A scoping review of core outcome sets and their ‘mapping’ onto real-world data using prostate cancer as a case study

Abstract

Background: A Core Outcomes Set (COS) is an agreed minimum set of outcomes that should be reported in all clinical studies related to a specific condition. Using prostate cancer as a case study, we identified, summarized, and critically appraised published COS development studies and assessed the degree of overlap between them and selected real-world data (RWD) sources. Methods: We conducted a scoping review of the Core Outcome Measures in Effectiveness Trials (COMET) Initiative database to identify all COS studies developed for prostate cancer. Several characteristics (i.e., study type, methods for consensus, type of participants, outcomes included in COS and corresponding measurement instruments, timing, and sources) were extracted from the studies; outcomes were classified according to a predefined 38-item taxonomy. The study methodology was assessed based on the recent COS-STAndards for Development (COS-STAD) recommendations. A 'mapping' exercise was conducted between the COS identified and RWD routinely collected in selected European countries. Results: Eleven COS development studies published between 1995 and 2017 were retrieved, of which 8 were classified as 'COS for clinical trials and clinical research', 2 as 'COS for practice' and 1 as 'COS patient reported outcomes'. Recommended outcomes were mainly categorized into 'mortality and survival' (17%), 'outcomes related to neoplasm' (18%), and 'renal and urinary outcomes' (13%) with no relevant differences among COS study types. The studies generally fulfilled the criteria for the COS-STAD 'scope specification' domain but not the 'stakeholders involved' and 'consensus process' domains. About 72% overlap existed between COS and linked administrative data sources, with important gaps. Linking with patient registries improved coverage (85%), but was sometimes limited to smaller follow-up patient groups. Conclusions: This scoping review identified few COS development studies in prostate cancer, some quite dated and with a growing level of methodological quality over time. This study revealed promising overlap between COS and RWD sources, though with important limitations; linking established, national patient registries to administrative data provide the best means to additionally capture patient-reported and some clinical outcomes over time. Thus, increasing the combination of different data sources and the interoperability of systems to follow larger patient groups in RWD is required.

Do existing real-world data sources generate suitable evidence for the HTA of medical devices in Europe? Mapping and critical appraisal

“…Importantly, for some registries, mostly in Scandinavian countries, data linkage is possible and, therefore, even if registries do not directly include economic outcomes, these can be obtained and/or expanded through other administrative data sets. It is very important to enable as much as possible data linkage (20), to avoid enlarging existing data sets with additional variables, which is expensive in terms of costs and time, and to capitalize on existing sources.…”

Patient-reported outcome measures in core outcome sets targeted overlapping domains but through different instruments

“…Patients, as well as health care professionals administering PROMs, are unlikely or unable to spend a considerable amount of time filling out questionnaires. In a recent mapping exercise from COS studies in prostate cancer to existing real world data sources, we found that self-reported outcome measures are a dimension not typically covered in routinely collected data sources (58). However, the current technological landscape would allow for a wide scale, standardized, continuous collection of PROMs (2).…”

A Systematic Review of Heterogeneity in Outcome Definition and Reporting in Localised Renal Cancer

“…Across many clinical areas including urology, patient-reported outcomes and clinical outcomes are reported inconsistently, with variability in definitions and measurement, for instance in the settings of localised prostate cancer and bladder cancer [8] , [9] , [10] . This makes it very difficult to compare and synthesise outcomes to improve guidelines to better direct and support patients and clinicians during treatment decision-making and ultimately improve results in clinical practice [11] , [12] .…”