What is ‘Bayesian Hierarchical Modelling’ and how can it be used to evaluate oncology treatments studied in basket trials?

Should payers cover a new oncology treatment targeting specific biomarkers across multiple tumor types? One the one hand, one could require a separate trial for each tumor type. While this would be convincing evidence, it also is very expensive to conduct clinical trials for every tumor type, particularly if treatment efficacy is homogenous across tumor…

Methods for Including Adverse Events in Economic Evaluations

That is the title of a great review paper by Salah Ghabri, Dalia Dawoud and Michael Drummond. They discuss that guidance on the incorporation of adverse events (AEs) into economic models has not been standardized. Nevertheless, this paper provides helpful guidance. Specifically, the paper explores: (1) the incorporation of AEs in economic decision models, (2)…

HTA and cell and gene therapy

A paper by Drummond et al. (2023) examines how health technology assessment bodies deal with the challenge of value assessment for cell and gene therapies (also known as Advanced Therapy Medicinal Products (ATMPs) in Europe). To do this the authors: …undertook i) a targeted review of the literature on the clinical and economic evidence needs…

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.…