World Orphan Drug Congress 2026: Advancing Rare Disease Therapies and Patient Access

The World Orphan Drug Congress convenes on 9–11 June at the Thomas M. Menino Convention & Exhibition Center in Boston. The event aims to advance therapeutic development, regulatory pathways, and patient access for rare diseases.

Central themes on 11 June focus on translating scientific innovation into measurable patient benefit through coordinated efforts across advocacy, commercialization, and regulatory science.

Organizers have structured the day to link early-stage discovery with post-approval access, emphasizing evidence generation and cross-stakeholder alignment.

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Morning Leadership Sets Strategic Tone

Morning keynotes establish the day’s emphasis on leadership and value demonstration.

A conversation among next-generation CEOs explores patient-centered strategies for therapy development.

This is followed by a Johnson & Johnson-hosted panel on global access and a presentation on disease modeling that connects limited datasets to launch evidence.

Subsequent parallel tracks examine these key areas:

• Patient access services
• Insurance coverage patterns
• Regulatory acceleration
• Ethical AI applications
• Expanded-access program transitions
• Newborn screening policies

Afternoon sessions continue with start-up pitches, registry-data publication guidance, and panels on Medicaid access barriers and HTA-driven pricing differences between the United States and Europe.

The sequence moves from strategic framing to operational tactics, ending with networking that reinforces practical collaboration.

Voices Shaping the World Orphan Drug Congress

Key speakers on June 11 include:

• Emil Kakkis of Ultragenyx Pharmaceutical
• Charlene Son Rigby of Global Genes
• Yael Weiss of Mahzi Therapeutics
• Alex Hersham of ReadOn Tx
• Nathan Walters of Johnson & Johnson
• Daria Julkowska
• Jayson Slotnik
• Ron Bartek of the Friedreich’s Ataxia Research Alliance
• Sandra Talbird of RTI Health Solutions
• Sophia Zilber
• Pamela Gavin

These leaders address therapeutic advancement, CEO-level patient-centered strategies, market-access policy, advocacy, disease-modeling methods, and co-creation of educational resources.

Representatives from FDA, CMS, HTA bodies, patient organizations, and emerging companies provide diverse perspectives spanning regulatory science, payer engagement, and early-stage innovation.

Pathways to Faster Patient Access

Discussions on disease modeling, coverage variation, and ethical AI use suggest that integrated evidence strategies may reduce uncertainty for payers and regulators.

Sessions addressing Medicaid barriers, HTA comparisons, and expanded-access transitions indicate that earlier alignment among manufacturers, patient groups, and public programs could shorten the time between approval and reimbursement.

The presence of start-up showcases and investor panels highlights continued capital interest in platforms for ultra-rare conditions when development and commercialization models show both clinical and economic viability.

Overall, the agenda positions collaborative data practices and patient-centric trial design as key mechanisms to strengthen the rare-disease ecosystem.