14th Annual Meeting Working Group Sessions
(Selecting the working group session link will direct you to specific working group’s page.)
ADAPTIVE DESIGN
Chairs: Judith Kando, PharmD; Ron Marcus, MD
The workshop will discuss a recent, innovative adaptive design study methodology ‘platform trials‘ that has been implemented in the European Prevention of Alzheimer’s Disease Study (EPAD).
ADDRESSING METHODOLOGICAL CHALLENGES IN INTERNATIONAL CNS CLINICAL TRIALS
Chairs: Amir Kalali, MD; Elizabeth Pappadopulos, PhD
Keeping with the focus on critical challenges facing international clinical trials, this work group will finalize our next topic and objectives.
ALGORITHMS/FLAGS TO IDENTIFY CLINICAL INCONSISTENCY IN THE USE OF RATING SCALES IN CNS RCTs
Chairs: Jonathan Rabinowitz, PhD; Nina R. Schooler, PhD
The working group will complete the compilation of the flags for the Montgomery-Asberg Depression Scale (MADRS) and begin working on flags for the Personal and Social Performance (PSP) scale.
ASSESSING ABUSE LIABILITY POTENTIAL
Chairs: Marta Sokolowska, PhD; Michael Klein, PhD; Beatrice Setnik, PhD
The functional excipient conundrum: Identifying nonclinical and clinical approaches to characterize the safety and effectiveness of excipients in abuse deterrent formulations
The development of abuse deterrent formulations is an important consideration in mitigating the risks associated with inappropriate use of opioid drug products. Many such formulations contain functional excipients that render the formulation less attractive, particularly when administered via non-intended routes. Additional studies may be required to examine safety of functional excipients administered via the intended as well as unintended (intranasal or intravenous) administration routes.
This workshop will 1) discuss the profiles of several different excipients used for abuse deterrent formulations, 2) discuss the changing regulatory framework with the need to evaluate safety of excipients administered via non-intended routes, 3) identify key study types that could be used to elucidate the safety and efficacy of an excipient, and 4) identify when such studies can be safely conducted in humans.
BEHAVIORAL AND PSYCHIATRIC SYMPTOMS IN DEMENTIA (BPSD)
Agitation Subgoup Session
Chairs: Cedric O’Gorman, MD; Paul Rosenberg, MD
The Agitation associated with Alzheimer’s Disease subgroup of the ISCTM BPSD working group will convene to further advance its plans to develop a consensus amongst academia, industry and regulators pertaining to recommendations for optimal trial methodology that could allow for the discovery of medications which may help relieve symptoms of agitation associated with AD. Specific objectives for discussion at the meeting will include the development of a publication plan (including the writing of a white paper), ongoing conference attendance considerations, as well as the possibility of multi-partner initiatives to draw attention to the considerable unmet need associated with agitation in Alzheimer’s disease.
BEHAVIORAL AND PSYCHIATRIC SYMPTOMS IN DEMENTIA (BPSD)
Apathy Subgroup Session (Note- session takes place Tuesday MORNING – 8:30-11:30)
Chairs: Krista Lanctot, PhD; David Miller, MD
During this session we will:
-Discuss the apathy consensus meeting survey process
-Discuss the results of the preliminary survey:
-identification of areas of agreement
-identification of areas of disagreement
-Review next steps for diagnostic criteria for diagnostic criteria consensus conference
PREVENTION TRIALS IN ALZHEIMER’S DISEASE – DESIGN CONSIDERATIONS
(formerly called – COGNITIVE ASSESSMENT IN ALZHEIMER’S DISEASE)
Chairs: Holly Posner, MD; Philip Harvey, PhD
Current treatment efforts in Alzheimer’s disease (AD) are focused on prevention of the illness, given the consistent failures in treatments targeted at existing disease. Prevention trials are challenging for several reasons, including in domains of subject selection, outcomes measurement, and trial duration. Subject selection in previous trials has included participants who were related to AD cases or cases with subclinical cognitive changes not meeting criteria for AD. Outcomes measurement is challenging because participants who do not have detectable cognitive changes cannot be assessed with standard outcomes measures. Trial duration is challenging because of the unclear time course of development of AD in the targeted populations and the cost of longer trials.
The current working group meeting will discuss alternative strategies for selection of participants, outcomes assessments, and duration considerations for trials. Brief presentations, followed by discussion, will be focused on these issues and other considerations raised by the attendees.
ESTIMANDS AND MISSING DATA
Chairs: Pilar Lim, PhD; Elena Polverejan, PhD
The Estimands and Missing Data Working Group will continue to develop the structured framework with which to implement the ICH E9(R1) draft guidance on “Estimands and Sensitivity Analysis in Clinical Trials”. This structured framework can be used by clinical teams when designing a clinical study according to the guidance. The first indication that is being tackled is Major Depressive Disorder.
LATE-ONSET DEPRESSION: A Distinct Indication?
Chair: Heddie Martynowicz, DM, MS
“Major depressive disorder (MDD), is a serious, recurrent, and disabling psychiatric illness associated with excess mortality and with years of potential life lost. This session will explore clinical distinctions between late-onset depression and adult MDD and potential regulatory implications.”
NEGATIVE SYMPTOMS
Chairs: Stephen Marder, MD; David Daniel, MD
The workgroup will re-examine prior recommendations on clinical trial methodology for studying treatments for negative symptoms. The re-examination will be informed by data from recent trials. In addition, the group will explore designs for drugs that may be used as monotherapy for psychotic and negative symptoms.
ORPHAN DISEASES (Closed)
Chairs: Joan Busner, PhD; Judy Dunn, PhD; Ravi Anand, MD
SESSION PREPARATION-OPEN TO 14TH ANNUAL MEETING ORPHAN DISEASE CHAIRS, SPEAKERS AND PANELISTS ONLY.