More Than 25% of New Molecular Entities Approved in 2016 Are Personalized Medicines

The Personalized Medicine Coalition (PMC) recently reported that for the third year in a row, personalized medicines accounted for more than 25% of all new molecular entities (NMEs) approved by the US Food & Drug Administration (FDA). Personalized Medicine at FDA, 2016 Progress Report (Report). Personalized medicines are new drugs, agents or therapeutic biologics where a diagnostic test is used to determine which medical treatments will work best for a specific patient. For purposes of the Report, the PMC categorized personalized medicines as those therapeutic products for which the label includes reference to specific biological markers.

PMCs Report specifically indicates that of the 22 NMEs approved by the FDA in 2016, 6 of them are personalized medicines, half of which are oncology drugs. This continues the 3-year trend that the PMC first documented in 2014 when it reported that 9 of the 41 approved NMEs were personalized medicines.

The 6 new FDA-approved drugs are:

1. Rubraca (rucaparib) for the treatment of advanced ovarian cancer;
2. Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy;
3. Epclusa (sofosbuvir and velpatasvir) for the treatment of chronic hepatitis C infection;
4. Tecentriq (atezolizumab) for the treatment of advanced or metastatic urotherlian cancer and metastatic non-small cell lung cancer;
5. Venclexta (venetoclax) for the treatment of chronic lymphocytic leukemia; and
6. Zepatier (elbasvir and grazoprevir) for the treatment of chronic hepatitis C infection.

The Report concludes that while challenges in the areas of diagnostic regulatory policy, reimbursement, and clinical adoption remain, the FDAs approval of these new drugs that rely on diagnostic medicine signals a shift toward a more personalized health care system.