Use Of Patient-reported Outcome Instruments Research Articles

Application of Patient-Reported Outcome Measurements in Adult Tumor Clinical Trials in China: Cross-Sectional Study.

International health policies and researchers have emphasized the value of evaluating patient-reported outcomes (PROs) in clinical studies. However, the characteristics of PROs in adult tumor clinical trials in China remain insufficiently elucidated. This study aims to assess the application and characteristics of PRO instruments as primary or secondary outcomes in adult randomized clinical trials related to tumors in China. This cross-sectional study identified tumor-focused randomized clinical trials conducted in China between January 1, 2010, and June 30, 2022. The ClinicalTrials.gov database and the Chinese Clinical Trial Registry were selected as the databases. Trials were classified into four groups based on the use of PRO instruments: (1) trials listing PRO instruments as primary outcomes, (2) trials listing PRO instruments as secondary outcomes, (3) trials listing PRO instruments as coprimary outcomes, and (4) trials without any mention of PRO instruments. Pertinent data, including study phase, settings, geographic regions, centers, participant demographics (age and sex), funding sources, intervention types, target diseases, and the names of PRO instruments, were extracted from these trials. The target diseases involved in the trials were grouped according to the American Joint Committee on Cancer Staging Manual, 8th Edition. Among the 6445 trials examined, 2390 (37.08%) incorporated PRO instruments as part of their outcomes. Within this subset, 26.82% (641/2390) listed PRO instruments as primary outcomes, 52.72% (1260/2390) as secondary outcomes, and 20.46% (489/2390) as coprimary outcomes. Among the 2,155,306 participants included in these trials, PRO instruments were used to collect data from 613,648 (28.47%) patients as primary or secondary outcomes and from 74,287 (3.45%) patients as coprimary outcomes. The most common conditions explicitly using specified PRO instruments included thorax tumors (217/1280, 16.95%), breast tumors (176/1280, 13.75%), and lower gastrointestinal tract tumors (173/1280, 13.52%). Frequently used PRO instruments included the European Organisation for Research and Treatment of Cancer Quality of Life Core Questionnaire-30, the visual analog scale, the numeric rating scale, the Traditional Chinese Medicine Symptom Scale, and the Pittsburgh Sleep Quality Index. Over recent years, the incorporation of PROs has demonstrated an upward trajectory in adult randomized clinical trials on tumors in China. Nonetheless, the infrequent measurement of the patient's voice remains noteworthy. Disease-specific PRO instruments should be more effectively incorporated into various tumor disease categories in clinical trials, and there is room for improvement in the inclusion of PRO instruments as clinical trial end points.

PCR80 Analysis of the Use of Instruments for Collecting Patient-Reported Outcomes (PROS) in Pediatric Asthma Research

Children and adolescents with asthma are distressed by disease symptoms and may experience limitations in daily activities. Patient-reported outcome (PRO) instruments provide added insights on the patient’s health status when evaluating interventions for pediatric asthma. This study examined trends in the use of PRO instruments in pediatric asthma trials.

Real-World Health-Related Quality of Life in Patients with Diffuse Large B-Cell Lymphoma: Comparisons with Reference Populations and By Line of Therapy

