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…