Unbiased Analysis of Today's Healthcare Issues
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”…
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.…
Let’s say that you have an international clinical trial that shows a new drug (SuperDrug) perform better than the previous standard of care (OldDrug). Also assume that individuals with a specific comorbidity–let’s call it EF–respond less well to the SuperDrug treatment. If you live in a country where comorbidity EF is common, how well do…
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…
On September 25, ICER released its 2023 value assessment framework. What is new? What stays the same? Here are a few key points you need to know. Special cases for ultra-rare and single or short-term therapies (SST): These did not change from previous. Adaptations for ultra-rare disease (from Jan 2020) Single or short-term therapies (from…