Unbiased Analysis of Today's Healthcare Issues
That is a title of a new white paper from FTI Consulting and Alexion. The key findings from the article are: HTA practices are designed to be used for medical technologies and pharmaceuticals that treat commonly-occurring diseases, and are not fit to determine the value of orphan drugs for rare disease, which face unique challenges.…
Check out an event this Friday, December 10, at 10am EST/7am PST on at The Hill on Rare Diseases: A Conversation on Value Assessments. A summary of the topic and the speaker list are below. One in 10 Americans suffers from a rare disease—that’s 30 million Americans who need access to treatment. According o the NIH,…
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.…
A new drug comes on the market. Is the health benefit worth the cost? What is it’s value? Value assessment frameworks are one way to answer this question. Value frameworks take the evidence of the treatments benefits, risk and cost and output some notion of the treatment’s value. Ideally, if anyone applied a given value…
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…