NICE Allows Rethink on Primary Progressive MS Drug

Peter Russell

November 02, 2018

Multiple sclerosis charities have welcomed a decision by the National Institute for Health and Care Excellence (NICE) to delay final guidance that would have rejected a drug for treating a form of MS on cost grounds.

Among other guidance from NICE this week were announcements on a treatment option for thyroid cancer, and updated guidelines to help prevent antibiotic resistance.

Ocrelizumab for Primary Progressive MS

NICE delayed issuing final guidance on the use of ocrelizumab (Ocrevus, Roche) for treating primary progressive multiple sclerosis (PPMS).

The final decision was due to be published on October 31st and would have rejected the drug on the grounds of cost effectiveness.

NICE said it had 'paused' the appraisal process to allow for commercial discussions between the company and NHS England to take place.

The Multiple Sclerosis Trust welcomed the move, saying it demonstrated a willingness on both sides to engage further in the debate around the disease modifying therapy, which has been shown in clinical trials to slow the worsening of disability in people with PPMS.

In July, NICE recommended ocrelizumab as an option for treating relapsing–remitting MS.

Antimicrobial Resistance

NICE updated its visual summary table for managing common infections and reducing antimicrobial resistance.

Four bacterial infections were added. They were:

The table already covered acute otitis media, acute sore throat, and acute sinusitis.

NICE said its antimicrobial prescribing guidelines "aim to ensure prescribing recommendations are consistent with antimicrobial resistance patterns, trends and data, and the best available published evidence."

Rejection for Hyperkalaemia Drugs

A negative assessment was made for the use of sodium zirconium cyclosilicate (Lokelma, AstraZeneca) or patiromer (Veltassa, Vifor Pharma) to treat hyperkalaemia.

Between 1% and 10% of hospital inpatients have hyperkalaemia. It is most common in people with chronic kidney disease, heart failure, liver disease, and adrenal insufficiency. Many people do not have symptoms but some severe cases can lead to cardiac arrest.

Evidence presented by both manufacturers assumed people would start treatment at lower blood potassium levels than in current NHS clinical practice.

The appraisal committee decided there was also no evidence to show that either drug extended life or improved quality of life compared with standard care.

In the absence of sufficient clinical evidence it decided that the drugs could not be recommended as a cost-effective use of NHS resources. The draft guidance is now with stakeholders who may comment until November 19th, 2018.

Final guidance on both drugs is expected in March next year.

Vandetanib for Thyroid Cancer

NICE published draft guidance which did not recommend vandetanib (Caprelsa, Sanofi) for treating aggressive and symptomatic medullary thyroid cancer (MTC) in adults with unresectable, locally advanced, or metastatic disease.

Vandetanib is a tyrosine kinase inhibitor which works by limiting new vessel growth and so blocking cancer cell growth and division.

The appraisal committee decided that although vandetanib might delay the disease getting worse compared with best supportive care, the benefit was uncertain. Also, the evidence about whether it increased the overall length of time people lived was unreliable, it said.

Clinical experts accepted that vandetanib was likely to be as effective as cabozantinib (Cabometyx and Cometriq, Ipsen) which NICE recommended as a treatment option earlier this year, but decided that vandetanib would not be a cost-effective use of NHS resources.

An appeal period for the draft guidance closes on November 14th, 2018.

Annually in the UK there are around 3400 cases of newly diagnosed thyroid cancer. MTC is a rare type of thyroid cancer and NICE says there are around 80 people diagnosed with it each year in England.

Darvanstrocel in Crohn's Disease

Darvadstrocel (Alofisel, Takeda) was not recommended for previously treated complex perianal fistulas in adults with non-active or mildly active luminal Crohn's disease, NICE announced in a final appraisal.

Darvadstrocel is injected directly into the perianal fistula. It is the first stem-cell treatment specifically developed to promote healing of fistulas caused by Crohn's disease.

The appraisal committee said that only an additional 14% of people showed a beneficial effect from darvadstrocel over and above placebo, based on available evidence from a single clinical trial. It also noted that specialist training for the pharmacist and surgeon would be needed for the treatment to take place.

Consultees have until November 15th, 2018 to respond to the final appraisal document, and NICE expects to publish final guidance early next month.


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