Gene Therapy Was Hailed as a Revolution. Then Came the Bill.

By James Paton: For More Info, Go Here…

Dozens of revolutionary gene therapies that mend faulty strands of DNA are on their way, bringing the power to eliminate lethal childhood diseases, rare blood disorders and other severe illnesses.

Beneath the excitement about these potential cures lies an important catch: No one knows how much to charge for them.

The new therapies aim to fix the root causes of disease with a single dose, in which the correct genetic material is introduced into the patient’s cells. If the treatments can replace a lifetime of conventional costly drugs, they may slash overall spending, even at multimillion-dollar prices. Yet the prospect of high costs is already stirring pushback.

“Most health-care payers in the world just haven’t had experience with this, and they’re going to have to come up with new models, especially when there are so many of these therapies coming,” said Vas Narasimhan, chief executive officer of Novartis AG. “We need the system to be able to adapt.”

The Swiss pharma giant says one answer could be installment payments, reducing the upfront cost. That’s what it plans for Zolgensma, a therapy for a devastating muscle disease that’s expected to get approval soon.

More drugmakers are betting gene therapies will have a big impact on patients and profits, with Pfizer Inc. last month agreeing to collaborate with Paris-based Vivet Therapeutics on a treatment for a rare liver disorder. The U.S. Food and Drug Administration forecasts as many as 20 cell- and gene-therapy approvals each year by 2025. Doubts remain about whether the treatments will sustain their dramatic results, making it difficult to determine their value.


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