What is orphan drug status in Europe?
The European Commission decides whether to grant an orphan designation for the medicine based on the COMP’s opinion. An orphan designation allows a pharmaceutical company to benefit from incentives from the EU, such as reduced fees and protection from competition once the medicine is placed on the market.
What did the orphan drug law allow for?
ch. 9 § 301 et seq. The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington’s disease, myoclonus, ALS, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States.
What is required for orphan drug designation?
Criteria for Orphan Drug Designation The product must be intended for use in a rare disease or condition. Adequate documentation or prevalence data must demonstrate that the intended condition is rare.
How long is orphan drug exclusivity?
The exclusivity granted to orphan drugs provides seven years without generic competition for the approved orphan designation but does not prevent generic competition for other approved uses of the medicine. Orphan exclusivity continues longer than patent protection in only 60 of the 503 orphan-designated medicines.
How many orphan drugs have been approved EU?
In the EU, 1264 Orphan Drug Designations have been granted and 133 medicinal products were approved covering a total of 179 indications and 122 rare conditions.
When was the Orphan Drug Act approved?
Congress passed the Orphan Drug Act of 1983 to stimulate the development of drugs for rare diseases. A rare disease is defined as a disease that affects fewer than 200,000 people in the United States.
Why has the Orphan Drug Act been successful?
Many researchers and policy makers have credited the Orphan Drug Act—in particular, its guarantee of seven years of market exclusivity—with this success, citing the surge in drugs for rare diseases as evidence that legislative grants of market exclusivity work to generate pharmaceutical innovation.
Are orphan drugs profitable?
Once approved and marketed, several companies have shown that profits can be made on orphan drugs and patients can be served, despite small numbers of potentially treatable patients. Gross profit margins of over 80% are reported in the rare disease industry, whereas the pharmaceutical industry average is 16%.
What happens after orphan drug designation?
Under the Orphan Drug Act, drug companies can apply for Orphan Drug Designation (ODD), and if granted, the drug will have a status which gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug.
What is the EU Orphan Medicinal Products Regulation?
The Regulation: lays down the EU procedure for designation of orphan medicines; defines incentives for the development and placing onto the market of designated orphan medicines; establishes the Committee for Orphan Medicinal Products (COMP).
When did the orphan regulation come into effect?
On 16 December 1999, the European Parliament adopted Regulation (EC) No 141/2000 (the Orphan Regulation). This was published in the Official Journal of the European Communities on 22 January 2000.
What makes a drug an orphan drug?
First of all, a product can gain orphan drug status if it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10,000 people in the European Community (EC) when the application is made.
What is the European Medicines Agency?
The European Medicines Agency (EMA) plays a central role in facilitating the development and authorisation of medicines for rare diseases, which are termed ‘ orphan medicines ‘ in the medical world. Sponsors of designated orphan medicines can benefit from a number of incentives in the EU.