Introduction: Diffuse large B-cell lymphoma (DLBCL) is the most common type of aggressive non-Hodgkin's lymphoma (NHL) constituting 30-58% of all NHL (Tilly et al, 2015; Thieblemont et al, 2020). Treatment can include intensive multiagent chemotherapy and other novel therapies which carries risk for toxicities. Despite this, we lack data comprehensively depicting the quality of life of real-world patients with DLBCL, particularly in the modern era with novel therapies. Therefore, we aimed to compare Quality of Life (QoL) to reference populations and assess real-world DLBCL patients across multiple countries and lines of therapy.Methods: Real-world data were drawn from the Adelphi DLBCL Disease Specific Programme™ (DSP), a point-in-time survey of hematologists, hemato-oncologists, oncologists and their patients with DLBCL conducted in France (FR), Germany (DE), Italy (IT), Spain (SP), the United Kingdom (UK) and the United States of America (US) between Jan-May 2021. Patients were asked to voluntarily complete a patient self-completion form (PSC) capturing demographics and QoL data through the use of patient-reported outcome instruments: the European Organization for Research and Treatment of Cancer quality of life questionnaire (EORTC QLQ-C30), EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L), EQ-5D-5L Visual Analogue Scale and Work Productivity and Activity Impairment questionnaire. Bivariate analysis was conducted to compare all cancer and NHL-specific reference values from the EORTC QLQ-C30 manual (Scott et al, 2008) to DLBCL DSP data, and to review QoL across lines of therapy, defined as first line and second line (1L+2L) and third line and above (3L+). Statistical significance level was set at p<.05. If functional scores were lower, when compared to reference values, within the DSP, this was indicative of a worse QoL in patients with DLBCL. For symptomatic scores the opposite was true; should the DSP value be higher, this was indicative of a worse QoL in patients with DLBCL.Results: Data analysis was conducted on 441 patients with DLBCL who completed a PSC (FR: n=80, DE: n=150, IT: n=54, SP: n=43, UK: n=34, US: n=80); at data collection, mean (standard deviation) age was 64.6 (12.39) years, 36% of patients were female, 19% working full- or part-time and 80% were relapsed/refractory, 29% were 3L+. 8%, 24%, 28% and 40% were at Ann Arbor disease stage I, II, III and IV respectively at the time of data collection.When comparing DLBCL DSP values to EORTC QLQ-C30 all cancer reference values for functional scores (Table 1), global health status, physical functioning, role functioning, emotional functioning, cognitive functioning and social functioning were significantly worse than all cancer reference values.In terms of symptoms, DSP values for fatigue, nausea and vomiting, dyspnea, appetite loss and diarrhea were significantly worse than all cancer reference values (Table 2).Results were mixed when comparing with EORTC QLQ-C30 NHL-specific reference values (Table 1) for functional scores; global health status was significantly worse for the DLBCL DSP population, whilst role functioning, cognitive functioning and social functioning were significantly better than NHL reference values. Significantly worse symptom scores were observed in the DLBCL DSP population (Table 2) for nausea and vomiting, pain, dyspnea and diarrhea when compared with the reference values.Functioning scores were significantly worse in 3L+ patients vs 1L+2L for global health status, physical functioning, role functioning, cognitive functioning and social functioning (Table 3).Fatigue, dyspnea and diarrhea symptomatic scores were significantly worse in 3L+ vs 1L+2L patients. Symptom burden was high across all lines of therapy (Table 4).Conclusion: Real-world patients with DLBCL demonstrated significantly worse QoL when compared with a general cancer reference population with respect to all functional scores, as well as fatigue, nausea and vomiting, dyspnea, appetite loss and diarrhea, underscoring the high symptom burden experienced by patients with DLBCL.Patients with DLBCL on 3L+ had significantly worse QoL than those on earlier lines of therapy with respect to global health status, physical functioning, role functioning, cognitive functioning and social functioning, fatigue, dyspnea and diarrhea indicating an unmet need in novel treatment options to help improve QoL in later lines. [Display omitted] DisclosuresMa: Regeneron Pharmaceuticals Inc.: Current Employment, Current holder of individual stocks in a privately-held company. Quek: Regeneron Pharmaceuticals Inc.: Current Employment, Current holder of individual stocks in a privately-held company.

A Systematic Review of Patient-Reported Outcomes in Primary Biliary Cholangitis and Primary Sclerosing Cholangitis.

Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are associated with decreased health‐related quality of life and debilitating symptoms. These experiences can be defined as patient‐reported outcome (PRO) concepts and measured using PRO instruments. We identified all PRO concepts and instruments used in the PBC and PSC literature. This systematic review identified PBC and/or PSC studies from January 1, 1990, to May 6, 2019, that measured at least one PRO concept. Study population, design, PRO concept, PRO instrument, and validation data for PRO instruments were investigated. We provided descriptive statistics of PRO concepts and instruments used, stratified by population type. Use of PRO concepts and instruments were assessed over time. The search yielded 318 articles (69% in PBC, 18% in PSC, 13% in both, and 24% in drug trials). Forty‐nine unique PRO concepts were identified. The five most common PRO concepts included pruritus (25%), fatigue (19%), broad health‐related quality of life (16%), gastrointestinal adverse events (6%), and physical adverse events (6%). Only 60% of PRO concepts were measured with a PRO instrument, most of which were nonvalidated visual analogue or numeric rating scales. Only three of 83 PRO instruments were developed with feedback from the target populations (one for PBC, one for PSC, and one for both), and only six documented any psychometric testing in the target populations. Use of PRO instruments increased over time from 30% in the 1990s to 67% by 2019. Conclusion: The overwhelming majority of PRO instruments used in PBC/PSC were nonspecific and lacked patient validation or empirical justification. Significant opportunities exist to use qualitative methods to better understand patient experiences, and translate this knowledge into meaningful, patient‐driven study outcomes.

