Search

While it is important to treat symptoms, there is growing recognition that in order to help people with mental health problems lead meaningful and fulfilling lives, it is crucial to capture the impact of their conditions on wider aspects of their social lives. We constructed two versions of the Recovering Quality of Life (ReQoL) measure&mdash, ReQoL-10 and ReQoL-20&mdash, for use in routine settings and clinical trials from a larger pool of items by combining qualitative and quantitative evidence covering six domains. Qualitative evidence was gathered through interviews and focus groups with over 76 service users, clinicians, and a translatability assessment. Psychometric evidence generated from data from over 6200 service users was obtained from confirmatory factor models and item response theory analyses. In this paper we present an approach based on a traffic light pictorial format that was developed to present qualitative and quantitative evidence to a group of service users, clinicians, and researchers to help to make the final selection. This work provides a pragmatic yet rigorous approach to combining qualitative and quantitative evidence to ensure that ReQoL is psychometrically robust and has high relevance to service users and clinicians. This approach can be extended to the development of patient reported outcome measures in general.

Background: Several instruments are widely used for assessing dependence, craving, withdrawal symptoms, and reinforcing effects in users of tobacco- and nicotine-containing products (TNP), including the Fagerström Test for Nicotine Dependence (FTND), Questionnaire of Smoking Urges, original (QSU) and brief (QSU-b) versions; Minnesota Nicotine Withdrawal Scale, original (MNWS) and revised (MNWS-R) versions; and Cigarette Evaluation Questionnaire, original (CEQ) and modified (mCEQ) versions. Although these instruments have been translated extensively, their translations and corresponding measurement properties have not been systematically assessed. This study aimed to (1) identify the translations of these instruments for which psychometric properties have been published, (2) describe the methods used for translation, and (3) describe the measurement properties and the context in which these translations were evaluated (e.g., target population and TNP used). Methods: Embase and MEDLINE databases were systematically searched. Results: While no information could be found for the CEQ/mCEQ, several translations were available for the remaining instruments: FTND, 25; QSU and QSU-b, 4 each; QSU (12-item version), 1; MNWS, 4; and MNWS-R, 1. Cigarette smokers represented the main target population in which the validation studies were conducted. Information about the translation process was reported for 25 translations. In most cases, the properties of the translations mirrored those of the originals. Differential item functioning was explored in only one case. Conclusions: There are few publications describing the measurement properties of the translations of the FTND, QSU/QSU-b, and MNWS/MNWS-R. None of these translations have been validated for TNPs other than cigarettes, which suggests the need for greater development and validation of instruments in this area.

The purpose of the study is to evaluate the extent to which a retrospectively conducted translatability assessment (TA) could identify the items previously singled out during the validation study as having poor content validity or poor measurement performance. This study was performed with the intent of supporting evidence of the usefulness of TA early in the PRO development process. The Weight module of the Youth Quality-of-Life Instrument (YQOL-W) was used for this appraisal of translatability.Two linguists, blinded to the design and results of content validation and psychometric analyses, conducted a TA on the 32-item version of the YQOL-W taken into cross-sectional validation for item reduction. TA results were categorized into (1) issues relating to target culture (cross-cultural issues) and (2) issues relating to the structure of the original questionnaire (structural issues). Items for possible revision or deletion were identified. We compared the results of the TA with the content validity and psychometric results and decisions to eliminate items after cross-sectional validation.Content validation identified seven of the 32 items to be dropped, and psychometric analyses including the 25 remaining items highlighted an additional four to be eliminated, yielding a final instrument with 21 items. Out of these 11 dropped items, TA had identified nine as problematic (82 %) and the developer was advised to drop five of them (45.4 %). In addition, TA results highlighted the need to change the original formulation of eight items for semantic reasons and identified two instances where alternative wording should be used for translation purposes without any change to the original formulation.Our study showed that translatability assessment confirmed problematic issues in items previously identified as having poor content validity or poor measurement performance. In general, a translatability assessment offers the possibility for the identification of alternative formulations for translation purposes, modifications of original formulations to optimize subsequent translations efforts, and the early detection and discussion of irrelevant or inappropriate items.

