Are Special Programs Driving the FDA Approval Bus?

• The FDA reported approval of 48 novel investigational drugs in 2019, lower than the 59 approved in 2018, but still the third highest total in the past 25 years.​
• Historically, it has been noted that December is the highest volume month for approvals, with 15% of total approvals coming during the month. In 2019 seven drugs were approved in December and 21 approvals (43%) were made in the fourth quarter. The therapeutic areas with the most approvals were oncology and neurology.
• Special Approval Programs have influenced an increase in the pace of approvals of new drugs over the past 40 years.
• The time for the FDA to complete a review of an investigational drug decreased from 3 years in 1983 to 1 year in 2017.
• From 1986 to 1996, 48% of drugs qualified for special approval program, while 80% of drugs qualified for a program in 2018.
• In 2019, priority reviews were given to 58% of the drugs with 44% of the priority reviews being designated an orphan drug, 27% as breakthrough therapies and 19% received accelerated approvals.
• Use of these programs (Orphan Drug, Accelerated Approval, Fast Tracking, Priority Review, and Breakthrough Therapies) has increased, which led to a decrease in FDA Approval time.
• Already this year the FDA has approved two orphan drugs: Aimmune Therapeutics AR101 (Palforzia) and Horizon Therapeutics’ teprotumumab (Tepezza).
• In the mid-90’s, 81% of drugs were approved based on evidence from at least two pivotal trials, but only 53% have data from two trials today. 
• Also there has been an increase in the use of surrogate endpoints with the rate rising to almost 60% of trials reviewed by the FDA.
• Below are links to two studies which analyzed the process for drug approval
• FDA Approval and Regulation of Pharmaceuticals, 1983-2018.
• Compliance with legal requirement to report clinical trial results on ClinicalTrials.gov: a cohort study.