Investigating Trends in the Quality of Reporting of Patient-Reported Outcomes in Oncology Over Time: Analysis of 631 Randomized Controlled Trials Published Between 2004 and 2019

ObjectivesIncomplete reporting of key information on patient-reported outcomes (PROs) in randomized controlled trials (RCTs) in oncology has been highlighted repeatedly as a major barrier to the use of study findings in clinical practice. We investigated whether the quality of reporting of PRO data in cancer RCTs has improved over the last 15 years.MethodsWe identified all cancer RCTs with PRO endpoints conducted across the most prevalent solid tumor types worldwide published between 2004 and 2019. The quality of PRO reporting was assessed using the International Society for Quality of Life Research recommended standards, which include important aspects related to assessment methodology, statistical analyses, and interpretation of data.ResultsWe assessed a total of 631 cancer RCTs in breast (n = 187), lung (n = 131), prostate (n = 120), colorectal (n = 107), and gynecological (n = 86) cancer. We observed a higher adherence to the International Society for Quality of Life Research reporting criteria in the more recently published studies. In a multivariable linear regression analysis, we observed a statistically significant improvement in the quality of PRO reporting over time (P<.001), and this relationship was independent of other measured confounding factors, such as sample size and study sponsorship. Overall, the quality of PRO reporting was higher for studies published after the publication of the Consolidated Standards of Reporting Trials-PRO Extension.ConclusionsThe quality of PRO reporting in cancer RCTs published in the last 15 years has improved significantly. Our findings are encouraging because better reporting of PRO results may translate into a greater impact of study findings on real-world practice.     

Inclusion of Patient-Reported Outcomes in Adolescent and Young Adult Phase III Therapeutic Trials: An Analysis of Cancer Clinical Trials Registered on ClinicalTrials.gov

ObjectivesThere is a paucity of research on the impact of cancer treatment on the health-related quality of life (HRQOL) of adolescent and young adult (AYA) patients with cancer. Patient-reported outcomes (PROs) are self-report measures used to assess HRQOL and symptom burden. The extent to which PROs have been included in trials that include common AYA cancer types has not been previously assessed.MethodsTherapeutic phase 3 trials among common AYA cancer types (Hodgkin lymphoma, non-Hodgkin lymphoma, acute lymphoblastic leukemia, sarcomas, and germ cell tumors) initiated between 2007 and 2020 were identified on ClinicalTrials.gov. The proportions and characteristics of trials including a PRO endpoint were assessed. For comparison with an older population, the proportion of breast and colorectal therapeutic phase 3 trials including PRO endpoints were assessed.ResultsEighty-seven studies met the inclusion criteria. Overall, 20.7% of therapeutic phase 3 AYA trials included a PRO endpoint, and only one trial published PRO data. Germ cell tumors (42.9%) and non-Hodgkin lymphoma (40%) trials had the highest proportions of PRO inclusion. The European Organization for Research and Treatment of Cancer generic, cancer-specific quality of life questionnaire was the most commonly used PRO measure; nevertheless, the measures used varied within and between cancer types. The proportion of trials including a PRO endpoint did not change significantly between 2007 to 2013 and 2014 to 2020 (18.6% vs 22.7%, P=.79).ConclusionsFew therapeutic phase 3 AYA cancer trials include PRO endpoints, fewer publish PRO data, and there is no homogeneity in the measures administered. Therapeutic trials represent an underused opportunity to capture PRO data in the AYA population with the goal of improving HRQOL outcomes.     

