NEW YORK: The term “precision medicine’’ is one that most people had likely never heard before President Barack Obama’s State of the Union address, but it is an area of exciting promise in biomedical research that will eventually improve health outcomes and potentially reduce healthcare costs.
The concept revolves around understanding the specific genetic changes that underlie such diseases as cancer, diabetes and cystic fibrosis. In general, patients with these types of diseases fall into different subgroups based on the genetic changes that can cause the diseases. Drugs tailored to address the changes are likely to be more effective. A good example is the cancer drug Gleevec, which inhibits an enzyme that is mutated in a subtype of leukemia and has had remarkable success with few side effects.
Scientists have been sequencing the DNA of many types of cancers to identify the specific patterns of mutations that can lead to disease and to try to find new drugs to treat them. While significant advances have been made recently in our understanding of these diseases, new investment is needed to turn discoveries made in the laboratory into drugs that can be tested in patients.
Some have argued that developing drugs for small groups of patients will increase the costs of drugs because developmental costs will be borne by fewer patients taking them. This is most likely not the case, for two reasons. First, skeptics need only look to the success of Gleevec to realize that these drugs are sometimes effective in subpopulations other than those for whom they were developed. Second, using precision medicine to target therapies to patients will greatly improve the odds that a drug will succeed in clinical trials and gain FDA approval; the failure rate and the associated costs of drug development will decline. Of course, there will be additional tests to determine the genetic profile of patients, but the costs of DNA sequencing have dropped dramatically in the past decade, making these types of tests much more affordable.
Others have argued that genomic information is less important than patient behavior in predicting and preventing disease. Precision medicine is not about predicting the probability that someone will develop a disease. It is the improved understanding of the underlying causes of a disease already present, and how best to treat and respond to the genetic changes.
Besides helping develop more effective drugs, precision medicine will help doctors prescribe the right drug for the right patient. Patients won’t get drugs that won’t work; they will be spared the wasted time and side effects that an ineffective drug might produce. Getting patients treated more quickly with the best drug for them based on their genetic profiles will result in better health outcomes, and overall treatment costs will fall.
Of course, challenges remain. Identifying the sets of biomarkers that will best predict which drug will work for which patient is essential and will require time and investment in the president’s Precision Medicine Initiative. And in diseases such as cancer, the tumors can mutate to develop resistance to targeted therapies, which is why combinations of drugs are often needed for the most advanced cancers.
Whether the president’s proposal will result in additional funding for the National Institutes of Health is unclear, but new investment in research is sorely needed. Since 2003, funding at the medical research agency has been essentially flat, with its overall budget purchasing power eroding by more than 10 percent. This despite the fact that the return on investment in overall economic growth spurred by agency spending is as high as 30 percent to 100 percent, and every dollar invested in research produces more than two dollars in new state business activity.
While the Precision Medicine Initiative is an exciting prospect, we should not be robbing Peter to pay Paul. We should increase investment in scientific biomedical research that can create new jobs, improve medical outcomes, reduce healthcare costs and save lives. (Reuters)