Background: The BHQ is a new health-related quality of life questionnaire, developed in British English for patients with bronchiectasis. Objectives: The objectives of this study were to present the results of the translation of the BHQ into 11 languages, i.e., 9 Indo-European (Dutch for Belgium and The Netherlands, English for the USA, French for Belgium and France, German, Italian, and Spanish for Spain and the USA), one Japonic (Japanese), and one Sino-Tibetan (Mandarin). Methods: In each country, the following translation method (i.e., linguistic validation) was used: 1. In-depth analysis of the original (i.e., concept definition) and suggestion of translation alternatives; 2. Forward/backward translation step (or adaptation for English and Spanish versions); and 3. Review by a clinician. Results: The translation process did not reveal any cultural issues since most of the concepts assessed were cross-culturally relevant. The main difficulties consisted in finding conceptual equivalents to the response choices designed as Likert-type scales in 7 points (e.g., “All of the time” to “None of the time”). In all countries, translators ensured that the continuity of time was kept between all versions using either adverbs or synonyms (e.g., “Always” for “All of the time”). Items 2, 5 and 10 generated most discussion across languages for idiomatic or syntactic reasons. The clinicians9 review proved useful to clarify the concept of “clear chest” in item 4, i.e., meaning “chest clear of sputum or congestion,” and led to changes in wording. Conclusion: The rigorous translation methodology was essential in producing 11 translations of the BHQ conceptually equivalent to the original.

The objectives of this study were to finalize, develop the scoring, and explore the psychometric properties of the ACCEPTance by the Patients of their Treatment (ACCEPT©) questionnaire, as well as to provide the first elements for its interpretation and guidance for its future use. ACCEPT© was finalized according to reference methods including testing in a pilot study, i.e., a multi-center, observational, longitudinal study conducted in France, in collaboration with a network of pharmacists. Principal component analysis using Varimax rotation was performed. The loadings of items on components in the principal component analysis were used to inform item selection. Validity of the measurement model of ACCEPT© was confirmed using Multi-trait/Multi-item Analysis based on item-scale Spearman correlations. Internal consistency reliability of the questionnaire was assessed by determining the Cronbach’s α coefficient. Linear and logistic regressions were used to identify predictors of general acceptance, and to study predictors of persistence. A total of 189 patients were included. The final version of ACCEPT© is composed of 25 items, distributed in seven dimensions providing a comprehensive appraisal of acceptance of long-term medication, with six scores measuring acceptance of treatment specific attributes and one score measuring general treatment acceptance. The measurement properties of ACCEPT© were overall fairly satisfactory. Regressions showed that Acceptance/Effectiveness is a predictor of general acceptance. However, no predictor of persistence could be identified. The self-administered ACCEPT© questionnaire is a valid and reliable instrument for the assessment of patients’ acceptance of long-term medication. Disease-specific and large prospective studies are needed to assess the ability of ACCEPT© to predict persistence with treatment.

The importance of patient perspectives on disease impact and response to therapy is increasingly recognized. Patient-reported outcomes (PROs), including health-related quality of life (HRQL), provide information on the effectiveness of medical interventions that cannot be obtained using objective clinical measures. For drug development, PROs such as symptom questionnaires are accepted by the European Medicines Agency (EMEA) and US Food and Drug Administration (FDA) in the approval process. HRQL assessments can help to establish the niche for a new product to support marketing efforts. Utilities can also be derived from health outcomes measures for cost-effectiveness analyses required by health plans for granting drug reimbursement. Because they more closely reflect routine clinical care, effectiveness studies are often a superior setting than traditional randomized controlled trials (RCTs) for conducting comprehensive PRO assessments to determine the real-world impact of therapy on symptoms and other aspects of everyday life. Screening, quality of care, self-reported adherence to treatment, and patient monitoring are important potential applications of PROs in effectiveness studies. However, well-known limitations of effectiveness studies also apply to PROs; these sometimes include a lack of randomization, controls, and blinding. It may be difficult to obtain patient compliance and clinician assistance with PRO assessments in a real-world setting. PRO data may be biased by nonrandom patient dropout, errors in timing of PRO assessments, and the environment in which questionnaires are administered. Additionally, the drug development schedule often requires outcomes results before effectiveness studies can be conducted. Addressing these difficulties during study design and the use of short and targeted questionnaires will improve the usefulness and feasibility of PRO measures in effectiveness studies. Drug Dev. Res. 67:193–201, 2006. © 2006 Wiley-Liss, Inc.

