Legal regulations, ethical guidelines and recent policies to increase transparency of clinical trials

Abstract

Timely and accurate dissemination of outcomes is essential to accomplish main benefits of scientific research including clinical trials. Clinical trial results can be disseminated in two main ways: by publication in a peer‐reviewed journal and by posting on a publicly available clinical trial register. The credibility of the literature on clinical trials is significantly diminished because a high percentage of trials is not published. While current legal regulations both in the European Union (EU) and the USA impose a duty to submit summary results of clinical trials to a respective register (EU Clinical Trial Register and http://ClinicalTrials.gov, respectively), the compliance with this requirement has been generally inadequate. Trial outcomes can be also made accessible by data sharing. However, in spite of the wide promotion of this idea, the access of investigators to participant‐level datasets remains limited. The main objective of this review is to discuss current legal regulations, international standards, ethical guidelines and recent policies pertaining to dissemination of clinical trial results.

Public availability of results of ClinicalTrials.gov‐registered expanded access studies

“…In the USA legal regulations have been introduced to mandate posting on CT.gov of summary results of applicable clinical trials. 8 However, this requirement does not concern expanded access studies; the Final Rule clearly states that expanded access studies are not applicable clinical trials and thus are exempt from the statutory requirements pertaining to those trials. 19 While currently there are no laws to mandate publishing of expanded access studies, this problem has been addressed by the Declaration of Helsinki (DH), the principal guidance document of international scope concerning the conduct of biomedical research.…”

“…introduced to mandate prospective registration and posting of trial results. 7,8 As a result, currently, CT.gov is a very important source of information about clinical trials. However, it also provides a possibility to register other types of studies, especially observational studies and expanded access studies.…”

From publication bias to lost in information: why we need a central public portal for clinical trial data

“…The resulting controversies accelerated data transparency initiatives propagating trial registration and the reporting of summary results. 5 However, even though mandatory implementation in several countries markedly improved reporting, deficits still exist, 6 7 which triggered calls to release more extensive clinical trial data such as clinical study reports (CSRs). These documents are traditionally used to inform regulatory decision making and were previously always confidential, but public access has increased, 8 9 and analyses of CSRs have challenged conclusions based on published evidence alone.…”

Affordable Prices Without Threatening the Oncological R&D Pipeline—An Economic Experiment on Transparency in Price Negotiations

“…The pharmaceutical market is characterized by conspicuous informational asymmetries, with pharmaceutical companies having a more precise assessment of key factors such as actual prices (3), research and development (R&D) costs (4), and clinical trial data (5,6). In the present system, the outcomes of bilateral negotiations remain confidential.…”