The Scottish Medicines Consortium (SMC) issued a negative recommendation on the use of Fampyra (fampridine; marketed as Ampyra in the U.S.) in the National Health System (NHS), for the treatment of multiple sclerosis (MS) patients who have walking disabilities, citing the medicine’s poor cost-effectiveness.
After reviewing the available clinical data on Fampyra’s effectiveness, safety and economical value, and gathering healthcare professionals and patient group opinions, the SMC board, an official adviser to NHS Scotland, decided not to approve the medicine for the NHS.
“After careful consideration, SMC decided the evidence provided by the company was not strong enough to be certain that fampridine offers value for money to NHS Scotland,” the advisers said in a public summary of the decision.
This decision means that, in Scotland, this therapy should not typically be prescribed on the NHS, and other treatment options should be considered. However, in cases where a doctor believes Fampyra would benefit a certain patient, he or she can still request its prescription.
The medicine had already been rejected once by NHS Scotland in 2016, but Biogen, the company marketing the treatment, asked for a re-evaluation after submitting additional data from clinical trials and real-world studies earlier this year.
Fampyra (prolonged-release tablets of fampridine) is the first approved treatment to improve walking in adults with MS and walking disability (EDSS score between 4 and 7). It is approved in the U.S. under the brand name Ampyra, marketed by Acorda Therapeutics, and in Europe, where Biogen holds the marketing license.
The medicine is a potassium channel blocker that improves the conduction of nerve impulses. In other words, it helps signals that come from the brain to continue along damaged nerves in MS patients and command the muscles, which can help ease gait problems.
According to a related press release by the the U.K. Multiple Sclerosis Trust, Fampyra is efficient for only a part of the MS population. In clinical trials, approximately one-third of the patients had an average 25% improvement in walking speed, compared with those given placebo.
“Many people living with MS are struggling to cope with the impact the condition has on their mobility. Walking problems can affect many of the things we all take for granted,” Linden Muirhead, director of information and engagement of MS Trust, said in the release. Therefore, “even small improvements in walking can bring many benefits to someone living with MS.”
“Today’s disappointing decision means that people with MS are being left to self-fund prescriptions,” an effort that is beyond the reach of many, “especially those who are unable to work,” Muirhead said. “We know that times are tight for the NHS, but people with MS deserve access to the independence and dignity this treatment can bring.”