The importance of clinical outcome assessments (COA) in healthcare decision making is increasingly recognised. However, evidence on associations between non-mandatory COA inclusion, health technology assessment (HTA) decision making, pricing, and sales of medicinal products is scarce. This study aimed to generate new insights into these associations. HTA reports published in 2012–2022 by bodies in Germany (G-BA), France (HAS), and England (NICE) were retrieved and combined with pricing and sales data. Multiclassification machine learning models were built to analyse the associations between non-mandatory COA inclusion and HTA-related decision making and sales against other relevant features. For Germany, the association with pricing was analysed in addition. Of 1506 identified HTA reports, 40.5
Health-related Quality-of-life is a key indicator in aged and long-term care, but disabled-older-adults cannot always self-report, making proxy-assessment necessary. However, evidence on self-proxy agreement from China is limited. We aimed to assess agreement between self-reported and proxy-reported EQ-5D-5L among disabled-older-adults by comparing proxy-proxy (P1) and proxy-person (P2) ratings with self-report, characterise individual-discrepancy patterns, and examine factors associated with self-proxy discrepancies. In a cross-sectional survey in Nanjing, China, disabled-older-adults completed the self-report EQ-5D-5L and proxies completed proxy-proxy and proxy-person versions in fixed order. Perspective differences and agreement were evaluated using Friedman-tests, weighted kappa, intraclass-correlation-coefficients, and Bland–Altman plots. Individual-discrepancy-patterns were examined using three-stage EQ-5D health profile analysis, and multiple linear regression identified correlates of proxy–self discrepancies. Data from 641 dyads were analysed. Utility and EQ-VAS differed across perspectives overall and in most subgroups. Agreement was higher for observable dimensions (mobility, self-care and usual activities, k ≥ 0.75) than for subjective dimensions (pain/discomfort and anxiety/depression, k = 0.62–0.65). Agreement was high for utility (ICC 0.846–0.849) but lower for EQ-VAS (ICC 0.637–0.698). P2 showed slightly closer agreement with self-report than P1 among nursing-home, severe-cognitive-impairment, and formal-caregiver subgroups. Education, caregiver burden, depressive symptoms, and cognitive impairment were associated with self-proxy discrepancies. Self- and proxy-reported EQ-5D-5L showed high utility agreement, with stronger agreement for observable than subjective dimensions. Proxy-person perspective aligned slightly more closely with self-report than proxy-proxy perspectives in specific care contexts. Proxy perspective should be selected according to care context in long-term care quality assessment and economic evaluation.
Fatigue is a multidimensional patient-reported symptom and an important determinant of health-related quality of life (HRQoL) in adults with anaemia. This review aimed to map the use of selected, validated instruments for measuring fatigue in adults with anaemia, describe their frequency of use, and summarise reported associations with clinical, biological and other patient-reported outcomes. A scoping review was conducted in accordance with PRISMA-ScR and Joanna Briggs Institute guidance. MEDLINE, PubMed, Embase, CENTRAL, the Cochrane Database of Systematic Reviews and Cumulative Index to Nursing and Allied Health Literature (CINAHL) were searched for English-language studies published from January 2000 to June 2025. Eligible studies included adults described by study authors as having anaemia and used at least one pre-specified validated fatigue instrument. Study selection and data charting were undertaken independently by two reviewers, and findings were synthesised descriptively. Of 5,481 records identified, 373 studies were included. The SF-36 vitality subscale (n = 207, 55.5
Rare alloantibody-related conditions of pregnancy, including hemolytic disease of the fetus and newborn (HDFN) and fetal and neonatal alloimmune thrombocytopenia (FNAIT), are serious, potentially life-threatening conditions. Currently, little is known about the experience, well-being, and unmet needs of the maternal patients affected by these conditions. Furthermore, the relevance and suitability of disease-agnostic patient-reported outcome (PRO) instruments in capturing these experiences remains to be evaluated. Qualitative, semi-structured interviews were conducted with participants who had ≥ 1 pregnancy diagnosed with HDFN or FNAIT. Open-ended concept elicitation interviews provided direct insights into the patient experience. From these, condition-specific thematic analyses allowed for concepts to be applied to existing PRO measures, such as the Short Form-36 Health Survey v2 Acute (SF-36v2 Acute) and the EuroQoL-5 Dimension 5 Levels (EQ-5D-5L), to evaluate the suitability of these disease-agnostic measures in capturing the maternal participant experience. Further cognitive debriefing with participants on additional PRO measures provided insight into item-level suitability, relevance, and feasibility of recall periods from the participants’ perspective. The Mother-Generated Index (MGI) was also provided to participants to complete at home. Ten participants with pregnancies affected by HDFN and 8 participants with FNAIT were included. Median age was 35.5 to 40.5 years, with most being White/Caucasian, college-educated, and employed. During pregnancy, impacts on emotional well-being were most prevalent across HDFN and FNAIT, and these were accompanied by physical, social, and work-related impacts. Postnatal experiences frequently involved emotional impacts and experiences with the neonatal intensive care unit. Long-term effects and concerns about future pregnancies were also commonly reported. Most concepts were represented by the SF-36v2 Acute/EQ-5D-5L, except for future family planning. Cognitive debriefing of additional PRO measures showed overall high relevance and suitability, with minor item-specific issues. Furthermore, MGI findings mirrored interview themes and provided broader perspectives on meaningful impacts in other areas. These findings add valuable insights into the patient experience of pregnancies affected by rare alloantibody-related conditions, revealing broad and long-lasting effects on emotional, physical, social, and other well‑being domains. These results support the suitability of SF-36v2 Acute and EQ-5D-5L for capturing patient-reported experiences during pregnancies affected by HDFN or FNAIT.
Elexacaftor/Tezacaftor/Ivacaftor (ETI) has transformed the clinical management of cystic fibrosis (CF), with marked improvements in clinical outcomes. However, its overall impact on patient-reported outcomes (PROs) has not been comprehensively synthesized. To systematically review and meta-analyze PROs following initiation of ETI in people with CF. A systematic search of MEDLINE, EMBASE, SCOPUS, the Cochrane Library, and other online databases was conducted from January 2018 to May 2025. Eligible studies reporting PROs following ETI initiation were included. Risk of bias was assessed using appropriate appraisal tools. Narrative synthesis and meta-analyses with random-effects models were used to pool PRO changes, and meta-regression explored factors associated with observed effects. Sixty-seven studies met the inclusion criteria, including five randomized controlled trials and 62 observational studies. The majority of observational studies were prospective pre-post studies of fair quality, mostly biased due to small sample sizes. Health-related quality of life PROs, including meta-analyses of the Cystic Fibrosis Questionnaire-Revised, demonstrated consistent and substantial improvements following ETI initiation, especially for the respiratory (+ 13.6 points), physical (+ 16.8 points), weight (+ 12.5 points), and vitality (+ 12.5 points) domains. System-specific PROs showed clinically meaningful reductions in sinonasal symptoms (SNOT-22: -16.1 points), whereas gastrointestinal/abdominal outcomes demonstrated mixed results. Mental health-related PROs showed no evidence of overall worsening following ETI initiation. This systematic review and meta-analysis suggests that ETI is associated with broad improvements in PROs, including quality of life and other multi-system benefits in people with CF. Findings are promising but would benefit from confirmation through additional studies, given the predominance of pre-post observational studies addressing non-respiratory PROs.
