Rare Disease Initiative

Goodwin stands proudly with the rare disease community.

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.

Our Pillars

Our rare disease initiative has four pillars:
1. Legal Excellence
We serve as a resource for the unique legal, regulatory, and compliance needs of research organizations; patient advocates; biotech, medtech, and diagnostic companies and service providers.
2. Regulatory and Policy Support
We respond to and help shape regulatory and public policy developments, whether at the FDA, CMS, HHS, or Congress, that support the rare disease community.
3. Educational Resources
We provide educational, benchmarking, and networking opportunities, such as our annual Goodwin Rare Disease Day, for stakeholders in this space — from research organizations to therapeutic and medical products companies and their investors.
4. Pro Bono Advocacy
We give back through meaningful pro bono relationships with patient advocacy and research organizations, which allows our lawyers to contribute their time, energy, and expertise to the rare disease community.

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 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

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!

Goodwin Boston Office Goodwin DC Office Goodwin Philadelphia Office Goodwin Silicon Valley Office Goodwin Santa Monica Office

Connect with us

Join our rare disease initiative distribution list to receive more information about our annual Goodwin Rare Disease Day event and other periodic updates.

Contact our team to learn more about our rare disease initiative or to discuss your specific needs.