How Much are Gene-Therapy Treatments Likely to Cost?

An article about the potentially high costs for cures for genetic diseases reveals a subtle shift in how pharmaceutical companies intend to justify their high prices.

In the past, the usual argument was that high drug costs were justified by the high costs of drug development. The standard argument was that since it costs upwards of a billion dollars to take a drug all the way from initial research through Phase I, II, and III clinical trials, drug companies justifiably needed to charge high prices to recoup their costs. In some cases, drug companies needed to recoup losses incurred by drugs that never made it to market at all.

The paradigm has shifted with the advent of genetic therapy. Now, cells can be removed from a patient with a specific genetic mutation, modified by gene therapy techniques for that patient (and only for that patient) and then returned to the patient, affecting a complete cure. Since these cures are specific to individual patients, the FDA has begun to approve these new gene therapy techniques when they have been shown to work on just a few patients (perhaps several dozen). Gone are those lengthy Phase II and Phase III clinical trials, with their thousands of patient at dozens of medical centers. And gone, too, most of the costs of drug development.

As a result, drug companies are being forced to come up with new arguments to justify their high prices. And their new argument is shaping up to be something along the lines of "What's it worth to you?" How much will you or your insurance company pay for what one drug company spokesperson calls "long-term transformative benefits"?

Potential prices ranging from about $475,000 to $900,000 have been floated by some companies. At some point we may have wrestle with the question, "What is the value of life"?