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Rare & Orphan Disease Endpoints
Challenges and Examples from Neurodevelopment

A WCG | CenterWatch FREE Webinar
Thursday, Feb. 15, 2018 • 3:00 p.m. - 4:00 p.m. EST

Studies of rare disorders have gained traction and prominence in clinical research as applications for orphan drug designation increase dramatically. While numerous rare disease studies are being developed and launched, some face significant challenges from an unexpected quarter — selection and management of endpoints and outcome measures for rare disorders.  

The core issue: While more common diseases often have well-established outcome measures that are valid, sensitive to treatment effects, and accepted by regulators for submission, rare diseases are less likely to have a clear, well-marked path for endpoint selection. While appropriate measures may exist, they may be infrequently used — perhaps developed at a single center and not widely disseminated or accepted. In some instances, researchers for rare disorders may “borrow” an outcome measure from another area of research or attempt to adapt one that is not purpose-built. Or perhaps researchers may even attempt to develop, test and validate a new outcome from the ground up. Each strategy has risks. 

One instructive example for outcome selection in rare and orphan diseases may be seen in disorders of neurodevelopment. Clinical trials in Fragile X, autism, Down Syndrome and lysosomal storage disorders, such as metachromatic leukodystrophy, have all wrestled with this problem.  Along the way, multiple approaches have been combined to help address the need for sensitive, yet specific measures.

But you’re probably wondering, where to find out more about these instructive examples and how to learn more about rare and orphan disease endpoints?

Not to worry — we have a webinar for you. In just 60 minutes, from the comfort of your own office, you can take a deep dive into the sometimes murky waters of rare and orphan disease outcomes and endpoints and emerge, through the aid of practical, real-world examples, with a more comprehensive understanding of the challenges you face in this growing area. Save the date Feb. 15, 2018, to hear several leading authorities explain the challenges and opportunities you face in neurodevelopment and other areas of rare and orphan diseases.

Here’s what you’ll learn during this interactive session:

  • How to deal with the current challenges in outcome measure selection for rare and orphan disease clinical trials
  • How the use, adaptation and development of new and existing outcome measures in neurodevelopment disorders is affecting selection for rare and orphan disease clinical trials
  • How to optimize the advantages and minimize the limitations of adapting existing versus developing new outcome measures

Your presenters will provide you with a more complete and practical understanding of the science, potential and risks involved in rare and disease orphan endpoints and outcomes.

Take advantage of this free, timely presentation.


Who Will Benefit

This session is appropriate for professionals who conduct research on rare or orphan diseases in neurodevelopment, including individuals holding these job titles:

  • Patients and patient advocacy representative
  • IRB members
  • IRB administrators
  • Clinical project manager
  • Clinical research associates
  • Clinical research team member
  • Biosafety professional
  • Clinicians
  • Clinical scientists

Sign Up for FREE Below

Meet Your Presenters

Gahan Pandina, PhD

Senior Director, Venture Leader
Janssen Research and Development, Pharmaceutical Companies of Johnson & Johnson

Gahan Pandina, PhD, is a Senior Director, Venture Leader in the Janssen Research and Development, Pharmaceutical Companies of Johnson & Johnson, in Titusville, New Jersey. His current role is as the solution development team leader focused on developing tools and technologies to optimize novel medication development for Autism Spectrum Disorder (ASD), as well as innovative technologies to measure outcomes in mood disorders including treatment-resistant depression and bipolar disorder.

Brian Rothman, PhD

Vice President of Clinical Services
MedAvante-ProPhase

Brian Rothman, PhD, is vice president of clinical services at MedAvante-ProPhase. Rothman is responsible for the day-to-day development and maintenance of the organization’s scientific, clinical and operational resources in support of training and calibration services on current and prospective projects. Since joining the MedAvante-ProPhase team in 2010, Rothman has led training efforts for various clinical trial programs in many disease areas and has been a valuable contributor to psychometric research and scale development.

Mark Opler, PhD, MPH

Chief Research Officer, Managing and Directing Scientific Research and Development
MedAvante-ProPhase

Mark Opler, PhD, MPH, serves as chief research officer, managing and directing scientific research and development at MedAvante-ProPhase. Opler holds the titles of Adjunct Assistant Professor of Psychiatry at New York University and Assistant Professor of Clinical Neuroscience at Columbia University’s College of Physicians and Surgeons. His academic research focuses on the etiology, phenomenology and treatment of serious and persistent mental disorders.

Michael Aman, Ph.D.

Professor Emeritus of Psychology
Ohio State University

Michael Aman is Professor Emeritus of Psychology at Ohio State University. Aman was co-developer of the Aberrant Behavior Checklist (ABC), a scale for assessing treatment effects in people with intellectual and developmental disabilities (IDD). The ABC has been used in over 440 scientific studies, and it has been translated into more than 35 languages. He was also co-developer of the Nisonger Child Behavior Rating Form (NCBRF), another widely used and translated IDD instrument.

Abi Bangerter

Clinical Research Manager
Janssen Research and Development, Pharmaceutical Companies of Johnson & Johnson

Abi qualified and worked for 15 years as an Educational Psychologist in the UK, providing consultation, assessment and intervention advice to schools and families for individuals with a range of complex learning and behavior needs. From 2011-2013 she was a Senior Educational Psychologist with a responsibility for Research and Evidence Based Practice, across Buckinghamshire Local Authority, UK. In 2013 she joined the team at Janssen Research and Development, to assist in the development of Janssen Autism Knowledge Engine (JAKE) a set of tools and technologies for measuring changes in ASD symptoms and behavior following intervention.