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President Obama’s nomination of Dr. Francis S. Collins as director of the National Institutes of Health bodes well for the field of personalized medicine. Known for his role as the director of the Human Genome Project, Dr. Collins helped pave the way for advances that target treatments to patients based on their genetic makeup.

The nomination comes as recent advances show the continued progress and promise of personalized medicine. Once just grazing the surface of cancer treatments, personalized medicine research has extended to cardiovascular disease, infectious diseases, psychiatric disorders, and transplant medicine.^[i] For example, researchers from the Children’s Hospital of Pittsburgh found that certain transplant patients with a genetic mutation are more likely to reject their new organs. This discovery allows doctors to personalize anti-rejection medicine for individual children and potentially reduce liver transplant rejection rates from 50% to under 20%.^[ii]

A newly approved blood-thinner also demonstrates the increasing frequency with which personalized treatment information is being included in labels of new medicines. Prasugrel offers an alternative to an existing blood-thinner, clopidogrel, for patients who do not metabolize clopidogrel normally due to a genetic variation. This means that physicians can screen patients at risk for heart attack and stroke to determine which treatment approach is most likely to work for them.^[iii]

A recent label change highlights the potential of personalized medicine to make health care more efficient. The FDA approved labeling changes for both panitumumab and cetuximab to indicate that these drugs should not be used in patients with mutations to the gene known as KRAS. Avoiding use of cetuximab as a first-line treatment for patients with the mutation could save about $600 million each year.^[iv]

Advances like prasugrel and the addition of KRAS information to panitumumab and cetuximab labels demonstrate how the biopharmaceutical sector is embracing personalized medicine. Companies are researching the genetic underpinnings of disease and searching for effective new personalized treatments and diagnostics. Here are just a few examples of the extent to which the approach has taken hold:

 

• A taskforce at AstraZeneca including representatives from drug discovery, development, and commercial divisions was formed to manage the personalized medicine program on a global basis. Ten drugs are currently in clinical development with over 20 more being considered.^[v]

   

• According to former Eli Lilly and Company CEO and Chairman Sidney Taurel, "Today, there is hardly a molecule or an approved product anywhere in Lilly’s pipeline or portfolio that is not the subject of tailoring. Our goal is to give doctors the ability to prescribe for individual patients – with a much higher level of confidence – the right dose of the right medicine at the right time."^[vi]

   

• Dr. Paul Stoffels, the Company Group Chairman of Global Pharmaceutical Research and Development, Johnson & Johnson said, "The pharmacogenetic approach is used for almost every compound we develop…We are now looking for markers for response and for adverse events to better understand our current compounds to improve effectiveness of future compounds."^[vii]

Recent advances in personalized medicine approvals and labeling, combined with the commitment of biopharmaceutical companies around the world to the approach suggests that these types of treatments will only become more common and more effective in the coming years. Personalized medicine will help improve outcomes, prevent side effects and make health care more efficient in years to come.