Part 2: Rare Disease Clinical Research – A Deep Dive into Regulatory Strategies & Considerations, Upcoming Webinar Hosted by Xtalks

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In this free webinar, Medpace’s global regulatory experts will discuss some of the key considerations for accelerating development in rare disease clinical research.

Xtalks Life Science Webinars

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.

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:

Part 1: Rare Disease Clinical Research – Spotlight on the Patient and Caregiver

Part 2: Rare Disease Clinical Research – A Deep Dive into Regulatory Strategies & Considerations

Part 3: Rare Disease Clinical Development – Strategies for Ensuring Endpoint Integrity

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.com
For information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

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Mira Nabulsi
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