Novartis plans to give away up to 100 doses annually of Zolgensma, its $2.1 million spinal muscular atrophy treatment, under a new managed-access program, according to TreatSMA.
Starting Jan. 2, Novartis' AveXis unit, which developed Zolgensma, will offer free doses of the world's most expensive drug to patients meeting specific criteria.
The program is only available in countries where Zolgensma is not an approved drug, intended as a way to improve SMA treatment in those countries. Once a country approves Zolgensma, submissions from that country will no longer be accepted.
The qualifications to receive the drug include: not requiring respiratory support for more than 16 hours a day and a certain baseline functional score yet to be clarified, according to TreatSMA, a patient support association based in England. Patients must also be under 2 years old.
Clinicians can submit email requests to AveXis for Zolgensma for their patients. Clinicians will receive an answer within a week or so.
An independent commission will conduct biweekly drawings for free Zolgensma doses, according to TreatSMA. Patients not selected in one drawing will be automatically entered into future drawings until they are either selected or no longer eligible.
Novartis said it plans to run the program globally for several years and give up to 100 free doses per year.
"As much as we applaud AveXis’s initiative to offer Zolgensma globally at no cost, given the lack of access to any SMA treatment in many places, we are yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease," TreatSMA wrote.
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