Pro92 Estimating Disutilities in Spinal Muscular Atrophy Using a Stated Preference Survey: A Uk General Public Study

Abstract

with and without PNH. Full-texts (no date restriction) and conference abstracts (2017 or later) of clinical trials or observational studies conducted in the US or Western Europe that examined patient-reported fatigue or associations between fatiguescales and hemoglobin were included. Data (i.e., relationship between improvements in fatigue-scales and clinical outcomes; clinically meaningful fatigue-scale thresholds) were extracted using a standardized form. Results: Among 161 search results, 16 peer-reviewed publications (4 [25%] PNH and 12 [75%] cancer studies) were included. Four clinical trials conducted in patients with PNH receiving ravulizumab or eculizumab reported that patients achieved and sustained clinically meaningful improvements in fatigue (i.e., $3 points). However, these studies did not examine the association between fatigue and hemoglobin. Twelve studies conducted in patients with cancer (anemic; with or without chemotherapy) demonstrated a statistically significant association between increased hemoglobin and improvements in fatigue (p,0.05). Two studies showed the greatest incremental gain (i.e., .3 mm in Linear Analog Scale Assessment) in improvement in fatigue was observed when hemoglobin increased from 11-12 g/dL. Conclusions: Substantial evidence in non-PNH literature (i.e., anemia of chronic disease) demonstrates that increased hemoglobin levels are associated with clinically significant improvements in fatigue (e.g., an increase of $3 points as measured by Functional Assessment of Chronic Illness Therapy-Fatigue). Future studies establishing this connection should be further validated among patients with PNH.

Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA)

“…Other challenges for economic evaluations in SMA that could be addressed in the future include lack of consideration for patient preference regarding treatment benefit versus risk, potential for improvement in particular clinical domains (e.g. motor function, respiratory function) and treatment attributes such as route of administration and age indications [83,84].…”

Systematic Literature Review to Identify Utility Values in Patients with Spinal Muscular Atrophy (SMA) and Their Caregivers

“…In total, this search identified four full-text publications [46][47][48][49] and one conference abstract and associated poster [50] that reported HSUVs for the population of interest. The updated search conducted in March 2021 yielded an additional nine publications, including five full-text publications [51][52][53][54][55] and four abstracts [56][57][58][59]; three abstracts had an associated poster [56,57,59]. Across the original and updated SLR search, 17 studies considering other (non-SMA) neuromuscular disorders and 463 studies reporting HRQoL data only (i.e.…”

“…The final list of 14 publications that met the eligibility criteria to be included in the SLR (Fig. 1) consisted of nine full-text publications [46][47][48][49][51][52][53][54][55] and five abstracts [50,[56][57][58][59]; four of the abstracts had an associated poster [50,56,57,59].…”

“…Countries/regions from which utility data were derived included (Table 3) Australia (n = 1) [53], Canada (n = 2) [54,58], Germany (n = 1) [52], Spain (n = 1) [47], the UK (n = 4) [46,56,57,59], and Europe, including studies covering France, Germany, Spain, and the UK (n = 2) [49,50], and France, Germany, and the UK (n = 1) [55]. Two publications derived global utility data using a database (n = 1) [51] and a global clinical trial (CHERISH; NCT02292537) (n = 1) [48].…”

“…The studies included the following patient populations (Table 3): patients with Types 1, 2, and 3 SMA (n = 9) [47, 49-51, 53-55, 58, 59]; Types 1 and 2 SMA (n = 1) [46]; Type 1 SMA (n = 1) [48]; Types 2 and 3 (non-ambulant) SMA (n = 1) [57]; and Types 2, 3, and 4 SMA (n = 1) [52]. One study did not report SMA type because the study consisted of a DCE in which a sample of the UK population was surveyed about SMA health outcomes and health burden [56].…”