Personalized medicine approvals experience sharp increase

The number of personalized medicines approved over the last two years has increased sharply and continues to increase, representing a trend in medical development towards therapeutics that can be tailored to individual patient efficacy and safety, according to a new analysis.

The analysis, 2015 Progress Report: Personalized Medicine at FDA, from the Personalized Medicine Coalition (PMC) documents an upward trend in the number of personalized medicine approvals at FDA, with personalized medicines accounting for more than 1 in 4 novel new drugs approved in 2015.

The analysis also found that:

• 13 of the 45 novel new drugs approved by the FDA in 2015 are personalized medicines (approximately 28%).

• Five of the 14 approved oncology indications in 2015 are personalized medicines (approximately 35%).

Additionally, FDA approved a number of significant new personalized medicine indications for previously existing drugs that redefine and expand their intended populations.

“With these new approvals, there are increasingly more tools in the managed care toolbox,” says analysis author Daryl Pritchard, PhD, vice president, science policy, PMC. “Personalized medicines and managed care go hand-in-hand as both focus on understanding and providing the most effective and valuable therapeutic option to individual patients. For many health conditions, diagnostic tests can help guide treatment decisions so as to provide safer more effective care.”

Personalized medicine is an evolving field in which physicians use diagnostic tests to determine which medical treatments will work best for each patient, according to Pritchard.

“By combining the data from those tests with an individual’s medical history, circumstances and values, healthcare providers can develop targeted treatment plans,” he says. “This in turn can bring down costs as downstream expenses are avoided because many patients will have improved outcomes and/or will have avoided adverse events.” 

More and more personalized medicines are becoming available for use in managed care, he says. An emerging concept just 10 years ago, personalized medicines went from an occasional annual therapeutic product approval to more than 25% of all FDA approvals in 2015.

“While the acceleration in product approvals demonstrates the increasing prominence of personalized medicine in healthcare, many significant challenges remain including discovery and translational research, diagnostic regulatory policy, coverage and reimbursement of diagnostic tests, and clinical adoption of new technologies,” Pritchard says.

“Efforts to educate healthcare providers about personalized medicine and to help understand its clinical and economic value will help to advance personalized medicine and improve managed care,” he says.

Next: Health execs hesitate to jump on board

Health execs hesitate to jump on board

Fifty-nine percent of respondents to a recent survey by Health Catalyst said precision medicine will not play a significant role in their organizations in the next five years. Among respondents from nonacademic hospitals and health systems, the number who said precision medicine will play an average, small or non-existent role in their organizations between now and 2020 jumped to 68%.

After President Obama announced a $215 million precision medicine initiative in his State of the Union address last year, many expected healthcare systems to increase precision medicine efforts. “As-historically-expected, academic medical centers seem to be leading the healthcare industry when it comes to clinical use of DNA sequencing and precision genomics,” says David Crockett of Health Catalyst.  “Is this really spreading into mainstream healthcare?  Maybe not as fast as the hype suggests.”

The tricky balance is between “standard care” and “tailored care” (precision medicine), according to Crockett. “It’s hard to standardize to evidence-based best practices, reducing variation and waste to save money-while expecting every patient to get treated a little differently because of DNA test results.”