The FDA approved 46 drugs in 2017, tying a record set for approvals in 2015. Cancer medications led the way, with 16 oncology and 11 hematology drugs approved. There were also 11 approvals for medications to treat infectious diseases, along with drugs to treat a cross-section of conditions, from cardiovascular disease to rheumatoid arthritis, Parkinson’s disease to constipation. Final decisions were coming through right up until the end of December. An important question, then, is whether this trend will continue into 2018 and beyond. Another is why this explosion in approvals occurred at all. Perhaps another is how practicing clinicians can keep pace, with nearly an approval a week coming through.
Andrew Powaleny, director of Public Affairs at PhRMA, speculated on the 2017 boom. “FDA commissioner Scott Gottleib focused on improving efficiency and enhancing competition in the marketplace,” he said. “He expedited the generic approval process, promoting more high-quality generic applications.”
Christopher P. Molineaux, president & CEO of Life Sciences Pennsylvania, agreed. “Under Dr. Gottlieb’s proactive leadership, the FDA has made changes to further improve and enhance the FDA approval process,” he said. “Those changes include robust engagement with the patient population at certain points in the approval process, which is an exciting new component in review of new drug applications. It will be interesting to see how that affects approvals and the process overall.”
Molineaux added that embedding experts within the review teams and making more use of real-world evidence will bring positive results to the process. “It is important to note that there are over 7,000 drugs in various stages of development in America’s pipeline,” he said. “Having an FDA ready and able to cope with these future applications is necessary and needed.”
Whether these changes, and the record number of approvals seen in 2017, will translate to a similar pattern remains to be seen. Healio.com reached out to the FDA for comment. The administration announced in an end-of-year press release that goals for 2018 include dealing with nicotine addiction, advancing drug and food safety, empowering consumers with more information and increased access to non-prescription medications, modernizing standards for regulations — including syncing U.S. standards with those in other parts of the world — and making greater use of technology. How those goals might translate to the approval process, however, remains to be seen.
Expectations for 2018
Neither Molineaux nor Powaleny would hazard a guess as to whether 2018 would break records yet again, but both referenced the Prescription Drug User Fee Agreement (PDUFA) as an important factor in the FDA approval process. Powaleny noted that the law was borne from the HIV/AIDS crisis in the 1990s. “The law was built out of a need to bring treatment to patients in a timelier manner without compromising safety and efficacy,” he said. “With more than 1,500 new drugs and biologics for many specialties approved, it has been a successful program. It has allowed the FDA to hire and maintain the best and brightest talent, who are developing new and innovative ways to structure clinical trials, how we diagnose things. This is moving the agency to a more efficient process and speaks to what we saw in 2017.”
Molineaux added that forecasting how applicants will fare in the FDA approval process is difficult. “We are hopeful that with the reforms found in the 21st Century Cures legislation and the new funding provided by PDUFA, the FDA will continue to make the process more efficient and improve the timeliness of the application review,” he said.
However, Molineaux noted that it is too soon for the changes in the 21st Century Cures legislation to show any meaningful impact on 2017 approvals, as it was only signed into law in December 2016. “Furthermore, the new funds provided by PDUFA VI have had even less time to make an impact since becoming law in the summer of 2017,” he said. “However, I do anticipate positive impact on the process in 2018 and hopefully more approvals.”
Both experts stressed that it is not necessarily about the numbers. “There is ebb and flow every year,” Powaleny said. “In 2016, there were 22 approvals.”
“I will point out that in 2016 — the year following the record approvals of 2015 — FDA approvals fell to a 6-year low,” Molineaux added.
In the pipeline
There is “a lot of promise in the pipeline,” according to Powaleny, who suggested that nearly three-quarters of the medications moving toward approval are first-in-class. “There are 822 projects designated by the FDA for treating rare diseases,” he said. “We have great hope for 2018.”
Molineaux suggested that the FDA is not the only player in the process. “An efficient FDA is key, but good applications applying good science to human health illnesses cannot be ignored,” he said. “There are lot more factors at play here.”
The diversity of drugs approved in 2017 reflects advances in science in general, according to Powaleny. “As science becomes more challenging and complex, biopharma entities are continuing to persevere,” he said. “They are recognizing that there are great unmet needs Alzheimer’s disease and other complicated conditions. We are also seeing companies focusing on personalized medicine, which could bring a whole new wave.”
Chimeric antigen receptor T-cell therapy and immunotherapy are groundbreaking approaches, according to Powaleny. “We are in an exciting time in science,” he said. “What is interesting is that what we are seeing in the lab is playing out in patient outcomes. Significant medical needs that were previously unmet are being met.”
Regarding physicians staying up to date with the pace approvals, Molineaux was forthright. “Like many information/data intensive professions, we believe the burden is on the medical professional to keep apprised in the latest drugs available for patients,” he said. – by Rob Volansky
For more information:
Christopher P. Molineaux can be reached at 650 E. Swedesford Rd. Suite 190, Wayne, PA 19087; email: email@example.com.
Andrew Powaleny can be reached at 950 F Street NW Suite 300 Washington, DC 20004; email: APowaleny@phrma.org.
Disclosures: Healio.com was unable to determine relevant financial disclosures at time of publication.