On May 25, more than 95 participants gathered in Boston for ASBMT’s “Value Assessment in Engineered T Cell Therapies,” an invitation-only forum to share perspectives on value assessment frameworks that will be needed for engineered T cell therapies. Attendees reflected the diverse stakeholder groups required to determine how this novel therapy can best be integrated into available therapeutic options, including researchers, clinicians, health economists, hospital administrators, payers, and patient advocacy groups.
ASBMT President Krishna Komanduri, MD welcomed the group, reviewed the agenda, and then introduced the first speaker, James Kochenderfer, MD, of the National Cancer Institute. Dr. Kochenderfer explained that T cells are genetically engineered to produce special receptors on their surface -chimeric antigen receptors (CARs) -that allow them to recognize and target tumor cells. He reviewed both outcomes and toxicities of CAR T cells in clinical trials for patients with relapsed and refractory leukemia and lymphoma.
Jonathan Gruber, PhD, Ford Professor of Economics at the Massachusetts Institute of Technology (MIT), gave a provocative keynote presentation about the health economics and reimbursement of innovative therapies. Gruber is popularly known as an architect of Obamacare and its 2006 predecessor, the Massachusetts health care reform. His views on “risk sharing” for the economic feasibility of the plans have been hotly debated.
Next, Mary Horowitz, MD, Chief Scientific Director of the Center for International Blood and Marrow Transplant Research (CIBMTR), presented a thoughtful review entitled, “Outcomes Analyses in Engineered T Cell Therapies.” She noted the long history of the CIBMTR in effectively capturing data for the conduct of observational research, demonstrated the challenges of collecting data for the purposes of identifying the cost/value of cellular therapies, and showed attendees the progress already made in developing CIBMTR forms for cellular therapy data capture.
In the afternoon, Lou Garrison, PhD, President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and Adrian Towse, MA, MPhil, of the Office of Health Economics (UK), led a presentation and group discussion entitled “Value Assessment Frameworks and T Cell Therapies.” These assessments will be influential in determining which therapies are chosen by doctors and patients, as well as how the cost of therapies that will be covered. Four existing frameworks were outlined and attendees held small group discussions to determine the pros and cons of using each framework for T cell therapies. The groups reported that, while none of the existing frameworks completely meets the needs of T cell therapies, the frameworks may serve as platforms for building a value assessment framework that does.
The day ended with a “stakeholder” panel discussion about implementation of T cell therapies in clinical settings and a “payer” panel discussion in which value determinations were discussed in more detail.
Dr. Komanduri concluded the meeting and spoke about next steps. The ASBMT Value and Health Economics Special Interest Group will be authoring a “white paper” as a follow-up to the forum to serve as a foundation for continuing discussions in developing a value assessment framework for T cell therapies.
The ASBMT would like to give a special thank you to our corporate partners: Kite Pharma, Novartis, Juno Therapeutics, MaxCyte, Atara Bio, Adaptimmune and Celgene Corporation. Without their support, this meeting would not have been possible.
Read the entire June 2017 ASBMT eNews here.