Rare Disease Disorders and CNS Drug Development – Paving the Way for Precision Medicine

Life Sciences, Pharmaceutical, Drug Discovery & Development,
  • Wednesday, November 15, 2017

In CNS drug development, more and more diseases once viewed as common are now known to be collections of rare variants. Our deepening understanding of the human genome and pathophysiology has led to fragmented classifications of common neurological or psychiatric disorders based on identified genetic markers. This trend of replacing larger classifications of common disorders with a more precisely defined spectrum of individual rare and ultra-rare diseases is changing the face of clinical research and development, not only in CNS but across virtually all therapeutic areas. What were once syndromes are now collections of diseases with common expression. Rare diseases are showing the way toward precision medicine for common disorders.

Join medical experts from Medpace’s neuroscience team as they explore:

  • The shifting clinical development landscape – Discussion of new genetic findings in Parkinson’s Disease, Dementia, and ALS, as well as the heritability of psychiatric disorders
  • The history of treatment of rare diseases with CNS manifestations – successes and failures
  • Operational lessons-learned in identifying, enrolling and retaining rare disease populations in clinical trials
  • New approaches to deliver disease specific targeted therapies to the CNS

Family history has long been central to clinical assessment of neurological and psychiatric diseases. Genetics has moved from disease risk factor to potential etiology in small patient subgroups, demanding a tailored approach to drug development. In neurodegenerative diseases, this shift is well underway. In psychiatry, responsible genes have been found in fewer syndromes, but the evidence for heritability and new ways of dividing populations by symptom complex, rather than classic diagnosis, are gaining ground and pointing the way to new approaches.

This improved understanding of the genetic basis and biochemical pathology of disease in genetic variants has combined with a revolution in bioengineering to potentially deliver innovative products directly to the CNS, thereby addressing unmet needs in neurological and psychiatric diseases with highly targeted therapy. This rare disease approach is beginning to impact patient care, and provide important opportunities for future clinical development in neuroscience and psychiatry.


James Vornov, MD, PhD. Vice President of Medical Affairs, Neurology, Medpace

Dr. Vornov is an internationally known clinician-scientist with broad experience in both neuroscience and CNS drug development. He has worked in multiple CNS therapeutic areas having directed programs in depression, suicidal ideation, Parkinson’s disease, stroke, neuropathic pain, diabetic and chemotherapy-induced peripheral neuropathy, anesthesia and brain tumors. He has particular expertise in the rapid transition of compounds from the laboratory to clinical proof of concept through the use of technologies such as biomarkers, PK/PD modeling, adaptive design and clinical trial simulation. Dr. Vornov received his B.A. in Biology from Columbia University and his M.D. and Ph.D. from Emory University School of Medicine. He trained in Neurology at the Johns Hopkins Medical School where he served on the faculty for 10 years prior to transitioning to industry.

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Richard Scheyer, MD. Vice President of Medical Affairs, Neurology, Pharmacology, Medpace

Dr. Richard Scheyer is a board certified neurologist with over 30 years of professional medical experience which includes 20 years dedicated to clinical drug development.  He is a pioneer in translational medicine and Phase I/II drug development, with special interest in early demonstration of clinical efficacy. Dr. Scheyer received his B.S. in Physics from Stanford University, his M.D. from The State University of New York, Upstate Medical University, and completed residency training in Neurology and fellowship training in Epilepsy and Clinical Pharmacology at Yale University before joining the Yale faculty, serving as Associate Professor of Neurology. Prior to joining Medpace, Dr. Scheyer held a number of leadership roles at biopharmaceutical firms where he was responsible for clinical, biomarker, and pharmacogenomic strategy and execution for more than 70 development candidates across therapeutic areas. Experience includes small molecule, macromolecular, and genetic therapies for common and rare neurologic disease.


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Michelle Petersen, MS, Sr. Associate Director, Clinical Trial Management, Medpace

Michelle Petersen has 10 years’ experience in preclinical and clinical research which includes managing global studies in Phases I through III of development. She has managed multiple global rare/ultra-rare disease trials with a focus on neuromuscular and pediatric disorders. Ms. Petersen is a member of Medpace’s specialized Rare Disease Consortium and Patient Recruitment Team, and she is adept in developing strategies necessary to address the potential challenges of recruitment and logistical requirements for studies of this nature. Ms. Petersen holds a Master of Science degree in Physiology from the University of Cincinnati College of Medicine.

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Who Should Attend?

VP’s, Directors, Managers and Department Heads working within:

  • Clinical Affairs
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Outsourcing
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

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Medpace is a scientifically-driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its physician-led, high-science, and disciplined operating approach that leverages local regulatory and deep therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective. Headquartered in Cincinnati, Ohio, Medpace employs approximately 2,500 people across 35 countries.

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