Treatments for rare diseases have generally been able to avoid the most aggressive payer management strategies, like step therapy. Chalk up this immunity to limited treatment options for these diseases, relatively few patients needing treatment for them, and vocal advocacy communities. So when pharmacy benefit managers introduced exclusion lists earlier this decade, it wasn’t much of a surprise that rare diseases were virtually absent from them.
That immunity is being challenged. In 2017, CVS Caremark began excluding drugs in the chronic myelogenous leukemia category. Patients who want to use excluded drugs may be required to pay full cost unless an exception is allowed, which often requires the patient to try a preferred drug — and fail on it — first.