Spring Intro 2023

03/03/2023

When should I use the Centralised Procedure?

Because you want to  Optional Scope (Article 3(2) of Regulation (EC) No 726/2004): 1. Medicinal Product with a NAS not authorised in EU

Because you have to  Mandatory Scope (Article 3(1) of Regulation (EC) No 726/2004): 1. Medicines derived from certain biotechnology processes, such as recombinant DNA technology • Incl Biosimilar products developed using the specified biotechnological processes 1.1 Advanced therapy medicinal products such as gene-therapy and somatic cell-therapy 2. New active substances for AIDS, cancer, neurogenerative disorder, diabetes, auto-immune diseases and other auto-immune dysfunctions and viral diseases

2. Significant therapeutic, scientific or technical innovation (e.g. new alternative to patients, utilises new technology, etc)

‘ Automatic Access’: • Generic/Hybrid of reference medicinal product authorised via CP (Article 3(3)) • Duplicate or informed consent applications • Generics of products approved by CP Paediatrics: • Paediatric indication in compliance with agreed PIP • Paediatric Use Marketing Authorisations (PUMA)

3. Orphans medicines (medicines for rare diseases)

Regardless of whether the product falls into the mandatory or optional scope, an 'eligibility request’ must be submitted ahead of filing the Marketing Authorisation Application to EMA using the specific form and accompanied by a justification of eligibility

EMA link: Definitions for mandatory scope of Centralised Procedure

The Organisation for Professionals in Regulatory Affairs

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Research and Development Phase

Orphan Drug Designation for rare diseases • Eligibility criteria are: – Treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating – The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development – No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition . • Applications are made to the EMA’s Committee for Orphan Medicinal Products (COMP) and take 90 days from validation (runs to a fixed timetable). • Generally early clinical data are needed, though can try on basis of compelling non-clinical data • Fee reduction is among benefits of orphan drug designation if designation is in place at the time of the MAA submission

Access

Submission and validation of MAA

Research and development (several years before MAA)

Pre submission (~18 months before MAA)

EU Commission decision

Evaluation of MAA (~ 1 year)

EMA opinion

Continuous Safety Monitoring

EMA link: Overview of Orphan designation

The Organisation for Professionals in Regulatory Affairs

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