Number of drugs approved by the FDA reaches record-breaking level

A record 56 drugs were approved by the FDA last year, a number that topped the previously held record of 53 approvals from 1996, according to a new analysis by Express Scripts.

A majority of the drugs approved last year were considered by the FDA to be novel new drugs — those which are considered innovative and are seen as advancing clinical care beyond where it has been in the past. Of the 45 novel new drug approvals in 2015, about 36% were first-in-class medicines, including Addyi, which treats hypoactive sexual desire disorder in women; Corlanor, a tablet aimed at reducing hospitalization due to worsening heart failure; and the first U.S. biosimilar, Zarxio. In addition, 64% of 2015’s novel new drugs are treatments that were approved in the United States before being approved in any other country.

In addition to the number of approved drugs that were the first-of-their-kind or first approved in the United States, the FDA touted the large number of special designations that its approved drugs received. Fourteen of the novel new drugs were given Fast Track status, applied to drugs that meet an unmet medical need. These medications in 2015 ranged from treating aggressive nonresponsive colorectal cancer to perinatal, infantile and juvenile onset hypophosphatasia.

Twenty-four drugs were given Priority Review status — which the FDA grants to drugs that it hopes to review in the span of six months rather than the standard 10 months — and 10 were designated Breakthrough therapies, which showed clinical evidence that it might result in a substantial improvement in at least one clinically significant endpoint. Additionally, six were given accelerated approval for treating serious or life-threatening illnesses that offer benefits over a current treatment.

“These new products contribute to quality of care, greater access to medication, more consumer choice and a competitive marketplace that enhances affordability and public health,” the FDA’s director of the Center for Drug Evaluation and Research Janet Woodcock wrote in the introduction to the FDA’s novel new drug overview, adding that the approvals “will offer much to patients in need.”

Many of the patients in need in 2015 were part of small patient populations — about 47% of the novel new drugs were approved for the treatment of orphan diseases, which affect fewer than 200,000 people. Among the highlights, Express Scripts pointed out in its analysis of approved drugs was the introduction of the first biosimilar in the United States, Zarxio (filgrastim-sndz), which was approved by the FDA in March 2015.

Additionally, Express Scripts noted the approval of a new class of specialty cholesterol treatments known as PCSK9 inhibitors, which count among its ranks Amgen’s Repatha (evolocumab) and Sanofi’s and Regeneron’s Praluent (alirocumab). Though innovative, the injectable drugs face high levels of scrutiny based on cost, as one year of treatment is estimated to cost as much as $14,000 per patient per year. “Numerous ongoing clinical trials are looking at their use in concert with statins,” Express Scripts wrote. “If trials show they improve cardiovascular outcomes, use of these therapies could be extensive. Anywhere from 10 million to 20 million Americans may be candidates for therapy with a PCSK9 inhibitor.”

Part of what the FDA’s former acting director Dr. Stephen Ostroff attributed the high number of approvals of new therapies to is the streamlining of the approval process that the agency has undertaken in recent years.

“The number of approvals, and the agency’s ability to review products efficiently, continue to be buoyed by the FDA’s expedited development and review programs,” Ostroff wrote. “When we talk to drug and device makers at the early stages of development, and apply better regulatory science to our ultimate review of their applications, products that are likely to fail are weeded out, allowing manufacturers to focus on those more likely to attain approval.”

This can be a boon for manufacturers, as the Pharmaceutical Research and Manufacturers of America has pointed out that only 12% of drugs that enter clinical trials end up making it to patients — and that’s after the average 10 years and $2 billion it costs to develop a new medicine.

“We are seeing these exciting new medicines because companies are continuing to identify new ways to improve the R&D process,” PhRMA said in an analysis of the FDA’s novel new drug approvals. “The new medicines approved in 2015 demonstrate researchers’ success in delivering on the promise of science. But they also remind us how important it is to continue to foster innovation so that we can continue to deliver for patients in the coming years.”