White Paper: Navigating the Landscape of Compassionate Use

This white paper summarizes insights on compassionate use developed at Siren Interactive’s workshop,

Critical Issues in Patient Advocacy Engagement & Collaboration: How Do We Find Common Ground?, at

the World Orphan Drug Congress in April 2014. Participants included representatives from the industry

in commercialization and patient advocacy roles, rare disease patient advocacy group (PAG) leaders, and

patient opinion leaders (POLs). 

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Special thanks are due to our moderator Wendy White, founder and CEO

of Siren Interactive, and panelists:

• Barbara Wuebbels, vice president of patient advocacy and medical affairs
• at Audentes Therapeutics
• Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy
• Ben Lenail, co-founder and business strategy officer of ALD Connect
• Julie Raskin, executive director of Congenital Hyperinsulinism International
• Carrie Burke, director of alliance development at Shire Pharmaceuticals
• Nicole Boice, founder and president of Global Genes/Rare Project

Webinar: Expedited Programs for Drug Development

Leveraging Expedited Programs for the Development of Drugs and Biologics for Serious Conditions and Rare Diseases

October 8, 2014 @ 1PM EDT
Reserve your spot now

in partnership with Cardinal Health

FDA is committed to advancing the development of therapies for the treatment of serious conditions and rare (orphan) diseases.  As part of this commitment, FDA provides expedited pathways to decrease the development and review time for new drugs and biologics that address unmet medical needs.  In addition to these expedited pathways, which are fast track, priority review, and accelerated approval, FDA recently introduced the new breakthrough therapy designation.

This new designation will allow for more intensive guidance from FDA throughout the development process. This presentation will examine each of FDA’s four expedited programs and their impact on recently approved products indicated for serious conditions and rare diseases.

Includes a live Q&A session, and post-webinar OnDemand access for all who register.

Key Takeaways:

• FDA regulations, legal acts, and guidances governing expedited programs for serious conditions and rare diseases 
• The concepts of serious condition, available therapy, unmet medical need, and rare disease
• Understand and distinguish the differences between FDA’s four expedited development programs - fast track, breakthrough therapy, priority review designations, and accelerated approval pathway
• The qualifying criteria and features of each expedited program, as well as what is required for requesting a specified designation
• The impact of expedited programs on recent New Drug Application (NDA) and Biologic License Application (BLA) approvals
• How to leverage expedited programs to support the development of pharmaceuticals indicated for serious conditions and rare diseases

Who Should Attend:

• Professionals who wish to broaden their understanding of drug development in the pharmaceutical or biotechnology industry, including:
• Regulatory Affairs Directors/Managers
• Research and Development Directors/Managers
• Clinical Research Associates
• Medical Writers
• Quality and Manufacturing Director/Managers
• Quality Assurance Directors/Managers
• Project Managers

Reserve your spot now

PharmaVOICE is partnering with Cardinal Health Regulatory Sciences