Contemporary Practice and Considerations for Real-World Data Source Identification and Feasibility Assessment.

PURPOSE: There has been rapid growth in the variety and number of real-world data (RWD) sources, as well as the number of regulatory documents that provide guidance for assessing the suitability of RWD sources for pharmacoepidemiology studies. This study aims to assess differences in RWD guidance and variability in current practice for identifying and assessing RWD for studies with regulatory purpose. METHODS: Key criteria for feasibility assessment were mapped against relevant regulatory guidance documents across US, EU, and Asia-Pacific regions. An online survey was designed and deployed to International Society for Pharmacoepidemiology members to understand current practice. Findings were summarized and used to inform key considerations and recommendations. RESULTS: Eleven RWD guidance documents were identified and mapped against 14 RWD assessment criteria. Variability was seen across these documents in guidance for these criteria. Between December 2022 and January 2023, 37 survey respondents reported having used RWD for post-marketing commitments (34, 92%) and/or background epidemiology (28, 76%). RWD were mostly identified through literature (33, 89%) and data landscaping (26, 70%); guidance documents referenced included: Food and Drug Administration (20, 54%), European Network for Centres for Pharmacoepidemiology and Pharmacovigilance (17, 46%), European Medical Agency (16, 43%), and Structured Process to Identify Fit-For-Purpose Data (11, 30%). Challenges for conducting feasibility assessments included RWD accessibility, ability to complete validation, and RWD provider responsiveness. CONCLUSIONS: Existing guidelines are used extensively by researchers, but key criteria for RWD identification and feasibility assessment are not reflected consistently and challenges remain. Recommendations have been made reflecting study findings.

Clinical trial transparency update: an assessment of the disclosure of results of company-sponsored trials associated with new medicines approved in Europe in 2014.

BACKGROUND: The objective of this study was to assess the timely disclosure of results of company-sponsored clinical trials related to all new medicines approved by the European Medicines Agency (EMA) during 2014. This is the final extension of three previously reported studies of trials related to all new medicines approved in Europe in 2009, 2010 and 2011, and in 2012 and 2013. The original study found that over a three-year period over three-quarters of all trials were disclosed within 12 months and almost 90% were disclosed by the end of the study (31 January 2013). The extension studies (2012 and 2013 approvals) both showed an improvement in results disclosure within 12 months to 90%, and an overall disclosure rate of 92% and 93% respectively by the end of the studies. METHODS: The methodology used was exactly as previously reported. Various publicly available information sources were searched for both clinical trial registration and disclosure of results. All completed company-sponsored trials related to each new medicine approved for marketing by the EMA in 2014, carried out in patients and recorded on a clinical trials registry and/or included in an EMA European Public Assessment Report (EPAR), were included. Information sources were searched between 1 May and 31 July 2016. OUTCOME MEASURES AND RESULTS: The main outcome measure was the proportion of trials for which results had been disclosed on a registry or in the scientific literature either within 12 months of the later of either first regulatory approval or trial completion, or by 31 July 2016 (end of survey). Of the completed trials associated with 32 new medicines licensed to 22 different companies in 2014, results of 93% (505/542) had been disclosed within 12 months, and results of 96% (518/542) had been disclosed by 31 July 2016. CONCLUSIONS: The disclosure rate within 12 months of 93% suggests that industry is continuing to achieve disclosure in a timely manner. The overall disclosure rate at study end of 96% indicates that the improvement in transparency amongst company-sponsored trials has been maintained in the trials associated with new medicines approved in 2014.

A description of health care provision and access to treatment for women with urinary incontinence in Europe -- a five-country comparison.

BACKGROUND: Female urinary incontinence is a prevalent condition, but only about one-third of women seek treatment. OBJECTIVES: To describe the health care provision for women with urinary incontinence from a European perspective, selecting France, Germany, Spain, Sweden, and the United Kingdom as examples, and to investigate whether specific barriers for treatment exist. METHODS: Available health care system information, a literature review and clinical expert information identified patterns of treatment provision. RESULTS: In Spain, Sweden, and the UK, access to medical care in general is primarily through the general practitioners. However, in Spain and Sweden, women with urinary incontinence can directly visit specialists. In France and Germany, women have equal access to either general practitioners or specialists. Aside from general practitioners, gynaecologists play a major role in urinary incontinence care in all countries except the UK. In Germany, urologists are also involved in initial female urinary incontinence care; however, only in about 16% of women. There are no waiting lists in France and Germany for appointments with physicians or procedures, contrary to Spain, which has long waiting lists. Access to general practitioners in the UK is unrestricted whereas advanced diagnosis and treatment in secondary care requires long waits. A specific Swedish policy mandates that no woman is required to wait longer than 3 months for incontinence visits and related surgery. In Sweden and the UK, specialist nurses and other health care workers provide incontinence services. Almost all treatment options for urinary incontinence are at least in part reimbursed. However, various co-payments and fees in France, Germany, Spain and Sweden exist and constitute out-of-pocket expenses for women if no complementary additional private health insurance is available. In some countries, financial incentives for physicians to provide incontinence services are low, raising concerns about their interest to engage in continued patient care. CONCLUSIONS: Information about service provision in Europe for women with urinary incontinence is limited and makes it difficult to understand barriers to treatment seeking. A broad European perspective may promote optimised treatment access in the future for this widespread and under-recognised condition.