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…

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.…

IVI’s Research Framework to Understand the Full Range of Economic Impacts on Patients and Caregivers

The Innovation and Value Initiative (IVI) and AcademyHealth have developed a consensus document titled “A Research Framework to Understand the Full Range of Economic Impacts on Patients and Caregivers” Direct Medical Costs: Patient or caregiver costs paid to a healthcare provider or healthcare system. Non-Clinical Healthcare Costs: Costs that are a direct result of seeking…

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…