PURPOSE: Breakthrough therapies for rare conditions using novel mechanisms of action, e.g., CAR-T and CRISPR/Cas9, present unprecedented challenges to value assessment. This workshop will examine the application of current economic evaluation methods for emerging high-priced therapies for rare diseases, and discuss whether these methods are fit for purpose or require evolved paradigms. The audience will participate in interactive discussion, reflecting on and responding to distinct value perspectives of the health economist, patient, and decision-maker. DESCRIPTION: Dr. Garrison will present current practices from a health economics lens. Participants will learn about methodological challenges of assessing therapies for rare conditions i.e., unmet need, disease burden, direct and indirect costs (including productivity losses), conventional cost-effectiveness thresholds, budget impact, value of innovation, and shortcomings in evidence, i.e., small sample sizes, single arm study design, outcome selection, fatal disease course, and incomplete understanding of natural history of disease. Paul Melmeyer will present a patient perspective, conveying factors that may not be captured in traditional value assessments, i.e., rarity of disease, availability of alternative therapies, patient-reported outcomes, benefit-risk trade-offs, and value of hope. Also addressed will be societal relevance of cost-effectiveness thresholds Dr. Goodman will discuss challenges of decision makers who see expensive emerging therapies for an increasing number of rare diseases. While traditionally payers and providers have based their decisions on rigorous evidence, they must now recalibrate their assessment of value while facing limited evidence of effectiveness and safety, different trade-offs of pre- and post-market data collection, shifting societal expressions of willingness to pay, emerging value-based payment options, and heightened cost-containment pressures. With this background, presenters will share early experiences and outlook for value assessment for the diverse pipeline of “million dollar therapies” for rare conditions, with implications for innovation for unmet medical needs. Audience discussion will embrace related international developments, including low/middle-income countries.