Item banking and computer-adaptive testing in clinical trials: Standing in sight of the PROMISed land.

The use of patient-reported outcome instruments (PRO) in clinical trials in order to capture the impacts of treatment on patients is widespread. However, regulatory agencies have over the past decade highlighted the need for PROs that are fit for purpose and target relevant aspects of the patient's condition. Many legacy PROs were developed with little patient input, are lengthy, and may lack relevance having not been modified or adapted as medical treatments have advanced. Computer-adaptive test (CAT) systems provide the possibility of targeted approaches to capturing patient-centric data, while minimising patient burden. Coupled with greater patient input in the development of PROs, CAT offers the opportunity of overcoming the shortcomings of the previous generation of PROs. This paper describes the some of the issues facing legacy PROs, current regulatory guidance, and initiatives, such as the Patient-Reported Outcome Measurement Information System (PROMIS), as well as the early signs of use of CAT to capture PRO data in clinical trials.

Prospective Analysis of Satisfaction by Means of BREASTQ in 156 Patients Who Underwent Breast Reduction.

Sir: Greco and Noone have recently published in Plastic and Reconstructive Surgery a very interesting article entitled “Evidence-Based Medicine: Reduction Mammaplasty.”1 We would like to congratulate the authors for their article and we want to direct attention to the evaluation of the patient’s point of view about reduction mammaplasty. In our recent study,2 we have shown how patients require more attention in presurgical consultations and that clear communication should be prioritized to ensure that the surgeon understands the patient’s expectations; thus, the use of patient-reported outcome instruments could help us to study the correct way to approach the patient’s expectations. The assessment of quality of life before and after surgery cannot be based on personal considerations of the attending physician, but must necessarily be expressed through objective studies, such as evaluation by means of the BREAST-Q.3 The BREAST-Q is considered worldwide the best instrument with which to quantify the patient’s perception before and after breast surgical procedures.4 At our Department of Plastic, Reconstructive and Aesthetic Surgery from September of 2011 to September of 2015, 156 consecutive patients who underwent breast reduction answered both preoperative and postoperative modules of the BREAST-Q. This study was approved by the ethics committee of our hospital, and each subject provided written informed consent before participating in the study. We administered the questionnaire 2 weeks before surgery and almost 6 months after surgery, and we performed a statistical analysis to compare preoperative and postoperative answers. Statistically significant results were observed between the mean scores on all four BREAST-Q scales: satisfaction with appearance of breasts, psychosocial well-being, sexual well-being, and physical well-being. In all of the patients, the raw score considered in this study was greater than or equal to 4 (Table 1). The concept of modern surgery has evolved from strictly a science that deals with a “disease to treat” rather than the “patient to be cured” to a psychosurgery, in which we aim is to provide the patient with the greatest degree of satisfaction in the shortest possible time. This concept is feasible only if the satisfaction of the patient and the improvement of quality of life can be assessed objectively from a scientific point of view.5 The point of view of the patient must be made scientifically valid and must be provable. The use of questionnaires is the only way to analyze scientifically and objectively the perception of the patient’s own body and that he or she would like to correct the defect. We think that the use of the BREAST-Q questionnaire should become part of the routine management of patients who are candidates for mammary and reconstructive surgical procedures and should be discussed and compared according to the needs of the patient.Table 1.: BREAST-Q Preoperative and Postoperative Modules in 156 PatientsDISCLOSURE The authors do not have any commercial associations that might pose or create a conflict of interest with information presented in this communication. No intramural or extramural funding supported any aspect of this work. Annalisa Cogliandro, M.D., Ph.D.Mauro Barone, M.D.Paolo Persichetti, M.D., Ph.D.Plastic and Reconstructive Surgery UnitCampus Bio-Medico University of RomeRome, Italy

Trends in the use of patient-reported outcome instruments in neurosurgical adult thoracolumbar deformity and degenerative disease literature.

