How to Apply for a Breakthrough Therapy Designation and Win It

Carolyn Troiano

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Background: 

A drug intended to treat a serious condition, and for which preliminary clinical evidence indicates it may demonstrate substantial improvement over currently available therapy on a clinically significant endpoint(s) may be designated as a breakthrough therapy. Making this determination is subjective, as it depends on the treatment effect, and may include the duration of the effect. It is also dependent on the importance of the observe clinical results. To gain this designation, there should be a clear and distinct advantage of using the new therapy.

A clinically significant endpoint refers to one that measures the effect on irreversible morbidity or mortality (IMM), or on symptoms indicating the serious impact of the disease. It can also refer to evidence that there is an effect on IMM or serious symptoms.

A drug designated as a breakthrough therapy is eligible for benefits related to the FDA approval process.  

The goal should be to request the designation no later than the end-of-phase-2 meetings, with a response from FDA within sixty (60) days of receiving the request.

Why should you 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 requests 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.

Description of the topic:

FDA provides 4 programs for accelerated drug approval, each with a specific set of requirements and considerations. Drugs must be for serious conditions that fill an unmet medical need and can be approved based on a surrogate endpoint, or biomarker.

A drug qualifies for BTD review if it is intended to treat a serious condition, and preliminary clinical evidence indicates it may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. BTD status affords the requestor more frequent meetings and written communications with FDA, rolling review, intensive guidance on efficient drug development and involvement of FDA senior managers to expedite the process.

Rolling Review enables a company to submit completed sections of its BLA or NDA for review by the FDA. The eCTD format enables a company to account for all components, interdependencies, linkages and the lifecycle of a submission, facilitating the process.

Only 70% of all BTD requests are met with FDA approval.  The standard for BTD is not the same as for drug approval, and preliminary clinical data are key. Specific study designs can aid in demonstrating substantial improvement. Early and effective communication with the FDA is key to success.

Areas Covered in the Session :

This webinar includes the following key objectives:

1. Objectives

2. FDA’s Expedited Programs

  • Fast-Track Designation
  • Breakthrough Therapy Designation (BTD)
  • Accelerated Approval
  • Priority Review Designation

3. Expedited Program Highlights

4. BTD Details

5. BTD Process

6. Additional Considerations

7. Recap

8. Q&A

Who will benefit:

This webinar is intended for those working in the FDA-regulated industries, including pharmaceutical, medical device, biological, animal health and tobacco. Functions that are applicable include research and development, manufacturing, Quality Control, distribution, clinical testing and management, adverse events management and post-marketing surveillance.

You should attend this webinar if you are responsible for planning, executing or managing the processes related to applying to FDA for a breakthrough therapy designation. Examples of who will benefit from this webinar include:

  • Regulatory Affairs Managers and Professionals
  • Compliance and Audit Managers
  • R&D Professionals
  • Clinical Data Management Professionals
  • Laboratory Professionals
  • Manufacturing Professionals
  • Computer System Validation Specialists
  • GMP Training Specialists
  • Business Stakeholders/Subject Matter Experts
  • Business System/Application Testers

This webinar will also benefit any consultants working in the life sciences industry who are involved in computer system implementation, validation and compliance.

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Training CD-DVD

Physical CD-DVD of recorded session will be despatched after 72 hrs on completion of payment


Recorded video

Recorded video session



Speaker: 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, providing very creative and interactive presentations. 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.


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