The RI-CART Registry Project Summary
The number of children diagnosed with autism spectrum disorders is large and growing. The federal Centers for Disease Control and Prevention estimates that 1 in 88 U.S. children are born with Autism Spectrum Disorders (ASDs), conditions that involve minor social deficits to severe communication impairments. In Rhode Island, between the 1994-2011 school years, the number of children aged 3 to 21 identified with ASDs rose from 41 to 1,937 a nearly 50-fold increase in 17 years.
Rhode Island is poised to establish itself as a national leader in innovative, integrated research for ASDs. Rhode Island is well-positioned to lead due to:
A long tradition of comprehensive care for people and families with developmental disorders
A nationally representative number of children and teens diagnosed with ASDs
A broad bench of experts who conduct a variety of research studies genetic, imaging, behavioral, clinical, and epidemiological on ASDs and developmental disorders
A less complex, and more collaborative, system of autism treatment and research than other states, having one medical school and strong relationships between academia, government, educators, service providers, and families
A relatively stable population located within the smallest state in the nation, creating ideal conditions for large, long-term studies and for easy convening of research meetings and trial sessions, as well as training and outreach activities
RI-CART members believe that the best way to create healthier, happier lives for children with ASDs is to improve and expand knowledge about ASDs through widespread, rigorous testing; collaborative, creative research; and inclusive, data-driven education and advocacy. This strong belief in the power of knowledge, and the importance of partnerships, drives RI-CART's long-term goal of enrolling every child in Rhode Island with an ASD into a rich, rigorously-managed, nationally representative research registry. From the start, the registry will contain a detailed set of demographic, medical, treatment, and diagnostic data, including results of a professionally-administered Autism Diagnostic Observation Schedule, or ADOS, the "gold standard" autism assessment. Within five years, the registry will also include genetic and imaging data based on blood samples and brain scans. This registry would be the first in the nation to include such a varied, reliable, and representative sample of children and teens with ASDs.
By standardizing and improving diagnostic protocols for individuals with an autism spectrum disorder, by bridging disciplinary silos, and by providing an informatics infrastructure, RI-CART will be well-positioned, in the long term, to successfully compete for national funding that will establish a world-class ASD research enterprise in Rhode Island, one that serves as a national model and resource.
From established senior investigators to energetic postdoctoral fellows, RI-CART researchers have projects underway with the potential to reshape what is known about autism. However, investigators have been carrying out research individually using relatively small samples, laboriously obtained without the advantage of a robust, secure shared database. The first step in creating this registry, and enrolling patients in ASD studies, is a research diagnosis. Yet, the ADOS the assessment required in federally-funded research studies, is used in only a fraction of cases in Rhode Island, and across the country. Only a handful of Rhode Island psychologists and physicians use the ADOS assessment, and it's estimated that fewer than 10 percent of children with autism in the state are ADOS tested.
The first objective for RI-CART is to expand ADOS testing in Rhode Island and to create a Rhode Island autism research registry in order to significantly increase statewide collaborative research as well as serve as a national model and resource for autism research. Family participation in research and partnerships with investigators are key to a successful research enterprise. Providing support and education for the family based upon the diagnostic evaluation results is likely to enhance participation in the research registry. Thus RI-CART proposes to meet this objective by establishing a family support team of 3 highly skilled members: a Ph.D. level, ADOS certified psychologist, a Masters prepared Educational Specialist and a parent Outreach/Resource Specialist.
One of RI-CART's partners, Bradley Hospital, the nation's first psychiatric hospital for children and adolescents, will hire the psychologist to administer the ADOS at no cost. A total of 480 children, whose families express interest in autism-related research, will be enrolled over the course of the three-year proposed project. That's about one quarter of the estimated 1,800 Rhode Island children identified with ASDs. Once assessed and with informed consent established, children will be entered into the web-based registry, which all RI-CART researchers can use.
Another RI-CART partner, The Autism Project, the primary statewide parent advocacy group, will join with Bradley to recruit a parent outreach/resource specialist who will give families access to community resources appropriate for their child, and facilitate and support parents in enrolling their children in clinical trials, behavioral and epidemiological studies, and other research opportunities in Rhode Island and across the United States. A third RI-CART partner, The Paul V. Sherlock Center on Disabilities at Rhode Island College, a university-affiliated program in developmental disabilities will, with Bradley Hospital, hire the educational specialist for the family support team. The educational specialist will be available to meet with school personnel, at parents' request, to help develop appropriate educational objectives based on results of the ADOS and other diagnostic tools. The specialist will also enlist teacher and school administrator support to recruit children with ASDs for entry into the RI-CART registry.
Autism is a complex developmental disability that causes social interaction and communication problems. Symptoms usually start before age three and can delay or impede skills that develop from infancy to adulthood, including the ability to share and control emotions, start and hold a conversation, or display empathy.
In the U.S., prevalence of ASDs which include classic autism, Asperger syndrome, and Pervasive Developmental Disorder has increased dramatically in the last decade. The Centers for Disease Control and Prevention calls autism a significant public health problem. Yet the causes for ASDs aren't completely clear, genetic and neurobiologic mechanisms are only beginning to be understood, and there are no cures. There are no medical tests or biologic markers for ASDs, and parents frequently report conflicting diagnoses. Research also shows that services for children with autism are often inconsistent and uncoordinated. As a result, children and their families struggle socially, emotionally, educationally, and financially.
