This document discusses the rise of personalized medicines and their impact on drug development. It notes that nearly 1 in 4 new drugs approved in recent years were precision medicines, and the proportion of personalized medicines in clinical development is expected to increase to 70% over the next five years. The use of genomics is also increasing in clinical trials, with over half of phase 1 trials and nearly two-thirds of phase 2 trials including some aspect of genomic medicine. The role of biomarkers, pharmacogenomics, and other approaches are enabling more targeted patient selection and dosing in clinical development. While promising, precision medicines also present challenges including complex trial designs, regulatory hurdles, data management needs, and the integration of multiple types of genomic and clinical
Drug development pipelines are full of targeted treatments that offer new hope for patients.
42% of all compounds and 73% of oncology compounds in the pipeline have the potential to be personalized medicines
Biopharmaceutical companies nearly doubled their R&D investment in personalized medicines over the past five years, and expect to increase their investment by an additional 33 percent in the next five years
Biopharmaceutical researchers also predict a 69% increase in the number of personalized medicines in development over the next five years
Tufts Center for the Study of Drug Development. Personalized medicine gains traction but still faces multiple challenges. Impact Report. 2015;17(3).
Pharmacogenomics is the study of genes in the context of drug response. Pharmacogenomics can help us understand why people who receive the same medicine respond differently to it. Some individuals may benefit from the drug or feel better while others may not respond—they experience no change in their condition. Some who take the drug may experience side effects such as rash or nausea. Some may benefit from the medicine but only after they have received a different dose. Pharmacogenomics can provide insight into the drug target and mechanism of action. It may also help us understand better how the drug is transported in the body or how it is used or metabolized by the body. It helps us understand how genomic variation, or differences in DNA, relates to safety or efficacy of a drug.
Approved drugs may have benefit to treat related diseases or in some instances very different conditions. For example, sildenafil was first developed for angina and then found to work in erectile dysfunction as well as pulmonary arterial hypertension. On average it takes about 12 years to bring a novel drug treatment to market. Repurposing that drug after approval for a new indication may be possible in as little as 3-4 years with accumulated safety and clinical data enabling pharma companies to get needed therapies to patients faster. Genomic evidence may provide evidence for drug repurposing or repositioning. In this example, our scientists leveraged transcriptomics data or RNA signatures from patients with various dermatological diseases to identify mechanistic connections or commonalities in the drug target pathways to demonstrate that a drug originally indicated to treat psoriasis may also benefit patients suffering with atopic dermatitis and even acne enabling the sponsor to initiate a new drug/indication program