STAT+: Pharma companies and their allies seek to exempt orphan drugs from state pricing limits

As more states create dedicated boards to cap the costs of medicines, some drugmakers and their allies are pushing back with a controversial tactic — lobbying for legislation to set exemptions for so-called orphan drugs, which are used to combat rare diseases that afflict relatively small groups of patients.

The efforts, which are being joined by some some lawmakers, reflect concerns that patients may lose access to these medicines if pharmaceutical companies halt sales or decide not to invest in developing such drugs. But opponents argue blanket exceptions would unnecessarily extend to numerous big-selling treatments for common conditions that — thanks to regulatory endorsements — also happen to have an orphan designation.

As a result, consumer advocates complain these legislative maneuvers, which have been proposed in several states, would only increase the risk that countless patients could have trouble paying for a wide variety of medicines. They further argue that the legislation would preserve profits for drug companies at the expense of states trying to cope with budgetary strains.

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