In France, decisions for reimbursement taken by the Ministry of Health are based on appraisal by an independent Health Technology Assessment body (HTAb): the ‘Haute Autorité de santé’ (HAS). HAS grades the clinical added value of any medicinal product for which a manufacturer seeks reimbursement. This appraisal considers different types of clinical and patient-centred outcomes, including patient-reported ones. Under certain conditions, a concomitant economic assessment which accounts for patients’ preferences in the form of utility values is also performed.
As providing fast access to breakthrough therapies is a critical expectation from patients, clinicians and health policy makers, the European Medicines Agency and the Food and Drug Administration have established various accelerated approval pathways. These procedures lead to conditional approvals frequently based on uncontrolled (ie, single arm) pivotal trials.
WHAT IS ALREADY KNOWN ON THIS TOPIC
Publication reporting bias is known to be a major threat for evidence-based medicine, but it is less appreciated that an inverse bias may operate sometimes, favouring the publication and dissemination of non-significant, null results.
Inverse publication reporting bias may operate in diverse settings including non-inferiority studies, adverse events literature, other reassurance-oriented narratives, the Proteus phenomenon and reproducibility checks.
More transparency in protocols, statistical analysis plans and registration, reduction of conflicts and heightened awareness may help diminish the impact of inverse publication reporting bias.
Classic publication (‘file drawer’) bias and related reporting biases threaten the validity of the scientific literature. Theoretical considerations and empirical data have demonstrated that in many fields and settings, there is preference for publishing studies...]]>
Choosing wisely (CW) is a campaign for healthcare professionals and patients to engage in conversations about unnecessary tests, treatments and procedures, and to help them make smart and effective choices to ensure high-quality care.
In the last decades, researchers, governments and public campaigns have increased awareness about healthcare overuse.
We aimed to systematically identify and scrutinise published empirical evidence about overdiagnosis in malignant melanoma and examine how frequent overdiagnosis of melanoma is and whether this is related to different types of interventions or diagnostic technologies.
Empirical studies that discussed overdiagnosis in malignant melanoma were eligible, including qualitative and quantitative studies in any type of population, age group and geographical location. We excluded studies that did not include empirical data, studies that only mentioned ‘overdiagnosis’ without addressing it further and studies that used the term overdiagnosis for cases of misdiagnosis or false positives.
We developed the search strategy in cooperation with an information specialist. We searched five databases on 21 April 2022: MEDLINE, Embase, CINAHL, PsycINFO and Cochrane Library.
This scoping review adheres to The JBI methodology and Prefered Reporting Items for Systematic Reviews and Meta-analyses extension for Scoping reviews (PRISMA-ScR). Two reviewers independently screened titles, abstracts and full texts for inclusion and extracted data from the included studies. The data extracted include study characteristics, population details, research question, the context and the study’s main results.
Our search resulted in 1134 potentially relevant studies. 35 studies were included: 29 register studies, 3 cohort studies, 1 case–control study, 1 survey study and 1 randomised controlled trial. Most register studies examined trends in melanoma incidence and/or mortality and found a significant increase in incidence between 0.39% and 6.6% annually and a little or no increase in mortality. Three cohort studies and one case–control study showed that skin screening was associated with increased detection of melanoma; especially in situ or thin invasive melanoma. Three studies estimated the degree of overdiagnosis which ranged from 29% to 60%.
Epidemiological data suggest a high degree of overdiagnosis in malignant melanoma. Studies that examined the association between skin screening and malignant melanoma all found increased detection of melanomas, mostly thin and in situ melanomas, which raises concern about overdiagnosis.
To investigate medical students’ ability to interpret evidence, as well as their self-assessed understandability, perceived usefulness and preferences for design alternatives in an interactive decision support tool, displaying GRADE evidence summaries for multiple treatment options (Making Alternative Treatment CHoices Intuitive and Trustworthy, MATCH-IT).
A combined randomised controlled trial and survey. Participants were presented with a clinical scenario and randomised to one of two versions of the MATCH-IT tool (A/B), instructed to explore the evidence and decide on a recommendation. Participants answered a questionnaire assessing interpretation, treatment recommendation self-assessed understandability and perceived usefulness before exposure to the other MATCH-IT version and asked questions on design preferences.
Online lecture in an evidence-based medicine (EBM) introductory course.
