Minutes of the Interagency Autism Coordinating Committee (IACC) Subcommittee for Basic and Translational Research Strategic Plan Question 4 Planning Group, Conference Call on September 26, 2013
The Interagency Autism Coordinating Committee (IACC) Subcommittee for Basic and Translational Research Strategic Plan Question 4 Planning Group convened a conference call on Thursday, September, 2013, from 1:37 p.m. to 3:15 p.m.
In accordance with Public Law 92-463, the meeting was open to the public. Thomas Insel, M.D., Chair, presided.
Thomas Insel, M.D., Chair, IACC, National Institute of Mental Health (NIMH); Susan Daniels, Ph.D., Executive Secretary, IACC, Office of Autism Research Coordination (OARC), NIMH; Idil Abdull, Somali American Autism Foundation; Anshu Batra, M.D., Our Special Kids; Tiffany Farchione, M.D., U.S. Food and Drug Administration (FDA); Catherine Rice, Ph.D., Centers for Disease Control and Prevention (CDC) (representing Coleen Boyle, Ph.D., M.S. Hyg.)
Roll Call and Opening Remarks
Dr. Susan Daniels welcomed the Planning Group and members of the public and called roll. She said that this Planning Group was charged with the update for Question 4 of the IACC Strategic Plan (Which Treatments and Interventions Will Help?). She noted that goal of this call of the Planning Group was to evaluate the status of the Question 4 objectives, by reviewing the funding and project information documents prepared by the Office of Autism Research Coordination (OARC).
Discussion of Question 4 IACC Portfolio Analysis Documents and Strategic Plan Progress
Dr. Daniels reviewed these materials, all of which are available online. The materials included: a Compiled Objective Chart for Question 4 (2008 to 2012), an IACC Strategic Plan Funding Summary Sheet for Question 4, and Project Lists for Question 4 from the IACC Portfolio Analyses (2008-2012). She briefly explained the process used to develop the Portfolio Analyses. For the Compiled Objective Chart, objectives were roughly categorized into a "stoplight" chart. Green highlighting indicates that funding fully met the recommend budget. Yellow highlighting denotes that funding for a particular objective partially meets the IACC recommended budget, while red highlighting indicates that there has been no funding /projects towards the particular objective. She noted that the Portfolio Analysis data provided for 2011 and 2012 is still subject to minor changes because the report has not yet been finalized.
Dr. Daniels clarified that the Strategic Plan is an advisory document that advises the agencies of the priorities identified by the IACC, but that the agencies are not bound to the project number and funding recommendations for the objectives in the IACC Strategic Plan. Also, she reminded the group that the IACC recommended budgets for the objectives represent the minimal amount of funding that the IACC thought would be needed to accomplish the objectives (a floor). She said that the Strategic Plan did not limit the agencies to only funding projects and programs that are responsive to the Strategic Plan. Dr. Thomas Insel noted that the Compiled Objective Chart showed that there had been no funding or projects for objective 4.S.E. (Convene a workshop to advance the understanding of clinical subtypes and treatment personalization (i.e., what are the core symptoms to target for treatment studies) by 2011). He said that he believed that Autism Speaks had sponsored similar meetings in 2011 and one that was sponsored by the Foundation for NIH in 2012. He asked that OARC check this information and make any needed updates to the information.
There was a brief discussion of why objective 4.S.B (Standardize and validate at least 20 model systems (e.g., cellular and/or animal) that replicate features of ASD and will allow identification of specific molecular targets or neural circuits amenable to existing or new interventions by 2012) was included under Question 4. Dr. Daniels explained that the Question 4 group in 2009 had discussed the need for animal models, so the objective ended up in Question 4. Dr. Insel also explained that animal models were important for the identification of biomarkers that could be used in drug development. Dr. Batra expressed concern that she perceived an imbalance between funding for objective 4.S.B. that supports basic research and for objectives that are more practically relevant to families.
Ms. Idil Abdull said that research spending was lacking for the development of methods to teach nonverbal children and individuals with ASD to communicate (including electronic/augmentative devices). Dr. Daniels said that they could get an estimation of the funding on technology-based interventions in Question 4 from the information provided to them. This was likely to be largely communications devices. Dr. Insel reminded the Group that they had a project list and they could take a closer look at projects related to a topic. Dr. Batra said that more funding also was needed for alternative and nutritional treatment approaches, instead of behavioral treatments.
Dr. Insel said that the purpose of this update of the Strategic Plan was to evaluate the progress achieved on existing objectives; new objectives would not be added. He noted that the objectives with the most funding were not necessarily the ones with the most progress.
Ms. Abdull said that parents are most interested in treatments that work. Dr. Insel said that many of these treatments are in development or are being evaluated through randomized trials. It would take time before effective, evidence-based treatments could be recommended to parents and clinicians.
