Communicating clinical trial information about cancer drug products to patients and healthcare professionals: review of patient-reported outcome messaging

WHAT IS ALREADY KNOWN ON THIS TOPIC Patient-reported outcomes (PROs) are routinely included in oncology trials to assess symptoms, functioning and quality of life. However, prior evidence shows that PRO findings are infrequently incorporated into regulatory drug labels and are inconsistently communicated beyond regulatory submissions, limiting their practical use in patient-centred cancer care.WHAT THIS STUDY ADDS This review of 128 pivotal trials in breast, gastrointestinal and non-small cell lung cancers shows that although most trials included PRO endpoints, only a small proportion resulted in regulatory PRO label claims. Across sponsor websites, professional society platforms and patient advocacy resources, PRO messaging was generally sparse, inconsistently presented and rarely available on patient-facing product sites.HOW THIS STUDY MIGHT AFFECT RESEARCH, PRACTICE OR POLICY The findings highlight the need to design pivotal trials with prespecified, analytically robust PRO endpoints to support regulatory inclusion and responsible post-approval communication. Improved adherence to methodological standards and clearer, balanced PRO messaging may enhance the clinical and regulatory value of PRO data and support informed decision-making in oncology.Introduction Patient-reported outcomes (PROs) offer valuable insights into the effectiveness and tolerability of therapies from the patient’s perspective. 1 Promotional materials—including patient brochures, social media advertising, websites, promotional exhibits and materials distributed at major medical meetings and pharmaceutical conventions—incorporate clinical outcome assessment (COA) data to communicate information about drug products to physicians and patients. These data often highlight the impact of the disease (eg, symptoms) and therapies (eg, side effects) as experienced by patients, including PRO results from clinical trials. In the USA, such promotional content is subject to regulatory oversight by the Food and Drug Administration (FDA). The FDA’s Office of Prescription Drug Promotion is responsible for reviewing these materials and may take enforcement action against those deemed false, lacking fair balance or otherwise misleading.2 3 Unlike the FDA, the European Medicines Agency (EMA) does not directly review or approve promotional materials. Oversight of marketing communications in the European Union is largely delegated to self-regulatory codes and national competent authorities, resulting in greater variability in how PRO-related content is monitored across countries.From 2013 to 2021, the FDA issued 22 warning letters and 65 untitled letters. Warning letters are issued for violations of regulatory significance that require action and may lead to further enforcement if not addressed. Untitled letters are issued for violations that do not meet that threshold and for which a response is requested rather than mandated. In 2013, the highest number of letters was issued (n=24). Among the 22 letters citing COA-related violations, the most common issue was improper study design or misinterpretation of results (21 of 22; 95%), followed by problems with statistical analyses (3 of 22; 14%) and use of COA measures not deemed fit for purpose (3 of 22; 14%).3PROs play a critical role in cancer care by capturing patients’ experiences with symptoms, treatment side effects and overall quality of life—dimensions often not fully reflected in traditional clinical measures. For example, in metastatic breast cancer (mBC), where curative treatment is not an option, PROs help inform shared decision-making and guide symptom management to support improvements in quality of life. Therefore, PROs are incorporated into clinical trials, non-interventional studies and registries to better understand what matters most to patients.4Given their growing influence in drug development and regulatory approval, it is essential that PRO findings be communicated to both patients and healthcare professionals (HCPs) in a manner that is transparent, unbiased and tailored to the informational needs of each audience. While previous reviews have primarily focused on regulatory claims, violations and the comprehensibility of PRO content in labeling,5 there remains a critical gap in understanding how PRO trial data are translated into public-facing promotional materials, including branded websites, cancer society platforms and patient advocacy channels.This study aimed to (1) assess how pivotal oncology trials in BC, non-small cell lung cancer (NSCLC) and gastrointestinal (GI) cancers, including colorectal cancer (CRC) and gastro-oesophageal cancer (GEC), incorporated PRO endpoints as secondary or exploratory outcomes; (2) determine the extent to which these trials led to PRO-related labelling claims approved by the FDA and/or EMA; (3) analyse how PRO findings are leveraged in support of treatment claims and communicated through promotional channels including sponsor-branded websites, HCP portals, patient-facing webpages and advocacy or cancer society platforms and (4) identify any associated regulatory non-compliance issues related to PRO messaging.Methods This study followed a structured, multistage design to evaluate how PROs from oncology clinical trials are represented in regulatory and public communications. The approach combined regulatory review, online data extraction and qualitative synthesis across multiple communication domains. Each step and data source was