Resource Library

Advocacy, Regulatory, Research & Drug Development

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

Regulatory, Research & Drug Development

Published June 1, 2021

FDA’S Accelerated Approval Pathway: A Rare Disease Perspective

Advocacy, Legislative Advocacy, Rare Disease Advisory Councils (RDAC)

Webinar Recording: Your State Just Enacted an RDAC – Now What?

Legislative Advocacy, Rare Disease Advisory Councils (RDAC)

Toolkit: Advocating for a Rare Disease Advisory Council in Your State

Understanding Rare Disease

Published March 1, 2015

Huntington’s Disease: Emerging Concepts in Diagnosis and Treatment

Understanding Rare Disease

Published February 1, 2016

2016 Neurological Rare Disease Special Report

Understanding Rare Disease

Published March 1, 2017

2017 Neurological Rare Disease Special Report

Understanding Rare Disease

Published March 1, 2018

2018 Neurological Rare Disease Special Report

Understanding Rare Disease

Published March 1, 2019

2019 Neurological Rare Disease Special Report

Rare Cancer, Understanding Rare Disease

Published December 1, 2020

2020 Rare Diseases Report: Cancers

Understanding Rare Disease

Published November 1, 2020

2020 Rare Diseases Report: Rheumatology

RDCA-DAP, Research & Drug Development

C-Path Data Integration and Analytics Approaches

RDCA-DAP, Research & Drug Development

NORD Registry Presentation

RDCA-DAP, Research & Drug Development

Accelerating Drug Development for Rare Diseases

RDCA-DAP, Research & Drug Development

Current Challenges in Rare Disease Drug Development