Updated: 20th April 2023

Important Regulatory Pathways for Accelerated Approval

3 min read By Manuel Neukum

– Takes about 6 minutes to read this topic –

The U.S. FDA has implemented various programs that help to deliver safe and effective therapies to patients in need.These programs cover a broad spectrum of topics and provide additional pathways and/or support for innovative science. In this blog, we’ll discuss several important and well-known programs and their impact on research and development. 

Accelerated Approval

In response to the AIDS epidemic in the 1980s, the FDA initiated a process to accelerate drug delivery and cut costs for new drugs treating serious and life-threatening illnesses and conditions. The process would seek to grant approval to certain drugs after extended Phase II trials.

This approach resulted in the enactment of Subpart H, commonly referred to as accelerated approval, leading to an expedited review of investigational agents by the FDA. This legislation allowed New Drug Applications (NDAs) to be approved based on so-called surrogate endpoints in clinical trials, including markers that would be expected to confer a clinical benefit, such as improved overall survival and tumor shrinkage in cancer patients and prolonged suppression of viral load in the treatment of patients with HIV. This approval also extended to other, intermediate clinical endpoints, including the measurement of a therapeutic effect considered reasonably likely to predict clinical benefit, such as an effect on irreversible morbidity or mortality.


These developments were followed by the Prescription Drug User Fee Act (PDUFA) under which the FDA agreed to improve drug review timelines and created a two-tiered system of review times, priority review and standard review.[1]


In 2012 the FDA Safety and Innovation Act (FDASIA) amended the fast track designation to include the Generating Antibiotics Incentives Now Act (GAIN Act), creating incentives to help drug developers develop and bring new antibiotics and other antimicrobials to market.

Breakthrough Therapy Designation

Another popular program under FDASIA is the FDA’s breakthrough therapy designation for investigational drugs. This designation addresses new trends in drug discovery and development, focusing on precision medicine, which is often paired with companion diagnostics, for treatment of cancer, hematological malignancies, genetic diseases, and an increasing number of other diseases.

Breakthrough therapy designation requires that the investigational drug be used for a serious or life-threatening disease or condition. It allows a rolling review of drug development material being submitted to the FDA. It differs from the other expedited review processes in requiring the use of clinically significant endpoints demonstrating substantial superiority of the drug over currently available therapies. In addition, breakthrough therapy designation offers more expansive benefits than the other expedited processes in that once a drug receives this designation, it is assigned an FDA committee. The committee meets regularly with the drug developers to devise the most efficient way to generate additional safety and efficacy data that will move development forward. However, in cases where a standard best-of-care therapy exists, the FDA requires “innovative, groundbreaking” changes in order for this designation to be granted.


Only a few years later, following the adoption of the FDA Modernization Act (FDAMA) in 1997, the expedited fast track approval process allowed drug developers to enter drugs intended to treat serious conditions where available data demonstrates a potential to address an unmet medical need. Beyond oncology and hematological malignances, drug developers increased their research to include a broad range of serious diseases such as AIDS, Alzheimer’s, epilepsy, and diabetes.

Fast Track Designation

Under FDAMA, a fast track designation can be submitted at any time during the drug development process and can use preclinical or clinical data to show potential to address an unmet medical need. The legislation requires the FDA to respond to the application within 60 days, allowing drug developers to benefit from early and frequent interactions with the FDA review team and from the rolling review process where portions of an application can be submitted for review prior to submitting the complete application.

Today, the majority of applicants granted fast track designation are also eligible for priority review. And while these regulatory changes in the drug approval process are beneficial in bringing new drugs to market, these new developments also result in more data and a greater need to share it more quickly with the broader scientific community and the public.

These evolving FDA programs and the rapid data sharing trends highlight the importance of data standards across the clinical research continuum. EvidentIQ all-in-one, user-friendly eClinical software, Marvin, is built from the ground up on CDISC standards. This ensures that clinical data seamlessly flows through the Marvin platform, improves data analysis across clinical trial databases, and optimizes data exchange with external systems. 


Contact us to learn more!

1] Committee on Strategies for Responsible Sharing of Clinical Trial Data; Board on Health Sciences Policy; Institute of Medicine. Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk. Washington (DC): National Academies Press (US); 2015 Apr 20. 4, “The Clinical Trial Life Cycle and When to Share Data.” Available from: https://www.ncbi.nlm.nih.gov
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