The purposes of Undiagnosed Diseases Programs (UDPs) are to provide patients with an unknown genetic condition a diagnosis and to find the correlation between genotype and phenotype; to share globally the information to facilitate the diagnosis through a matchmaking for finding possible second cases

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The purpose of NBS is to detect potentially fatal or disabling conditions in newborns as early as possible and possibly before onset of symptoms. Such detection allows the early treatment which may significantly modify the natural history of the disease and potentially prevent developmental delays, physical disabilities and eventually death.

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

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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

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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

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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

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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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