From TACT to ACT: Expansion of the TACT model in other rare diseases
Since 2009, the TREAT-NMD Advisory committee for Therapeutics (TACT) has served the neuromuscular community by providing independent and objective advice on the developmental pathway of neuromuscular drug development programmes. Due to the success of TACT, staff from the John Walton Muscular Dystrophy Research Centre, Newcastle University have developed a toolkit to replicate the TACT model.
The Advisory Committee for Therapeutics (ACT) toolkit provides step-by-step procedural advice for other rare disease communities, who share similar challenges in therapy development, to establish their own ACT. The toolkit was developed through the European Joint Programme for Rare Diseases (EJP RD)* and is a freely available resource in the EJP RD Innovation Management Toolbox (IMT). The IMT is a free to access library of over 450 translational medicine resources on rare diseases. The IMT is listed as a resource to support sponsors on the European Medicines Agency (EMA) website.
One of the goals of EJP RD was to identify good practices and support adoption to other communities. With this in mind, an EJP RD EJP RD funded European Reference Network (ERN) training workshop was held in 2019 to introduce the ACT model and the toolkit to ERN representatives. After the workshop, meetings were held with ERNs who were identified as being interested in further exploring the feasibility of setting up an ACT in their respective fields. Another EJP RD workshop was organised in 2023 to support these ERNs to bring together key stakeholders in their field to discuss the benefits and challenges of adopting the ACT model in their field.
Since the workshop took place, The Ataxia Global Initiative has now set-up and launched their own ACT. The Ataxia Advisory Committee for Therapeutics (Ataxia ACT) aims to optimize the planning and execution of preclinical and clinical drug development programs for all forms of Ataxias. In March 2024, Joanne Lee (Newcastle University, former member of the TACT secretariat) and Annemieke Aartsma Rus (Leiden University Medical Centre, current TACT Chair) presented at a workshop organised by the Ataxia Global Initiative. This workshop was designed to provide members of the Ataxia ACT core committee with training and support before their first ACT review meeting.
The Ataxia ACT is now open for applications from both academia and industry. The first Ataxia ACT review meeting took place between 11th and 12th November in London, UK. Holm Graessner from the Ataxia Global Initiative spoke to us about the launch of their ACT:
There are only a few approved therapies for ataxia, a group of rare degenerative neurological diseases. Offering an Ataxia ACT review to therapy developers is a significant step toward improving trial readiness in this field.
We are grateful to the TACT team for allowing us to build on their experience and process flow, as well as for their active support in establishing our own ACT program.
Our first meeting underscored the need for an ACT initiative in ataxias, and we look forward to future meetings to advance ataxia therapy development.
For further information, please contact the following:
*This initiative has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement N°825575.
Author(s)
Joanne Lee