There is a lack of high-quality health state utility value (HSUV) data for spinal muscular atrophy (SMA), according to a recent systematic literature review published in PharmacoEconomics.

Researchers found that none of the 14 studies identified met the requirements of the Health Technology Assessment (HTA) bodies for the UK, US, or Canada —mostly due to the use of country-specific tariffs. One met the requirements for the UK and Canada, and 6 studies could not be assessed because they did not report their method of valuation.

All studies included did highlight the substantial burden SMA places on the health-related quality of life for patients and their caregivers.

Read more about SMA prognosis.

The review also identified utilities for other neuromuscular disorders that may provide useful surrogate values for studies of SMA and other rare diseases.

“The primary objective of this systematic literature review (SLR) was to identify published HSUVs associated with patients with SMA and their caregivers, with a secondary objective to broaden the scope to identify HSUVs associated with other (non-SMA) neuromuscular disorders,” the authors said.

According to the article, HSUVs are used by HTA bodies to calculate quality-adjusted life-years (QALYs) and subsequently the incremental cost-effectiveness ratio used to make healthcare resource-allocation decisions. With the approval of several disease-modifying therapies (DMTs), the development of SMA-focused HSUVs is critical for HTAs to be able to make cost-benefit assessments.

“The absence of robust utility data highlights the importance of global, regional, and/or local data collection platforms and disease registry networks and supports a recommendation for early planning in future clinical trial design to help generate utility data for economic evaluations in SMA,” the authors stressed.

Studies included in the review involved patient and/or proxy assessment of health states including questionnaires, structured forms of expert elicitation such as discrete choice experiments, vignette approaches including case history reviews, and clinical trial data.


Sutherland CS, Hudson P, Mitchell S, Paracha N. Systematic literature review to identify utility values in patients with spinal muscular atrophy (SMA) and their caregivers. Pharmacoeconomics. Published online December 15, 2021. doi:10.1007/s40273-021-01115-5