Seminar 15, 16, 17 and 18: MA and regulatory exclusivities Flashcards
What is the centralised procedure for marketing authorisation (MA) in the EU?
It is an EU-wide system allowing medicines to be marketed in all Member States through a single authorisation granted by the European Commission based on EMA scientific assessment.
For which products is the centralised procedure mandatory under Regulation (EC) No 726/2004?
Products derived from biotechnology, orphan medicines, and medicines for AIDS, cancer, neurodegenerative disorders, or diabetes authorised after 20 May 2004.
What is the evaluation timeline for the EMA under the centralised procedure?
The standard assessment timeline is 210 days, with extensions possible for additional questions.
What are mutual recognition and decentralised procedures for marketing authorisation?
Mutual recognition applies to products already authorised in one EU country, while the decentralised procedure is for products not yet authorised in any Member State, with one country acting as the Reference Member State (RMS).
What is a full application for MA under Article 8(3) of Directive 2001/83/EC?
It requires submission of pharmaceutical, pre-clinical, and clinical data, including test results on safety, efficacy, and quality.
When is a generic application under Article 10(1) of Directive 2001/83/EC used?
It is used when bioequivalence to a reference product is demonstrated, and the reference product has been authorised for at least 8 years.
What is a hybrid application under Article 10(3) of Directive 2001/83/EC?
It is used when the product differs from the reference product in active substances, indications, strength, form, or route of administration, requiring both reference data and new clinical/pre-clinical data.
What is the purpose of conditional marketing authorisation (MA)?
It addresses unmet medical needs for life-threatening conditions by granting temporary authorisation based on incomplete data, with the obligation to provide comprehensive data later.
What are the requirements for granting an MA under exceptional circumstances?
It is granted when comprehensive safety and efficacy data cannot be obtained due to rare indications, scientific limitations, or ethical barriers.
What is the difference between conditional MA and exceptional circumstances MA?
Conditional MA requires eventual provision of complete data and can transition to standard MA, while exceptional circumstances MA applies to rare cases where complete data cannot be obtained and remains incomplete.
What is pharmacovigilance in the context of MAs?
It is the science of monitoring and preventing adverse effects or medicine-related problems, coordinated by the EMA and Member States.
What is global marketing authorisation (GMA) under Directive 2001/83/EC?
GMA unifies the initial marketing authorisation and all subsequent variations, extensions, and related authorisations under the same exclusivity period.
What qualifies as a new active substance (NAS) for regulatory purposes?
A NAS is an active substance significantly different in safety or efficacy from previously authorised substances, triggering a new GMA and data exclusivity.
What is the SPC export waiver under Regulation 2019/933?
It allows EU manufacturers to produce generics/biosimilars during the SPC term for export to non-EU markets without SPC protection and to prepare for EU market entry immediately after SPC expiry.
What is orphan designation under Regulation (EC) No 141/2000?
It applies to medicines for rare diseases affecting fewer than 5 in 10,000 people in the EU or where investment is financially unviable, with the product offering significant benefit over existing treatments or addressing an unmet medical need.
What is indication stacking in orphan drugs?
Indication stacking allows a product to receive separate market exclusivity periods for different rare disease indications, potentially extending exclusivity across multiple overlapping periods.
What incentives are provided for orphan drugs under Regulation No 141/2000?
Incentives include 10 years of market exclusivity (plus 2 years for compliance with Paediatric Investigation Plans), fee reductions, and access to the centralised procedure.
How did the Glivec case (C-138/15) confirm overlapping exclusivity for similar orphan drugs?
The Court confirmed that similar drugs with overlapping indications can each receive separate 10-year exclusivity periods, reinforcing their independent market protection.
What was the ruling in the Shire Pharmaceuticals case (T-80/16) regarding orphan drug designation?
The Court held that a product offering significant clinical benefit for a subset of patients (e.g., Hunter Syndrome cognitive symptoms) could qualify for independent orphan designation despite containing the same active substance as an earlier approved product.
What are the proposed reforms to limit orphan drug indication stacking?
The EU proposes capping orphan exclusivity extensions to two additional indications, requiring significant clinical benefits for each, and ensuring submissions occur at least two years before existing exclusivity ends.
