Unbiased Analysis of Today's Healthcare Issues
If you are a payer or a policymaker, should you except the results of a global clinical trial or require additional evidence that a drug works in your country. There are a few options to consider. Delay approval until local confirmatory trial. Approach enacted in several Asian countries (see Habr et al. 2024). While this…
That is the title of a helpful article by Karnon and Haji Ali Afzali (Pharmacoeconomics 2014) with a subtitle A Review and Critique of the Costs and Benefits of DES. The paper starts by highlighting the 3 most common types of models: decision trees, Markovian cohort models, and individual level models. Decisions trees are useful…
That is the question asked in a new paper by Enright, et al. (2025). The authors use the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database current as of August 2023. This database contains publicly available specialty pharmacy coverage decisions as issued by 18 large US commercial health plans, which—collectively—cover 200m people (i.e.,…
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…
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)…
If a child dies during childbirth, should the impact of the child’s death on parental quality of life be considered? What about interventions that prevent miscarriages; should the health of the fetus be considered or only that of the mother? A paper by Lamsal et al. (2024) conducts a systematic review to examine which individual’s…
A message from the Innovation and Value Initiative (IVI) Center for Innovation & Value Research CEO Jason Spangler. I was the former Director of Research at IVI and I wish them luck with the new name as they continue with their mission to “To advance the science, practice, and use of patient-centered Health Technology Assessment…
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…
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”…