Barriers and Opportunities for Use of Patient Registries in Medicines Regulation

What Does It Take to Transform Real‐World Data Into Real‐World Evidence?

“…). Additionally, as data from patient registries evolve to generate high‐quality data to support regulatory decision making, Olmo et al . describe some of the barriers and opportunities identified from the EMA's Patient Registry Initiative that may enable a more systematic approach for utilizing registry‐based RWD to generate RWE.…”

From Laboratory to Patient: The Evolving Path to Become a Medicine

“…The level of uncertainty at the time of regulator approval will have consequences in the type of authorization achieved and determine the regulatory life along the life-cycle of the product. Post-authorization studies like registries help address that need for further information 15 .…”

“…It is never too early to start consideration and preparation for what happens after the therapy is on the market. Elements that require early consideration include identifi cation of potentially suitable existing registries, feasibility analysis and establishment of governance and data ownership 15 . For ATMPs, failure to do so in the early stages could at a minimum result in longer and more diffi cult approval process.…”

Inhibitor development in previously untreated patients with severe haemophilia: A comparison of included patients and outcomes between a clinical study and a registry‐based study

“…Within the regulatory field, the Patient Registry Initiative of the European Medicine Agency is exploring the use of patient registries. This initiative supports a systematic approach for a better use of registry data for the benefit‐risk evaluation of medicines, mostly post‐marketing 4,5 . To retrieve high‐quality registry data, key aspects are a comprehensive enrolment of patients, avoidance of selection bias, collection of essential core data and completeness of data 5 …”