Precision medicines have faster approvals based on fewer and smaller trials than other medicines.
A new study, led by researchers from The University of Queensland (UQ) in Brisbane, Australia, and the Program on Regulation, Therapeutics, and Law (PORTAL) at Boston’s Brigham & Women’s Hospital and Harvard Medical School reveals that clinical trials and approvals for precision medicines take, on average, two years less than traditional medicines in the US. Shorter development times means cost-savings during new drug development.
‘Precision medicines’ are medicines targeted at specific patients based on their genomic characteristics and mutations, aimed at addressing specific subsets of diseases and enhancing the prospect of effectiveness.
The study, conducted by an international team of seven researchers from institutions in Brisbane and Boston, compared 151 medicines approved by the U.S. Food and Drug Administration between 2013 and 2017, of which 118 were non-precision medicines (78 per cent) and 33 were precision medicines (22 per cent).
Dr Lisette Pregelj, lead author of the study and UQ Post-Doctoral Research Fellow at UQ Business School, said that precision medicines hold the prospect for numerous benefits, and the results from the study could translate to important positive outcomes for industry and patients alike.
“Our findings show that new precision medicines can reach patients faster, which is important when precision medicines offer substantial advantages in effectiveness over non-precision treatments,” Pregelj said.
Author Jonathan Darrow from Harvard Medical School and Brigham and Women’s Hospital suggested a need for cautious optimism: “Although speed-to-market can benefit patients if precision medicines are actually safer or more effective, our data show that the pivotal trials of precision medicines were less likely to be randomized, controlled, or blinded, and involved fewer patients—making actual benefit more uncertain and increasing the need for further evidence development after approval,“ he said.
The study and its findings were published in Health Affairs journal on 7 May 2018. View the original article here.
JOURNAL: Health Affairs
AUTHORS: Lisette Pregelj, Thomas J. Hwang, Damian C. Hine, Evan B. Siegel, Ross T. Barnard, Jonathan J. Darrow, and Aaron S. Kesselheim
