TACT: fully Approaching Drug Development

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

 
17 Aug 2018