Valuing treatments for rare disease

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”…

The Carer QALY Trap

The term “Carer QALY” was coined in a paper by Mott et al. (2023) and is identified as the case where “it is possible for an effective treatment that provides survival gains (with relatively little or no QOL gain) to appear less effective than the comparators when carer QOL is considered.” While the term “Carer…

Recommendations for incorporating equity into HTA evaluation

Many health policy experts–including myself–have noted that treatments that help reduce health disparities may be especially valuable whereas those that exacerbate inequalities may be somewhat less valuable than predicted by standard cost-effectiveness analysis. A key question is, health disparities over what dimension(s)? Is it race? Income? Education? O’Nell et al. (2013) developed the PROGRESS framework.…

Why you should include dynamic drug pricing in your CEA model

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…

CMS = HTA?

The U.S. is one of the the few developed nations without a government-run health technology assessment (HTA) body. Or are they? A recent perspective in the New England Journal of Medicine by Peter Neumann and Sean Tunis argues that the Centers for Medicare and Medicaid Services (CMS) already is serving as a de facto HTA…

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…