TreatSMA has received a formal response from NHS England to the letter sent by the UK medical community about the Expanded Access Programme of nusinersen.
We are bitterly disappointed to note that NHS England fails to understand the basics of spinal muscular atrophy and nusinersen EAP:
- that nusinersen has been reviewed by the European Medicines Agency and found effective in the entire spectrum of spinal muscular atrophy, that including in particular those with its most severe form,
- that clinical trial designs in rare disorders, especially in such a phenotypically varied disorder as SMA, cannot reasonably include all the possible patient categories in whom the treatment will have clinical benefit, for reasons of statistical power, group homogeneity, and cohort size limits, and are instead based on prevalence,
- that nusinersen is offered through EAP on a purely compassionate basis which by definition does not require providing hard proof of efficacy,
- that saving lives through EAP involves no physical costs nor any additional budget from the government since drug cost is fully covered by its manufacturer and drug administration can be done in a simple outpatient procedure,
- that nowhere in the world where EAP has been in operation – which is in nearly 20 countries – have the health authorities imposed such draconian restrictions on nusinersen EAP as in England and Wales.
Considering all of the above, we view the stated requirement of providing “published data” as deliberate obstruction being placed before doctors who have been striving to save the most precious lives of our children. We strongly reject the entire letter and call on NHS to stop focusing on policies and procedures and, for once, see the patient and listen attentively to the voice of medical experts.
We the UK SMA community will not give up our right to receive effective and life-saving treatments.