Orphan drugs marketing

Most pharmaceutical companies that market their own products can benefit from marketing orphan drugs. These benefits include the following. 

Includes combination drugs, new drugs, IND application, application for approval to market, orphan drugs, bioavailability, and bioequivalence. 

The 79 biological and drug applications approved for marketing by the FDA with orphan status as of September 1992 represent broad and extensive R D efforts for rare diseases. The test of the Orphan Drug Act s effectiveness, however, is whether it has led to the approval and marketing of drugs for orphan conditions that would otherwise have been unavailable to patients. If pharmaceutical companies would have developed and marketed orphan drugs even without these subsidies, then their tme effectiveness would be nil. 

The Orphan Drug Act of 1983 was passed to encourage die development and marketing of products used to treat rare diseases. The act defines a rare disease as a condition affecting fewer than 200,000 individuals in die United States. The National Organization of Rare Disorders reports that there are more tiian 6000 rare disorders that affect approximately 25 million individuals. Examples of rare disorders include Tourette s syndrome, ovarian cancer, acquired immunodeficiency syndrome (AIDS), Huntington s disease, and certain forms of leukemia... 

Orphan Drug Act passed, enabling FDA to promote research and marketing of drugs needed for treating rare diseases. 

Passage of the first Orphan Drug Amendment to encourage development of drugs for small markets. 

In September 2007, the EMEA approved the use of trabectidin against ovarian cancer (OC) and STS. In November 2009, Yondelis received its second marketing authorization from the European Commission for its administration in combination with pegylated liposomal doxorubicin (Doxil, Caelyx) for the treatment of women with relapsed ovarian cancer presently, trabectedin (36) is under Phase II trials for the treatment of paediatric sarcomas as well as breast and prostate cancers. The European Commission and the US Food and Drug Administration (FDA) have granted orphan drug status to trabectedin for soft tissue sarcomas and... 

In order to encourage pharmaceutical companies to invest in orphan drug development, legislation provides for a number of incentives. These include application fee waiver (the extent of reduction varies with the region of the world, and in the European Union it is 50% for all fees since 2002), market exclusivity and protocol assistance. In the European Union, there is 100% reduction in the fee applicable to the provision of any scientific advice. The fund made available by the Community for fee exemptions for orphan medicinal products amounts to= 3 700000 in 2005. 

An orphan drug is one that is intended for use in rare diseases and thus for which there is not a sufficient market to justify the investment needed to demonstrate safety and effectiveness in order to obtain approval of an NDA. Por more than 20 years PDA had permitted orphan drugs to be distributed through a permanent IND, with little or no thought that it would ever progress to an approved NDA. In 1983, Congress enacted the Orphan Drug Act to provide... 

The expected private benefit depends on a number of market characteristics and public policies, including market size, intellectual property rights, price regulation, and the magnitude of prizes for innovation. Some public policies, such as the Orphan Drug Act, may affect both the costs and the expected private benefits of R D investment. 

Figure 7.5. Number of orphan drug marketing approvals, 1980-2000.

Lichtenberg, F., and J. Waldfogel. 2003. Does Misery Love Company Evidence from Pharmaceutical Markets before and after the Orphan Drug Act. National Bureau of Economic Research Working Paper 9750. 

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