TACT : TREAT-NMD Advisory Committee for Therapeutics

TACT : TREAT-NMD Advisory Committee for Therapeutics
  • TACT Chair
  • Kathryn Wagner
    Kathryn Wagner
    Kathryn R. Wagner, M.D., Ph.D. is the Director of the Center for Genetic Muscle...
  • Latest publication
  • The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development", Heslop E et al. Orphanet Journal of Rare Diseases 2015, April

    DOI:10.1186/s13023-015-0258-1
  • Quote...
  • “The strength of TACT is its broad scientific and development expertise coupled with the rigour and independence of its review process, performed in a truly global context. This committee can provide objective and constructive guidance from world experts in the field that will both help researchers focus on areas for development and ensure the wider community is better informed about the readiness of new therapies for the next step.” 

    Cristina Csimma, previous TACT chair

 

Introduction

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.

Established in 2009, the TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary international group of well recognized academic and industry drug development experts as well as representatives of patient foundations and institutional governmental scientific research centers, who meet twice a year to review and provide guidance on the translation and development path of therapeutics programs in rare neuromuscular diseases with large unmet need, such as muscular dystrophies and amyotrophic lateral sclerosis (ALS). The confidential and comprehensive review provides recommendations including go-no-go milestones, is independent of any funding stream however it may enable subsequent funding.

The TACT Chair rotates on a 3 yearly basis. Dr Wagner took over from Professor Dominic Wells, in 2015.

TACT will:

  • Provide a one point in time multi-disciplinary review.
  • Meet 2 times per year (once every 6 months).
  • Accept applications for therapeutics targeted to any form of rare inherited neuromuscular disease.
  • Carry out reviews under a confidential disclosure agreement.
  • Review therapeutics that are presented as having a clear perspective within the translational process with the long-term goal of an intended clinical trial and potential registration.
  • Consider reviewing pre-reviewed programmes if they have substantially progressed or changed and if the committee felt that it could add further value.
  • Address issues of drug formulation, bioavailability and toxicology as well as possible regulatory requirements and marketing considerations.
  • Provide applicants with a comprehensive written review, including recommendations no later than 6 weeks following the meeting.
  • Publish a non-confidential summary to ensure the community receives expert feedback on the progress of the application.
  • Provides information about the projects reviewed and planned to be reviewed at TACT meetings and the timing of TACT’s recommendations report on the TREAT-NMD website and in TREAT-NMD newsletters.

TACT will not:

  • Provide on-going feedback to applicants on the development programme.
  • Respond to queries following the issue of the final report.
  • Amend or update the final report.
  • Engage with funding organisations on behalf of applicants.
  • Provide funding; TACT is not a funding organization but provides evaluation and recommendations which might facilitate development of a review into an application to a funding organization.

If you wish to find out more about TACT you may want to view a video of a presentation given at the Faster Cures Conference in New York City, November 2012. View video here.


Terms of Reference

Please read the TACT Terms of Reference carefully if you are considering submitting an application to TACT.

Download TACT Terms of Reference




 

 
11 Apr 2016