Unbiased Analysis of Today's Healthcare Issues
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…
Standard cost-effectiveness analysis assumes that any gain in quality-adjusted life years (QALYs) should be valued equally. This does not sound unreasonable, but is it true in practice? Consider two potential violations of constant value of QALY gains: scope insensitivity and severity independence. I define each of these below: Scope insensitivity. This assumes that individuals value…
Cost effectiveness analysis aims to capture identify (and monetize) all of a treatments benefits, risk and costs and then determine if the treatment is worth the money. Traditionally, however, benefits, risk and costs only related to benefits that accrue to the health care system. Many studies in the academic literature have advocated for a societal…
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…
When evaluating a new diagnostic, HTA agencies must assess two separate issues: analytical and clinical validity. Analytical validity basically indicates whether the test works; is it able to accurately predicts the presence or absence of a particular biomarker of interest. Clinical validity is whether the test matters in clinical practice. It could be the case…
A nice (pun intended) paper by Thokala et al. (2020) compares the Institute for Clinical and Economic Review (ICER) with the National Institute for Health and Care Excellence (NICE) along 4 dimensions: structure, methods, process, and use in decision-making. While ICER and NICE methods are fairly similar, ICER is a non-governmental body without any explicit…
Is it clinical benefit? Cost? Value? The availability of treatment alternatives? To answer this question, a paper by Wranik et al. (2019) conducted a discrete choice experiment DCE to determine HTA stakeholders stated preferences. The sample consisted of HTA stakeholders from 5 countries: Australia, Canada, Germany, Poland, and the United Kingdom. The stakeholders included not…
Many health technology assessment (HTA) agencies give additional consideration to treatments if there is unmet need. But what really defines unmet need? According to an article by Vreman et al. (2019), there are three key elements. First, there should be no or limited treatment alternatives. If there are lots of good treatment options available, then…
In general, health economists would like to have health insurers cover treatments that are welfare improving in the Pareto sense. This means, if a treatment provides more expected benefits than costs and no one is worse off (in expectation), then this treatment should certainly be covered. It could be the case, however, that people care…
Health technology assessment (HTA) is growing in popularity. Already widely entrenched in Europe, in the U.S. value frameworks are being used to measure the cost effectiveness of different therapies. Some of these frameworks, however, take a narrow view of societal benefits and value to include components of value to patients or society such as caregiver…