Wednesday, May 21, 2025
12PM – 1PM ET
Online (Zoom)
What are some challenges impeding the broad development of engineered cell-based therapies?
Friends of Cancer Research (Friends) is hosting a webinar, “Unlocking Next-Generation Therapies Meeting Recap: Sharing Next Steps and Opportunities with Advocates,” on May 21, 2025, as a follow-up to the upcoming public meeting titled, “Unlocking Next-Generation Therapies,” co-hosted by Friends and Parker Institute for Cancer Immunotherapy (PICI).
This webinar will revisit key takeaways from the recent public meeting and explore unique challenges currently impeding the progress and broad availability of engineered cell-based therapies for rare diseases and small patient populations. Panelists will examine key barriers to patient access, such as how limitations in current manufacturing systems and regulatory hurdles contribute to limited clinical availability of these therapies. Panelists will explore how alternative manufacturing approaches, including decentralized and point-of-care models, can help address these challenges and improve delivery of engineered cell-based therapies. Please see below for the agenda and essential questions for advocates. We encourage you to submit questions in advance for the live Q&A.
Agenda
12:00 PM – Welcoming Remarks
- Mark Stewart, Friends of Cancer Research
12:02 PM – Panel Introduction & Discussion
- Moderator: Natasha Kekre, Ottawa Hospital Research Institute
- Kristen Hege, Independent Board of Directors Member
- Patrick Hanley, Children’s National Hospital
- Holly Fernandez Lynch, University of Pennsylvania
12:25 PM – Audience Q&A Session
12:59 PM – Closing Remarks
- Mark Stewart, Friends of Cancer Research
1:00 PM – Meeting Adjourned
Attendees are encouraged to submit question(s) regarding the topics covered in the webinar.
Click the button below to submit your question before the event. Additional event details can be found below.
Essential Questions for Advocates
- What policy opportunities exist to overcome current barriers and ensure that patients, even those with rare diseases, can access the potentially transformative benefits of engineered cell-based therapies?
- Considering the complexities of manufacturing and development of engineered cell-based therapies, especially for small populations, how can expedited development pathways such as Breakthrough Designation and Regenerative Medicine Advanced Therapy (RMAT) designation support the timely delivery of these therapies to patients who can benefit from them?
- What kinds of regulatory flexibilities are needed to facilitate the development of engineered cell-based therapies in small, rare-disease populations while ensuring timely access, quality, and safety for patients
Background
Engineered cell-based therapies have demonstrated remarkable success in treating certain cancers and, given their personalized nature, hold promise for rare cancers and other difficult-to-treat diseases, especially when conventional therapies may be insufficient or unavailable. However, current development and manufacturing models often face challenges related to regulatory uncertainty, timeliness, accessibility, and production costs, which can be especially limiting for rare disease applications. Overcoming these challenges will be necessary to create a sustainable pathway that ensures 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 advancing regulatory and manufacturing approaches that can enable timely access to promising therapies without compromising quality or safety. This includes regulatory flexibilities for rare disease populations, reimbursement considerations to help recover development costs while facilitating sustained patient access to promising therapies, and alternative manufacturing approaches, including point-of-care models to enable timely therapy development, particularly for rare or hard-to-treat diseases. Together, these solutions aim to facilitate sustainable approaches and innovative policies that expand the availability of engineered cell-based therapies.