Concepts and Instruments for Patient-Reported Outcome Assessment in Celiac Disease: Literature Review and Experts’ Perspectives

BackgroundIn diseases where there is a large subjective component, such as celiac disease (CD), patient reported-outcomes (PRO) endpoints are highly relevant. However, there is a gap in knowledge about which PRO endpoints and instruments should be used for clinical trials for treatment of celiac disease.ObjectivesTo identify patient-centered symptom, impact, and health-related quality of life (HRQoL) concepts in CD and relevant PRO instruments, and to gather expert input on concepts and instruments to inform selection of PRO endpoints for use in clinical trials of new CD treatments.MethodsA targeted literature review was conducted to identify symptom, impact, and HRQoL concepts, including those captured in PROs further reviewed against U.S. Food and Drug Administration standards for development and validation as endpoints. US and European clinicians, payers, and a patient advocate (n = 21) were interviewed to assess the identified concepts’ relative importance in measuring treatment benefit and to gauge the value of potential PROs as endpoints for market access/reimbursement.ResultsThirty-four published studies were identified: 27 elucidated patient-centered concepts and 7 detailed the development or validation of PRO instruments. The Celiac Disease Symptom Diary and Celiac Disease Patient Reported Outcome instrument were deemed most appropriate for use as endpoints; however, each had limitations related to conceptual coverage, evidence for measurement properties, and feasibility for use in clinical trials. Experts reported gastrointestinal symptoms as most important to treat, with extra-intestinal symptoms burdensome from the patient perspective as well. Payers emphasized measuring both frequency and severity of symptoms and targeting patients nonresponsive to the gluten-free diet for treatment.ConclusionsWith emerging treatment options for CD, further work is needed to operationalize PRO symptom endpoints that are meaningful to patients, valued by payers, and acceptable to regulators in demonstrating efficacy.     

The Association Between Computed Tomography–Defined Sarcopenia and Outcomes in Adult Patients Undergoing Radiotherapy of Curative Intent for Head and Neck Cancer: A Systematic Review

BackgroundComputed tomography (CT)-defined sarcopenia is a demonstrated poor prognostic factor in patients with cancer; however, its influence on outcomes for patients with head and neck cancer (HNC) has not been established.ObjectiveThis review synthesizes current knowledge regarding the association between CT-defined sarcopenia and outcomes for adult patients undergoing radiotherapy with or without other treatment modalities of curative intent for HNC.MethodsA systematic review of the literature published between January 2004 and June 2019 was conducted in Medline, Embase, Cumulative Index of Nursing and Allied Health Literature, Allied and Complementary Medicine Database, and PubMed. Empirical studies of CT-defined sarcopenia in adult patients (≥18 years) with HNC who had completed radiotherapy of curative intent with or without other treatment modalities were included. Outcomes reported included survival, prolonged radiotherapy breaks, and chemotherapy toxicity. Study quality was assessed using the American Academy of Nutrition and Dietetics Quality Criteria Checklist. Synthesis of outcomes and clinical relevance was performed using the Grading of Recommendations Assessment, Development, and Evaluation system.ResultsOf 11 studies (n = 3,461) identified, 3 were positive and 8 were neutral quality. Studies were heterogeneous in HNC diagnosis, ethnicity, definition of sarcopenia, CT level of evaluation, and skeletal muscle index threshold value. Eight definitions for sarcopenia were identified with pretreatment prevalence of 6.6% to 70.9% and posttreatment prevalence of 12.4% to 65.8%. Pretreatment sarcopenia was independently associated with reduced: overall survival (OS), 3-year OS, disease-free survival, prolonged radiotherapy breaks, and chemotherapy-related toxicities. Posttreatment sarcopenia was independently associated with reduced OS and 5-year OS. The overall certainty of evidence according to Grading of Recommendations Assessment, Development and Evaluation criteria was low for OS; 3-year, 5-year, and 10-year OS; locoregional control; locoregional failure; progression-free survival; metastasis-free survival, disease-specific survival; and disease-free survival and very low for distant metastasis, prolonged radiotherapy breaks, and chemotherapy toxicity-related outcomes.ConclusionsCT-defined sarcopenia is independently associated with reduced OS and treatment completion in patients with HNC and holds a clinically meaningful prognostic value. The certainty of the evidence requires strengthening with further research. Understanding the impact sarcopenia has on outcomes for these patients has implications for informing potential nutrition interventions and facilitating individualized care.     

