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20-Aug-2019

Expedited Programs for Oncology Drug Development: accelerating the pathway to market

Expedited Programs for Oncology Drug Development: accelerating the pathway to market

Summary

Nearly half of oncology drugs recently approved for the US market have gone through one of the FDA’s expedited programs. This has helped place important new drugs into the hands of patients who need them while creating a robust pipeline of therapeutics. For cancer drug developers to effectively use these accelerated pathways they need a clear understanding of the differences between each program and what regulators are looking for based on current criteria and past approvals.  
Editor: Cort Hepler Last Updated: 16-Oct-2019

Nearly half of oncology drugs recently approved for the US market have gone through one of the FDA’s expedited programs. This has helped place important new drugs into the hands of patients who need them while creating a robust pipeline of therapeutics. For cancer drug developers to effectively use these accelerated pathways they need a clear understanding of the differences between each program and what regulators are looking for based on established criteria and past approvals.  

Key Strategic Points:

  • An expedited program requires greater attention to the features of any malignancy being studied, as well as to trial design and regulatory requirements.
  • The agency’s two new pilot programs, with their ability to shorten approval times by months, are already seeing increased popularity, especially when granted in combination with other expedited programs.
  • For companies choosing to pursue one of these pathways, early and regular discussions with FDA personnel are recommended, including about clinical trial design

Background

Adopted in 1992, The Accelerated Approval pathway was the first of the FDA’s expedited programs. Bicalutamide (Casodex) is one of the earliest examples of an oncology drug to take this pathway, in 1995, for the treatment of prostate cancer, is one of the earliest examples of an oncology drug to gain approval via this pathway. for the treatment of prostate cancer. One of the fastest oncology approvals has been imatinib mesylate (Gleevec), in May 2001, for the treatment of chronic myeloid leukemia. The FDA’s review and approval of Gleevec took approximately a mere 2.5 months.

More recently in 2019, the FDA granted Accelerated Approval for the immunotherapy atezolizumab (Tecentriq) in combination with chemotherapy for initial treatment of advanced triple-negative breast cancer. This is the first FDA-approved regimen for breast cancer with an immunotherapy. Shortly afterward, the drug also received approval for treatment of small-cell lung cancer (SCLC), another condition that is particularly challenging to treat.

These examples show the typical drug candidate that FDA regulators look for - therapeutics that address major unmet needs and that seem to offer a significant advantage over currently available treatments for patients. Although the types of products being studied have greatly expanded, these features remain central to the granting of an expedited review.

Expedited Programs

The FDA has four established and two pilot expedited programs from which drug developers can select from.  Although at first look, the differences between these programs may seem subtle, they each have important distinctions. In addition, some can be used in conjunction with one another.

When developing a regulatory strategy and plan for applying for an expedited program, it can be advantageous to seek out an experienced life science consulting firm. By taking advantage of their specialized knowledge of oncology drug development and regulatory strategy, you can improve your odds of success and significantly decrease the chance of a costly error in time or financial capital. Ultimately, teaming up with an expert will help ensure your drug program is setting itself up to avoid surprises and succeed with its application in the shortest timeframe possible.

Below each of the FDA’s expedited programs are summarized.

Accelerated Approval is a pathway specifically intended for drugs with long-term end points, such as increased survival or decreased morbidity, which are hard to measure in clinical trials. It also allows approval based on surrogate end points such as tumor shrinkage. Post-market clinical trials are required, and such drugs can be subject to expedited withdrawal.  Developed in 1992, this was the first FDA expedited program.

Fast Track designation facilitates development and expedites review of drugs to treat serious conditions and fill an unmet medical need. A Fast Track designation can be requested as early as during the Investigational New Drug (IND) application.

Breakthrough Therapy designation applies to drugs for treatment of serious conditions that show a significant early clinical effect. Implemented in 2012, drugs granted this designation may be able to skip portions of the standard FDA review process.

Priority Review cuts the FDA review period of a drug from 10 to 6 months. This designation should be requested with the submission of a BLA or NDA. Drugs that qualify for Fast Track, Breakthrough Therapy, and Accelerated Approval are also eligible for Priority Review.

The Real-Time Oncology Review Pilot Program (RTOR) began in mid-2018. It is designed for drugs that are “likely to demonstrate substantial improvements over available therapy” and are undergoing “straightforward study designs.” It allows the FDA access to key data prior to the official application for approval.

The Assessment Aid Pilot Project (AAid) is a voluntary submission to support assessment of the NDA/BLA. The goal of the program is to focus the FDA’s review on critical thinking while increasing review efficiency and consistency, as well as to decrease review time spent on administrative tasks such as formatting. The AAid template is sent to the applicant during the IND stage and submitted with the NDA/BLA. AAid and ROTR can be pursued simultaneously.

Conclusion

In conclusion, the FDA expedited regulatory pathways provide oncology drug developers with an avenue for getting needed cancer drugs to market and into the hands of patients more quickly. But the process is complex, requires early planning and a solid regulatory strategy, if success is to be achieved and precious time-to-market gained. With nearly half of recently approved oncology drugs gaining approval through an expedited program, the data shows that it has never been more crucial to have these programs as a key part of your strategy. 

View a more detailed white paper on the topic here: https://www.alacrita.com/whitepapers/fda-expedited-programs-for-cancer-drug-development-dont-believe-the-doubters

About us: 

Alacrita is a life science consulting firm providing drug development, commercialization and business development services to the pharmaceutical and biotech industries. We have a presence in both the United States and Europe and serve clients worldwide.