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

Autism Speaks submitted a nomination on treatments for autism spectrum disorders and it was forwarded to an EPC who was refining a related topic nomination. Three primary areas of the Autism Speaks nomination will not be covered in the current review and are resubmitted in rank order of priority. This revised nomination is based on a discussion with the nominator, the EPC, and the TOO after the Key Questions for the current review were finalized.

1. For children with autism spectrum disorder (ASD), does evaluation and treatment of commonly associated medical conditions (such as sleep disorders, dietary and/or nutritional deficiencies, GI conditions, and allergies) improve the effectiveness of behavioral interventions in reducing core symptoms? What are the most effective treatments, components of treatments, or combinations of treatments to treat these medical comorbidities? (Ib and III of original nomination, current HRSA review may answer some of these questions)

2. What are the components and the effectiveness of treatments designed to develop vocational and employment related skills of adolescents and young adults with ASD transitioning to work and college? At what age should these treatments begin? (IV c and d of the original nomination)

3. For children with suspected autism spectrum disorders younger than 24 months, what is the effect of behavioral interventions on early signs of autism? What are the modifiers of outcomes for these children? (key trials in this area are in progress, so delaying a review in this area will allow them to be published).

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:

1. treatments, components of treatments, or combinations of treatments to treat medical comorbidities associated with ASD

2. treatments, components of treatments, or combinations of treatments to develop vocational and employment related skills in ASD

3. treatments, components of treatments, or combinations of behavioral treatments on very young children with early signs of ASD

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.)

Children and adults of any age with suspected or diagnosed ASD

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

1. any age individual with medical comorbidities associated with ASD

2. adolescents and young adults with milder forms of ASD transitioning to work and college

3. children younger than 24 months with suspected ASD

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

1. improved symptoms associated with sleep disorders, dietary and/or nutritional deficiencies, GI conditions, and allergies as well as core symptoms of ASD such as social skills/interaction, language and communication, repetitive and other maladaptive behaviors, psychological distress, adaptive independence/self care, interpersonal relationships/community involvement

2. academic and vocational skills and abilities

3. reduction in core symptoms suggestive of ASD

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

Adverse behavioral or psychosocial reactions to behavioral or other therapies (e.g. increased aggression or anxiety)

Regression of language, skills, or behaviors

Increases in/worsening of co?morbid symptoms

Adverse reactions to drug therapies (e.g. somnolence, weight gain)

Reduction in/negative influences on quality of life

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
• Functional limitations and disability

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

• Medicaid
• State Children's Health Insurance Program (SCHIP)

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. (From prior Autism Speaks nomination.)

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.)

These topics were identified as the three largest priorities for review after discussion with the nominator from Autism Speaks following posting of the Key Questions for the current review of behavioral interventions for ASD.

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. (From prior Autism Speaks nomination.)

Potential Impact

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

A systematic review of the literature will provide objective information on the benefits and potential harms of available interventions and assist patients, their families and their providers in selecting optimal care. The disability associated with ASD is lifelong and the costs associated with care high, so these decisions are difficult and the consequences significant for both individuals with ASD and their families. The variation in care across providers also should decrease as evidence?based recommendations for best treatment practices become available.

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

As soon as possible for questions 1 and 2. As soon as relevant research is published for question 3.

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. (From prior Autism Speaks nomination.)

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)

A systematic review of the literature will provide objective information on the benefits and potential harms of available interventions and assist patients, their families and their providers in selecting optimal care. The disability associated with ASD is lifelong and the costs associated with care high, so these decisions are difficult and the consequences significant for both individuals with ASD and their families. The variation in care across providers also should decrease as evidence?based recommendations for best treatment practices become available.

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

Organization

Please tell us how you heard about the Effective Health Care Program