Trials with patient-reported outcomes registered on the Australian New Zealand Clinical Trials Registry (ANZCTR)

Aims

It is important to understand the number, types and regions of trials that include patient-reported outcomes (PROs) to appreciate how patient experiences have been considered in studies of health and interventions. Twenty-seven percent of trials registered with ClinicalTrials.gov (2007–2013) included PROs; however, a regional breakdown was not provided and no reviews have been conducted of the Australia New Zealand Clinical Trials Registry (ANZCTR). We aimed to identify trials registered with ANZCTR with PRO endpoints and describe their characteristics.

Methods

ANZCTR was systematically searched from inception (2005) to 31 March 2017 for trials with PRO endpoints. Search terms included PRO measures listed in Patient-Reported Outcomes Quality of Life Instrument Database and Grid-Enabled Measures, as well as generic PRO terms (e.g. “quality of life” (QOL)). Trial endpoints were individually coded using an established framework to identify trials with PROs for the analysis.

Results

Of 13,666 registered trials, 6168 (45.1%) included a PRO. The proportion of studies including PROs increased between 2006 and 2016 (r?=?0.74, p?=?0.009). Among the 6168 trials, there were 17,961 individual PRO endpoints, including symptoms/functional outcomes/condition-specific QOL (65.6%), generic QOL (13.2%), patient-reported experiences (9.9%), patient-reported behaviours (7.9%). Mental health was the most common category (99.8% included PROs), followed by physical medicine/rehabilitation (65.6%), musculoskeletal (63.5%), public health (63.1%), and cancer (54.2%).

Discussion

Our findings suggest growing use of PROs in the assessment of health and interventions in ANZ. Our review identifies trial categories with limited patient-reported information and provides a basis for future work on the impact of PRO findings in clinical care.

     

A Review of Patient-Reported Outcome Labeling of FDA-Approved New Drugs (2016-2020): Counts,Categories, and Comprehensibility

ObjectivesA review of new drug approvals (NDAs) by the US Food and Drug Administration (FDA) for 2006 to 2015 showed that approximately 20% of new drugs had labeling based on patient-reported outcomes (PROs). The purpose of this study was to review labeling text based on PRO endpoints for NDAs from 2016 to 2020, with a special focus on the comprehensibility of such statements when included.MethodsWe reviewed drug approval reports on the Drugs@FDA web page of the FDA website to determine the number of NDAs from 2016 to 2020. For all identified NDAs, drug approval package and product labels were reviewed. NDAs from 2016 to 2020 were grouped by disease category as per International Classification of Diseases 10th Revision. Data were summarized for diseases that traditionally rely on PROs for evaluating treatment benefit (PRO dependent) and for diseases that traditionally do not rely on PROs (non-PRO dependent). Results were compared with NDAs from 2006 to 2010.ResultsNDAs amounting to 228 were identified from 2016 to 2020, 26.3% of which had labeling statements based on PRO endpoints. From 2006 to 2015 and from 2016 to 2020, PRO labeling statements were included in 46.5% (46 of 99) and 50.0% (47 of 94), respectively, of NDAs for PRO-dependent new molecular entities and in 6.0% (12 of 199) and 9.7% (13 of 199), respectively, of NDAs for non–PRO-dependent new molecular entities. Comprehensibility of labeling statements based on PRO endpoints was judged to be complex in 56.7% of product labels.ConclusionsThe increase in labeling text based on PRO endpoints in product labels is encouraging. However, there is room for improvement on the comprehensibility of labeling statements based on PRO endpoints.     

Measuring,Analyzing, and Presenting Work Productivity Loss in Randomized Controlled Trials: A Scoping Review

