The National Institute for Health and Care Excellence, better known as NICE, issued a final decision against including Ocrevus (ocrelizumab) as a treatment for primary progressive multiple sclerosis (PPMS) in the subsidized public health system for England and Wales.
The agency’s “final appraisal,” which mirrors its draft June ruling, is based on the cost-effectiveness of the Genentech therapy — its price versus its benefits as a PPMS treatment offered patients using the public-funded National Healthcare System (NHS).
“The most plausible cost-effectiveness estimates for ocrelizumab compared with best supportive care alone are much higher than those NICE normally considers an acceptable use of limited NHS resources,” the final appraisal states. “Because of this, ocrelizumab cannot be recommended for treating early primary progressive multiple sclerosis in adults.”
A positive NICE opinion obligates the NHS to offer the therapy at no or low cost.
Reaction by the MS Trust was swift and critical of the ruling, and the U.K. patient advocacy organization said it would continue to push for Ocrevus’ inclusion for PPMS patients.
“There are currently no approved treatments for PPMS and people with this form of MS experience disability significantly quicker than those with other forms. The lack of treatments that can modify their disease often forces them to rely on wheelchairs and mobility aids sooner, impacting on their independence,” Jo Sopala, director of Health Professional Programmes at the MS Trust, said in a press release.
“People do everything they can to minimize the impact of PPMS has on their lives, but what they really want is access to treatment which will slow down the progression of their disease,” Sopala added, calling on NICE “to overturn the decision for ocrelizumab as soon as possible.”
But in its release, the Trust acknowledged that a reversal at this stage was unlikely. The decision, it added, came despite the “nearly 500 responses from people with PPMS, their families and specialist MS health professionals” that the group had collected in support a NICE ruling in favor of Ocrevus for PPMS patients.
Ocrevus is approved across the EU and in the U.S. to treat both relapsing-remitting MS (RRMS) and PPMS, but is costly. NICE initially ruled against its NHS inclusion for relapsing MS patients as well, but reversed that position in June, after it reached an agreement with Genentech (part of the Roche group) on an undisclosed price. Ocrevus, administered as a total 600 mg intravenous infusion every six months, sells in the U.S. at an estimated annual list price of $65,000.
Genentech also offered a reduced price for PPMS patients in the NHS, the MS Trust noted, but was again rejected.
NICE acknowledge that its decision left PPMS patients without easy access to any disease-modifying treatment, and that a pivotal study in these patients — especially those with early stage disease — showed benefit in terms of slower disability worsening. But the agency added, “the size and duration of this effect are uncertain.”
Roche noted in its press release that NICE also recognized Ocrevus as an “innovative treatment that provides a step change in the treatment of PPMS” even as it rejected it. The company said it remained open to working with NICE for NHS inclusion.
A specialist quoted in the Roche release was scathing in his criticism of NICE and its final appraisal.
“As doctors, we are left feeling powerless when we deliver the devastating diagnosis of PPMS to people because we know there is currently no disease-modifying treatment available to help them,” said Gavin Giovannoni, a neurologist at Barts and the London School of Medicine and Dentistry. “It is even more frustrating that an effective treatment that can help slow the disease has been developed and made available across the globe yet people in England and Wales will continue to suffer disability worsening because of an archaic and inflexible medicine assessment system.”