Lessons learned on the design and the conduct of Post-Authorization Safety Studies: review of 3 years of PRAC oversight.
Pierre Engel,Mariana Ferreira Almas,Marieke L. de Bruin,Marieke L. de Bruin,Kathryn Starzyk,Stella Blackburn,Nancy A Dreyer +6 more
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TLDR
The results show that both EMA and PASS sponsors could respectively increase the availability of protocol assessments and documents in the EU‐PAS and raise awareness among PASS stakeholders to design more thoughtful studies according to pharmacoepidemiological principles and existing guidelines.Abstract:
Aims
To describe and characterize the first cohort of Post-Authorization Safety Study (PASS) protocols reviewed under the recent European pharmacovigilance legislation.
Methods
A systematic approach was used to compile all publicly available information on PASS protocols and assessments submitted from July 2012 to July 2015 from Pharmacovigilance Risk Assessment Committee (PRAC) minutes, European Medicines Agency (EMA) and European Network of Pharmacovigilance and Pharmacoepidemiology (ENCePP) webpages.
Results
During the study period, 189 different PASS protocols were submitted to the PRAC, half of which were entered in the ENCePP electronic register of post-authorization studies (EU-PAS) by July 2015. Those protocols were assessed during 353 PRAC reviews. The EMA published only 31% of the PRAC feedback, of which the main concerns were study design (37%) and feasibility (30%). Among the 189 PASS, slightly more involved primary data capture (58%). PASS assessing drug utilization mainly leveraged secondary data sources (58%). The majority of the PASS did not include a comparator (65%) and 35% of PASS also evaluated clinical effectiveness endpoints.
Conclusions
To the best of our knowledge this is the first comprehensive review of three years of PASS protocols submitted under the new pharmacovigilance legislation. Our results show that both EMA and PASS sponsors could respectively increase the availability of protocol assessments and documents in the EU-PAS. Protocol content review and the high number of PRAC comments related to methodological issues and feasibility concerns should raise awareness among PASS stakeholders to design more thoughtful studies according to pharmacoepidemiological principles and existing guidelines.read more
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References
More filters
Journal ArticleDOI
Guidelines for good database selection and use in pharmacoepidemiology research.
Gillian C. Hall,Brian C. Sauer,Alison Bourke,Jeffrey S. Brown,Matthew W. Reynolds,Robert Lo Casale +5 more
TL;DR: This guidance is presented as a detailed text with a checklist for quick reference and covers six areas: selection of a database, use of multiple data resources, extraction and analysis of the study population, privacy and security, quality and validation procedures and documentation.
Journal ArticleDOI
European Medicines Agency review of post-authorisation studies with implications for the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance.
Kevin Blake,Stefanie Prilla,Sophie Accadebled,Marie Guimier,Monica Biscaro,Ingemar Persson,Ingemar Persson,Peter Arlett,Stella Blackburn,Henry Fitt +9 more
TL;DR: A review of post‐authorisation studies requested in 2007 by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency was undertaken to determine compliance and the need for research capacity in the European Union.
Journal ArticleDOI
Evaluation of post-authorization safety studies in the first cohort of EU Risk Management Plans at time of regulatory approval.
Thijs J. Giezen,Aukje K. Mantel-Teeuwisse,Sabine M. J. M. Straus,Toine C. G. Egberts,Stella Blackburn,Ingemar Persson,Ingemar Persson,Ingemar Persson,Hubert G. M. Leufkens,Hubert G. M. Leufkens +9 more
TL;DR: The need for more complete study proposals to be submitted earlier on in the evaluation period and for the inclusion of EU inhabitants in PASS is emphasized, as well as the need for individualized tailored PASS depending on the type of drug.
Journal ArticleDOI
Data Extraction and Management in Networks of Observational Health Care Databases for Scientific Research: A Comparison of EU-ADR, OMOP, Mini-Sentinel and MATRICE Strategies
Rosa Gini,Martijn J. Schuemie,Jeffrey S. Brown,Patrick B. Ryan,Edoardo Vacchi,Massimo Coppola,Walter Cazzola,Preciosa M. Coloma,Roberto Berni,Gayo Diallo,José Luís Oliveira,Paul Avillach,Gianluca Trifirò,Peter R. Rijnbeek,Mariadonata Bellentani,Johan van der Lei,Niek S. Klazinga,Miriam C. J. M. Sturkenboom +17 more
TL;DR: A conceptual framework was created distinguishing three steps in local data processing: data reorganization into a data structure common across the network; derivation of study variables not present in original data; application of study design to transform longitudinal data into aggregated data sets for statistical analysis.
Journal ArticleDOI
Increasing scientific standards, independence and transparency in post-authorisation studies: the role of the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance.
TL;DR: The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP), an initiative coordinated by the European Medicines Agency, aims to build capacity for and increase trust in post‐authorisation studies to further support medicine decision making.
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