Rare Disease Resource Library

Advocates, Medical Professionals, Pediatric, Physician, Researchers, Advocacy, Research & Drug Development, Publication, Medical Affairs, Research,

December 13, 2024

How To Advocate For Rare Disease-Related Policies: A Guide for Professionals Involved in Diagnosis, Care, or Research of Rare Diseases

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

October 27, 2022

What You Need to Know About the Rare Disease Cures Accelerator-Data and Analytics Platform

Advocates, Patient & Caregiver, Patient Organizations, Researchers, Students & Volunteers, Advocacy, RDCA-DAP, Research & Drug Development, Infographic, PDF, Education, Policy,

October 20, 2022

The Five Steps in Drug Development (Spanish version)

Advocates, Patient & Caregiver, Patient Organizations, Researchers, Students & Volunteers, Advocacy, RDCA-DAP, Research & Drug Development, Infographic, PDF, Education, Policy,

The Five Steps in Drug Development

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, Medical Professionals, Patient Organizations, Students & Volunteers, Advocacy, Legislative Advocacy, Research & Drug Development, PDF, Publication, Education, Policy,

Study on Orphan Drug Costs & Use

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

Orphan Drug Costs & Expenditures

Advocates, Patient & Caregiver, Patient Organizations, Students & Volunteers, Advocacy, Research & Drug Development, Understanding Rare Disease, Infographic, PDF, Publication, Education,

NORD RareInsights^® 5 Myths About the ODA

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

Orphan Drug Trends in the United States 2018