Encouraging new drug development: laws, incentives and advocacy
Orphan drug legislation, aimed at providing incentives for pharmaceutical companies to develop and market drugs to treat rare diseases, is a relatively recent phenomenon.
A groundbreaking precedent was set by the United States in 1983 with its Orphan Drug Act. In the 1990s, first Japan, then Australia adopted orphan drug legislation. The revolution reached Europe in 1999, with the adoption by the European Parliament of the Regulation on Orphan Medicinal Products. Eurordis represented the rare disease patient association community in Europe in lobbying for the adoption of this legislation. Following the adoption of the law, a committee named COMP (the Committee for Orphan Medicinal Products) was created to review applications for drugs that wished to qualify for the label "orphan" in Europe. Eurordis has held two of the three seats assigned to patient organisation representatives on this committee since its creation in 2000.
Complete information on the Regulation on Orphan Medicinal Products (Regulation EC n° 141/2000), adopted by the European Parliament on December 16th 1999 and published on January 2000, and on Europe's orphan drug strategy is available on the European Commission's DG Public Health website.
Orphan drugs in Europe: Incentives and regulation
The purpose of EU orphan drug regulation is spelled out in article 1:
"...to lay down a Community procedure for the designation of medicinal products as orphan medicinal products and to provide incentives for the research, development and placing on the market of designated orphan medicinal products."
The Incentives
Among the most important incentives provided for by the regulation are:
Exclusivity
One of the main incentives included in the legislation for the development of orphan drugs is market exclusivity for a period of 10 years after the grant of marketing authorisation. During this period directly competitive similar products cannot usually be placed on the market. This exclusivity is a substantial competitive advantage, which mainstream pharmaceutical products do not enjoy.
Lower Fees
Fees are reduced for all the steps of the market authorization centralized procedure at the EMEA (European Medicines Agency).
A full listing of incentives for developing and marketing orphan drugs is available on the EMEA site.
Regulation
In order to be eligible for these economic advantages, a drug must meet certain criteria that then allow it to carry the label "orphan." This process is called "designation," and it is the role of the COMP (Committee for Orphan Medicinal Products) to decide whether or not a drug is eligible to be called "orphan."
Author: Fabrizia Bignami
Editor: Julia Fitzgerald
