The Rare Disease Drug Dilemma: Great Value, High Price, Limited Access?

Drugs to treat rare diseases were once immune to patient access management strategies due to the high unmet need, few, if any, treatment options, and small patient populations. However, as the cost of these treatments continues to spiral upwards due to the high cost of drug development, patient access is no longer a given. Pricing pressures on payers to afford and manage costs of treating rare diseases and drug companies to demonstrate the value of their products are mounting. How do we determine the “value-based price” of a drug?

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