European Medicines Agency issues qualification opinion that Enroll-HD can provide data and infrastructure support for post-authorization monitoring of medical products for Huntington’s disease
New York, NY, September 21, 2022; The European Medicines Agency (EMA) has issued a qualification opinion that details facets of the Enroll-HD registry, core observational study, and clinical research platform that are especially suited to post-authorization regulatory approval. Scientific advice on Enroll-HD was sought in March 2021 under a recently launched EMA initiative to facilitate the use of data from well-developed and comprehensive registries in post-authorization medical product monitoring. Importantly, the EMA opinion states that “Enroll-HD is well-suited” and “can be a source of secondary data for drug utilization studies and post-authorisation safety and efficacy studies (PASS and PAES)”; these latter studies “can also be nested in Enroll-HD” to provide primary data.
Enroll-HD was launched in 2012 by CHDI Foundation, Inc. to expedite Huntington’s disease research and therapeutic development and has recruited more than 28,000 participants (over 21,000 are currently actively engaged) at over 150 clinical sites in 23 countries around the world. Participants undergo standardized core assessments and biosample collection at their annual visits, with extended or optional assessments conducted at the discretion of the principal investigator or participant, respectively.
“This advice from the EMA is really good news for drug development for Huntington’s disease since it will expedite the collection of post-authorization data,” said Cristina Sampaio, MD, PhD, Chief Clinical Officer at CHDI. “This adds substantially to what the whole community is working towards – a rigorous, streamlined drug approval pathway for Huntington’s disease.”
The EMA advice noted favorable aspects of Enroll-HD, including its comprehensive participant representation and data collection across the disease spectrum, high participant retention, the platform’s flexibility to adapt to regulatory requirements, systematic data quality control that ensures integrity and reliability, and a broad mechanism for sharing data.
Read the EMA qualification opinion in full.
For further information please contact Tim McLean (Tim.McLean@Enroll-HD.org) or Jenny Townhill (Jenny.Townhill@Enroll-HD.org).