Regeneron’s newly approved gene therapy Otarmeni will be made available at no cost to eligible patients in the United States.
That is hopeful.
Because rare diseases often come with a difficult reality.
The patient group is small.
Development is expensive.
Manufacturing is complex.
If only a small number of patients can receive a treatment, the price often has to be very high to make development commercially viable.
That makes the traditional pharma model difficult.
In this case, Regeneron is taking a different route.
Of course, this does not happen without political, commercial and strategic context.
But still.
For patients and families dealing with this rare genetic form of hearing loss, it matters.
Sometimes innovation is not only in the therapy itself.
It can also be in the way you make the therapy available.