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. 2024 Apr;33(4):1075-1084.
doi: 10.1007/s11136-023-03587-8. Epub 2024 Jan 24.

Administering selected subscales of patient-reported outcome questionnaires to reduce patient burden and increase relevance: a position statement on a modular approach

Daniel Serrano  1   2 , David Cella  3 , Don Husereau  4 , Bellinda King-Kallimanis  5 , Tito Mendoza  6   7 , Tomas Salmonson  8 , Arthur Stone  9 , Alexandra Zaleta  10   11 , Devender Dhanda  12 , Andriy Moshyk  12 , Fei Liu  12 , Alan L Shields  13 , Fiona Taylor  13 , Sasha Spite  13   14 , James W Shaw  12 , Julia Braverman  12   15
Affiliations

Administering selected subscales of patient-reported outcome questionnaires to reduce patient burden and increase relevance: a position statement on a modular approach

Daniel Serrano et al. Qual Life Res. 2024 Apr.

Abstract

Patient-reported outcome (PRO) questionnaires considered in this paper contain multiple subscales, although not all subscales are equally relevant for administration in all target patient populations. A group of measurement experts, developers, license holders, and other scientific-, regulatory-, payer-, and patient-focused stakeholders participated in a panel to discuss the benefits and challenges of a modular approach, defined here as administering a subset of subscales out of a multi-scaled PRO measure. This paper supports the position that it is acceptable, and sometimes preferable, to take a modular approach when administering PRO questionnaires, provided that certain conditions have been met and a rigorous selection process performed. Based on the experiences and perspectives of all stakeholders, using a modular approach can reduce patient burden and increase the relevancy of the items administered, and thereby improve measurement precision and eliminate wasted data without sacrificing the scientific validity and utility of the instrument. The panelists agreed that implementing a modular approach is not expected to have a meaningful impact on item responses, subscale scores, variability, reliability, validity, and effect size estimates; however, collecting additional evidence for the impact of context may be desirable. It is also important to recognize that adequate rationale and evidence (e.g., of fit-for-purpose status and relevance to patients) and a robust consensus process that includes patient perspectives are required to inform selection of subscales, as in any other measurement circumstance, is expected. We believe that the considerations discussed within (content validity, administration context, and psychometric factors) are relevant across multiple therapeutic areas.

Keywords: Clinical trial; Modular approach; Oncology; Patient-reported outcome; Questionnaire.

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Conflict of interest statement

A.Z.: Cancer Support Community, Research and Training Institute (employment); institutional research funding from Astellas, Boston Scientific Foundation, Gilead Sciences, Novartis, Pfizer Inc., and Seagen Inc.; Bristol Myers Squibb (BMS) (consultancy). A.S.: Adelphi Values (consultancy) and the Gallup Organization (senior scientist). B.K.: LUNGevity Foundation (employment), AstraZeneca (grants), G1 Therapeutics (grants), BMS (grants), Merck (grants), Blueprint Medicine (grants), Eli Lilly (grants and consultancy), Genentech (grants), Takeda (grants), Jazz Pharmaceuticals (grants), Novartis (grants), Jansen (grants), Health Outcome Solutions (consultancy), and Atheneum (consultancy). D.S.: The Psychometrics Team (employment), Pharmerit International (employment), Vedanta Research (consultancy), and BMS (consultancy). D.C.: FACITtrans, LLC, Functional Assessment of Chronic Illness Therapy (FACIT.org), PROMIS Health Organization, AbbVie, Inc. (consultancy), Alexion Pharmaceuticals, Inc. (consultancy), Amgen, Inc. (consultancy), Astellas Pharma Global Development, Inc. (Astellas Pharma US, Inc.) (consultancy), Black Diamond Therapeutics (consultancy), Boehringer Ingelheim Pharmaceuticals, Inc. (Boehringer Ingelheim GmbH) (consultancy), BMS (consultancy), Celcuity, Inc. (consultancy), Corcept Therapeutics (consultancy), Daiichi Sankyo Company, Limited (consultancy), Disc Medicine (consultancy), Dompe (consultancy), Eisai, Inc. (consultancy), Eli Lilly and Company (consultancy), Evidera (consultancy), Genzyme Corporation (consultancy), Georgetown University (consultancy), GlaxoSmithKline (consultancy), H. Lee Moffitt Cancer Center and Research Institute (consultancy), Inmagene LLC (consultancy), Kiniksa Pharmaceuticals (consultancy), Leidos (consultancy), MD Anderson (consultancy), MEI Pharma (consultancy), Merck and Co., Inc. (consultancy), National Cancer Institute (consultancy), Novartis (consultancy), RAND Corporation (consultancy), Rafael Pharmaceuticals (consultancy), Takeda Pharmaceuticals North America, Inc. and Novo Nordisk A/S (consultancy), University of Arkansas for Medical Sciences (consultancy), UpToDate, Inc. (consultancy), and Yale School of Medicine (consultancy). D.H.: University of Ottawa (faculty), ISPOR (consultancy), and BMS (consultancy). T.M.: University of Texas MD Anderson Cancer center (faculty), Amgen, Inc (consultancy), and BMS (consultancy). T.S.: Consilium Salmonson and Hemmings (employment) and BMS (consultancy). J.B.: BMS (employment and stock ownership). A.M.: BMS (employment and stock ownership). D.D.: BMS (employment and stock ownership). F.L.: BMS (employment and stock ownership). J.S.: BMS (employment and stock ownership). A.S.: Adelphi Values (employment). F.T.: Adelphi Values (employment). S.S.: Adelphi Values (employment).

Figures

Fig. 1
Fig. 1
Administration of select subscales of the FACT-G using a modular approach. aGP5 item within PWB subscale can be administered as a standalone item to assess the concept of perceived bother with side effects of treatment
See this image and copyright information in PMC

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References

    1. Mercieca-Bebber R, King MT, Calvert MJ, Stockler MR, Friedlander M. The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Related Outcome Measures. 2018;9:353–367. doi: 10.2147/prom.s156279. - DOI - PMC - PubMed
    1. Coleman RL, Beck JT, Baranda JC, Jacobs I, Smoyer KE, Lee LJ, Askerova Z, McGinnis J, Ganti AK. The use of patient-reported outcome measures in phase I oncology clinical trials. Oncology. 2021;99(7):444–453. doi: 10.1159/000514874. - DOI - PubMed
    1. Atkinson TM, Schwartz CE, Goldstein L, Garcia I, Storfer DF, Li Y, Zhang J, Bochner BH, Rapkin BD. Perceptions of response burden associated with completion of patient-reported outcome assessments in oncology. Value in Health. 2019;22(2):225–230. doi: 10.1016/j.jval.2018.07.875. - DOI - PMC - PubMed
    1. Aiyegbusi OL, Roydhouse J, Rivera SC, Kamudoni P, Schache P, Wilson R, Stephens R, Calvert M. Key considerations to reduce or address respondent burden in patient-reported outcome (PRO) data collection. Nature Communications. 2022;13(1):6026. doi: 10.1038/s41467-022-33826-4. - DOI - PMC - PubMed
    1. Williamson PR, Altman DG, Bagley H, Barnes KL, Blazeby JM, Brookes ST, Clarke M, Gargon E, Gorst S, Harman N, Kirkham JJ, McNair A, Prinsen CAC, Schmitt J, Terwee CB, Young B. The COMET handbook: version 1.0. Trials. 2017;18(3):280. doi: 10.1186/s13063-017-1978-4. - DOI - PMC - PubMed

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