The MHRA will offer incentives in the form of market exclusivity and full or partial refunds for marketing authorisation fees to encourage the development of medicines in rare diseases.
Questions concerning the fulfilment of the orphan designation criteria will be raised with the company. If the MHRA concludes that the criteria for orphan designation are not met, there will be an opportunity to appeal the decision to the CHM. The applicant should inform the MHRA of the intention to appeal as soon as possible.
For non-orphan indications, a new marketing authorisation application is required. There are no additional fees to apply for orphan designation. Information on fee waivers and refunds for orphan medicines is provided in the amended MHRA application fees.