A decade of optimizing drug development for rare neuromuscular disorders through TACT

Kathryn R. Wagner; Annamaria De Luca; Didier Caizergues; James Dowling; Nathalie Goemans; Heather Gordish-Dressman; Miranda D. Grounds; Michael Kelly; Anna Mayhew; Elizabeth M. McNally; Tracey Zoetis; Joanne Lee; Cathy Turner; Dominic J. Wells; Cristina Csimma; Volker Straub

doi:10.1038/d41573-019-00199-1

A decade of optimizing drug development for rare neuromuscular disorders through TACT

Kathryn R. Wagner, Annamaria De Luca, Didier Caizergues, James Dowling, Nathalie Goemans, Heather Gordish-Dressman, Miranda D. Grounds, Michael Kelly, Anna Mayhew, Elizabeth M. McNally, Tracey Zoetis, Joanne Lee, Cathy Turner, Dominic J. Wells, Cristina Csimma, Volker Straub

Medicine, Cardiology Division

Research output: Contribution to journal › Comment/debate › peer-review

4 Scopus citations

Abstract

This year marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare neuromuscular diseases and identifies pitfalls. Here, we discuss the experience with TACT based on its reviews of more than 50 applications and its potential as a model for other rare disorders.

Original language	English (US)
Journal	Nature Reviews Drug Discovery
Volume	19
Issue number	1
DOIs	https://doi.org/10.1038/d41573-019-00199-1
State	Published - Jan 1 2020

Funding

The TACT committee aims to apply the TACT model to other disease areas. This will be explored through two projects — conect4children (c4c) and the European Joint Programme for Rare Diseases (see Related links). The aim of the pan-European project c4c, which is funded by the Innovative Medicines Initiative, is to facilitate the development and availability of new medicines for neonates, paediatric patients and adolescents, through the creation of a large collaborative paediatric network. Through the European Joint Programme for Rare Diseases, an Advisory Committee for Therapeutics (ACT) toolkit will be created based on the TACT model to enable other rare disease networks to develop their own ACT. TACT has received funding from Parent Project Muscular Dystrophy, Cure Duchenne, Muscular Dystrophy UK, MDA, Joining Jack, Duchenne UK, Duchenne Ireland, Myotubular Trust, Duchenne Now, Duchenne Children’s Trust and SMA Europe.

ASJC Scopus subject areas

Pharmacology
Drug Discovery

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10.1038/d41573-019-00199-1

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Wagner, K. R., De Luca, A., Caizergues, D., Dowling, J., Goemans, N., Gordish-Dressman, H., Grounds, M. D., Kelly, M., Mayhew, A., McNally, E. M., Zoetis, T., Lee, J., Turner, C., Wells, D. J., Csimma, C., & Straub, V. (2020). A decade of optimizing drug development for rare neuromuscular disorders through TACT. Nature Reviews Drug Discovery, 19(1). https://doi.org/10.1038/d41573-019-00199-1

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author = "Wagner, {Kathryn R.} and {De Luca}, Annamaria and Didier Caizergues and James Dowling and Nathalie Goemans and Heather Gordish-Dressman and Grounds, {Miranda D.} and Michael Kelly and Anna Mayhew and McNally, {Elizabeth M.} and Tracey Zoetis and Joanne Lee and Cathy Turner and Wells, {Dominic J.} and Cristina Csimma and Volker Straub",

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Wagner, KR, De Luca, A, Caizergues, D, Dowling, J, Goemans, N, Gordish-Dressman, H, Grounds, MD, Kelly, M, Mayhew, A, McNally, EM, Zoetis, T, Lee, J, Turner, C, Wells, DJ, Csimma, C & Straub, V 2020, 'A decade of optimizing drug development for rare neuromuscular disorders through TACT', Nature Reviews Drug Discovery, vol. 19, no. 1. https://doi.org/10.1038/d41573-019-00199-1

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AU - Wagner, Kathryn R.

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AU - Caizergues, Didier

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AU - Goemans, Nathalie

AU - Gordish-Dressman, Heather

AU - Grounds, Miranda D.

AU - Kelly, Michael

AU - Mayhew, Anna

AU - McNally, Elizabeth M.

AU - Zoetis, Tracey

AU - Lee, Joanne

AU - Turner, Cathy

AU - Wells, Dominic J.

AU - Csimma, Cristina

AU - Straub, Volker

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A decade of optimizing drug development for rare neuromuscular disorders through TACT

Abstract

Funding

ASJC Scopus subject areas

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