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    Money-back guarantee on $665K gene-therapy, the most expensive drug in history

    A gene therapy will be offered for sale in Europe with a money-back guarantee, according to GlaxoSmithKline, the company commercializing it.
    According to MIT Technology Review, The one-time treatment, called Strimvelis, is the first outright cure for a rare disorder to emerge from gene therapy. Priced at 594,000 euros ($665,000), it’s among the most expensive treatments ever sold by a drug firm. The specific therapy is a cure for severe immunodeficiency stemming from a lack of adenosine deaminase (ADA-SCID), rather than an ongoing treatment as other rare disease drugs are.
    “The drug has to deliver what you say or we don’t pay,” says Luca Pani, director general of the Italian Medicines Agency, known as AIFA, which set the price and terms during negotiations with the British drug giant. “If it does not work, they will return the money.”
    The treatment employs a virus to add a missing gene to the bone marrow of children with ADA-SCID, a sometimes fatal inability to fight infections. In a study involving eighteen children, carried out at a Milan hospital, all but three were cured outright. GSK bought rights to the Strimvelis in 2010 and won approval earlier this year to sell it in Europe, but because of its complexity the company will offer it only in Milan, requiring families to travel and spend weeks there. That means the Italian price will apply to all of Europe, says Pani. He also said that GSK would also be paid in installments over a period of years.
    GSK says it won’t make much money off Strimvelis. Instead, it sees the treatment as a way to help patients and gain experience with treatments involving cells and genes. It’s also working with a small company, Adaptimmune, to genetically alter immune cells to battle cancer. “We do not expect to recover all of the costs of building a platform to deliver gene and cell therapy from Strimvelis alone,” says Anna Padula, a spokesperson for GSK’s rare-diseases group. “We hope that Strimvelis will be the first of a number of innovative gene-therapy medicines that we will bring to patients.” The company recognizes, she adds, “that the industry will need to adapt the way in which medicines are priced and funded.”