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Accelerating Access to Life-Saving Treatments: Breakthrough Therapy Designation Demystified

Updated: Dec 30, 2023


Welcome to an in-depth exploration of breakthrough therapy designation—an influential regulatory framework that expedites the approval of life-saving treatments. As regulatory affairs professionals, we understand the vital role breakthrough therapy designation plays in providing timely access to innovative therapies for serious or life-threatening conditions. In this comprehensive article, we will delve into the historical origins of breakthrough therapy designation, its regulatory framework, statistical insights, real-world case studies, and debunk common misconceptions. Whether you are a beginner in the industry or a seasoned professional, join us as we navigate the complexities of this groundbreaking regulatory pathway.

I. Historical Origins: A Paradigm Shift in Regulatory Thinking

A. The Evolution of Expedited Drug Approval

The journey toward expedited drug approval began with the realization that certain patient populations face urgent and unmet medical needs. Regulatory agencies recognized the need to provide faster access to treatments for serious or life-threatening conditions.

B. The Birth of Breakthrough Therapy Designation

In response to this pressing need, the FDA introduced breakthrough therapy designation as part of the FDA Safety and Innovation Act (FDASIA) of 2012. This innovative pathway aimed to accelerate the development and review of promising therapies by facilitating increased collaboration between regulatory authorities and pharmaceutical companies.

II. Regulatory Framework: Navigating the Path to Accelerated Approval

A. FDA's Breakthrough Therapy Designation Program

The FDA's breakthrough therapy designation program offers a streamlined regulatory pathway for drugs demonstrating substantial improvement over existing treatments. The designation is granted based on preliminary clinical evidence, allowing for expedited development, review, and potential accelerated approval.

B. EMA's PRIME (PRIority MEdicines) Scheme

The European Medicines Agency (EMA) established the PRIME scheme to foster the development of innovative medicines addressing high unmet medical needs. PRIME provides early regulatory support and enhanced interaction between the EMA and developers of breakthrough therapies, aiming to expedite access to patients in need.

C. PMDA's Sakigake Designation

In Japan, the Pharmaceuticals and Medical Devices Agency (PMDA) introduced the Sakigake designation to promote the development of innovative therapies. Similar to breakthrough therapy designation, it aims to expedite regulatory review and approval, enhancing patient access to transformative treatments.

III. Statistical Insights: Illuminating the Impact of Breakthrough Therapy Designation

A. Proportion of FDA-Approved Drugs with Breakthrough Therapy Designation

Statistics reveal a significant impact of breakthrough therapy designation on the drug approval landscape. From 2018 to 2020, approximately 40% of novel drugs approved by the FDA received breakthrough therapy designation, showcasing the prevalence and importance of this pathway.

B. Analysis of Approval Times for Breakthrough Designated Drugs

Breakthrough therapy designation has been associated with reduced approval times, allowing patients to benefit from innovative therapies sooner. On average, drugs granted breakthrough therapy designation have shown a 50% reduction in the review time compared to non-designated drugs. Analyzing approval timelines highlights the efficiency gains and expedited access made possible by this regulatory pathway.

IV. Real-World Case Studies: Transforming Patient Care

A. Imatinib (Gleevec) - Revolutionizing Chronic Myeloid Leukemia Treatment

Imatinib, a breakthrough therapy for chronic myeloid leukemia (CML), exemplifies the transformative power of breakthrough therapy designation. Its accelerated approval paved the way for targeted therapies, significantly improving patient outcomes and quality of life. Since its approval in 2001, it has achieved an estimated 10-year survival rate of over 80% for CML patients.

B. Pembrolizumab (Keytruda) - Empowering Immunotherapy for Cancer

Pembrolizumab, a breakthrough therapy for certain types of cancer, showcases the potential of immunotherapy in oncology. Its expedited development and approval have revolutionized cancer treatment, providing new hope for patients with limited options. It has demonstrated impressive response rates in various cancers, including a 45% objective response rate in advanced melanoma patients.

V. Debunking Misconceptions: Separating Fact from Fiction

A. Balancing Expedited Access with Safety and Efficacy Standards

Despite the accelerated nature of breakthrough therapy designation, safety and efficacy remain paramount. Regulatory affairs professionals work diligently to ensure that expedited access does not compromise rigorous scientific evaluation. The designation does not bypass the need for robust evidence supporting the benefits and risks of the therapy.

B. Ensuring Rigorous Evaluation in Breakthrough Therapy Designation

Breakthrough therapy designation does not imply a relaxation of regulatory standards. Regulatory authorities maintain stringent review processes to evaluate the safety, efficacy, and quality of breakthrough therapies. Robust data and well-designed clinical trials continue to be essential for successful regulatory approval.

VI. Advantages of Breakthrough Therapy Designation:

  1. Expedited development and review process: Breakthrough therapy designation expedites the development and review process for life-saving treatments. It allows for faster access to innovative therapies, addressing urgent unmet medical needs and potentially saving lives. This streamlined pathway significantly reduces the time between initial development and regulatory approval.

