Rare Diseases And Orphan Drugs – Access & Reimbursement – Special Topics: Partnering With Patient Advocacy Groups To Accelerate Orphan Drug Development (US)

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.

QUESTIONS ANSWERED

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

  • Rare Diseases And Orphan Drugs - Access & Reimbursement - 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
      • Expert Insight
    • Mission and Goals of Advocacy Groups in Rare Diseases
      • Expert Insight
    • R&D Support
      • Key Findings
      • Patient Registries and Natural History Studies
        • Expert Insight
      • Clinical Trial Enrollment
        • Expert Insight
      • Development of Novel Clinical Trial End Points
        • Expert Insight
      • Diagnosis
        • Expert Insight
      • Advisory Services for Clinical Program Development
        • Expert Insight
      • Funding of R&D
        • Expert Insight
    • Interaction with Key Stakeholders
      • Key Findings
      • FDA
        • Expert Insight
      • Patients
        • Expert Insight
      • Biopharma Companies
        • Expert Insight
      • Payers
        • Expert Insight
        • Expert Insight
      • Government
        • Expert Insight
    • Appendix
      • List of Abbreviations
      • Bibliography
      • About the Author