ASSESSING THE VALUE OF RARE DISEASE TREATMENTS- GLOBAL LESSONS FROM RECENT CASE STUDIES
Author(s)
Discussion Leaders: James Wu, MSc, MPH, Principal Health Economist, Global Health Economics, Amgen, Thousand Oaks, CA, USA Clifford Goodman, PhD, Senior Vice President and Director, Center for Comparative Effectiveness Research, The Lewin Group, Falls Church, VA, USA; Louis P. Garrison, PhD, Principal, Strategic Pharmacy Innovations, Seattle, WA, USA; Paul Melmeyer, MPP, Director of Federal Policy, National Organization for Rare Disorders (NORD), Washington, DC, USA
Presentation Documents
PURPOSE
: Therapies for rare conditions that utilize novel mechanisms of action, such at CAR-T or gene therapies, present unique challenges to value assessment. This workshop will examine the contemporary economic evaluation of high-cost therapies for rare diseases (RD) by probing 3 case examples of recent value assessments, followed by audience voting. The voting results will then be discussed within the context of global value assessment.DESCRIPTION
: Dr. Garrison will present recent recommendations from the ISPOR Task Force on Value Frameworks and their implications when applied to RD1. Participants will learn about the challenges of assessing the value of RD therapies, with a focus on how conventional cost-effectiveness analyses are limited by the disease and the evidence (i.e., incomplete understanding of natural history of disease and appropriate endpoints, small sample sizes, and single-arm trials). Paul Melmeyer will present the patient perspective, highlighting factors not well represented in traditional and newly proposed value assessment frameworks, and those especially relevant to RD patients, such as the severity of disease, lack of therapies, benefit-risk trade-offs, and the value of hope. Also addressed will be the societal relevance of cost-effectiveness thresholds. Dr. Goodman will present three case studies of contemporary value assessments for RD therapies: CAR-T therapies, hemophilia A, and inherited retinal disease, focusing on three products Kymriah™ (Novartis), Hemlibra® (Genentech), and Luxturna™ (Spark), respectively. Each will highlight the assessment approaches taken in the UK, Germany and US (including the long-term value assessment by the Institute for Clinical and Economic Review). The audience will then vote electronically on (1) the relevance and impact of non-conventional elements of value, (2) an appropriate cost-effectiveness threshold, and (3) the long-term value of each therapy (high, medium, or low). Results will be discussed with a focus on deriving insights on essential elements of value and feedback for advancing global value assessment.Conference/Value in Health Info
2019-05, ISPOR 2019, New Orleans, LA, USA
Code
W8