Synergy in Action

16th Annual Meeting Working Group Session Descriptions

You may select one working group for each session.
Working Group Session A: (Wednesday, 4:25-6:00 pm)

Artificial Intelligence and Machine Learning for CNS Clinical Trials (Butler, Alphs)
Waste and inefficiency in drug development are big problems. Some experts believe that as much as 85% of biomedical research may be wasteful due to biases in study design, lack of publication, unnecessary duplication, or investigating questions of little importance. It is also estimated that only about one (or maybe two) of every 10 drugs that enter into clinical testing will turn out to be effective. These problems in drug development and clinical trial design can be hard to spot, especially when you are in the midst of the process. New approaches are needed to make the process of drug development more effective and efficient.
Application of machine learning and artificial intelligence to drug development offer great promise to make this possible, but many questions remain on how to do this. The AI/ML working group will be focused on continuing our efforts to determine guidelines and best practices for implementing AI and ML in clinical trials. The group will initially be focused on opportunities to use AI/ML in support of:
• Clinical Outcome Development
• Trial Enrichment
• Exploring Placebo Response
• Companion Diagnostics or Digital Biomarkers
• Trial Optimization
Work in this group will be to help address these and other issues and so shape more efficient and informative trials for the future.

Autism Spectrum Disorder (Mantua, Farchione, Arango)  Available to Manuscript Authors
The manuscript team will meet for further development of articles for a special issue of European Neuropsychopharmachology, a series of opinion papers on issues relevant for improving clinical trial methodology in ASD, addressing initiatives in the field to foster drug development.

Behavioral and Psychiatric Symptoms in Dementia (BPSD)
Agitation Subgroup (O’Gorman, Rosenberg)
The Alzheimer’s Disease Agitation Working Group of the BPSD working group will provide an update on the publication process and outcomes for the submitted ISCTM recommendations paper: “A Framework for Developing Pharmacotherapy for Agitation in Alzheimer’s disease”.  The meeting will provide the working group an opportunity to brainstorm and discuss next steps. 

Developing Integrated Interventions: Clinical Trials for Drug/Neuromodulation – Psychotherapy Combinations (Dunn, Yaseen)
Recent years have seen a steady increase in the new applications of somatic therapies that target symptom change in interaction with psychotherapeutic interventions. Examples range from use of deep repetitive transcranial magnetic stimulation combined with symptom provocation for obsessive compulsive disorder, to intranasal oxytocin combined with social cognition training for young people with early psychosis, to use of MDMA to enhance non-directive psychotherapy grounded in psychodynamic principles to treat adults with post-traumatic stress disorder.  In clinical trials settings such integrated interventions pose a variety of technical and conceptual problems. This innaugural meeting seeks to gather those who are currently investigating such integrated intervention approaches, to engage trialists who would like to investigate such approaches, and bring to the table those who are cautious or concerned about such approaches or their regulation, and begin an effort to better understand how integrated interventions can best be studied to support understanding of mechanisms, demonstrations of efficacy, and regulatory decision-making.

Estimands and Missing Data (Lim, Polverejan)  Available to Working Group Members and Session Speakers
A closed working group session will be held for the members of the ISCTM Estimands Working Group as well as the chairs, speakers and panelists of the parallel session on estimands. In this session, the attendees will have the opportunity to connect and discuss various topics related to estimands.

Prevention Trials in Alzheimer’s Disease (Posner, Harvey) 
This working group will be meeting to continue the writing of the two papers on Primary and Secondary prevention addressed at the last US meeting in February, 2019. There will be no planned presentations, but all pre-registrants will be provided with the latest drafts of the papers and will be welcome to participate in their finalization, targeted for before the Autumn 2020 meeting.

Working Group Session B: (Friday, 7:30-9:15 am)

Assessment Methods and Endpoints for Rapid-Acting Antidepressants-RAADs (Ballard, Yavorsky, Opler)
This workgroup is focused on evaluating the validity and reliability of current and prospective future methods of assessment of efficacy, effectiveness of the “next wave” of treatments in mood disorders, utilizing the rapid acting antidepressants (RAADs) as a model. Do the instruments developed to evaluate the efficacy of older generations of treatments (e.g. MAOIs, TCAs, SSRIs) meet the needs of this new class of treatments?
For this meeting, the RAAD workgroup will focus on the following:
– A review of data / findings from EFA performed by Dr. Elizabeth Ballard and colleagues on NIH studies of ketamine;
– Present and discuss results of an IRT analysis from a large RAAD Phase III trial;
– Review and critique a proposed consensus statement on the relative utility of select MADRS items for RAAD studies;
– Discuss findings on novel assessment methods and innovative technologies for RAAD trials.

Behavioral and Psychiatric Symptoms in Dementia (BPSD)
Apathy Subgroup (Lanctôt, Miller)
In July of 2019, key stakeholders including the leadership of the ISCTM BPSD WG, along with regulators, and academics from both N. America and the EU met at the AAIC meeting in Los Angeles. At that meeting, final wording was agreed upon for the newly revised diagnostic criteria for Apathy in the context of Cognitive Impairment. We aim to present the criteria and discuss next steps including operationalization with the larger BPSD WG.

Curriculum Development (Canuso)  Available to Curriculum Development Committee
The ISCTM/ASCP Curriculum Development Committee will meet to continue course development.

Innovative Technologies for Clinical Trials (Keefe, Davis)  Available to Working Group Members and Previous Attendees of WG
This working group is developing a white paper to describe key challenges in clinical trials that could potentially be addressed with new technological approaches, including placebo effects, assessment of clinical meaningfulness, and patient recruitment. The WG will proceed from work completed in the past two meetings. Since the WG is at capacity, in order to proceed effectively, only those members who have participated in one or both of the previous two meetings or working group calls may attend.

Orphan Diseases (Busner, Pandina, Anand)
Over the next year, the Orphan Disease Working Group will tackle the following areas, with a specific deliverable for each. During this WG session, we will focus discussion on two of the points. The remaining two will be addressed over the next months on a series of monthly calls. We will target the WG session at the Autumn Conference in Boston for updates from each of the 4 small groups for feedback from the full Working Group. Volunteers to spearhead each deliverable will be sought.
• Methods for obtaining stakeholder feedback on study design
• Methods for improving access to clinical trials in orphan disease
• Approaches to endpoint development and validation for Orphan Diseases – recent examples of data in the public domain with endpoints
• Best practices for training and standardizing assessments for orphan disease trials
The last 15 minutes will be devoted to refining the agenda of the upcoming 2 day Pediatric/Orphan Disease Autumn Session in Boston