Friday, May 9, 2025
10AM – 2PM ET
The Ritz Carlton
1150 22nd St NW, Washington, DC 20037
Conference will also be streamed virtually
Friends of Cancer Research (Friends) and the Parker Institute for Cancer Immunotherapy (PICI) are proud to announce our upcoming joint public meeting, Unlocking Next-Generation Therapies: Exploring Innovative Development and Manufacturing Models for Cell Therapies.
Cell therapies have demonstrated remarkable success in treating certain cancers and, given their personalized nature, hold particular promise for rare cancers and other difficult-to-treat diseases, especially when conventional therapies may be insufficient or unavailable. However, current manufacturing models often face challenges related to timeliness, accessibility, and production costs, which can be especially limiting for rare disease applications. Overcoming these challenges could help ensure that patients—particularly those with rare diseases—benefit from these promising therapies.
To explore solutions, Friends and PICI launched a multi-stakeholder working group to develop a practical framework for evolving manufacturing approaches. This includes decentralized models for cell therapies, as well as regulatory and cost recovery considerations to facilitate continued patient access and real-world evidence generation prior to full approval. This initiative will examine operational, safety, and quality control strategies to support manufacturing models that enhance flexibility while maintaining rigorous standards.
Through collaboration, this effort aims to identify sustainable approaches that expand the availability of cell therapies—particularly for patients with rare diseases—by enabling earlier access and developing scalable, adaptable manufacturing solutions. Check back for more details.
Draft Agenda
10:00 AM | Welcoming Remarks
10:05 AM | Morning Keynote
Session 1: Development and Reimbursement Models for Rare Disease Therapies
10:30 AM | Panel Discussion and Q&A
This session will explore the challenges in bringing rare disease therapies to patients and discuss potential solutions to address gaps in development and access. Panelists will examine models that could facilitate cost recovery and evidence generation in the pre-market space, while also discussing regulatory pathways that balance flexibility with ensuring sufficient data to support patient access.
11:45 AM | Lunch Break
12:15 PM | Lunch Keynote
Session 2: Manufacturing Models for Rare Disease Therapies
12:45 PM | Panel Discussion and Q&A
Manufacturing remains a critical challenge for advancing rare disease therapies, particularly when both low throughput and commercial viability are concerns. This session will explore proposals that could reduce upfront costs, opportunities to leverage partnerships, and scalable manufacturing solutions.
2:00 PM | Meeting Adjournment
Draft agenda is subject to change.
