HTA Quarterly | Summer 2017

While health technology assessments (HTAs) aim to produce policies that balance clinical benefit alongside the increased expense, the entity ultimately most affected by these decisions is the patient. Patient and patient advocacy groups are increasingly laying pressure on regulatory and HTA agencies to have more input into the decision-making process. This article will provide a brief snapshot as to how Australia, Canada, England, and Scotland involve patient or patient groups in product assessment decision making.

Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease that affects only a small number of mostly adolescent males, but the rare disease is making international headlines as new treatments are stimulating current discussions on evidentiary requirements, pricing, and access of pharmaceuticals. In this article, we review the burden of DMD, treatment, and disease management approaches in development, and we examine recent regulatory and health technology assessment (HTA) appraisals.

Colombia is the second-most populated country in South America after Brazil. The Colombian health sector has been in transition since bold healthcare reform was introduced in 1993, experiencing an increase of overall life expectance and a decreased infant mortality rate. We examine key market considerations of Colombia's unique healthcare model.