FDA Tallies 53 Novel Drug Approvals in 2020
Despite challenges from balancing resources to navigate the Covid-19 pandemic, FDA approved 53 new molecular entities in 2020, which is five (10%) more than it approved in 2019 and six shy of 2018’s record 59 approvals. A quick look at the approvals shows that many were for treating cancer and rare diseases and benefited under FDA’s fast track program. This speeds new drug development and review by increasing communication between FDA and drug developers, and by enabling CDER to review portions of a drug application before it is fully submitted. CDER also designated many novel drugs in 2020 as breakthrough therapies. A breakthrough therapy designation includes all the fast track program features, as well as more intensive FDA guidance on an efficient drug development program.
FDA has benefited in resource growth during the pandemic and saw about 1,000 net growth in staff due to a streamlined hiring spree authorized by the recent CURES Act. “For FY 20, the FDA hired 2,308 staff (and lost 1,281) with a total net gain of 1,027, compared to a FY19 net gain of 135,” FDA commissioner Stephen Hahn told a Friends of Cancer Research virtual annual meeting 11/13. “We have also made significant progress,” he said, “with the percentage of CURES hires from outside the agency, as 54% of our CURES hires have been external. This represents an important achievement for the agency especially at a time when our focus has rightfully been on the global pandemic.”
Hahn noted that drug approvals had not slowed down due to Covid-19. As of 10/30, CDER had approved 42 novel drugs, including 15 new drugs to treat patients with various forms of cancer. “Several of these approvals have been ‘firsts’ for oncology,” he said, “including: The first FDA-approved therapy, Tabrecta (capmatinib), to treat non-small cell lung cancer with specific mutations; Retevmo (selpercatinib), the first therapy approved specifically for lung and thyroid cancer patients with the RET gene alterations; and Tazverik (tazemetostat), the first treatment option specifically for patients with epithelioid sarcoma, a rare soft tissue cancer that often occurs in young adults.”
Concern about an increase in complete response letters due to facility inspection delays during the pandemic led to a new FDA guidance entitled Review Timelines for Applicant Responses to Complete Response Letters When a Facility Assessment is Needed During the Covid-19 Public Health Emergency. The document applies to applicant responses to complete response letters when a facility assessment is necessary before FDA can take action on a marketing application. Specifically, the guidance explains how FDA will determine review timelines following the issuance of a complete response letter when a facility assessment is necessary for the agency’s regulatory decision on an original or supplemental application. The guidance applies to inspections of manufacturing facilities and also to bioresearch monitoring program sites conducting clinical, analytical, and nonclinical studies.
Last month, CDER Office of Compliance director Donald Ashley said that FDA is continuing to explore using “remote regulatory assessments” in lieu of conducting physical facility inspections during the Covid-19 public health emergency. Speaking during a virtual Food and Drug Law Institute conference 12/16, Ashley discussed how CDER has adapted to the changing times by increasing its use of alternative tools to conduct oversight and take regulatory actions, such as sampling of drugs, relying on information shared by its foreign regulatory partners, requesting records and other information from drug manufacturers, clinical investigators and any other parties that are normally subject to inspection, and remote regulatory assessments.