Drug Policy
How laws, regulators and markets shape which CMT treatments get developed and approved, from orphan drug incentives to priority review vouchers. More in the glossary
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One Patient, One Drug—and a Possible Voucher
A drug made for a single patient is one of the most hopeful ideas in rare disease medicine. Drug companies hope to profit from them, if public funding and exclusive rights defray the costs of developing these drugs. People with rare diseases hope for a cure or better health outcomes. Can both interests align?