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“The second big area is the ethical question and dilemma of how to get these treatments, these life-saving treatments, to patients across the globe. But the cost of the most recently launched gene therapy in the market, in the US, at over $2 million per patient. This is a very important question because only the wealthiest countries and those within the wealthiest countries with the best insurance coverage are currently able to access these treatments. One method that has been used by one of the developers so far is a lottery for international patients or global patients in which they can enter and try to have their infant treated with the gene therapy, free of cost. However, we think that we can do better, and it involves more than financial engineering, which what has been used thus far in the US market is essentially amortizing the payments over time.
Probably the biggest question to wrestle with is, is there a single price for these therapies that all should pay? Are there differential prices as there are for different therapies for different places across the globe? Does it get adjusted somehow? Should there be a different intellectual property protection paradigm for these therapies that cure rare disorders? What should be the method for trying to ensure that patients, again, across the globe, since there’s a relatively small number of them in an often that number is in the hundreds or low thousands, that need treatment, can access that treatment. These are questions that we’re still wrestling with, and we don’t have great answers yet, but it can’t be that only the wealthiest patients in the wealthiest countries can access these new treatments.”
