BOX 4-1

Examples of Business Models for Funding the Development of Drugs to Treat Rare and Neglected Diseases

INSTITUTE FOR ONEWORLD HEALTH (IOWH)


Business Model

Not-for-profit pharmaceutical company


Structure

A small team of pharmaceutical company experts, founded in 2000 with seed funding from the Bill and Melinda Gates Foundation.


Approach

Identify promising drug candidates, complete clinical trials, secure local manufacturing and regulatory approval in countries in which target diseases are endemic, and form partnerships to ensure drug distribution. IOWH strives to be opportunistic and flexible, engage industry partners, bridge industry and the public sector, focus on development, not duplicate available global resources, and ensure local government support.


Strategies

  • Find new approaches to old diseases.

  • Focus on high-risk, high-reward projects.

  • Start with parasitic diseases (for which there are no vaccines).

  • Seek to find new uses for older, off-patent drugs.

Portfolio

  • Public health tools—for disease elimination programs (e.g., paromomycin, a cure for visceral leishmaniasis)

  • Consumer products—the mother as the decision maker and purchaser (e.g., antidiarrheal medication)

  • Prescription drugs—accessed through the formal health care system (e.g., Chagas drug)

  • Active pharmaceutical ingredients—supplier to industry (e.g., biosynthetic artemisinin analogues)

CYSTIC FIBROSIS FOUNDATION THERAPEUTICS, INC. (CFFT)


Business Model

“Venture philanthropy”


Structure

Wholly owned, nonprofit drug discovery and development subsidiary of the Cystic Fibrosis Foundation with eight staff members.



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