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Wednesday, June 18
 

8:30am EDT

Rare but Real: Navigating Rare Disease Product Development and Market Access Challenges
Wednesday June 18, 2025 8:30am - 9:30am EDT
TBD
Component Type: Session
Level: Intermediate

Experts representing industry, non-profit, research, regulatory, and payor perspectives will explore key hurdles and solutions to rare disease treatment development, from recruitment and diagnosis to late-stage development and access after accelerated approval.

Learning Objectives

Identify hurdles and solutions to clinical trial recruitment, including early diagnosis and newborn screening; Identify approaches to patient stratification, disease understanding, and novel biomarkers, while de-risking late-stage development in competitive and high-failure landscapes; Examine challenges of market access and pricing after accelerated approval, and gain insights into addressing payer-specific concerns to ensure equitable access.

Speaker

Panelist
Annie Kennedy


Speakers
avatar for Annie Kennedy

Annie Kennedy

Chief of Policy, Advocacy, and Patient Engagement, EveryLife Foundation for Rare Diseases, United States
Annie has served within the community for nearly three decades through her roles with Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA). In that time she helped lead legislative efforts around passage and implementation of the MD-CARE Act (2001... Read More →
Wednesday June 18, 2025 8:30am - 9:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  14: DIAmond, Session
 
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    00: Plenary
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    01: ClinSafety-PV
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    02: ClinTrials-Ops
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    03: Data-Tech
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    04: MedAffairs-SciComm
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    05: Patient-Impact-Product-Dev
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    06: PersonalizedMed-ComboProd-Diagnostics
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    07: ProjectManagement-StrategicPlanning
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    08: RD-Quality-Compliance
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    10: RegCMC-Product Quality
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    11: Statistics-Data Science
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    12: ProfDevelopment
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    13: Spotlight
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    14: DIAmond
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    15: Content-Hubs
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    16: Community-Rounds
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    17: Posters
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    18: InnovationTheaters
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    20: Short-Courses
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  • Level
    Advanced
    Basic
    Intermediate
  • Keyword
    Advanced Analytics
    Artificial Intelligence (AI)
    Bioethics
    Biomarkers
    Cell and Gene Therapy
    Clinical Trial Design
    Diagnostics
    Digital Technology
    Global Perspectives
    Non-CE
    Patient Experience
    Payers
    Pediatrics
    Rare Disease
    Real-World Data (RWD) / Real-World Evidence (RWE)
    Regulator Perspectives
    Regulatory Collaboration
    Regulatory Convergence and Harmonization
    Regulatory Reliance
    Student Programming
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EH
TBD
Virtual
  • 00: Plenary
  • 01: ClinSafety-PV
  • 02: ClinTrials-Ops
  • 03: Data-Tech
  • 04: MedAffairs-SciComm
  • 05: Patient-Impact-Product-Dev
  • 06: PersonalizedMed-ComboProd-Diagnostics
  • 07: ProjectManagement-StrategicPlanning
  • 08: RD-Quality-Compliance
  • 09: Regulatory
  • 10: RegCMC-Product Quality
  • 11: Statistics-Data Science
  • 12: ProfDevelopment
  • 13: Spotlight
  • 14: DIAmond
  • 15: Content-Hubs
  • 16: Community-Rounds
  • 17: Posters
  • 18: InnovationTheaters
  • 20: Short-Courses
  • Level
  • Advanced
  • Basic
  • Intermediate
  • Keyword
  • Advanced Analytics
  • Artificial Intelligence (AI)
  • Bioethics
  • Biomarkers
  • Cell and Gene Therapy
  • Clinical Trial Design
  • Diagnostics
  • Digital Technology
  • Global Perspectives
  • Non-CE
  • Patient Experience
  • Payers
  • Pediatrics
  • Rare Disease
  • Real-World Data (RWD) / Real-World Evidence (RWE)
  • Regulator Perspectives
  • Regulatory Collaboration
  • Regulatory Convergence and Harmonization
  • Regulatory Reliance
  • Student Programming