Decentralised pregnancy registry study

Generating pregnancy safety evidence for rare disease therapies presents unique challenges, including extremely low exposure rates and geographically dispersed patient populations. To address these challenges, ICON designed and implemented a fully decentralised pregnancy registry that supported post-approval evidence requirements while maximizing global reach, patient access, and long-term study sustainability.

The sponsor needed to satisfy a post‑approval pregnancy safety commitment across multiple countries despite a small number of exposed pregnancies Given the low number of exposed cases, traditional enrolment models were not operationally efficient and risked limiting patient identification and data collection opportunities. A scalable, patient-centric solution was required to facilitate global case capture while maintaining robust scientific and regulatory oversight.

ICON implemented a fully decentralised pregnancy registry, serving as the central scientific and operational hub. A dedicated country-level structure was established, with oversight provided either directly by ICON or through local Key Opinion Leaders acting as Principal Investigators.

The registry leveraged targeted patient and healthcare professional outreach to support enrolment and data collection, integrating information from both patients and treating physicians. Independent teratology experts conducted pregnancy outcome adjudication to ensure consistent and scientifically rigorous assessment across participating countries.

To maximise enrolment and long-term retention in a low-exposure population, ICON implemented optimized recruitment and retention strategies through a flexible, multi-pronged approach, including engagement with Patient Advocacy Groups (PAGs), targeted outreach to medical communities and professional societies, tailored stakeholder communications, and robust awareness campaigns directed at potentially exposed women and their healthcare providers. Digital and social media channels were utilized to expand reach and enhance engagement.

The model was supported by ICON's innovation and technology-enabled solutions, facilitating efficient patient identification, engagement, and long-term follow-up while minimizing participant burden.

Designed with scalability in mind, the registry could be seamlessly expanded into additional markets as the product launched in new geographies, without requiring significant changes to study infrastructure, governance, or operational processes.

The decentralised registry enabled compliant, long-term pregnancy safety monitoring across multiple geographies while efficiently capturing rare exposures. The approach reduced burden on patients and healthcare providers, supported both prospective and retrospective case capture, and ensured continuity of scientific oversight in a low-exposure rare disease setting.