TACT : TREAT-NMD Advisory Committee for Therapeutics
Introduction and background
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 trials. Evaluating the therapeutic potential of drugs seemingly ready for this step is a challenge not only for the patients who build hope on non-clinical results and for the potential funders and industry sponsors of the research, but also for the researchers themselves. When research does move into trials, poor design or lack of thorough understanding across all areas can mean that trials fail or are sub-optimal.
Established in 2009, the TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary international group of internationally recognized academic and industry drug development experts as well as representatives of patient foundations and regulatory experts, 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 including:
Duchenne Muscular Dystrophy (DMD)
Spinal Muscular Atrophy (SMA)
Becker Muscular Dystrophy (BMD)
Congenital Muscular Dystrophies (CMD)
Congenital Myasthenic Syndromes (CMS)
Myotonic Dystrophy (DM)
Charcot Marie Tooth (CMT)
Myotubular and Centronuclear Myopathies (MTM-CNM)
Limb Girdle Muscular Dystrophy (LGMD)
Facioscapulohumeral muscular dystrophy (FSHD)
Amyotrophic lateral sclerosis (ALS)
The confidential and comprehensive review provides recommendations including go-no-go milestones and is independent of any funding stream. However, applicants often use TACT reports to support subsequent funding applications or discussions.
The TACT Chair rotates on a 3 yearly basis. Prof. Annemieke Aartsma-Rus took over from Prof. Annamaria De Luca in 2021.
Previous TACT chairs:
If you are interested in submitting an application to TACT or would like to learn more, please contact Cathy Turner (email@example.com).
Upcoming TACT Review Meetings
4–5 November 2022 – Online
18–19 November 2022 – Online
21–23 April 2023 – Philadelphia, PA, USA
- Provide a one point in time multi-disciplinary review.
- Meet 2 times per year (once every 6 months) – the location changes for each meeting.
- Accept applications for therapeutics targeted to any form of rare inherited neuromuscular disease.
- Carry out reviews under a confidential disclosure agreement.
- Convene a bespoke panel of experts from the extensive committee for each review based on the needs of an application, recruiting new key opinion leaders as needed.
- Undertake a check for any conflict of interest with all reviewers for all applications and to communicate the details of the panel members with applicants in advance of sharing the full application.
- Review therapeutics at any stage of development, so long as a lead compound has been identified, 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 of each reviewed application to ensure the community is aware that a review has taken place.
- Provide information about upcoming meetings on the TREAT-NMD website and in TREAT-NMD newsletters.
TACT will not:
- Provide on-going feedback to applicants on their 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.
Terms of Reference
Please read the TACT Terms of Reference carefully if you are considering submitting an application to TACT.
Support for TACT
Industry applicants are asked to pay a contribution towards meeting costs based on size of company. TACT has received generous support from:
PPMD, Cure Duchenne, MDUK, MDA, Joining Jack, Duchenne UK, Duchenne Ireland, Myotubular Trust, Duchenne Now, SMA Europe, Association Française contre les Myopathies, Jesse’s Journey, Duchenne Research Fund, Action Duchenne, Cure CMD and Foundation to Eradicate Duchenne
If you represent a patient organisation and would be interested in supporting TACT, please contact Cathy Turner.
“I feel very privileged to be involved in TACT, which combines multidisciplinary expertise with a constructive attitude. The goal is not to discredit therapeutic approaches, but to make sure preclinical and clinical studies are designed and conducted optimally because as a field we owe this to the patients. The multidisciplinary teamwork for each application is a unique, motivating and educational experience for everyone involved. Each expert tries to improve an approach based on their own background and knowledge to provide the applicant with very comprehensive feedback for future development.“
Annemieke Aartsma-Rus, TACT Chair
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
A Newcomer’s View
By Fabiola M. Bertinotti
It’s not easy to write about the complex range of feelings that have prevailed in me during my week in Vienna in early July where I had the privilege to enter the world of TREAT-NMD and to act as a member of TACT, the team of neuromuscular experts who form the TREAT-NMD Advisory Committee for Therapeutics.
A Welcoming Environment
First and foremost, I have to say that everyone was particularly kind and welcoming to me, while I felt honored (and admittedly a bit nervous at the same time) to sit amongst an array of highly competent people voluntarily lending their expertise in different disciplines with one common goal: that of providing high-end advice and feedback to pharma companies and academic-led groups during the challenging and perilous pre-clinical phase leading into clinical studies or on clinical trial plans themselves, ultimately leading to drug development. As my first experience as a patient representative, I felt overwhelmed with a sense of responsibility towards my TACT colleagues as well as all the stakeholders of the rare disease community, people all working very hard in order to help a drug design programme more successfully move forward into the “real world” of a clinical trial and eventually treatment, involving patients.
