Module 1 2021

28/06/2021

Post-approval efficacy and safety: A pro-active, strategic approach At the time of approval

• limited data and more uncertainties

• CHMP will ask for a commitment to generate post-approval data to address the uncertainties.

• CHMP final assessment depends on agreement on post-approval commitments, mostly on safety, but also efficacy

Company strategy: be prepared to present “on the spot” company-approved scenarios, e.g.

• ... follow the patients more closely, with questionnaires, PSURS with a higher frequency..

• …. participate in an existing Real World Evidence program where patients-data can be compared to other treatments

• … set up an own registry in which you can control the data

• … but, ideally the CHMP would like to see a long term randomized double blind controlled trial.  Whatever the outcome, it is work for the years to come!

The Organisation for Professionals in Regulatory Affairs

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Life Cycle Management The volume of post-approval efficacy + safety

Infections in the elderly

Sarcoidosis

Nail Psoriasis, Quality of Life

Invasive fungal infections, aspergilosis Paediatric PK

TB, skin and subcutaneous tissue disorders PsA structural damage, physical function

Cardiomyopathy, parasitic infections

Delayed Hypersensitivity

Shortened infusion time

Invasive fungal infections

Fistulizing CD maintenance

Opportunistic Infections

ILD, AEs with fatal outcome

Agranulocytosis, Pancreatitis

CD: 2 nd to 3 rd line therapy

Oligosaccharides

RA anaemia

Crohn’s Disease US

Moderate CD

Demyelinating disorders

Heart failure Early progressive RA

UC colectomy, hospitalization, surgeries

RA joint damage

HSTCL

Interstitial pneumonitis /fibrosis

Peripheral demyelination

Severe infections

Paediatric UC

Vasculitis Ulcerative Colitis Psoriasis

Crohn’s Disease

Extrapulmonary TB

HSTCL

Pregnancy

RA physical function

1998 1999 2000 2001 2002 2003 2004

2005

2006

2007 2008 2009 2010 2011 2012

2013

Readministration

HBV reactivation

Serum sickness, Pericardial effusion

HBV

Pancytopenia Listeriosis

Hepatotoxicity RA shortened infusion

Rheumatoid arthritis signs and symptoms

Luminal CD maintenance

ADR frequencies

Multiorgan complications, opportunistic infections CD time to resp nse, dose escalation, mucosal healing, reduction of surgeries, hospitalization, and corticosteroid use

Malignancies in COPD

Transverse myelitis, Anemia, Hepatocellular damage

Safety in juvenile RA

RA dose escalation

Ankylosing spondylitis

Malignancies

Pneumonia

New onset psoriasis, pustular psoriasis, hepatobiliary events

Psoriatic Arthritis

1 st indication US EU indications

Paediatric malignancy, leukaemia

Paediatric CD

BIOSIMILARS

AS change of population

CD 3 rd to 2 nd line

Viral infections, witching between biological DMARDs, neutropenia, hematologic reactions

Every box represents a regulatory procedure, with a clinical report, a safety analysis, several summaries, a revised label etc. A truly multi-disciplinary team work and within a fixed timeframe.

The Organisation for Professionals in Regulatory Affairs

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