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 to consider, such as the domains on the ISPOR Value Flower, and he provided examples from Sweden, Scotland, and NICE who incorporated other considerations outside of QALYs. For example, Drummond noted that NICE considered “modifiers for innovation, magnitude of treatment benefit, severity, health inequality, and uncertainty.” On the topic of QALY shortfall, Drummond illustrated how NICE applied a severity modifier to address proportional and absolute QALY shortfall. These modifiers may be helpful to assess gene therapy, but it is unclear how these modifiers are used.

Sean Sullivan noted that most HTA agencies do not use special methods for evaluating gene therapies.

He added that, “Novel valuation strategies should be considered to encompass the contextual consideration and other benefits of gene therapy in patients with rare, severe, debilitating, hereditary disease.” Sullivan shared that the ISPOR Value Flower has several elements that should be considered (eg, incorporating severity of illness, accounting for equity, quantifying insurance value, quantifying hope, quantifying option value). According to Sullivan, treatments for low-severity illness are likely overvalued by a factor of >2 and treatments for high-severity conditions are likely undervalued by a factor of >5. He also pointed out that using alternatives to QALYs may address the equity issue. Equal Value of Life (EVL) and Health Years in Total (HYT) may offer advantages over the QALY.

The full V&O spotlight article is here.