Module 1 2021

28/06/2021

Post-approval Efficacy

 Usually planned ● Extension of the indication to different diseases or to extend the treatment duration ● After approval the target patient population is broadened and/or treatment duration extended .  Planned, and/or ● A requirement imposed at the time of initial approval. ● In procedures like Conditional Marketing Authorization, Adaptive Licensing, PRIME (and US Breakthrough designation): ● Post-approval data generation is required to get a better picture of the efficacy and safety in a specific patient population.  More often these data come from other, external sources (e.g. disease registries, Real World Data etc.)

The Organisation for Professionals in Regulatory Affairs

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Post-Approval Efficacy Example: The volume of post-approval efficacy activities of a TNF-blocker*

Nail Psoriasis, Quality of Life

Paediatric PK

PsA structural damage, physical function

Shortened infusion time Moderate CD

Fistulizing CD maintenance

Crohn’ s Diseas e USCrohn’s Disease

RA anaemia

UC colectomy, hospitalizatio n, surgeries

Early progressive RA

RA joint damage

Paediatric UC

Ulcerative Colitis Psoriasis

RA physical function

1998 1999 2000 2001 2002 2003 2004

2006 2005 2007 2008 2009 2010 2011 2012

2013

Rheumatoi d arthritis signs and symptoms

RA shortened infusion

CD time to response, dose escalation, mucosal healing, reduction of surgeries, hospitalization, and corticosteroid use

Luminal CD maintenance

RA dose escalation

Ankylosing spondylitis

Psoriati c Arthritis

1 st indication US EU indications

Paediatric CD

AS change of population

BIOSIMILARS

CD 3 rd to 2 nd line

* Ref: EPAR Remicade®

The Organisation for Professionals in Regulatory Affairs

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