Background The extent to which patient-based outcomes can be used to evaluate and communicate the effect of new drugs and devices is a subject of much debate. Criteria for evaluating the scientific quality of data to support health-related quality of life (HRQL) and other patient-based labeling and promotional claims in the United States and Europe have been proposed by various scientists and organizations. Since March 2000, a working group composed of members of the International Society for Quality of Life Research (ISOQOL), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the Pharmaceutical Manufacturer's Association Health Outcomes Committee (PhRMA-HOC), and the European Regulatory Issues on Quality of Life Assessment (ERIQA) met to discuss and coordinate the various recommendations by their respective groups and address the need to harmonize outcomes review criteria within and across United States and European regulatory agencies. Over time, the discussion expanded from HRQL outcomes to include any outcome based on data provided by the patient or patient proxy, that is, patient-reported outcomes (PROs). The working group therefore became known as the PRO Harmonization Group. Methods Working with a member of the US Food and Drug Administration (FDA), four key issues requiring clarification were identified: how PROs are defined and put into operation for research purposes; the added value of PROs in the drug review and evaluation process; selected questions related to the PRO measurement and research methodology; and the interest and demand for PRO information by decision makers. On February 15, 2001, all members of the PRO Harmonization Group attended a meeting in Rockville, Maryland, to discuss these four issues further, and on February 16, 2001, a formal presentation was made to representatives from various departments and reviewing divisions of the FDA. These presentations are summarized in this report. Results All participants agreed that PROs are important for understanding the impact of treatment on patient functioning and well-being. They also stressed the need to communicate PRO information to key decision makers, including regulatory agencies, clinicians, patients and their families, and payers. Finally, the meeting resulted in plans for continuing the dialogue on PRO measurement and interpretation. Summary The February 16, 2001, meeting represented an important step in harmonizing efforts across various organizations and in opening a dialogue with the FDA around major issues related to methodologic standards for measuring and interpreting PROs in the drug evaluation process.

Patient-reported outcome (PRO) measures must provide evidence that their development followed a rigorous process for ensuring their content validity. To this end, the collection of data is performed through qualitative interviews that allow for the elicitation of in-depth spontaneous reports of the patients’ experiences with their condition and/or its treatment. This paper provides a review of qualitative research applied to PRO measure development. A clear definition of what is a qualitative research interview is given as well as information about the form and content of qualitative interviews required for developing PRO measures. Particular attention is paid to the description of interviewing approaches (e.g., semi-structured and in-depth interviews, individual vs. focus group interviews). Information about how to get prepared for a qualitative interview is provided with the description of how to develop discussion guides for exploratory or cognitive interviews. Interviewing patients to obtain knowledge regarding their illness experience requires interpersonal and communication skills to facilitate patients’ expression. Those skills are described in details, as well as the skills needed to facilitate focus groups and to interview children, adolescents and the elderly. Special attention is also given to quality assurance and interview training. The paper ends on ethical considerations since interviewing for the development of PROs is performed in a context of illness and vulnerability. Therefore, it is all the more important that, in addition to soliciting informed consent, respectful interactions be ensured throughout the interview process.

