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Background: In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment benefit are often generic and may not be sensitive enough to detect change in specific patient populations. Thus, there is a need for disease specific COAs that track meaningful change among individuals. When developing such measures, input from clinicians, caregivers and patients is critical for assessing clinically relevant concepts and ensuring validity of the measure.Method: The aim of this study was to develop two Duchenne-specific global impression items for use in clinical trials. The development of the Duchenne Clinical Global Impression of Change (CGI-C) and Caregiver Global Impression of Change (CaGI-C) was informed by findings from concept elicitation (CE) interviews with clinicians, caregivers and individuals with Duchenne. Through cognitive debriefing (CD) interviews, clinicians and caregivers evaluated draft CGI-C and CaGI-C items to ensure relevance and understanding of the items and instructions. Suggestions made during the CD interviews were incorporated into the finalized CGI-C and CaGI-C measures.Results: The symptoms most frequently reported by clinicians, caregivers and individuals with Duchenne were muscle weakness, fatigue, cardiac difficulties and pain. Regarding physical functioning, all three populations noted that small changes in functional ability were meaningful, particularly when independence was impacted. Caregivers and clinicians reported that changes in speed, endurance and quality of movement were important, as was improvement in the ability of individuals to keep up with their peers. A change in the ability to complete everyday activities was also significant to families. These results were used to create two global impression of change items and instruction documents for use by clinicians (CGI-C) and caregivers (CaGI-C). Overall, both items were well understood by participants. The descriptions and examples developed from the CE interviews were reported to be relevant and appropriate for illustrating different levels of meaningful change in patients with Duchenne. Modifications were made based on caregiver and clinician CD feedback .Conclusions: As part of a holistic measurement strategy, such COA can be incorporated into the clinical trial setting to assess global changes in relevant symptoms and functional impacts associated with Duchenne. [ABSTRACT FROM AUTHOR]

Background: The Schizophrenia Caregiver Questionnaire (SCQ) was developed to provide a comprehensive view of caregivers' subjective experiences of the impacts of caring for someone with schizophrenia. The Caregiver Global Impression (CaGI) scales were designed to assess their perception of the severity of the schizophrenia symptoms, of change in schizophrenia symptoms and in the experience of caring since the beginning of the study. The objectives of the study were to translate the SCQ and CaGI scales in 11 languages [French (Canada, France), English (Canada, UK, Australia), German (Germany), Italian (Italy), Spanish (Spain), Dutch (the Netherlands), Finnish (Finland), and Swedish (Sweden)], to present evidence that the translations capture the concepts of the original questionnaires and are well understood by caregivers of patients with schizophrenia in each target country. Methods: The different language versions were developed using a standard or adjusted linguistic validation process fully complying with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) recommended procedures. Results: Interviews were conducted with 55 caregivers of patients with schizophrenia from 10 countries representing the 11 different languages. Participants ranged in age from 28 to 84 years and had 5 to 16 years of education. Women represented 69.1 % (38/55) of the sample. Fourteen out of the 32 items of the SCQ generated difficulties which were mostly of semantic origin (13 items). The translation of the CaGI scales did not raise any major difficulty. Only five out of the 55 caregivers had difficulty understanding the meaning of the translations of "degree" in the expressions "degree of change in experience of caring" and "degree of change in symptoms". Conclusions: Translations of the SCQ and CaGI scales into 11 languages adequately captured the concepts in the original English versions of the questionnaires, thereby demonstrating the conceptual, semantic, and cultural equivalence of each translation. [ABSTRACT FROM AUTHOR]