Project Scope 

This project aims to create a guidance about best data practices for patient foundations and for researchers in the form of decision trees, graphics, short printable materials or videos.

Project Statement 

The purpose of this Working Group is to create awareness and education about best practices for collecting regulatory quality data from the industry perspective, improving data quality, empowering patients and patient groups with knowledge and information, and building bridges between industry, academics and patient communities.

Project Impact 

This project will create greater partnerships between industry, researchers and the patient community. It will also help improve data collection, with the goal of the data being useful to the industry.

Project LeadsEmail

Sophia Zilber, Alexion AstraZeneca Rare Disease, Cure Mito Foundation

Danielle Boyce, Johns Hopkins University
Paul Howard, Amicus Therapeutics

Nicola Newton, PHUSE Project Assistant 


  • Drafted a lay language summary of recent FDA guidance

Objectives & Deliverables


Decide on a strategy to share all three resources, possibly through PHUSE Education webinars Q2 2023
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