On the Issue of Developing PROM as Part of the Value-based Healthcare

Background. Value-based healthcare is becoming the main theme of restructuring and improving modern healthcare systems around the world. The value of medical care for the patient is at the forefront; therefore, in order to make effective decisions, it is important to measure the effect of the treatment by taking into account the data reported by patients. This article provides a brief description of the concepts of patient-reported outcomes (PROs), indicators for measuring them (PROMs) and their significance in value-based healthcare. Based on the analysis of foreign publications, the basic principles and stages of the formation of PROMs with detailed descriptions of separate elements are considered, and a scheme of the PROM development process is generated. Since the use of PROM in Russian healthcare has not yet widely used, but have proven effective in supporting value-based healthcare initiatives abroad, exploring methods for developing and selecting these measures is becoming a relevant trend. The goal is to analyze the approaches of foreign researchers to the development of PROM and choosing it for implementation in Russian healthcare. Materials and Methods. This review is based on the content analysis of scientific literature published in the Springer Nature databases (BMC journal portfolio) and eLibrary.ru. It includes publications on the problems of measuring the PROs and the process of their development selected using the following keywords: outcome indicators reported by patients, PROM, development, and value-based healthcare. Additionally, general scientific methods of cognition were used: comparison, generalization, system-structural and analytical methods. Results. This article considers the main types of patient-reported outcomes — PROs — and types of indicators for their measurement — PROMs. It also analyzes the results of research and opinions of foreign scientists on the development of PROM. The principles and stages of PROM development are systematized, and a scheme of the PROM development process for any areas of measurement and application is generated — a possible methodological basis for the development and implementation of PROM in the Russian healthcare market.

1. Kharisov A.S., Sukhoterin A.F., Seleznev P.S., et al. Health Transformation to a Parent-Oriented Model. Bulletin of Semashko National Research Institute of Public Health. 2019;3-4:96-104. https://doi.org/10.25742/NRIPH.2019.03.011. (In Russ.)

2. Musina N.Z., Omel'janovskij V.V., Gostishhev R.V., et al. The concept of value-based healthcare. Farmakoekonomika. Modern Pharmacoeconomics and Pharmacoepidemiology. 2020;13(4):438-451. https://doi.org/10.17749/2070-4909/farmakoekonomika.2020.042. (In Russ.)

3. Muhina S.М., Orlova E.V. Patient-reported outcomes: an overview of application directions. Real-World Data & Evidence. 2022; 2(2):1-7. https://doi.org/10.37489/2782-3784-myrwd-12. (In Russ.)

4. Galletta M., Piazza M.F., Meloni S.L., Chessa E., Piras I., Arnetz J.E., D'Aloja E. Patient Involvement in Shared Decision-Making: Do Patients Rate Physicians and Nurses Differently? Int J Environ Res Public Health. 2022 Oct 31;19(21):14229. https://doi.org/10.3390/ijerph192114229. PMID: 36361109; PMCID: PMC9656720.

5. Moloczij N., Gough K., Solomon B., et al. Development of a hospital-based patient-reported outcome framework for lung cancer patients: a study protocol. Health Qual Life Outcomes. 2018;16:10. https://doi.org/10.1186/s12955-017-0837-z

6. Wilson R. Patient led PROMs must take centre stage in cancer research. Res Involv Engagem 2018;4:7. https://doi.org/10.1186/s40900-018-0092-4

7. Kargo A.S., Coulter, A., Jensen P.T., et al. Proactive use of PROMs in ovarian cancer survivors: a systematic review. J Ovarian Res 2019;12:63. https://doi.org/10.1186/s13048-019-0538-9

8. Moss, M.C.L., Aggarwal, A. Qureshi A., et al. An assessment of the use of patient reported outcome measurements (PROMs) in cancers of the pelvic abdominal cavity: identifying oncologic benefit and an evidence-practice gap in routine clinical practice. Health Qual Life Outcomes. 2021;19:20. https://doi.org/10.1186/s12955-020-01648-x

