We're unable to sign you in at this time. Please try again in a few minutes.
We were able to sign you in, but your subscription(s) could not be found. Please try again in a few minutes.
There may be a problem with your account. Please contact the AMA Service Center to resolve this issue.
Contact the AMA Service Center:
Telephone: 1 (800) 262-2350 or 1 (312) 670-7827  *   Email: subscriptions@jamanetwork.com
Error Message ......
Viewpoint |

Pricing for Orphan Drugs Will the Market Bear What Society Cannot?

Brian P. O’Sullivan, MD1; David M. Orenstein, MD, MA2; Carlos E. Milla, MD3
[+] Author Affiliations
1University of Massachusetts Medical School, Worcester
2University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania
3Stanford University School of Medicine, Palo Alto, California
JAMA. 2013;310(13):1343-1344. doi:10.1001/jama.2013.278129.
Text Size: A A A
Published online


Cystic fibrosis (CF) is an inherited condition that affects approximately 30 000 individuals in the United States.1 Rare genetic diseases such as CF are increasingly seen as unique niche markets for pharmaceutical companies. An unsustainable price structure for novel drugs developed by pharmaceutical companies for this rare condition and others is developing. In January 2012, the US Food and Drug Administration approved Vertex Pharmaceuticals’ drug ivacaftor (Kalydeco) for use in a subpopulation of patients with CF who carry a specific genetic mutation, G551D. Ivacaftor is the first drug that treats the underlying molecular defect in CF, and its introduction illustrates an application of personalized medicine. The development program that led to the discovery of this drug was firmly grounded and incrementally built on the many contributions made during at least 3 decades by a highly supportive community built by and around patients and their families affected by this disease. The work of the CF community has been used as an example of how to advance discovery toward a cure for rare conditions in partnership with industry.2

Sign in

Purchase Options

• Buy this article
• Subscribe to the journal
• Rent this article ?

First Page Preview

View Large
First page PDF preview




Also Meets CME requirements for:
Browse CME for all U.S. States
Accreditation Information
The American Medical Association is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. The AMA designates this journal-based CME activity for a maximum of 1 AMA PRA Category 1 CreditTM per course. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Physicians who complete the CME course and score at least 80% correct on the quiz are eligible for AMA PRA Category 1 CreditTM.
Note: You must get at least of the answers correct to pass this quiz.
Please click the checkbox indicating that you have read the full article in order to submit your answers.
Your answers have been saved for later.
You have not filled in all the answers to complete this quiz
The following questions were not answered:
Sorry, you have unsuccessfully completed this CME quiz with a score of
The following questions were not answered correctly:
Commitment to Change (optional):
Indicate what change(s) you will implement in your practice, if any, based on this CME course.
Your quiz results:
The filled radio buttons indicate your responses. The preferred responses are highlighted
For CME Course: A Proposed Model for Initial Assessment and Management of Acute Heart Failure Syndromes
Indicate what changes(s) you will implement in your practice, if any, based on this CME course.


Some tools below are only available to our subscribers or users with an online account.

30 Citations

Sign in

Purchase Options

• Buy this article
• Subscribe to the journal
• Rent this article ?

Related Content

Customize your page view by dragging & repositioning the boxes below.

Articles Related By Topic
Related Collections
PubMed Articles
PhRMA sues on 340B orphans. Mod Healthc 2013;43(40):17.