto-optimal screening strategies. Oper Res. 2010;58:1269–86.

Article  Google Scholar 

Cressman S, Browman GP, Hoch JS, et al. A time-trend economic analysis of cancer drug trials. Oncologist. 2015;20:729–36.

Article  PubMed  PubMed Central  Google Scholar 

de Mello-Sampayo F. The timing and probability of treatment switch under cost uncertainty: an application to patients with gastrointestinal stromal tumor. Value Health. 2014;17:215–22.

Article  PubMed  Google Scholar 

Vernon JA, Hughen WK. A primer on dynamic optimization and optimal control in pharmacoeconomics. Value Health. 2006;9:106–13.

Article  PubMed  Google Scholar 

Chick SE, Inoue K. New two-stage and sequential procedures for selecting the best simulated system. Oper Res. 2001;49:732–43.

Article  Google Scholar 

Murphy SA, Oslin DW, Rush AJ, et al. Methodological challenges in constructing effective treatment sequences for chronic psychiatric disorders. Neuropsychopharmacology. 2007;32:257–62.

Article  CAS  PubMed  Google Scholar 

Cain LE, Saag MS, Petersen M, et al. Using observational data to emulate a randomized trial of dynamic treatment-switching strategies: an application to antiretroviral therapy. Int J Epidemiol. 2016;45:2038–49.

Article  PubMed  Google Scholar 

Messner DA. Evaluating the comparative effectiveness of treatment sequences in oncology: a new approach. J Comp Eff Res. 2015;4:537–40.

Article  PubMed  Google Scholar 

Dekker J, Van HL, Hendriksen M, et al. What is the best sequential treatment strategy in the treatment of depression? Adding pharmacotherapy to psychotherapy or vice versa? Psychother Psychosom. 2013;82:89–98.

Article  PubMed  Google Scholar 

Weintraub M, Waldman E, Koplewitz B, et al. A sequential treatment algorithm for infants with stage 4s neuroblastoma and massive hepatomegaly. Pediatr Blood Cancer. 2012;59:182–4.

Article  PubMed  Google Scholar 

Trappe R, Oertel S, Leblond V, et al. Sequential treatment with rituximab followed by CHOP chemotherapy in adult B-cell post-transplant lymphoproliferative disorder (PTLD): the prospective international multicentre phase 2 PTLD-1 trial. Lancet Oncol. 2012;13:196–206.

Article  CAS  PubMed  Google Scholar 

Grigor C, Capell H, Stirling A, et al. Effect of a treatment strategy of tight control for rheumatoid arthritis (the TICORA study): a single-blind randomised controlled trial. Lancet. 2004;364:263–9.

Article  PubMed  Google Scholar 

Wang L, Rotnitzky A, Lin X, et al. Evaluation of viable dynamic treatment regimes in a sequentially randomized trial of advanced prostate cancer. J Am Stat Assoc. 2012;107:493–508.

Article  PubMed  PubMed Central  Google Scholar 

Briggs A, Sidhu M, Baker T. A framework for analysing treatment sequences: incorporating time dependent transitions through partitioned survival analysis. Value Health. 2014;17:A204–5.

Google Scholar 

Lewis RA, Hughes D, Sutton AJ, et al. Quantitative evidence synthesis methods for the assessment of the effectiveness of treatment sequences for clinical and economic decision making: a review and taxonomy of simplifying assumptions. Pharmacoeconomics. 2021;39:25–61.

Welton N, Phillippo D, Owen R, et al. CHTE2020 sources and synthesis of evidence; update to evidence synthesis methods. Available at: https://rees-france.com/wp-content/uploads/2020/12/CHTE-2020_synthesis-of-evidence.pdf. Accessed 29 Aug 2022.

Huang M, Ramsey S, Xue W, et al. Conceptual framework and methodological challenges for modeling effectiveness in oncology treatment sequence models. Pharmacoeconomics. 2022;40:257–268.

Simpson A, Ramagopalan SV. R WE ready for reimbursement? A round up of developments in real-world evidence relating to HTA: part 5. J Comp Eff Res. 2022;11:213–5.

Article  CAS  PubMed  Google Scholar 

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