World Orphan Drug Congress USA 2026

Date: June 9th-11th, 2026

Location: Boston, USA

PharSafer® is pleased to confirm our participation as a Gold Sponsor at the World Orphan Drug Congress USA 2026, one of the leading global events focused on orphan drugs, rare disease development, regulatory strategy, and patient-centred innovation.

Bringing together pharmaceutical companies, biotech organisations, regulators, patient advocacy groups, and industry experts from across the rare disease landscape, WODC USA provides an important platform for discussing the evolving challenges surrounding orphan-drug development and long-term patient safety. ​

Exclusive Speaker Session

Join Dr. Graeme Ladds, CEO of PharSafer®, for an insightful speaker session within the Clinical Development & Regulatory track:

“What Makes Orphan Drug Pharmacovigilance Different? Real-World Challenges and Practical Solutions”

Pharmacovigilance for orphan and ultra-orphan therapies carries the same regulatory expectations as any other product – if not more – yet the path to achieving compliant, sustainable safety oversight is often significantly more challenging.

In this session, Dr. Ladds will explore the practical realities of pharmacovigilance in rare disease settings, including how limited clinical exposure, fragmented real-world evidence, dispersed patient populations, and complex disease backgrounds can make safety interpretation disproportionately difficult.

Key discussion areas will include:

• Why traditional pharmacovigilance approaches often struggle in orphan-drug settings
• The challenges created by limited datasets, translation barriers, and complex disease profiles
• Practical, proportionate PV approaches that support compliance without overwhelming smaller teams
• Building credible, defensible safety oversight where every individual case can significantly influence patient understanding and programme confidence
• The wider role of pharmacovigilance in supporting regulatory confidence, programme sustainability, and long-term development success

Ideal for: Pharmacovigilance leaders, regulatory professionals, clinical development teams, biotech innovators, medical affairs professionals, and organisations involved in orphan and ultra-orphan drug development.

Attendees will leave with a deeper understanding of the operational and strategic challenges unique to orphan-drug pharmacovigilance, alongside practical insight into designing sustainable safety frameworks for low-data environments.

​Connect with PharSafer® in Boston

Our senior team will be available throughout the event and would welcome the opportunity to discuss your organisation’s challenges and explore tailored support across:

• Clinical & Post-Marketing Pharmacovigilance
• Rare Disease & Orphan Drug Safety Strategy
• Global Regulatory Strategy & Risk Management Planning
• Auditing, Training & Inspection Readiness
• Medical Affairs & Safety Communication
• Safety Automation & Workflow Innovation with RAPTAR®​

Schedule a Meeting

To ensure dedicated time with our team during the event, we encourage you to schedule your meeting with us in advance.

We look forward to connecting with industry colleagues and partners in Boston for what promises to be another valuable and insightful event.

Note: For more information about the event, please visit: World Orphan Drug Congress 2026.