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.

Explore the report

 

Return to Insights Center

Related Insights

Report

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

May 22, 2023

Video

Part 2: Risk-based Quality Management Video (RBQM) Series

Nov 11, 2021

Blog

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

May 2, 2024

Blog

Adapting the validation process for PROs for rare diseases and other diseases with large unmet need and/or rapid progression

Apr 23, 2024

Report

New Medicines, Novel Insights: Achieving patient-guided drug development

Oct 30, 2023

Blog

Celebrating 40 Years of Rare Disease Progress: WODC Highlights

Jun 6, 2023

Article

Bringing clinical trials to patients with the decentralized model

Jan 8, 2021

Webinar

China's Market Approval Policy and Medical Insurance Payment System for Rare Disease

Jul 21, 2022

Article

Three ways to work with the FDA for better patient-focused trials

Jul 29, 2021

Whitepaper

Overview of China's Market Approval Policy Med Insurance Payment System

Apr 7, 2022

Article

Part 1 - Preparing for a Risk-Based Future: What ICH Revisions Mean for Clinical Trial Design and Conduct

Sep 14, 2021

Article

EU Orphan Drug Designation – overcoming regulatory challenges

Jul 20, 2022