Putting 2018 medicine approvals in context

Andrew Powaleny
Andrew Powaleny January 23, 2019


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Last year, the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved a record number of novel new medicines with 59 new molecular entities (NMEs). While medicine approval numbers vary each year, 2018 was a particularly exciting year for drug development. These novel approvals demonstrate biopharmaceutical research companies’ continued commitment to developing innovative medicines and highlight the FDA and Commissioner Gottlieb’s commitment to strengthening the Agency’s human drug review program through ongoing implementation of the Prescription Drug User Fee Act (PDUFA). CDER met all its PDUFA goal dates for novel drugs in 2018, further increasing the Agency’s regulatory review efficiency and predictability and providing patients timely access to safe and effective new medicines.

Novel medicines are critical to advancing patient care, as they present new opportunities for treating previously unmet needs and diseases. 32 percent of NMEs approved in 2018, were deemed first-in-class, utilizing previously unavailable approaches to treat illnesses. These much-needed treatments approved by the FDA in 2018, include medicines for HIV, migraine, leukemia, endometriosis, malaria, prostate cancer and more.

Of the 59 novel medicine approvals, 43 were designated in one or more expedited approval categories, including approvals for 14 breakthrough therapies. Breakthrough therapies are a key component of PDUFA VI, with the FDA investing dedicated resources to prioritize the development and availability of medicines for patients with serious and life-threatening diseases. Furthermore, of these 59 NMEs, 42 were first approved in the U.S. before receiving approval in any other country.

CDER approved many unique treatment options for patients in need, including 34 new approvals for patients with rare diseases which are defined as affecting fewer than 200,000 people in the U.S. These approvals included treatments for such conditions as rickets, Fabry disease and phenylketonuria (PKU). It’s worth noting that while drug development is already challenging, developing a treatment for a rare disease is even more so, with a Tufts Center for the Study of Drug Development finding it takes 2.3 years, or 18 percent, longer to develop an orphan drug compared with medicines to treat more common conditions.

The 2018 approvals demonstrate that innovation takes many forms. Newly approved uses of existing medicines reflect ongoing research on medicines and provide new options to more patients. For example, a medicine for rheumatoid arthritis was approved to treat patients with ulcerative colitis, a chronic inflammatory bowel disease.

Other approvals built on existing options by offering new formulations or dosage forms. For example, a new formulation of a schizophrenia medicine treats symptoms more quickly by allowing patients to ramp up to full therapeutic dose immediately rather than the gradual increase that were required before.

As we look ahead to 2019, last year’s approvals serve as a reminder of the remarkable promise of the biopharmaceutical research pipeline. Biopharmaceutical companies are on the forefront of medical innovation – innovation that must be protected by strong intellectual property policies and a competitive market that rewards value.

Click here to learn more about innovation from America’s biopharmaceutical companies.

Topics: Research and Development, FDA, Rare Diseases, PDUFA, New Era of Medicine