Incentives for orphan drug research and development in the United States

Abstract

Background: The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions. This study analyzed the characteristics of OD designations, approvals, sponsors, and evaluated the effective patent and market exclusivity life of orphan new molecular entities (NMEs) approved in the US between 1983 and 2007.

Delays in the submission of new drugs in Canada

“…3 ) These FDA programs are heavily used for orphan drugs, as reported by Seoane-Vazquez and colleagues. 4 In 2004, the European Medicines Agency created a similar program (conditional marketing authorizations) that permits submissions for new drugs that address unmet medical needs, even when comprehensive clinical data have not been provided. However, conditional authorizations were not empirically important during the period of our analysis: as Joppi and colleagues 5 Note: SD = standard deviation.…”

Registro e incorporação de tecnologias no SUS: barreiras de acesso a medicamentos para doenças da pobreza?

“…For this reason, agencies such as the FDA and EMA have developed specific regulations to encourage research and development in this area with specific emphasis on the so-called "orphan drugs", developed specifically to treat rare diseases, and medicines and formulations for pediatric use 13,14 . It therefore follows that Brazil needs to improve the regulatory framework for conditions that are not attractive to the pharmaceutical industry, be they poverty-related diseases, rare diseases or childhood diseases.…”

“…The funding of this medication and its use on the SUS in the treatment of sickle cell disease is regulated on an off-label basis 19,20 . Seoane-Vazquez et al 14 show that the incentives provided by the FDA to stimulate the development of drugs that attract little industry interest resulted in 322 new approvals between 1983 and 2007. The study cited the following successful support mechanisms: (i) grants awarded to academic-based researchers; (ii) 50% tax credit for costs incurred during the clinical testing phase; and (iii) a seven-year period of marketing exclusivity granted upon approval of a drug.…”

Fair pricing of “old” orphan drugs: considerations for Canada’s orphan drug policy

“…No additional North American manufacturer of trientine has emerged since the drug's market exclusivity expired in 1992. Generic orphan drugs are rarely made; 17 there fore, the price of many orphan drugs often remains exceptionally high.…”