PURPOSE: To discuss challenges and opportunities in measuring patient preferences for treatment-process attributes to support the evaluation of gene therapy.

DESCRIPTION: Health technology assessment agencies often prefer that utilities used to calculate quality-adjusted life years are derived using standardized methods, such as generic preference-based measures completed by patients in clinical trials. However, there are situations when no standardized approach is feasible or appropriate for a specific treatment-process attribute, while distinct treatment-process attributes for gene therapies receive increasing attention in treatment preferences.

Dr. Garrison will introduce health utility measures. (5 minutes) and moderate the discussion.

Mark Skinner will discuss how the patient-centric approach is used in designing key treatment attributes for gene therapy in hemophilia. The core outcome set for hemophilia gene therapy (coreHEM) includes method and frequency of administration, mental health, chronic pain, and annual bleeding rate. (15 minutes)

Dr. Shi will discuss common methods of vignette-based utilities and discrete choice experiments with time tradeoff (DCE-TTO) utilities for treatment-process in gene therapies. His introduction will benefit the audience to understand new developments, nuances, opportunities, and challenges in measuring utilities for gene therapies. He will further provide a recently completed patient preference study using DCE-TTO among patients with hemophilia A as a case study. In addition to the DCE attributes based on the coreHEM, he will specifically describe the process of deriving the utility value of durability of gene therapy for hemophilia. (15 minutes)

Dr. Campbell will discuss ICER’s perspective and experience in including treatment-process utility in their considerations in ICER’s recent assessments of gene therapies. (15 minutes)

We will have a Q&A session at the end of the session. (10 minutes

The workshop will use real-time polling to work through the examples and conclude with an audience discussion about the opportunities and challenges of using DCE-TTO in the evaluation of gene therapies.