Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT).
Dis Model Mech
; 13(2)2020 02 07.
Article
in En
| MEDLINE
| ID: mdl-32066568
ABSTRACT
Clinical trials for rare neuromuscular diseases imply, among other investments, a high emotional burden for the whole disease community. Translation of data from preclinical studies to justify any clinical trial must be carefully pondered in order to minimize the risk of clinical trial withdrawal or failure. A rigorous distinction between proof-of-concept and preclinical efficacy studies using animal models is key to support the rationale of a clinical trial involving patients. This Review evaluates the experience accumulated by the TREAT-NMD Advisory Committee for Therapeutics, which provides detailed constructive feedback on clinical proposals for neuromuscular diseases submitted by researchers in both academia and industry, and emphasizes that a timely critical review of preclinical efficacy data from animal models, including biomarkers for specific diseases, combined with adherence to existing guidelines and standard protocols, can significantly help to de-risk clinical programs and prevent disappointments and costly engagement.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Advisory Committees
/
Translational Research, Biomedical
/
Neuromuscular Diseases
Type of study:
Guideline
Limits:
Humans
Country/Region as subject:
Europa
Language:
En
Journal:
Dis Model Mech
Journal subject:
MEDICINA
Year:
2020
Document type:
Article
Affiliation country: