Project Scope

Within this project we will make a list of available data sources and pros and cons. We will discuss what documents and requirements we need to show the FDA in the pre-alignment meeting and we will try to point to or create an outline and/or requirements for those documents. If possible, we will align with the FDA on this.

Problem Statement

Real-world data sources have been used for many years in the pharmaceutical industry. They are useful for validating research questions and protocol set-up, patient and centre selection, post-registry safety follow-up and market access. However, the use for regulatory submissions is new. For this, the requirements are more stringent and we need a robust process to assess quality, accuracy, appropriateness of the data and compliance to regulatory requirements. How are we going to set ourselves up for success?

We shall focus on:

selecting the appropriate data source
ensuring the data sources are accurate and traceable
how we assess the quality of the data

These are all questions that we will start to answer in this project.

Problem Impact

A number of (real-world evidence parts of) submissions have been rejected because of a lack of power or representativeness. The FDA indicates that aligning with them before the start of a study is key to assessing whether the approach and source we are using is acceptable. Then, we need to have good funding for our assumptions. If we do not have this, we need to restart our design.

Project Leads	Email
Berber Snoeijer, ClinLine	b.snoeijer@clinline.eu
Ashwin Rai, Evidera	ashwin.rai@evidera.com
Nicola Newton, PHUSE Project Assistant	nicky@phuse.global

CURRENT STATUS Q1 2025
Consolidated paper available for internal review of project group.

Objectives & Deliverables	Timelines
Project kick off	Q2 2023
List of documents and requirements validating the right data source	Q4 2023
Pre alignment meeting with regulatory agencies	Q4 2023

Deliverables and Blogs
Understanding the Data Quality Issues in Real World Data through Real World Examples
Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products
RWD: The FDA’s Guidance on Assessing Registry Data to Support Regulatory Decision-Making

Objectives & Deliverables

Timelines