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?
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
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 Ph.D. in medical genetics from Brescia University in Italy and a BS/MS degree in medical biotechnology from University of Milano in Italy.
