Unbiased Analysis of Today's Healthcare Issues
That is the subtitle of a paper by Vreman et al. (2020). The authors look at all treatments approved by FDA and EMA that were subsequently reviewed by HTA bodies in both jurisdictions between 1995 and 2018. The authors used ICER as the US HTA body; European HTA bodies included IQWiG (Germany), NICE (UK), ZIN…
“I’d like to see Generalized Cost Effectiveness Analysis become the norm for how we assess value.” – Rep. Jake Auchincloss (D-MA 4th District) See the full interview here. To learn more about GCEA, check out the GCEA User Guide published last year.
When conducting evidence synthesis, many individuals use the PICO framework. PICO stands for population; intervention; comparator(s); and outcome. This helps to describe the research question that the evidence synthesis aims to answer. However, recently Remiro‐Azócar (2024) proposed PICOSI framework. PICOSI stands for: Population Intervention Comparator(s) Outcomes Summary effect measure; Intercurrent events In fact, Canada’s Drug…
The answer is ‘yes’, but the question is in what manner? A paper by Che et al. (2024) examines the use of real-world data (RWD) across 2016-2023 HTA assessments. Over this time period, 11% of HTA submissions included real-world data. Further, they find that: The main sources of RWD considered in the submissions were disease…
Meng Li (2024) has a nice summary of the generalized cost effectiveness analysis (GCEA) user guide paper (see Shafrin et al. 2024) that was published last month. The GCEA methodology encompasses 15 ‘petals’ across four domains. The uncertainty domain captures how risk aversion and uncertainty in treatment outcomes impact value, including aspects such as outcome…
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…
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…
Why is it so hard to estimate the value of orphan drugs indicated for the treatment of rare diseases? There are a variety of reasons, but a scoping review by Grand et al. (2024) provides a nice summary of these issues. Key challenges include small sample sizes for nearly all parameters and lack of data…
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…
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.…