New FDA regulatory initiatives improving rare disease drug development
Listen to Parexel's SVP of Global Regulatory Strategy Mwango Kashoki, M.D., M.P.H., who is also a former FDA regulator, on how the FDA's new "plausible mechanism pathway" aims to advance individualized therapies like gene editing for serious rare diseases with no adequate treatments.
Building on recent CRISPR approvals and Rare Disease Evidence Principles, this promising development for drug innovation has the healthcare community eagerly awaiting further details to fully realize its potential for patients with very rare genetic conditions.
Related Insights
Article
China’s rare disease drug development landscape: Overview and insights for global biopharmaceutical companies
Oct 22, 2026
Webinar
FDA Rare Disease Innovation Hub: Enhancing Collaboration and Speed?
Jan 7, 2025
Podcast
Rare endpoints: Delivering on unmet patient needs
May 7, 2024
Blog
Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress
May 2, 2024
Blog
Celebrating 40 Years of Rare Disease Progress: WODC Highlights
Jun 6, 2023
Report
New Medicines, Novel Insights: Advancing rare disease drug development
May 22, 2023
CTB Blog
EMA’s new pre-submission interactions model pilot: A fundamental shift toward submission readiness and predictable reviews
Sep 30, 2026
Case Study
Speed meets strategy: Delivering a critical FDA 1572 package in just seven days
Sep 9, 2026
Blog
Actioning the commitment letter: The PDUFA VIII changes that matter most to sponsors
Aug 25, 2026
Blog
Data without borders: Assessing China-only evidence for EMA approval – insights from ex-EMA and seasoned subject matter experts
Aug 19, 2026
Blog
The Next Frontier of ADC Development: Navigating an Evolving Regulatory Landscape
Aug 12, 2026
Blog
Japan: the new strategic launchpad for accelerating global rare disease development
Jul 23, 2026
Related Insights
Article
China’s rare disease drug development landscape: Overview and insights for global biopharmaceutical companies
Oct 22, 2026
Webinar
FDA Rare Disease Innovation Hub: Enhancing Collaboration and Speed?
Jan 7, 2025
Podcast
Rare endpoints: Delivering on unmet patient needs
May 7, 2024
Blog
Accelerating Delivery and Patient Access to Rare Disease Treatments – Highlights from World Orphan Drug Congress
May 2, 2024
Blog
Celebrating 40 Years of Rare Disease Progress: WODC Highlights
Jun 6, 2023
Report
New Medicines, Novel Insights: Advancing rare disease drug development
May 22, 2023
CTB Blog
EMA’s new pre-submission interactions model pilot: A fundamental shift toward submission readiness and predictable reviews
Sep 30, 2026
Case Study
Speed meets strategy: Delivering a critical FDA 1572 package in just seven days
Sep 9, 2026
Blog
Actioning the commitment letter: The PDUFA VIII changes that matter most to sponsors
Aug 25, 2026
Blog
Data without borders: Assessing China-only evidence for EMA approval – insights from ex-EMA and seasoned subject matter experts
Aug 19, 2026
Blog
The Next Frontier of ADC Development: Navigating an Evolving Regulatory Landscape
Aug 12, 2026
Blog
Japan: the new strategic launchpad for accelerating global rare disease development
Jul 23, 2026