Introduction

ATMPs (Advanced Therapy Medicinal Products) have the potential to bring about significant changes in the field of medicine. These innovative therapies including gene therapies, cell therapies and tissue-engineering products. Treatment with ATMPs is different in many ways from conventional therapies (e. g. small molecules). ATMPs focus on manipulating genes, cells, or tissues to address the underlying causes of diseases. This personalized approach allows for more targeted and precise treatments, potentially offering improved efficacy and long-lasting effects. 

Despite the potential of this novel approach, many questions that are relevant for patients, healthcare systems and society in general are not answered yet. Although vigilance systems on ATMPs are under construction, knowledge about the long-term effect of therapeutic manipulation of genes, cells and tissues by ATMPs is limited. 

ATMPs involve companion diagnostics on individual level. Individualized manufacturing processes tailored to each patient requires specific expertise and infrastructure to manufacture and apply ATMPs. This presents logistical hurdles, scalability limitations, and hindering wider accessibility and affordability. Additionally, the high costs associated with ATMP development and production pose economic challenges for healthcare systems and reimbursement schemes. 

All of this creates complex technical, regulatory and societal requirements for these innovative technologies and new challenges for society to have answers.

Mission

Complexity can be addressed by collaboration, multidisciplinary skills and foresight. International cooperation supports to prepare people and systems for ATMPs in the best possible way. Involving all relevant disciplines including patients and the carer environment requires more attention and knowledge in this area.

The aim of the working group is to increase awareness, knowledge and data availability in the field of ATMP. The group brings together individuals, organisations, members and non-members with specific skills and different professional and cultural backgrounds to share information and knowledge related to new and emerging ATMPs, specifically from non-industrial development. The collaboration supports the building of a common understanding of needs, requirements, methods and standards of the different stakeholders involved. A common understanding forms the basis of a similar knowledge structure, improving communication and collaboration in the development and implementation of value-added technologies in comparison to existing standards.

The working framework

Early Awareness on emerging, unusual technologies

Permanent identification of emerging technologies in the field of ATMPs which, due to their characteristics, do not fit into given procedures and standards in development, production, evaluation and use. The working group is supported in this by the existing skills for identifying these technologies in the network.

Detection of gaps and disparities, determining as system and patient requirements

Uncovering local and regional gaps and disparities in healthcare provision will help to describe where Advanced Therapy Medicinal Products (ATMPs) are or can be available to people. The identification of inequalities and enabling targeted interventions to address them. 

The findings of the working group will be made available to all members and partners of international HealthTechScan according to their requirements via the network's information system.

Provision of information to members and partners

Information captured by the working group will provided to members and partners to promote the development support, evaluation, utilization, training and education in the field of Advanced Therapy Medicinal Products (ATMPs).

Membership

Membership of the working group is by invitation only. The selection of members is based on their expertise, knowledge and commitment to the particular topic or area of work of the group. Invitations will be extended to individuals or organisations who are willing to actively contribute to the work and objectives of the Working Group.

Invitation to the working group is a recognition of the expertise and potential contribution of the invited members. Selection by invitation ensures that the working group is composed of individuals and organisations who can contribute their specific knowledge and experience to the work.

Members who accept the invitation become active participants in the working group and contribute to the development of recommendations, solutions or other outcomes of the working group. The invitation basis makes it possible to assemble a team with different perspectives and areas of expertise.