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  • Richard Garr

The Novartis Lottery for a Rare-disease Lifesaving Drug

Reframing the conversation around the Novartis lottery for a rare-disease lifesaving drug


Novartis is reportedly in discussions with patient groups to “refine” its proposed lottery system for giving away 100 doses of Zolgensma, a treatment for spinal muscular atrophy in babies, a fatal disease. At $2.1 million for a single dose, it is likely the most expensive single dose gene therapy drug in the world.


This story is disturbing on many levels, but the biggest problem lies in how the issue is framed. When large biopharma companies hire bio ethicists to help them create “fair” processes for access to investigational drugs, and even approved drugs, the resulting recommendations are often based on protocols developed for organ donation programs, where there is a supply and demand problem. These are situations where the actual scarcity involved demands a system that takes that into account. However, in the case of a drug, the only scarcity is money. Now money is a real resource. Ask any CEO. And it is often a scarce resource. In the U.S., however, we never talk about it that way. We’re not allowed to talk about it that way. Instead, we throw the whole issue of expensive drug access into an organ donor-like ethical realm. The resulting “bioethics” discussion precludes us from tackling the real problem, the real scarcity: money. This is not the case in other parts of the world, including Europe, where innovative financial solutions are being modeled and implemented to address these critical needs.


Ironically, the field of ultra-expensive drugs for rare diseases might just create the payment

model for all “expensive” drugs in the future: Payments over time that are also, somehow, tied to the value created (if the benefit lasts less, the payment is less). People a lot smarter than I are working on these things, but I can’t help but feel that if we would just say this is about money, we would get to the solution a lot quicker. The Novartis supply problem is only limited by how quickly they want to spend how much, and by their desire to test the pricing market before they make that decision. I believe that should be their decision and that they will have their chance to justify the cost to payers the world over, both private and public.


This lottery, however, is a horrible way to launch life-saving medicines. The sooner we stop

pretending these decisions are like those involved in organ donations, involving some

unsolvable supply/demand shortage, and recognize that we are really talking about money, the sooner we will find answers that actually work for patients and the biopharma industry.


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