When Decisions Can't Wait: Delivering Rapid Real World Studies
At the Annual Meeting of the international Society for Pharmacoepidemiology our reseachers participated in a symposium organised by the European Medicines Agency on the topic of rapid real world studies for regulatory purposes. It was great to be able to demonstrate the power of the OMOP Common Data Model and standardised analytics for the Ranitidine use case led by Erasmus MC that included the IPCI data.
Regulatory decisions on medicinal products often require valid and generalisable real-world evidence on their utilisation and risks. In some conditions, data are needed wihin a few weeks or months, such as for identification of the population at risk of serious adverse effects or targeted for risk minimisation, rapid surveillance and prevention of potential safety concerns, or management of public health emergencies (e.g. pandemic influenza). In suchcases, multiple challenges need to be met with regard to study design, data access, collection and analysis and result integration, and some of these steps must be accelerated to support timely decisions without affecting internal and external validity. Given the increasing public expectation for fast regulatory decisions, solutions may need to be further developed and introduced in routine practice.
This symposium presented regulatory circumstances in the EU and the US where rapid real-world studies were needed and described the challenges to be addressed and some available solutions. It will contribute to define the infrastructure, research environment, methodological approaches and the analytical system necessary for rapid studies, and benefit regulators, researchers and industry who need to quickly answer regulatory questions.
Xavier Kurz (Data Analytics and Methods Task Force, EMA) introduced the topic and moderated the panel.
Sabine Straus (MEB, Chair of the EU Pharmacovigilance Risk Assessment Committee) presented examples of questions from the PRAC Pilot on rapid data analytics and how they were addressed.
Michel Kwa (Senior assessor, MEB) explained the need for timely data in the situation of the EU Referral procedure on H2 receptor antagonists assessing the potential risk associated with N-nitrosodimethylamine (a human carcinogen).
Peter Rijnbeek and Katia Verhamme (Department of Medical Informatics, Erasmus University Medical Center) presented the challenges met and solutions found for an EMA-funded multi-centre study from 5 EU countries on this topic which was completed within 4 months, and will discuss the opportunities of this approach to answer other types of regulatory questions.
Simone Pinheiro (Office of Surveillance and Epidemiology, FDA) and Judith Maro(Harvard Pilgrim Health Care Institute) discussed how the Sentinel system is used in situations where rapid analyses are needed, including sequential surveillance, cohort definition and acceleration of time segments for different study phases.
The session was ended with a panel discussion on the types of questions amenable (or not) to rapid studies and the research environment supporting them.