Adopting orphan drugs--two dozen years of treating rare diseases.
نویسنده
چکیده
445 ing or evaluating the work of so many volunteers. Some organizations conduct internal evaluations, and they tend to agree with experienced volunteers on some guidelines for effective overseas missions. It is best, they say, to send an advance team so that requirements such as lodging, an adequate electrical supply, clinic space, and surgical facilities can be anticipated. Many stress the importance of developing strong relationships with local physicians on the basis of respect for their skills, knowledge, and traditions and, whenever possible, using locally available medications. Surgeons recommend a conservative approach to choosing patients and operations to perform. Finally, says Migliori, “don’t operate the day before you leave.” An interview with Dr. Mark Migliori can be heard at www.nejm.org.
منابع مشابه
Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007–13) and prospective (2014–18) MIDAS sales data analysis
BACKGROUND Health Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons). An estimated 9 % of Canadians suffer from a rare disease. Drugs treating rare diseases (DRDs) are also known as orphan drugs. While Canada is currently developing an orphan drug framework, in the United Sta...
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BACKGROUND Manufacturers justify the high prices for orphan drugs on the basis that the associated R&D costs must be spread over few patients. The proliferation of these drugs in the last three decades, combined with high prices commonly in excess of $100,000 per patient per year are placing a substantial strain on the budgets of drug plans in many countries. Do insurers spend a growing portion...
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Rare diseases are an important public health issue and a challenge to medical care. Specific legislation to encourage research of rare diseases and development of orphan drugs has been adopted in the United States (US), the European Union (EU), and elsewhere. In recent years, much progress has been made in some parts of Asia, including Japan, South Korea, and Taiwan, with the enactment of legis...
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BACKGROUND Ten years have passed since Latvia became a Member State of the EU in 2004. As a result European regulations, including those related to rare diseases and orphan drugs, have been applied to Latvian legislative system. Orphan diseases have been recognized as a priority area for action in the public health system, though there are significant differences in the national healthcare serv...
متن کاملLucrative niches: how drugs for rare diseases became lifeline for companies.
When an experimental medicine helping her son was dropped because it didn’t seem profitable, Connecticut homemaker Abbey Meyers turned into a lobbyist. Her crusade: Change the law to create incentives to develop drugs for rare diseases. Congress responded with the Orphan Drug Act of 1983, giving companies a seven-year monopoly for bringing a new treatment for a rare disease to market. Within tw...
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عنوان ژورنال:
- The New England journal of medicine
دوره 354 5 شماره
صفحات -
تاریخ انتشار 2006