Orphan Drugs

Orphan Drugs are medicines for rare diseases which, under normal circumstances, would not be developed by pharmaceutical companies because they would be unable to recoup their costs. Globally a range of incentives have been implemented to encourage companies to develop orphan medicines. 

Example: Europe

To benefit from the incentives offered, individuals or companies have to submit an application to the EMA to request "orphan designation" for their medicine.  Once the medicine is awarded "orphan drug" status, the individual or company can then take full advantage of the incentives on offer. These incentives include:

  • Protocol assistance (scientific advice during the product development phase)
  • marketing authorisation (10 year marketing exclusivity)
  • financial incentives (fee reductions or exemptions)
  • country-specific incentives detailed in an inventory made available by the European Commission

Global Regulatory Services (GRS) can help you to determine whether or not you can apply for your medicine to have "orphan drug" status. You can then take advantage of the incentives listed above and maximise your return on investment. At the same time, you will be improving the quality of life of those patients who suffer rare diseases.

If a company is considering both the American and European markets for their Orphan Drugs, Global Regulatory Services (GRS) recommends that the Orphan Drug Designation applications should run in parallel.  This can be achieved by using the ‘common application’ for submissions to the European Medicines Agency (EMA) and the US FDA.  This reduces overall timescales and therefore, minimises financial risk and investment.

Please contact us with your specific regulatory compliance requirements.