defined a priori to ensure transparency and reproducibility.Defining the target drug scope We identified all drug products approved for adult patients with BC, GI cancers (GI, including colorectal and GECs), and NSCLC by the US FDA or the EMA between 1 January 2014 and 31 December 2024.Pivotal trials supporting each product’s approval were located through ClinicalTrials.gov using trial identifiers referenced in FDA or EMA labelling documents. When available, we extracted data directly from registered protocols; otherwise, information was taken from the ‘Study details’ and ‘Design’ sections. For every pivotal study, we recorded trial phase, blinding, comparator and whether a PRO instrument was prospectively included.A trial was coded as having ‘PRO evidence identified’ when at least one validated PRO instrument was listed in the registry or supporting documents as a primary, secondary or exploratory endpoint and summary results were publicly accessible through ClinicalTrials.gov, regulatory assessments or sponsor communications. Peer-reviewed journal articles were not systematically reviewed but were used for verification of instrument names or scoring methods when registry data were incomplete.Two analysts independently confirmed eligibility and PRO inclusion. Discrepancies were resolved by discussion. This ensured consistent identification of trials contributing to the subsequent analyses.Targeted search for pro-label claims and related messaging After establishing the analytic drug list, we conducted three coordinated searches to examine how PRO information was translated into public-facing content: (1) regulatory documentation, (2) sponsor-controlled websites and (3) external professional or patient-advocacy sources.Regulatory domain All FDA and EMA product labels were reviewed for explicit mention of PRO data within the ‘Clinical Studies,’ ‘Patient Counselling Information’ or other narrative sections. We noted the instrument type, endpoint designation, results summary and whether quantitative statistics (eg, HR or mean change) were provided. For the FDA, warning and untitled letters from 2013 to 2024 were retrieved from the Agency’s public database using each brand name. Instances where COA or PRO claims were cited as non-compliant were catalogued to understand patterns of regulatory enforcement.Sponsor-controlled domain Official branded websites directed to HCPs and patients were identified via Google searches restricted to the US domain (eg, ‘drug name site:.com hcp’ or ‘drug name site:.com patients’) supplemented by internal site-search tools. When global websites existed, they were also reviewed. We assessed whether PRO information appeared and, if so, how it was framed (symptom relief, functional improvement, quality of life or treatment burden). The presence of visuals, statistical context, disclaimers and alignment with labelled information was documented.External professional and advocacy domain To contextualise sponsor messaging, we searched professional association and patient-advocacy websites linked to each cancer type. Predefined organisations included American Society of Clinical Oncology (ASCO), European Society for Medical Oncology (ESMO) and National Comprehensive Cancer Network (NCCN) for all cancers; Breastcancer.org for BC; Colorectal Cancer Alliance and Gastric Cancer Foundation for GI; and the American Lung Association and Lung Cancer Europe for NSCLC. Selection of patient-advocacy organisations was guided by three criteria: (1) high web visibility and traffic metrics (based on Google ranking and citation frequency in peer-reviewed or policy documents); (2) established credibility through affiliation with recognised cancer consortia or long-standing educational initiatives and (3) regular publication of patient-directed resources relevant to oncology. Only English-language sites with publicly available content were included, while smaller regional or inactive groups were excluded. Each site was searched between January and June 2024 using combinations of the product’s brand and generic names, the relevant cancer type and PRO-related terms (‘quality of life,’ ‘patient-reported outcome,’ ‘HRQoL,’ ‘symptom’).All retrieved pages were archived with date stamps to ensure traceability, acknowledging that web content changes over time. All website searches across regulatory, sponsor and external domains were conducted between January and June 2024, and the captured versions were archived for verification. Subsequent updates to branded websites, such as the Trodelvy HCP page that later included quality-of-life content, occurred after this review period.Data screening and quality control Two independent raters screened all sources for eligible PRO mentions and extracted data into a structured spreadsheet. A subsample was cross-checked to ensure accuracy. Discrepancies were reconciled through consensus discussion; inter-rater reliability was not formally quantified because the aim was qualitative synthesis rather than quantitative meta-analysis.Case studies were selected purposively to exemplify variation across cancer types and information domains. A product qualified as a case study if at least one verifiable PRO message was identified in any of the three domains (regulatory, sponsor or external). Preference was given to cases illustrating regulatory compliance issues, innovative communication formats or divergent practices across audiences.Integration and rating framework All extracted data were triangulated to map the trajectory of PRO information from trial collection to external dissemination. The strength of PRO communication