Personalized Medicines Account for 42% of All Drug Approvals in 2018

In the last 2 years, more than one-third of FDA-approved therapies has included information on the label identifying patients would benefit the most or experience fewer side effects, found a recently published report from the Personalized Medicine Coalition (PMC). The report, “Personalized Medicine at FDA: A Progress & Outlook Report,” proposes that lawmakers could address rising drug prices and healthcare costs by instituting policies that encourage personalized medicines.

In 2018 alone, there were a record number of 25 personalized medicines approved, accounting for 42% of all 2018 drug approvals, according to the report. This uptick in approvals is due in part to policy initiatives advanced by the FDA in 2018 that “demonstrate the agency’s continued commitment to accelerating personalized medicine.”

The report also credits the FDA’s recent implementation of certain expedited review programs. Notably, of the 25 personalized medicines that received FDA approval in 2018, 24 underwent some form of FDA review. In the past year, the FDA also “took steps to advance digital health technologies; modernize the frameworks applicable to the oversight of emerging types of diagnostic tests; and facilitate the use of direct-to-consumer (DTC) genetic testing.”

Notably, DTC genetic testing has allowed, for the first time, a test to determine hereditary cancer risk without the need for a prescription. “To harness these opportunities in science and technology, FDA must create processes that are able to keep pace with [personalized] medicine innovation,” said FDA Commissioner Scott Gottlieb, MD, according to the PMC report.

More opportunities remain for personalized medicines to make a significant difference in patients’ lives in the coming years. To highlight the potential personalized medicine has, the report compiled various achievements these therapies have made in recent years, such as:

• A cure for hepatitis C
• Restored vision loss from congenital blindness
• Improvement in overall survival and progression-free survival rates for patients with breast, lung, and colorectal cancer

However, the findings of the report also demonstrated that challenges in this field of medicine also remain.

“Some emerging public policies still assume that providers and payers will continue to make decisions based on data from population averages, instead of embracing personalized medicine,” according to the report.

Specifically, reimbursement policies for these therapies continues to be an ongoing challenge. The report cited certain policy decisions that have the potential to limit patient access to these medications. For example, last year, CMS announced plans to apply step therapy, which requires a patient to first try cheaper treatments before moving on to more expensive therapies, to Medicare Part B drugs. PMC stated in the report that this proposed policy “ignores” the trend toward therapies that have informative labels about the populations likely to benefit from the treatment.

“Driving treatment decisions by cost considerations rather than determining what treatment would work best for an individual patient fundamentally conflicts with personalized medicine, and in many cases will increase downstream costs brought on by continued progression of disease and more adverse side effects associated with the wrong treatment,” according to the report.

In this instance, the report also cautioned that by the time a patient with cancer who would likely respond to a personalized therapy first completes the step therapy approach, the disease may have already progressed so far that the treatment may no longer provide benefit. “The key to advancing more sophisticated payment policies that recognize the value of personalized medicine tests and treatments is to accumulate evidence of the clinical and economic value of personalized medicine.”

In no uncertain terms, PMC is encouraging policy makers to create guidelines that support the continued use of personalized medicine. “The science, more than ever, is leading the health system away from one-size-fits-all, trial-and-error medicine, and toward the utilization of molecular information to improve outcomes and make the health system more efficient,” concluded the report.