As Prescribed

The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug exclusion, a once narrow exception that permitted manufacturers of rare disease drugs and biologics to be insulated from consideration in Medicare drug price negotiations. By redefining the qualifications for negotiation eligibility, the bill hopes to create opportunity for renewed innovation for orphan drug products.

The 340B Drug Pricing Program (the Program) has long been a cornerstone for healthcare providers seeking to deliver affordable care to underserved communities. However, as the regulatory and compliance landscape evolves, stakeholders—including covered entities, pharmaceutical manufacturers, and pharmacies—must navigate an increasingly complex array of legal requirements and enforcement trends.