The clinical development of Advanced Therapy Medicinal Products (ATMPs) can be a daunting task, but partnering with a team of experts can pave the way for success. In this blog, we highlight ten key considerations that can make the development process less risky and more efficient.

1) Addressing Variability in Starting Materials:

ATMP clinical trials often use donor or patient-based starting materials, leading to inherent variability. Defining release specifications for cell-based ATMPs must take this variability into account to ensure consistency and efficacy.

2) Optimizing Sampling Strategies:

Due to the scarcity of ATMP materials, retaining samples for reconfirmation may pose challenges. Developing a sampling strategy that aligns with product stability and shelf-life is essential to maintain quality assurance.

3) Adhering to Technical Requirements:

ATMP development involves bidirectional traceability and technical demands for coding, processing, preservation, storage, and distribution of human tissues and cells. Complying with these requirements ensures product safety and transparency.

4) Balancing Risks in Cell Extraction:

The process of taking biopsies or extracting cells may carry risks for the subject and impact the product's quality and safety. Careful consideration and mitigation strategies are necessary to safeguard both patients and product integrity.

5) Establishing Comprehensive Handling Procedures:

Detailed procedures for ATMP handling, disposal, preparation, and bedside administration are critical. Additionally, healthcare professionals should receive proper training to ensure safe and effective administration.

6) Complex Dose Determination:

Determining the appropriate ATMP dose and repeatability can be complex. Thorough research and scientific expertise are vital to achieve optimal treatment outcomes.

7) Accounting for Prior Treatments and Infections:

Previous or concomitant treatments and prior infections or vaccinations can significantly impact ATMP efficacy. A thorough assessment of these factors is essential for accurate clinical trial results.

8) Evaluating Long-Term Effects:

Considering potential long-lasting and/or irreversible effects on clinical trial subjects, their close contacts, and future pregnancies is crucial. Comprehensive risk analysis should be performed to ensure patient safety.

9) Implementing Long-Term Safety Follow-Ups:

Planning for long-term safety follow-ups after ATMP treatment is essential. This includes reporting and monitoring serious adverse reactions and events, with investigators well-trained in adverse event reporting.

10) Comprehensive Causality Assessment:

A differentiated causality assessment is necessary for each component of the ATMP, such as the cell-based and medical device components in combined ATMPs. Additionally, product administration procedures, concomitant medication, and product failure must be thoroughly evaluated.

By carefully considering these ten key aspects, the clinical development of ATMPs can become more manageable and successful. Collaboration with experienced experts and adherence to best practices will play a vital role in overcoming challenges and ensuring the safe and efficient advancement of ATMPs.