Catherine Acquadro

Patient-reported outcomes (PROs) are any outcome evaluated directly by the patient himself and based on the patient's perception of a disease and its treatment(s). PROs are direct outcome measures that can be used as clinical meaningful endpoints to characterize treatment benefit. They provide unique and important information about the effect of treatment from a patient's view. However, PROs will only be considered adequate if the assessment is well-defined and reliable. In 2009, the FDA has issued a guidance, which defines good measurement principles to consider for PRO measures intended to give evidence of treatment benefit in drug development. In hematologic clinical trials, when applied rigorously, they may be used to evaluate overall treatment effectiveness, treatment toxicity, and quality of patient's well-being at short-term and long-term after treatment from a patient's perspective. In situations in which multiple treatment options exist with similar survival...

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. The different language versions were developed using a standard or adjusted linguistic validation process fully compl...

Major contributors Acquadro, Catherine MAPI Research Institute, Lyon, France Alderson, Philip National Institute for Health and Clinical Excellence, London/Manchester, United Kingdom Altman, Douglas G Centre for Statistics in Medicine, University of Oxford, Oxford, United Kingdom Armstrong, Rebecca The McCaughey Centre: VicHealth Centre for the Promotion of Mental Health and Community Wellbeing, University of Melbourne, Melbourne, Australia Askie, Lisa M NHMRC Clinical Trials Centre, University of Sydney, Camperdown, Australia Becker, Lorne A ...

2.1 Translating and evaluating questionnaires: cultural issues for international research Patrick Marquis, Dorothy Keininger, Catherine Acquadro and Christine de la Loge Introduction Advances in new technologies (Schieber el al. ... Mathias, SD, Fifer, SK, Patrick, DL (1994). ...

Chronic leg wounds can represent a major health problem and have a negative emotional impact on patients' lives. The Cardiff Wound Impact Schedule (CWIS) was developed by the Wound Healing Research Unit at University of Wales College of Medicine to assess the quality of life in patients with chronic wounds. To perform the linguistic validation (translation) of the CWIS into German, French and US English as a first step in making the questionnaire available globally. The standard linguistic validation process employed by the Mapi Research Institute was used to translate the CWIS into French and German. An adjusted process was used for the US English translation. The linguistic validation of the US English version was straightforward as it shares the same root language with the original UK version. The problems encountered in the French and German translations were mostly related to semantics and syntax. The CWIS is now available for further validation testing by the international...

... 17.9 Chapter information Authors: Donald L Patrick, Gordon H Guyatt and Catherine Acquadro on behalf of the Cochrane Patient Reported Outcomes Methods Group. ... Guyatt 1997 Guyatt GH, Naylor CD, Juniper E, Heyland DK, Jaeschke R, Cook DJ. ...

Purpose: To review the extent to which HRQL and other PROs have played a role in drug approval and labeling since the FDA issued its draft guidance on the use of PRO measures and the EMA, its reflection paper on HRQL in 2006. Method: This research was conducted through a systematic manual review of therapy area-specific regulatory guidelines (US and EU) issued and product labels approved during the period of January 1st, 2006 to November 16th, 2010. Result: 15 FDA and 34 EMA guidance documents were released containing recommendations for the inclusion of PRO endpoints in clinical trials. The FDA referred to HRQL specifically (as a secondary endpoint) in three of 15 (20%) guidance documents mentioning PRO endpoints – for chronic obstructive pulmonary disease, oncology and weight management. The EMA recommended use of HRQL endpoints in 22 of the 34 (65%) guidances. Interestingly guidelines issued by the EMA during our period of analysis have gained in precision and clarity compared to...

DEVELOPMENT OF E-VERSIONS OF THE ASTHMA QUALITY OF LIFE QUESTIONNAIRE (STANDARDISED) [AQLQ(S)] IN 20 LANGUAGES FOR USE ON HANDHELD DEVICES Elizabeth F Juniper1, Géraldine Grataloup2, Catherine Acquadro3 1Dept. Clinical Epidemiology and Biostatistics, McMaster University, Canada. 2Mapi, Linguistic Validation, Lyon, France 3Mapi Research Trust, Lyon, France Objectives. Adapting validated paper questionnaires for handheld electronic devices often requires questions and response choices to be presented on two or more screens and instructions reformulated. Before use on handheld device, the original English version of the Standardized Asthma Quality of Life Questionnaire [AQLQ(S)] was required to be validated. The objective of this study is to present the methods used and the challenges faced during the adaptation of 20 language versions of the AQLQ(S) for a specific device, using the linguistically validated paper versions of these languages. Methods. The following steps were performed ...

Objectives. The objective of this research was to review the role of patient-reported outcomes (PROs) in drug approval and labeling of products authorized for the treatment of cystic fibrosis (CF) in Europe and in the USA. Methods. This research was conducted through a systematic manual review of CF specific regulatory guidelines and product labelings (US and EU). The PROLabels database was used for the label review. FDA medical reviews and EMA assessment reports were also used to review the clinical studies performed for regulatory approval. Results. In its guideline specific to CF, the EMA recommends the use of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) and labels it as a quality-of-life endpoint. The CFQ-R is a disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms. There is no specific FDA guidance on CF: the corresponding FDA guidance only covers exocrine pancreatic insufficiency drug products and recommend...