TACT: the TREAT-NMD Advisory Committee for Therapeutics

The need for TACT

Of the many promising research results presented at conferences, published in journals and hailed as the basis for possible future treatments and cures, few progress into clinical trial. Evaluating the therapeutic potential of drugs seemingly ready for this step is a challenge not only for the patients who build hope on preclinical results and for the potential funders and industry sponsors of the research, but also for the researchers themselves.

TACT, the TREAT-NMD Advisory Committee for Therapeutics, is an expert multidisciplinary body that provides the neuromuscular community (clinicians, researchers, patient advocacy groups and industry) with independent and objective guidance on advancing new therapies (whether novel or repurposed) for neuromuscular diseases. Its goal is to position the potential therapy along a realistic pathway to eventual clinical trial and registration by evaluating preclinical data as well as drug development considerations that are crucial for the conduct of studies that generate meaningful data. In close collaboration with the TREAT-NMD clinical trials coordination centre (Freiburg, Germany), TACT is also dedicated to providing information on optimal trial design and the resources available to investigators and industry for clinical trial planning and conduct.

The committee

Cristina Csimma, as TACT chair, is leading this exciting initiative with the support of the TACT core committee, nominated extended committee members and the TREAT-NMD secretariat.

The remit of the committee is to provide applicants with transparent and consistent guidance and advice, in an educational and directional context, on the readiness of drugs and/or therapeutic targets for trial. TACT advice will be helpful for preparing funding applications and investigational drug applications, while the publication of a non-confidential summary will ensure the community receives expert feedback on the progress of the application.

In addition to the expert analysis of preclinical data, TACT reviews also address issues of drug formulation, bioavailability and toxicology as well as possible regulatory requirements and marketing considerations. In close collaboration with the TREAT-NMD clinical trials coordination centre in Freiburg, TACT is also dedicated to providing information on rational and economical trial design. The ultimate goal of TACT is to help pave the pathway for successful drug registration by providing professional and independent advice.

The TACT committee convenes once every six months to consider selected applications. TACT will consider more than one drug at a time and therefore will have panels of experts, selected from the TACT members, reviewing the drugs concurrently, with the same chair and core committee for continuity.

Terms of Reference