The EUPATI Academy is the only in-depth training program for patient experts so far in EU that lasts 14months.
It includes an on online training program supported with two weeks of F2F training. The Academy is still operated by EPF, providing high quality training in drug development to patient from EMEA.

View this resource Bookmark this resource

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

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

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. 

View this resource Bookmark this resource