For a few years now, we have been hearing how personalized medicine is going to change the face of health care. Personalized medicine is about making the treatment as individualized as the disease. It involves tailoring the treatment to aspects of the disease that can be readily recognized. This may include identifying genetic, genomic, and clinical information that allows accurate predictions to be made about a person’s potential response to therapy or his or her risk to experience adverse events.
In order for personalized medicine to be used effectively by healthcare providers and their patients, these findings must be translated into precise diagnostic tests and targeted therapies. This has begun to happen in certain areas, such as the recent approval of the first drug to treat one of the underlying causes of cystic fibrosis (CF). The drug known as Kalydeco is specifically indicated for patients who carry a unique mutation (G551D) in the CF protein.
Kalydeco will cost $294,000 a year per patient. The high price is due to the fact that of the 30,000 patients in the US with CF, only 4% (roughly 1,200) carry the G551D mutation, which is not a large enough patient population to make it affordable, while adequately offsetting R&D and other associated costs. The question that arises is who will (or should) pay for this medication? Moreover, even if insurance covers 80% of costs, this still translates to an estimated co-pay of $60,000 per year.
Vertex, the maker of Kalydeco, has set up a few financial assistance programs to help patients make their treatment affordable:
- Free Medicine Program: Vertex will provide Kalydeco for free to people who do not have insurance and have an annual household income of $150,000 or less; and
Unfortunately, some patients are not eligible for company co-pay support because they have Medicare or Medicaid coverage or live in Massachusetts. For these individuals, we would suggest exploring independent non-profit co-pay assistance foundations that may be able to help those patients with their out-of-pocket costs. However, in cases like these, there are no certainties, and perhaps, more evidence that every silver lining does come with an obligatory cloud.
The promise and practicality of personalized medicine will not be a cheap proposition. The best way to support the cost and use will be better outcomes. Over time, if patients receiving expensive medications do better, live happier lives and actually use fewer healthcare resources, the benefits will outweigh the costs. If the opposite turns out to be true, we will find ourselves back in the therapeutic equivalent of the 1950s.
Only time will tell.