What is Breakthrough Therapy?
Breakthrough Therapy is a designation granted by the
U.S. Food and Drug Administration (FDA) to expedite the development and review of drugs intended to treat a serious condition. This designation is given when preliminary clinical evidence suggests that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.
Why is Breakthrough Therapy Designation Important?
The Breakthrough Therapy designation is crucial because it helps drugs reach patients faster. It provides all the features of the
Fast Track designation, including more intensive guidance on an efficient drug development program, potential eligibility for
priority review if supported by clinical data at the time of application, and organizational commitment involving senior managers. This can be particularly beneficial for patients with life-threatening conditions who have no other treatment options.
How Does Breakthrough Therapy Work?
When a drug is designated as a Breakthrough Therapy, the FDA collaborates closely with the drug developers to streamline the development process. This could include rolling review, where the FDA reviews portions of a New Drug Application (NDA) or Biologics License Application (BLA) before the complete application is submitted. The close communication and guidance from the FDA aim to identify the most efficient path to drug approval.What Criteria Must Be Met?
To receive Breakthrough Therapy designation, the following criteria must be met: The drug must be intended to treat a serious or life-threatening disease or condition.
Preliminary clinical evidence must indicate that the drug may offer substantial improvement over existing therapies on one or more clinically significant endpoints.
The goal is to identify the drugs that have the potential to offer significant benefits to patients facing serious health challenges.
What Are the Benefits for Drug Developers?
Drug developers benefit from the Breakthrough Therapy designation in several ways. The designation often leads to shorter development times and reduces costs associated with clinical trials. The close collaboration with the FDA helps in identifying potential issues early, minimizing the risk of unexpected challenges during the review process. Moreover, the accelerated path to market can provide a competitive advantage, allowing the drug to reach patients and generate revenue more quickly.Examples of Breakthrough Therapies
Several drugs have successfully received Breakthrough Therapy designation and gone on to make significant impacts in their respective fields. For example,
Kymriah (tisagenlecleucel) was one of the first gene therapies to receive this designation for the treatment of certain types of cancer. Another example is
Orkambi (lumacaftor/ivacaftor), which was designated for the treatment of cystic fibrosis in patients with specific mutations.
Challenges and Considerations
While Breakthrough Therapy designation offers many advantages, there are challenges and considerations for drug developers. The intense focus and rapid pace of development can be demanding. It requires substantial resources and commitment from the developers to meet the stringent requirements and timelines. Additionally, the preliminary data must be robust enough to justify the designation, which can be a high bar to meet.Future of Breakthrough Therapy Designation
The concept of Breakthrough Therapy continues to evolve as the FDA and other regulatory bodies look for ways to improve the drug development and review process. There is ongoing discussion about expanding similar designations globally to harmonize efforts and expedite drug approvals worldwide. The success of this program highlights the potential of innovative regulatory pathways to significantly impact public health.
