Wednesday, June 25, 2014

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 trials.

- Public Law 107-280, the Rare Diseases Act of 2002 directed the National Institutes of Health (NIH) to establish an Office of Rare Diseases and, through this office, to support regional centers of excellence or clinical research into the rare diseases [4]. The Act also increased funding for the development of diagnostics and treatments for the rare diseases [5].

- Public Law 108-155, the Pediatric Research Equity Act of 2003 is a somewhat ambivalent law that requires applications for new drugs to test for safety and effectiveness in relevant pediatric populations, while providing full and partial waivers from the law when such testing is considered impractical.

- Public Law 110-233, the Genetic Information Nondiscrimination Act of 2008 makes it illegal to discriminate against employees or applicants for employment based on their personal genetic information (i.e., whether individuals or family members have a genetic disease or condition, or whether individuals are at risk of developing a disease or condition based on genetic testing).

- Public Law 111-80, the Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act of 2010 authorized the FDA to appoint a review group to recommend design improvements for preclinical and clinical trials aimed at preventing, diagnosing, and treating rare diseases [3].

- Public Law 111-148, the Patient Protection and Affordable Care Act of 2010, known widely as ObamaCare requires insurance companies to cover all applicants regardless of pre-existing conditions. As generally interpreted, the Act will eliminate lifetime caps on benefits.

I urge you to read more about this book. There's a good preview of the book at the Google Books site. If you like the book, please request your librarian to purchase a copy of this book for your library or reading room.

- Jules J. Berman, Ph.D., M.D.

tags: rare disease legislation, rare disease laws, u.s. support for rare diseases, rare disease research, rare diseases, orphan diseases, orphan drugs, fda, drug development