The Evidence Base Post

Three themes shaping discussions at the World Orphan Drug Congress USA 2026: Market access, patient partnership and evidence generation

Joanne Walker

From evolving access and pricing pressures to new approaches to evidence generation and growing calls for meaningful patient partnership, the World Orphan Drug Congress USA 2026 highlights the issues shaping the future of rare disease development and access.

The rare disease landscape continues to evolve rapidly, with scientific advances creating new opportunities for patients with previously underserved conditions. At the same time, policymakers, regulators, payers, industry, and patient organizations are navigating increasingly complex questions around evidence generation, access, affordability, and patient engagement.

Taking place in Boston from June 9–11, the World Orphan Drug Congress USA 2026 will bring together stakeholders from across the rare disease ecosystem to explore these challenges and opportunities. As a media partner, The Evidence Base will be on site throughout the congress, with Executive Editor Joanne Walker attending to report on key discussions, emerging trends, and insights from across the rare disease community.

Reviewing this year's agenda, three interconnected themes stand out: market access in an evolving policy and reimbursement environment, the growing importance of innovative evidence generation strategies, and the continued shift from patient centricity toward meaningful patient partnership throughout the development lifecycle. Here, Joanne highlights a selection of sessions that reflect these themes and what attendees can expect from the discussions.

Market access in a changing policy and reimbursement environment

Access to rare disease therapies remains one of the most significant challenges facing the healthcare ecosystem. While scientific innovation continues to expand treatment possibilities, policymakers, payers, manufacturers, and patient advocates are navigating increasing pressure around affordability, reimbursement, and long-term sustainability.

Several sessions at the congress will explore how evolving policy and pricing frameworks are influencing access decisions across both the US and Europe. Discussions will examine topics including Most-Favored-Nation pricing, international reference pricing, emerging CMS policies, the growing influence of health technology assessment (HTA), and changing commercial payer coverage practices. Together, these sessions highlight the growing need for manufacturers to consider evidence generation, pricing strategy, reimbursement requirements, and patient support infrastructure much earlier in the development process.

Why I'm looking forward to these sessions: What makes this collection of sessions particularly interesting is the diversity of perspectives represented across industry, payers, patient advocacy groups, policymakers, and market access experts. Attendees will have the opportunity to hear how these stakeholders are interpreting the same challenges through different lenses, providing valuable insight into how access expectations are evolving and where future opportunities and barriers may emerge for orphan medicines.

Selected sessions

Most Favored Nation (MFN) and International Reference Pricing (IRP) for Orphan Medicines and the Future of European Access and Launch Strategy

Date and time: Tuesday June 9, 2026, 12:00pm
Speakers: Andrew Cummins (Sciensus Pharma Services ) and Denise Chaudhary (Clarivate Analytics)

Staying Ahead of the Curve: Strategic Responses to a Shifting Policy and Pricing Landscape for Orphan Drug

Date and time: Tuesday June 9, 2026, 3:00pm
Speakers: Amanda Forys, Amanda O'Hara and Tracy Baroni Allmon (Magnolia Market Access)

Designing Global Launches Before Constraints Lock In

Date and time: Tuesday June 9, 2026, 4:00pm
Speaker: Sonit Handa (Biovance Consulting)

Coverage That Keeps Up With Innovation: Access to Rare Disease Therapies

Date and time: Wednesday June 10, 2026, 11:30am
Moderator: Laura Henkhaus (Center for US Healthcare Policy Research, Johnson & Johnson)
Speakers: James Chambers (Tufts Medical Center), Deanna Steinle (Resilience is Beautiful) and Melissa Paige (National Association of Medication Access & Patient Advocacy)

Why is Collaboration in Expanded Access fundamental to Rare Patient Communities?

