Personalized medicines advanced by FDA in 2019 address root causes of rare diseases

In a report released today (February 21), the Personalized Medicine Coalition (PMC) explains how 12 personalized treatments the US Food and Drug Administration approved or cleared in 2019 will improve patient care and make the health system more efficient by addressing root causes of rare diseases, expanding treatment options for cancer patients, and targeting therapies to responder populations.

The FDA’s Center for Drug Evaluation and Research (CDER) approved 48 new molecular entities (NMEs) in 2019. All but four of these NMEs are therapeutic products (the others were diagnostic agents). Of the 44 therapeutic NMEs, 11 of them (25%) are personalized medicines as classified by the Personalized Medicine Coalition.

These approvals continue a trend that began in 2014, when PMC classified 21% of NMEs as personalized medicines. The trend accelerated in 2015, 2016, 2017 and 2018, when the Coalition classified 28%, 27%, 34%, 42% of NMEs, respectively, as personalized medicines.