European Medicines Agency stakeholder interaction on the development of medicinal products for chronic non-infectious liver diseases (PBC, PSC, NASH)

Event Human Medicines

Date: Monday, 3 December 2018, All day
Location: European Medicines Agency, Amsterdam, the Netherlands

This workshop on the development of medicines for chronic non-infectious liver diseases, including primary biliary cholangitis, primary sclerosing cholangitis and nonalcoholic steatohepatitis, provides a platform for discussion on appropriate endpoints including validation of surrogate endpoints/biomarkers, suitable study populations, potentially adequate trial designs and the specific challenges with paediatric medicine development. The workshop will support the drafting of a reflection paper on regulatory requirements for the development of medicines for the treatment of these diseases.

Documents

Report - European Medicines Agency stakeholder interaction on the development of medicinal products for chronic non-infectious liver diseases (PBC, PSC, NASH)

Reference Number: EMA/873574/2019

English (EN) (275.08 KB - PDF)

First published: 21/06/2019

Programme - European Medicines Agency stakeholder interaction on the development of medicinal products for chronic non-infectious liver diseases (PBC, PSC, NASH)

Reference Number: EMA/269375/2017

English (EN) (347.88 KB - PDF)

First published: 13/09/2018 Last updated: 30/10/2018

Invitation for expressions of interest - European Medicines Agency stakeholder interaction on the development of medicinal products for chronic non-infectious liver diseases (PBC, PSC, NASH)

Reference Number: EMA/457607/2018

English (EN) (68.53 KB - PDF)

First published: 13/09/2018 Last updated: 13/09/2018

Presentations

Presentation - Session 1: Patient perspective (Robert Mitchell-Thain)

English (EN) (391.55 KB - PDF)

First published: 14/12/2018

Presentation - Session 1: Authorisation of Ocaliva for PBC in the EU (J. Musaus)

English (EN) (203.87 KB - PDF)

First published: 14/12/2018

Presentation - Session 1: PBC: Definition, natural history and current therapeutic interventions (Gideon Hirschfield)

English (EN) (2.14 MB - PDF)

First published: 14/12/2018

Presentation - Session 1: Historical outcome parameters used in PBC and the search for potential alternatives (Bettina E Hansen)

English (EN) (1.42 MB - PDF)

First published: 14/12/2018

Presentation - Session 1: Potential trial designs and suitable study populations (Bettina E Hansen)

English (EN) (673.42 KB - PDF)

First published: 14/12/2018

Presentation - Session 2: EMA stakeholder interaction on the development of medicinal products for chronic non-infectious liver diseases (Martine Walmsley)

English (EN) (445.77 KB - PDF)

First published: 14/12/2018

Presentation - Session 2: Definition, natural history and the lack of approved therapeutic interventions (Douglas Thorburn)

English (EN) (2.43 MB - PDF)

First published: 14/12/2018

Presentation - Session 2: Currently proposed endpoints in PSC: the search for reliable surrogate outcome parameters (Cyriel Ponsioen)

English (EN) (1.94 MB - PDF)

First published: 14/12/2018

Presentation - Session 2: Potential trial design and suitable study populations (Michael Trauner)

English (EN) (2.84 MB - PDF)

First published: 14/12/2018

Presentation - Session 2: The need for paediatric developments in PSC, trial duration and endpoints (Henkjan J. Verkade)

English (EN) (1.2 MB - PDF)

First published: 14/12/2018

Presentation - Session 3: NASH - The patient perspective (Yvonne Gray)

English (EN) (306.38 KB - PDF)

First published: 14/12/2018

Presentation - Session 3: PDCO current approach (Chrissi Pallidis)

English (EN) (199.69 KB - PDF)

First published: 14/12/2018

Presentation - Session 3: Non-alcoholic steatohepatitis (NASH): Definition, natural history and current therapeutic interventions (Frank Tacke)

English (EN) (3.56 MB - PDF)

First published: 14/12/2018

Presentation - Session 3: Outcome in NASH trials: From histology and “hard outcomes” to less invasive reliable surrogates (Laurent Castera)

English (EN) (7.34 MB - PDF)

First published: 14/12/2018

Presentation - Session 3: Potential trial designs and suitable study populations (Quentin M. Anstee)

English (EN) (4.42 MB - PDF)

First published: 14/12/2018

Presentation - Session 3: NAFLD: Population in need, clinical trial duration and endpoints (Piotr Socha)

English (EN) (1.3 MB - PDF)

First published: 14/12/2018

Multimedia

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