A rare disease drug seems to work, but the health care system might not

Cristina Brennan can hear the clock ticking.

At the end of the year, her 12-year-old son Tristan will no longer have free access to a pricey medicine that has successfully helped him combat a rare form of epilepsy. This means that after being on the treatment — made available to him through a clinical trial since early 2022 — he may once again have to cope with seizures that will worsen his health, diminish his quality of life, and place a proverbial cloud over his future.

This looming dilemma reflects a complicated scenario, but one that may seem familiar to many people in the U.S. who have a rare disease and must navigate the rising cost of medicines and hurdles in health care coverage. And confronting such a challenge amounts to an existential struggle that involves battling the priorities of pharmaceutical companies and insurers.