Rare Disease Resource Library

Advocates, Patient & Caregiver, Insurance, Legislative Advocacy, Regulatory, Webinar, Policy,

November 27, 2023

Medicaid Unwinding

Advocates, Medical Professionals, Researchers, Advocacy, Gene Therapy, Regulatory, Research & Drug Development, Educational Video, Education, Policy, Research,

October 17, 2022

The FDA’s Role in Gene Therapy

Advocates, Medical Professionals, Researchers, Regulatory, Research & Drug Development, PDF, Publication, Policy,

FDA’s Flexibility in Regulating Therapies

Advocates, Patient Organizations, Researchers, Advocacy, Regulatory, Research & Drug Development, Collection, Publication, Education, Policy,

Orphan Drug Trends in the United States 2018

Advocates, Patient Organizations, Advocacy, Regulatory, Telehealth, Understanding Rare Disease, PDF, Publication, Education,

NORD RareInsights^® Ensuring Access to Telehealth for Rare Diseases

Advocates, Patient Organizations, Researchers, Advocacy, Regulatory, Research & Drug Development, PDF, Publication, Education, Policy,

Orphan Drugs in the United States: An Examination of Patents and Orphan Drug Exclusivity

Advocates, Medical Professionals, Researchers, Regulatory, Research & Drug Development, PDF, Publication, Education,

FDA’S Accelerated Approval Pathway: A Rare Disease Perspective

Advocates, Medical Professionals, Researchers, RDCA-DAP, Regulatory, Research & Drug Development, Educational Video, Webinar, Research,

From Data To Cures: Speeding The Way For Rare Diseases

Advocates, Medical Professionals, Researchers, RDCA-DAP, Regulatory, Research & Drug Development, Educational Video, Webinar, Research,

How Can Patients and Advocates Help Accelerate Drug Development?

Medical Professionals, Patient Organizations, Researchers, Regulatory, PDF, Other,

September 28, 2022

FDA’s Flexibility in Regulating Therapies