LONDON - With uncharacteristic speed, the National Health Service (NHS) has agreed to reimburse treatment with Novartis AG's Kymriah CAR T immunotherapy, little more than a week after the product received formal approval from the European Commission.
That makes NHS England the first health care provider in Europe to allow children and young adults under 25 years of age access to the treatment for B-cell acute lymphoblastic leukemia, which has a U.K. list price of £282,000 (US$361,000) per patient.
To secure access, Novartis has agreed to a lower price with the NHS, which, as is the case for such deals, was not disclosed.
Basel, Switzerland-based Novartis got Kymriah (tisagenlecleucel) to market in the U.K. days after Gilead Sciences Inc.'s CAR T therapy, Yescarta (axicabtagene ciloleucel), was rejected by the National Institute for Health and Care Excellence (NICE), as not being cost effective.
The U.K. has been laying the ground for the arrival of advanced cell and gene therapies for some time, setting up specialist medical centers where they will be administered. The first three NHS hospitals to go through the international accreditation process for the provision of CAR T therapy for children are in London, Manchester and Newcastle. (See BioWorld, March 13, 2018.)
Subject to passing accreditation requirements the first treatments could begin in a matter of weeks, according the head of NHS England, Simon Stevens, who announced the Kymriah access agreement in a speech in Manchester Wednesday.
"CAR T therapy is a true game-changer, and NHS cancer patients are now going to be amongst the first in the world to benefit," said Stevens. The approval "is proof-positive" the NHS is leading from the front on innovative new treatments.
The "constructive fast-track" negotiation also shows how responsible and flexible life sciences companies can succeed in making revolutionary treatments available to patients, Stevens said.
It is expected that around 30 patients per year who have failed other therapies will be eligible for treatment with Kymriah.
For now, patient T cells will be processed at Novartis' facility in Morris Plains, N.J. However, following marketing approval in Europe, the company announced plans to build a new plant for manufacturing cell and gene therapies at Stein in Switzerland, and also sealed an agreement with Cellforcure, a French contract manufacturing company.
Reaching agreement on access to Kymriah only 10 days after its regulatory approval may look like breakneck speed, but pulling the rabbit from the hat has required extensive groundwork.
Much of that effort has been orchestrated by the Cell and Gene Therapy Catapult center, a government-funded organization set up in 2012 to support process development, commercialization and market access for advanced therapies.
In addition to lobbying for the setting up of the Advanced Therapy Treatment Centers, for which it provides a central management function, the Catapult has commissioned economic assessments and formulated new payment methods for one-off expensive therapies.
In particular, the Catapult worked with health economists at York University to model adoption of a hypothetical T-cell cancer immunotherapy (bearing close resemblance to Kymriah). (See BioWorld, March 16, 2018.)
However, as Yescarta and other expensive therapies such as Biogen Inc.'s Spinraza (nusinersen) treatment for spinal muscular atrophy illustrate, the key to loosening NHS purse strings lies in what reduction to the listed price manufacturers are persuaded to offer.
In draft guidance published Aug. 28, NICE recommended against the use of Yescarta, saying the cost per quality adjusted life year (Qaly) was above the upper limit of £50,000 (US$64,800) per year allowed for specialist cancer treatments.
There is the prospect of some arm twisting ahead, with Meindert Boysen, director of the center for health technology evaluation at NICE, calling Yescarta "an exciting innovation" in a very difficult to treat cancer, with a promise of cure for some patients. "Unfortunately, in this case, we are not able to recommend [Yescarta] for use in the NHS in England at the cost per patient set by [Gilead]," Boysen said.
The case is open for submission of further information up to Sept. 15, with the NICE committee due to meet and mull any new evidence on Sept. 27.
Similarly, on Aug. 14, NICE said it would "welcome further discussions" about the price of Spinraza after concluding that, based on the list price, its cost-effectiveness ranged between £400,000 and £600,000 per Qaly gained. Even with a proposed confidential price reduction, the cost is too high for Spinraza to be considered a cost-effective use of NHS resources, Boysen said.