Module 1 2021

30/06/2021

Similarity vs MAA vs Orphan designation

OD



Comply to EC 141/2000 Art 3*?

MAA





No OD

Similar EC 847/2000 Art 3

MAA



Derogation Consent EC 141/2000 Art 8(3)a



MAA



Derogation Insufficiënt supply EC 141/2000 Art 8(3)a





Derogation Clinical Superior EC 141/2000 Art 8(3)a

MAA



No MAA



• Similar = identical active substance, or an active substance with the same principal molecular structural features (but not necessarily all of the same molecular structural features) and which acts via the same mechanism (EC 847/2000 Art 3.3(c), amended by EC 2018/781). • OD = orphan designation • Clinical superior: e.g. Greater efficacy, less frequent and/or less severe adverse effects, more favourable PK properties, Compliance-promoting features, Fewer interactions with food or other drugs • MAA = marketing authorization application, a marketing authorization will only be granted provided quality, safety & efficacy data are positive • *criteria for orphan status – incl significant benefit!

The Organisation for Professionals in Regulatory Affairs

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Demonstrate significant benefit

Criteria for OD: No other method authorised in EC for this condition OR significant benefit over existing satisfactory methods Significant benefit defined as: new treatment would generally be of “significant benefit” if it provides a clinically relevant advantage or a major contribution to patient care:

• improved efficacy • improved safety • major contribution to patient care

At time of MAA, demonstration of significant benefit by data and critical review by the applicant

COMP to evaluate probability for the patients to experience a clinically relevant benefit

If no evidence for significant benefit at the time of MA, the product will lose its OD status

The Organisation for Professionals in Regulatory Affairs

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