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

Published by

Advisory Committee for Therapeutics

2020 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.

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Once the pre-application form has been accepted, the applicant will be asked to submit a more detailed application form. This will ensure that the review panel will have sufficient information to be able to comprehensively review the application for advice. Depending on the disease area, the core committee should agree consensus on the questions to be included as part of an ACT application form.

This report to the applicant template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit. 

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SPIRIT PRO Extension for inclusion of patient-reported outcomes in clinical trials protocols

Published by ECRIN

Clinical Trials ToolkitPCOM

Extension of the SPIRIT (Standard protocol items: recommendations for interventional trials) guideline, SPIRIT PRO provides guidelines for inclusion of patient-reported outcomes in clinical trial protocols.

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PedCRIN list of Tools

Published by ECRIN

PediatricClinical Trials Toolkit

PedCRIN developed a series of tools (WP3) and procedures to support the setup and the management of multinational neonatal and paediatric clinical trials in Europe. These tools are developed to train and support researchers and clinicians so they can establish, run, and manage both paediatric and neonatal multinational clinical trials in a more effective and easier way. The removal of barriers to trial management will speed up the evaluation of new and improved therapies.

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Advisory Committee for Therapeutics (ACT) toolkit – Report to applicant template

Published by UNEW

Translational ResearchResearch and Drug DevelopmentTREAT-NMDClinical DevelopmentClinical Trial DesignPreclinical ResearchTemplatesAdvice

After the review meeting, the lead reviewer, with input from the review panel, will create a report to the applicant which will include recommendations, advice and answers to the applicant’s specific questions. The lead reviewer will send the report to the secretariat to be formatted, reviewed and added to the report template before being sent out to the applicant.

This report to the applicant template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit. 

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Advisory Committee for Therapeutics (ACT) toolkit – Pre-application form template

Published by UNEW

Translational ResearchResearch and Drug DevelopmentTREAT-NMDClinical DevelopmentClinical Trial DesignPreclinical ResearchTemplatesAdvice

The pre-application form should be issued to potential applicants when they enquire about an ACT review. This form is designed to gain more information about their intended drug development plan and to allow the ACT Chair and secretariat to decide if their application is at a suitable stage for review. If accepted, this form will also be used by the secretariat to identify potential reviewers.

This report to the applicant template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit. 

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Advisory Committee for Therapeutics (ACT) toolkit – Conflict of interest (CoI) form template

Published by UNEW

Translational ResearchResearch and Drug DevelopmentTREAT-NMDClinical DevelopmentClinical Trial DesignPreclinical ResearchTemplatesAdvice

To ensure that a review panel includes independent and objective reviewers, each reviewer is required to complete a conflict of interest form for each application they review. The conflict of interest form must be submitted when a reviewer agrees to provide advice at a particular ACT meeting. Each form will be reviewed by the secretariat, in consultation with the ACT Chair. If a conflict of interest is identified for a specific application, they will not be able to review the application form or provide advice on the application.

This conflict of interest form template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit. 

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Advisory Committee for Therapeutics (ACT) toolkit – Applicant agreement template

Published by UNEW

Translational ResearchResearch and Drug DevelopmentTREAT-NMDClinical DevelopmentClinical Trial DesignPreclinical ResearchTemplatesAdvice

Once the pre-application form has been accepted for review, an applicant agreement form should be issued to the applicant to ensure that they are committed to attending an ACT review meeting. The agreement will include a request for donation. Please note that this agreement is only to be completed by industry applicants. You may wish to create a modified version for academic applicants.

This applicant agreement template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit. 

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This template includes a suggested schedule of timings, based on the TACT model. This agenda is intended for reviewers, observers and the secretariat. A modified version should be created for each applicant informing them of timings of the review, venue and a list of reviewers on their review panel. The modified version for each applicant will not include information about other applicants.

This agenda template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit. 

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