ObjectivesThis study aimed to conduct a scoping review of randomized controlled trials (RCTs) and investigate which work productivity loss outcomes were measured in these RCTs, how each outcome was measured and analyzed, and how the results for each outcome were presented.MethodsA systematic search was conducted from January 2010 to April 2020 from 2 databases: PubMed and Cochrane Central Register of Controlled Trials. Data on country, study population, disease focus, sample size, work productivity loss outcomes measured (absenteeism, presenteeism, employment status changes), and methods used to measure, report, and analyze each work productivity loss outcome were extracted and analyzed.ResultsWe found 435 studies measuring absenteeism or presenteeism, of which 155 studies (35.6%) measured both absenteeism and presenteeism and were included in our final review. Only 9 studies also measured employment status changes. The most used questionnaire was the Work Productivity and Activity Impairment Questionnaire. The analysis of absenteeism and presenteeism data was mostly done using regression models (n = 98, n = 98, respectively) for which a normal distribution was assumed (n = 77, n = 89, respectively). Absenteeism results were most often presented in time whereas presenteeism was commonly presented using a percent scale or score.ConclusionsThere is a lack of consensus on how to measure, analyze, and present work productivity loss outcomes in RCTs published in the past 10 years. The diversity of measurement, analysis, and presentation methods used in RCTs may make comparability challenging. There is a need for guidelines providing recommendations to standardize the comprehensiveness and the appropriateness of methods used to measure, analyze, and report work productivity loss in RCTs.     

Reasons for Rejection of Patient-Reported Outcome Label Claims: A Compilation Based on a Review of Patient-Reported Outcome Use among New Molecular Entities and Biologic License Applications, 2006–2010

ObjectivesPrevious analyses of patient-reported outcome (PRO) label claims concentrated only on successful label claims. The goal of this research was to explore the reasons why PRO label claims were denied and to compile regulatory feedback regarding the use of PROs in clinical trials.MethodsBy using the Food and Drug Administration's Drug Approval Report Web page, all new molecular entities and biologic license applications approved between January 2006 and December 2010 were identified. For identified drug products, medical review sections from publicly available drug approval packages were reviewed to identify PRO end-point status and any Study Endpoints and Label Development team comments.ResultsOf the 116 new molecular entities and biologic license applications with accompanying drug approval packages identified and reviewed, 44.8% of the products included PROs as part of the pivotal studies; however, only 24.1% received PRO label claims. Primary reasons for denial included issues of fit for purpose, issues of study design, data quality or interpretation, statistical issues, administrative issues, and lack of demonstrated treatment benefit.ConclusionsBased on drug approval packages, nearly half (45%) of new molecular entitity/biologic license application products in the years 2006 to 2010 included PROs in the clinical trials supporting their approval, yet this rate is not reflected by claims granted. Understanding the nature of PRO claims granted under the current regulatory guidance is important. In addition, a clear understanding of denied claims yields valuable insight into where sponsors may improve implementation of PROs in clinical trials and submission of PRO evidence to increase the likelihood of obtaining PRO label claims.     

Efficacy,Patient-Reported Outcomes (PROs), and Tolerability of the Changing Therapeutic Landscape in Patients with Metastatic Prostate Cancer (MPC): A Systematic Literature Review

ObjectiveNew therapies have attempted to improve on efficacy outcomes observed with docetaxel in patients with metastatic prostate cancer (MPC) who are hormone-therapy refractory or castration-resistant. In addition to the efficacy, patient-reported outcomes (PROs) and tolerability need to be assessed to define treatment benefit, as PROs measure the patient’s subjective experience and can be correlated with hard outcomes. The main objective of this study was to evaluate the survival benefit of new therapies and secondary efficacy-related outcomes. Assessment of the number of studies reporting PROs and tolerability was also conducted.MethodsA predefined search strategy was conducted on major academic/governmental databases and conference proceedings (2007–2011). Exclusion criteria were applied.ResultsOf 77 studies identified, 26 (34%) evaluated survival as an end point; 14 (18%) assessed PROs/tolerability. In chemotherapy-naive patients (no/minimal symptoms), median overall survival (OS) was 26 months for sipuleucel-T. In relapsed patients, the survival benefit of cabazitaxel/abiraterone was 15 months and that of enzalutamide was 18 months. Denosumab prolonged time to first on-study skeletal-related event (20.7 months denosumab, 17.1 months zoledronic acid; P = 0.0002, noninferiority; P = 0.008, superiority). Similar benefit was documented with radium-223, a new bone-targeted α-particle–emitting radiopharmaceutical. Radium-223 also significantly improved the OS (two-sided P = 0.00185). Specific to PROs, they were incorporated primarily as secondary end points, and improvements in pain response (most commonly evaluated) were variable among the agents. Last, the therapies were associated with unique toxicities requiring careful consideration.ConclusionsThe results of this review demonstrate that the therapeutic landscape of MPC has changed dramatically and many therapies in MPC now show OS improvements of about 4 months in the postdocetaxel setting.     

Using patient-reported outcomes in clinical practice: challenges and opportunities

Purpose

Introduce and explore issues at an international conference about the use of patient-reported outcomes (PROs) in clinical practice.

Methods

Review of salient literature, clinical and personal experiences, conference presentations and discussions, and post-conference comments from outside experts.

Results

PROs (information from patients about a health condition and its management) have been assessed through self-reports for at least four decades. Traditional applications are in clinical and health services research. Uses in clinical practice, although increasing, are less common and more challenging. PROs can enhance the understanding of patients’ experiences and responses to therapy and inform clinical practice.

Conclusions

We pose and discuss four main questions: (1) Will clinicians accept PRO measures? (2) Will clinicians use PRO measures? (3) Will measuring PROs actually improve those outcomes? (4) Will PROs be perceived as having other, less salutary purposes? A patient-centered perspective on PRO measurement presents issues about the extent to which PROs can accurately capture patient experiences and assess psychosocial and environmental factors that influence communication with clinicians and eventual outcomes. We end with comments about the intersection of PROs and bioethics, noting contributions that PROs may make to beneficence, patient autonomy, and distributive justice.     

Health-Related Quality of Life and Other Patient-Reported Outcomes in the European Centralized Drug Regulatory Process: A Review of Guidance Documents and Performed Authorizations of Medicinal Products 1995 to 2003

OBJECTIVES: The objective of this study was to review and analyze the use of health-related quality of life (HRQL) and other patient-reported outcome (PRO) evaluations for the approval of new pharmaceutical products by the European Medicines Agency (EMEA). METHODS: All published EMEA guidance documents and regulatory information for products authorized at the EMEA and appearing in the European Public Assessment Report (EPAR) database between 1995 and 2003 were examined for reference to HRQL and other PROs. RESULTS: More than half of the guidance documents for clinical investigation of pharmaceutical products in specific disease areas included reference to HRQL or other PROs. Guidance notes for 10 conditions indicated PROs can serve as primary endpoints in clinical trials, among which three included HRQL outcomes. The review of EPAR documentation uncovered HRQL and other PRO data for 34% of the drugs registered during the period of the review, with cancer-related treatments most frequently including PRO data. There was a trend toward increasing HRQL and other PRO claims in regulatory documents of pharmaceutical products in recent years, with the proportion exceeding 30% from 1999 to 2003. CONCLUSIONS: There is further scope for health outcomes researchers and regulatory decision-makers to contribute to the more efficient utilization of PROs and HRQL outcomes. Health researchers need to better justify the inclusion of these outcomes in clinical trials and highlight the added value of PRO data; while the regulators should develop harmonized procedures and capacities to adequately appraise the submitted information.     

Assessment of PRO Label Claims Granted by the FDA as Compared to the EMA (2006–2010)

BackgroundThe US Food and Drug Administration (FDA) provides formal guidance for the use of patient-reported outcomes (PROs) in support of labeling claims, whereas the European Medicines Agency (EMA) offers insight in a reflection paper relating to health-related quality of life in lieu of formal guidance.ObjectivesPRO label claims granted for new molecular entities and biologic license applications from 2006 through 2010 were reviewed to evaluate consistencies and discrepancies in PRO label claims granted by the FDA and the EMA and to highlight trends in the acceptance of PRO claims across agencies.MethodsProducts approved by both the FDA and the EMA were identified. By using US Drug Approval Packages and European Public Assessment Reports packages, any PRO label claims made for the same product by the same company were compared.ResultsBoth agencies approved a total of 75 products. Of these, 35 (47%) had at least one EMA-granted PRO label claim compared with 14 (19%) by the FDA. Most FDA-grated claims focused on symptoms; however, EMA-granted claims were more likely to include higher order concepts. Few (~12%) were granted the same label claims. Despite this discordance between the two agencies, where PRO label claims were granted by both the FDA and the EMA, there was similarity in the type of label claim.ConclusionsThe EMA is more likely than the FDA to grant PRO claims and for higher order constructs. On a macro level, there appears to be poor concordance between claims granted by both agencies. On close examination, however, there appears to be greater concordance than previously recognized, which may be instructive in formulating future PRO strategies. Further research to create strategic alignment across agencies may be beneficial.     