Date and time: Wednesday June 10, 2026, 10:10am
Speakers: Rachel Harrison (Argenx), Thomas Bartlett (Rare Disease Patient Advocate) and Naomi Litchfield (Bionical Emas)

Make Day-One Access Inevitable: A Practical Blueprint for EAP Transitions

Date and time: Wednesday June 10, 2026, 12:30pm
Speaker: Kristine McGaughey (ConnectiveRx)

Value in Rare Disease: A Global View of Trials, Endpoints, and Access in 2026

Date and time: Thursday June 11, 2026, 9:50am
Moderator: Jayson Slotnik (Health Policy Strategies, LLC)
Speakers: Nathan Walters (Johnson & Johnson), Daria Julkowska (ERDERA – INSERM) and Charlene Son Rigby (Global Genes)

Coverage of Rare Disease Therapies in U.S. Commercial Insurance: Patterns and Trends

Date and time: Thursday June 11, 2026, 11:40am
Speaker: James Chambers (Tufts Medical Center)

Barriers to Access and Implementing Strategies for Patient Centric Policies

Date and time: Thursday June 11, 2026, 12:40pm
Moderator: Michael Eging (Rare Access Action Project)
Speakers: Jayson Slotnik (Health Policy Strategies, LLC), Alex Mexiner (The ALS Association), Melissa Horn (Arthritis Foundation) and Jon Campbell (National Pharmaceutical Council)

Collaborating for Coverage: How Industry, CMS, and Patients Can Advance Access to Orphan Drugs

Date and time: Thursday June 11, 2026, 12:40pm
Moderator: Karin Hoelzer, (Biotechnology Innovation Organization)
Speakers: Thomas Bartlett (Rare Disease Patient Advocate), Melanie Lendnal (The ALS Association) and Yuri Maricich (CAMP4 Therapeutics)

From Designation to Decision: Fast Tracking UK, Ireland & EU Access for Orphan Medicines

Date and time: Thursday June 11, 2026, 12:40pm
Moderator: Brenda Dooley (AXIS - The Reimbursement Experts)
Speakers: Isabelle Lousada (Amyloidosis Research Consortium), Jenna Dilkes (NICE) and Patricia Synnott (Tufts Medical Center)

From Value to Volume: Comparing HTA and Market-Driven Pricing for Orphan Drugs in Europe and the United States

Date and time: Thursday June 11, 2026, 2:40pm
Speakers: Alexander Natz (EUCOPE - European Confederation of Pharmaceutical Entrepreneurs) and Amanda Forys (Magnolia Market Access)

Advancing patient partnership in rare disease research and decision-making

The importance of incorporating the patient voice into rare disease research and development is now widely recognized. Increasingly, however, the conversation is moving beyond patient centricity toward a more comprehensive model of partnership, where patients, caregivers, and advocacy organizations actively contribute to decisions throughout the development and access journey.

Several sessions at this year's congress will explore how patient perspectives can be integrated more effectively into regulatory engagement, policy development, clinical trial design, evidence generation, and access discussions. Topics range from strengthening engagement between patient advocacy groups and the FDA to addressing mental health needs within rare disease communities and ensuring patient experiences inform both development and commercialization strategies.

The congress's commitment to patient engagement extends beyond the formal agenda. Throughout the event, attendees will also be able to visit the dedicated Patient Zone, home to the Rare Community Hub, which will bring together patient advocacy organizations, community groups, and industry stakeholders. Featuring organizations including CheckRare, RTW Foundation, and Pathways to Trust, the space will provide opportunities to connect with the rare disease community, explore educational resources, and participate in interactive activities designed to amplify patient voices and foster collaboration.

Why I'm looking forward to these sessions: Patient centricity is a term that is frequently used across healthcare, but these sessions go a step further by examining what meaningful patient partnership looks like in practice. I'm particularly interested in hearing directly from patient advocates, caregivers, and community leaders alongside industry and policy stakeholders. Their perspectives should help attendees better understand how patient experiences can be translated into more relevant research, more informed policy decisions, and ultimately better outcomes for rare disease communities.

Selected sessions

Multidimensional Pathways: Helping PAGs of All Sizes Plan for Engagement with FDA

Date and time: Tuesday June 9, 2026, 12:00pm
Speakers: Melody Kisor (FamilieSCN2A Foundation) and Erica Barnes (State Of Minnesota)

Supporting the Mental Health Needs of Rare Disease Communities: Programs That Work

Date and time: Tuesday June 9, 2026, 2:00pm
Speakers: Albert Freedman (Rarecounseling.com) and Julie Wells (Give an Hour)

Building a Patient-Centered Policy Framework for Rare Disease Drug Development

Date and time: Wednesday June 10, 2026, 9:10am
Moderator: Julianna LeMieux (Genetic Engineering & Biotechnology News)
Speakers: Wendy Erler (Sarepta Therapeutics), Pamela Gavin (National Organization for Rare Disorders (NORD)) and Annie Kennedy (EveryLife Foundation for Rare Diseases)

