EMA: aggiornamento Q&A - variazioni di tipo II

Sono stati aggiornati i quesiti n. 14 e 21 del documento Q&A per le variazioni di tipo II 

3.14. What fee do I have to pay for a type II variation?
For information on the fee applicable for type II variations, please refer to the explanatory note on fees payable to the European Medicines Agency. Such fee covers all authorised strengths, pharmaceutical forms and presentations of a given medicinal product. Reduced Type II fees may apply to certain variations, as specified in the Explanatory note on fees payable to the EMA.
For Type II variations which introduce additional presentation/pack-size(s), each additional presentation/pack-size attracts separate fees (x additional presentations x separate fees). Each presentation/pack-size should therefore be declared as a separate variation on the variation application form.
Grouped Type II variations, whether consequential or not, will each attract a separate Type II fee.
The fee will become due on the date of the notification of the administrative validation to the applicant and fees will be payable within 45 calendar days of the date of the said notification. After approximately 15 days an invoice will be sent to the applicants billing address held on the Agency’s file.
The invoice will contain details of the product and type of procedure involved, the fee amount, the financial information and the customer purchase order number associated with the procedures invoiced (if provided in the eSubmission delivery file). The Agency does not accept stand-alone notifications of purchase order numbers that are not associated with a dossier.
Guidance on how to pay an invoice can be found on our website.
For type II variations, if the variation is finally considered ‘invalid’ (i.e. an assessment process cannot be started), an administrative fee will be charged by the Agency (see also Explanatory note on fees payable to the EMA).
In case an inspection is required, please note that in addition an inspection fee will be requested (see also Pre-submission Guidance – “What is the fee for a GMP/GCP/GLP pharmacovigilance inspection?”).

References
• Fees payable to the European Medicines Agency

3.21. Do I need to confirm the maintenance of my orphan designation when applying for a type II variation? Rev. Jun 2022
If the product has been designated as orphan and the application concerns a new therapeutic indication or a modification of an existing one, in order to ensure that the Marketing Authorisation only covers indications that fulfil the orphan designation criteria foreseen in Art 3 of Regulation (EC) No 141/2000, a COMP review may be required as following:
• for a new therapeutic indication falling within a new orphan designation, i.e. an orphan designation other than the one(s) related to the already approved indication(s), the COMP will have to confirm the maintenance of the orphan designation before authorisation of the new indication. In this case, the sponsor should provide at the time of submission a maintenance report using the template provided on the EMA website. The maintenance report should be submitted via the IRIS Platform.
• for a new therapeutic indication falling within an already authorised orphan designation, the COMP will have to consider if the specific scope of the variation raises justified and serious doubts in respect to the fulfilment of the orphan designation criteria and indicate if a formal review process of the maintenance of the orphan designation is needed
To support this process, the MAH/sponsor is requested to provide at the time of submission of the variation either a justification that the variation does not raise doubts on the fulfilment of the orphan criteria or a maintenance report to justify that the orphan criteria are still met. The justification/ maintenance report should be submitted via the IRIS Platform.
Further to the COMP preliminary discussion based on the sponsor’s justification/ maintenance report, a formal review process of the maintenance of the orphan designation for the applied indication will be triggered if this raises justified and serious doubts on the maintenance of the orphan designation. In this case, if previously only a justification was submitted, the MAH/sponsor will be requested to provide a maintenance report. The procedure for assessment will follow the usual procedure, as described in Orphan Medicinal Product Designation and Maintenance SOP/H/3534.
For the purpose of defining what is a new therapeutic indication or a modification of an existing one for the COMP review for post-authorisation extensions of indications, the Guideline on the elements required to support the significant clinical benefit in comparison to existing therapies of a new therapeutic indication in order to benefit from an extended (11-year) marketing protection should be followed.
In case of doubts, the Agency encourages applicants to contact the Orphan Medicines Office in advance of a planned submission in order to clarify orphan requirements. Please submit your message via EMA-info: Send a question to the European Medicines Agency.
Further information can be found on the dedicated EMA Website on Orphan designation.

References

• Regulation (EC) No 141/2000
• Commission Notice on the application of Articles 3,5 and 7 of Regulation (EC) No 141/2000 on orphan medicinal products
• Post-orphan medicinal product designation procedures - Guidance for sponsors (EMA/62801/2015)
• Orphan Medicinal Product Designation and Maintenance SOP/H/3534