Health Technology Assessment (HTA) is a multidisciplinary process that uses explicit methods to assess the value of a health technology at different points in its lifecycle with the aim to inform decision-making to promote an equitable, efficient, and high-quality health system. While these systematic appraisal processes can make decision making for most medical technologies easier and more transparent, technologies aimed at rare diseases, such as “orphan drugs,” present unique challenges to the HTA process. This class of drugs often carries high costs, aims to fill an unmet demand, and often lacks clear comparators. Other aspects such as disease severity, data constraints, and small population size all require payers and assessors to take special approaches to value assessment.
This course will outline the unique situation that orphan drugs pose to health technology assessment processes and will teach the participant different protocols and criteria for conduct, citing real-world cases globally. Issues surrounding evidence requirements, cost-effectiveness, and stakeholder value judgment will be addressed.