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The JOIN4ATMP project aims to accelerate and de-risk European ATMP development and ensure wide-spread access of ATMPs, through the mapping of obstacles to such development, the audit of real-world-based solutions and the definition of new paths forward.


What are ATMPs?

Are you new to ATMPs or are you interested in the JOIN4ATMP project but you do not have a clear overview of its European context? In this article, we offer you the keys to better understand this innovative type of medicine through 7 questions.

  1. How does the EU define ATMPs?
    Advanced Therapy Medicinal Products (ATMPs) are medicines which consist of gene therapy, cell therapy or tissue-engineered products that can sustainably improve diseases that currently have inadequate standard-of-care options. These diseases include genetic disorders, cancers, degenerative diseases and autoimmune diseases. ATMPs are particularly suitable for conditions that lack tailored and effective treatment options.

  2. What are the specific requirements for the approval of ATMPs in the European Union?
    To put an ATMP on the European market, developers need to fulfil specific requirements and receive a single authorization from the European Medicines Agency (EMA). Such requirements include preclinical and clinical data demonstrating safety and quality of the developed medicine and its compliance with Good Manufacturing Practice (GMP) standards. Applicants can seek scientific advice for the authorization procedure from the EMA.

  3. How are preclinical and clinical studies for ATMPs designed and conducted in Europe?
    Preclinical studies involve in vitro testing (outside the human body or any other living organism, in a controlled laboratory environment) and animal testing to assess safety and biological activity. Clinical studies are conducted in phases:

    • Phase I: assess safety and dosage ranges
    • Phase II: evaluate efficacy and side effects
    • Phase III: confirm efficacy and monitoring of side effects, collect safety data

    Studies must comply with Good Clinical Practice (GCP) guidelines and take ethical considerations into account, including informed consent and patient safety monitoring.

  4. Who are the major actors involved in the ATMP sector in Europe?
    Various actors take part in the development, approval and distribution of ATMPS. Major stakeholders include academic and not-for-profit research institutions, pharmaceutical and biotechnology companies, developers, regulatory bodies like the EMA, policy makers, healthcare providers, patient advocacy groups and reimbursement institutions. Collaboration among these stakeholders is crucial for advancing research, ensuring regulatory compliance, and facilitating patient access to ATMPs.

  5. What are the main challenges for the development of ATMPs?
    Despite the opportunities they present in the domain of health, ATMPs face several challenges in their development from early research through to clinical trials (CT) and further challenges in the assessment and approval, resulting on limited access across Europe.

    Many of these challenges stem from the small number of people who need these therapies, as this impacts the viability to run clinical trials, which in turns results in limited evidence available used to inform the approval of a new therapy.

    The relatively small scale of populations affected by the conditions these innovative treatments try to solve and the complexity of their development and manufacturing, impacts on the sustainability of the costs for these therapies. This is further compounded by a lack of transparency on pricing and no consistent methodology to demonstrate value between countries. Finally, even when an ATMP has been approved, it is still not feasible to make them available in all countries as you need a highly specialised centres who sees a critical mass of patients to maintain their expertise. This leads to inequities in access as there are also challenges to accessing these therapies in another country.

  6. What are the anticipated future trends and challenges for ATMPs in Europe?
    Future trends include the development of more targeted and personalized therapies, advancements in gene editing and regenerative medicine, and optimization of manufacturing processes. Challenges involve regulatory harmonization across EU countries, ensuring affordability and access, and addressing ethical issues related to genetic modifications. Ongoing innovation and collaboration will be one of the key factors for overcoming these challenges and unleashing the potential of ATMPs.

  7. What is JOIN4ATMP’s mission?
    The JOIN4ATMP project was born from the necessity to expand ATMP development opportunities in Europe, through the mapping of obstacles to such development, the audit of real-world-based solutions and the definition of new paths forward.

    Therefore, JOIN4ATMP aims at facilitating the translation of ATMPs from preclinical studies to the market and clinical use. Clinical, regulatory, and economic barriers preventing ATMP access will be identified, and recommendations for overcoming these obstacles will be provided, along with strategies for integrating different requirements across European countries.

Discover the project