Clinical and Regulatory Considerations for Orphan Drug Applications

Clinical and Regulatory Considerations for Orphan Drug Applications

Date: March 10, 2016, 3:00 p.m. EST

By Emma Barrett, MD

Overview

The development of Orphan Drugs - those developed specifically to treat rare medical conditions - is growing at an astounding rate. In 2014, nearly 41%1 of all new product approvals from the FDA were for the treatment of rare diseases - a trend that is expected to continue as the exclusivity rights for current blockbuster drugs expire over the next five years. This reality, coupled with a lack of adequate replacements in the development pipeline, has persuaded biopharmaceutical companies large and small to turn to orphan drugs to fill the gap and drive profitability moving forward.

As a result, the development and marketing of orphan products is one of the fastest-growing sectors of the pharmaceutical, biologic and device industries, representing over $115B in worldwide sales in 2013 and expected to eclipse $200B by 20202.

Successfully navigating the orphan drug approval process requires addressing issues and challenges typically not encountered in traditional biopharmaceutical product development. In this webinar, Dr. Emma Barrett will provide expert clinical and regulatory guidance for the development of products that treat rare diseases.

This webinar will guide participants through the latest Agency thinking and Industry standards on the following:

  • Review of Latest Agency Guidance on Orphan Drug Submissions
  • Best practices for creating a strategic plan to bring an orphan product to market
  • Discussion of clinical and regulatory considerations for clinical trial design and enrollment procedures
  • Case studies & strategies for navigating the challenges of orphan drug development


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About the Host

Dr. Emma Barrett comes to Biologics Consulting with 15 years of clinical development experience in the pharmaceutical industry. She has held positions of increasing responsibility in a number of pharmaceutical companies, most recently as Senior Director, Clinical Research at Array BioPharma. In her most recent role, Emma oversaw the development of binimetinib from IND filing to pivotal Phase 3 studies. She was intimately involved in the strategic planning for the binimetinib program and for the studies conducted by Array BioPharma and their former co-development partner Novartis Pharmaceutical. In addition, she was the clinician overseeing all binimetinib clinical trials at Array Biopharma during her 6 years with the company. This involved protocol designs, sites selection, CROs selection, medical monitoring, data cleaning and analysis, clinical input on regulatory submissions and inquiries, CRA/study team/site/investigator education and training, organization of steering committees, data safety monitoring boards and advisory meetings, study and program level safety monitoring and participation in the global strategy team. Emma was the clinical lead for 4 additional oncology programs and 1 cardiology program during this time. She has extensive experience in the design, planning, implementation, oversight and analysis of first-in-human trials through global Phase 3 trials.

About The Biologics Consulting Expert Edge Webinar Series

The Expert Edge Webinar Series was created by Biologics Consulting in an effort to share the latest Agency guidance and best practices with leaders throughout the biopharmaceutical and medical device industries. Each interactive, web-based workshop in the series is hosted by an accomplished subject-matter expert and designed to provide participants with a pathway to success through some of the most complex regulatory and product development challenges in the Industry today.