Big pharma is taking notice; the most recent gene therapy deal signed in February, between Sanofi’s Genzyme unit and Third Rock Ventures’ Voyager Therapeutics, both in Cambridge, Massachusetts, is worth up to 5 million.
But as gene therapies start to provide solutions for highly penetrant genetic diseases that had been intractable, the hand-wringing over their value and how government and private insurers around the globe will pay for them will likely begin.
Bluebird’s BB305 may be among the first products that meet those criteria.
The impact of the company’s gene therapy on beta-thalassemia patients who would otherwise require chronic blood transfusions is “measurable, with the ability to track on a patient-by-patient basis their level of hemoglobin, which has a direct correlation to whether they’re anemic,” says Walsh.
Uniqure has six-year follow-up data showing a 50% reduction in pancreatitis in Glybera-treated patients, he said, and dividing that up-front price by six years “you get to a [per-year] price that’s actually lower than [typical] orphan drug pricing,” he said.
Pricing gene therapies might follow one of three general schemes.
BB305 treats those diseases by using a lentiviral vector to transfect a patient’s own hematopoietic stem cells ex vivo with a functional human beta-globin gene, then returning those cells to the patient.In addition, he says, “quality of life is another major component of value,” in particular when you think about how a one-time treatment can replace a lifetime of medical interventions.And lastly, he says, “if a patient is having challenges maintaining an education or a job, and a curative therapy alters that equation, there’s societal value” to the therapy as well.As the first and only approved gene therapy in Europe or the US, Glybera (alipogen tiparvovec) will likely become a bench-mark product.Glybera was recommended for approval by the European Medicines Agency in 2012 (Nat. 30, 1153, 2012), and Chiesi plans to launch the product in Europe in early 2015, though it has yet to receive a nod from the US Food and Drug Administration (FDA).