Orphan Drugs in Oncology.

Rare diseases represent a group of conditions affecting a very limited number of patients. Low profitability resulting from the small size of target population coupled with difficulties in conducting the research causes the lack of interest from the pharmaceutical industry. In order to promote research and development of medicines for rare diseases, a special 'orphan' legislation was introduced in a number of regions.

Economic impact of new active substance status on EU payers' budgets: example of dimethyl fumarate (Tecfidera(®)) for multiple sclerosis.

Background: Recently, collaboration between regulators and payers was set up and was mainly focused on evidence generation along product clinical development. However, neither the regulatory path nor the new active substance status (NASs) was considered. Granting NASs will provide the product with 8 years of data protection and 2 years of market exclusivity during which no generic could enter the market.