Objectives Overactive bladder (OAB) affects millions of people worldwide. Identifying patients and measuring OAB’s impact on symptom severity and patients’ health-related quality-of-life is necessary to ensure proper treatment and facilitate communication among patients, clinicians, and caregivers. To accomplish this, the Overactive Bladder questionnaire (OAB-q) and its subsets instruments, the OAB-q Short-Form and the OAB-V8, were developed in U.S. English. To measure the impact of OAB cross-culturally, we performed the linguistic validation of these instruments in Danish, English (Canada), French (Canada and France), Italian (Switzerland), German (Switzerland), Korean, Norwegian, Polish, Portuguese (Brazil), Romanian, Swedish, and Turkish. The linguistic validation was conducted following a rigorous method to ensure conceptual equivalence between the original and its translations. Methods In each country, a specialist monitored the process, which included six steps: (1) two forward translations; (2) comparison and reconciliation of the translations; (3) back-translation; (4) comparison of the source and back-translation; (5) review by one urologist or gynecologist; and (6) a comprehension test using patients. Results The translation of symptom-related adjectives such as “uncomfortable,” “sudden,” “accidental,” “uncontrollable” proved challenging. The subtle differences in the meaning of symptomatic items increased the difficulties to find equivalents. Issues regarding the appropriateness of certain concepts and idiomatic terms emerged during cognitive debriefing. The terms “urge,” “desire,” “urination,” “commute,” “drowsy,” and “escape routes” were not retained literally and were replaced by colloquial expressions. Conclusions The 14 versions of the OAB-q, OAB-q Short-Form, and OAB-V8 were successfully validated linguistically to facilitate data collection cross-culturally and the international comparison of symptom bother and health-related quality of life in patients with OAB.

The exponential development of Patient-Reported Outcomes (PRO) measures in clinical research has led to the creation of the Patient-Reported Outcome and Quality of Life Instruments Database (PROQOLID) to facilitate the selection process of PRO measures in clinical research. The project was initiated by Mapi Research Trust in Lyon, France. Initially called QOLID (Quality of Life Instruments Database), the project's purpose was to provide all those involved in health care evaluation with a comprehensive and unique source of information on PRO and HRQOL measures available through the Internet. PROQOLID currently describes more than 470 PRO instruments in a structured format. It is available in two levels, non-subscribers and subscribers, at http://www.proqolid.org. The first level is free of charge and contains 14 categories of basic useful information on the instruments (e.g. author, objective, original language, list of existing translations, etc.). The second level provides significantly more information about the instruments. It includes review copies of over 350 original instruments, 120 user manuals and 350 translations. Most are available in PDF format. This level is only accessible to annual subscribers. PROQOLID is updated in close collaboration with the instruments' authors on a regular basis. Fifty or more new instruments are added to the database annually. Today, all of the major pharmaceutical companies, prestigious institutions (such as the FDA, the NIH's National Cancer Institute, the U.S. Veterans Administration), dozens of universities, public institutions and researchers subscribe to PROQOLID on a yearly basis. More than 800 users per day routinely visit the database.

Multiple Sclerosis (MS) is a neurodegenerative disease which runs its course for the remainder of the patient's life frequently causing disability of varying degrees. Negative effects on Health-related quality of life (HRQOL) are well documented and a subject of clinical study. The Multiple Sclerosis QOL 54 (MSQOL-54) questionnaire was developed to measure HRQOL in patients with MS. It is composed of 54 items, and is a combination of the SF-36 and 18 disease-specific items. The objective of this project was to translate the MSQOL-54 into French Canadian, and to make it available to the Canadian scientific community for clinical research and clinical practice. Across all French speaking regions, there are occurrences of variation. They include the pronunciation, sentence structure, and the lexicon, where the differences are most marked. For this reason, it was decided to translate the US original MSQOL-54 into French Canadian instead of adapting the existing French version. The SF-36 has been previously validated and published in French Canadian, therefore the translation work was performed solely on the 18 MS specific items. The translation followed an internationally accepted methodology into 3 steps: forward translation, backward translation, and patients' cognitive debriefing. Instructions and Items 38, 43, 45 and 49 were the most debated. Problematic issues mainly resided in the field of semantics. Patients' testing (n = 5) did not reveal conceptual problems. The questionnaire was well accepted, with an average time for completion of 19 minutes. The French Canadian MSQOL-54 is now available to the Canadian scientific community and will be a useful tool for health-care providers to assess HRQOL of patients with MS as a routine part of clinical practice. The next step in the cultural adaptation of the MSQOL-54 in French Canadian will be the evaluation of its psychometric properties.