Novartis has responded to criticism over its managed access programme for spinal muscular atrophy (SMA) gene therapy Zolgensma, with patient groups accusing the company of exercising an unfair ‘health lottery’.
Novartis made history when its brought its gene therapy Zolgensma to market, for mixed reasons. Although it is the first therapy which promises a one-shot cure for a deadly neurodegenerative disease – SMA – it is also the most-expensive drug in the world, costing $2.1m for that single dose.
That posed a problem to payers and patients alike, with many criticising the price for making the innovative treatment almost impossible to access.
To salve this criticisms, Novartis announced plans for a managed access scheme for the gene therapy, offering a lottery draw for 100 infants and children suffering with SMA to gain Zolgensma treatment.
The Swiss pharma said it will draw one child every few weeks, who will then go on to have free treatment with the gene therapy. Around one in 8,000 to 10,000 people across the globe have SMA, which often overwhelmingly affects boys and can lead to death before a child reaches teenage-hood.
The plan has been heavily criticised by patient groups, including SMA Europe. The organisation said in a statement that the MAP was received with “mixed feelings”, saying that it “fully appreciates the fact that the programme will offer access to this promising therapeutic option to a number of infants across the globe”.
However, its highlighted the unfairness of the “health lottery”, which it deemed to be an “inappropriate way of addressing the unmet medical need in this severe disease”.
Importantly, the group also presented concerns over the lack of involvement of neuromuscular experts as well as patient organisations in consulting on the design and implementation of the programme.
For SMA Europe, the fact that it is Novartis rather than a treating clinician who will be making the decisions over medical treatment for children with SMA is “unacceptable”.
“We are alarmed that the programme will make thousands of SMA babies compete with each other for a life-saving treatment, splitting tightly knit communities and turning this experimental drug into a coveted prize,” it added.
In response to the criticism, Novartis said that it “has heard the feedback from the SMA community on the design of the MAP and are actively engaging with patient advocacy groups and healthcare professionals to capture their insights”.