Modeling strategies to improve parameter estimates in prognostic factors analyses with patient-reported outcomes in oncology

Purpose

The inclusion of patient-reported outcome (PRO) questionnaires in prognostic factor analyses in oncology has substantially increased in recent years. We performed a simulation study to compare the performances of four different modeling strategies in estimating the prognostic impact of multiple collinear scales from PRO questionnaires.

Methods

We generated multiple scenarios describing survival data with different sample sizes, event rates and degrees of multicollinearity among five PRO scales. We used the Cox proportional hazards (PH) model to estimate the hazard ratios (HR) using automatic selection procedures, which were based on either the likelihood ratio-test (Cox-PV) or the Akaike Information Criterion (Cox-AIC). We also used Cox PH models which included all variables and were either penalized using the Ridge regression (Cox-R) or were estimated as usual (Cox-Full). For each scenario, we simulated 1000 independent datasets and compared the average outcomes of all methods.

Results

The Cox-R showed similar or better performances with respect to the other methods, particularly in scenarios with medium–high multicollinearity (ρ?=?0.4 to ρ?=?0.8) and small sample sizes (n?=?100). Overall, the Cox-PV and Cox-AIC performed worse, for example they did not select one or more prognostic collinear PRO scales in some scenarios. Compared with the Cox-Full, the Cox-R provided HR estimates with similar bias patterns but smaller root-mean-squared errors, particularly in higher multicollinearity scenarios.

Conclusions

Our findings suggest that the Cox-R is the best approach when performing prognostic factor analyses with multiple and collinear PRO scales, particularly in situations of high multicollinearity, small sample sizes and low event rates.

     

Prognostic value of patient-reported symptom interference in patients with late-stage lung cancer

Purpose

Patient-reported outcomes (PROs) have been found to be significant predictors of clinical outcomes such as overall survival (OS), but the effect of demographic and clinical factors on the prognostic ability of PROs is less understood. Several PROs derived from the 12-item Short-Form Health Survey (SF-12) and M. D. Anderson Symptom Inventory (MDASI) were investigated for association with OS, with adjustments for other factors, including performance status.

Methods

A retrospective analysis was performed on data from 90 patients with stage IV non-small cell lung cancer. Several baseline PROs were added to a base Cox proportional hazards model to examine the marginal significance and improvement in model fit attributable to the PRO: mean MDASI symptom interference level; mean MDASI symptom severity level for five selected symptoms; SF-12 physical and mental component summaries; and the SF-12 general health item. Bootstrap resampling was used to assess the robustness of the findings.

Results

The MDASI mean interference level had a significant effect on OS (p = 0.007) when the model was not adjusted for interactions with other prognostic factors. Further exploration suggested the significance was due to an interaction with performance status (p = 0.001). The MDASI mean symptom severity level and the SF-12 physical component summary, mental component summary, and general health item did not have a significant effect on OS.

Conclusions

Symptom interference adds prognostic information for OS in advanced lung cancer patients with poor performance status, even when demographic and clinical prognostic factors are accounted for.     

French Patients with Hepatitis C Treated with Direct-Acting Antiviral Combinations: The Effect on Patient-Reported Outcomes

Background

In addition to high efficacy, new anti–hepatitis C virus (HCV) regimens improve patient-reported outcomes (PROs), which must be considered by policymakers in different countries when deciding upon treatment coverage.

