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Definition:

Breakthrough therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on clinically significant endpoint(s).

What is a ‘Serious Condition’?

A disease or condition is considered serious if it significantly impacts day-to-day functioning due to associated morbidity. While temporary and self-resolving morbidity may not meet this criterion, persistent or recurrent morbidity does, even if it is reversible. All conditions that are defined as life-threatening are also classified as serious.

What is Preliminary Clinical evidence?

Preliminary clinical evidence is early proof that a new treatment might work well. For the FDA’s Breakthrough Therapy Designation, it means the treatment shows signs in early studies that it could be much better than current options for a serious condition. This early evidence suggests the treatment could make a big difference for patients.

What is a substantial improvement over an available therapy?

A substantial improvement means that a new treatment works much better than the existing ones. For the FDA’s Breakthrough Therapy Designation, this means the new treatment can show it has significantly better results in treating a serious condition compared to what’s already available. It could help patients more effectively or reduce symptoms more than current treatments.

What is a clinically significant endpoint?

For Breakthrough Therapy designation, a clinically significant endpoint is a key measure of how a treatment impacts a serious disease. This could mean showing it affects:

1. Irreversible Morbidity or Mortality (IMM): The treatment can improve outcomes for conditions that cause permanent damage or death.
2. Serious Symptoms: The treatment can alleviate symptoms that have severe consequences for the patient.

Essentially, it’s about proving that the treatment makes a real difference in important health outcomes.

Benefits:

Drugs that receive Breakthrough Therapy Designation from the FDA are eligible for some or all of the following benefits:

• More frequent meetings with the FDA or more frequent written communication with the FDA
• Eligibility for Accelerated Approval and Priority Review (if criteria are met)
• Rolling review
• Intensive guidance on an efficient drug development program
• Involvement of FDA senior managers to expedite drug development

Timing: The FDA recommends that the Breakthrough Therapy Designation request should occur before the end-of-phase-2 meetings.

What is the difference between Fast Track Designation and Break Through Designation?

The most significant difference in these two programs is related to the type of data needed to substantiate the request.

Fast Track Designation can be granted based on preliminary data, such as activity in a nonclinical model or pharmacological data, or a mechanistic rationale.

Breakthrough Therapy Designation must use preliminary clinical data, and therefore activity in a nonclinical model or a mechanistic rationale alone would not be sufficient.

Fast Track Designation must only have the potential to address an unmet medical need, while drugs seeking Breakthrough Therapy Designation must have preliminary data which demonstrate substantial improvement on clinically significant endpoint(s)

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