Gene therapy challenges
When the Food and Drug Administration approves the first gene therapy for a disease with one gene, which is expected in January, insurers and sponsoring companies will have plans to ensure that patients can actually receive the new treatments and not only have access to these treatments. to them? That is currently a matter of intense focus.
The first clinical test for gene therapy was already in 1990. But in the past year or two, the treatments are approaching the end of the regulatory path. Some claims are incremental and incomplete, others are amazing, perhaps no more than the treatment of a form of inherited blindness that was the subject of an FDA advisory committee on 12 October.
Patients who had received a Luxturna injection in each eye reported that they could see the facial expressions for the first time and stared at the sun, the moon and the stars in awe.
Christian Guardino, then 16, demonstrated the success of gene therapy when he sang Jackson's Who's Lovin 'You' on America's Got Talent in June. Prior to gene therapy he could not recognize faces, he could not walk through the corridors of his school or he could not stay safe when the sun went down. Gene therapy has made my world so brighter. I can even walk around on stage and perform and not just stand in one place. My vision has been stable for 4 years and if I had not got it, I would have been completely blind now, \he told the FDA.
It took only a few seconds for the committee to pass the new treatment to the next phase. But how long does it take for the first patients to be treated for these and other emerging gene therapies outside the clinical research institutions?
Is $ 1 million too much?
The initial costs are a problem for every pioneering medical procedure or treatment. But unlike insurance coverage of chronic treatments, such as cholesterol-lowering drugs, gene therapy can disrupt pathology. It can possibly heal. So the best comparison can be organ transplants. Consider their costs and annual markets in the US:
• Heart: $ 138,240 for 2,725 patients
• Kidney $ 414,800 for 16,804 patients
• Bone marrow (from donor) $ 892,700 for 9,284 patients
• Deliver $ 812,800 for 6,158 patients
Acute lymphocytic leukemia
So far, gene therapies are in the same cost cage. Kymriah, the recently approved treatment that delivers a manipulated immune system protein in gene therapy packages, is a one-off treatment for a form of leukemia that costs $ 475,000. Yescarta, for another blood cancer, is also priced.
The seven-digit cap may come from experience with Glybera, the first gene therapy approved in Europe. Despite decades of development, the drug was drawn after only two patients had it, the $ 1 million plus costs that were considered excessive. The second gene therapy approved in Europe, Strimvelis, to treat an inherited immune deficiency, costs $ 665,000.
Another story
Gene therapies bring different challenges than conventional drugs, even organ transplants.
The main costs for gene therapy at R u0026 D for gene therapy are provisional, with the vectors designed to deliver the healing genes. That can cost $ 500,000 to $ 1 million, but the delivery can be optimal, almost anti-climax.
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