New Medicines, Novel Insights: Advancing rare disease drug development
Novel therapies for rare diseases offer hope for thousands of patients and their families. This interactive report presents insights from Parexel experts doing everything humanly possible to deliver on the promise of patient-focused drug development – and to bring more rare disease treatments to market, faster.
Related Insights
Report
New Medicines, Novel Insights: Accelerating development of cell and gene therapies
May 22, 2023
Report
New Medicines, Novel Insights: Achieving patient-guided drug development
Oct 30, 2023
Video
Part 2: Risk-based Quality Management Video (RBQM) Series
Nov 11, 2021
Blog
Celebrating 40 Years of Rare Disease Progress: WODC Highlights
Jun 6, 2023
Blog
Adapting the validation process for PROs for rare diseases and other diseases with large unmet need and/or rapid progression
Apr 23, 2024
Blog
Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress
May 2, 2024
Podcast
Enabling Successful Sites, Episode 2: Empowering Sites to be More Inclusive Through Cultural Sensitivity Training
Jun 28, 2024
Podcast
Enabling Successful Sites, Episode 3: Taking the complexity out of oncology clinical trials
Jun 28, 2024
Blog
Including patients in DCT design
Sep 13, 2022
Webinar
FDA Rare Disease Innovation Hub: Enhancing Collaboration and Speed?
Jan 7, 2025
Podcast
Rare endpoints: Delivering on unmet patient needs
May 7, 2024
Article
Part 1 - Preparing for a Risk-Based Future: What ICH Revisions Mean for Clinical Trial Design and Conduct
Sep 14, 2021
Related Insights
Report
New Medicines, Novel Insights: Accelerating development of cell and gene therapies
May 22, 2023
Report
New Medicines, Novel Insights: Achieving patient-guided drug development
Oct 30, 2023
Video
Part 2: Risk-based Quality Management Video (RBQM) Series
Nov 11, 2021
Blog
Celebrating 40 Years of Rare Disease Progress: WODC Highlights
Jun 6, 2023
Blog
Adapting the validation process for PROs for rare diseases and other diseases with large unmet need and/or rapid progression
Apr 23, 2024
Blog
Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress
May 2, 2024
Podcast
Enabling Successful Sites, Episode 2: Empowering Sites to be More Inclusive Through Cultural Sensitivity Training
Jun 28, 2024
Podcast
Enabling Successful Sites, Episode 3: Taking the complexity out of oncology clinical trials
Jun 28, 2024
Blog
Including patients in DCT design
Sep 13, 2022
Webinar
FDA Rare Disease Innovation Hub: Enhancing Collaboration and Speed?
Jan 7, 2025
Podcast
Rare endpoints: Delivering on unmet patient needs
May 7, 2024
Article
Part 1 - Preparing for a Risk-Based Future: What ICH Revisions Mean for Clinical Trial Design and Conduct
Sep 14, 2021