Rare Disease Legislation in the U.S.

In June, 2014, my book, entitled Rare Diseases and Orphan Drugs: Keys to Understanding and Treating the Common Diseases was published by Elsevier. The book builds the argument that our best chance of curing the common diseases will come from studying and curing the rare diseases. Here is a short excerpt from Chapter 14. Many countries have passed legislation ensuring that the rare diseases receive research funding, that pharmaceutical companies are encouraged to produce medications for the rare diseases, and that individuals and families receive necessary medical and emotional support. In the U.S., some of the most important political milestones have been the following: - Public Law 97-414, the Orphan Drug Act of 1983 defines rare diseases and provides sponsors of drugs intended to treat rare diseases with protection from competition (i.e., 7 years of market exclusivity), tax credits, and various other incentives. - Public Law 101-629, the Safe Medical Devices Act of 1990 provides incentives and exemptions for devices, much as prior legislation covered drugs. The Act applies to devices to treat or diagnose diseases affecting fewer than 4000 individuals [3]. - Public Law 105-115, the FDA Modernization Act of 1997 grants an exemption for orphan drugs from drug approval application fees that would otherwise apply [3]. Amendments to the Act in 2007 include the Best Pharmaceuticals for Children Act (Public Law 110-85), which encourages the recruitment of children into clinical ...
Source: Specified Life - Category: Pathologists Tags: drug development fda genetic disease orphan diseases orphan drugs rare disease laws rare disease legislation rare disease research rare diseases u.s. support for rare diseases Source Type: blogs