Aspects of dissemination and communication of study results include, but are not limited to, reports to health authorities and study sponsors, presentations in scientific fora, scientific publications, patient-focused communications, and websites dedicated to publishing study reports.
Important specific points relating to reporting of study results that are common to the various guidelines cited in this Chapter are that:
Sources of research funding should always be disclosed whether in oral or written presentation of results.
A dissemination and communication strategy should be pre-defined as part of the funding contract for a given study.
All results with a scientific or public health impact must be reported to relevant authorities and made publicly available without undue delay.
Quantitative measures of association should be reported rather than just results of statistical testing.
The Declaration of Helsinki provides overarching guidance on the registration, publication and dissemination of research results. Every research study involving human subjects must be registered in a publicly accessible database before recruitment of the first subject. A means to achieve this with pharmacoepidemiology and pharmacovigilance studies is through registration of protocols and reports of studies in the European Union electronic Register of Post-Authorisation Studies (EU PAS Register), ideally before they start. Making protocols and study results public in this Register is compulsory only for study imposed by regulators, however, transparent communication of research outside of regulatory requirements is encouraged. In addition to the EU PAS Register, study protocols and reports can also be registered and posted on other platforms: ClinicalTrials.gov includes specific guidelines for the posting of non-interventional research, while since 2020, the Open Science Forum has a specific registration portal for observational studies. The prospective register PROSPERO can be used for systematic reviews and meta-analyses.
Transparency of real-world evidence (RWE) studies starts with transparency of the study protocol. The European Medicines Agency (EMA) Guidance for the format and content of the protocol of non-interventional post-authorisation safety studies has been available since 2012 and provides the template required for study protocols submitted to EU regulators. Derived from an international consensus, the HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: A good practices report of a joint ISPE/ISPOR task force (Pharmacoepidemiol Drug Saf. 2023;32(1):44-55) became available in 2023 to guide structure and content of RWE study protocols with a focus on providing best possible detail of the operational study parameters used to create analytic datasets from the data collected for the study. A key component for design transparency, also included in the HARPER template, is the Graphical Depiction of Longitudinal Study Designs in Health Care Databases (Ann Intern Med. 2019;19;170(6):398-406), a framework of graphical representations that clarifies critical design choices. It helps researchers and reviewers to think systematically about time-related aspects in the context of typical study designs when designing studies or preparing manuscripts, which may increase confidence in evidence generated from non-randomised database studies.
Authors of publications should conform to the guidelines, including authorship criteria, established by the International Committee of Medical Journal Editors (ICJME) ‘Recommendations for the Conduct, Reporting, Editing, and Publication of Scholarly work in Medical Journals’.
The ENCePP Code of Conduct states that “the (primary) lead investigator shall be ultimately responsible for the design of the protocol, the conduct of the study, the analysis and interpretation of the study results and the preparation and publication of the study outcome.”
The ISPE GPP contain a section on communication (section V) which includes a statement that there is an ethical obligation to disseminate findings of potential scientific or public health importance and that research sponsors (government agencies, private sector, etc.) shall be informed of study results in a manner that complies with local regulatory requirements. The European Medicines Agency (EMA) Guidance for the format and content of the protocol of non-interventional post-authorisation safety studies states that plans for disseminating and communicating study results are to be described as part of study planning activities.
The EMA Guidance for the format and content of the final study report of non-interventional post-authorisation safety studies (PASS) provides a template for final study reports that may be applied to any non-interventional study, including meta-analyses and systematic reviews. The FDA’s Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Health Care Data Sets includes a description of all the elements that should be addressed and included in the final study report of such studies.
The Strengthening the Reporting of Observational studies in Epidemiology (STROBE) Statement Guidelines for reporting observational studies has established recommendations for improving the quality of reporting of observational studies and seeks to ensure a clear presentation of what was planned, done, and found. Of note, the aim of these guidelines was not to require the reporting of observational research in a rigid format, but to address what should be the essential information contained in a publication on an observational study. The REporting of studies Conducted using Observational Routinely-collected health Data (RECORD) Statement (PLoS Med. 2015;12(10):e1001885) was created as an extension to the STROBE statement to address reporting items specific to observational studies using routinely collected health data. RECORD makes additional recommendations on the reporting of methods of selection of study populations, exposures, outcomes and covariates (including codes or algorithms used), whether validation has been conducted, the level of access to databases used, and data linkages that were required to conduct the study. The RECORD-PE statement (BMJ. 2018;363:k3532) aims to extend existing STROBE and RECORD guidelines providing guidance for the reporting of pharmacoepidemiological studies using routinely collected data. Retraction of COVID-19 Pharmacoepidemiology Research Could Have Been Avoided by Effective Use of Reporting Guidelines (Clin Epidemiol. 2020;12:1403-1420) evaluated two retracted articles on the effectiveness and risk of hydroxychloroquine in COVID-19 patients, and demonstrated that transparent and complete reporting would have provided peer-reviewers and editors with sufficient information to question the methods used and the validity of the results.
The Good ReseArch for Comparative Effectiveness (GRACE) guidance includes recommendations on reporting comparative effectiveness studies. The STARD guidelines (BMJ Open 2016;14;6(11):e012799) focus on reporting diagnostic accuracy studies.
The Meta-analysis of Observational Studies in Epidemiology (MOOSE) group (JAMA. 2000;283(15):2008-15) has developed a consensus statement and recommendations for reporting meta-analyses of observational studies. It is equivalent to the STROBE Statement and the Consolidated Standards of Reporting Trials Consolidated Standards for Reporting Trials (CONSORT) 2010 Statement for RCTs, in focusing primarily on communication and list the minimum requirements for adequate reporting. The authors recommend a broad inclusion of studies and conduct of post-hoc sensitivity testing on the dependence of the results on factors such as quality of underlying papers, design, accounting for confounders, etc. The authors comment on the particular problems in merging observational studies with highly variable sets of confounders that were or were not controlled for, but they do not suggest any solution or give any references to possible ways to address it. As pragmatic trials increase in our field, another CONSORT extension focused on this type of studies, Improving the reporting of pragmatic trials: an extension of the CONSORT Statement (BMJ. 2008;337:a2390) might be also relevant.
The Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement (BMJ. 2009;339:b2535) is an evidence-based minimum set of items for reporting in systematic reviews and meta-analyses. While focused on randomised trials, PRISMA can also be used as a basis for reporting systematic reviews of other types of research, particularly evaluations of interventions. PRISMA may also be useful for critical appraisal of published systematic reviews, although it is not designed as a quality assessment instrument.
Module VI of the Guideline on good pharmacovigilance practices (GVP) addresses the legal requirements which are applicable regards submission of individual reports of suspected adverse reactions associated with medicinal products authorised in the European Union. The Guidelines for Submitting Adverse Event Reports for Publication (Pharmacoepidemiol Drug Saf. 2007;16(5): 581–7) also list key elements that have to be included when publishing a report of one or more adverse events. These guidelines have been endorsed by the International Society for Pharmacoepidemiology (ISPE) and the International Society of Pharmacovigilance (ISoP) and are available on their websites.
A preprints is an author’s research manuscript prior to formal peer review which is deposited on a public server. Preprint publication can speed the sharing of work with important and immediate public health implications, and stimulate pre-publication comment from a wide research audience. Guidance on preprints can be found at journal and institution sites, including, for example, The Lancet and Nature.