Precision medicine, which exists at the confluence of pharmacology, various ‘omics’ fields and data science, is the latest buzzword promising the eventual solution to all health problems by the personalization of the treatment process. The approach accepts the complexity of and variance between humans, and attempts to develop drugs for people with particular genetic makeups. Here are 3 hurdles to the precision medicine revolution.
1. Next Generation of Scientists?
First, the field has reached a hurdle rooted in the fact that, as the Baylor Charles A. Sammons Cancer Center Oncologist Dr. Mennel puts it, “most physicians are not fluent in the principles of precision medicine.” Eric P. Winer, the director of the Breast Oncology Program at the Dana-Farber Cancer Institute, explains that “our ability to sequence genes has gotten ahead of our ability to know what it means.” We are therefore left with many oncologists who are unsure what to make of the genetic information provided to them by extensive screenings.
Earlier iterations of precision medicine have led to the development of Gleevec and several other drugs that have helped many. In these success cases, particular mutations and genetic abnormalities (such as the Philadelphia Chromosome) were identified, directly associated to a particular phenotype and were addressed by a targeted therapy (for information on targeted approaches to cancer therapy see here). This process requires an immense amount of feedback between clinic, physician and laboratory. If physicians do not have the proper training to use the genetic information provided to them, or if the association between a genetic abnormality and a specific phenotype cannot be determined due to a deficiency in data, the complex chain that serves as the backbone of precision medicine is effectively broken. For precision medicine to truly be the way of the future, physicians need to be trained to analyze and make use of provided genetic information and ‘omics’ scientists need to be able to turn their data into something that could be used to develop targeted drugs (i.e. the creation of what Collins and Varmus call the “next generation of scientists”—perhaps medicine, like science will “advance one funeral at a time”).
2. The Economics of Precision Medicine
Additionally, a large set-back in precision medicine is its very precision. Its precision means that a particular drug will help only a fraction of the population, but will need a similar approval time (slightly shorter than usual due to the President’s Initiative) to one that can treat the general population. The Tufts Center for the Study of Drug Development’s most recent analysis of the average cost of drug development from discovery to market (including opportunity cost) is over $2.5 billion. This figure has been contested, but nonetheless indicates the vast costs associated with drug development. Unless government incentives are increased to an unprecedented level (i.e. even beyond the incentives for Orphan Drug development), it seems unlikely that a pharmaceutical company will recoup their investment in a drug that is specifically targeting a mutation found in a small percentage of the population. It is difficult, therefore, to imagine the precision medicine revolution without a similar revolution in the pharmaceutical and biotech industries.
The Precision Medicine Initiative is fundamentally different than the War on Cancer. Its success will be in the implementation of a methodology as opposed to curing a seemingly incurable class of diseases. Success in the next few years, therefore should likely not be measured by the drugs brought to market, but rather by the increase in general awareness of the budding field and the increased implementation of its strategies.
3. Personalized Medicine has Outpaced the Science
Finally, a major problem with government research initiatives is that they are frequently compared to the moonshot. Most medicinal work, however, is like so inexact that the medicinal chemist Derek Lowe suggests: “you wouldn’t jump off a chair if our knowledge of physics was as inexact as our knowledge of human biology.”
We cannot assume that if enough money is thrown at an issue that an eventual solution will be met in the near future. While the ultimate goal is noble and has provided true reasons for optimism, it is important to realize that given the sheer complexity of biological systems no advancement is inevitable. Precision medicine is surely the future, but whether it is the revolutionary upheaval in treatment that some claim (at least in the near future) remains to be seen.
In a piece in the New Yorker from last year, the science writer Cynthia Graber put it best: “The excitement surrounding personalized medicine has outpaced the science.” That does not mean, however, that there is nothing to be excited about.
- Collins, Francis S., and Harold Varmus. “A New Initiative on Precision Medicine.” New England Journal of Medicine N Engl J Med 372.9 (2015): 793-95.
- Dimasi, Joseph A., Henry G. Grabowski, and Ronald W. Hansen. “Innovation in the Pharmaceutical Industry: New Estimates of R&D Costs.” Journal of Health Economics47 (2016): 20-33.
- Kolata, Gina. “When Gene Tests for Breast Cancer Reveal Grim Data but No Guidance.” The New York Times[New York] 11 Mar. 2016.
- Mennel RG. Precision Medicine: Hype or Hope? Proceedings (Baylor University Medical Center). 28.3 (2015): 397-400.