A Science Editorial on the Cost of Gene and Cell Therapies.
As it happens, I am working on a few of gene therapy development projects professionally; the instruments to conduct my part are very, very, very expensive, take significant training and time to use, and frankly, I like to get paid for what I do.
A Science editorial discusses the issue of the cost of these therapies, which save lives and reduce overall medical costs, but in our system of randomized payers, the burden of cost is not uniformly distributed. For example, an insurance company may not be willing out to shell out a huge sum for a ten year old girl who will survive to retirement age with treatment, rather than die in her teens, particularly as she is likely to be insured by someone else after cure.
Many of these therapies are designed to be "one and done;" a single dose may result in a cure. This means all of the development costs need to be captured in that dose. While this is obviously medically desirable, it is undesirable from the standpoint of recovering the costs of development by spreading them over many doses.
I don't have an answer to this problem, but I note the editorial, which I welcome:
Cell and gene therapy accessibility
Rayne H. Rouce and Matthew H. Porteus Science 1 Aug 2024 Vol 385, Issue 6708 p. 475
Excerpts:
In CGT research and development, newer, more complex techniques are more likely to be funded, published, and confer prestige than discoveries that simplify older methods. Accessibility is hardly considered in such “discovery” science. Process engineering, which aims to improve productivity in biopharmaceutical manufacturing, is ripe for rethinking and could be creatively focused on lowering patient cost. As well, clinical care innovation can be a game changer, particularly in low- and middle-income countries. For example, India has decreased the cost of hematopoietic stem cell transplantation by 30- to 50-fold compared to the US while maintaining excellent clinical results.
CGTs are fundamentally custom-made therapies, with small batches or even single doses manufactured, which is why they are inherently expensive. Further, supply chain costs can account for up to 50% of total manufacturing costs. The supply chain and manufacturing for CGTs have not had their “assembly line” moment and thus lack the “economies of scale” required to make goods available to global populations...
...Companies prioritizing shareholder value have been successful at developing safe and effective drugs, but complementary approaches are needed. Many CGTs that will affect a small number of patients are halted in their clinical development or even removed from market owing to limited commercial viability. Complementary approaches in which various institutions—not-for-profit foundations, privately held corporations, governmentsupported entities, or benefit corporations—take on clinical development and commercial distribution for CGTs with low revenue streams are being considered. The next step is to develop and test these other business models.
Only a subset of patients who could benefit from CGTs receive them because their complexity limits the number of institutions that can safely provide them, thus creating geographical barriers to access. Also, high prices place the patient and treating institution at financial risk, which means that in most cases, treatment does not proceed without prior payor authorization...
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