As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when all of them are equally in need. That’s the case for the new gene therapy ‘Zolgensma’, famously the most expensive medicine ever, with a $2.1 million price tag. This extremely high cost is supposedly justified because of its momentous results. The treatment corrects the mutated gene, in turn halting spinal muscular atrophy (SMA) from running its course, which is terminal without intervention. The genetic disease causes nerve cells to deteriorate, leading to paralysis within the first couple of years of life. The therapy cannot recover already damaged neurons, but it stops any further erosion by curing the mutation responsible for the nerve cell deterioration. After the therapy, patients can expect relatively normal health, and no further repercussions from the disease.
The drug manufacturer has announced a distribution plan that’s receiving criticism from patient groups: a lottery will decide which lucky patients will be gifted with their lives by receiving a free dose. It will give the live-saving treatment to 100 lucky children with the disease under the age of two. This has sparked criticism from patients, who value the possibility of saving lives but feel that deciding through a lottery is cruel.
The Backlash
Patient groups argue that using a lottery to allocate the drug gives the whole population hope when there is still little, and divides them into a competition for their lives. These groups prefer that the patients be selected for specific clinical needs.
The patient organization points out it shouldn’t be a pharma company deciding who gets the drug as they are not the appropriate actor to make greater ethical decisions that could be the difference between a future and death in infancy. The drug developer AveXis responds to this criticism, stating that they used independent ethical experts in efforts to create a fair distribution. President Davion Lennon said,
“Unfortunately there are many babies out there who deserve this medication, and we can’t produce quickly enough to treat them all,” and so, he said, “This was the only way we could think about making a fair and equitable allocation of the product that is available.”
With rare diseases, each patient can potentially be impactful to research. Some patients display different variations of symptoms or have different mutations causing the onset. These details are important to future improvements. There are not many data points, so each one can add a whole new shape to the understanding of the disease.
A lottery can not have been the only ‘fair’ and equitable allocation of the product. A lottery is fair in the sense that it gives patients equal chance, but fairness and chance are different. Giving every patient a random chance is not the same as distributing fairly. If we treat the SMA patients and families as a community in need of further medical research and accessibility, it is most fair to give it to those most likely to aid that mission or who are in the most urgent need. Chance, on the other hand, is why the lottery is so charged; it is a game that plays off participants’ hope. Like gambling, lotteries give participants the anticipation and hope that their bid has as good of as a chance as any. This sets patient families on an emotional rollercoaster. This is inherent in lottery systems: everyone has a chance and can develop expectations, but the actual winners are only a fraction. For this therapy, 100 lucky patients will be regifted their lives, while thousands of others will receive the penalty of eminent health decline.
Rare disease patients know the how vital unity is; a rare disease community must work together to get cures. A lottery is a game that drug manufacturers should not be playing.
