The genomics revolution over the past three decades has led to great strides in rare disease
(RD) research, which presents a major shift in global policy landscape. While RDs are …

Background: Decision-makers have implemented a variety of value assessment frameworks
(VAFs) for orphan drugs in European jurisdictions, which has contributed to variations in …

Abstract Background and Objective Enthusiasm for the use of outcomes-based managed
entry agreements (OBMEAs) to manage uncertainties apparent at the time of …

Objectives With complex health technologies entering the market, methods for health
technology assessment (HTA) may require changes. This study aimed to identify challenges …

Context Positive health technology assessment (HTA) outcomes can have important
implications for equity, efficiency and timely patient access to novel therapies. Several …

Background The aims of this research were to provide a better understanding of the specific
evidence needs for assessment of clinical and cost-effectiveness of cell and gene therapies …

Conventional cost-effectiveness analysis—ie, assessing pharmaceuticals through a cost per
quality-adjusted life year (QALY) framework—originated from a societal commitment to …

Abstract Background The Canadian government has committed to developing a national
strategy for drugs for rare diseases starting in 2022. Considering this announcement, we …

Precision oncology has made remarkable strides in improving clinical outcomes, offering
hope to patients with historically difficult-to-treat, as well as rare or neglected cancers …

Access to medicines treating rare diseases ('orphan medicines') has proven challenging due
to high prices and clinical uncertainty. To optimise market access to these medicines, some …