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FDA Announces Availability Of The Draft Guidance, “Developing Targeted Therapies In Low-Frequency Molecular Subsets Of A Disease”

December 21, 2017

News from the FDA

December 21, 2017

On December 15, 2017, the U.S. Food and Drug Administration (FDA) announced the availability of the draft guidance for industry entitled "Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease". This draft guidance describes:
  • The FDA's current recommendations on how to group patients with different molecular alterations for eligibility in clinical trials thereby increasing the likelihood of finding viable treatment options for those with less common alterations; and
  • General approaches to evaluate the benefits and risks of targeted therapies within a clinically defined disease where some molecular alterations may occur at low frequencies which could lead to more consistent development and approval of targeted therapies for patients who are likely to benefit from them.

Insights into the molecular basis of disease have led to the development of targeted therapies. Targeted therapies are drugs that act on the molecular changes that cause or contribute to a disease (e.g., changes in DNA, RNA, or proteins). Some diseases can be caused by many different molecular changes, some of which can be very uncommon and too infrequent in the disease population to feasibly study in the context of a traditional randomized controlled clinical trial. While drug developers have been able to develop targeted therapies to treat many genetic diseases and cancers, it is often difficult to determine whether or not the drug is effective for certain patients whose disease is caused or affected by uncommon molecular changes. This draft guidance will provide drug developers greater clarity on the FDA's recommendations for researching and developing new targeted therapies.

The "Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease" draft guidance is available at Please refer to the guidance for more details.

FDA is publishing this draft guidance to collect public comments. You may submit your comments to this guidance by February 16, 2018 to the Docket No. FDA-2017-D-6617 available at Your comments do make a difference and can impact the outcomes of FDA regulatory policy. Share your knowledge and experience, and make your voice count.

We always welcome your thoughts regarding the format, content, and utility of the communication. Comments may be sent via email to

This communication was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.

In cooperation with the Food and Drug Administration (FDA), and as a service to our members, ACCP will periodically distribute information about newly approved therapies or significant changes to approved therapies. This helps the FDA to inform professionals in the patient care arena of recent approvals in a timely manner. Included in the message from the FDA is a link to the product label, which will provide the relevant clinical pharmacology information on the indication, contraindications, dosing, and safety. In sharing this information, ACCP does not endorse any product or therapy and does not take any position on the safety or efficacy of the product or therapy described.