Module 6

04 October 2024

Failure/Success of MAA: associated factors

111 MAAs between 1997-2001

Peak of 41% in 1998

29% failed MAA in 1997-2001

28% failed MAAs in 1995-1999

Variables: Che/Bio,ATC, start year, MO presence

Most common MO: Clinical safety (inadequate safety database, SAE) Clinical efficacy (lack of RCT)

Failed MAA contains unresolvable Major Objections (MO) : deficiencies incompatible with a positive outcome

188 MAAs between 2004-2007

- Lower approval rate for ODD - SA compliance and company size are success factors

27% failed MAA in 2004-2007

Variables: Company Size, Che/Bio,ODD,Therapeutic Areas, SA compliance (choice of primary endpoint, selection of control, statistical methods)

34% failed MAAs in 2009-2010

64 MAAs between 2009-2010

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

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Classified as public by the European Medicines Agency

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Analysis of ODD MAA : Method

• ODD between 2000-2013, prevalence

• MAA Outcome (year, positive, negative, withdrawn), MA level of evidence (RCT:

Y/N) , SA request/compliance , Major Objections (MO)

• OD maintenance: positive, negative

• Comparison orphan vs non orphan: MAA outcome, regulatory pathways, type of

active substance, Therapeutic Areas, SA/Protocol Assistance request,

company size

• Factors associated with MAA success for ODD?

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

Classified as public by the European Medicines Agency

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