Dr. Daniels suggested that the Group review the levels of funding for each Question 4 objective, based on the stoplight indicators from the Compiled Objective Chart for Question 4 and identify areas that need more funding/research. They could also review the list of projects for the objectives to get a better sense of how funds have been spent.
Dr. Batra re-emphasized the imbalance between funding for objective 4.S.B and other objectives. Dr. Insel noted that part of this process was to identify gap areas. Dr. Batra also raised the question of how they could distinguish which trials/projects were for children or adults. Dr. Insel said that sometimes this information was available from the titles or summaries. Dr. Daniels noted that OARC had provided project lists that the Group could review for some of this information.
Dr. Insel noted that there was a large number of projects, but that many of the projects were small in funding amount. Since large trials are often the ones that provide the most definitive results, he suggested that perhaps many of the projects were inadequately powered to provide meaningful results. Dr. Tiffany Farchione said that at the FDA, they see a lot of small pilot studies and very few larger-scale pivotal studies. Dr. Insel said that they needed to consider whether any of the funded projects had resulted in concrete and useful results. Perhaps they could recommend funding fewer projects, but providing each project with a larger funding amount to increase the chances of trials providing meaningful results.
Ms. Abdull and Dr. Batra both expressed concern that there has not been enough funding/projects on evaluating the efficacy of existing treatments (the focus appears to be on target identification and development of new treatments). The Group looked at specific projects related to treatments for minimally verbal individuals. Ms. Abdull said that more research was needed in this area – on facilitating communication for nonverbal kids. The Group agreed that this could be identified as a gap area in the Question 4 text. Dr. Daniels said that the Planning Group's task was to draft a description of funding needs or accomplishments. She summarized the Guidance for the 2013 IACC Strategic Plan Updating Process. (PDF – 100 KB)
Dr. Daniels noted that they could also identify barriers to research on specific objectives. They could look up the specific projects and/or ask the invited experts who would be joining the next call why certain gaps exist. Dr. Insel noted that in general they have met or exceeded the number of projects for many objectives but the recommended funding has not been met.
Dr. Batra said that perhaps they are not targeting the right factors to assess, particularly early sensory and motor issues, in order to intervene early. Dr. Insel agreed that it is important to adequately assess risk factors in order to target potential interventions. He noted that there are two projects in 2012 that are underway for objective 4.L.B (Develop interventions for siblings of people with ASD with the goal of reducing the risk of recurrence by at least 30% by 2014). It was considered appropriate to classify progress on this objective as "yellow." Dr. Insel said that it was difficult to develop interventions for individuals at risk unless they could be identified. It is necessary to understand the trajectory of autism.
With regard to objective 4.L.D (Support at least five community-based studies that assess the effectiveness of interventions and services in broader community settings by 2015. Such studies may include comparative effectiveness research studies that assess the relative effectiveness of: Different and/or combined medical, pharmacological, nutritional, behavioral, service-provision, and parent-or caregiver-implemented treatments; Scalable early intervention programs for implementation in underserved, low-resource, and low-literacy populations; and studies of widely used community intervention models for which extensive published data are not available.
Outcome measures should include assessment of potential harm as a result of autism treatments, as well as positive outcomes), Dr. Batra said the number of projects appears to be adequate. However, the objective is very broad. She said that individual projects do not appear to evenly address all of the aspects of this objective. She suggested reviewing the specific projects under this objective.
Wrap-Up and Next Steps
Dr. Insel noted that many projects had no funding listed in certain years. Dr. Daniels said that this may be due to projects where all the funding for a multiyear project was allocated in the first year, which is the practice of some agencies and organizations. Dr. Insel questioned whether some projects were designed with enough rigor to disprove an intervention. Dr. Batra said that it is important to first identify the subtypes of autism in order to identify and test treatments. She said that the projects listed do not cover the scope of interventions in community.
Dr. Batra noted that for objective 4.S.A (Support at least three randomized controlled trials that address co-occurring medical conditions associated with ASD by 2010), there too few comorbidities under investigation (only three in 2012). This group should focus on treatments. Ms. Abdull agreed that this was an important topic.
Dr. Insel volunteered to draft a summary document for the Group to review and suggest changes. He said that this discussion had raised two concerns. The first was the overall balance of the objectives. The second was the number of projects relative to amount of funding, as it appeared that many studies may be underpowered.
The conference call was adjourned at 3:15 p.m.
I hereby certify that this meeting summary is accurate and complete.
/Susan Daniels/ November 12, 2013
Susan A. Daniels, Ph.D.
Executive Secretary, Interagency Autism Coordinating Committee