An analysis of characteristics of post-authorisation studies registered on the ENCePP EU PAS Register

Robert Carroll, Sreeram V. Ramagopalan, Javier Cid-Ruzafa, Dimitra Lambrelli, Laura McDonald
2017 F1000Research  
The objective of this study was to investigate the study design characteristics of Post-Authorisation Studies (PAS) requested by the European Medicines Agency which were recorded on the European Union (EU) PAS Register held by the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). Methods: We undertook a cross-sectional descriptive analysis of all studies registered on the EU PAS Register as of 18 October 2016. Results: We identified a total of 314 studies on
more » ... e EU PAS Register, including 81 (26%) finalised, 160 (51%) ongoing and 73 (23%) planned. Of those studies identified, 205 (65%) included risk assessment in their scope, 133 (42%) included drug utilisation and 94 (30%) included effectiveness evaluation. Just over half of the studies (175; 56%) used primary data capture, 135 (43%) used secondary data and 4 (1%) used a hybrid design combining both approaches. Risk assessment and effectiveness studies were more likely to use primary data capture (60% and 85% respectively as compared to 39% and 14% respectively for secondary). The converse was true for drug utilisation studies where 59% were secondary vs. 39% for primary. For type 2 diabetes mellitus, database studies were more commonly used (80% vs 3% chart review, 3% hybrid and 13% primary data capture study designs) whereas for studies in oncology, primary data capture were more likely to be used (85% vs 4% chart review, and 11% database study designs). Conclusions: Results of this analysis show that study objectives and therapeutic area influence PAS design in terms of type of data capture used. PubMed Abstract | Publisher Full Text 2. Maasland L, Van Oostenbrugge RJ, Franke CF, et al.: Patients enrolled in large randomized clinical trials of antiplatelet treatment for prevention after transient ischemic attack or ischemic stroke are not representative of patients in clinical practice: the Netherlands Stroke Survey. Stroke. 2009; 40(8): 2662-2668. PubMed Abstract | Publisher Full Text 3. McDonald L, Lambrelli D, Wasiak R, et al.: Real-world data in the United Kingdom: opportunities and challenges. BMC Med. 2016; 14(1): 97. PubMed Abstract | Publisher Full Text | Free Full Text 4. Gale EA: Post-marketing studies of new insulins: sales or science? BMJ. 2012; 344: e3974. PubMed Abstract | Publisher Full Text 5. Borg JJ, Aislaitner G, Pirozynski M, et al.: Strengthening and rationalizing pharmacovigilance in the EU: where is Europe heading to? A review of the new EU legislation on pharmacovigilance. Drug Saf. 2011; 34(3): 187-197. PubMed Abstract | Publisher Full Text 6. Blake KV, Prilla S, Accadebled S, et al.: European Medicines Agency review of post-authorisation studies with implications for the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance. Pharmacoepidemiol Drug Saf. 2011; 20(10): 1021-9. PubMed Abstract | Publisher Full Text 7. Engel P, Almas MF, De Bruin ML, et al.: Lessons learned on the design and the conduct of Post-Authorization Safety Studies: review of 3 years of PRAC oversight. Br J Clin Pharmacol. 2017; 83(4): 884-893. PubMed Abstract | Publisher Full Text | Free Full Text 8. Hoekman J, Klamer TT, Mantel-Teeuwisse AK, et al.: Characteristics and follow-up of postmarketing studies of conditionally authorized medicines in the EU. Br J Clin Pharmacol. 2016; 82(1): 213-26. PubMed Abstract | Publisher Full Text | Free Full Text 9. The article is well written and provides a descriptive analysis of the EU PAS Register. This type of research is welcome as an evaluation of the usefulness of the EU PAS Register. The article would benefit from a couple of clarifications:
doi:10.12688/f1000research.12198.1 pmid:29188016 pmcid:PMC5698914 fatcat:zo2g62hi2jd2bb3mnsikwauobq