Patient advocacy groups play many roles in orphan drug development, including funding and propelling early development and interacting with key stakeholders such as the patients, their caregivers, regulatory bodies, elected officials, and payers. By engaging with these organizations throughout the drug development process, drug developers may have a smoother development path and be able to launch new therapies for these rare diseases faster.


How do patient advocacy groups support research & development efforts in the rare disease space? How can biopharma companies support these endeavors?

How do patient advocacy groups interact with key industry stakeholders?

How can biopharma companies support nascent advocacy groups, and how should they engage established organizations?

Table of contents

  • Special Topics: Partnering with Patient Advocacy Groups to Accelerate Orphan Drug Development (US)
    • Overview and Actionable Recommendations
      • Actionable Recommendations to Optimize Interactions with Patient Advocacy Groups
    • Mission and Goals of Advocacy Groups in Rare Diseases
    • R&D Support
      • Key Findings
      • Patient Registries and Natural History Studies
      • Clinical Trial Enrollment
      • Development of Novel Clinical Trial End Points
      • Diagnosis
      • Advisory Services for Clinical Program Development
      • Funding of R&D
    • Interaction with Key Stakeholders
      • Key Findings
      • FDA
      • Patients
      • Biopharma Companies
      • Payers
        • Activities to Support Value Demonstration
        • Activities to Improve Access to Available Medications
      • Government
    • Methodology
      • Primary Market Research
    • Appendix
      • About the Author

Author(s): Claudia Dall'Osso

Claudia is a Senior Business Insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing on niche and rare indications. Before joining DRG, she held a management and strategy consultant position at Precision Medicine Group, where she worked for clients in the biopharmaceutical, medical device and diagnostic industries. Claudia completed her Master’s in Management at Harvard University; she also holds a in medical genetics from Brescia University in Italy and a BS/MS degree in medical biotechnology from University of Milano in Italy.

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