FDA Approval, Regulation Process Accepts Less Data, Shortens Review Times

Report demonstrates an increase in generic and new drug approvals, Orphan Drug designations, and an increase in the use of expediated approval programs.

Over the past 4 decades, the FDA’s approval and regulation process has evolved and increased in complexity, causing the federal agency to accept less data and more surrogate measures as well as shortening its review times.

US law requires testing of new drugs before approval to ensure that they provide a well-defined benefit proportionate to risk. In recent years, the FDA has had challenges in achieving an appropriate balance between rigorous testing and the need for timely drug approvals with benefits that outweigh risk.

The analysis examined the evolution of laws and standards affecting drug testing, the use of new approval programs and standards, expansions of the FDA’s role and authority, and changes in the number of drug approvals between 1980 and 2018.

Researchers used data from principal federal laws and FDA regulations (1962-2018); FDA databases of approved new drugs (1984-2018); generic drugs (1970-2018); biologics (1984-2018); vaccines (1998-2018); special development and approval programs; and user fees paid to the FDA by industry (1993-2018).

Drugs that benefited from expediated development or review programs were then identified by examining the FDA’s annual new drug approval reports (2011-2018) and the Drugs@FDA database (1984-2010).

The researchers found that from 1983 to 2018, legislation and regulatory initiatives substantially changed drug approvals at the FDA. Additionally, the annual number of generic drugs approved has increased to more than 700 per year, and the mean annual number of new drug approvals, including biologics, increased from 34 from 1990-1999 to 41 from 2010-2018.

The number of new drug approvals has remained at or below 60 per year, whereas the proportion of drugs approved using at least 1 special review program increased substantially over time, exceeding 80% in 2018. The proportion of drugs approved with an Orphan Drug Act designation also increased from 18% in 1984-1995 to 41% in 2008-2018.

The proportion of new approvals supported by at least 2 pivotal trials decreased from 80.6% in 1995-1997 to 52.8% in 2015-2017, based on 124 and 106 approvals, respectively.

The use of Accelerated Approval, Fast-Track, and Priority Review programs for new drugs has increased over time, with 81% of new drugs benefiting from at least 1 expedited review program in 2018.

The study authors noted that these expedited approval programs create substantial administrative costs and allow the approval of new drugs based on fewer, smaller, and earlier-stage clinical trials that may not be randomized, controlled, blinded, or based on traditional measures of how a patient feels, functions, or survives, according to the study authors.

“Achieving the proper balance between the speed of development and rigorous evidence generation requires careful construction of regulatory requirements, proper execution of these rules, and evidence-based evaluation of whether new programs are truly benefitting the public,” the study authors concluded in the report.


Darrow JJ, Avorn J, Kesselheim AS. FDA Approval and Regulation of Pharmaceuticals, 1983-2018. JAMA. 2020; 323(2):164—176. Published January 14, 2020. doi:10.1001/jama.2019.20288. Accessed January 24, 2019.

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