So I went looking and found that there’s a new most expensive drug treatment. The cost / benefit market analysis is cold enough to become superfluid and flow without friction:

Lenmeldy becomes world’s most expensive drug

So far, Lenmeldy has only generated $12.7m in product sales across Europe, meaning just a handful of children with the disease received the treatment.

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The efficacy of the drug was assessed using data from 37 children who received Lenmeldy in two single-arm, open-label clinical trials and in an expanded access program, with the average follow-up time being 6.76 years, making this the longest follow-up for a newly approved gene therapy. Lenmeldy was found to have significant positive outcomes in the clinical trials, where 100% of PSLI MLD patients who were treated with Lenmeldy were alive at six years of age, compared to only 58% of children in the comparator data (who did not receive the treatment). At five years of age, 71% of treated children were able to walk without assistance. Groundbreakingly, 85% of the children treated had normal language and performance IQ scores.

Lenmeldy is the first curative treatment for this disease, and will significantly reduce the burden of the disease if it is adequately accessible. Family and carers of children with MLD will be significantly unburdened as children will be able to live relatively normal lives, reducing the time and money spent on care and therapy, and reliance on the healthcare system will reduce dramatically. However, patients will have to return for life-long monitoring for haematologic malignancies, including a complete blood count (with differential) every year and integration site analysis, as warranted, for at least 15 years after treatment. Additionally, one analysis by Mohajer and colleagues found that the current average yearly cost of care for a patient with MLD is over $125,000. The high price point of the drug therefore reflects the quality-adjusted life years (QALY) gained from treatment with Lenmeldy as patients will be able to live a relatively normal life, and potentially work in the future.