for each product was rated as low, medium or high, based on predefined qualitative criteria: low: PRO data collected but absent or minimally represented in communications; medium: PRO data present and discussed in ≥2 communication forms or accompanied by a regulatory label claim; high: PRO data integrated into labelling and consistently communicated across multiple public channels.Figure 1 summarises the overall workflow linking identification of pivotal trials, retrieval of regulatory materials, web-based data extraction and synthesis across domains. This three-tier framework was adapted from prior oncology communication analyses6 and refined through iterative consensus between two independent reviewers to ensure consistency.Figure 1Overview of data sources and analytical workflow sequential process used to evaluate PRO integration and communication across regulatory, sponsor-controlled and external platforms: (1) identify oncology drugs approved 2014–2024 (FDA/EMA); (2) retrieve pivotal trials from ClinicalTrials.gov and extract PRO endpoints; (3) collect regulatory labels and FDA compliance letters; (4) search sponsor HCP and patient websites; (5) search professional and advocacy sources (ASCO, ESMO, NCCN, etc); (6) triangulate data and rate PRO messaging strength and (7) develop illustrative case studies. ASCO, American Society of Clinical Oncology; BC, breast cancer; EMA, European Medicines Agency; FDA, Food and Drug Administration; GI, gastrointestinal; HCPs, healthcare professionals; NSCLC, non-small cell lung cancer; PRO, patient-reported outcome.Insights synthesis The final phase of the analysis involved synthesising findings across the three cancer types to characterise the landscape of PRO messaging in oncology. This synthesis included an evaluation of the extent and nature of PRO data collection in pivotal trials, the presence and specificity of PRO-related claims in FDA and EMA labels, and the use of PRO messaging on branded websites available to HCPs and patients.We also analysed how PROs were communicated by medical associations and Patient Advocacy Organizations (PAGs), identifying patterns in terminology, emphasis and alignment with regulatory and clinical trial data. For each cancer type, we developed in-depth case studies for products, medical associations and PAGs that featured PRO messaging content. These case studies were designed to answer the three key topics outlined below.First, we examined how manufacturers share PRO messaging on brand websites. This included an assessment of whether PROs were presented as central to the product’s value proposition, whether they were framed in terms of symptom relief, functional improvement or improved quality of life, and whether the messaging was tailored to specific audiences.Second, we assessed how PROs were used to support treatment claims and whether these messages were backed by robust clinical evidence. This included evaluating whether the messaging was consistent with the data presented in pivotal trials and regulatory labels.Third, we explored whether PROs were used to differentiate products within crowded therapeutic classes. In doing so, we categorised PRO messaging strength into three levels. A low rating indicated limited or no PRO data collection and minimal or no related communications. A medium rating reflected the presence of PRO data from clinical trials, accompanied by either at least two distinct forms of communication or one form that included a PRO label claim. A high rating was assigned when PRO data collection was supported by PRO label claims and comprehensive communication efforts across multiple channels.Results Overall PRO features PRO measures were identified in 128 pivotal trials (28 breast, 34 GI and 66 NSCLC) approved between 2014 and 2024. Most trials (79) were open-label and 49 were double-blind. PRO endpoints appeared in 105 trials (82%), primarily as secondary (84) or exploratory (40) outcomes. The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30- item (EORTC QLQ-C30) and its disease-specific modules were the most frequently used tools, followed by the EQ-5D. Table 1 summarises these characteristics by cancer type.Table 1Overall PRO features of approved pivotal breast, colorectal and gastro-oesophageal cancers approved by the regulatory between 2014 and 2024Cancer and subtypeNumber of trialsPRO as secondary endpointPRO as exploratory endpointOpen label versus double blindApplied PRO instrumentsEORTC-QLQ-C30Cancer-specific EORTCEQ-5DOther instrumentsBreast cancer TNBC4421 vs 34320 Early Ca. (1)3202 vs 11010 HR+/HER2−8831 vs 77474 Metastatic HER2+6425 vs 14461 BRCA ½ Mutated HER2, locally advanced, or metastatic3212 vs 13101 PIK3CA-mutated3310 vs 33201 Other mutation set1101 vs 01011All breast Ca.2824912 vs 162314178GI cancers Colorectal (2)13529 vs 47172 Gastro-oesophageal (3)211279 vs 121210157All GI Ca.3417918 vs 161911229NSCLCAll NSCLC (4)66432249 vs 1741353326All studied cancers128844079 vs 49836072431. One trial did not have information on PRO use.2. No PRO endpoint was identified in 6 of 13 trials.3. No PRO endpoint was identified in 3 of 21 trials.4. No PRO endpoint was identified in 13 of 66 trials.GI, gastrointestinal; NSCLC, non-small cell lung cancer; PRO, patient-reported outcome; TNBC, triple negative breast cancer.BC PRO messaging All 16 approved BC medications included some PRO evidence (neratinib limited to metastatic HER2+ disease), though only four contained regulatory label information: ribociclib (Kisqali), abemaciclib (Verzenio) and palbociclib (Ibrance) (EMA only), and inavolisib (Itovebi) (FDA only). PRO narratives