By Alexandria Kyle-Hammer
The use of one’s genetic profile to inform and guide the diagnosis, treatment and even prevention of disease in individuals (i.e. personalized medicine),1 has made great strides over the last decade and is becoming increasingly popular and common as a treatment tool around the world. Although its use applies to the treatment of many diseases in various fields of medicine, it is worth noting that particularly in oncology, the role of personalized medicine has been revolutionary.
A recent Nature Reviews article, Defining and Quantifying the Use of Personalized Medicines2 , where the authors use the following definition for personalized medicine, 1) “A medicine that has a US Food and Drug Administration (FDA) or a European Medicines Agency (EMA) label stating that its choice as a treatment must be governed by results from a companion diagnostic test” 2) “A medicine that has a label that recommends (but does not require) the companion diagnostic, and at least one authoritative professional organization also recommends use of the test to guide treatment”2 , analyzes the growth in the use of personalized medicine from 1998 to 2009.
When personalized medicine first began to be used in the 1990´s the United States quickly became the world leader in the per capita use of biological personalized medicine. However by 2007 the US was overtaken by the EU, specifically the 5 major markets of France, Germany, Italy, Spain and the United Kingdom (the EU5), and by 2009 the United States had also been passed by Japan. By the end of 2009 the U.S. market was 25% smaller than that of the EU5. In 2011 and 2012 only 8 out of the 69 new molecular entities (NME) approved by the FDA were personalized medicines (i.e. 11.5% of the NMEs).
With the great potential and many befits of personalized medicine it is important for the U.S. not to fall farther behind in this field. The article proposes that by studying over time the various geographical regions where the use of personalized medicine has been the most successful we may be able to draw conclusions about the policies and healthcare systems used that might be most useful in supporting the further adaptation of personalized medicine in the United States.
The article also offers several other interesting findings. One being that the majority of the usage and sales of personalized medicine has been focused in the area of Oncology, suggesting a great unmet need in the field. Many older drugs that were not previously used as personalized medicine have since been converted into personalized medicines, as it has become clear that they are more effective when used as such. An example of this is the drug Tamoxifen, which was commonly given to women with ER+ breast cancer. It became a personalized medicine when it was discovered that 65% of women taking the drug developed resistance due to a mutation in their CYP2D6 gene. Now women are genotyped for that specific mutation so that the right treatment is given to each breast cancer patient. This conversion of medicines already on the market to personalized medicines has impacted the growth of the field.
Although the overall use of personalized medicine is growing at a 22% Average Annual Growth Rate (AAGR) globally between 1998 and 2009, the United States is lagging behind when compared to Japan and the EU5.
The FDA and the US National Institutes of Health (NIH) have promised to invest heavily in the field to support the growth of personalized medicine and the effort to make it a reality in the USA. As part of this effort the FDA has released a set of guidelines to regulate the field of personalized medicine. Within the Agency they have also created a Personalized Medicine Staff (http://goo.gl/7FCkcr) dedicated to “addressing the opportunities and challenges associated with diagnostics used in personalized medicine”3.
While these efforts, along with others, are a step in the right direction the progress of personalized medicine in the US is still slow. Further investigation is needed to identify the essential driving factors of the growth of personalized medicine in order for it to reach its full potential as a treatment method. It is also possible that more “public funding for translational research, greater use of electronic medical records to better access patients’ test information, and incentives for developers to personalize both approved and investigational therapeutics”4 are needed to further drive the advancement of personalize medicine.
2 Hu, Sean X., Murray L. Aitken, Arnold L. Epstein, Mark R. Trusheim, and Ernest R. Berndt. “Defining and Quantifying the Use of Personalized Medicines.” Nature Reviews Volume 12 (2013): 896-97. 1 Dec. 2013. Web.
4 See footnote 2