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- All HBS Web
(66)
- News (30)
- Research (19)
- Multimedia (1)
- Faculty Publications (8)
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- March 2014
- Editorial
Limits on Use of Health Economic Assessments for Rare Diseases
By: Hanna I. Hyry, Ariel Dora Stern, Jonathan CP Roos and Timothy M. Cox
Funding of expensive treatments for rare ('orphan') diseases is contentious. These agents fare poorly on 'efficiency' or health economic measures, such as the QALY, because of high cost and frequently poor gains in quality of life and survival. We show that... View Details
Hyry, Hanna I., Ariel Dora Stern, Jonathan CP Roos, and Timothy M. Cox. "Limits on Use of Health Economic Assessments for Rare Diseases." hcu016. QJM: An International Journal of Medicine 107, no. 3 (March 2014): 241–245.
- 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
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.
- 02 Jun 2021
- Research & Ideas
A Rare Find in Health Care: A Simple Solution to Racial Inequity
are within our grasp." “Although we don’t have as much data on the quality of COVID care, I’m quite sure there are much greater quality differences across hospitals in treating COVID than heart attacks,” he says. “In Boston, the leading academic medical centers, with a... View Details
- 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
Objective: To estimate Medicare spending and global... View Details
Vogel, Matthew, Olivia Zhao, William B. Feldman, Amitabh Chandra, Aaron S. Kesselheim, and Benjamin N. Rome. "Cost of Exempting Sole Orphan Drugs from Medicare Negotiation." JAMA Internal Medicine 184, no. 1 (January 2024): 63–69.
- 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
Ghosh, Shikhar, and Shweta Bagai. "Verve Therapeutics: Taking DNA Editing to Heart." Harvard Business School Case 823-113, June 2023.
- 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
Keywords: Moral Sensibility; Investment; Emerging Markets; Negotiation; Corporate Social Responsibility and Impact; Business and Government Relations; Sales; Commercialization; Expansion; Value Creation
Bartlett, Christopher A., and Andrew N. McLean. "Genzyme's Gaucher Initiative: Global Risk and Responsibility." Harvard Business School Case 303-048, September 2002. (Revised August 2003.)
- 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
- 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
Bagley, Nicholas, Benjamin Berger, Amitabh Chandra, Craig Garthwaite, and Ariel Dora Stern. "The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century." Chap. 4 in Innovation Policy and the Economy, Volume 19, edited by Josh Lerner and Scott Stern, 97–137. University of Chicago Press, 2018.
- 09 Mar 2009
- Research & Ideas
How to Revive Health-Care Innovation
effective solution. In the past, business model innovation was common in health care. When the technological enablers for the diagnosis and treatment of infectious diseases emerged, most patient care was transferred away from hospitals to... View Details
- 24 Apr 2018
- First Look
First Look at New Research and Ideas, April 24, 2018
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... View Details
Keywords: Sean Silverthorne
- 22 Aug 2022
- Research & Ideas
Can Amazon Remake Health Care?
we desperately need more meaningful innovation for a whole host of diseases—diabetes, cardiovascular disease, Parkinson’s, Alzheimer’s, and ALS. We want cures, not chronic disease management. How does Amazon’s foray into health care help... View Details
- 30 Jul 2007
- Research & Ideas
Repugnant Markets and How They Get That Way
was get some of the gains from exchange without exciting the repugnance that forbids markets. One could imagine that a law about kidney sales might come about if the courts decided that it was unreasonable for someone who was dying of kidney View Details
Keywords: by Martha Lagace
- 15 Dec 2003
- Research & Ideas
The New Global Business Manager
Massachusetts. Their Cerezyme plant runs twenty-four hours a day, 365 days a year, and produces six kilos of product in all that time. You could fit it in a little six-pack bag. But it is sufficient to treat the 6,000 people around the world—and that's all there... View Details
Keywords: by Cynthia Churchwell
- 17 Dec 2007
- Research & Ideas
The Rise of Medical Tourism
What used to be rare is now commonplace: traveling abroad to receive medical treatment, and to a developing country at that. So-called medical tourism is on the rise for everything from cardiac care to plastic surgery to hip and knee... View Details
- 23 Nov 2009
- Research & Ideas
Management’s Role in Reforming Health Care
experiments are underway with professionals trying different ways of configuring and managing services. On that list I include experiments with disease management programs, substituting nurse practitioners for physicians in certain... View Details
- 07 Jan 2008
- Research & Ideas
Pursuing a Deadly Opportunity
entrepreneurial venture. The paper, which offers a rare glimpse into an area where little consolidated data is available, is forthcoming in the journal Social Science & Medicine. "This study happens to focus on cadavers, but it... View Details
- 18 Dec 2018
- First Look
New Research and Ideas, December 18, 2018
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 orphan drugs... View Details
Keywords: Dina Gerdeman
- 28 Apr 2009
- First Look
First Look: April 28, 2009
consumer-oriented model for drug development and use has attracted attention in recent years as an alternative to the much-maligned approach of mass-marketing blockbuster drugs. In a parallel development, patients and disease-based organizations have assumed greater... View Details
Keywords: Martha Lagace
- 29 Apr 2014
- First Look
First Look: April 29
utilizes a very rare "no haggle pricing" strategy and extended sales cycle when selling pieces to collectors. Though it remains profitable and very respected, the size and scope of the gallery will be brought into question when... View Details
Keywords: Sean Silverthorne