The Value of Orphan Drug Designation in your Drug Development Program

Original Air Date:  Tuesday, July 28, 2020
Time:  11:00 AM EST/4:00 PM London

As a drug developer you may wonder if you have a portfolio product or development candidate that qualifies as an orphan drug and thus provides options unique to orphan drug development.

There are approximately 7000 known rare diseases and more expected to be identified in the future and your drug may be effective against a rare disease. A number of these orphan diseases are well known, and the epidemiology well characterized, however, there are still numerous rare diseases still in need of an effective treatment.

This webinar will provide an overview of Orphan Drug Designation and the benefits from both a development and commercial viewpoint. It will explore the benefits of early planning and early engagement with the FDA.

A panel of CATO SMS experts will review the key issues encountered during the development of a product receiving Orphan Designation and explore what challenges and options for sponsors may be seen in the coming months.

Webinar Key Learning Objectives

  • What unique development options does Orphan Drug Designation offer?
  • What are the typical timelines and milestones in an orphan development program?
  • How can FDA’s development designations (Fast Track, Breakthrough, Accelerated Review, Priority Review) be applied in an Orphan program?
  • What are the regulatory nuances of Orphan Drug Designation as it relates to PREA?
  • Case studies on what has and has not been an effective strategy to gain Orphan Drug Designation

Featured Speakers


Gregory A. Hileman, Ph.D.,
Vice President, Regulatory Affairs

Dr. Hileman more than 35 years’ research and industrial experience, including 25 years in strategic regulatory affairs. He has led drug development meetings with most FDA divisions and contributed to submission of multiple fast track designation requests, breakthrough therapy designation requests, FDA meeting packages, INDs, NDAs, and BLAs. Dr. Hileman currently leads Cato’s regulatory strategy group and serves as primary FDA contact for multiple sponsors. Dr. Hileman has clinical and product development experience in neurology, psychiatry, oncology, gastroenterology, cardiology and women’s health.


William Lee, Ph.D., R.A.C.,
Vice President, Regulatory Affiars

Dr. Lee has more than 25 years’ research and industrial experience in gene therapy, cellular immunology, and therapeutic vaccines, including more than 15 years in regulatory affairs. Dr. Lee was involved in the writing of patents and the design and execution of preclinical studies. Dr. Lee has been involved in studies for experimental medications for oncology, peripheral arterial disease, dermatology, and myocardial ischemia. He has also been involved in combination drug/device products. He has been involved in the design of Phase 1 and Phase 2 clinical studies for devices, small-molecule drugs, and biologics. He has also participated in the preparation and submission of various INDs, New Drug Applications, and Food and Drug Administration meeting packages. Dr. Lee has experience in pharmacogenetics and the therapeutic areas of infectious diseases and oncology.


Nicholas Osborne, Ph.D., R.A.C.,
Clinical Writing Scientist, Medical Writing

Dr. Osborne has more than 24 years’ experience in biological and clinical research, with extensive experience in molecular biology, biochemistry, embryology, genetics, epigenetics, and stem cell biology. He has extensive drug and biologic development experience in a variety of therapeutic areas, with particular emphasis on rare diseases.

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