Putting the Patient First, How to Partner with Advocacy Organizations

Date and time: Wednesday June 10, 2026, 11:50am
Speaker: Jonathan Kornstein (Caidya)

Voice of the Patient: Gaps in Genetic Diagnosis, Access, and Precision Medicine

Date and time: Wednesday June 10, 2026, 11:50am
Moderator: Sara Riordan (Genome Medical)
Speaker: Colleen Caleshu (Genome Medical)

Fireside Chat: Empowering Patients as Partners: Driving Orphan Drug Market Success Through Advocacy and Engagement

Date and time: Wednesday June 10, 2026, 12:15pm
Speakers: Bonnie Kiesling (ITF Therapeutics) and Susan (Patient Caregiver)

Evolving evidence generation strategies to meet the challenges of rare disease development

Generating robust evidence for rare disease therapies has never been straightforward, with small patient populations, heterogeneous disease presentations, and evolving treatment paradigms continuing to challenge traditional development approaches. At the same time, regulators, payers, and patients are seeking evidence that not only demonstrates safety and efficacy but also reflects outcomes that are meaningful in real-world settings.

Several sessions at the congress will examine how stakeholders are responding to these challenges through innovative trial designs, patient-relevant endpoints, real-world data (RWD), patient-reported outcomes, and novel approaches to data collection and analysis. Discussions will also explore efforts to strengthen global alignment around evidence expectations and consider how emerging technologies, data sources, and individualized therapies may reshape future evidence generation strategies. Many of these sessions ultimately focus on a common question: what constitutes decision-ready evidence in rare diseases, and how can it be generated efficiently without compromising scientific credibility?

Why I'm looking forward to these sessions: What makes this collection of sessions particularly interesting is the balance between strategic and practical discussions. From regulatory expectations and patient-relevant endpoints to RWD and individualized therapies, attendees will gain insight into both the opportunities and challenges associated with generating evidence in rare diseases. The sessions will also offer a valuable opportunity to better understand where consensus is emerging and where important questions remain unresolved.

Selected sessions

Bridging the Gap Between Patient Relevance and Regulatory Reliability- Sharing the outcomes of the Expanding Regulatory Agility and Evidentiary Integrity in Developing Treatments for Rare Diseases

Date and time: Tuesday June 9, 2026, 10:00am
Moderator: Jennifer McNary (Canary Concierge)
Speaker: Susan Winckler (Reagan Udall Foundation for the FDA)

Global Alignment in Rare Disease Drug Development

Date and time: Wednesday June 10, 2026, 10:10am
Moderator: Rachel Smith (Parexel International LLC)
Speakers: Christopher Juenger (CSL), Kinnari Patel (Rocket Pharma) and Yann Le Cam (VOZ

Beyond AI Predictions: Identifying Early Rare Disease Patients Through Real-Patient-Evidence Data

Date and time: Wednesday June 10, 2026, 12:30pm
Speaker: Arndt Rolfs (subrosa Health & Capital Group)

From Claims to Commercial Strategy in Rare Disease: Signal vs. Noise

Date and time: Wednesday June 10, 2026, 3:40pm
Speaker: Lucas Paine (Forian)

Generating Evidence that Counts: Data Strategies for Individualized Medicines

Date and time: Wednesday June 10, 2026, 3:40pm
Speaker: Cynthia Cassandro (Vigil Neuroscience)

Making Rare Diseases Count: How Better Data Can Unlock a Multi-Trillion-Dollar Opportunity

Date and time: Thursday June 11, 2026, 11:40am
Speaker: Will Greene (Foundation for Prader-Willi Research)

Accelerating Rare Disease Diagnosis: Leveraging Health Claims Data Early in Clinical Development

Date and time: Thursday June 11, 2026, 11:40am
Speaker: Connie Lee (Alliance to Cure Cavernous Malformations)

From Symptoms to Decisions: How Patient-Reported Outcomes Shape Trustworthy Clinical Evidence

Date and time: Thursday June 11, 2026, 2:00pm
Speaker: Joan Mendivil (Pharvaris)

Coverage of the World Orphan Drug Congress USA 2026 by The Evidence Base

We’ll be on site in Boston attending some of these key sessions and providing key insights at the end of the conference. Follow The Evidence Base on LinkedIn for updates and for exclusive post-event analysis and session round-ups, register on our site.