Archives: Agenda

The master protocol approach is transforming the landscape of drug development by allowing multiple treatments to be evaluated across different disease states or patient populations within a single trial structure. An approach like this can offer significant advantages, including increased efficiencies, faster decision-making and improved coordination but poses challenges to operationalization without the proper planning and support. ​In this session, explore the key aspects of master protocols, it’s benefits and challenges, and provide practical insights into implementation and operationalization.

Whether you are a researcher, clinician, or part of the pharma industry, this session will equip you with the knowledge to leverage master protocols effectively in your clinical research. ​

Key Takeaways: ​

• Understand the benefits, optimal use cases and best scenarios for master protocols. ​
• Maximize trial operations with improved coordination and more sub-study opportunities. ​
• Enhance data collection and sharing, broadening patient access to new therapies. ​
• Navigate strategic relationships for safe and successful master protocol research.

• Identifying and creating criteria that addresses exactly what your study needs to make the selection process easier
• Strengthening relationships with CROs by communicating effectively throughout your study
• Understanding and assessing CROs capabilities tailored to your specific trial needs to avoid running into roadblocks

Apple Prize Draw taking place in exhibition room – please ensure your cards are stamped and submitted by 1:15pm

• Comparing outcomes of a DCT model versus non DCT model
• Sharing benefits and lessons learned from experiences within rare disease DCTs

Rare disease innovation has always required both scientific courage and regulatory flexibility. Today, under the Trump Administration’s FDA, sponsors are seeing new opportunities for accelerated approvals, digital biomarkers, and innovative endpoints—but also greater scrutiny around confirmatory trials and uncertainty over key incentives like the pediatric voucher program. Meanwhile, political turbulence at FDA and CDC leadership levels underscores just how intertwined policy and science have become.