A planning committee of the National Academies of Sciences, Engineering, and Medicine will organize and conduct a public workshop to examine barriers in access for cell-based gene therapies. The overarching goal of the workshop is to explore current challenges that limit the accessibility, affordability, and delivery of cell-based gene therapies, and to discuss promising opportunities to accelerate access to these therapies for patients.
The workshop may include invited presentations and panel discussions to:
• Examine the current landscape of access to cell-based gene therapies, including economic, logistical, and infrastructural barriers that affect patients, clinicians, and health systems, and highlight lived experiences that illustrate real-world challenges to delivering these therapies.
• Explore how a variety of factors shape access and affordability, which may include regulatory pathways, reimbursement, workforce capacity, clinical site readiness, geographic obstacles to accredited service and treatment centers, and manufacturing models, and identify how decisions made early in therapy development can influence scalability and patient reach.
• Learn about existing and emerging groups working on patient access to these therapies and understand the factors that prevent many eligible patients from receiving these therapies.
• Consider lessons learned from both successful and unsuccessful efforts to expand access (e.g., alternative funding and payment models), manage long-term follow-up, and consider how these experiences can inform future strategies (e.g., novel public-private partnerships, platform technologies).
The planning committee will organize the workshop, develop the agenda, select and invite speakers and discussants, and moderate or identify moderators for the discussions. A proceedings-in-brief of the workshop presentations and discussions will be prepared by a designated rapporteur in accordance with institutional guidelines.