149 third-year medical students. 52% (n=77) had 6 months of clinical training and 48% (n=72) had preclinical training only.
The MATCH-IT tool version A uses colour coding to categorise interventions by magnitude and direction of effects and displays all outcomes in a table on entry. Version B has no colour coding, and the user must decide which outcomes to display in the table.
Interpretation of evidence, treatment recommendation, perceived usefulness and understandability, preference for format and design alternatives.
82.5% (n=123) of medical students correctly answered ≥4 out of 5 multiple choice questions assessing interpretation of data. 75.8% (n=114) of students made a treatment recommendation in accordance with an expert panel for the same clinical scenario. 87.2% (n=130) found the tool understandable while 91.9% perceived the tool as useful in addressing the clinical scenario.
Medical students with no prior training in EBM can interpret and use the MATCH-IT tool. Certain design alternatives were preferred but had no bearing on interpretation of evidence or understandability of the tool.
To systematically collect and analyse diverse definitions of ‘evidence’ in both health and social sciences, and help users to correctly use the term ‘evidence’ and rethink what is the definition of ‘evidence’ in scientific research.
Scoping review.
Definitions of evidence in the health sciences and social sciences were included. We have excluded the definition of evidence applied in the legal field, abstracts without full text, documents not published in either Chinese or English and so on. We established a multidisciplinary working group and systematically searched five electronic databases including Medline, Web of Science, EBSCO, the Chinese Social Sciences Citation Index and the Chinese Science Citation Database from their inception to 26 February 2022. We also searched websites and reviewed the reference lists of the identified studies. Six reviewers working in pairs, independently, selected studies according to the inclusion and exclusion criteria, and extracted information. Any differences were discussed in pairs, and if there was disagreement, it was resolved via discussion or with the help of a third reviewer. Reviewers extracted document characteristics, the original content for the definitions of ‘evidence’, assessed definitions as either intensional or extensional, and any citations for the given definition.
Forty-nine documents were finally included after screening, and 68 definitions were obtained. After excluding duplicates, a total of 54 different definitions of ‘evidence’ were identified. There were 42 intensional definitions and 12 extensional definitions. The top three definiens were ‘information’, ‘fact’ and ‘research/study’. The definition of ‘evidence’ differed between health and social sciences. The term ‘research’ appeared most frequently in the definitions.
The definition of ‘evidence’ has gradually attracted the attention of many scholars and decision-makers in health and social sciences. Nevertheless, there is no widely recognised and accepted definition in scientific research. Given the wide use of the term, we need to think about whether, or under what circumstances, a standardised, clear, meaningful and widely applicable definition of ‘evidence’ might be helpful.
If a new clinical trial is to be justifiable both scientifically and ethically it should be designed in the light of an assessment of relevant previous research, ideally a systematic review. When its findings are reported, these should be set in the context of updated reviews of other, similar research.
When reading the report of a new controlled trial, interpretation will be greatly aided if the Discussion sets the results in the context of the results from available similar research.
Consider the aspirational goal of the UK’s National Health Service (NHS): to increase the proportion of all cancers diagnosed at an early stage from approximately 50%–75%.
If time or other resources do not permit the full implementation of GRADE, the following recommendations can be considered: (1) limit rating COE to the main intervention and comparator and limit the number of outcomes to critical benefits and harms; (2) if a literature review or a Delphi approach to rate the importance of outcomes is not feasible, rely on informal judgements of knowledge users, topic experts or team members; (3) replace independent rating of the COE by two reviewers with single-reviewer rating and verification by a second reviewer and (4) if effect estimates of a well-conducted systematic review are incorporated into an RR, use existing COE grades from such a review. We advise against changing the definition of COE or the domains considered part of the GRADE approach for RRs.
]]>Although my treatment was deemed to be curative, the diagnosis had defined the previous few years of my life. I had been warned that I was at risk of developing more melanomas in the future. Sun cream became a permanent feature of my bag; every time the sun shone I was reminded of its carcinogenic potential. After six months of checkups, responsibility for preventing future deadly disease...]]>
Non-publication of completed clinical trials has been identified as a major contributor to research waste.
Here, we report the results of a representative, multinational survey of primary care physician perspectives on how virtual care changed their clinical practices. The Commonwealth Fund’s 2022 International Health Policy Survey of Primary Care Physicians reflects the experiences and perceptions of...]]>
The domain of large language and transformer...]]>