Health Care Experts Recommend Accelerated Path to Market for Repurposed Drugs Used to Treat Rare Diseases

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New Kauffman Foundation paper highlights solutions to speed patients’ access to drugs with therapeutic value for more than one disease.

Our goal was to find solutions for repurposing existing or abandoned drugs that can be used for both rare diseases and other conditions where they may be of benefit.

For patients with rare medical conditions, the typical 12 to 15 years it takes to bring a new drug to market may be too long to wait.

Repurposed drugs – those already approved for a specific condition that can be used to treat another – offer a solution: Because their safety profile is already established, they can be brought to market more quickly. However, pharmaceutical and biotech firms have not aggressively pursued repurposing because of barriers to exclusivity in the current regulatory process.

A group of 15 health care industry, government and education stakeholders participated in the Ewing Marion Kauffman Foundation’s “Design Day” panel to brainstorm solutions to facilitate a faster path to market for these critical drugs. Their recommendations are presented in a new paper released today.

“Lack of a clear exclusivity path is a major challenge for most repurposed drugs, and makes it difficult for innovator firms and investors to fund the drug development activities required for market approval,” said Dominique Pahud, Kauffman Foundation director of Research and Policy. “Our goal was to find solutions for repurposing existing or abandoned drugs that can be used for both rare diseases and other conditions where they may be of benefit.”

The group’s proposed solution: to enable diagnosis-based differential pricing and reimbursement, allowing premium pricing for the drug when used for its new indication for a market exclusivity period of seven years, while maintaining generic pricing for its old (original) use. Key components of this recommendation include:

  •     Market exclusivity – repurposed drugs may benefit from seven years of market exclusivity under the Orphan Drug Act, and achieve regulatory approval through the “orphan drug development” paths created by the FDA.
  •     Market segmentation – the manufacturer or sponsor of a repurposed drug would be allowed to differentially price by treatment indication.
  •     Pricing flexibility – drug manufacturers or sponsors could opt in to a Payer-Sponsor Rare Disease Program, in which they cap R&D investment in the repurposed drug, in exchange for payers keeping out-of-pocket costs the same, regardless of diagnosis.
  •     Distribution – prescriptions for the repurposed drug would go through a prior authorization process to confirm diagnosis, enabling differential reimbursement.

The Kauffman Design Day participants also discussed the potential creation of a new agency, run jointly by the FDA and the Centers for Medicare & Medicaid Services (CMS), which would preapprove clinical development plans for repurposed drugs and be able to determine market exclusivity and pricing flexibility on a case-by-case basis.

“These proposed solutions would help us determine the right balance of incentives and efficiency to drive development of new clinical data to support new uses,” Pahud said. “There are enormous potential benefits in treating a variety of diseases, and in supporting health care innovation.”

About the Kauffman Foundation
The Ewing Marion Kauffman Foundation is a private, nonpartisan foundation that aims to foster economic independence by advancing educational achievement and entrepreneurial success. Founded by late entrepreneur and philanthropist Ewing Marion Kauffman, the Foundation is based in Kansas City, Mo., and has approximately $2 billion in assets. For more information, visit, and follow the Foundation on and

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Lacey Graverson
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