My Patient Perspective
Around the U-shaped table, I could see experts in neurology, pharmacology, immunology, physiotherapy, genetics, toxicology, regulatory affairs, statistics, pediatrics, neuroscience, molecular genetics, biologics, damage and repair of the skeletal muscle, biopharmaceuticals, and industry specialists in therapy development, as well as patient organization representatives like me, all engaged in a fast-moving dialogue around an applicant’s proposal for drug development. Was I prepared for all this “scientific tsunami”? I’d say yes, although I have to admit that I was feeling like a rookie in many ways. Certainly, I will never bless enough the day in which I decided to enroll in the EURORDIS Summer School which had kept me awake long nights over the last six months studying all the different subjects which needed to be learned in order to obtain a certificate as Patient Expert. This really equipped me well to be able to follow the reasoning being discussed around this TACT table.
Understanding the impact of a disease is never easy, whether one is a patient’s carer or even if the disease for which a drug or therapy is being debated is not specifically affecting a close family member or friend. I personally sat on the Committee to evaluate two different applications which I cannot discuss because I am, of course, covered by a non-disclosure agreement. One of them was touching me very closely and, for a moment, I thought I would react emotionally, but after the first three minutes, all the fear disappeared and I was up and running, fully immersed in the debate and especially interested in the interaction of the various experts who were expressing their constructive criticism towards the applicant’s proposal.
An In-depth and Fast-Moving Dialogue
How does it all work? First of all, the application is brought to the attention of the experts through the TACT Secretariat, the TACT secretariat put together a multidisciplinary panel of experts which is bespoke to the application being reviewed. Reviewers are asked to submit feedback on the application being reviewed. All the feedback is collected by the Secretariat and on the day of the meeting with the applicant(s), TACT experts get together beforehand and discuss the proposal, highlighting its strengths and limitations. The applicant(s) are then invited to join the committee in the meeting room and have approximately ten minutes to give an overview presentation of their application and present any new data. One of the TACT members acts as a lead reviewer and expresses the various concerns of the committee whose members also contribute to the conversation by posing questions to the applicant. That is when the real debate begins. One of the many fascinating dynamics I was able to witness was the intensity of the team work carried out by my colleagues who were raising all the key questions while staying open to hear the applicants (from academia or industry) providing answers and making a wide range of enquiries. The whole process was fast-moving, thanks to the invaluable TACT Secretariat and the moderator who helped carry on the debate in a seamless manner until it was time for the applicant representatives to leave the meeting, with the committee then reconvening to work for another hour to sum up all the feedback and main indications.
As previously mentioned, on the same day I attended two different meetings where two applications were respectively assessed. At my second session, I could really understand and appreciate the seamless and harmonized design by which the TACT meeting functioned, working in a fast and coordinated way which made the questions and answers session with the applicant extremely effective. The conclusive piece lasted just one hour! WOW. I was both ecstatic and full of admiration at the same time.
A Steaming Locomotive
It is such an invaluable service that the TACT team brings to science, industry and – most of all to the patients! It is really such a relief for someone like me with an affected son at home to be able to rely on this wonderful ‘steaming locomotive’ made of a well-oiled, highly competent, independent battery of experts. But, as they say in the entertainment business in which I work, the best was yet to come! All the experience I gained during the TACT committee meetings the first day, found its perfect completion the following day, when I sat amongst the audience participating to the “TREAT-NMD Teaching Course in Drug Development” within the ICNMD (International Congress on Neuromuscular Diseases) in Vienna. In fact, the aim of this congress is to present a wide spectrum of neuromuscular diseases from the perspectives of advances in research, diagnosis and treatment. There, my TACT colleagues were to teach what the actual benefits of TACT are vis-à-vis the science community.
First of all, as an expert in corporate communication and social responsibility, I truly applaud the effective way in which the entire course was structured, with a moderator keeping the various presentations more in the form and tone of interviews and therefore flowing in a very fluid and intriguing way for the audience. Last but not least, a mock committee was put on the stage with all the experts acting out their roles like movie characters. I must say that this was truly the best part. In fact, I learned that these highly respected experts are so humble and passionate that they can even jump onto the stage and actually “perform” their teaching in a clear, but also very fun way. Kudos!
What I truly appreciated above everything else is that the TACT members are all volunteers that do their job with love and passion for the good of the patients.
Fabiola M. Bertinotti