Challenge in HTA for gene therapies

Last month, the Office of Health Economics published a report titled “Health Technology Assessment of Gene Therapies: Are Our Methods Fit for Purpose?” I summarize some of the key challenges and solutions below. Challenge #1: Initial assessment of clinical effectiveness. Since gene therapies often target rare disease, the sample size from clinical trials is often…

Gene therapies and the ISPOR value flower

What considerations beyond quality adjusted life years and cost should be considered when evaluating gene therapies? A panel from ISPOR provided just such a discussion. As summarized an ISPOR’s Value and Outcomes Spotlight article titled “Can Pharmaceutical Pricing Move Beyond Cost QALY for Value Consideration?“, Michael F. Drummond pointed out that: …there are other values…

Coverage with evidence development for medical devices in Central and Eastern Europe

The value of medical devices caries with it less certainty than pharmaceuticals for a variety of reasons.  As described in Kovács et al. (2022) medical devices: often have multiple applications, frequently, undergo product modifications and during their product lifecycle, multiple incremental technological innovations take place affecting both clinical and economic consequences of their adoption into…

NICE’s new Severity Adjustment

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…

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.…

Is the value of a QALY constant?

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…