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…

ICER’s 2022 “Barriers to Fair Access Assessment” Protocol

How does ICER plan to determine whether health plans offer fair access to pharmaceuticals? I summarized their approach last year as outlined in their white paper titled “Cornerstones of ‘Fair’ Drug Coverage: Appropriate Cost-Sharing and Utilization Management Policies for Pharmaceuticals.” Last month, final protocol for their “Barriers to Fair Access Assessment“. While the approach follows…

ICER’s updated process for determining unsupported price increases (UPI)

Last month, ICER updated their protocol for identifying drugs that have unsupported price increases (UPI). Not only is ICER examining rising drug prices, but California and Vermont now have laws tracking substantial drug price increases, requiring drug manufacturers to submit information that might justify increases above a certain threshold. ICER says that the UPI reports…

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…