A new report published in the Journal of Neuromuscular Diseases highlights the impact of the TREAT-NMD Advisory Committee for Therapeutics (TACT) in advancing and de-risking drug development for neuromuscular diseases (NMDs).

The paper, “TREAT-NMD Advisory Committee for Therapeutics: Preclinical and Clinical Learnings from 15 Years of TACT,” reflects on lessons learned from 90 therapeutic development programmes reviewed by TACT between 2009 and 2024, including 68 industry-sponsored applications.

TACT was established to provide academic and industry developers with confidential, multidisciplinary expert advice across all stages of drug development. Each review panel brings together internationally recognised experts in preclinical research, clinical trial design, biostatistics, toxicology, regulatory science, biomarkers, manufacturing, and patient advocacy.

The publication outlines common challenges seen across neuromuscular drug development programmes, including weaknesses in preclinical study design, endpoint selection, statistical planning, and regulatory strategy. By sharing these learnings, the authors aim to help developers avoid common pitfalls and improve the quality and readiness of therapies entering clinical trials.

Annemieke Aartsma-Rus, PhD, of Leiden University Medical Centre and corresponding author shares:

“TACT was created to help derisk therapeutic development programmes and to increase the chance of effective therapies reaching the clinic. Fifteen years later, the continued demand for TACT, where some applicants return multiple times, reflects the value of bringing together broad, experienced expertise to support neuromuscular drug development.”

Since its launch, the neuromuscular field has evolved from having a single approved therapy to multiple approved treatments and a rapidly expanding development pipeline. The authors note that while major progress has been made, significant unmet need remains across many rare neuromuscular diseases, so more treatments are needed.

The review emphasises that TACT’s role is not only to support successful programmes, but also to identify gaps early, helping companies and academic groups make better-informed development decisions and ultimately improving the likelihood of clinical and regulatory success.

The full article is freely available in the Journal of Neuromuscular Diseases.

 About TREAT-NMD

TREAT-NMD is a global network of experts in the neuromuscular field. Established in 2007 and headquartered at Newcastle University, the network today comprises more than 100 research centres and patient organisations across 54 countries, alongside independent academics and patient representatives. TREAT-NMD’s mission is to accelerate the development of effective treatments and improve diagnosis and care for people living with neuromuscular diseases worldwide. The network achieves this by improving global trial readiness, delivering high quality educational programmes, advancing patient diagnosis and standards of care, and supporting preclinical research. 

For further information please contact: 

Samara Laboriel | Marketing and Communications Officer and Business Partner, TREAT-NMD 

Email: info@treat-nmd.org 

Website: www.treat-nmd.org 

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