Excluding orphan drugs from Medicare pricing pilots could erase most savings, analysis shows

A Harvard researcher has calculated that removing rare disease treatments from two Medicare pilot programs-GLOBE and GUARD-would dramatically cut the projected cost savings.

The pilots are designed to bring U.S. drug prices in line with those of other wealthy nations through a “most‑favored‑nation” pricing model. Biotech firms are lobbying the administration to keep orphan drugs out, arguing they require special pricing considerations.

The analysis suggests that the savings from retail drugs would be largely wiped out if orphan therapies are exempted, undermining the pilots' intended impact.

The report also notes that some large drugmakers have already secured exemptions through undisclosed agreements with the White House, though the specifics remain opaque.