Leading UK physicians and SMA advocacy groups have sent a strongly worded letter to Sir David Haslam, Chair of the National Institute of Health and Care Excellence (NICE), objecting to the proposed negative recommendation on nusinersen therapy.
We are writing to you as clinicians involved in the care and translational research of children with a devastating neuromuscular disease, spinal muscular atrophy (SMA), to express our strong dissatisfaction with the recent decision of NICE technology appraisal committee not to recommend nusinersen for reimbursement in England.
The signatories, who include a number of renowned neuromuscular experts, underline the clinical effectiveness of nusinersen therapy.
Nusinersen has clearly demonstrated a very robust therapeutic effect with highly significant positive results on the functional outcome of affected SMA children. (…) Severely affected children with SMA1 (…) now have the prospect of a therapy that – especially if initiated close to the onset of disease – can very substantially reduce the respiratory comorbidities and deaths; allows a proportion of affected children the ability to acquire the sitting position; to stand, and to participate to life by speaking, as they, for the first time can move sufficient air to generate speech.
The experts took a jab at NICE’s complaint about “lack of data” and reminded that NHS itself has prevented collection of nusinersen efficacy data by deliberately obstructing the opening of the Nusinersen Expanded Access Programme, which also forced countless UK families to seek life-saving treatment abroad.
We are disappointed to report that England was one of the last countries to enable the EAP to be initiated (August 2017). At that time the EAP had been running in 23 other countries – 17 from Europe – and the investigators and clinicians were reporting data on feasibility and efficacy on their EAP treated population. This led dozen of patients and their families to go abroad to get access to the EAP (for example to France; Austria, Germany).
We should stress here that treatment abroad for UK families was only possible through the good will of foreign clinicians coupled with funding from foreign SMA organisations. Then, it was a co-ordinated push from TreatSMA, other SMA charities and the medical community that forced NHS England to finally agree to start EAP as the last country in the world.
The clinicians then mention ethical ramifications which go contrary to medical ethics:
We are the physicians in the frontline who manage these patients and are currently and reluctantly needing to manage the frustration of families who are faced with accepting the deterioration and death of their children despite an effective treatment being available.
The experts then criticise the appraisal mechanism of novel drugs for orphan diseases:
We also feel that the complete lack of flexibility in the way NHSE and NICE appraise drugs for these groups of patients, fails to provide an effective mechanism to respond to the needs of subgroups of children with devastating conditions.
Finally, the signatories issue a stern warning to the policy makers:
We finally wish to point out that while the research infrastructure of England, heavily supported via NIHR, is one of the most efficient in the world; the mechanism of assessment and potential adoption of novel drugs is antiquated and unfit for purpose of the 21st century. This has started to alienate industrial partners from considering this country as “the place” to perform experimental studies, with detrimental consequences for our research environment and eventually the health of our patients.
The letter, signed by Professor Dr med. Francesco Muntoni and Dr med. Rosaline Quinlivan, was co-signed by a number of clinical experts representing the SMA REACH initiative and the British Myology Society as well as the representatives of TreatSMA, SMA Support UK, the SMA Trust, and MD UK.