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Objective: Tolerability and safety of treatments are important in oncology trials and should be informed by patient assessments. We identified the most relevant patient‐reported symptomatic adverse events (AEs) to measure in patients with non‐small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations. Methods: This study selected relevant symptomatic AEs from 78 AEs available in the Patient‐Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO‐CTCAE) measurement system. Initially, symptomatic AEs were selected based on literature and product labeling reviews, and then core sets of symptomatic AEs were identified by patient and clinician interviews. Qualitative and descriptive analyses were performed using the data collected from three iterative rounds of patient interviews. Results: During concept elicitation interviews involving 29 patients, 12 symptomatic AEs were identified and were then adapted into a 25‐item PRO‐CTCAE form for use in future clinical trials along with commonly used PRO measures. Cognitive interviews showed that the PRO‐CTCAE items were easy to answer and appropriate for assessing the patients' experience with symptomatic AEs. This study also assessed disease symptoms, impacts, and overall patient experience. Conclusions: The 25‐item PRO‐CTCAE form captures the most relevant symptomatic AEs in this patient population, and it is available for future studies. Baseline characterization of AEs associated with this distinct patient group contributes to our broader knowledge about NSCLC and through platforms like Project Patient Voice will expand our understanding of treatment tolerability and safety for NSCLC. Ultimately, this data collection will help inform decision‐making for patients, caregivers, healthcare providers, and regulators. [ABSTRACT FROM AUTHOR]

Objective: Pantothenate kinase‐associated neurodegeneration (PKAN) is an autosomal‐recessive, neurodegenerative disorder with a mixed‐motor phenotype caused by a defective PanK2 enzyme, for which there are few adequate treatment options. Clinimetrically sound measures of patient‐reported outcomes are necessary to facilitate therapeutic development for this debilitating disease. This study's objective was to develop such a scale and assess its clinimetric properties. Methods: A conceptually driven, iterative, content development process incorporating input from experts, caregivers, and patients was used. Scale items were initially adapted from the Unified Parkinson's Disease Rating Scale (UPDRS) Part II resulting in the 12‐item Pantothenate Kinase‐Associated Neurodegeneration Activities of Daily Living (PKAN‐ADL). The PKAN‐ADL scale was administered to caregivers (n = 37) and patients (n = 2) twice over 2 weeks, along with selected Quality of Life in Neurological Disorders (Neuro‐QoL) measures, selected attributes of the Health Utilities Index (HUI)‐2/3, and the Stroke Aphasia Depression Questionnaire (SADQ‐10) to assess construct validity. Results: Internal consistency was 0.93, with excellent test‐retest reliability (intraclass correlation coefficient = 0.99). Of the 12 items, 25% (n = 3) showed a ceiling effect >30% (range, 31–54) and 42% (n = 5) showed a floor effect >30% (range, 31–46), reflecting disease heterogeneity. Convergent validity was shown with Neuro‐QoL measures (rs > 0.90) and HUI‐2/3 attributes (rs ≥ 0.48); divergent validity was demonstrated with the SADQ‐10 (r = 0.11). Participants reported a high level of comprehension (98%), and average item relevance ratings (0–10 scale) ranged from 7.0 to 9.9. Conclusion: The PKAN‐ADL scale demonstrated acceptable content validity, with evidence of construct validity and excellent reliability. Overall results support the use of the PKAN‐ADL scale in clinical trials. [ABSTRACT FROM AUTHOR]

Introduction As drug development research efforts move toward studying patients earlier in the course of Alzheimer's disease (AD), it is important to incorporate the patient's perspective into measurement of outcomes. Methods This article summarizes the qualitative work of the Patient-Reported Outcome Consortium's Cognition Working Group in the development of a new self-reported outcome measure in persons with mild cognitive impairment (MCI) due to suspected AD, herein referred to as MCI. Results The draft measure captures the patient's voice for two functional domains, complex activities of daily living and interpersonal functioning. Discussion This work represents a series of initial steps in the development of this rating scale. The next steps are to conduct psychometric analysis and evaluate the role of insight. [ABSTRACT FROM AUTHOR]

Background: Sleep‐wake disturbances appear early in the course of Alzheimer's disease (AD) and other dementias and are not uncommonly associated with disruption in circadian rhythms, such as Irregular Sleep‐Wake Rhythm Disorder (ISWRD; ICD‐10 code G47‐23). ISWRD is also associated with behavioural and functional symptoms. The objective of this study (E2006‐A001‐111; NCT04300569) was to identify signs and symptoms of ISWRD important to patients and caregivers of patients with dementia and ISWRD. The data informed a novel questionnaire, filling a measurement gap as no questionnaires specific to ISWRD in the AD population are available. Method: This cross‐sectional, qualitative, non‐interventional, telephone study interviewed patients and their caregivers. Caregivers were interviewed apart from the patient; patients were interviewed with caregivers present. All participants were from the US; data were collected in English and Spanish. Interview transcripts were analysed using ATLAS.ti software. Result: 15 patient/caregiver dyads and 5 caregiver‐only subjects were interviewed. For the patient group (n=20; 5 not interviewed), mean age was 79.0 (8.3)y and 75% were male. For the caregiver group (n=20), mean age was 67.5 (14.5)y and 20% were male. Both caregivers and patients noted fragmented nighttime and daytime sleep‐wake patterns, but also reported functional consequences of ISWRD. Nighttime examples included waking, wandering, and refusing to go back to sleep. Daytime examples included falling asleep at inappropriate places; appearing sleepy; losing concentration due to sleepiness; sleepiness interfering with personal care and leisure activities; needing to postpone, change, or avoid activities due to sleepiness; adverse emotional impacts, and sundowning. Few differences in terms, or their meaning, were identified between language of data collection. Based on the differing quality of patient and caregiver reports, the potential for patient anosognosia, and the ability of caregivers to observe signs and behaviours, the data suggested a caregiver‐completed observer‐reported outcome (ObsRO) measure is most appropriate for ISWRD evaluation. Conclusion: Concepts reported by patients and caregivers uncovered ISWRD observations and functional consequences appropriate for a novel ObsRO measure that can be used in future studies of ISWRD. Support: Eisai Inc. [ABSTRACT FROM AUTHOR]