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Breakthrough Therapy


Breakthrough therapy is a designation given to certain drugs or treatment by the U.S. Food and Drug Administration (FDA) for expedited development and review. This status is assigned to drugs that show significant clinical improvements over existing treatments for serious or life-threatening conditions. The aim of this designation is to ensure promising therapies reach patients faster by providing a more efficient and collaborative evaluation process.

Key Takeaways

  1. Breakthrough Therapy is a designation by the U.S. Food and Drug Administration (FDA) that expedites the development and review of promising drug treatments for serious or life-threatening conditions.
  2. This designation aims to provide patients with quicker access to new therapies with substantial improvements over currently available options, by fast-tracking the approval process and ensuring close collaboration between the FDA and the drug developers.
  3. To qualify for Breakthrough Therapy designation, the treatment must demonstrate substantial, preliminary clinical evidence that it offers significant advantages in efficacy and/or safety over existing treatments, addressing unmet medical needs for a specific disease or condition.


The term Breakthrough Therapy is important in the context of VA benefits as it refers to a medical intervention or treatment that is deemed highly significant in addressing severe or life-threatening medical conditions affecting veterans, especially when compared to available therapies.

This designation, given by the Food and Drug Administration (FDA), ensures that the therapy receives expedited development, review, and approval processes to make it accessible more quickly to the veterans who need it.

By making it a priority, the VA aims to provide breakthrough therapies to veterans, thus significantly improving their quality of life and ensuring a prompt and effective intervention for their medical conditions.


Breakthrough Therapy is a designation granted by the U.S. Food and Drug Administration (FDA) to expedite the development and review of potential medications or therapies that demonstrate substantial improvement over existing options for severe or life-threatening conditions.

The primary purpose of this designation is to accelerate the availability of such groundbreaking medical treatments to patients who desperately need them, ultimately enhancing their quality of life and potentially increasing their survival rates. This expedited process is possible because the FDA provides intensive guidance and greater access to its expert advice, enabling researchers and pharmaceutical companies to quickly identify the most efficient pathway to bring effective, innovative treatments to patients.

The utilization of Breakthrough Therapy designation is also beneficial for medical professionals and various stakeholders involved in the healthcare industry. It encourages investment and research in novel therapeutic options, offering an incentive to develop solutions to address unmet medical needs.

Moreover, the intensive collaboration between researchers, pharmaceutical companies, and regulatory authorities ensures a robust assessment of the safety and efficacy of the treatments, while maintaining a rigorous scientific foundation. Overall, Breakthrough Therapy is instrumental in fostering the development of life-changing medical treatments, correlating to better health outcomes and improved patient care.

Examples of Breakthrough Therapy

The term “Breakthrough Therapy” does not directly relate to VA Benefits, as it refers to a special designation given by the US Food and Drug Administration (FDA) to expedite the development and review of drugs that treat serious or life-threatening conditions. Breakthrough Therapy designation is given to those drugs that have shown preliminary clinical evidence demonstrating the potential for significant improvement over available therapies.However, if we are considering real-world examples of drugs that have received Breakthrough Therapy designation, there are many benefits that veterans could receive from these therapies for various conditions. Here are three examples:

Esketamine (Spravato) for Treatment-resistant Depression: In 2019, the FDA approved Spravato as a breakthrough therapy for treatment-resistant depression. Veterans suffering from depression often have high rates of treatment-resistant forms of the condition, and this nasal spray, used in conjunction with other oral antidepressant medications, could provide significant improvements for those who have not responded to standard treatments.

Axicabtagene ciloleucel (Yescarta) for Non-Hodgkin Lymphoma: This immunotherapy, also known as CAR-T cell therapy, received Breakthrough Therapy designation in 2017 for the treatment of certain types of aggressive non-Hodgkin lymphomas. Veterans diagnosed with these complex and challenging-to-treat cancers could potentially benefit from this breakthrough immunotherapy drug.

Glecaprevir/Pibrentasvir (Mavyret) for Hepatitis C: Hepatitis C infection is a significant health issue affecting many veterans. In 2017, the FDA approved Mavyret as a breakthrough therapy for treating all six major genotypes of Hepatitis C. The medication regimen is shorter than traditional treatments and offers a higher cure rate, which both can directly benefit veterans living with this chronic and potentially life-threatening infection.

VA Benefits: Breakthrough Therapy

What is Breakthrough Therapy?

Breakthrough Therapy is a designation by the U.S. Food and Drug Administration (FDA) that intends to expedite the development and review of drugs for serious or life-threatening conditions. The goal is to ensure that patients have access to new therapies as soon as possible, especially when no satisfactory treatment options exist.

What are the benefits of Breakthrough Therapy?

Breakthrough Therapy designation offers several benefits for drug manufacturers, including more intensive FDA guidance on efficient drug development, eligibility for Accelerated Approval and Priority Review, and the involvement of senior FDA managers in the process. It helps in faster development, approval, and availability of the designated therapy for patients who need it.

Who is eligible for Breakthrough Therapy?

Drugs that are intended to treat serious or life-threatening conditions and have demonstrated substantial improvement over available therapies on a clinically significant endpoint based on preliminary clinical evidence can be eligible for Breakthrough Therapy designation. Drug manufacturers need to apply for this designation with their supporting data, and the FDA reviews their request before granting the designation.

How to apply for Breakthrough Therapy designation?

Drug manufacturers can request Breakthrough Therapy designation for their product by submitting an application to the FDA. The application must include preliminary clinical evidence indicating that the drug demonstrates substantial improvement over current therapies on one or more clinically significant endpoints. Additionally, a detailed description of the serious or life-threatening condition, the proposed treatment population, and the available treatment options must be provided.

What is the difference between Breakthrough Therapy and other FDA designations?

Breakthrough Therapy is one of several FDA programs aimed at expediting the development and review of drugs for serious or life-threatening conditions. Other programs include Fast Track, Priority Review, and Accelerated Approval. The main difference between these designations lies in the level of FDA involvement, review speed, and specific benefits. Breakthrough Therapy, in particular, offers more intensive guidance and involvement of senior FDA managers in the process, ensuring a faster and more collaborative approach toward drug development and approval.

Related VA Benefit Terms

  • Expedited Drug Approval
  • Innovative Treatment
  • Clinically Significant Improvement
  • Advanced Therapy Designation
  • Patient Access to New Medications

Sources for More Information