FDA Rare Disease Innovation Hub: Enhancing Collaboration and Speed?

In 2023, over half of all the novel drugs and biologics approved by the FDA’s Center for Drug Evaluation and Research (CDER) and the FDA’s Center for Biologics Evaluation and Research (CBER) were to prevent, diagnose or treat a rare disease or condition. The recently-formed FDA Rare Disease Innovation Hub is a new model of collaboration between CDER and CBER – and a key indicator of their commitment to explore new approaches for the development and approval of safe and effective drugs and biologics for rare diseases. 

This webinar discussion focuses on the desired priorities and potential outcomes of this initiative from the perspectives of the rare disease clinical research community – including those of a rare disease clinician, former FDA regulator and rare disease clinical research scientists. Key questions raised in this discussion include:

  • What outcomes are most important for the research community? For patients and their families? What are the top priorities for improvement from the drug development ecosystem in support of rare disease?
  • Which longstanding challenges are most critical for the FDA to address and why? What resources are needed to help solve these challenges?
  • Are there  similar regulatory initiatives that are helping the FDA keep pace with drug development complexity and the recent breakthroughs in genetic science, personalized medicine and novel therapies? What learnings and advancements are currently being applied to the rare disease space or can be? 
  • What emerging practices and development approaches are likely here to stay? What expanded role can the research community take in partnering with the FDA and how?

Read transcript

 

Return to Insights Center

Related Insights

Blog

Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress

May 2, 2024

Report

New Medicines, Novel Insights: Advancing rare disease drug development

May 22, 2023

Blog

Celebrating 40 Years of Rare Disease Progress: WODC Highlights

Jun 6, 2023

Podcast

Rare endpoints: Delivering on unmet patient needs

May 7, 2024

Article

Regulation pathway/strategy: Key considerations for foreign medical product registration in China

Jul 1, 2025

Video

How to transition existing trials under EU-CTR

Feb 1, 2023

Webinar

How to navigate China’s regulatory environment

Feb 15, 2023

Playbook

Insights from the 2022 R&D Innovation Survey

Feb 17, 2023

Whitepaper

The Chinese Pharmaceutical Market: Size, R&D, Regulations, Market Access and Innovations

Mar 9, 2023

Playbook

Mitigating risk, protecting potential: Practical strategies that position cell and gene therapy development for success

Mar 28, 2023

Report

New Medicines, Novel Insights: Accelerating development of cell and gene therapies

May 22, 2023

Webinar

Assessing appropriate use of ECAs in clinical trials

May 28, 2023

Related Insights

Blog

Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress

May 2, 2024

Report

New Medicines, Novel Insights: Advancing rare disease drug development

May 22, 2023

Blog

Celebrating 40 Years of Rare Disease Progress: WODC Highlights

Jun 6, 2023

Podcast

Rare endpoints: Delivering on unmet patient needs

May 7, 2024

Article

Regulation pathway/strategy: Key considerations for foreign medical product registration in China

Jul 1, 2025

Video

How to transition existing trials under EU-CTR

Feb 1, 2023

Show more