Background and importance Most decisions made in clinical practice are based on the results of published clinical trials (CT). A widely used tool for the evaluation of the methodological quality of publications of randomised clinical trials (RCTs) are the guidelines of the CONSORT 2010 declaration. These guides are a checklist of 25 items that allow the evaluation of the publications of RCTs from the point of view of transparency, design, abstract, flowchart of participants and analysis of the results.
Aim and objectives The main objective was to evaluate the methodological quality of all pivotal RCT publications of orphan drugs authorised during 2018 in the European Union.
Material and methods The pivotal CT publications were found in the ClinicalTrials.gov and PubMed databases. Methodological quality was examined using the guidelines of the CONSORT 2010 statement on the publication of RCTs, assigning a score of 0 or 1 to each of the sections that comprised it. They were also evaluated following the CONSORT for abstracts guidelines because many clinical decisions are made based on the conclusions from these sections.
Results Of the 21 orphan drugs authorised in 2018, 24 pivotal CT were located and 33% were not randomised. The pivotal RCTs analysed complied with only 66.13% of the items in the CONSORT guidelines, compared with 82% in high impact journals; 60% of abstracts analysed fulfilled more than 70% of the items in the CONSORT for abstracts declaration. Only 26.6% of the RCTs described the randomisation method selected. Regarding masking, only 40% of the RCTs detailed who remained blinded after performing the corresponding interventions. As for access information to the complete protocol of the RCT, only 20% declared where it can be located.
Conclusion and relevance The pivotal publications of RCTs of orphan drugs met most of the items in the CONSORT 2010 guidelines, particularly the abstracts. However, many pivotal CT were not randomised and compliance with the guidelines was not as high as that of other high impact journal publications. Therefore, more quality and transparency criteria should be required in pivotal CT publications of orphan drugs, such as randomisation, detailed masking and where to locate the complete protocol.
References and/or acknowledgements No conflict of interest.
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