One of the most innovative medications of recent years is Novartis’s imatinib, which is marketed as Gleevec. Introduced in 2001, it set a record for the fastest approval time by the U.S. Food and Drug Administration (FDA), and in 2011 had US$4.3 billion in sales. Imatinib was the first of a new class of drugs that act by inhibiting a specific enzyme — in this case, a receptor called tyrosine kinase — that is characteristic of particular cancer cells. Because only certain cancers will respond to imatinib, it is prescribed only for patients identified by specific diagnostic tests (for example, the Philadelphia chromosome abnormality test).
Imatinib is an example of the products emerging from a new pharmaceutical field known as personalized medicine, or PMx: the tailoring of drugs and other treatments to specific populations, based on their genetic profiles or other differentiating factors. (We use the abbreviation PMx to echo Rx for prescription medicine and Dx for diagnostic tests.) The underlying technology for PMx marries advances in diagnostic biotech, such as rapid gene sequencing, with a profound expansion of scientists’ understanding of molecular disease pathways. Another example is Genomic Health’s Oncotype DX breast cancer test, introduced in 2004, which uses an analysis of multiple genetic variations to determine which cancers are likely to recur for a particular individual. It also indicates which patients are likely to benefit from chemotherapy. The results of this test can help physicians avoid prescribing drugs with no medical value; because those drugs often have severe side effects, the test greatly benefits patients as well.
The concept of personalized medicine is only 15 years old, and it does not have a perfect track record; some of its medications have failed to get regulatory approval or to be profitable in the marketplace. But where it has proven successful, it is creating a fundamentally new value proposition for healthcare businesses. It represents a game-changing trend for healthcare providers (hospitals and individual physicians); payors, including Medicare and private insurance; and patients, who can have a far more participative role in their own care than they would without PMx. When physicians and patients can draw upon a wealth of genetic and biological data, personalized therapy will become the norm, not the relatively rare occurrence it is today.
The greatest impact of this new approach to individualized diagnosis and treatment will probably be felt in the pharmaceutical industry. Leading pharmaceutical companies have long relied on innovation to expand the frontiers of better health — and to financially sustain their businesses. But that capacity for launching new products is under severe strain today. Many pharmaceutical companies have recently seen their research and development productivity erode. Biotech startups have struggled to turn their scientific advances into viable drugs. Companies have resorted to alternatives such as vaccines, preventive care, and geographical outreach for profits, but no pharma industry breakthrough has come along recently to create a promising pipeline of new products. PMx offers the potential for just that sort of breakthrough.
Thus, despite the disruptive nature of personalized medicine, the pharmaceutical industry is emerging as a driving force in this field. The list of early PMx adopters includes some industry giants: Roche, Pfizer, Novartis, and Lilly. The field is also populated with upstart companies such as Genomic Health, XDx, and Prometheus Labs that offer specialized approaches to segmenting disease, as well as the recognized pioneers in personal genomics: 23andMe, deCODE Genetics, and DNA Direct. In adopting PMx, they are all embracing a new healthcare model that emphasizes segmentation of patients, and decisions and practices tailored to individuals or small groups. Although this approach might lead to smaller revenue pools for pharmaceutical producers, PMx can actually improve their profits. A business model based on differentiated products can reduce the system-wide costs and complications of one-size-fits-all medications, provide new opportunities for marketing, and establish new efficiencies in production and distribution.