Expediting Approval for Regenerative Drugs and Breakthrough Therapy Designation

The FDA has paved the way for four distinct programs designed to fast-track drug approvals, each tailored to specific criteria and considerations. These programs are exclusively available for drugs targeting severe medical conditions where unmet needs persist, and where approval can be based on surrogate endpoints or biomarkers.

The Breakthrough Therapy Designation (BTD) is bestowed upon drugs intended for serious conditions, showing promising preliminary clinical evidence of substantial improvements over existing therapies on clinically significant endpoints. BTD status unlocks a host of advantages, including increased interaction and communication with the FDA, a rolling review process, comprehensive guidance for streamlined drug development, and the engagement of FDA senior management to expedite the approval journey.

The Rolling Review mechanism empowers companies to submit completed sections of their Biologics License Application (BLA) or New Drug Application (NDA) for ongoing assessment by the FDA. Utilizing the electronic Common Technical Document (eCTD) format ensures a systematic approach that takes into account all submission components, their interdependencies, linkages, and the submission's lifecycle, streamlining the entire process.

It's worth noting that not all Breakthrough Therapy Designation requests receive FDA approval, as the criteria differ from standard drug approval. Success hinges on compelling preliminary clinical data and specific study designs that demonstrate substantial improvement. Effective and early communication with the FDA stands as the linchpin to achieving your goals in this accelerated journey.

WHY YOU SHOULD ATTEND:

During this presentation we will discusses FDA expectations from BTD requests, the best practices to write a BTD request, and pros and cons of applying for a BTD. The session will highlight key regulatory requirements, dos and don’ts, common areas of concern, and trouble-shooting. The format and rules governing BTD request will be discussed along with success and challenge factors. The training is designed to provide industry best practices related to all aspects of the BTD application process.

AREAS COVERED IN THE SESSION:

• Objectives
• FDA’s Expedited Programs
  • Fast-Track Designation
  • Breakthrough Therapy Designation (BTD)
  • Accelerated Approval
  • Priority Review Designation
• Expedited Program Highlights
• BTD Details
• BTD Process
• Additional Considerations
• Recap and Q&A

WHO SHOULD ATTEND:

This 90-minute online course is intended for professionals in the Medical Device, Biotechnology, Pharmaceutical Industry. Although not presently stated in the draft , the same guide could be used by FDA Regulated Industries personnel.

Anyone who is involved in the development, testing, manufacturing, storage, handling and distribution of product must understand and conform to FDA requirements for data quality and integrity.

Finally, anyone who is acting as a consultant or contractor to a company in an FDA-regulated industry should attend to ensure they are able to bring the most current knowledge and expertise to their assignment.

Course Director: CAROLYN TROIANO

Carolyn Troiano has more than 30 years of experience in computer system validation in the pharmaceutical, medical device, animal health, tobacco and other FDA-regulated industries. She is currently an independent consultant, advising companies on computer system validation and large-scale IT system implementation projects. During her career, Carolyn worked directly, or on a consulting basis, for many of the larger pharmaceutical companies in the US and Europe. She developed validation programs and strategies back in the mid-1980s, when the first FDA guidebook was published on the subject, and collaborated with FDA and other industry representatives on 21 CFR Part 11, the FDA’s electronic record/electronic signature regulation. Carolyn has participated in industry conferences. She is currently active in the PMI, AITP, and RichTech, and volunteers for the PMI’s Educational Fund as a project management instructor for non-profit organizations.