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  6. Focus Area: Individualized Therapeutics and Precision Medicine
  1. Focus Areas of Regulatory Science Report

Focus Area: Individualized Therapeutics and Precision Medicine

Bacteriophage Virus.
Artificial Intelligence DNA molecule

Importance to FDA

Most medical treatments are designed for the average patient, which may be successful for most but not all patients. Individualized therapeutics are products to designed to treat one to a few individuals to address unmet health needs. Individualized therapies have become increasingly feasible due to improved understanding of individual variability and identifying new rare genetic diseases with next generation sequencing (NGS) technologies. The challenges and opportunities for utilizing FDA-regulated products as individualized therapeutics span the product lifecycle: the development of robust manufacturing and assurance of product quality, extent of preclinical testing to support regulatory evaluation, and the collection of clinical evidence with a very small number of patients worldwide (e.g., populations as small as one patient). These issues impact safety and effectiveness evaluation and sustainability.

Precision medicine—sometimes known as personalized medicine—tailors disease prevention and treatment for individual variability (e.g., genetic and lifestyle differences among patients). The goal of precision medicine is to match the right treatments at the right dosages for each individual patient at the right time. The challenge for precision medicine is identifying the mechanistic basis for adverse events, such as why the body reacts negatively to a treatment (e.g., breaking out in a rash) and differences in efficacy (e.g., why a drug works better in some patients than it does in others).

To realize the promise of precision medicine and individualized therapeutics, FDA sees a critical need for more mechanistic understanding, improved manufacturing capabilities, and additional tools. FDA is exploring new technologies (e.g., omics) to advance major breakthroughs in diagnosis, prognosis, and treatment of diseases. FDA created precisionFDA, a cloud-based portal for community research and development that allows users world-wide to share data and tools to test, pilot, and validate existing and new bioinformatics approaches to NGS processing.

For example, pharmacogenetics studies how individuals respond differently to drug therapies based on their genetic make-up using technology such as NGS which allows sequencing of a human’s entire genome in a short period of time (as short as one day). This technology combined with others enables researchers to identify precise genetic, mechanistic, or lifestyle reasons to understand why certain individuals or subpopulations respond positively or negatively when treated for the same disease with the same drug. Being able to more precisely classify the genetic basis of diseases and drug responses through diagnostic tests and devices enables development of mechanistically targeted therapeutics.


  • Bacteriophage (phage) therapy (i.e., the use of viruses that invade and kill bacterial cells) is being investigated as a novel antimicrobial approach to treat antibiotic resistant bacterial infections. To overcome bacterial resistance of conventional antibiotic treatment, personalized bacteriophage cocktails are used as treatment for the patient’s unique bacterial strain. FDA is developing and evaluating animal models to assess safety and effectiveness of bacteriophage cocktails for treating antibiotic resistant bacterial infections.
  • Gene therapies, such as adeno-associated virus (AAV) vectors and CAR T cells show promise for treating several types of rare genetic and intractable diseases, with some products already approved, and ex vivo modified cells, often using lentiviral vectors for gene delivery, have been found to be effective at treating disorders of the hematopoietic system. In addition, genome editing tools that can directly repair genetic defects, are being explored to allow rapid development of individualized therapies. However, there are still challenges that need to be addressed before these complex therapeutic strategies can be deployed for more routine use. This includes predicting or avoiding immunogenicity of the vector (e.g., AAV) or therapeutic gene, creating more efficient and scalable manufacturing methods, and developing more reliable ways to identify and understand the potential for unintended changes to the genome that may have negative consequences.
  • Pharmacogenetic tests are of increasing interest to healthcare practitioners in selecting therapeutic agents and avoiding harmful treatments. FDA published a Table of Pharmacogenetic Associations to increase the quality of scientific evidence supporting clinically available tests. This resource provides transparency into FDA’s view of the state of scientific evidence in pharmacogenetic gene-drug associations, and where the evidence is enough to support therapeutic management recommendations for patients with certain genetic variants, or genetic variant-inferred phenotypes that are likely to have altered drug metabolism, and in certain cases, differential therapeutic effects. This is an important step toward supporting well-qualified therapeutic decisions by medical professionals.
  • FDA researchers answer critical regulatory science questions related to drug approval in use of immunotherapy, as well as other new drugs for acute myeloid leukemia (i.e., a cancer of the blood and bone marrow), lung cancer, and other malignant conditions. This includes understanding the genetics of immune-related harmful events and investigating genetic signatures (i.e., information about a group of genes) associated with the impact of drug toxicity or efficacy.




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