The CARS Scale to Assess Pharmacological Therapies in Autism Spectrum Disorder: A Targeted Literature Review
Author(s)
Schmid R1, Beillat M1, Breau-Brunel M2, Ethgen O3
1Servier International, Suresnes, France, 2Amaris, Barcelona, Spain, 3University of Liège, Liège, Belgium
OBJECTIVES The Childhood Autism Rating Scale (CARS) is a standardized, comprehensive, and reliable observational rating scale of Autism Spectrum Disorder (ASD). It has been cited by the European Medical Agency (EMA) as a scale used for assessment of symptoms in ASD and a potential scale to measure treatment-related changes in the ASD core symptoms. This study aims at documenting how the CARS has been used to assess clinically relevant response to investigational drug treatments in clinical trials and observational studies. METHODS We conducted a targeted literature review to identify studies assessing at least one pharmacological intervention with the CARS (or its second version the CARS-2) in a pediatric population with ASD. The Embase® database was searched over the 2000-2018 time span. This electronic search was followed by hand searches. All searches were conducted in June 2019. RESULTS In total, 507 abstracts were screened for eligibility. Nineteen studies were included in our review. Fourteen (74%) studies were randomized trials and 10 (53%) studies enrolled less than 50 patients. Together, the studies focused on a total of 9 pharmacological interventions, including secretin, risperidone and bumetanide. The CARS was the primary outcome measure in 5 studies (26%). Among those 5 studies, 4 specifically used the CARS to assess treatment response, and 2 suggested to interpret a score variation beyond -4 points from baseline as clinically significant. Yet, no study purposely justified a clinically relevant response definition with a Minimal Clinically Important Difference (MCID) for the CARS. CONCLUSIONS The CARS was used in a quarter of the few studies that assessed a pharmacological intervention in ASD. Despite this and the recent recognition by the EMA of the CARS merit in assessing effect on ASD core symptoms, our review indicates the need to define a clinically meaningful treatment response and a MCID for the CARS.
Conference/Value in Health Info
2021-05, ISPOR 2021, Montreal, Canada
Value in Health, Volume 24, Issue 5, S1 (May 2021)
Code
PND1
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
Neurological Disorders, Pediatrics