Legal and regulatory aspects of orphan drugs.

Segev Shani, Zohar Yahalom

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Rare (orphan) diseases are defined as diseases whose prevalence is significantly low. Many of these diseases are diagnosed at childhood by pediatricians. Rare diseases pose many obstacles for health care systems in general and patients specifically. As they are rare, they are less investigated, there is less knowledge about the disease and less professionals specializing in it. Furthermore, as for most diseases there is no specific treatment, diagnosis is not relevant. From industry perspective, as the market potential is small, there is no financial incentive to invest in developing treatments for rare diseases. All the above led patients, researchers and policymakers around the world to legislate specific laws designated to encourage and provide incentives for researchers and for the pharmaceutical industry to develop scientific and clinical knowledge as well as potential treatments for these diseases. The objective of this article is to describe the initiation and current status of public health policy concerning orphan disease and drugs.

Original languageEnglish
Pages (from-to)110-115
Number of pages6
JournalPediatric Endocrinology Reviews
Volume11 Suppl 1
StatePublished - 1 Jan 2013
Externally publishedYes

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Endocrinology, Diabetes and Metabolism


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