were identified on HCP websites for ribociclib (Kisqali) (early BC and metastatic HR+/HER2- BC) and alpelisib (Piqray) (PIK3CA-mutated BC), both of which lacked regulatory PRO labelling in at least one BC indication. No information elicited from PRO measures was found on patient-facing websites for any of the 16 medications. However, PRO-related messages were published for 10 of the 16 medications on external platforms such as ESMO, ASCO and BreastCancer.org. The content of PRO information was categorised as low for 13 medication-indication pairs, medium for 7, and high for none. Table 2 presents a visual summary of the distribution of PRO messaging across stakeholder groups, along with additional details on the nature and content of PRO communications directed towards each stakeholder.Table 2Breast cancer PRO messaging stakeholder distributions by type of pivotal trial between 2014 and 2024IndicationTrialPRO evidencePRO FDA claimPRO EMA claimPRO messaging for HCPPRO messaging for patientsPRO messaging for professional associationContent of PRO messaging*Triple negative breast cancer sacituzumab govitecan-hziy (Trodelvy)ASCENT✓✗✗✗✗✗Low pembrolizumab (Keytruda)KEYNOTE 355, 522✓✗✗✗✗✓8Low atezolizumab (Tecentriq)IMpassion 130✓✗✗✗✗✗LowEarly breast cancer ribociclib (Kisqali)NATALEE✓✗✗✓5✗✓9Medium abemaciclib (Verzenio)MONARCHE✓✗✗✗✗✗Low neratinib (Nerlynx)EXTENET✗✗✗✗✗✗LowMetastatic HR+/HER- breast cancer sacituzumab govitecan-hziy (Trodelvy)TROPICS-02✓✗✗✗✗✓10Medium ribociclib (Kisqali)MONALEESA 2,3,7✓✗warn✓2✓6✗✓11Medium abemaciclib (Verzenio)MONARCH-2, 3✓✗✓3✗✗✓12Medium palbociclib (Ibrance)PALOMA-2, 3✓✗✓4✗✗✓13MediumMetastatic HER+breast cancer fam-trastuzumab deruxtecan-nxki (Enhertu)DESTINY-BREAST 02, 03, 04✓✗✗✗✗✓14Medium tucatinib (Tukysa)HER2CLIMB (phase 2)✓✗✗✗✗✓15Low margetuximab-cmkb (Margenza)SOPHIA✓✗✗✗✗✗Low neratinib (Nerlynx)NALA✓✗✗✗✗✗LowGermline BRCA1/2 Olaparib (Lynparza)OLYMPIAD, OLYMPIA✓✗✗✗✗✓16Low talazoparib (Talzenna)EMBRACA✓✗✗✗✗✓17MediumPIK3CA-related breast cancer Inavolisib (Itovebi)INAVO120✓✓1✗✗✗✗Low capivasertib (Truqap)CAPITELLO-291✓✗✗✗✗✗Low Alpelisib (Piqray)SOLAR-1✓✗✗✓7✗✗LowOther elacestrant (Orserdu)EMERALD✓✗✗✗✗✓18Lowwarn. FDA TV Ad non-compliance.1. Reporting PRO-CTCAE results.2. No difference in GHS/QoL (ML-2 only).3. No difference in HRQoL (MONARCH-2 only).4. Benefit in delaying time to deterioration in pain.5. US and Global: EORTC QLQ-C30.6. US and Global: time to deterioration results.7. Outside US only—time to deterioration GHS/QoL results.8. ESMO daily reporter.9. ESMO virtual plenary with AACR experts.10.ESMO daily reporter, oncology news, Breastcancer.org research news and podcasts.11. ASCO daily news.12. ASCO daily news, Breastcancer.org podcasts.13. Breastcancer.org research news.14. ESMO daily reporter, oncology news, ASCO daily news on ePRO study, Breastcancer.org research news and podcasts.15. Breastcancer.org research news.16. NCCN guidelines.17. ESMO oncology news, Breastcancer.org podcasts.18. ESMO daily reporter news. Of note, the Trodelvy (sacituzumab govitecan-hziy) HCP website was reviewed between January and June 2024. Quality-of-life content currently available online was not present during the data-collection period.*Low rating: Limited or no PRO data have been collected, and minimal or no PRO communications are available, medium rating: PRO data have been collected in trials, accompanied by some level of PRO communication (≥2 different form of communication or 1 form of communication with PRO label claim). High rating: PRO data have been collected, which is complemented by PRO label claims and comprehensive PRO communication efforts. AACR, American Association for Cancer Research; ASCO, American Society of Clinical Oncology; CTCAE, Common Terminology Criteria for Adverse Events; EMA, European Medicines Agency; FDA, Food and Drug Administration; GHS, global health status; HCP, healthcare professional; HRQoL, health-related quality of life; PRO, patient-reported outcome; QoL, quality of life; TV, television.GI cancer PRO messaging Among 12 approved GI cancer medications (8 CRC and 8 GEC), 10 included PRO evidence, but only ramucirumab (Cyramza) carried a PRO label claim with the EMA in CRC. Limited PRO narratives appeared on HCP websites for two drugs, while only fruquintinib (Fruzaqla) featured patient-facing content. External platforms such as ESMO and ASCO mentioned PROs for two medications. Overall evidence strength was rated low for nearly all products, with fruquintinib (Fruzaqla) in CRC rated medium. Table 3 summarises PRO messaging patterns across stakeholder groups.Table 3GI cancer PRO messaging stakeholder distributions by type of pivotal trial between 2014 and 2024IndicationStudyPRO evidencePRO FDA claimPRO EMA claimPRO messaging for HCPPRO messaging for patientsPRO messaging for professional associationsContent of PRO messagingCRCs pembrolizumab (Keytruda)KEYNOTE-164 (phase 2), KEYNOTE-177✓1✗✗✗✗✓14Low nivolumab (Opdivo)CHECKMATE-8HW, CHECKMATE-142 (phase 2)✗✗✗✗✗✓15Low ramucirumab (Cyramza)RAISE✓✗✓6✗✗✗Low fruquintinib (Fruzaqla)FRESCO, FRESCO2✓2✗✗✓11✓13✗Medium encorafenib (Braftovi)BEACON, BREAKWATER✓✗✗7✗✗✗Low tucatinib (Tukysa)MOUNTAINEER (phase 2)✓✗✗8✗✗✗Low adagrasib (Krazati)KRYSTAL-1 (phase 1/2)✗✗✗9✗✗✗Low trifluridine and tipiracil (Lonsurf)RECOURSE, SUNLIGHT✓3✗✗✗✗✗LowGastro-oesophageal cancers pembrolizumab (Keytruda)KEYNOTE-859, 590, and 181KEYNOTE-811, 158 (phase 2) and 164 (phase 2)✓4✗✗10✗✗✓16Low nivolumab (Opdivo)CHECKMATE 649, 577, 648 and ATTRACTION-3✓✗✗✗✗✓17Low tislelizumab-jsgr (Tevimbra)RATIONALE-302, 305, and 306✓✗✗✗✗✗Low toripalimab-tpzi (Loqtorzi)JUPITER-06✓✗5✗✗✗✗Low Ramucirumab (Cyramza)RAINBOW, REGARD✓✗✗✓12✗✗Low fam-trastuzumab deruxtecan-nxki (Enhertu)DESTINY-Gastric01 (phase 2), DESTINY-Gastric02 (phase 2)✓✗✗✗✗✗Low zolbetuximab-clzb (Vyloy)SPOTLIGHT, GLOW✓✗✗✗✗✗Low trifluridine and tipiracil (Lonsurf)TAGS✓✗✗✗✗✗Low1. Evidence was absent from KEYNNOTE-164 (phase 2).2. Evidence was absent from FRESCO.3. Evidence was absent from RECOURSE.4. Evidence was absent from KEYNOTE-811, 158 (phase 2) and 164 (phase 2).5. The indication is not approved in the US.6. The EORTC QLQ-C30.7. The indication of Braftovi in combination with Erbitux (cetuximab) and mFOLFOX6 (fluorouracil, leucovorin and oxaliplatin), for the treatment of patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation is not approved in the EU (only in the USA)–BREAKWATER trial.8 and 9. The indication for mCRC is not approved in the EU (only in the USA).10. The indication of 2 L locally advanced or metastatic oesophageal squamous cell carcinoma for Keytruda is not approved in the EU (only in USA)–KEYNOTE-181 trial.11,12 and 13. EORTC QLQ-C30.14. ESMO oncology news.15. ESMO oncology news, ASCO Connection.16. ASCO daily news.17. ESMO daily reporter.*Low rating: Limited or no PRO data have been collected, and minimal or no PRO communications are available, Medium rating: PRO data have been collected in trials, accompanied by some level of PRO communication (≥2 different form of communication or 1 form of communication with PRO label claim). High rating: PRO data have been collected, which is complemented by PRO label claims and comprehensive PRO communication efforts.ASCO, American Society of Clinical Oncology; EMA, European Medicines Agency; EU, European Union; FDA, Food and Drug Administration; GI, gastrointestinal; HCP, healthcare professional; PRO, patient-reported outcome.NSCLC PRO messaging Of 28 approved NSCLC medications, 21 included PRO evidence and 6 carried regulatory PRO label claims. The EMA recognised durvalumab (Imfinzi), atezolizumab (Tecentriq), osimertinib (Tagrisso), ceritinib (Zykadia), ramucirumab (Cyramza), and nintedanib (Vargatef); the FDA also granted a claim to ceritinib (Zykadia). Limited HCP-facing PRO narratives appeared for two drugs, and only brigatinib (Alunbrig) featured patient-facing content. External platforms such as ESMO and ASCO referenced PROs for seven medications. PRO communication strength was generally low, with medium ratings for durvalumab (Imfinzi), atezolizumab (Tecentriq) and brigatinib (Alunbrig). Table 4 summarises these distributions.Table 4NSCLC PRO messaging stakeholder distributions by type of pivotal trial between 2014 and 2024IndicationStudyPRO evidencePRO FDA claimPRO EMA claimPRO messaging for HCPPRO messaging for patientsPRO messaging for professional associationsContent of PRO messagingcemiplimab-rwlc (Libtayo)EMPOWER-Lung 1, EMPOWER-Lung 3✓✗✗✓18✗✗Lowdurvalumab (Imfinzi)AEGEAN, PACIFIC, POSEIDON✓✗✓10✗✗✓30Mediumatezolizumab (Tecentriq)IMPOWER110, IMPOWER130, IMPOWER150, OAK, IPSOS IMPOWER010✓1✗✓11✗✗✓31Mediumpembrolizumab (Keytruda)KEYNOTE-189, 407, 024, 010, 671, 091 042✓2✗✗✗✗✗Lownivolumab (Opdivo)CHECKMATE-9LA, 017, 057, 227, 816, 77T✓✗✗✗✗✓32Lowtislelizumab-jsgr (Tevimbra)RATIONALE-303, 304, 307✓✗7✗✗19✗24✗LowSugemalimab (Cejemly)GEMSTONE-302✗✗8✗✗20✗25✗Lowosimertinib (Tagrisso)ADAURA, LAURA, FLAURA, FLAURA-2, AURA-3✓✗✓12✗✗✗Lowamivantamab-vmjw (Rybrevant)CHRYSALIS (phase 1) MARIPOSA-2, PAPILLON, MARIPOSA✓3✗✗13✗✗✗Lowlazertinib (Lazcluze)MARIPOSA✓✗✗14✗21✗26✗Lowdacomitinib (Vizimpro)ARCHER 1050✓✗✗✗✗✗Lowlorlatinib (Lorbrena/Lorviqua)B7461001 (phase 1/2), CROWN✓✗✗✗✗✓33LowAlectinib (Alecensa)ALINA, ALEX, ALUR✓✗✗✗✗✗Lowceritinib (Zykadia)ASCEND-4, ASCEND-5✓✓9✓15✗22✗27✗Lowbrigatinib (Alunbrig)ALTA (phase 2), ALTA-1L✓✗✗✓23✓28✗Mediumncorafenib+binimetinib (Braftovi+Mektovi)PHAROS (phase 2)✗✗✗✗✗✗Lowabrafenib+trametinib (Tafinlar+Mekinist)BRF113928 (phase 2)✗✗✗✗✗✗Lowrepotrectinib (Augtyro)TRIDENT-1 (phase 1/2)✗✗✗✗✗✓34Lowentrectinib (Rozlytrek)STARTRK-2 (phase 2) STARTRK-1 (phase 1) ALKA-372-001a✓4✗✗✗✗✗Lowtepotinib (Tepmetko)VISION (phase 2)✓✗✗✗✗✗Lowcapmatinib (Tabrecta)GEOMETRY (phase 2)✗✗✗✗✗29✗Lowselpercatinib (Retevmo/Retsevmo)LIBRETTO-001 (phase 1/2) LIBRETTO-431✓5✗✗✗✗✗Lowpralsetinib (Gavreto)ARROW (phase 1/2)✗✗✗✗✗✗LowOtorasib (Lumakras/Lumykras)CODEBREAK 100 (phase 1/2)✓✗✗✗✗✓35Low(Krazati)KRYSTAL-1 (phase 1/2) KRYSTAL-12✓6✗✗✗✗✓36Lowtrastuzumab deruxtecan (Enhertu)DESTINY-Lung01✗✗✗✗✗✗Lowramucirumab (Cyramza)REVEL, RELAY✓✗✓16✗✗✗Lownintedanib (Vargatef)LUME-Lung-1✓✗✓17✗✗✗Low1. Evidence was absent from IMPOWER010.2. Evidence was absent from KEYNOTE-042.3. Evidence was absent from CHRYSALIS (phase 1).4. Evidence was absent from STARTRK-2 (phase 2).5. Evidence was absent from LIBRETTO-001 (phase 1/2).6. Evidence was absent from KRYSTAL-1(phase 1/2).7. No marketing authorisation in the USA, indication not listed on the product-specific site.8. No marketing authorisation in the USA, product-specific site not identified.9. ASCEND-4 (1 L ALK+advanced NSCLC): EORTC QLQ-LC13, TTD, LCSS.10. PACIFIC (NSCLC): EORTC QLQ-C30 and EORTC QLQ-LC13.11. OAK (NSCLC): EORTC QLQ-LC13.12. ADAURA (Adjuvant treatment stage IB-IIIA NSCLC): SF-36, FLAURA, AURA-3 (Metastatic NSCLC): EORTC QLQ-C30 and EORTC QLQ-LC1.13. Recent marketing authorisation in the EU–product label not yet available.14. Recent marketing authorisation in the EU–product label not yet available.15. ASCEND-4 (1 L ALK+advanced NSCLC): EORTC QLQ-LC13, TTD, LCSS, GHS/HRQoL (EORTC QLQ-C30), EQ-5D-5L.16. REVEL (1 L treatment of NSCLC): LCSS.17. LUME-Lung-1 (locally advanced or metastatic NSCLC): HRQoL.18. EMPOWER-Lung 3 (NSCLC, combo tx): EORTC QLQ-C30 and EORTC QLQ-LC1.19. No