WASHINGTON. D.C.—The Personalized Medicine Coalition (PMC) in late January released a report documenting the record number of new personalized medicines the U.S. Food and Drug Administration (FDA) approved last year, making 2017 the fourth consecutive year that personalized medicines accounted for more than 20 percent of all new drug approvals.
The annual analysis, titled “Personalized Medicine at FDA: 2017 Progress Report,” shows that FDA approved a record number of 19 personalized medicines—16 new molecular entities and three gene therapies—in 2017.
Specifically, the report lists a total of six regulatory precedents FDA set last year, as follows: (1) A record number of 16 personalized medicines approved as new molecular entities; (2) the approval of first three gene therapies; (3) the first approval of a tissue-agnostic indication for cancer therapy; (4) the first authorization for marketing of health-related genetic tests directly to consumers; (5) the first approval of a personalized medicine biosimilar; and (6) the first FDA/CMS joint approval and coverage decision for a next-generation sequencing test.
PMC President Dr. Edward Abrahams said the precedents demonstrate how personalized medicine has reshaped drug development in the decade since 2007, when targeted therapies accounted for less than 10 percent of new drug approvals.
As the PMC points out, an influential article published in 2007 in the Harvard Business Review titled “Realizing the Promise of Personalized Medicine,” for example, suggested that FDA was not yet committed to the paradigm. The pharmaceutical industry, the article noted, was at that time hesitant to develop medicines for smaller patient populations, preferring instead to develop “blockbuster” medications that could earn approval for one-size-fits-all applications.
This obviously is no longer true, the PMC remarks, though there remain many obstacles—notably regarding regulation, reimbursement, access and clinical adoption—that complicate the commercialization of personalized medicine products.
“Despite myriad challenges, the diagnostic and pharmaceutical industries are deeply invested in making healthcare more effective and efficient by developing products that guide treatments to only those patients who will benefit from them,” Abrahams explained. “As this report shows, FDA is increasingly committed to supporting that effort.”