(Re)Defining Value in Rare Disease Through Clinical and Commercial Insight

With unprecedented candor, health insurers are questioning how drugs for rare diseases are defined, regulated and priced in the U.S. market. Their concerns, echoed in the media, the halls of government and other public forums, have profound implications for biopharmaceutical companies developing and marketing new medicines.

Yet, the issues payers raise are not intractable. In the pages that follow, we summarize the concerns and show how innovations in drug discovery, real world evidence, value-based contracts, and other business processes can address some of the most divisive issues.

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