rare diseases - Publications - Faculty & Research

January 2024
Article

Cost of Exempting Sole Orphan Drugs from Medicare Negotiation

By: Matthew Vogel, Olivia Zhao, William B. Feldman, Amitabh Chandra, Aaron S. Kesselheim and Benjamin N. Rome

Importance: The Inflation Reduction Act (IRA) requires Medicare to negotiate prices for some high-spending drugs but exempts drugs approved solely for the treatment of a single rare disease.
Objective: To estimate Medicare spending and global... View Details

Keywords: Health Care and Treatment; Insurance; Price; Health Industry

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June 2023
Case

Verve Therapeutics: Taking DNA Editing to Heart

By: Shikhar Ghosh and Shweta Bagai

Verve Therapeutics, a public biotech company based in Boston, created a novel approach to addressing cardiovascular disease (CVD) - a leading cause of deaths globally. The company's approach was a single shot treatment to permanently lower cholesterol, thus reducing... View Details

Keywords: AI; Genetic Engineering; Medicine; Health Care and Treatment; Genetics; Innovation Strategy; Business and Stakeholder Relations; Medical Specialties; Innovation and Invention; Entrepreneurship; Biotechnology Industry

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May 2021
Case

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

By: Amitabh Chandra, Spencer Lee-Rey and Caroline Marra

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... View Details

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

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2018
Chapter

The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century

By: Nicholas Bagley, Benjamin Berger, Amitabh Chandra, Craig Garthwaite and Ariel Dora Stern

On the 35th anniversary of the adoption of the Orphan Drug Act (ODA), we describe the enormous changes in the markets for therapies for rare diseases that have emerged over recent decades. The most prominent example is the fact that the profit-maximizing price of new... View Details

Keywords: Health Care and Treatment; Laws and Statutes; Research and Development; Investment; Markets; Monopoly

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January 2018 (Revised January 2019)
Case

ZappRx

By: Jeffrey J. Bussgang and Olivia Hull

In October 2015, ZappRx founder Zoe Barry is deciding between two business models for her health technology start-up. Her product, a software application that aims to expedite the prescription fulfillment process for patients with rare diseases, has attracted interest... View Details

Keywords: Speciality Drugs; Hub Services; Pivot; Speciality Prescriptions; Health Care and Treatment; Customization and Personalization; Internet and the Web; Business Model; Decision Choices and Conditions; Pharmaceutical Industry; Massachusetts

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September 2002 (Revised August 2003)
Case

Genzyme's Gaucher Initiative: Global Risk and Responsibility

By: Christopher A. Bartlett and Andrew N. McLean

In Egypt, Genzyme's humanitarian commitment to treat all sufferers of the rare Gaucher disease worldwide first confronts its commercial imperative to recoup the huge investment required to bring the drug Cerezyme to market. Here Tomye Tierney must decide how to balance... View Details