By Sam Fillingham | CEO
Real-world data from registries and other real-world evidence sources remain a fertile ground of opportunity for advocacy groups and biopharma companies to accelerate rare disease research and better characterize and manage these diseases, their progression and treatment.
OBJECTIVES
To answer this challenge, PIP-UK was focused on ensuring that the data were collected in a standardized way, met regulatory requirements and aligned with their vision to build global collaboration, with the technology as a nucleus for patients to convene and share their experiences.
METHODS
PIP-UK started working with Pulse Infoframe in 2021 to launch the Poland Syndrome Community Register. Utilising the Pulse Platform, PIP-UK were able to deploy the registry within 4 months with a recruitment target of 100 participants in the first 3 months. Common data elements to form the central hub for data to be characterized and curated were created. PIP-UK and Pulse addressed the regulatory requirements that would be needed for drug development by ensuring that the data are collected in a platform that adheres to FDA and EMA data standards and further provides confidence to participants that their data are stored safely due to compliance with HIPAA and GDPR.
RESULTS SO FAR
The registry currently has 280 active participants enrolled into the platform where 31 of them are under the age of 10. The majority of participants are female and right side affected. The majority of participants are diagnosed between ages 0-9 years but a significant number aged 10 and over with the latest reported diagnosis aged 60+.
CONCLUSIONS
Our community is engaging regularly with the register and updating their quality-of-life survey every 6 months. We are just getting our scientific advisory board off the ground to complete the first major analysis of the data. We have doubled the number of participants in the first year and entrants are steadily growing still. We will need major investment in research and advancements of the register to tackle the research the community needs. We continue to work on funding applications to support this but have yet to be successful.
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