A registry-based study is an investigation of a research question using the infrastructure of (a) new or (an) existing registry(ies) for patient recruitment and data collection. A registry-based study may be a clinical trial, to which the provisions of Directive 2001/20/EC or of Regulation (EU) No 536/2014 (when it becomes applicable) apply, or a non-interventional study if it fulfills the corresponding requirements specified in Directive 2001/20/EC (see Annex of Questions & Answers document, Version 11.0, May 2013) or Regulation (EU) No 536/2014 1 (1). A registry-based study may apply primary data collection and/or secondary use of data collected in a patient registry for another purpose than the given study (see definitions in Appendix 1). A patient registry is defined in this Guideline as an organized system that collects data and information on a group of people defined by a particular disease or condition, and that serves a pre-determined scientific, clinical and/or public health (policy) purpose. The use of the term ‘patient’ in combination with ‘registry’ the dataset on health information. The terms ‘people’ and ‘patients’ used in this definition and Guideline are synonyms, independently of the health status of the individual.
The objective of this Guideline is to provide recommendations on key methodological aspects that are specific to the use of patient registries by marketing authorization applicants and holders (MAAs/MAHs) planning to conduct studies. To support these recommendations, aspects of patient registries that regulators consider important for their use in registry-based studies are included in the Annex. Relevant legal basis and regulatory requirements that apply to these studies are listed in Chapter 4.
Read the pdf