The European Medicines Agency (EMA) has issued a new guideline on registry-based studies, giving detailed advice for use of patient registries in both the pre- and post-authorization phases for medicinal products.
After committee approval and a consultation period with the EU Regulatory Network, the guideline has been released for public consultation, with comments due before the end of 2020.
The guideline’s focus is on the use of patient registries for studies by marketing authorisation applicants and holders (MAAs/MAHs). The guideline’s scope includes disease and condition registries, such as those for patients with a particular disease or disease characteristic, a condition such as pregnancy, or molecular or genomic features.
Registries are particularly important, notes the guideline, for patients with rare diseases who may receive advanced therapy medicinal products (ATMPs) such as gene therapy…