Module 6

04 October 2024

Analysis of SME MAA : Results

• MO associated with MAA failure for SME:

• Quality: batch to batch consistency

Efficacy: choice of endpoints

•

Safety

•

• Applications with unresolved MOs (N=22)

Efficacy: 20 (91%)

•

Safety: 11 (50%)

•

Quality: 8 (36%)

•

Non Clinical: 1 (5%)

•

An Agency perspective: the success and failure of a medicinal product in the EU

44

Classified as public by the European Medicines Agency

44

Benefit-Risk Balance of a medicinal product

From Quality, Safety, Efficacy to Benefit Risk Balance

Legal framework: An application is to be refused if…

65/65/EEC

75/318/EEC

Harmful or, Therapeutic efficacy is lacking

2004/27/EC The risk-benefit balance is not considered to be favourable Therapeutic efficacy is

Harmfulness and therapeutic efficacy can only be examined in relation to each other; Therapeutic advantages must outweigh potential risks

insufficiently substantiated

45

An Agency perspective: the success and failure of a medicinal product in the EU

Classified as public by the European Medicines Agency

45

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