marketing authorisation in the USA, indication not listed on the product-specific site.20. No marketing authorisation in the USA, product-specific site not identified.21. Medical information site (product label and other documents but no format brand site).22. Product label (no other information).23. ALTA-1L (ALK+metastatic NSCLC): GHS/HRQoL (EORTC QLQ-C30).24. No marketing authorisation in the USA, indication not listed on the product-specific site.25. No marketing authorisation in the USA, product-specific site not identified.26. Product label (no other information).27. Product label (no other information).28. ALTA-1L (ALK+metastatic NSCLC): GHS/HRQoL (EORTC QLQ-C30).29. Product label (no other information).30. ESMO daily reporter; ESMO oncology news; ASCO daily news.31. ASCO daily news.32. ESMO ECC 2015 press release, ASCO daily news.33. ESMO oncology news.34. ESMO oncology news.35. ESMO daily reporter.36. ASCO daily news.*Low rating: Limited or no PRO data have been collected, and minimal or no PRO communications are available, medium rating: PRO data have been collected in trials, accompanied by some level of PRO communication (≥2 different form of communication or 1 form of communication with PRO label claim). High rating: PRO data have been collected, which are complemented by PRO label claims and comprehensive PRO communication efforts.ASCO, American Society of Clinical Oncology; ECC, European Cancer Congress; EMA, European Medicines Agency; EU, European Union; FDA, Food and Drug Administration; GHS, global health status; HCP, healthcare professional; LCSS, Lung Cancer Symptom Scale; NSCLC, non-small-cell lung cancer; PRO, patient-reported outcome; TTD, Time to Deterioration.Case studies Regulatory labeling Nine oncology products were identified: ribociclib (Kisqali), inavolisib (Itovebi), palbociclib (Ibrance), abemaciclib (Verzenio), ramucirumab (Cyramza), durvalumab (Imfinzi), atezolizumab (Tecentriq), osimertinib (Tagrisso), ceritinib (Zykadia) and nintedanib (Vargatef). EMA labels for all products included some reference to PRO. In contrast, US FDA labels referenced PRO data in only two cases—inavolisib (Itovebi) and ninetedanib (Vargatef). Ribociclib (Kisqali) was flagged for regulatory non-compliance related to promotional materials.PRO instruments cited in EMA labels included the EORTC QLQ-C30, European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Lung Cancer 13 (QLQ-LC13), Lung Cancer Symptom Scale (LCSS), 36-Item Short Form (SF-36), EuroQol 5-Dimension 5-Level questionnaire (EQ-5D-5L) and Patient-Reported Outcome-Common Terminology Criteria for Adverse Events (PRO-CTCAE). Endpoints typically focused on symptoms, global health status or time to deterioration in Health-Related Quality of Life (HRQoL). Reporting formats varied: some labels presented detailed statistical results such as HRs and CIs, while others offered only brief summaries. PRO completion rates were inconsistently reported but generally exceeded 70%–80%. Several EMA labels noted limitations such as open-label designs. The FDA compliance letter for ribociclib (Kisqali) raised concerns over PRO endpoint interpretation, measurement intervals and presentation format in the context of a Direct-to-Consumer (DTC) television commercial, a communication channel not evaluated for the other products in this review.Healthcare professionals PRO content for healthcare professionals (HCPs) was typically located under ‘Efficacy’ or ‘Quality of Life’ on sponsor websites, with variation in depth and format across sponsors and indications. Six case studies were identified: ribociclib (Kisqali), alpelisib (Piqray), fruquintinib (Fruzaqla), ramucirumab (Cyramza), cemiplimab (Libtayo) and brigatinib (Alunbrig).For ribociclib (Kisqali), both US and global HCP sites provided PRO results for early and mBC, citing the NATALEE7 and MONALEESA8–10 trials. Messaging emphasised EORTC QLQ-C30 domains such as physical functioning and global health, with disclaimers on statistical validity and symptom scope. Alpelisib (Piqray) included a single PRO result from the SOLAR-1 trial11 available only outside the USA. Fruquintinib (Fruzaqla) featured subscale-specific time-to-deterioration findings from the FRESCO-2 trial, presented with infographics and explicit disclaimers on trial power.Ramucirumab (Cyramza) displayed whisker plots from the RAINBOW trial,12 including completion rates and statistical caveats. Cemiplimab (Libtayo) included detailed forest plots from the EMPOWER-Lung 3 trial,13 accompanied by statistical methodology and limitations. Brigatinib (Alunbrig) used infographics to show time to worsening in quality of life from the ALTA-1L trial,14 while noting the open-label design and absence of prespecified endpoints. Across all products, interpretation guidance was minimal.Patient Patient-directed messaging of PROs was scarce across major oncology patient-advocacy platforms. Breastcancer.org stood out as the only site consistently translating PRO clinical trial findings into accessible content for patients. Four podcast episodes featured discussions with clinical investigators on quality-of-life outcomes presented at major conferences, focusing on abemaciclib (Verzenio), talazoparib (Talzenna), trastuzumab deruxtecan (Enhertu), and sacituzumab govitecan (Trodelvy), with three episodes supported by Eli Lilly. Topics included symptom management (eg, diarrhoea with abemaciclib (Verzenio)), quality of life advantages of talazoparib (Talzenna) over chemotherapy, and functional benefits of trastuzumab deruxtecan (Enhertu).A dedicated publication on trastuzumab deruxtecan (Enhertu) (Kikawa 