There are now five drugs on the market to treat genetic disorders that are each priced at over $2 million.
They have been approved by the FDA to treat illnesses that had no curative therapy. Some, such as spinal muscular atrophy, were fatal while others such as hemophilia and sickle cell anemia led to repeated crises and frequent hospitalizations.
Are these astronomical prices justified? The pharmaceutical companies justify the prices by citing two factors: the high cost of drug development and the long-term financial benefits.
The problem with using research costs as a justification for the prices is that much of the basic research is funded by NIH grants or academia rather than by industry.
In the case of Zolgensma, Novartis’ $2.1 million drug for spinal muscular atrophy, the bulk of the early funding came from a private charity founded by parents whose child had the disease. ProPublica documented how their efforts, both financial and emotional, were ignored when the commercial potential of the drug began to become apparent. They hoped for a cure for their daughter and others similarly afflicted, not a windfall for the executives of the start-up they helped fund. See: www.propublica.org/article/zolgensma-sma-novartis-drug-prices-gene-therapy-avexis
Research published in JAMA provided details of the sponsorship and funding of 341 trials of gene therapy. Fewer than half were industry-funded. Academic hospitals, universities and the NIH were more often the funders, and yet the financial rewards almost all go to the pharmaceutical industry.
One could make a case that the sky-high one-time cost of gene therapy pays for itself by preventing the numerous emergency room visits and hospitalizations that it will eliminate. The estimated lifetime cost of hemophilia is $20 million! This makes the one-time $3.5 million cost of Hemgenix seem a bargain.
The big if is that the only data we have is that the benefits last 3 years – hopeful, but only a short span in a life-long disease. If the benefits fade after 4-5 years will CSL Behring refund the cost?
Will health insurance companies be willing to pay for a drug that may benefit the patient for 50 years or more when they know that their average subscriber is probably going to change insurers in a few years?
We need a different way to pay for these “miracle” drugs. One option is to have them paid for on an annual basis, spreading the cost over the patient’s estimated lifespan, with payment to stop if the drug stops working. A plan must devised soon, or scientific advances plus pharmaceutical greed will exceed society’s ability to pay.
Edward Hoffer MD is Associate Professor of Medicine, part-time, at Harvard.
What Does The Doctor Say?
By Dr. Edward Hoffer