9. Sørensen N.L., Hammeken L.H., Thomsen J.L., et al. Implementing patient-reported outcomes in clinical decision-making within knee and hip osteoarthritis: an explorative review. BMC Musculoskelet Disord. 2019;20:230. https://doi.org/10.1186/s12891-019-2620-2

10. Pronk Y., van der Weegen W., Vos R., et al. What is the minimum response rate on patient-reported outcome measures needed to adequately evaluate total hip arthroplasties?. Health Qual Life Outcomes. 2020;18:379. https://doi.org/10.1186/s12955-020-01628-1

11. Oliveira H.C., Hayashi D., Carvalho S.D.L., et al. Quality of measurement properties of medication adherence instruments in cardiovascular diseases and type 2 diabetes mellitus: a systematic review and metaanalysis. Syst Rev. 2023;12:222. https://doi.org/10.1186/s13643-023-02340-z

12. Coles T.M., Hernandez A.F., Reeve B.B., et al. Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials. Health Qual Life Outcomes. 2021;19:164. https://doi.org/10.1186/s12955-021-01800-1

13. Hat'kov I.E., Minaeva O.A., Domrachev S.A., et al. PROM is a modern approach to assessing the quality of life of patients with cancer. Therapeutic Archive. 2022; 94(1):122-128. https://doi.org/10.26442/00403660.2022.01.201343. (In Russ.)

14. van Silfhout N.Y., van Muilekom M.M., van Karnebeek C.D., et al. PROs for RARE: protocol for development of a core patient reported outcome set for individuals with genetic intellectual disability. Orphanet J Rare Dis. 2024;19:354. https://doi.org/10.1186/s13023-024-03264-0.

15. Methods for assessing the quality of life of patients with cardiac arrhythmias / V. I. Steklov, E. A. Kim, P. E. Krajnjukov et al. // Journal of Arrhythmology. 2021;28(1):55-63. https://doi.org/10.35336/VA-2021-1-55-63. (In Russ.)

16. Borovskaja V.G., Kasimova A.R. Review of guidelines from the FDA for patient involvement in the process of developing medical products and making regulatory decisions. Real-World Data & Evidence. 2023;3(2):1-9. https://doi.org/10.37489/2782-3784-myrwd-31. (In Russ.)

17. Narita A., Koto Y., Noto S., et al. Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan. Orphanet J Rare Dis. 2024;19:11. https://doi.org/10.1186/s13023-024-03264-0

18. Koto Y., Narita A., Noto S., et al. Qualitative analysis of patient interviews on the burden of neuronopathic Gaucher disease in Japan. Orphanet J Rare Dis. 2022;17:280. https://doi.org/10.1186/s13023-022-02429-z.

19. Kirkham J.J., Gorst S., Altman D.G., et al. Core Outcome Set-STAndardised Protocol Items: the COS-STAP Statement. Trials. 2019;20:116. https://doi.org/10.1186/s13063-019-3230-x

20. Müller A.R., Luijten M.A.J., Haverman L., et al. Understanding the impact of tuberous sclerosis complex: development and validation of the TSC-PROM. BMC Med. 2023;21:298. https://doi.org/10.1186/s12916-023-03012-4

21. Xiaojuan Hu, Zhiqiang Zhao, Shao-Kai Zhang, Yanhong Luo, Hongmei Yu, Yanbo Zhang. CA-PROM: Validation of a general patient-reported outcomes measure for Chinese patients with cancer. Cancer Epidemiol. 2020;67:101774. https://doi.org/10.1016/j.canep.2020.101774

22. Spennacchio P., Senorski E.H., Mouton C., et al. A new patient-reported outcome measure for the evaluation of ankle instability: description of the development process and validation protocol. J Orthop Surg Res. 2024;19:557. https://doi.org/10.1186/s13018-024-05057-4

23. Guruparan Y., Navaratinaraja T.S., Selvaratnam G., et al. Development and validation of a set of patient reported outcome measures to assess effectiveness of asthma prophylaxis. BMC Pulm Med. 2021;21:295. https://doi.org/10.1186/s12890-021-01665-6