Project Goals and Objectives
RI-CARTs long-term goal is to enroll every Rhode Island child with an ASD diagnosis into a research-caliber, de-identified, secure registry from which accredited investigators can recruit subjects for large-scale interdisciplinary research projects.
To get there, the inaugural project has three goals with supporting objectives:
1. Expand ADOS testing in Rhode Island
Hire a full-time, ADOS-certified Ph.D. psychologist to administer at least 160 annual ADOS assessments (4 per week for 40 weeks/year) to children from member RI-CART clinical and education programs, with at least 480 children tested when the project is complete. Purchase testing kits, observation coding forms and a scoring CD needed to use the ADOS test. By agreeing to ADOS testing, families also agree to enroll their child in the research registry.
2. Create and populate an autism research registry
Purchase computer hardware and software needed for Lifespan IT staff, in consultation with RI-CART clinicians, to build a confidential, secure, ethical, expandable, web-based patient registry for research purposes. Hire a full-time research assistant to manage the registry database, which will include at least 480 children when the project is complete. The registry has several components: 1) A de-identified data set that contains ADOS results plus results from other key assessments and family/sociodemographic items; 2) A secure patient identifier key (using the GUID for future compatibility with national databases) with 4 levels of consent; and 3) A research project component that identifies which subjects are enrolled in which projects, which subjects drop out and which subjects were unable to participate.
Pre-certified RI-CART investigators will be able to query the database using pre-specified criteria derived from their individually funded, IRB-approved studies to recruit selected subjects that meet the query criteria. The subjects' level of consent will determine how the investigator contacts the family to solicit participation. The functional analysis specifications and initial set of variables for the RI-CART registry are attached as an Appendix.
3. Provide support to families and schools that will enhance research participation
Provide a full-time parent to serve as an outreach/resource specialist who supports participating families by linking them to appropriate community resources, advocate with them as necessary, and encourage participation in research projects by RI-CART investigators. The specialist will serve as the liaison between RI-CART and family members interested in research opportunities. Taking advantage of Rhode Island's compact size, and parents' desire to have direct contact with ASD experts, the specialist will conduct three major outreach activities across the state each year that bring families together with physicians, service providers, educators, academic experts and advocates who can answer questions, talk about key challenges in diagnosis and treatment, and explain research projects. These regional events are expected to create a community supportive of ASD research, and drive registry enrollment. The specialist will maintain contact with area diagnosticians, hospital staff, and state officials to ensure families are connected to resources quickly, gather and maintain information on resources appropriate for ASDs and related disabilities, and meet with families one-on-one as needed. It is projected that the outreach/resource specialist will individually consult with 240 families or 50% of those whose children are ADOS-tested under the proposed project. Joanne Quinn, executive director of The Autism Project, the primary parent advocacy program in Rhode Island, will supervise the specialist.
Hire a full-time educational specialist to meet with teachers and school administrators at parents' request to discuss the potential role of the ADOS and additional assessments for planning of a child's educational program and support services. The educational specialist will also lead teacher training workshops and, at parents' request, provide consultation to educational teams about use of assessment results in developing educational programming. Finally, the specialist will encourage school districts to recruit families to join RI-CART's registry. School district cooperation is critical for meeting RI-CART's long-term goal of enrolling all ASD families in Rhode Island. RI-CART expects that half the 480 children who enroll in the registry over the course of the project will be directly recruited from school districts. When the project is complete, the specialist will have worked in all 26 Rhode Island school districts. Amy Grattan, from the Paul Sherlock Center on Developmental Disabilities at Rhode Island College, and Sue Constable of the Rhode Island Technical Assistance Program, will jointly hire and supervise the education specialist.
Project Impact & Future Directions
At the end of the three-year project, RI-CART will have significantly expanded Rhode Island's capacity for collaborative and innovative autism research. With a large-scale registry to seed basic science, clinical and epidemiological studies, and with a multi-disciplinary, multi-institutional team to execute this research, Rhode Island will be well-positioned to successfully pursue follow-on funding from government agencies, private foundations, non-profit advocacy groups, and individual donors. For example, RI-CART members would, at project's end, apply for a five-year, $2M Centers for Biomedical Research Excellence award from the National Institutes of Health to establish shared, statewide facilities for use in basic, clinical, and epidemiological research projects. This major infrastructure award will foster a variety of new research projects, including studies to test the efficacy of educational interventions based on ADOS results, and/or to test clinical and home-based interventions that use strategies derived from ADOS performance.
The project would also bolster existing national research efforts. The registry would be compatible with, and feed into, the National Database for Autism Research, an NIH-funded secure data repository to accelerate data sharing and collaboration among ASD researchers. RI-CART would also like to join the International Autism Epidemiological Network, an initiative of the Centers for Disease Control and Prevention and Autism Speaks that supports registry trials around the world. In addition, RI-CART's parent outreach/resource specialist will direct participants into publicly and privately funded ASD research studies across the country.
The RI-CART model of collaborative, interdisciplinary research and sustained, comprehensive family support can not only serve as a resource and model for ASD research nationally, but also serve as a model for the investigation of other diseases and conditions in defined geographic catchment areas. Gathering data comprehensively and systematically will also informs public policy and improve treatment for children with ASDs and their families.