{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T02:12:58Z","timestamp":1791339178314,"version":"4.3.1"},"reference-count":56,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2022,8,12]],"date-time":"2022-08-12T00:00:00Z","timestamp":1660262400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Med."],"abstract":"<jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Cancer and corresponding available treatments are associated with substantial symptoms and functional limitations. In this context, collection of patient-reported outcomes (PRO) in clinical trials gained special interest and is recommended by regulatory authorities. Within clinical trials framework, PRO may provide evidence to support medicines approval, labeling and marketing claims. This study aims to analyze the existing evidence based on PRO as part of new oncology indications receiving positive opinions issued by the European Medicines Agency (EMA) between 2017 and 2020 and to identify PRO related label claims granted.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methodology<\/jats:title>\n                    <jats:p>Oncology medicinal products and indications approved by the European Commission following a positive opinion from the EMA between 2017 and 2020 were identified. European Public Assessment Report (EPAR) and Summary of Product Characteristics (SmPC) were reviewed for each medicinal product to identify use of PRO and PRO label claims.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>A total of 128 oncology indications, corresponding to 76 medicines, were approved; of those, 100 (78.1%) included PRO in the confirmatory clinical trials. Thirty-seven indications were supported by double-blind randomized trials and the remainder 63 by open-label trials. Out of the 104 confirmatory trials analyzed, PRO were defined as a secondary endpoint in 60 studies (57.7%), exploratory in 31 (29.8%) and as both in 13 (12.5%). In total, 54 different PRO measures (PROM) were used, of those 41 (75.9%) were disease-specific measures. Nevertheless, PROM selected relied on the EORTC (41.3%), FACIT (17.1%) and EQ-5D (29.2%) measures. A total of 76 indications (59.4%) had PRO reviewers comments included in the EPAR, however only 22 indications (17.8%) included label claims in the SmPC. The reasons identified in the EMA assessment supporting the exclusion of PRO claims were described for 34 indications (44.7%).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Despite growing recognition of the value of PRO data for the development of improved cancer therapies, PRO implementation remains challenging. The main reasons identified in our study are related with study design, missing data, study conduct and PROM selection.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3389\/fmed.2022.968272","type":"journal-article","created":{"date-parts":[[2022,8,12]],"date-time":"2022-08-12T01:28:25Z","timestamp":1660267705000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":36,"title":["A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020"],"prefix":"10.3389","volume":"9","author":[{"given":"Maria Manuel","family":"Teixeira","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"F\u00e1bio Cardoso","family":"Borges","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula Sousa","family":"Ferreira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jo\u00e3o","family":"Rocha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bruno","family":"Sepodes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla","family":"Torre","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2022,8,12]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"203","DOI":"10.1016\/j.jval.2018.09.2842","article-title":"A Review of patient-reported outcomes labeling for oncology drugs approved by the FDA and the EMA (2012\u20132016)","volume":"22","author":"Gnanasakthy","year":"2019","journal-title":"Value Heal"},{"key":"B2","doi-asserted-by":"publisher","first-page":"67","DOI":"10.1200\/EDBK_159514","article-title":"Patient-reported outcomes in cancer clinical trials : cancer institute' s patient-reported outcomes version","volume":"35","author":"Kluetz","year":"2016","journal-title":"Am Soc Clin Oncol"},{"key":"B3","doi-asserted-by":"publisher","first-page":"41","DOI":"10.1111\/hex.12997","article-title":"Including the patient voice in the development and implementation of patient-reported outcomes in cancer clinical trials","volume":"23","author":"Addario","year":"2020","journal-title":"Heal Expect."},{"key":"B4","doi-asserted-by":"publisher","first-page":"61","DOI":"10.4137\/HSI.S11093","article-title":"Article commentary: patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs)","volume":"6","author":"Weldring","year":"2013","journal-title":"Heal Serv Insights."},{"key":"B5","doi-asserted-by":"publisher","first-page":"57","DOI":"10.1007\/978-3-030-01207-6_5","article-title":"Patient-reported outcomes in oncology, beyond randomized controlled trials","volume":"213","author":"Toumi","year":"2019","journal-title":"Recent Results Cancer Res."},{"key":"B6","first-page":"1","article-title":"EMEA Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man","volume":"44","year":"2014"},{"key":"B7","volume-title":"Understanding Patient-Reported Outcomes in Cancer Trials: A Beginner's Guide","author":"Desai","year":"2017"},{"key":"B8","doi-asserted-by":"publisher","first-page":"353","DOI":"10.2147\/PROM.S156279","article-title":"The importance of patient-reported outcomes in clinical trials and strategies for future optimization","volume":"9","author":"Mercieca-Bebber","year":"2018","journal-title":"Patient Relat Outcome Meas"},{"key":"B9","doi-asserted-by":"publisher","first-page":"65","DOI":"10.1245\/s10434-019-07749-2","article-title":"Translation of patient-reported outcomes in oncology clinical trials to everyday practice","volume":"27","author":"Ivatury","year":"2020","journal-title":"Ann Surg Oncol."},{"key":"B10","volume-title":"Patient-Reported Outcome Measures Literature Review","author":"Williams","year":"2016"},{"key":"B11","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1007\/s11136-015-1099-z","article-title":"Reflection Paper on the use of patient reported outcome (PRO) measures in oncology studies","volume":"44","year":"2014","journal-title":"Eur Med Agency Sci Med Heal"},{"key":"B12","doi-asserted-by":"publisher","first-page":"1015","DOI":"10.1111\/hex.13254","article-title":"Patient-reported outcome measures (PROMs): a review of generic and condition-specific measures and a discussion of trends and issues","volume":"24","author":"Churruca","year":"2021","journal-title":"Heal Expect."},{"key":"B13","unstructured":"Patient-reported outcome measures: an overview.1. 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