Rare Disease Initiative

Goodwin’s Life Sciences Regulatory & Compliance team handles a host of legal and regulatory issues for rare disease companies to help surmount the unique challenges faced by patients, providers, companies, and investors seeking to find diagnostic, therapeutic, and other treatment for rare diseases. Together, we help our rare disease clients by:

• Seeking and securing expedited program and orphan designations with the FDA
• Understanding the dynamics of orphan and pediatric exclusivity
• Navigating the complexities of FDA interactions and trial design
• Handling clinical trial site setup, recruitment, safety reporting and investigator compensation issues
• Preparing marketing and market access plans for launch readiness
• Developing expanded access programs and supporting investigator-initiated research
• Supporting efforts to secure payor coverage, and navigating specialty pharmacy arrangements
• Understanding the implications of and navigating clients through the Medicare Drug Price Negotiation Program, the Medicaid Drug Rebate Program, and 340B Program
• Engaging with patient and caregiver advocacy groups
• Counseling on patient support and other financial assistance programs  
• Managing inspection, audit, and warning letter responses
• Providing legislative counseling and support on policy developments

Read insights from our Life Sciences Perspectives blog for the latest legal happenings in the rare disease community:

• The Inflation Reduction Act Resource Page
• The European Commission Proposes First Major Overhaul of the EU Medicines Regulatory Framework in 20 Years: Orphan Medicines
• Leveraging Investigator-Initiated Trials in Rare Disease Drug Development
• Clinical Trial Diversity Plans and Rare Diseases
• Understanding Data Monitoring Committee Conflict of Interest Limitations
• For Clinical Trial Recruiting, Words Matter

To learn more about rare diseases and the patients that they affect, consider the following external resources:

• List of Rare Diseases
• Genetic and Rare Diseases Information Center
• Search Orphan Drug Designations and Approvals 
• Office of Orphan Products Development
• Funding Opportunities for Rare Disease Research
• Specific Public Health Genomics and Knowledge Base for Rare Diseases
• NORD “Rare Disease Drug Development: What Patients and Advocates Need to Know” Learning Series
• NORD State Report Cards
• NORD #LightUpForRare
• EveryLife Foundation for Rare Diseases ICD Code Roadmap
• EveryLife Foundation for Rare Diseases Guide to Patient Involvement In Rare Disease Therapy Development