Position Statement regarding Edaravone/Radicava®
The Federal Drugs Agency (FDA) licensed Edaravone (Radicava®) for people with Motor Neurone Disease (pwMND) of the ALS type in the USA in 2017 [FDA, May 5, 2017]. A number of expert consensus reviews/position statements have followed in Europe and the United Kingdom. This position statement summarises the views of MND Specialists in Scotland.
A recent clinical trial reported in a post hoc analysis that a subgroup of pwMND experienced beneficial effects from edaravone treatment. This subgroup consisted of patients with early-stage ALS in whom disease progression was rapid. A second randomised, double-blind, parallel-group trial of 137 patients, in which inclusion criteria were restricted to a group of early stage patients, investigated a dosage of 60 mg Edaravone; initiation of riluzole, the current standard therapy for ALS, was prohibited in this trial. This trial confirmed the results of the post hoc analysis by demonstrating a significant slowing of disease progression measured using the established clinical score, the ALS Functional Rating Scale (ALS-FRS).
In our expert view the small number and subtype of pwMND investigated and the short duration of follow up (24 weeks) mean that the study results can only be interpreted in relation to a minority of pwMND Furthermore effect of the drug on survival, and advantage exerted over the current standard of care with Riluzole, has not been determined. Administration is challenging (delivered as an infusion 2 weeks out of 4), and side effects include bruising, gait disturbance, hives, swelling, shortness of breath and anaphylaxis to sodium bisulphite.
The drug has yet to receive approval from the European Medical Agency for licensing in Europe (including the UK), the National Institute for Clinical Excellence, or the Scottish Medicines Consortium. An expert grouping of clinical specialists in the United Kingdom has advocated caution in prescribing Edaravone until there is a stronger body of evidence for its use from further clinical trials, and appropriate European and National regulatory approvals are in place (ENCALS statement on Edaravone, 2017). The drug company, Mitsubishi Tanabe Pharma Corporation have confirmed there are no plans for implementing a drug trial of Edaravone in the United Kingdom (May 2018).
A Managed Access Program for pwMND is available, however a named MND Specialist must be prepared to prescribe, monitor and administer Edaravone. MND specialists from across NHS Scotland agree that such a programme given the very limited benefit in a fraction of the MND population and logistical challenges of safe long-term delivery is not presently viable. Administration of the drug, applications or requests for the Managed Access Program are not being supported at present in NHS Scotland. This is similar to the position in England and Wales. We remain very enthusiastic to identify safe and effective therapies and will ensure these are made available to pwMND in a timely way once the evidence for their use is secure.
For further information please go to the MNDA blog.
Reviewed by Scottish MND Steering Committee , June 2018
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