Public Release: 

Precision medicine approvals and rare disease treatment incentives evaluated

Brigham and Women's Hospital

WHO: Aaron Kesselheim, MD, MPH, Director, Program On Regulation Therapeutics And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital

WHAT: Kesselheim, the senior author of two separate articles published in the May issue of Health Affairs, members of the PORTAL research group and co-authors, examine the fast approvals of precision medicines in one paper while evaluating the impact of the Orphan Drug Act's seven-year market exclusivity in the other.

"Precision Medicines Have Faster Approvals Based on Fewer and Smaller Trials Than Other Medicines," Lisette Pregelj et al, and Aaron Kesselheim

The outcome of clinical trials evaluating the effectiveness of precision medicines suggests such medicines, which can offer successful individualized treatments benefitting patients, will reach the market more rapidly than traditional drugs.

In this study, researchers examined the development and review of precision medicines by the FDA from 2013-2017. They found the process to be almost two years faster than comparable medicines, in part due to various special accelerated or priority pathways like the "breakthrough therapy" designation for which these drugs qualify.

In addition, however, the authors found that precision medicines were approved based on fewer pivotal trials of these drugs that enrolled fewer patients, and the trials were less likely to be blinded, randomized or controlled by comparing to placebo. The authors note that these trends raise separate questions about the quality and reliability of the data used for FDA approval.

Kesselheim also authored a study published in April in the Journal of Clinical Oncology, that showed how breakthrough-designated cancer drugs were associated with faster review and approval times, but no apparent improvement in safety or effectiveness when compared with traditional drugs.

"Evaluating The Impact Of The Orphan Drug Act's Seven-Year Market Exclusivity Period," Ameet Sarpatwari, PhD, assistant director, PORTAL, BWH et al, and Aaron Kesselheim

Among other incentives, the Orphan Drug Act of 1983 provides seven years of market exclusivity, which many believe acts as a strong incentive for pharmaceutical manufacturers to invest in drugs to treat rare diseases.

This study reviewed a cohort of drugs approved in the period from 1985-2014 that had at least one indication for an orphan-designated disease as of January 1, 2017. The investigators found that patent protection for these rare disease drugs often far outlasts the seven-year period provided under the Orphan Drug Act. The authors concluded that the explosion in new drugs for rare diseases is being incentivized by scientific advances and other features of the market, and not the statute's market exclusivity provision. The authors recommend that policymakers looking to replicate the Orphan Drug Act's success in other fields should leverage these other factors and not replicate the Orphan Drug Act's exclusivity provision.


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