What is a rare disease? The definition varies across countries, but according to the European Medicines Agency (EMA) rare diseases are those with a prevalence of <5 cases per 10,000 people; ultra-rare diseases are those with <1 case per 50,000 people. In most cases, rare… Read More »Technology assessment in rare diseases
Should the US have a formal, centralized health technology assessment (HTA) organization? In the United Kingdom, the National Institute for Health and Care Excellence (NICE) plays this roles and has a significant impact on drug pricing in the UK. However, the UK relies on a… Read More »What kind of HTA does the US need?
Created by the Affordable Care Act (ACA) in 2010, the Patient-Centered Outcomes Research Institute (PCORI) has funded a wide variety of research comparing different treatments to one another. One thing PCORI was forbidden from funding, however, was cost-effectiveness analyses. Section 1181 of the Social Security… Read More »PCORI to incorporate cost but not cost-effectiveness in its new principles
At drug launch, treatments have some benefit and cost. However, drug prices may change over time. Additionally, treatment benefits may rise or fall depending on how well efficacy translates into effectiveness and how well physicians learn to manage adverse events. Mattingly and Love (2020) provide… Read More »The need for continuous cost-effectiveness analysis