Testimony on Comparative Effectiveness Research
The full completed topic nomination form has been submitted to the Effective Health Care Program and will appear in the Effective Health Care Program Web site public reading room at http://effectivehealthcare.ahrq.gov/getInvolved.cfm?involvetype=rs.
In the area of autism research, the vast majority of clinical trials conducted to date have addressed only the questions of how an individual treatment compares to a placebo. Extremely few studies have been conducted that make head-to-head comparisons of two or more treatments. Indeed, in reviewing the list of existing Effective Health Care Program funded projects, it is noteworthy that none addresses autism spectrum disorders (ASD). Given the very high prevalence of ASD and the fact that ASD is a life-long condition that costs the US approximately $35 billion annually, it is crucial that research identifying the most effective treatments of ASD be conducted. The following are some of the high priority questions that such research could address:
- Understanding the combined effectiveness of medical and behavioral treatments for improving outcomes for individuals with ASD. To date, there have been few, if any, studies that have examined the comparative effectiveness of psychopharmacological or other medical treatments when they are administered alone versus combined with behavioral/psychosocial interventions for reducing core or associated symptoms. The following are examples of projects that are needed:
- For individuals with ASD who present with severe aggression and irritability, what is the comparative efficacy of a pharmacological treatment versus a pharmacological treatment combined with functional behavioral analysis versus functional behavioral analysis alone for reducing such challenging behaviors?
- For children with ASD whose condition is not improving in response to behavioral intervention, what is the comparative effectiveness of conducting a comprehensive medical evaluation to detect and treat commonly associated medical conditions (e.g. sleep disorder, dietary and/or nutritional deficiencies, GI conditions, and allergies) versus continuing behavioral intervention without such medical assessments and interventions?
- Understanding the key effective ingredients of early behavioral intervention. Tremendous financial, feasibility, and accessibility challenges are involved in the provision of early intensive behavioral intervention for young children with ASD. Yet, no studies to date have examined the comparative effectiveness of key elements of early behavioral intervention. Among the high priority questions that need to be addressed are:
- What is the comparative effectiveness of lower versus higher "doses" of early behavioral intervention (e.g. 15 versus 25 hours per week; 2 versus 4 years) for improving long term outcome?
- What is the comparative effectiveness of early behavioral intervention implemented by highly trained professionals (e.g. Ph.D. Psychologist) versus parents for reducing autism symptoms and improving language, cognitive, and social abilities?
- What is the comparative effectiveness of two different models of early behavioral intervention (for example, traditional applied behavior analytic early intervention programs versus developmental behavioral early intervention programs) for reducing autism symptoms and improving language, cognition, and social behavior in children with ASD?
- For children with ASD who fail to develop communicative speech in response to early behavioral intervention, what is the comparative effectiveness of continuing with traditional early intervention approaches versus adding computer-assisted communication augmented devices for improving communication skills?
- Treating commonly associated medical conditions in ASD. ASD is commonly associated with medical conditions, such as sleep, GI, psychiatric, and other co-morbidities, that have a significant impact on quality of life. Yet, little is known regarding the most effective treatments to address such co-morbidities. The following are examples of the types of studies that need to be conducted:
- What is the comparative effectiveness of cognitive behavioral treatment versus cognitive behavioral treatment plus a pharmacological treatment versus pharmacological treatment alone in reducing depressive or anxiety symptoms in adolescents and adults with ASD?
- For individuals with ASD presenting with sleep difficulties, what is the comparative effectiveness of sleep hygiene program versus a combination of sleep hygiene with melatonin versus melatonin alone in reducing sleep-related problems?
- Assessment of the comparative effectiveness of currently implemented community/school based treatment/educational programs. There are many treatment/educational programs that are currently being utilized in the community. Although individually many of these programs have been shown to be effective for promoting positive outcomes, there have been no studies that have compared whether one program is more effective than another. Examples of such programs include:
- Early behavioral intervention programs administered through the public schools or private agencies
- Programs for promoting social, language, and cognitive skills for school age children
- Programs to facilitate transition to adulthood, focusing on vocational and employment related skills
- Programs designed to promote success for high functioning adults with ASD attending college