FDA Approvals Advance Precision Medicine, Genomics Treatments

The FDA approved or cleared 12 personalized medicines and seven diagnostics in 2019, helping providers address the root causes of disease and further integrating precision medicine with clinical care, a report from the Personalized Medicine Coalition (PMC) stated.

Personalized Medicine at FDA: The Scope & Significance of Progress in 2019 examined how the approved personalized medicines will improve patient care, target therapies to certain patient populations, and expand treatment options for cancer patients.

Personalized medicines now account for more than one out of every four drugs the FDA has approved since 2014. This is a significant increase from 2005, when personalized medicines accounted for just five percent of new therapies approved each year.

“The transformation of healthcare from one-size-fits-all, trial-and-error medicine to a targeted approach utilizing each patient’s molecular information continues to accelerate as the US Food and Drug Administration more regularly and rapidly approves diagnostic tools and treatments that expand the frontiers of personalized medicine,” the report stated.

In addition to the newly approved personalized medicines, the agency approved new personalized medicine indications for previously existing drugs in 2019. The approvals redefine the drugs’ intended populations and provide patients with more personalized treatment options, PMC noted.

Moreover, five of the approved personalized treatments are the first to address the root causes of debilitating chronic diseases, and four provide new treatment options for cancer patients.

“Many patients with spinal muscular atrophy (SMA), Duchenne muscular dystrophy, acute hepatic porphyria, cystic fibrosis, and sickle cell disease now have treatments available that target the underlying molecular mechanisms of diseases where there were no options before,” PMC wrote.

The agency also approved or cleared diagnostics to help target treatments to patients who need them. The use of companion or complementary diagnostics is an essential part of precision medicine, PMC said, and the approval of these diagnostics will enable providers to make more informed drug decisions.

“In 2019, FDA’s Center for Devices and Radiological Health (CDRH) approved or cleared seven new or expanded in vitro diagnostic tests that underpin personalized medicine strategies. Each of the diagnostics will help inform targeted treatment decisions to improve drug safety and efficacy,” the report said.

The FDA has made significant strides in advancing the use of precision medicine and genomics in healthcare. The agency recently released six final guidance documents on gene therapy manufacturing and clinical development of products. The documents include input from stakeholders across the healthcare industry and aim to design a modern structure for gene therapy development.

“As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said at the time.

“Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.”

Payers, providers, and policymakers across the industry increasingly recognizing the potential for gene therapies and precision medicine to transform disease treatments. However, considerable barriers still exist: A 2019 report found that although health systems are increasing their investments in precision medicine technologies, many still struggle with limited resources and a lack of reimbursement.

PMC noted that while these challenges need to be addressed, FDA approvals of personalized medicines will play a big part in advancing the use of these therapies in routine clinical care.

“These significant developments reflect the extraordinary pace of scientific innovation in personalized medicine, which is leading to improved health outcomes and having a tremendous impact on health care delivery efficiency and efficacy,” the report said.

“While ongoing challenges in the areas of scientific discovery, diagnostic regulatory policy, coverage and reimbursement, and implementation of new technologies into clinical practice must be addressed, the science 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.”

In order to further promote innovative precision medicine treatments and gene therapies, officials should continue to approve and clear these drugs for clinical use.

“To ensure that industry leaders continue to develop groundbreaking personalized medicine tests and treatments and that patients have access to these products, policymakers, as they have in the past, must favor policies that encourage the advancement of the field,” PMC concluded.