Elevating the Future of Rare Disease Medication Access and Patient Support

Building Sustainable Access for Breakthrough Therapies

Sept 8th, 2026 | Las Vegas

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Call For Speakers

Space is limited — secure your spot early!

⚠️

Space is limited — secure your spot early! ⚠️

The Rare Disease Summit USA brings together payers, providers, manufacturers, and access experts to tackle one of healthcare’s most urgent challenges.

This one-day summit goes beyond the headlines — diving straight into the real-world challenges of affordability, reimbursement, and delivery for groundbreaking rare disease therapies. Together, we’ll confront the critical question:

How do we ensure patients can access transformative treatments without bankrupting families or the healthcare system?

Through a powerful day of keynotes, panels, patient case studies, and collaborative roundtables, the program explores the entire ecosystem shaping the future of advanced therapies — from payer and provider coordination to HUB innovation, patient navigation, and long-term sustainability.

Why the Summit on Rare Disease Matters

The future of medicine is being shaped right now and it can’t be written by science alone. Breakthrough therapies only change lives if patients, providers, payers, and policymakers work together to remove barriers and build new models of care. This summit is a catalyst for that collaboration, bringing every voice to the table so progress in the lab becomes progress in the real world.

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What Attendees will Gain

At the Rare Disease Summit USA, you’ll gain the insights, strategies, and connections to accelerate real-world progress in this rapidly advancing field. Every session is built for action — turning complex challenges into practical solutions.

  • A deeper understanding of the current state of rare disease access and affordability

  • Insights into payer perspectives, reimbursement approaches, and sustainability models

  • Awareness of patient assistance and financial navigation gaps in advanced therapies

  • Perspective on specialty pharmacy, distribution, and HUB service challenges

  • Real-world case studies and lessons learned from patient and provider experiences

  • A forward look at where the field is heading and how stakeholders can prepare together

Together, we’ll move beyond innovation to implementation — ensuring that access keeps pace with advancement.

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Who Should Attend

This summit is built for innovators shaping the future of advanced therapies — those bringing treatments from development to delivery.

You’ll find immense value if you’re focused on:

  • Navigating access and reimbursement realities for high-cost, one-time therapies

  • Designing patient support strategies that align with payer expectations and policy shifts

  • Building launch frameworks that integrate affordability, sustainability, and stakeholder trust

  • Collaborating across providers, payers, and partners to strengthen real-world implementation

  • Translating innovation into impact — ensuring every approved therapy reaches the patients it was created for

If you’re ready to move beyond the science to solve the access equation, this summit is where the right conversations — and the right connections — happen.

    • Vice Presidents and Directors of Market Access

    • Managed Markets and Reimbursement Strategy Executives

    • Commercial Operations and Field Deployment Leaders

    • Franchise Heads and Brand Directors by Therapeutic Area

    • Business Development and Strategic Partnerships Leaders

    • Patient Access Services Executives

    • HUB Program Directors and Managers

    • Co-Pay and Affordability Strategy Leaders

    • Specialty Pharmacy Integration Managers

    • Medical Affairs Directors and Field MSL Leads

    • Clinical Development and Investigator Relations Teams

    • Scientific Communications and Evidence Generation Leaders

    • HEOR (Health Economics & Outcomes Research) Executives

    • RWE (Real-World Evidence) and Outcomes Analytics Directors

    • Payer Evidence & Value Communication Leads

    • Government Affairs and Public Policy Executives

    • Advocacy Relations Directors

    • Public-Private Partnership Development Leaders

    • Trade and Distribution Executives

    • Specialty Pharmacy Relations Managers

    • Digital Therapeutics and Telehealth Commercial Teams

  • Specialty pharmacies, HUBs, technology platforms, and access partners that work alongside pharmaceutical manufacturers to support every step of the patient journey—streamlining medication access, enhancing affordability support, and removing barriers to ensure timely, coordinated care.

  • Pharmacies that manage the distribution and support of high-cost, complex therapies—ensuring patients receive timely access, education, and coordinated care.

  • Mission-driven organizations that provide critical support services, financial assistance, education, and advocacy to reduce barriers and improve access for underserved patient populations.

  • If your organization is navigating medication access, reimbursement, or patient affordability challenges — this is the meeting for your team.

    Recommended roles include:

    • Executive Leadership: Chief Medical Officer, Chief Pharmacy Officer, Chief Operating Officer, Chief Nursing Officer, VP of Specialty Services, or VP of Population Health

    • Pharmacy & Clinical Management: Pharmacy Director, Specialty Pharmacy Manager, Infusion Center Manager, Ambulatory Care Director, or Clinical Pharmacist Leader

    • Access & Financial Navigation Teams: Director of Patient Access, Financial Counselor Manager, Reimbursement Manager, Medication Assistance or Financial Navigation Program Lead

    • Practice Administration: Practice Manager, Billing and Coding Lead, Compliance Officer, Revenue Cycle Director, or Payer Relations Manager

    • Independent Practices & Specialty Clinics: Physicians, Nurse Practitioners, Office Managers, or Access Coordinators working directly with patients and payers

  • Payer and health plan leaders responsible for coverage, formulary, and utilization management decisions for high-cost and complex therapies. This includes medical and pharmacy directors, policy and strategy leaders, and teams shaping specialty drug management, reimbursement, and value-based care models.

Agenda Overview

At the Rare Disease Summit USA, you’ll gain the insights, strategies, and connections to accelerate real-world progress in this rapidly advancing field. Every session is built for action — turning complex challenges into practical solutions.

Key topics include:

  • The state of affordability and access for rare disease therapies in 2026 and beyond

  • Innovative reimbursement frameworks and sustainable funding models

  • Patient affordability and financial navigation for high-cost, one-time therapies

  • Distribution and specialty pharmacy coordination across complex treatment pathways

  • Next-generation HUB design integrating data, technology, and care team collaboration

  • Real-world case studies from early-access programs and treatment centers

  • Cross-functional collaboration to scale equitable, sustainable access

  • Future-focused leadership roundtables on policy, innovation, and system readiness

If you’re shaping the strategy behind tomorrow’s therapies — this is where your next breakthrough conversation begins.

Final agenda coming soon — get ready for a full day of strategy, innovation, and real-world insight.

Why It Matters

As a commercial planning anchor for the specialty ecosystem, The Rare Disease Summit USA delivers unmatched value through its real-time dialogue, multi-stakeholder engagement, and therapeutic-specific focus. With limited registration and a curated audience, this summit is tailored for organizations ready to act, innovate, and elevate patient outcomes through strategic access planning.

Download the White Paper: Navigating the Rare Disease Patient Journey

Go beyond the science and uncover the real-world systems that make advanced therapies possible. This exclusive white paper offers a comprehensive framework for navigating access, reimbursement, and coordination across the rare disease ecosystem — insights that set the stage for the Rare Disease Summit USA.

📥 Download Now and Join Us at the Summit to Continue the Conversation

Stay Up-to-Date with the Rare Disease Summit

Under the Specialty Summit USA umbrella, we are proud to launch an expansive conference series covering 11 therapeutic areas, each designed to deliver specialty-specific insights while fostering cross-functional collaboration and knowledge sharing.

The Specialty Summit USA convenes leaders and decision-makers from biopharma, specialty pharmacy, technology, policy, and care delivery — bringing together the stakeholders who drive access, innovation, and operational excellence across the healthcare continuum.

These summits are where strategic conversations become actionable solutions, uniting experts who are redefining how therapies reach the people who need them.

We hope you will join us for this event and across the Specialty Summit USA series as we shape the next era of specialty care and access.

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