Objective:Shifting national healthcare trends place increased emphasis on patient-centered care and value-based outcomes, and thus, patient-reported outcome instruments (PROIs) are often used. We sought to characterize the trends in PROI use over the past decade with regard to thoracolumbar degenerative spine disease and spinal deformity in major neurosurgical journals.Methods:Articles were screened for PROI use through a PubMed search among five major neurosurgical journals from 2006 to 2016. Articles focusing on adult thoracolumbar deformity and degenerative disease were selected with stringent criteria to further characterize PROI use.Results:A total of 29 different PROIs were used among 102 articles identified from 2006 to 2016 using our search strategy. Journal of Neurosurgery: Spine contained the most articles utilizing PROIs with 35.3% of all articles meeting search criteria. The most frequently used PROIs were Oswestry Disability Index, visual analog scale, and the European Quality of Life Five-dimension questionnaire used in 79.4%, 59.8%, and 29.4% of articles, respectively. Linear regression identified a significant increase in the number of articles employing PROIs from 2006 to 2016 (Y = 1.85, R2= 0.77, P < 0.01). The total number of PROIs per article was relatively stagnant over time and did not significantly change (Y = 0.03, R2= 0.05, P = 0.51).Conclusions:PROI use as an outcome tool in the adult thoracolumbar disease literature has increased during the past decade, which may be an indicative of PROI use to define patient expectations. This may also represent a trend toward PROI use as a surrogate measure of value-based care.

Montreal Accord on Patient-Reported Outcomes (PROs) use series–Paper 7: modern perspectives of measurement validation emphasize justification of inferences based on patient reported outcome scores

BackgroundObtaining the patient's view about the outcome of care is an essential component of patient-centered care. Many patient-reported outcome (PRO) instruments for different purposes have been developed since the 1960s. Measurement validation is fundamental in the development, evaluation, and use of PRO instruments. ObjectivesThis paper provides a review of modern perspectives of measurement validation in relation to the followings three questions as applied to PROs: (1) What evidence is needed to warrant comparisons between groups and individuals? (2) What evidence is needed to warrant comparisons over time? and (3) What are the value implications, including personal and societal consequences, of using PRO scores? DiscussionMeasurement validation is an ongoing process that involves the accumulation of evidence regarding the justification of inferences, actions, and decisions based on measurement scores. These include inferences pertaining to comparisons between groups and comparisons over time as well as consideration of value implications of using PRO scores. Personal and societal consequences must be examined as part of a comprehensive approach to measurement validation. The answers to these three questions are fundamental to the the validity of different types of inferences, actions, and decisions made on PRO scores in health research, health care administration, and clinical practice.

Current Trends in the Use of Patient-Reported Outcome Instruments in Degenerative Cervical Spine Surgery.

Study Design Bibliometric analysis. Objective To determine trends, frequency, and distribution of patient-reported outcome instruments (PROIs) in degenerative cervical spine surgery literature over the past decade. Methods A search was conducted via PubMed from 2004 to 2013 on five journals (The Journal of Bone and Joint Surgery, The Bone and Joint Journal, The Spine Journal, European Spine Journal, and Spine), which were chosen based on their impact factors and authors' consensus. All abstracts were screened and articles addressing degenerative cervical spine surgery using PROIs were included. Articles were then analyzed for publication date, study design, journal, level of evidence, and PROI trends. Prevalence of PROIs and level of evidence of included articles were analyzed. Results From 19,736 articles published, 241 articles fulfilled our study criteria. Overall, 53 distinct PROIs appeared. The top seven most frequently used PROIs were: Japanese Orthopaedic Association score (104 studies), visual analog scale for pain (100), Neck Disability Index (72), Short Form-36 (38), Nurick score (25), Odom criteria (21), and Oswestry Disability Index (15). Only 11 PROIs were used in 5 or more articles. Thirty-three of the PROIs were appeared in only 1 article. Among the included articles, 16% were of level 1 evidence and 32% were of level 4 evidence. Conclusion Numerous PROIs are currently used in degenerative cervical spine surgery. A consensus on which instruments to use for a given diagnosis or procedure is lacking and may be necessary for better communication and comparison, as well as for the accumulation and analysis of vast clinical data across multiple studies.

Statistical challenges in drug approval trials that use patient-reported outcomes

This article describes challenging aspects of the use of patient-reported outcome instruments in clinical trials for drug approval, in our perspective as statistical reviewers at the US Food and Drug Administration. We discuss aspects of planning and interpreting results in clinical trials (1) adapting an existing patient-reported outcome instrument for use in clinical trials, (2) using multi-item patient-reported outcomes and (3) missing patient-reported outcome values from many subjects over time. These challenges are illustrated with multiple examples from different clinical trials for different indications. We finally discuss important considerations in labeling.

Patient-reported outcome instruments used to assess pain and functioning in studies of bisphosphonate treatment for bone metastases

PurposeWhen treating metastatic bone disease, relief of bone pain is often a key outcome. Because pain cannot be quantified with objective clinical measures, patient-reported outcome (PRO) measures are required to assess patients' subjective experience. The goal of the current review was to examine measures used to assess pain, as well as the impact of pain on functional status and health-related quality of life (HRQL), in trials of bisphosphonates for the treatment of bone metastases.MethodsA literature search focused on articles published from January 1999 to April 2009.ResultsA total of 49 articles were located that used PROs to assess pain-related outcomes of bisphosphonate treatment for bone metastases. The Brief Pain Inventory was the most commonly used multi-item instrument. However, the most common approach for assessing pain was to administer a single-item scale such as a visual analog scale, numerical rating scale, or verbal rating scale. Of the 49 studies, 19 included a PRO assessing functional status or HRQL.ConclusionsAlthough pain is an important outcome of trials examining treatment for bone metastases, the current review suggests that there is little consistency in PRO measurement across studies. Furthermore, presentation of measures often lacked clear description, information on measurement properties, citations, clarity regarding method of administration, and consistent instrument names. Recommendations are provided for instrument validation within the target population, assessment of content validity, use of PRO instruments recently developed for patients with bone metastases, clear description of instruments, and implementation of measures consistent with recommendations from instrument developers.

Use of pain instruments as endpoints in regulatory submissions: implications of the US FDA's new Patient Reported Outcomes (PRO) Guidance

The goal of this poster is to review aspects of the FDA's PRO Guidance which have implications for measuring pain as an endpoint in clinical trials. This information will be of immediate interest to researchers developing or using pain instruments to evaluate pain treatments for eventual regulatory submission and subsequent approval. Pain management across therapeutic categories remains a key area for the use of PRO instruments and the interpretation of PRO endpoints to inform drug and device labeling decisions. While academic and medical recommendations exist to inform the development and use of pain instruments (e.g., IMMPACT, OARSI), it remains unclear as to how these recommendations harmonize with emerging regulatory guidelines. In 2009 the US FDA finalized a guidance for the use of PROs for regulatory submissions/approval (FDA, 2009). This guidance outlines criteria for the development, implementation, and interpretation of PROs when used to support primary and secondary endpoints in clinical trials. Moreover, the FDA has indicated that PRO instruments will not be “grandfathered” (i.e., accepted for present or future use based on previous regulatory approvals) and, therefore, will require the same level of validation and documentation to defend and support their use to regulators. To meet regulatory expectations, researchers will have to substantiate their measurement strategy decisions within the context of the PRO Guidance. We will cite the salient aspects of the PRO Guidance, summarize methods and provide citations that can be used to help researchers successfully achieve these new standards set by the FDA. Specific areas of focus will include, but not be limited to: - Recall period and retrospective recall issues - Frequency and timing of assessments - Validation or psychometric evaluation of existing pain scales for use in other pain therapeutic areas - Translation and linguistic validation requirements - Paper to electronic platform migration.

