Rare diseases represent a diverse range of medical conditions that impact only a small percentage of the global population. This small but strong community is supported by patient advocates and champions, caregivers, healthcare providers, and companies leveraging innovations and their collective commitment to addressing unmet needs — including advanced testing, technology, software, and therapy options — to help rare disease patients.
Spearheaded by our Life Sciences practice, our rare disease initiative works with biotech, pharmaceutical, medical technology, and diagnostic companies and their investors, partners, and key stakeholders focused on developing solutions for countless rare medical conditions.
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MORE ON OUR 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 orphan designation with the FDA
- Understanding the dynamics of orphan exclusivity
- Navigating the complexities of drug repurposing
- Addressing patient recruitment needs in light of small patient populations
- Handling site and investigator compensation issues
- Attaining FDA market authorization support
- Developing expanded access programs as patients with unique rare disease needs transition from research product to commercially covered product
- 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
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:
Goodwin Rare Disease Symposium 2024
A Look Back at Goodwin Rare Disease Day 2023
In global observance of Rare Disease Day 2023, the Goodwin Life Sciences Regulatory & Compliance group hosted an event to spotlight the critical work being done to address the 7,000+ rare diseases that impact more than 300 million people globally. Learn more.
Goodwin Rare Disease Day
On Rare Disease Day 2024 Goodwin lit our offices from coast-to-coast in Boston, DC, Philadelphia, Santa Monica, and Silicon Valley in Rare Disease Day colors!
Contact our team to learn more about our rare disease initiative or to discuss your specific needs.
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Julie Tibbets
PartnerChair, Life Sciences Regulatory & Compliance Practice - /en/people/w/wetzel-matt
Matt Wetzel
PartnerLife Sciences Regulatory & Compliance