FDA Provides Guidance on Development of Targeted Therapies


The FDA issued new draft guidances to expedite the approval of novel targeted drugs.

The uptake of targeted therapies has drastically changed the field of medicine. Previously, treatments for certain conditions may have been standard for all patients, but now, physicians are able to tailor therapy based on the unique genetic profile of individual patients.

This innovative approach to treatment has resulted in more targeted treatments and includes those that are developed for a specific patient, according to a statement from FDA Commissioner Scott Gottlieb, MD.

The FDA plays a significant role in encouraging the development of novel targeted therapies by developing the necessary framework and guidance.

Dr Gottlieb noted that the FDA needs to clarify and extend the current pathway that allows manufacturers to create drugs based on biomarkers, whereas traditional drug development focuses on the targeted phenotype, according to the release.

Current research indicates that many diseases are caused by a molecular change, so novel drugs are developed to target the subtypes. There is an urgent need for the development of a new pathway that allows innovative drugs to be evaluated for approval in each of the targeted settings, Dr Gottlieb wrote.

Providing updated guidance on the regulatory and scientific framework for manufacturers, targeted therapies can be assessed by validated tests and be offered to patients faster. For these reasons, the FDA issued 2 draft guidances that seek to increase the clarity of the expectations for the research and development of individualized medicine, according to the release.

Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease

The first draft guidance tackles the issue of discovering therapies which target genetic mutations that drive diseases, including uncommon alterations, according to the release. The guidance suggests a novel strategy for drug developers to enroll patients in clinical trials based on the presence of mutations.

The proposal also highlights the need to explore the efficacy of molecular subsets within a certain disease. This could lead to better development and approval of targeted therapies for patients who would experience the most benefit from the drugs, according to the release.

The FDA said the principles discussed in the proposed guidance would also support regulatory approval based on targeting molecular subtypes common in different phenotypes rather than just a disease state. This may lead to the discovery of effective therapies for patients with less common mutations, according to the release.

Investigational In Vitro Diagnostic Devices Used in Clinical Investigations of Therapeutic Products

This proposed guidance gives clinical researchers a framework to follow when exploring whether an in vitro diagnostic (IVD) device needs to be reviewed by the FDA separately from a drug that is used with it, according to the release.

IVDs help assess biomarkers and help researchers develop an optimal treatment strategy for patients in clinical trials, Dr Gottlieb noted.

When finalized, this guidance will determine the pathway for investigational IVDs used in clinical trials. This may strengthen the results of clinical trial results for a novel targeted therapy because it will not be affected by a diagnostic test that did not meet regulatory criteria, according to the release. The goal of the guidance is to make the process more efficient and simplified.

Additionally, the FDA is considering novel ways to streamline the approval of oncology drugs and associated IVDs, with another draft guidance to be proposed in the future.

“By proposing streamlined approaches for our colleagues in the research and development communities, the FDA hopes to enable more efficient access to safe and effective, novel targeted therapies for the patients who need them,” Dr Gottlieb wrote.

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