2024) emphasised validated quality of life tools, delayed deterioration and key quotes from trialists. Additional brief PRO mentions appeared in coverage of palbociclib (Ibrance), tucatinib (Tukysa) and sacituzumab govitecan (Trodelvy), citing preserved or improved quality of life. Despite breastcancer.org’s efforts, overall patient-facing messaging remains limited, fragmented and often sponsor-dependent—highlighting a significant communication gap in relaying quality of life outcomes to patients.Professional associations (ESMO and ASCO) In the 14 case studies, PRO messaging was disseminated through professional association oncology platforms, particularly ESMO and ASCO, using several communication channels.The ESMO-Magnitude of Clinical Benefit Scale (MCBS) recognised PRO significance when quality of life was a prespecified and statistically significant endpoint. Agents such as sacituzumab govitecan (Trodelvy), palbociclib (Ibrance) and olaparib (Lynparza) received MCBS credit, while others did not qualify due to exploratory or insufficient data.Editorial synthesis through ESMO Oncology News and the Daily Reporter highlighted how PROs complemented survival and safety data, often focusing on symptom-specific benefits such as improved global health status, delayed deterioration or maintenance of functioning.Scientific plenary sessions, such as the ESMO plenary discussion of the NATALEE study (Salmon 2024), formally presented health-related quality of life results for ribociclib (Kisqali), contextualised by leading investigators.Comparative reporting was prominent in ASCO Annual Meeting coverage, where PROs were contrasted across trials. Ribociclib (Kisqali) showed superior quality of life outcomes compared with abemaciclib (Verzenio) in HR+/HER2− BC. Electronic symptom monitoring with trastuzumab deruxtecan (Enhertu) in the PRO-DUCE study15 was associated with improved fatigue scores and maintenance of quality of life. Additional examples and contextual case studies of PRO messaging across regulatory, HCP, academic, and patient settings are provided in the online supplemental appendix 1.SP110.1136/bmjonc-2025-000942.supp1Supplementary dataDiscussion Despite the increasing recognition of PROs in oncology 16–18 drug development, our review highlights a substantial gap between the collection of PRO data in pivotal trials and its translation into regulatory labelling and public-facing communication. Across 128 pivotal trials for BC, GI and NSCLC approved between 2014 and 2024, PROs were included in 105 trials—most frequently as secondary endpoints. Yet only a small subset of these trials (n=14) resulted in regulatory PRO claims. A systematic review found that although 85% of BC drug submissions to the FDA included PRO data, none included PRO content in the FDA label.6 This gap is not unique to BC. In another study examining oncology drug approvals between 2012 and 2016, PROs were used in trials but were rarely included in product labels.3 These findings support our observation of a persistent disconnect between trial-level PRO collection and its integration into formal labelling and external communication. Although PROs offer invaluable insights into the therapeutic impact from the patient’s perspective, their inclusion in regulatory labelling has often been constrained by trial design rather than regulatory conservatism. During the period reviewed, many pivotal studies incorporated PROs only as exploratory or non-prespecified endpoints, limiting their interpretability and qualification for label inclusion.17 19 The limited integration of PROs into labelling therefore reflects shortcomings in study planning and endpoint hierarchy rather than reviewer reluctance. Strengthening the prespecification and analytical rigour of PRO measures within pivotal protocols is essential to ensure that these data can meaningfully inform both regulatory evaluations and post-approval communication.The FDA’s Project Patient Voice initiative, as well as findings from Symonds et al,2 highlight the importance of PROs in informing tolerability and quality of life, especially when survival benefits are marginal or toxicity risks are substantial. Notably, recent FDA labels in 2024 have focused primarily on tolerability outcomes, such as side effect reporting, reflecting a shift toward PROs that assess treatment burden rather than overall quality of life.Although regulatory labelling provides the most compliant framework for sharing PRO data, our findings indicate that sponsor communications are not always confined to products with formal PRO claims. In several cases, limited PRO narratives appeared on HCP or patient-facing websites despite the absence of label content, typically referencing exploratory endpoints or congress presentations. This highlights an unresolved regulatory question: how far PRO information may be communicated responsibly when it is not label-endorsed. Current FDA guidance requires that promotional statements remain consistent with labelling, yet sponsors may perceive value in presenting patient-experience data for contextual understanding rather than promotional intent. A clearer international consensus is needed to delineate boundaries between informative and promotional communication of PROs, ensuring both transparency for patients and compliance with regulatory standards.Most of the pivotal trials employed validated instruments such as the EORTC QLQ-C30 or EQ-5D, and most reported high completion rates, frequently exceeding 80%. However, PRO endpoints were often not prespecified as key measures and were instead categorised as exploratory. This