Resource Library

Rare Disease Data & Document Anonymization

"ULTRAGENYX is committed to sharing information about our research to help advance future development in rare diseases. Performing robust, accurate and reliable Anonymization while maintaining data utility in rare patient populations is challenging. Our objective is to protect participant privacy while adhering to our internal standards and those of global regulatory bodies. Real Life Sciences guided us through every step of the process with comprehensive technology driven approaches to quantitative risk assessments for rare and ultra-rare populations."

-Director, Clinical Transparency
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ULTRAGENYX is a biopharmaceutical company committed to bringing novel products to patients for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases.

Avoiding delays when seeking regulatory approvals for rare disease populations

The challenges facing ULTRAGENYX required the Real Life Sciences and the ULTRAGENYX teams to work through complex issues together.  Working with rare disease populations requires compliance strategies be thought through carefully in advance.

Regulators prefer anonymization methods that maintain data utility over those that rely on redacting large portions of pertinent information. Working together, ULTRAGENYX and Real Life Sciences performed anonymization and quantifiable risk modelling ahead of the first meeting with regulators.  This approach has historically proven to enable alignment of expectations between Sponsor and Regulator and has avoided downstream rework and time-consuming iterations (a concern expressed repeatedly across the industry).

An efficient cross-functional process to simplify submission complexity when working with rare disease population data

To achieve sustained success, cross-functional teams need to be aligned when solving transparency and anonymization challenges.  This is particularly true in situations involving advanced data anonymization complexity, such as rare disease populations. By leveraging the quantifiable risk modeling capabilities offered by Real Life Sciences and implementing an internal cross-functional decision-making process spanning Transparency, Regulatory, Biostats, Clinical Development, Compliance/Legal and Program Management, ULTRAGENYX was able to proactively address the concerns associated with disclosure of clinical trial results.

Real Life Sciences generated a white paper for ULTRAGENYX that defined best practices including roles and responsibilities needed in transforming small population data on a trial-by-trial basis. ULTRAGENYX leveraged these best practices to streamline processes and align internal teams. 

One critical success criteria surrounding data anonymization for rare-disease trial populations at ULTRAGENYX stemmed from the ability to share a detailed plan and strategy for review by the regulators at the outset. Proactive planning allows for a more streamlined process that helps avoid rework and leads to a more efficient disclosure approval cycle.

Transparency And Data Anonymization For Rare Disease Populations

ULTRAGENYX cares deeply about data transparency in its commitment to advance life-changing treatments .  Regulatory bodies and pharmaceutical organizations are seeing an increase in data sharing on a global basis -- and Rare Disease populations are not exempt. New regulations such as Clinical Trial Regulation (Regulation (EU) No 536/2014) will continue to make it even more critical  for sponsors, like ULTRAGENYX, to maintain a reliable method of data transformation, and a business process that is repeatable, efficient and applicable across trials and adheres to the needs of multiple governing bodies.

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