“It’s a lottery where we’re leaving children’s lives up to chance. I don’t think it’s a game that any of us want to play.” This is a parent’s response to the news that Novartis has started to receive applications for a lottery-based program to give away 100 doses of its $2.1m gene therapy treatment Zolgensma for the debilitating spinal muscular atrophy (SMA) in countries where it is currently unlicensed.
Spinal muscular atrophy (SMA) is a genetic, progressive, and sometimes fatal neuromuscular disease. The In the natural history of the disease, individuals with type 1 will never achieve the ability to roll over or sit on their own and their life expectancy is under two years.
This global lottery aims to give patients an equal chance of being selected to receive the breakthrough treatment for this expensive and hard to produce therapy. The company was advised to take the lottery approach by a group of bioethics experts, who were concerned that creating complicated criteria could unfairly discriminate against some patients. Novartis might have been better served to also speak with patient groups and secure their buy-in to how the managed access program would be administered.
The SMA patient community demand for Zolgesma and its potential to add years to life far outstrips the supply. Desperate parents are looking for novel ways to get the therapy for their children, including this example of crowdfunding to purchase Zolgensma for a 9 month old. Although it’s clear Novartis is trying to offer a fair system to supply a life changing therapy to patients in countries where they may never get access, given the outcry of patient groups it is not clear that Novartis has benefitted.
Due to the geographical spread of the lottery, Novartis cannot guarantee lottery recipients a comprehensive medical follow-up at an expert center. They are simply offering the therapy. Patients will be relying on their primary caregivers who may be inexperienced with the novel gene therapy and the management of potential side effects or adverse reactions and there is little chance for follow up and collection of real-world evidence which could benefit not just the patient but the whole community?
While not every patient lives in an area close to an expert center, wouldn’t it make more sense to tie the compassionate use therapy distribution to expert centers with the ability to track patient experience and provide holistic care in the real world?