The use of patient-reported outcomes instruments in registered clinical trials: Evidence from ClinicalTrials.gov

Background Investigators of clinical trials in which the list of outcomes include patient-reported outcomes (PROs) – usually labeled quality of life (QoL) – have a large number of instruments from which to choose. The extent and manner in which PRO instruments are used in clinical trials can be assessed using data from clinical trial registries. Most medical journals now require a clinical trial be registered before its results are considered for publication. This requirement is intended to discourage publication bias, such as the reporting of tests of hypotheses different from those stipulated at the start of the trial and selective reporting of partial results. Purpose To assess the usage of PRO instruments in registered trials by various trial characteristics and to determine if the instruments are adequately identified in the registry. Methods A local copy of the ClinicalTrials.gov database was made in September 2007. The outcomes of all interventional trials registered since September 2004 were assessed for usage of a PRO instrument. Odds ratios of PRO usage were estimated by a logistic regression model. Results Of 17,704 interventional trials, 2481 (14.0%) used at least one PRO instrument. However, less than half of those trials (41.0%) identified the instrument to be used. PRO usage is positively associated with phase (III), randomization type (randomized), intervention type (behavior) and sponsorship type (university/research organization). Conclusions PRO instruments are used in a significant percentage but minority of clinical trials. Trial registries should require that all PRO instruments be identified, including the concepts or outcomes they are intended to measure.

What Can We Learn from the Time Course of Untreated and Partially Treated Community‐OnsetStreptococcus pneumoniaePneumonia? A Clinical Perspective on Superiority and Noninferiority Trial Designs for Mild Community‐Acquired Pneumonia

There are no well-designed placebo-controlled clinical trials in the recent era that precisely define the magnitude of the drug effect of antimicrobial therapy for mild community-acquired pneumonia (CAP). However, there is evidence that ineffective therapies, selected on the basis of the ratio of 24-h area under the concentration curve to minimum inhibitory concentration, associated with a discordant (nonsusceptible in vitro) specific agent (or no therapy) for mild CAP due to Streptococcus pneumoniae are associated with increased risk of progression to serious CAP. The relatively high rate of clinical success associated with appropriate antimicrobial treatment of mild CAP renders a standard outcome measure of clinical success an unlikely way to differentiate new agents. However, there may be an advantage in composite outcome assessments for mild CAP. Composite-outcomes end points that include time to resolution of morbidity, the use of patient reported-outcomes instruments, and biomarkers are recommended for future studies. Because the composite rate of success in recent randomized clinical trials exceeds 90%, it would seem that a noninferiority margin of 10% is reasonable for trials for mild CAP.

Accounting for the effect of GERD symptoms on patients’ health-related quality of life: supporting optimal disease management by primary care physicians

AimTo review, from a primary care physician (PCP) perspective, the use of patient-reported outcome (PRO) instruments for assessment of gastro-oesophageal reflux disease (GERD) symptoms, their impact on health-related quality of life (HRQL) and the effectiveness of therapy.ResultsWhile generic and disease-specific PRO instruments have been used in the assessment of GERD, the latter can be considered to be more appropriate as they focus only on problems relevant to the disease in question (and therefore tend to be more responsive to change). Such instruments include the Quality of Life in Reflux and Dyspepsia (QOLRAD) questionnaire and the Gastrointestinal Symptom Rating Scale and the Reflux Disease Questionnaire (RDQ). Their use indicates that GERD symptoms are troublesome and significantly reduce patients’ HRQL, and that effective treatment of GERD improves HRQL. The GERD Impact Scale (GIS) questionnaire, primarily developed for use within primary care, can also help to determine the impact of symptoms on patients’ everyday lives and, in turn, the benefit of appropriately targeted therapy. Notably, these PRO instruments were developed from focus groups of GERD patients, and only aspects rated of highest importance are used in the final instruments. Consequently, PCPs can feel confident that these questionnaires encompass the most relevant points that they are likely to ask in terms of how symptoms affect patients’ everyday lives.ConclusionsPrimary care physicians are encouraged to make wider use of PRO instruments within routine practice to improve communication with their GERD patients that, in turn, could lead to improved clinical outcomes and greater patient satisfaction.Review CriteriaWe conducted a review of validated patient-reported outcome (PRO) instruments used in gastro-oesophageal reflux disease (GERD), based on the authors’ expertise in the field and a supplementary MEDLINE search with the terms ‘health-related quality of life’, ‘quality of life questionnaire’, ‘patient-reported outcomes’, ‘patient satisfaction’ and ‘gastro-oesophageal reflux disease’.We reviewed papers reporting health-related quality of life (HRQL) data in patients with GERD, including the effect of GERD on HRQL, treatment efficacy and HRQL, patient satisfaction and physician-patient agreement, to help primary care physicians to incorporate PRO instruments into their day-to-day management of patients with GERD.Message for the ClinicAn evidence-based review shows that PRO instruments can accurately assess the nature of GERD symptoms, their impact on HRQL and the efficacy of treatment.Primary care physicians are encouraged to make wider use of PRO instruments as part of their management of patients with GERD, given that such questionnaires can facilitate patient communication and help physicians understand and satisfy the therapeutic needs of their patients.Among validated PRO instruments, the GERD Impact Scale represents a practical tool that is easy for primary care physicians to incorporate into their everyday practice.

Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance

This guidance describes how the FDA evaluates patient-reported outcome (PRO) instruments used as effectiveness endpoints in clinical trials. It also describes our current thinking on how sponsors can develop and use study results measured by PRO instruments to support claims in approved product labeling (see appendix point 1). It does not address the use of PRO instruments for purposes beyond evaluation of claims made about a drug or medical product in its labeling. By explicitly addressing the review issues identified in this guidance, sponsors can increase the efficiency of their endpoint discussions with the FDA during the product development process, streamline the FDA's review of PRO endpoint adequacy, and provide optimal information about the patient's perspective of treatment benefit at the time of product approval.A PRO is a measurement of any aspect of a patient's health status that comes directly from the patient (i.e., without the interpretation of the patient's responses by a physician or anyone else). In clinical trials, a PRO instrument can be used to measure the impact of an intervention on one or more aspects of patients' health status, hereafter referred to as PRO concepts, ranging from the purely symptomatic (response of a headache) to more complex concepts (e.g., ability to carry out activities of daily living), to extremely complex concepts such as quality of life, which is widely understood to be a multidomain concept with physical, psychological, and social components. Data generated by a PRO instrument can provide evidence of a treatment benefit from the patient perspective. For this data to be meaningful, however, there should be evidence that the PRO instrument effectively measures the particular concept that is studied. Generally, findings measured by PRO instruments may be used to support claims in approved product labeling if the claims are derived from adequate and well-controlled investigations that use PRO instruments that reliably and validly measure the specific concepts at issue.The glossary defines many of the terms used in this guidance. In particular, the term instrument refers to the actual questions or items contained in a questionnaire or interview schedule along with all the additional information and documentation that supports the use of these items in producing a PRO measure (e.g., interviewer training and instructions, scoring and interpretation manual). The term conceptual framework refers to how items are grouped according to subconcepts or domains (e.g., the item walking without help may be grouped with another item, walking with difficulty, within the domain of ambulation, and ambulation may be further grouped into the concept of physical ability).FDA's guidance documents, including this guidance, do not establish legally enforceable responsibilities. Instead, guidance documents describe the Agency's current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidance documents means that something is suggested or recommended but not required. First publication of the Draft Guidance by the Food and Drug Administration- February 2006.

Patient reported outcomes in gastroesophageal reflux disease: An overview of available measures

Gastroesophageal reflux disease (GERD) is a common, chronic disorder. The main symptom of GERD is heartburn, although a diverse range of symptoms can be associated with the disease including acid regurgitation and epigastric pain. GERD is also a risk factor for Barrett's oesophagus and esophageal adenocarcinoma. The impact of GERD symptoms on patients' lives can be profound and is unrelated to the presence or absence of esophagitis. The impact of GERD can be measured by assessing the patient perspective using Patient Reported Outcomes (PROs). There are two categories of questionnaires that can be used to measure the effect of GERD on health-related quality of life (HRQoL), namely generic and disease or treatment specific. The use of PRO instruments has become more accepted in the assessment of disease treatment. Well-designed instruments that assess physical, psychological and emotional factors can provide clinicians with the data that will promote effective management decisions for the treatment of GERD. The most frequently used instruments in GERD are reviewed here, in terms of their psychometric properties.