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  • May 2021
  • Case
  • HBS Case Collection

The SMA Foundation: Steering Therapeutic Research and Development in a Rare Disease

By: Amitabh Chandra, Spencer Lee-Rey and Caroline Marra
  • Format:Print
  • | Language:English
  • | Pages:19
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Abstract

This case explores incentives for rare disease drug development by chronicling the role of the Spinal Muscular Atrophy (SMA) Foundation in forming strategic partnerships with the scientific research community and pharmaceutical developers to transform the trajectory of innovation for the disease. In the 17 years since its inception, the Spinal Muscular Atrophy (SMA) Foundation has helped transform SMA from an underfunded and poorly researched disease with no available treatments to one with three novel drugs approved by the Food and Drug Administration (FDA). While these therapies greatly improved the quality of life for many newly diagnosed patients, there was still a substantial portion of existing patients with unmet medical needs. Though many in the medical community, and even at the FDA, felt that SMA had been all but cured, the Foundation could not accept that their work was done until every patient had a treatment option.

Keywords

Innovation and Invention; Strategy; Business or Company Management; Society; Health; Public Administration Industry; Health Industry; United States

Citation

Chandra, Amitabh, Spencer Lee-Rey, and Caroline Marra. "The SMA Foundation: Steering Therapeutic Research and Development in a Rare Disease." Harvard Business School Case 621-112, May 2021.
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About The Author

Amitabh Chandra

Technology and Operations Management
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More from the Authors
  • Vertex and the Cure for Type 1 Diabetes By: Amitabh Chandra, William J Anderson and Silvia Mare
  • Achieving Universal Health Insurance Coverage in the United States: Addressing Market Failures or Providing a Social Floor? By: Katherine Baicker, Amitabh Chandra and Mark Shepard
  • Regulatory Incentives for Innovation: The FDA's Breakthrough Therapy Designation By: Amitabh Chandra, Jennifer Kao, Kathleen Miller and Ariel D. Stern
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