Recall viewing the features of your newborn child for the first time and the warm emotions it inspired -- or perhaps laying eyes on your crush, spurring the butterflies and that tingling sensation. Sight is a gateway to a wealth of emotions. Many people, however, have never seen the faces of their loved ones or are slowly, frighteningly losing that ability due to disease.
The FDA recently approved the new gene-therapy, Luxturna, for the treatment of heritable retinal degeneration. The illness most often begins with nighttime blindness and nystagmus, but eventually progresses to complete vision loss. Luxturna offers hope by restoring the gift of sight.
The product treats retinal dystrophy in those with a mutation to the RPE65 gene, which currently affects 1000 to 2000 citizens in the U.S. RPE65 produces a protein involved in the visual cycle, which converts light into electrical signals to transmit to the brain for optical recognition.
The procedure is surprisingly simple, with physicians injecting a benign virus containing nourishing copies of RPE65 just below the retinas of each eye. The injection permits the production of the proteins, enabling repair to the visual cycle.
The makers of Luxturna, Spark Therapeutics, found that although sight is incompletely restored and long-term effects are unknown, the intervention leads to significant improvements in comparison to control groups. Financial stress, however, may disable numerous people from undergoing the life-changing rehabilitation.
According to the Institute for Clinical and Economic Review, the current predicted value of Luxturna treatment is between $650,000 to $1 million. Spark Therapeutics chief executive Jeffrey Marrazzo detailed similar estimates, noting the culprit of such figures being the “indirect costs” of the procedure. He defined the indirect costs as including improved quality of life, diminished medical expenses for caregiving, and ability to obtain a career.
Does Marrazzo have a logical point? Researchers at the University of Chicago noted that such impairments estimate a total economic burden in the U.S. of $139 billion. Also, blind individuals currently have an unemployment rate of 58%, with 29% living below the poverty line, as found by Cornell University. The method may allow patients to diminish previous caregiving expenditures and finally pursue their goals.
While the benefits are exemplary, would $1 million be affordable and fair? As reported by Liberty Nation, an estimated 10 – 15% of adults are unable to receive proper care for their illnesses resulting from the fees for medical care. Furthermore, earlier this year, LN reported the FDA’s approval of their second ever gene-therapy, the Car T - cell method for increasing longevity in 94% of multiple myeloma patients. However, as a result of the fees, 25% of those with cancer cannot obtain proper treatment.
If Spark Therapeutics decides to rate their product in such a way, most with the heritable disease will likely be unable to find a remedy. The advent of gene therapies is relatively new, and although they have proven effective, the prices disallow many from experiencing the benefits.
Indeed, research is an expensive task requiring years of devotion to uncovering cures. Nonetheless, the purpose of the pharmaceutical industry is to create relief for the millions suffering from numerous conditions. One could argue that lower charges are the moral choice.
While many continue to endure retinal degeneration, Americans can only hope that remedies will someday become more reasonably priced. Give us a call on our LIVE author chat to tell us if you believe the current estimates for Luxturna are fair.
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