The U.S. Food and Drug Administration (FDA) approved 18 new personalized medicines in 2024, highlighting the continued advancement of precision medicine approaches in healthcare, according to a new report released by the Personalized Medicine Coalition (PMC).
These personalized therapies, which reference specific biological markers in their labeling, represented approximately 38% of all newly approved therapeutic molecular entities last year. The FDA also authorized six new gene and cell-based therapies for rare genetic diseases and certain cancers, while approving or clearing 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," said PMC President Daryl Pritchard.
Expanding Treatment Options Across Multiple Disease Areas
The newly approved personalized medicines target a diverse range of conditions, including cancer, Alzheimer's disease, cystic fibrosis, Niemann-Pick disease, and alopecia. This broad spectrum of applications demonstrates the versatility and growing adoption of precision medicine approaches across multiple therapeutic areas.
The report, titled "Personalized Medicine at FDA: The Scope & Significance of Progress in 2024," reveals that personalized medicines have consistently accounted for at least a quarter of all drug approvals for each of the past 10 years. This represents a significant increase from a decade ago when such treatments constituted less than 10% of newly approved therapies.
Key Regulatory Developments
Beyond new drug approvals, the FDA implemented several important regulatory initiatives in 2024 to further advance personalized medicine:
- Expanded indications for previously authorized personalized therapies, including the first-ever expanded indication for an approved gene therapy treatment
- Safety label changes for certain cancer treatments based on pharmacogenomic (PGx) 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- Release of a draft guidance document on Diversity Action Plans to improve enrollment of participants from underrepresented populations in clinical studies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 Council to provide oversight and coordination of AI activities
- Creation of the CDER Center for Real-World Evidence Innovation to 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 states.
The Growing Impact of Personalized Medicine
The consistent growth in personalized medicine approvals reflects a fundamental shift in the healthcare paradigm. By targeting treatments to specific patient populations based on genetic, molecular, or cellular characteristics, these approaches aim to improve efficacy, reduce adverse effects, and optimize therapeutic outcomes.
This transition from traditional one-size-fits-all approaches to more tailored interventions represents one of the most significant advancements in modern medicine. The increasing proportion of personalized medicines among new drug approvals suggests this trend will continue to shape the future of healthcare delivery.
Maintaining Momentum
Despite the encouraging progress, PMC President Pritchard emphasized the importance of continued support for personalized medicine initiatives. "We can't take progress for granted, however," he cautioned. "The FDA should continue to promote policies and practices that push forward the field so we can realize the true potential of personalized medicine."
The PMC, a nonprofit education and advocacy organization comprising more than 200 institutions across the healthcare spectrum, works to promote the understanding and adoption of personalized medicine concepts, services, and products. Their annual report serves as an important benchmark for tracking progress in this rapidly evolving field.
As personalized medicine continues to gain traction, collaboration among regulators, researchers, clinicians, and industry stakeholders will be essential to overcome remaining challenges and fully realize the potential of precision healthcare approaches.
