The cost of high-end, breakthrough medications traumatizes patients and their families. According to USAfacts, “national spending on personal health care has increased 1,629% since 1980 — more than $3 trillion.” Medicaid, Medicare and private health insurance bare much of the cost, but it follows naturally that out-of-pocket spending shoots upward. These “cash” payments handle roughly 13% of total medical spending.
The most intense pain is felt from those specialty medications that cost a fortune and that work for a small body of sufferers. Among these is tafamidis, which comes in at $225,000 a year. The minuscule number of users — an estimated 120,000 adults in the U.S. — and the cost to develop these breakthroughs proves overwhelming. This medication does battle with transthyretin amyloid cardiomyopathy (ATTR-CM) and falls under the Pfizer PFE umbrella using the brand names Vyndaqel and Vyndamax.
Among the budget busters are gene therapies used to treat rare, deadly genetic disorders. This category includes the world’s most expensive drug: Lenmeldy, which runs $4.25 million a year. Manufactured by Orchard Therapeutics ORTX , this miracle drug treats a condition that affects approximately one in 40,000 to 100,000 live births in the U.S.
Not far behind in cost is Elevidys at $3.2 million annually. This treats Duchenne muscular dystrophy (DMD), a genetic disease that leads to progressive muscle weakness and which affects approximately 300,000 people worldwide.
Tafamidis, Lenmeldy and Elevidys have many siblings: Hemgenix, made by Sarepta Therapeutics SRPT , treats hemophilia B and costs $3.5 million a year. Skysona, a product of Bluebird Bio BLUE , is used for a deadly neurodegenerative disease and runs $3 million annually. Zynteglo is priced at $2.8 million a year. Also manufactured by Bluebird, it is used to treat a particular genetic blood disorder. Bluebird finds itself in a peculiar niche owning several of the most expensive medications.
The situation reveals the challenge: people need these cures. They are very expensive. Cutting down the price through legislation will cause the giant pharmaceutical companies to shy away from searching for new cures.
Solutions are few and far between. Some improvement might come from a general insurance plan to deal with the growing patient appetite for these products. If the cost of these specific life savers could be averaged out in company insurance plans, some relief would be gained by the individuals and families involved.
However, if the government were to try to cut the price, the pharmaceutical leaders would likely back off research on diseases that affect small percentages of the population.
Insurance companies also do not want their revenue or profit to shrink. In recent weeks, UnitedHealth Group UNH and Elevance ELV have seen their share prices dip.
A search of what the big companies are saying about this subject finds more references to “value” and “transparency” than to any clear plan to reduce cost. The expression “affordable access” appears in discussions but without any detail. While cost remains a critical issue, no clear solution shows up.
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