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Innovation comes in many forms, including new and expanded uses of existing medicines

Emma Van Hook   |     January 29, 2018   |   SHARE THIS

2017 was a record-breaking year for new drug approvals, with the U.S. Food and Drug Administration (FDA) approving 56 new drugs and biologics. But the innovation did not stop there. As the FDA highlighted in their annual report on new medicines, many new and expanded uses of already approved medicines are providing patients and prescribers with important new treatment options.

While the initial approval of a new medicine is a landmark milestone, based on rigorous demonstration of safety and efficacy, research does not stop there. Additional knowledge and understanding of a medicine continues to build over time, through additional study and collection of data. This ongoing research can culminate in expanded uses of medicines, in new patient populations, new indications (e.g., different types of cancer), as well as new dosage forms or alternative delivery systems (e.g., making a previously injectable medicine available in an inhalable form).

These new medicines can have a significant impact on patient care. Some notable approvals in 2017 include:

  • First Cancer Treatment Approved for Tumors with a Common Biomarker
    • An immunotherapy, originally approved for a specific form of advanced melanoma in 2014, has since received approval for a number of types of cancer. In 2017, a new indication was approved, marking the first time a medicine has been approved for tumors with a specific genetic biomarker, regardless of the site of the cancer in the body.

  • First Therapy for Ultra-Rare Inflammatory Disease
    • Although initially approved as a maintenance treatment for asthma, researchers discovered that a medicine also treats an extremely rare disease called eosinophilic granulomatosis with polyanglitis (EGPA). This is the first treatment specifically approved for this disease, which causes extensive damage to organ systems.

  • Expanded Use of Breakthrough Cystic Fibrosis Treatment
    • Initially approved to treat a narrow population of cystic fibrosis (CF) patients, a medicine received a significantly expanded indication this year, to treat patients with one of 33 CF mutations (increased from ten previously).

  • Pain Medications Formulated to Help Prevent Abuse
    • Three abuse-deterrent formulations of opioid medications were approved in 2017, designed to deter abuse by making abuse of the drug more difficult or less rewarding.

  • New Sensor Technology
    • A product combining a medicine to treat serious mental illness with an electronic sensor that allows tracking of when medicine has been ingested was approved this year. This new technology has the potential to help patients take their medicine as prescribed, which is critically important in patients with these difficult to treat conditions.

  • New Combination Treatment for Blood Cancer Reduces Treatment Burden
    • A new formulation of a cancer medicine, combined with an enzyme, can be delivered in a single injection under the skin in just five to seven minutes. The medicine, which previously took hours to administer via an intravenous infusion, offers a significant quality of life improvement for patients while maintaining comparable efficacy.

From developing entirely new medicines to finding new dosage forms and new ways to use medicines, biopharmaceutical researchers are committed to finding innovative ways to prevent and treat disease. To learn more about the innovation happening inside America’s biopharmaceutical companies, visit innovation.org.

Emma Van Hook

Emma Van Hook Emma Van Hook is a director of Policy & Research at PhRMA focusing on the promise of the biopharmaceutical pipeline, the value of medicines, the R&D process, and personalized medicine. Emma is a biology nerd at heart and has worked in both bench science and in regulatory affairs, helping to get new medicines through the development and FDA approval processes. In her free time, Emma likes to get outside the Beltway for running and hiking with her family and her dog Blue.

Topics: Research and Development, FDA

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