10 Facts on Orphan Designation
- Obtaining Orphan Designation early in the development process encourages the development of medicines for rare diseases as it offers incentives for development.
- Once an orphan medicine is authorised, it will qualify for 10 years market exclusivity. The market exclusivity becomes effective once the Manufacturing Authorisation (MA) is granted. This blocks similar competitive medicines from entering the market for 10 years. However, it does not necessarily block medicines that are targeted at the same disease if they differ from the first medicine on the basis of their molecular characteristics, the way they work or the way they are used. Also, in some circumstances, even a similar medicine could be marketed if, for example, it is manufactured so that it works better than the first medicine.
- Obtaining Orphan Designation does not necessarily speed up the development time or the evaluation of the MA application.
Can The Drug Demonstrate ‘Significant Benefit’?
- For a medicine to obtain Orphan Designation, it must be demonstrated that the medicine will bring “significant benefit” to the patient when compared to existing treatments for the targeted rare disease. “Significant benefit” should not be confused with the “benefit-risk” assessment carried out for the MA application which demonstrates that benefits from a medicine are outweighed by the risk it poses.
- The use of biomarkers to identify a subset of patients for whom medicine could be used is generally not accepted for Orphan Designation.
Obtaining Designation Does Not Permit Use
- The obtaining of Orphan Designation does not in itself permit the use of the medicine. An MA will only be granted once enough evidence has been obtained to demonstrate that the medicine is effective and acceptably safe. In exceptional circumstances, a doctor in consultation with their patient may consider the use of an unauthorised medicine through a compassionate use programme or the patient may have the possibility to enroll in a clinical trial.
- Medicines are evaluated for Orphan Designation by the Committee for Orphan Medicinal Products (COMP). It is also their responsibility to determine if Orphan Designation medicines can continue to be classified as Orphan medicines at the time of granting MA. This is separate to the evaluation of the MA application which is conducted by the Committee for Medicinal Products for Human Use (CHMP). The final decision on granting of Orphan Designation and MA is the responsibility of the European Commission. COMP publishes their assessment once Orphan status is designated and the Orphan designation is entered into the Community Register of Designated Orphan Medicinal Products.
- The medical condition specified in the Orphan Designation is often broader than the “indication” approved in the MA as it will be based on evidence obtained during the development process and post-Orphan Designation.
- Assessment of Orphan medicines is based on scientific assessment of available evidence. The assessment of costs associated with Orphan medicines is the responsibility of National Health Authorities. Individual countries within the EU will decide whether they will provide an Orphan medicine within their country’s health system.
Differences Between Authorities
- The rules for granting Orphan Designation can vary between different authorities, therefore, a medicine classified as an Orphan in the EU may not necessarily be classified as an Orphan medicine in another jurisdiction. However, the EMA encourages parallel applications for Orphan Designation with regulatory authorities outside the EU. A common application form is available when applying to the EMA and FDA in parallel. The EMA also has a special arrangement in place with the Japanese health authorities. The application to the EMA is free of charge.