The cost of experimenting

There are some key differences between expanded access, which allows seriously ill people to apply for access to experimental drugs that might not have been through any human trials, and Montana’s approach. In theory, a person doesn’t need to be seriously ill to access experimental drugs in Montana. “In Montana, patients may be eligible for preventive or earlier-stage interventions if they provide informed consent and meet the program’s requirements, so the breadth of potential therapies and situations is much broader,” says Kaeberlein, the Montana ETRB member, who is an affiliate professor at the University of Washington in Seattle. Kaeberlein also highlights another key difference, which is cost. Companies that make their treatments available through expanded access are only able to charge for the costs of making, transporting, and monitoring the drug, and they must justify the eventual price to the FDA.

In Montana, they can charge whatever price they want. Stanley of WinSanTor says he plans to sell his drugs “at cost. ” But Ceres’s Joudinaud says that he’d be more interested in selling his at a market price. When asked what that might be, he hinted that the prices of new drugs for rare diseases can be high. In recent years, the median price of such drugs was $218,872.

“Instead of simply creating a legal pathway for patients, it also creates a business model that companies may actually be willing to use,” says Kaeberlein. Beyond the financial cost, there will be risks associated with any experimental drug. Phase I trials don’t conclusively reveal whether a drug is safe. Around 17% of drugs are found to be inadequately safe during phase III trials. “The idea that a drug has been proven safe because it’s been subject to a phase I study is very, very wrong,” says Kesselheim.

Bioethicists have raised concerns about the ethics of promoting and selling unproven treatments and the risk of harm should something go wrong.