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Drug Development Designation for FDA Accelerated Approvals - Part 2

This blog is part two, in a two-part series, about FDA approvals. In Part 1, we discussed the approval framework. In this installment, we’ll discuss the various opportunities for accelerated approval.

What are FDA Accelerated Approvals?

In certain situations, the FDA may expedite the approval process through Accelerated Approval.[1] This pathway applies to drugs that treat serious or life-threatening conditions and show therapeutic benefits over existing therapies. FDA Accelerated Approvals allow drugs demonstrating an effect on a "surrogate endpoint" that predicts clinical benefit or an earlier clinical endpoint (though less robust) to gain approval. Post-marketing clinical trials are then required to verify and describe the drug's benefit. If subsequent trials fail to confirm the predicted benefit, the FDA may withdraw approval.

The FDA also employs various drug development designations to encourage the development of certain drugs, particularly those that address unmet medical needs or offer significant benefits over existing treatments. These designations include fast track, breakthrough therapy, and priority review.

What is Fast Track Designation?

This process expedites the development and review of drugs that treat serious conditions and fill unmet medical needs based on promising animal or human data.[2] The drug company must request Fast Track designation. The criteria for Fast Track designation include:

  1. The drug is intended to treat a serious condition.

  2. The drug addresses an unmet medical need.

  3. Preliminary clinical evidence indicates that the drug may provide a significant improvement over existing therapies.

If the FDA determines that the drug meets these criteria, it grants Fast Track designation. Fast Track designation does not guarantee FDA approval of the drug. However, it signifies the FDA's recognition of the drug's potential to address an unmet medical need and their commitment to facilitating its development and review process in an expedited manner.

Breakthrough Therapy Designation: Definition and Benefits

This designation accelerates the development and review of drugs intended to treat serious conditions, provided preliminary clinical evidence indicates substantial improvement over available therapies.[3] Drugs with Breakthrough Therapy designation are also eligible for Fast Track. The drug company must request Breakthrough Therapy designation.

The Breakthrough Therapy designation offers significant benefits to drug development and review processes.

Expedited Development and Review

The primary benefit of Breakthrough Therapy designation is the expedited development and review of the drug. The FDA provides intensive guidance and assistance to the drug company throughout the development process, including advice on clinical trial design, data collection, and regulatory requirements. This collaborative approach helps accelerate the drug's development timeline.

More Frequent FDA Interactions

Drug companies with Breakthrough Therapy designation have increased opportunities for communication and interaction with the FDA. This enables timely discussions on various aspects of the drug's development, such as clinical trial endpoints, surrogate markers, and potential challenges. Regular FDA input and guidance can help streamline the development process and address any concerns in a timely manner.

Rolling Review and Priority Review

Similar to the Fast Track designation, the Breakthrough Therapy designation allows for rolling reviews. This means that the drug company can submit sections of the New Drug Application (NDA) as they become available, instead of waiting for the complete application. This facilitates an earlier review process, potentially leading to faster approval. Breakthrough Therapy-designated drugs are also eligible for Priority Review, which expedites the FDA's review timeline from the standard ten months to six months.

Increased Probability of Approval

Breakthrough Therapy designation indicates that preliminary clinical evidence suggests the drug has the potential to provide a substantial improvement over existing therapies for a serious condition. While it does not guarantee approval, the designation signals the FDA's recognition of the drug's significance. Breakthrough Therapy-designated drugs have shown a higher likelihood of eventual approval compared to drugs without this designation.

Supportive Regulatory Flexibility

The FDA may be more flexible in its requirements for Breakthrough Therapy-designated drugs, considering the urgent medical need and the potential benefits they offer. This flexibility may be seen in clinical trial design, endpoints, and data collection, allowing drug companies to adopt innovative and efficient approaches to demonstrate safety and efficacy.

The Breakthrough Therapy designation provides valuable support, guidance, and expedited processes to facilitate the availability of promising treatments to patients in need.

Priority Review

Drugs with Priority Review designation undergo an expedited review process, with the FDA aiming to take action on the application within six months, compared to the standard review period of 10 months. Priority Review focuses attention and resources on drugs that significantly improve the treatment, diagnosis, or prevention of serious conditions. These may include therapies that offer enhanced efficacy, safety, or convenience compared to existing treatments. The designation is intended to prioritize the review of drugs that can address critical medical needs and make a substantial impact on patient care.

Priority Review receives increased attention and resources from the FDA. This may involve additional staff and expertise dedicated to reviewing the application, facilitating efficient communication between the FDA and the drug company, and expediting the decision-making process. The FDA encourages early and frequent communication between the drug company and the review team during the Priority Review process. This facilitates discussions regarding the drug's development, clinical trial design, data collection, and any potential issues or questions that may arise.

Accelerated approvals play a vital role in expediting the development and availability of promising treatments for serious and life-threatening conditions. These pathways recognize the urgent medical needs of patients and the potential benefits offered by innovative drugs. By allowing for faster evaluations, increased FDA support, and flexibility in the regulatory process, accelerated reviews aim to bring safe and effective therapies to patients in a timely manner. While maintaining rigorous standards for efficacy and safety, these designations facilitate collaboration between drug companies and the FDA, fostering a more efficient and collaborative approach to drug development. Ultimately, accelerated reviews have the power to positively impact patients' lives by accelerating the availability of breakthrough treatments and addressing unmet medical needs.

[1] [2] [3]

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