Briefly describe a specific question, or set of related questions, about a health care test or treatment that this program should consider.

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:

1. 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:
   1. 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?
   2. 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?
2. 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:
   1. 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?
   2. 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?
   3. 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?
   4. 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?
3. 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:
   1. 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?
   2. 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?
4. 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:
   1. Early behavioral intervention programs administered through the public schools or private agencies.
   2. Programs for promoting social, language, and cognitive skills for school age children
   3. Programs to facilitate transition to adulthood, focusing on vocational and employment related skills
   4. Programs designed to promote success for high functioning adults with ASD attending college

Does your question include a comparison of different health care approaches? (If no, your topic will still be considered.)

yes

If yes, explain the specific technologies, devices, drugs, or interventions you would like to see compared:

See #1 above

What patients or group(s) of patients does your question apply to? (Please include specific details such as age range, gender, coexisting diagnoses, and indications for therapy.)

1. Understanding the combined effectiveness of medical and behavioral treatments

• Applies to individuals with ASD throughout the lifespan

Understanding the key effective ingredients of early behavioral intervention

• Applies to children with ASD less than 6 years of age

Treating commonly associated medical conditions in ASD

• Applies to individuals with ASD throughout the lifespan

Assessment of the comparative effectiveness of currently implemented community/school based treatment/educational programs

• Applies to individuals with ASD throughout the lifespan

Are there subgroups of patients that your question might apply to? (For example, an ethnic group, stage or severity of a disease.)

Describe the health-related benefits you are interested in. (For example, improvements in patient symptoms or problems from treatment or diagnosis.)

Outcomes include (1) reduction in ASD core symptoms, (2) language, cognitive, and social abilities, (3) reduction in associated medical conditions and symptoms, such as sleep, GI, and psychiatric disorders, (4) improvement in quality of life and vocational outcome.

Describe any health-related risks, side effects, or harms that you are concerned about.

Potential risks include adverse or side effects from medications

Appropriateness for EHC Program

Does your question include a health care drug, intervention, device, or technology available (or likely to be available) in the U.S.?

yes

Which priority area(s) and population(s) does this topic apply to? (check all that apply)

EHC Priority Conditions (updated in 2008)

• Developmental delays, attention-deficit hyperactivity disorder, and autism

AHRQ Priority Populations

• Children
• Individuals with special health care needs, including individuals with disabilities or who need chronic care or end-of-life health care

Federal Health Care Program

None

Importance

Describe why this topic is important.

ASDs are a group of severe developmental disabilities characterized by lifelong impairments in communication and social interaction. Many individuals with ASD never speak or live independently, have severe cognitive disabilities, and suffer a wide range of associated medical conditions. ASD is a highly prevalent disorder, estimated to occur in 1 out of every 150 individuals. The total annual societal per capita costs of caring for and treating a person with ASD in the U.S. are estimated to be $3.2 million and approximately $35 billion for the entire birth cohort of people with ASD.

What specifically motivated you to ask this question? (For example, you are developing a clinical guideline, working with a policy with large uncertainty about the appropriate approach, costly intervention, new research you have read, items in the media you may have seen, a clinical practice dilemma you know of, etc.)

I am writing on behalf of Autism Speaks as the Chief Science Officer for the organization. As the nation’s largest autism science and advocacy organization whose mission is to promote autism awareness, increase services, and fund biomedical research on the causes, prevention, treatments, and cure for autism, Autism Speaks has a strong stake in the priorities of the Agency for Healthcare Research and Quality.

Does your question represent uncertainty for clinicians and/or policy-makers? (For example, variations in clinical care, controversy in what constitutes appropriate clinical care, or a policy decision.)

yes

If yes, please explain:

The high priority research questions described above represent areas in which very little is known about best clinical practices. As a result, clinical care for individuals with ASD varies greatly across care providers, and clinicians must make important health care decisions that will have tremendous impact on outcome with little guidance or knowledge. Lawmakers are struggling with how to make the best decisions about policy and funding due to lack of an adequate knowledge base regarding what are the most effective treatments. Parents, similarly, are left on their own to make decisions about their children’s health care and, as a result, often fall victim to misinformation because of the void of empirically-tested effective treatments.

Potential Impact

How will an answer to your research question be used or help inform decisions for you or your group?

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). As more clinicians are faced with the challenges of caring for individuals with ASD, it is essential that evidence-based recommendations for best treatment practices become available. Thus, studies that can identify the most effective treatments for improving outcomes for individuals with ASD are sorely needed. Such studies would have wide-ranging effects on clinical practice.

Describe the timeframe in which an answer to your question is needed.

Answers to the questions outlined above are urgently needed.

Describe any health disparities, inequities, or impact on vulnerable populations your question applies to.

Individuals with ASD and their families represent a highly vulnerable and underserved population. The World Health Organization considers factors such as prevalence, functional impairment, chronicity, age of onset, and cost in determining the health burden of a disease. Using the WHO criteria, autism’s burden (Disability Adjusted Life Year) is higher than Type 1 Diabetes, Childhood leukemia, and Cystic Fibrosis, among other childhood illnesses. A recent study on the health care experience of individuals with ASD and their families demonstrated that, compared to other children with special health care needs, children with ASD are more likely to have unmet needs for health care and family support services, have difficulty receiving appropriate medical referrals and coordinated health care, and have families that experience more financial problems, are required to reduce or stop work to care for their child, and spend over 10 hours a week providing special health care for their child. Many of these burdens could be addressed if knowledge about best practices and the most effective medical treatments was available. To this end, comparative effectiveness research that can address this knowledge gap is critically needed.

Nominator Information

Other Information About You: (optional)

Please choose a description that best describes your role or perspective: (you may select more than one category if appropriate)

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). As more clinicians are faced with the challenges of caring for individuals with ASD, it is essential that evidence-based recommendations for best treatment practices become available. Thus, studies that can identify the most effective treatments for improving outcomes for individuals with ASD are sorely needed. Such studies would have wide-ranging effects on clinical practice.

Are you making a suggestion as an individual or on behalf of an organization?

Organization

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