  2. Increased regulatory support and guidance: Designated breakthrough therapies benefit from enhanced support and guidance from regulatory authorities. This includes more frequent interactions with regulatory agencies, providing opportunities for early advice, feedback, and guidance throughout the development and approval process. The close collaboration between regulatory authorities and sponsors ensures alignment of expectations and facilitates efficient decision-making.

  3. Collaborative approach to regulatory decision-making: Regulatory agencies take a collaborative approach when reviewing breakthrough therapy designations. This collaborative environment fosters constructive engagement and allows sponsors to work closely with regulators to address any questions or concerns. The joint effort between sponsors and regulators aims to ensure that the therapy meets regulatory requirements while considering the urgent needs of patients.

  4. Priority review status for regulatory submissions: Breakthrough therapy designation grants priority review status to the therapy's regulatory submission. This status expedites the evaluation process, reducing the time taken for regulatory decisions. The priority review designation signifies the therapy's potential to provide a substantial improvement over existing treatments and underscores the importance of timely patient access to life-saving therapies.

  5. Market exclusivity period: Breakthrough therapy designation provides a period of market exclusivity once the therapy is approved. During this exclusivity period, no other company can market a similar therapy for the same indication. Market exclusivity offers a competitive advantage to the sponsor, allowing them to recoup their investment and promote continued innovation. This exclusivity period ensures that the sponsor has the opportunity to establish a strong market presence and benefit from their investment in research and development.

  6. Flexibility in evidence generation: Breakthrough therapy designation allows for flexibility in evidence generation, recognizing that traditional clinical trial designs may not always be feasible for certain conditions or populations. This flexibility enables sponsors to explore innovative approaches, such as adaptive trial designs and surrogate endpoints, to demonstrate the therapy's effectiveness. It promotes efficient development while ensuring rigorous evaluation of the therapy's benefits and risks.

  7. Implementation of expanded access programs: Breakthrough therapy designation facilitates the implementation of expanded access programs, also known as compassionate use programs. These programs provide early access to therapy for patients who have no other treatment options. By allowing eligible patients to access the therapy while it undergoes regulatory review, breakthrough therapy designation addresses the urgent needs of patients who would otherwise have limited or no treatment alternatives.

  8. Global recognition and harmonization: Breakthrough therapy designation has gained recognition globally, with regulatory authorities in various countries adopting similar expedited pathways. This global recognition and harmonization of regulatory processes enhance the efficiency of drug development and patient access to breakthrough therapies across borders. It fosters collaboration and knowledge-sharing among regulatory agencies, streamlining the regulatory pathway for innovative treatments and benefiting patients worldwide.

VII. Regulatory Affairs Strategies: Nurturing Breakthrough Therapies

A. Collaborative Efforts between Regulatory Affairs and R&D Teams

Effective collaboration between regulatory affairs and research and development teams is crucial for the success of breakthrough therapy designation. Regulatory affairs professionals play a pivotal role in guiding development strategies, ensuring compliance with regulatory requirements, and facilitating effective communication with regulatory authorities throughout the process.

B. Leveraging Real-World Evidence in Regulatory Submissions

Real-world evidence plays an increasingly important role in supporting regulatory submissions for breakthrough therapies. Regulatory affairs professionals harness this evidence to enhance the understanding of the therapy's benefits and risks, optimize patient selection criteria, and provide insights into long-term safety and effectiveness.

VIII. Future Perspectives: Advancing Breakthrough Therapy Designation

A. Emerging Trends in Precision Medicine and Personalized Therapies

Breakthrough therapy designation will continue to evolve in response to advancements in precision medicine and personalized therapies. Regulatory affairs professionals will navigate the regulatory challenges associated with targeted treatments and companion diagnostics, ensuring timely access to tailored therapies.

B. Exploring Global Harmonization of Breakthrough Therapy Designation

As the impact of breakthrough therapy designation extends globally, regulatory harmonization becomes increasingly important. Collaboration between regulatory agencies worldwide aims to align processes, standards, and expedited pathways, facilitating efficient development and access to breakthrough therapies across borders.


Breakthrough therapy designation has revolutionized the regulatory landscape, paving the way for expedited approval of life-saving treatments. By understanding its historical context, regulatory framework, and statistical impact, we can appreciate the significance of this regulatory pathway. Real-world case studies exemplify the tangible benefits breakthrough therapy designation offers to patients, while debunking misconceptions clarifies its limitations. As regulatory affairs professionals, let us continue to navigate the complexities of breakthrough therapy designation, shaping the future of expedited drug approval and ultimately improving patient outcomes. Together, we can forge a path toward even greater advancements in regulatory affairs and the development of life-saving therapies.


Credits: <a href="">Image by master1305</a> on Freepik

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