Breakthrough Therapy

Breakthrough Therapy

A Breakthrough Therapy is a designation granted by the United States Food and Drug Administration (FDA) to expedite the development and review of drugs that are intended to treat a serious condition and have shown preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). This designation is part of the FDA's efforts to facilitate and accelerate the development of promising new treatments for serious or life-threatening conditions.

Criteria for Designation

To qualify for Breakthrough Therapy designation, a drug must meet the following criteria:

1. Serious Condition: The drug must be intended to treat a serious or life-threatening disease or condition. 2. Preliminary Clinical Evidence: There must be preliminary clinical evidence indicating that the drug may offer substantial improvement over existing therapies on one or more clinically significant endpoints.

Benefits of Breakthrough Therapy Designation

Drugs that receive Breakthrough Therapy designation are eligible for:

• Intensive Guidance: The FDA provides intensive guidance on an efficient drug development program, beginning as early as Phase 1.
• Organizational Commitment: The FDA commits to involving senior managers and experienced review staff in a proactive and collaborative manner.
• Rolling Review: The drug sponsor can submit completed sections of the New Drug Application (NDA) or Biologics License Application (BLA) for review by the FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed.

Impact on Drug Development

The Breakthrough Therapy designation can significantly impact the drug development process by:

• Reducing the time required to bring a drug to market.
• Increasing the likelihood of approval for drugs that show early promise in clinical trials.
• Encouraging innovation and investment in the development of new therapies for serious conditions.

Examples of Breakthrough Therapies

Several drugs have received Breakthrough Therapy designation, leading to accelerated approval and availability to patients. Examples include:

• Imatinib (Gleevec) for certain types of cancer.
• Sofosbuvir (Sovaldi) for the treatment of hepatitis C.
• Pembrolizumab (Keytruda) for various types of cancer.

Also see

• Fast Track (FDA), another FDA program to expedite drug development.
• Priority Review, a designation that shortens the review period for drugs that offer significant improvements in treatment.
• Accelerated Approval, a pathway for drugs that treat serious conditions and fill an unmet medical need.

This article is a stub related to pharmacology. You can help WikiMD by expanding it!

Template:FDA