PMC’s FDA Report Shows Continued Progress for Personalized Medicine

The annual analysis documented approvals of 18 new personalized medicines in 2024, important regulatory milestones and establishment of the Center for Drug Evaluation and Research AI Council

/EIN News/ -- Washington, D.C., May 19, 2025 (GLOBE NEWSWIRE) -- The Personalized Medicine Coalition (PMC) has released a new report documenting the U.S. Food and Drug Administration’s approval last year of 18 new personalized medicines. The agency also approved six new gene and cell-based therapies for rare genetic diseases and some cancers and approved or cleared new or expanded indications for 11 diagnostic testing systems.

“The new personalized medicine approvals and policy developments at the FDA in 2024 helps demonstrate that scientific innovation continues to move the health system away from one-size-fits-all, trial-and-error medicine, toward the utilization of molecular information to improve patient outcomes and make clinical care more efficient," says PMC President Daryl Pritchard.

The annual report, Personalized Medicine at FDA: The Scope & Significance of Progress in 2024, also highlighted several regulatory developments in the field of personalized medicine. 

Other 2024 achievements include: 

• expanded indications for previously authorized personalized therapies, including the first ever for an approved gene therapy treatment

• safety label changes for certain cancer treatments based on pharmacogenomic (PGx)-based biomarker testing

• release of a draft guidance document on Diversity Action Plans to improve enrollment of participants from underrepresented populations in clinical studies 

• establishment of the Center for Drug Evaluation and Research (CDER) Artificial Intelligence (AI) Council to provide oversight, coordination and consolidation of CDER activities around AI use

• creation of the CDER Center for Real-World Evidence Innovation to coordinate, advance and promote the use of real-world evidence in regulatory decision-making

• establishment of the Rare Disease Innovation Hub to enhance collaboration across the FDA for rare disease product development

“The newly approved products, and these important regulatory milestones, will help innovators and clinicians provide safer and more efficacious treatments and prevention regimens based on the principles of patient-centered care,” the report reads. 

The scope of breakthroughs underscored the acceleration of the personalized medicine field. The 2024 approval of 18 personalized medicines – meaning the label references specific biological markers – represent approximately 38 percent of all newly approved therapeutic molecular entities. Treatment areas include: cancer, Alzheimer’s disease, cystic fibrosis, Niemann-Pick disease and alopecia.

 Personalized medicines now account for at least a quarter of drug approvals for each of the last 10 years. That’s a notable increase from a decade ago when such treatments made up less than 10 percent of new therapies that were approved.

 “We can’t take progress for granted, however,” says Pritchard. “The FDA should continue to promote policies and practices that push forward the field so we can realize the true potential of personalized medicine.” 

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About the Personalized Medicine Coalition:
The Personalized Medicine Coalition is a nonprofit education and advocacy organization comprised of more than 200 institutions from across the health care spectrum, including innovators, scientists, patients, providers and payers. PMC works closely with business executives, clinicians, lawmakers and government agency officials to promote the understanding and adoption of personalized medicine concepts, services and products. For more information, please visit www.personalizedmedicinecoalition.org.

Kayla Brown
                    Personalized Medicine Coalition
                    kbrown@personalizedmedicinecoalition.org