In the Spotlight: Recent FDA Updates and Guidance for Rare Disease Drug Development

Drug Discovery & Development, Life Sciences, Pharmaceutical Regulation,
  • Wednesday, April 24, 2019

The US Food and Drug Administration (FDA) just updated its 2015 draft guidelines for drug discovery in rare diseases. The January update, Rare Diseases: Common Issues in Drug Development, seeks to help pharmaceutical companies and other sponsors perform more efficient development programs for drugs and biological products and provides new insights on complex elements of the development process.

In this webinar, participants will learn about the core elements of the new guidance plus the latest recommendations and instructions from the agency. Participants will hear about practical examples and applications. 

 Subjects will include: 

  • Natural history studies
  • Issues for evaluation and validation of surrogate biomarkers
  • Nonclinical flexibility
  • External controls and early randomization
  • Additional agency considerations and interactions

 

Speakers

http://Angi%20Robinson,%20Premier%20Research

Angi Robinson, Vice President, Rare Disease & Pediatrics, Premier Research

Angi Robinson has been conducting pediatric and rare disease studies for more than 17 years. She has provided leadership and strategic oversight for rare disease studies at Premier Research for over 10 years, including global programs in ultra-rare indications. Ms. Robinson has supported FDA Pre-IND meetings, IND submissions and NDA project directorship.

At Premier Research, Ms. Robinson was involved with the launch of the first Best Pharmaceuticals for Children Act – Coordinating Center, collaborating with leadership staff at the National Institute of Child Health and Human Development (NICHD) in this capacity. Ms. Robinson’s experience includes multiple study designs including PK/PD, adaptive design and she has worked with products granted expedited designations by the agencies including FDA Fast Track, Breakthrough Therapy and PRIME designations. Specifically in orphan drugs Ms. Robinson has supported four orphan drug products resulting in FDA and European Medicines Agency (EMA) approval including two enzyme replacement therapies approved in 2015.   

Message Presenter
http://Abie%20Ekangaki,%20Premier%20Research

Abie Ekangaki, PhD, Vice President, Statistical Consulting, Premier Research

Dr. Abie Ekangaki is responsible for providing strategic planning, coordination, knowledge and expertise in Biostatistics for projects and/or standalone consultative services for both internal as well as external clients. He supports thebusiness development and operations efforts for key customers with regard to designing and managing effective and efficient clinical trials/programs bringing specific statistical, drug development, and operational knowledge in the respective area of focus. 

With over 23 years of experience as a biostatistician, Dr. Ekangaki has worked in different capacities around the globe, including as research scientist with the World Health Organization in Switzerland, as a statistics lecturer at Macquarie University, Australia and has held several technical and senior leadership roles in the pharmaceutical industry. In his 17+ years of experience in the pharmaceutical industry, he has applied and advised on the statistical design and analysis of clinical trials in various therapeutic areas, mostly ranging from proof-of-concept and beyond. He has led, overseen or consulted on the implementation of advanced statistical approaches in trial design and execution, such as advising on multiplicity issues in multicenter mega-trials and considerations for using a clinical utility index in the dose decision criteria for seamless phase 2/3 adaptive trials.

Prior to joining Premier Research, Dr Ekangaki was head of Global Statistical Sciences for Immunology at UCB BioPharma, where he supported the implementation of sound statistical methods for trials in psoriasis, psoriaticarthritis, arthritis, lupus, Crohn’s disease and others. He was previously head of North America Biostatistics at Quintiles, where he was instrumental in providing statistical guidance and consultation on sponsors’ trial protocols across different therapeutic areas and phases. Prior to Quintiles, Dr. Ekangaki spent 10 years at Eli Lilly in both technical and leadership roles and briefly held the position of head of statistics for the Diabetes and Osteoporosis platform.

Message Presenter
http://Kristi%20Miller,A%20Division%20of%20Premier%20Research

Kristi Miller, Head of Regulatory Affairs, Regulatory Professionals Inc., A Division of Premier Research

Kristi Miller leads a team of over 20 regulatory professionals to deliver both regional and international regulatory strategy for pharmaceutical and biotech drug development teams. With over 15 years of experience in the pharmaceutical industry, she has experience in providing regulatory leadership and execution of strategy for products across development (pre-IND through post-marketing). Her accomplishments include leading teams to successful global health authority interactions, INDs, orphan drug applications, fast track applications, and marketing applications. Her experience encompasses multiple therapeutic areas across immunology/inflammation, ophthalmology, oncology and other rare diseases and includes incorporating pediatric and companion diagnostic strategies.

She was previously director of global regulatory affairs at Pharmacyclics LLC, where she contributed to regulatory strategy for several indications for a marketed product in oncology and early development products in oncology or autoimmune indications. Prior to joining Pharmacyclics she was part of the clinical regulatory affairs group at Genentech with multiple roles including global regulatory lead for products in development for asthma and ophthalmology indications.

Message Presenter

Who Should Attend?

VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:

  • Clinical Affairs
  • Clinical R&D
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Operations
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

What You Will Learn

In this webinar, attendees will gain insights on the new FDA guidance for rare disease drug development regarding:

  • Natural history studies
  • Issues for evaluation and validation of surrogate biomarkers
  • Nonclinical flexibility
  • External controls and early randomization
  • Additional agency considerations and interactions

Xtalks Partner

Premier Research

Premier Research, a mid-size clinical research company, is dedicated to helping biotech, specialty pharma, and device innovators transform life-changing ideas and breakthrough science into new medical treatments.

As a global company, Premier specializes in the use of innovative technologies for smart study design and trial management to deliver clean, conclusive data to sponsors.

Whether it’s developing product lifecycle strategies, reducing clinical development cycle times, securing access to patients, navigating global regulations, maximizing the impact of limited rare disease data, or providing expertise in specific therapeutic areas, Premier is committed to helping its customers answer the unmet needs of patients across a broad range of medical conditions.

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