Michael Werner, executive director of the Alliance for Regenerative Medicine, says a gene therapy should be priced based on its value to the patient and to society, which he says includes quality of life improvements for the patient and potential cost savings. Several new cancer drugs cost upwards of $100,000 per year. Over a year, that comes to about $440,000. To stay healthy, he goes to his doctor 22 times a year for an injection of $20,000 worth of drugs. David Mitchell, founder of the advocacy group Patients for Affordable Drugs, has multiple myeloma, which is incurable but treatable. Some types of cancer are already incredibly expensive to treat and require that patients take drugs to keep cancer cells at bay and manage side effects of the disease. ![]() Gene therapy trials for SCID are ongoing in the U.S., and U.K.-based Orchard Therapeutics is developing a therapy that would rival GlaxoSmithKline’s. But GlaxoSmithKline has said these can cost more than $4 million for a single patient over the course of a decade. ![]() A bone marrow transplant also offers a cure, and the disease can be managed with enzyme replacement therapies. It costs 594,000 euros, or about $712,000, but comes with a money-back guarantee if patients aren’t cured. A gene therapy is already available in Europe, marketed as Strimvelis by GlaxoSmithKline. ![]() This makes babies born with it extremely vulnerable to infectious diseases, and without treatment, children will often die within the first two years of life. Severe combined immunodeficiency, also known as SCID, or “bubble boy disease,” is a rare, inherited disorder that renders the immune system virtually useless against pathogens. UniQure and Baxalta are also testing gene therapies for hemophilia, while BioMarin is investigating one for hemophilia A. Early clinical trial results from Spark Therapeutics have shown that gene therapy can essentially cure hemophilia B. Those with hemophilia A had slightly higher health-care costs-at $162,054 per year-than those with hemophilia B, which were about $127,194 per year. A 2016 analysis in the American Journal of Managed Care found that the annual average cost per patient for men with hemophilia was $155,136. As a result, patients need regular injections of clotting factor, which can cost tens to hundreds of thousands of dollars a year based on a person’s weight. People with hemophilia-mostly men-lack the necessary protein the body needs to clot blood. Earlier this year, biotech company Bluebird Bio reported that its experimental gene therapy cured a teenage boy in France. Lifetime health-care costs for a 45-year-old with sickle cell disease reached $953,640. A 2009 study in the American Journal of Hematology looked at data from the Florida Medicaid program and found that the average annual cost of health care for sickle cell patients ranged from $10,704 for children up to nine years old to $34,266 for those aged 30 to 39 years. ![]() Patients with sickle cell, an incredibly painful blood disorder, frequently end up in hospital emergency rooms, costing the U.S.
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