limits both their interpretability and their eligibility for regulatory claims. Our review also found that presentation of PRO data, when present, often lacked statistical detail, omitted limitations or relied on visuals without contextual guidance, particularly in HCP materials. For example, only the Kisqali (ribociclib) website included multilevel PRO messaging for both early and metastatic indications, and even this was flagged by the FDA for issues of endpoint designation and message framing.20 Further work is needed to present PRO data to HCPs and patients in an actionable way that can inform care.Emerging best practices suggest a broader role for PROs beyond registrational endpoints. Friends of Cancer Research and other organisations have emphasised the importance of including PROs in early-phase studies to improve dose optimisation and assess tolerability.21 22 The FDA’s Project Optimus similarly supports this approach, advocating for the early integration of PROs to refine dosing strategies and enhance evaluations that focus on the patient experience.23 Despite this, few products in our review leveraged early-phase PROs in their communication or regulatory strategies.While robust methodological guidance exists, including freely available resources on the PROTEUS Consortium website,24 such as Standard Protocol Items: Recommendations for Interventional Trials–Patient-Reported Outcome,25 Consolidated Standards of Reporting Trials–Patient-Reported Outcomes,25 Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life–Innovative Medicines Initiative,26 27 and FDA’s Patient-Focused Drug Development guidance,28 adherence to these standards remains variable. Giesinger et al29 highlighted notable discrepancies in the reporting of PRO results, particularly in relation to missing data handling, clarity of scoring rubrics and interpretation of scores and score changes. These inconsistencies may undermine the reliability of PRO findings and reduce their utility in regulatory decision-making.30 Notable inconsistencies have been identified in how PRO endpoints are reported, especially in relation to handling missing data, applying scoring methods and interpreting scores and their changes. Variability may jeopardise the reliability of PRO results and erode their value in regulatory decision-making.Several limitations should be considered when interpreting the findings of this study. First, variability in the accessibility and completeness of clinical trial protocols and website content may have influenced the consistency and depth of data extraction across products. For example, full information on analysis plans and exploratory endpoints was not always available on ClinicalTrials.gov, and no additional effort was made to obtain protocols from alternative sources. As a result, some PRO measures and endpoints may have been missed.Second, although two reviewers independently applied the three-tier communication rating framework, its qualitative nature introduces some interpretive subjectivity that may influence classification consistency.Third, our evaluation of public-facing communication was limited to sponsor websites, medical association platforms and patient advocacy group content. We did not include peer-reviewed publications or health technology assessment submissions, where more extensive PRO data reporting may occur. Therefore, our findings reflect only a subset of the broader landscape of PRO communication.Fourth, the identification of PRO messaging relied on targeted search terms (eg, brand/generic name, cancer type, ‘quality of life (QoL),’ ‘patient-reported outcomes (PRO)’) within the internal search tools of selected websites. This approach may have missed relevant content not captured by these terms or not indexed within site search functions—although this limitation mirrors the real-world challenges faced by patients and HCPs seeking this information.Additionally, our review of branded and external communications was focused primarily on US-based sources, which may not reflect global practices or regional variations in how PRO data are communicated. The selection of medical societies and patient advocacy organisations, while methodologically structured, was not exhaustive and may not represent the full range of stakeholder perspectives in oncology.Finally, this study did not evaluate how patients or clinicians interpret or use PRO information in practice, which limits our ability to assess the real-world impact of PRO messaging on decision-making.Taken together, these findings suggest that PROs, while increasingly embedded in trial protocols, are still underleveraged as tools for postapproval communication and decision-making. Incorporation of PROs into protocols has improved over time, driven by regulatory expectations and global initiatives.31 32 However, converting this embeddedness into actionable, interpretable and communicable evidence remains a significant hurdle. To bridge this gap, it is essential to enhance coordination among regulatory bodies and for sponsors to actively commit to integrating PRO data into the core narrative of their products. As the emphasis on patient-centred care and real-world outcomes grows stronger, the success of future oncology communications will depend on the clear, precise and customised presentation of PRO information that is accessible not only for regulatory review but also for HCPs and patients to help guide decision-making. Despite these limitations, we have focused on brand websites and patient advocacy resources that are key for patients who are attempting to find information about products.