How to pay for very high cost, curative drugs? ICER and NICE put their heads together

When Novartis’ hinted last year that its SMA gene therapy Zolgensma could cost $4m per patient, it put healthcare systems on notice that a medicines revolution is fast approaching.

Like many other cell and gene therapies, it could represent a cure for the disease – but comes with a huge upfront cost that healthcare systems aren’t currently set up to handle.

Now three health technology assessment (HTA) organisations are collaborating on how to adapt their cost effectiveness and value for money assessments.

The US-based Institute for Clinical and Economic Review (ICER), England’s National Institute for Health and Care Excellence and the Canadian Agency for Drugs and Technologies in Health (CADTH) are to work together on the ‘Valuing a Cure’ project.

ICER's Steven Pearson

“Patients and those who care for them eagerly anticipate the coming wave of treatments that may cure a wide range of illnesses,” said Steven D. Pearson, MD, MSc, President of ICER.

“The science is undeniably exciting. But when these treatments are first launched, which is when pricing and coverage decisions have to be made, the evidence on the long-term value of these treatments may be extremely limited. Given the stakes for patients, and the high prices that one-time curative treatments are likely to command, we are taking the lead now to assemble and work with key experts to ensure we are using the best possible methods for assessing the value of these treatments."

Pearson said that without this work, HTA bodies risked over or undervaluing these potential cures, which could harm patient care and healthcare systems.

He added that attempts to build a consensus around new economic models would be “essential to arm the policymaker, payer, and manufacturer communities with a platform that can reward innovation while supporting a sustainable health insurance system.”

ICER, a privately funded institute based in Boston, Massachusetts first came to prominence in 2015 when it called for Gilead to lower its US price of Sovaldi, which represented the first cure for hepatitis C. England's NICE has also become famous for its run-ins with pharma companies, but has also shown itself willing to try out new market acess models. It is now working closely with NHS England (which negotiates directly on price with manufacturers), and last year gave the green light to the first CAR-T therapies, Novartis' Kymriah and Gilead's Yescarta.

The three HTA organisations are now inviting stakeholders to comment on the Valuing a Cure plans, and to address some of the biggest questions about future value and payment models.

The collaboration between the HTA bodies reflects similar moves in Europe, where groups of countries are coming together to make joint assessments. These include the Beneluxa initiative, currently including five countries, alongside a EU goal of harmonising HTA across member states via new legislation this year.

Meanwhile, Novartis says it has already begun early-stage talks with healthcare systems about new approaches to paying for very high cost curative treatments like Zolgensma (now filed with regulators), including a reinsurance model to underwrite the cost.