[co-author: Alexander Wenzel]

Introduction

On 5 November 2018, the EMA published a discussion paper concerning the use of patient disease registries for regulator purposes. The discussion paper was published within the context of the EMA’s Patient Registries Initiative (PRI). The consultation period is open to stakeholders until 29 June 2019. It has been published to allow stakeholders to participate in the discussion by commenting on the paper.

In 2015, the EMA established the PRI and the Cross-Committee Task Force (CCTF) on registries to explore ways of expanding the use of patient disease registries.[1] The EMA wishes to support a more systematic and standardised approach so patient disease registries can become useful to the benefit-risk evaluation of medicinal products.

The initiative aims to provide long term access to high quality data for research purposes through patient disease registries. Patient disease registries include a large amount of clinical information that has not been extensively used by national competent authorities. The EMA would like competent authorities to use the patient registries for the benefit-risk evaluation of medicinal products.

The main objective of this initiative is to facilitate use of patient registries to support regulatory decision-making. The paper discusses methodological and operational aspects of the use of patient disease registries and registry studies for regulatory purposes, including the methodological aspects of studies performed with registry data to increase regulators’ confidence in the results of the studies.

The paper

The paper explains that regulators generally prefer disease registries to product registries. This is because patient disease registries gather insights into clinical outcomes in patients receiving different treatments. Patient disease registries may support a wider range of study designs than product registries. An example given is “controlled designs without an external data source”.

As the paper makes comparisons with other types of registries, the considerations also concern product registries and registries of patients defined by a specific condition.

The paper highlights the differences between a registry study and a registry. It also provides considerations regarding best registry practices for registries as well as considerations for registry studies. The paper provides that a registry is a routine, long-term, data collection system. A registry study, is a time-limited investigation of a specific research question. The paper explains that as such, a different methodological approach is needed for patient disease registries and registry studies.

Regarding registry studies the paper provides considerations regarding the choice of the patient population, the recording of time data, the keeping track of core data elements collected and a list with recommended international terminology. The paper also suggests measures and data indicators to improve data quality, considerations regarding safety analyses and recommendations regarding the collaboration between registries, Market Authorisation Holders (MAH) and competent national authorities.

Regarding registry studies, the paper provides considerations regarding regulatory legal compliance obligations such as post-authorisation safety studies, the design of study protocols and timelines, the choice of study populations, limitations to data collection, data quality control, data analysis, safety reporting and reporting of study results.

The EMA further encourages early dialogue between the MAH and the competent national authority. The EMA is also willing to provide tools to facilitate the recognition of disease registries as data sources, to conduct studies for regulatory purposes. The Scientific Advice procedure on study protocols and the Qualification procedures of registries are available to pharmaceutical companies and registry coordinators to provide advice and opinions on the validity of registries and study protocols.

[1]               The paper uses the following definition of a patient registry: “A patient registry is defined as an organised system that uses observational methods to collect uniform data on a patient population defined by a particular disease, exposure or condition (e.g. age, pregnancy, specific patient characteristics), and which is followed over time.”

[View source.]