Drug companies experiencing faster reviews from FDA: JAMA

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Trine K. Tsouderos HRI Regulatory Center Leader, PwC US January 24, 2020

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Federal initiatives to speed patients’ access to pharmaceutical products have resulted in faster FDA reviews and a shift in the level of evidence needed to gain approval over the past 40 years, a study in the Journal of the American Medical Association (JAMA) has found.

Still, the number of annual new drug approvals hasn’t increased substantially, the study notes. And while FDA review times are shorter, overall clinical development times have not shrunk, perhaps because of increased emphasis on drugs in more challenging therapeutic categories and for rare diseases.

Eighty-one percent of new drugs benefited from at least one of the expedited-approval programs in 2018, the last year included in the study. FDA review times have dropped from more than three years in the early 1980s to just over 10 months for standard applications and 7.6 months for priority applications.

The initiatives also loosened the required level of evidence by allowing the FDA to approve drugs after a single clinical trial and to use surrogate measures, such as lab tests and imaging studies, to assess results.

About half of new approvals were supported by at least two pivotal trials in 2015-17, down from almost 81 percent in 1995-97, the study notes. Surrogate measures were used in the evaluation of almost 60 percent of new drugs approved in 2015-17, up from 44 percent of those approved in 2005-12.

HRI impact analysis

In all, the programs “allow the approval of new drugs based on fewer, smaller, and/or earlier-stage clinical trials that may not be randomized, controlled, blinded, or based on traditional measures of how a patient feels, functions, or survives,” the authors write.

The programs – focused on new therapies for rare, serious and life-threatening conditions – were a response to growing concern that the slow review process was failing patients. The changes began with the Orphan Drug Act of 1983 and include the Fast-Track, Accelerated Approval, Priority Review and Breakthrough Therapy programs.

Aiding in their success is funding generated by the Prescription Drug User Fee Act of 1992, which gives the FDA the resources needed to meet the new demands. Fees have jumped from $29 million in its first year to $908 million in 2018, the study finds.

The benefits of quicker access to new drugs should be weighed periodically in an evidence-based manner against the potential risk of relying on less substantial evidence for approval, the authors say. Drug efficacy might vary between clinical trial patient panels and broader post-approval patient populations, and established drugs might work better in certain patient populations than new therapies, they add.

Real-world data – information harvested from insurance claims and bills, electronic health records, and even wearable medical devices – could serve as a valuable tool in assessing the impact of quicker approvals on patient risk. Already the federal government and industry players recognize and are investing in the use of real-world evidence not only to study the efficacy of approved drugs, but also as part of the approval process for new drug indications.

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Trine K. Tsouderos

HRI Regulatory Center Leader, PwC US

Tel: +1 (312) 241 3824

Crystal Yednak

Senior Manager, Health Research Institute, PwC US

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