Developing a sound strategy to navigate the regulatory landscape for rare diseases can literally save (or cost) a sponsor months, or even years, of development.
TORONTO (PRWEB) February 26, 2020
Join the live session on Wednesday, March 18, 2020 at 11am EST (3pm GMT/UK) with speakers Christina Vonderhaar, MBA, Sr. Director, Regulatory Affairs at Medpace and Sargon Daniel, Associate Director, Regulatory Affairs at Medpace.
Regulatory bodies have demonstrated flexibility in applying the standards of safety and efficacy to rare disease trials given the unique challenges that drug sponsors face. Developing a sound strategy to navigate the regulatory landscape for rare diseases can literally save (or cost) a sponsor months, or even years, of development. That translates into lives saved and quality of life for those suffering with the disease, and financial gains or losses as well as a competitive advantage for the drug sponsor.
Topics will include:
Which way should we go? A deep dive into the available designations and regulatory pathways that can be used to expedite and accelerate treatments for rare diseases in the US and EU including: orphan drug designation, fast track designation, breakthrough designation, rare pediatric disease vouchers, PRIME and accelerated and conditional approvals.
What is required and how do we prepare? An in-depth assessment of the requirements for these designations and accelerated or expedited pathways, how to prepare your application and what to expect when you’re granted a designation or an expedited or accelerated pathway, as well as considerations around best-timing for submission will be provided from a global perspective. The presenters will also describe how to prepare for meetings with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and what to expect when engaging with regulatory authorities, particularly in a face-to-face meeting.
With or without pediatric populations? The pros and cons of including pediatric patients in adult trials for rare diseases will be presented, including suggestions for when it is time to engage regulators in discussions about including pediatrics patients earlier in the clinical development program. The webinar will wrap up with an overview of requirements for pediatric plans in the US and EU, and how these plans fit into the overall development program for rare diseases.
This webinar is second in a 3-part series on rare disease clinical research:
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