Unbiased Analysis of Today's Healthcare Issues
Progression-free survival (PFS) is a surrogate endpoint is defined as follows (by ChatGPT): In essence, PFS refers to the length of time during a clinical study or treatment regimen where a patient’s disease doesn’t worsen or progress. For instance, in cancer research, it measures the time from the start of treatment until the cancer shows…
While robust data is preferred when parameterizing economic models, this information is not always available. For instance, it may be unclear the degree to which clinical trial results would translate to the real world. Or, clinical trial results may need to be extrapolated beyond the time period of the trial. Or, there may simply be…
That is the topic of a recent white paper from the Innovation and Value Initiative titled “Valuing Rare Disease Treatments in Healthcare: Real Experience, Real Impact“. The report notes a number of challenges in assessing treatment value in rare disease (see my white paper “Challenges in Preserving Access to Orphan Drugs Under an HTA Framework”…
A Health Affairs Forefront paper by researchers Melanie Whittington, Peter Neumann, Joshua Cohen, and Jonathan Campbell makes a compelling case for incorporating drug price dynamics into cost effectiveness analysis. The first questions many people may have is ‘how do drug prices usually change over time?’ A drug’s net price often increases following launch and may…
In the United Kingdom, the National Institute for Health and Care Excellence (NICE) made an update to their health technology evaluations manual in January 2022. Of particular interest, section 6.2 of the manual states that the review committee “will consider the associated absolute and proportional QALY shortfall.” The committee defined QALY shortfall two ways: Absolute…
Standard quality approach compares the difference in QALYs which is a function of survival and quality of life during each survival period. Consider the comparison of treatments A and B, where treatment A is the novel treatment and treatment B is the standard of care. Under the QALY based approach this is calculated as Where…
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…
The New York Times has a very interesting piece on the caregiver experience. Americans spend so much time debating so many aspects of health care, including insurance and access. Almost none of that covers the actual impossibility and hardship faced by the many millions of friends and family members who are caregivers. It’s hugely disrupting…