Gene Therapy...Quíen paga la cuenta?

Novartis has said 

its forthcoming spinal muscular

 atrophy gene therapy 

could be cost-effective 

at a price of up to $5 million, 

but now the company's CEO says 

to think "far lower." (Ver)

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.

Pricing a Cure




The question for health systems: how much are one-time therapies worth?

Rational prices are critical to ensure the breakthroughs get to patients who desperately need them, said Steven Pearson, president of the Institute for Clinical and Economic Review, a nonprofit whose reports on pricing have gained influence in the debate over U.S. drug spending. If they’re too low, it could deter investment, while exorbitant costs would strain health networks.

“We have to create a system that means we don’t have a speeding freight train hitting a brick wall, and really high prices that seem disconnected from the value to patients,” he said in an interview. Without that, “it’s likely the system will freeze up.”

Novartis’s Zolgensma is a test case for the budding field. The treatment for spinal muscular atrophy awaits a regulatory decision in the U.S., where UBS Group analysts assume it will carry a price tag of about $2 million. That would make it the country’s first multimillion-dollar therapy.

In a February report, Boston-based ICER highlighted figures showing the treatment could be worth as much as $1.5 million -- and as little as $310,000. Novartis, however, focused on different measures in the analysis suggesting a much greater value of as much as $5 million. 

Ver:
Novartis prices Zogensma at $2.125M


ICER responded last week, saying that the cost of Zolgensma “should be lower than the hypothetical $4-5 million price the manufacturer has suggested could be justified.” 

Ver:
Zolgensma / Novartis: Titulares de "consenso"...

Installment Plans

The alternative to Zolgensma is the current treatment for spinal muscular atrophy: Biogen Inc.’s Spinraza, which patients take for life. Biogen said its treatment, launched in the U.S. two years ago, is backed by more evidence than Zolgensma, though Novartis said it now has almost five years of data. Spinraza costs about $4.1 million over a decade.

Novartis’s installment plans would spread out payments over five years, Narasimhan said. The company would also offer reimbursement if a patient dies or the treatment otherwise fails within that period.

“We ask for payment over five years, then it goes to zero,” he said. “Think about the value provided as the medicine continues to keep children alive. It’s enormous.”(Ver)

Ahead of Novartis' likely Zolgensma approval, 

Narasimhan called for changes 

to current drug pricing models. 

Drugs are now sold 

on a “pay-as-you-go” basis, 

the CEO wrote 

in a recent CNBC commentary.

(Ver)