Baby Pia, the Belgian toddler whose family worked furiously to crowdfund to raise $2 million for the world’s most expensive drug, in fact, received an infusion of the drug which can potentially help her avoid a life-threatening condition. TrialSite News actually made a plea to Novartis and surely glad Baby Pia has been treated.
Baby Pia suffers from spinal muscular atrophy (SMA)—she made the headlines earlier when her family successfully completed a crowdfunding campaign that raised $2 million required for access to the drug. Due to a massive outpour, especially large numbers in the Belgian community, the family was able to raise the funds reports the Brussels Times.
Next Steps Watch and Wait
Ellen de Meyer, Pia’s mom, now knows she must sit and wait as “The drug must do its work.” The first sign of success will be that Pia would be able to sit down on her own. The hope and prayer are that she will be able to site down in a wheelchair and live a “beautiful and independent life.”
Novartis Changes Position
TrialSite News first observed that Novartis wasn’t willing to allow for any compassionate use even with the funds available from the family’s crowdfunding effort. However, something obviously changed. Principals within TrialSite News have put together technology deals in the past with Novartis and know that there are incredibly good, caring people working in Novartis. Obviously, the company must satisfy shareholder requirements but it must also continuously build a powerful brand. By changing its position we commend Novartis.
Balancing Economy & Humanity
However, the incident in Belgium raised the issue of price points for this powerful new gene therapy. It is difficult for health systems to adjust to the economics behind the pricing of Zolgensma. On the one hand, Novartis must profit as a publicly-traded company beholden to shareholders and on the other hand, it has a responsibility as a global multi-national to contribute to the world’s health—finding a way for a win-win—could be quite compelling for its brand.