MedicalResearch.com Interview with: Dr. Agnes Dechartres MD, PhD Centre de Recherche Epidémiologie et Statistique Paris, France MedicalResearch.com: What is the background for this study? What are the main findings? Dr. Dechartres: In general, the availability of trial results is not optimal, with about half of the results from clinical trials, especially those with negative results, not being published in journals. To improve the availability of results, the FDA Amendment Act (FDAAA) requires results to be posted at ClinicalTrials.gov for all trials evaluating FDA-approved drugs, devices, or biologics within one year of trial completion. However, some previous methodological studies showed poor compliance with this law^1-3. Rare diseases are defined by a prevalence of fewer than 200,000 people, but overall, they may affect up to 25 million people in North America. The limited number of patients affected by each rare disease makes this research particularly challenging, and the access to information on all trials conducted and their results is even more necessary. In this study, we aimed to describe the characteristics of completed phase 3 or 4 trials of rare diseases registered at ClinicalTrials.gov and assess whether their results were publicly available. We found a substantial proportion of single-group trials and trials not randomized. Many trials were also conducted in a single-center. Concerning the availability of results, 36 months after trial completion, results were posted at ClinicalTrials.gov for 35% of trials, published in journals for 47%, and publicly available (ie, posted at ClinicalTrials.gov or published in journals) for 63%. Among the trials likely subject to the FDAAA, 68% had results posted at ClinicalTrials.gov: 4% had results posted at 12 months and 57% at 36 months. We also found that 21% of studies not concerned by the FDAAA had results posted. (more…)