Gene therapy pushes the US $1 million price barrier (2024)

Chiesi and uniQure have proposed a price tag of roughly US$1.1 million for their alipogene tiparvovec gene therapy.

The lowdown: Gene therapies pose a pricing dilemma for industry. If drugs are only administered once and, in many cases, to small patient populations, how can drug companies recoup development costs and turn a profit? Pricing negotiations for uniQure and Chiesi's alipogene tiparvovec — which is approved in Europe for lipoprotein lipase (LPL) deficiency — provide a first insight into how drug developers and payers are thinking about the problem.

The firms have opened the negotiations for their therapy — an adeno-associated virus (AAV) vector that carries the LPL gene — at around US$1.1 million per dose. The companies reportedly considered pursuing an annuity-pricing model, under which payers would make small upfront payments followed by annual payments for a fixed amount of time or for as long as the treatment continued to provide benefit. But while the firms have shown that their treatment reduces the incidence of pancreatitis in LPL-deficient patients, patients have to be hospitalized for up to 3 days to assess a battery of biomarkers to confirm benefit. Confounding factors such as diabetes and diet also complicate the analysis. Other gene therapies, with simpler disease outcomes, may be better candidates for annuity-pricing models.

A single dose of the gene therapy can provide benefit for at least 6 years, says the company. This brings its annualized cost down to $183,000, which is cheaper than the annual price of many enzyme-replacement therapies that are needed for the lifetime of patients with rare diseases. There are thought to be 150–200 patients in Europe who would be eligible for the gene therapy.

The negotiations are ongoing.