A Review of Labeling Based on Patient-Reported Outcome Endpoints for New Oncology Drugs Approved by the European Medicines Agency (2017-2021)

ObjectiveA review of new oncology indications approved by the European Medicines Agency (EMA) for 2012-2016 showed that 33% of new drugs had labeling based on patient-reported outcomes (PROs). We reviewed labeling text based on PRO endpoints for new oncology indications approved during 2017-2021.MethodsNew oncology drugs approved by EMA to treat indications of cancers during 2017-2021 were identified from the EMA website. PRO-related language reported in EMA summaries of product characteristics (SmPCs) were summarized and compared with similar findings reported for oncology indications approved during 2012-2016.ResultsReview documents by the EMA during 2017-2021 were available for 49 new oncology drugs for 70 cancer indications. Submissions for 52 (74.3%) of the 70 indications included PRO data for EMA review. Of all submissions, 14 (20.0%) approvals contained PRO-related language in the SmPC. Broad concepts such as health-related quality of life were most common and found in 8 of 14 (57.1%) PRO-related labels.ConclusionPRO-related language appeared in SmPCs for 20% of all indications of new oncology drugs approved by EMA during 2017-2021 compared with approximately 33% of EMA approvals during 2012-2016. PRO-related labeling during the same periods showed a greater decline (from 47% to 27%) for indications of new oncology drugs that also included PRO data. One possible reason for this decline may be the increase in open-label studies from 62% between 2012 and 2016 to approximately 79% between 2017 and 2021.     

Methods for Developing Patient-Reported Outcome-Based Performance Measures (PRO-PMs)

ObjectiveTo recommend methods for assessing quality of care via patient-reported outcome-based performance measures (PRO-PMs) of symptoms, functional status, and quality of life.MethodsA Technical Expert Panel was assembled by the American Medical Association–convened Physician Consortium for Performance Improvement. An environmental scan and structured literature review were conducted to identify quality programs that integrate PRO-PMs. Key methodological considerations in the design, implementation, and analysis of these PRO-PM data were systematically identified. Recommended methods for addressing each identified consideration were developed on the basis of published patient-reported outcome (PRO) standards and refined through public comment. Literature review focused on programs using PROs to assess performance and on PRO guidance documents.ResultsThirteen PRO programs and 10 guidance documents were identified. Nine best practices were developed, including the following: provide a rationale for measuring the outcome and for using a PRO-PM; describe the context of use; select a measure that is meaningful to patients with adequate psychometric properties; provide evidence of the measure’s sensitivity to differences in care; address missing data and risk adjustment; and provide a framework for implementation, interpretation, dissemination, and continuous refinement.ConclusionMethods for integrating PROs into performance measurement are available.     

Common Patient-Reported Outcomes Within the Food and Drug Administration Voice of the Patient Reports

ObjectivesProponents of disease-specific patient-reported outcome measurements (PROMs) often argue disease-agnostic measures do not adequately capture their patient population’s experience. Patient-Reported Outcomes Measurement Information System (PROMIS) provides a disease-agnostic domain set that may adequately cover many diseases. This study seeks to investigate whether PROMIS’s quality of life domain coverage can span patient-reported outcomes (PROs) elicited from patients across unrelated diseases.MethodsThe Food and Drug Administration Voice of the Patient reports were an initiative to elevate patient voices regarding their condition and associated treatments. Two reviewers extracted patient-reported health-related (quality of life) domains from the reports and categorized them into PROMIS domains or non-PROMIS domains. Domain coverage was summarized for each report. Any extracted PROs not covered by PROMIS domains were placed in an “other” category and analyzed for common themes.ResultsAcross 26 reports, PROMIS covered 216 of 374 (70%) of the reports’ PRO domains. The heritable bleeding disorders report had the highest coverage (82%). Human immunodeficiency virus had the lowest coverage (50%). The most common PROMIS domain, “ability to participate in social roles,” appeared in 25 reports (96%). The most common domains not included in PROMIS were stigma, sensitivities, and sensory deficits as evident in 19 (73%), 18 (69%), and 18 reports (69%), respectively. If the top 3 unincluded domains were amended into PROMIS, the total domain coverage would increase to 84%.ConclusionsPRO domains elicited in the Food and Drug Administration Voice of the Patient reports were widely captured by PROMIS, suggesting domains patients experience contain enough overlap to be recorded by appropriate PROMIS domains. PROMIS could increase its coverage by adding domains.