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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

    • December 2021
    • Faculty Research

    Value-Based Insurance Design at Onex

    By: Joshua Schwartzstein, Amitabh Chandra and Amram Migdal
    • New England Journal of Medicine

    Supporting Value-Based Health Care—Aligning Financial and Legal Accountability

    By: Mark M. Zaki, Anupam B. Jena and Amitabh Chandra
    • June 2021
    • Faculty Research

    Who Lives & Who Dies: Expanded Access for Experimental Drugs at Chimerix (B)

    By: Amitabh Chandra and Spencer Lee-Rey
More from the Authors
  • Value-Based Insurance Design at Onex By: Joshua Schwartzstein, Amitabh Chandra and Amram Migdal
  • Supporting Value-Based Health Care—Aligning Financial and Legal Accountability By: Mark M. Zaki, Anupam B. Jena and Amitabh Chandra
  • Who Lives & Who Dies: Expanded Access for Experimental Drugs at Chimerix (B) By: Amitabh Chandra and Spencer Lee-Rey
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