Four ways to increase rare disease drug development success
There are more than 7,000 identified rare diseases today, 71.9% of which are genetic. And only around 5% of rare diseases have treatments. Development is complex and presents specific challenges. For example, many rare disease patients are children, since nearly 70% of rare diseases are exclusively pediatric-onset. The patient pool is often very limited for any one disease, and patients are often geographically dispersed, highlighting the importance of selecting a development partner with strong patient recruitment and global operational capabilities.
In this playbook, Parexel shares insights on maximizing the chances of success in rare disease drug development, providing our perspectives on four of the most significant challenges:
- Easing trial burdens on sites, and enhancing the clinical staff experience
- Ensuring that pediatric trials are ethical and feasible for patients and families
- Getting to market faster and stronger with natural history studies and real-world evidence
- Learning from past successes and failures in orphan indications
We hope you find these articles helpful on your journey.
Related Insights
Blog
Optimize your post-approval CMC change management in China with the ICH Q12 tool
Nov 24, 2025
Playbook
Are you using real-world evidence?
Feb 1, 2023
Podcast
Get Recruited to Be a Clinical Research Associate: The Benefits of Parexel’s FSP Program
Oct 27, 2022
Article
Relyvrio Approval: Lessons Learned
Oct 31, 2022
Article
Five strategies for meeting the requirements of Project Optimus and improving the chances of approval
Nov 10, 2022
Blog
New CNS Franchise Leader brings experience from pharma and as a clinician and researcher
Nov 14, 2022
Podcast
FSP Podcast Series | Episode 2: Celebrating a 25-Year Career with Kerri McCaul-Claus
Dec 6, 2022
Blog
Innovative modeling method could speed patient access to critical IO therapies
Jan 4, 2023
Blog
The New Frontier of Innovative Therapies: Navigating What's Next
Jan 12, 2023
Video
How to transition existing trials under EU-CTR
Feb 1, 2023
Article
Q&A Project Optimus: What you need to know
Oct 11, 2022
Whitepaper
Optimizing Biosensor-based Trials
Feb 1, 2023
Related Insights
Blog
Optimize your post-approval CMC change management in China with the ICH Q12 tool
Nov 24, 2025
Playbook
Are you using real-world evidence?
Feb 1, 2023
Podcast
Get Recruited to Be a Clinical Research Associate: The Benefits of Parexel’s FSP Program
Oct 27, 2022
Article
Relyvrio Approval: Lessons Learned
Oct 31, 2022
Article
Five strategies for meeting the requirements of Project Optimus and improving the chances of approval
Nov 10, 2022
Blog
New CNS Franchise Leader brings experience from pharma and as a clinician and researcher
Nov 14, 2022
Podcast
FSP Podcast Series | Episode 2: Celebrating a 25-Year Career with Kerri McCaul-Claus
Dec 6, 2022
Blog
Innovative modeling method could speed patient access to critical IO therapies
Jan 4, 2023
Blog
The New Frontier of Innovative Therapies: Navigating What's Next
Jan 12, 2023
Video
How to transition existing trials under EU-CTR
Feb 1, 2023
Article
Q&A Project Optimus: What you need to know
Oct 11, 2022
Whitepaper
Optimizing Biosensor-based Trials
Feb 1, 2023