Development and validation of a patient-reported outcome tool to assess cancer-related financial toxicity in Italy: a protocol

Silvia Riva, Jane Bryce, Francesco De Lorenzo, Laura Del Campo, Massimo Di Maio, Fabio Efficace, Luciano Frontini, Diana Giannarelli, Lara Gitto, Elisabetta Iannelli, Claudio Jommi, Vincenzo Montesarchio, Francesca Traclò, Concetta Maria Vaccaro, Ciro Gallo, Francesco Perrone, Silvia Riva, Jane Bryce, Francesco De Lorenzo, Laura Del Campo, Massimo Di Maio, Fabio Efficace, Luciano Frontini, Diana Giannarelli, Lara Gitto, Elisabetta Iannelli, Claudio Jommi, Vincenzo Montesarchio, Francesca Traclò, Concetta Maria Vaccaro, Ciro Gallo, Francesco Perrone

Abstract

Introduction: Financial toxicity (FT) is a well-recognised problem in oncology. US-based studies have shown that: (a) cancer patients have a 2.7 times risk of bankruptcy; (b) patients who declare bankruptcy have a 79% greater hazard of death; (c) financial burden significantly impairs quality of life (QoL) and (d) reduces compliance and adherence to treatment prescriptions. The aim of the project is to develop and validate a patient-reported-outcome (PRO) measure to assess FT of cancer patients in Italy, where, despite the universal health coverage provided by the National Health Service, FT is an emerging issue.

Methods and analysis: Our hypothesis is that a specific FT measure, which considers the relevant sociocultural context and healthcare system, would allow us to understand the main determinants of cancer-related FT in Italy, in order to address and reduce these factors. According to the International Society for Pharmaco-economics and Outcomes Research guidelines on PROs, the project will include the following steps: (1) concept elicitation (from focus groups with patients and caregivers; literature; oncologists; nurses) and analysis, creating a coding library; (2) item generation (using a format that includes a question and a response on a 4-point Likert scale) and analysis through patients' cognitive interviews of item importance within different coding categories to produce the draft instrument; (3) factor analysis and internal validation (with Cronbach's alpha and test-retest for reliability) to produce the final instrument; (4) external validation with QoL anchors and depression scales. The use of the FT measure in prospective trials is also planned.

Ethics and dissemination: The protocol is approved by the ethical committees of all the participating centres. The project will tentatively produce a validated tool by the spring 2021. The project might also represent a model and the basis for future cooperation with other European countries, with different healthcare systems and socioeconomic conditions.

Trial registration number: NCT03473379.

Keywords: Cancer; Financial toxicity; Health economics; Italy; Patient Reported Outcome Measures.

Conflict of interest statement

Competing interests: SR has received personal fees from CSL-Behring and GlaxoSmithKline Foundation. JB has received personal fees from Novartis, AstraZeneca, Merck Sharp & Dohme. MDM has received personal fees from Bristol Myers Squibb, Merck Sharp & Dohme, Roche, AstraZeneca, Janssen. FE has received personal fees from Bristol Myers Squibb, Incyte, Orsenyx and Amgen. LG has received personal fees from Bristol Myers Squibb. CJ has received personal fees from Amgen, Astra Zeneca, Biogen, Boehringer Ingelheim, Celgene, Gilead, GSK, Ipsen, Janssen-Cilag, Takeda and Sanofi. VM has received personal fees from Bristol Myers Squibb and Italfarmaco; a member of his family is employee in Bayer. CMV has received personal fees from Baxter, MSD, Novartis, Sanofi, Sanofi Genzyme. FP has received personal fees from Bayer, Ipsen, Astra Zeneca, Bristol Myers Squibb, Sandoz, Incyte, Celgene, Pierre Fabre, Janssen-Cilag.

© Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Figures

Figure 1
Figure 1
Endpoint models for future use of PROFFIT. PROFFIT, Patient Reported Outcome for FIghting Financial Toxicity of cancer; QoL, quality of life.

