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FDA issues 2 guidances to advance development of new therapies

In an effort to modernize clinical trials and advance the development of safe and effective drugs and biologics, the Food and Drug Administration on Monday released two guidance documents to assist drug companies that are developing these next generation of therapies and treatments.

The FDA’s draft guidance entitled, “Hematologic Malignancies: Regulatory Considerations for Use of Minimal Residual Disease in Development of Drug and Biological Products for Treatment,” is aimed at sponsors planning to use minimal residual disease, or MRD, as a biomarker in clinical trials of drugs or biologics to treat specific blood cancers.

“MRD as a general measure of tumor burden has multiple potential regulatory and clinical uses as a biomarker to help with more informed drug development,” FDA commissioner Scott Gottlieb said in a statement. “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.”

The FDA also issued a final guidance, “Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease,” which was issued as draft guidance in December 2017. The guidance addresses the topic of finding treatments that address the underlying molecular changes, e.g., genetic variants, that often cause or contribute to diseases, including uncommon or rare molecular changes that are present in a small subset of patients.

“By providing clarity on the regulatory and scientific frameworks for product developers, safe and effective targeted treatments can be identified with scientifically valid tests and, ultimately, made available to patients more efficiently,” Gottlieb said.
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