Carla J Jonker1, Marcel S G Kwa2, H Marijke van den Berg3, Arno W Hoes4, Peter G M Mol5. 1. Dutch Medicines Evaluation Board (CBG-MEB), Utrecht, the Netherlands; Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, the Netherlands. Electronic address: c.jonker@cbg-meb.nl. 2. Dutch Medicines Evaluation Board (CBG-MEB), Utrecht, the Netherlands. 3. PedNet Hemophilia Research Foundation, Baarn, the Netherlands. 4. Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, the Netherlands. 5. Dutch Medicines Evaluation Board (CBG-MEB), Utrecht, the Netherlands; Department of Clinical Pharmacy and Pharmacology, University Medical Center Groningen, Groningen, the Netherlands.
Abstract
PURPOSE: As part of the approval process, regulatory authorities often require postauthorization studies that involve patient registries; it is unknown, however, whether such registry studies are adequately completed. We investigated whether registry studies for new drugs were performed as agreed at time of approval. METHODS: This study reviewed protocols and follow-up reports for 73 registry studies that were proposed for 43 drugs approved by the Committee for Medicinal Products for Human Use in Europe in the period 2007 to 2010. RESULTS: The data lock point of January 1, 2016, was taken to allow a 5-year follow-up period for each drug after approval. At that time, 2 studies (3%) in registries had been finalized, 19 registries (26%) had not enrolled any patients, and 52 studies (71%) were ongoing. The median enrollment was 31% (interquartile range [IQR], 6-104) of the required number of patients for 41 registry studies that had a predefined sample size, 30% (IQR, 2-101) for nonimposed registries, and 61% (IQR, 18-144) for imposed registries. IMPLICATIONS: Enrollment of patients into postapproval registries is poor, although the results for imposed registries seem better. Currently, registries only have a limited impact on resolving gaps in the knowledge of a drug's benefits and risks at time of marketing authorization.
PURPOSE: As part of the approval process, regulatory authorities often require postauthorization studies that involve patient registries; it is unknown, however, whether such registry studies are adequately completed. We investigated whether registry studies for new drugs were performed as agreed at time of approval. METHODS: This study reviewed protocols and follow-up reports for 73 registry studies that were proposed for 43 drugs approved by the Committee for Medicinal Products for Human Use in Europe in the period 2007 to 2010. RESULTS: The data lock point of January 1, 2016, was taken to allow a 5-year follow-up period for each drug after approval. At that time, 2 studies (3%) in registries had been finalized, 19 registries (26%) had not enrolled any patients, and 52 studies (71%) were ongoing. The median enrollment was 31% (interquartile range [IQR], 6-104) of the required number of patients for 41 registry studies that had a predefined sample size, 30% (IQR, 2-101) for nonimposed registries, and 61% (IQR, 18-144) for imposed registries. IMPLICATIONS: Enrollment of patients into postapproval registries is poor, although the results for imposed registries seem better. Currently, registries only have a limited impact on resolving gaps in the knowledge of a drug's benefits and risks at time of marketing authorization.
Authors: Monica Mazzucato; Cinzia Minichiello; Andrea Vianello; Laura Visonà Dalla Pozza; Ema Toto; Paola Facchin Journal: Front Pharmacol Date: 2022-09-30 Impact factor: 5.988
Authors: Carla J Jonker; Sieta T de Vries; H Marijke van den Berg; Patricia McGettigan; Arno W Hoes; Peter G M Mol Journal: Drug Saf Date: 2021-06-06 Impact factor: 5.606