References

    1. Mailankody S, Prasad V. Five years of cancer drug approvals: innovation, efficacy, and costs. JAMA Oncol 2015;1:539–40. 10.1001/jamaoncol.2015.0373
    1. Saltz LB. Perspectives on cost and value in cancer care. JAMA Oncol 2016;2:19–21. 10.1001/jamaoncol.2015.4191
    1. Ramsey S, Blough D, Kirchhoff A, et al. . Washington state cancer patients found to be at greater risk for bankruptcy than people without a cancer diagnosis. Health Aff 2013;32:1143–52. 10.1377/hlthaff.2012.1263
    1. Chino F, Peppercorn J, Taylor DH, et al. . Self-Reported financial burden and satisfaction with care among patients with cancer. Oncologist 2014;19:414–20. 10.1634/theoncologist.2013-0374
    1. Zafar SY, Peppercorn JM, Schrag D, et al. . The financial toxicity of cancer treatment: a pilot study assessing out-of-pocket expenses and the insured cancer patient's experience. Oncologist 2013;18:381–90. 10.1634/theoncologist.2012-0279
    1. Lathan CS, Cronin A, Tucker-Seeley R, et al. . Association of financial strain with symptom burden and quality of life for patients with lung or colorectal cancer. J Clin Oncol 2016;34:1732–40. 10.1200/JCO.2015.63.2232
    1. Zafar SY, McNeil RB, Thomas CM, et al. . Population-Based assessment of cancer survivors' financial burden and quality of life: a prospective cohort study. JOP 2015;11:145–50. 10.1200/JOP.2014.001542
    1. Delgado-Guay M, Ferrer J, Rieber AG, et al. . Financial distress and its associations with physical and emotional symptoms and quality of life among advanced cancer patients. Oncologist 2015;20:1092–8. 10.1634/theoncologist.2015-0026
    1. Ramsey SD, Bansal A, Fedorenko CR, et al. . Financial Insolvency as a risk factor for early mortality among patients with cancer. Journal of Clinical Oncology 2016;34:980–6. 10.1200/JCO.2015.64.6620
    1. Büttner M, König H-H, Löbner M, et al. . Out-of-pocket-payments and the financial burden of 502 cancer patients of working age in Germany: results from a longitudinal study. Support Care Cancer 2019;27:2221–8. 10.1007/s00520-018-4498-1
    1. Ezeife DA, Morganstein BJ, Lau S, et al. . Financial burden among patients with lung cancer in a Publically funded health care system. Clin Lung Cancer 2019;20:231-236 10.1016/j.cllc.2018.12.010
    1. Honda K, Gyawali B, Ando M, et al. . A prospective survey of comprehensive score for financial toxicity in Japanese cancer patients: report on a pilot study. Ecancermedicalscience 2018;12:847 10.3332/ecancer.2018.847
    1. Newton JC, Johnson CE, Hohnen H, et al. . Out-Of-Pocket expenses experienced by rural Western Australians diagnosed with cancer. Support Care Cancer 2018;26:3543–52. 10.1007/s00520-018-4205-2
    1. de Souza JA, Yap BJ, Hlubocky FJ, et al. . The development of a financial toxicity patient-reported outcome in cancer: the cost measure. Cancer 2014;120:3245–53. 10.1002/cncr.28814
    1. Altice CK, Banegas MP, Tucker-Seeley RD, et al. . Financial Hardships experienced by cancer survivors: a systematic review. J Natl Cancer Inst 2016.
    1. de Souza JA, Yap BJ, Wroblewski K, et al. . Measuring financial toxicity as a clinically relevant patient-reported outcome: the validation of the comprehensive score for financial toxicity (cost). Cancer 2017;123:476–84. 10.1002/cncr.30369
    1. Honda K, Gyawali B, Ando M, et al. . Prospective survey of financial toxicity measured by the comprehensive score for financial toxicity in Japanese patients with cancer. J Glob Oncol 2019;5:1–8. 10.1200/JGO.19.00003
    1. Scott F Huntington BMW, DAN T Vogl, ADAM D Cohen, Alfred L Garfall, Patricia a Mangan, Jalpa a Doshi, Edward a Stadtmauer. financial toxicity in insured patients with multiple myeloma: a cross-sectional pilot study. Lancet Haematol 2015;2:e408–16.
    1. Perrone F, Jommi C, Di Maio M, et al. . The association of financial difficulties with clinical outcomes in cancer patients: secondary analysis of 16 academic prospective clinical trials conducted in Italy. Annals of Oncology 2016;27:2224–9. 10.1093/annonc/mdw433
    1. Patrick DL, Burke LB, Gwaltney CJ, et al. . Content Validity—Establishing and reporting the evidence in newly developed patient-reported outcomes (pro) instruments for medical product evaluation: ISPOR pro good research practices Task force report: part 2—Assessing Respondent understanding. Value in Health 2011;14:978–88. 10.1016/j.jval.2011.06.013
    1. Patrick DL, Burke LB, Gwaltney CJ, et al. . Content validity--establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: part 1--eliciting concepts for a new PRO instrument. Value Health 2011;14:967–77. 10.1016/j.jval.2011.06.014
    1. Onwuegbuzie AJ, Dickinson WB, Leech NL, et al. . A qualitative framework for collecting and analyzing data in focus group research. Int J Qual Methods 2009;8:1–21. 10.1177/160940690900800301
    1. Glaser BG. Theoretical sensitivity. Mill Valley, CA: Sociology Press, 1978.
    1. Glaser BG. Discovery of grounded theory. Chicago, IL: Aldine, 1992.
    1. Stull DE, Leidy NK, Parasuraman B, et al. . Optimal recall periods for patient-reported outcomes: challenges and potential solutions. Curr Med Res Opin 2009;25:929–42. 10.1185/03007990902774765
    1. Heritage J. Conversation analysis and institutional talk: Analyzing data : Silverman D, Qualitative research: theory, method, and practice. Thousand Oaks, CA: Sage, 2004: 222–45.
    1. Onwuegbuzie AJ. Typology of mixed methods sampling designs in social science research. The Qualitative Report 2007;12:281–316.
    1. Walter SD, Eliasziw M, Donner A. Sample size and optimal designs for reliability studies. Stat Med 1998;17:101–10. 10.1002/(SICI)1097-0258(19980115)17:1<101::AID-SIM727>;2-E
    1. Aaronson NK, Ahmedzai S, Bergman B, et al. . The European organization for research and treatment of cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst 1993;85:365–76. 10.1093/jnci/85.5.365

Source: PubMed

Patrocinadores y Colaboradores

Condiciones médicas

Intervenciones de drogas

3
Suscribir