New guidance aims to modernize FDA approval process

Mar 12, 2019
New guidance aims to modernize FDA approval process

Two new pieces of clinical trial guidance from the Food and Drug Administration (FDA) aim to make the drug development and approval process as innovative as today’s medical science.

A final version of Developing Targeted Therapies in Low-Frequency Molecular Subsets of Disease and a draft of Hematologic Malignancies: Regulatory Considerations for Use of Minimal Residual Disease in Development of Drug and Biological Products for Treatment were published in October 2018.

In an online statement, FDA Commissioner Scott Gottlieb, M.D., says the “impressive field of new therapies” approved in recent years, such as cell- and gene-targeting treatments, has grown out of scientists redefining diseases based on their “molecular underpinnings”.

The statement says the agency is focusing on modernizing its approach to the design of clinical trials, as well as making the overall drug development and regulatory process more efficient and less costly.

As Dr. Gottlieb notes: “By using more sophisticated approaches to learn about the safety and efficacy of treatments, we can reduce the barriers to bringing new science forward and make sure more patients can benefit sooner from improved treatments.”

Final guidance

Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease is published in its final form, following draft guidance issued in December 2017.

It provides developers with greater clarity on the FDA’s recommendations for researching and developing the next generation of individualized therapies, Dr. Gottlieb points out.

He adds: “The guidance discusses an approach for drug developers to enroll patients based on the identification of rare variants into clinical trials for targeted therapies when reasonable scientific evidence suggests the drug could be effective in patients with these genomic findings.

“It also discusses the evidence needed to demonstrate effectiveness for a variety of molecular subsets within a particular disease. This approach could lead to more consistent development and approval of targeted therapies for patients who are likely to benefit from them.”

Draft biomarker advice

The Hematologic Malignancies draft guidance, developed through a series of stakeholder workshops, offers sponsors advice on the use of minimal residual disease (MRD) as a biomarker in marketing applications.

A general measure of tumor burden, MRD is often used in studies of drugs or biologics intended to treat blood cancers, but is applied inconsistently across clinical trials.

“Depending upon the clinical setting, MRD may reflect a patient’s response to treatment or it may be used as a prognostic tool to assess the risk of future relapse.

“As such, it may be considered for use to enrich clinical trial populations, or to guide allocation into specific treatment arms in clinical trials and could be developed as a potential surrogate endpoint,” observes Dr. Gottlieb.

“Improving every stage of drug development”

The two documents are part of the FDA’s wider aim to modernize its programs to ensure ground-breaking new drugs are made available to the patients who need them as quickly as possible, notes the statement.

“Our comprehensive efforts are aimed at improving every stage of drug development. We’re focused on making the process of generating pre-clinical and clinical evidence required for making risk-based regulatory decisions more modern, more scientifically rigorous, and more efficient.

“The scientific opportunities we’re seeing today demand that we make sure our policies are as sophisticated as the treatments that are being developed,” explains Dr. Gottlieb.


New guidance aims to modernize FDA approval process

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