Comparative Effectiveness

What is wrong with QALYs?

A paper by Rand and Kesselheim (2021) in Health Affairs this month conducts a systematic literature review to answer this question. Based on 113 articles they identified in peer-reviewed journals, they identify the following 10 criticisms categories. The graph above has each criticism category and the number of peer-reviewed articles that mention this critique type. …

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Do most Medicare Part D drugs have CEA studies?

That is the question that a recent study by Tisdale et al. (2021) aim to answer. They first look at the top 250 Medicare Part D drugs in terms 2016 total spending based on the 2016 Medicare Part D Prescriber Public Use File. Drug characteristics were identified from the 2016 FDA Orange Book. The authors …

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Technology assessment in rare diseases

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 disease must not only be rare but also severe, often …

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What kind of HTA does the US need?

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 single payer system and NICE’s recommendations can be directly implemented …

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PCORI to incorporate cost but not cost-effectiveness in its new principles

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 Act, however, has expanded the scope of the types of …

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The need for continuous cost-effectiveness analysis

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 a case study of how cost-effectiveness analysis can change over …

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