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.…
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.…
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…
Standard cost-effectiveness measures the value of a treatment based on the cost for an additional unit of health where units of health are measured in quality-adjusted life years (QALYs). For Alzheimer’s disease, however, this approach may be limited. A paper by Garrison et al. (2021) identifies the petals of the value flower that are particularly…
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…
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…
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…
Clinical trials are typically of (relatively) short duration, but innovative treatments may impact patient survival over multiple years. Health economists and outcomes researchers often are faced with the challenge of extrapolating clinical trial survival curves to estimate long-term survival gains for the typical patient. These estimates may be done parameterically (e.g., exponential, Weibull, Gompertz, log-logistic,…