Overcoming the challenges of clinical trials for cell and gene therapies

Requirements for clinical trials of gene and cell therapies

The following requirements apply to clinical trials of gene and cell therapies:

• Part IV of the EU GMP guidelines must be fulfilled
• A manufacturing authorization is required (starting in phase I)
• The responsible ethics committee must have examined the procedure and assessed it as ethically acceptable
  

Manufacturers can implement alternative approaches as long as they can prove that quality, safety, efficacy, and traceability of the product is ensured. Adjustments to the application of GMP requirements, particularly in the early stages of clinical development, are possible if they are justified in a risked-based manner. Find out more about the risk-based approach and the resulting flexibility in GMP manufacture here. 

Challenges in clinical trials with gene and cell therapeutics

Only small patient groups can be treated with novel gene and cell therapeutics. At the same time, many of these therapies offer therapeutic concepts for the treatment of rare diseases. Since only limited patient recruitment is possible, an orphan designation status can be applied for from the European Medicines Agency (EMA). This scenario must be considered when planning the pharmaceutical development of the of gene and cell therapies. This requires a guideline on clinical trials in small populations and clear specifications on biostatistics.

Despite all challenges, numerous gene and cell therapeutics as well as bioengineered tissue products have already been approved in the EU and the U.S. The number of gene and cell therapies in clinical trials is increasing continuously on a global scale.

Your benefits

In Germany, the Paul Ehrlich Institute (PEI) is responsible for reviewing clinical trials in the field of medicinal products, vaccines, and biomedical drugs. The PEI offers a demand-oriented, step-by-step consulting concept. We will gladly assist you in the preparation and follow-up of the PEI consultation. Besides we also support you with:

• Evaluating, planning, and setting up a clinical trial
• The initial application for approval of your study
• Quality-related issues for preclinical and clinical study phases regarding regulatory requirements
• Creating study documentation

• Organizing clinical studies
• Study logistics (concept for supplying the study centers) 

You can find more information about our service packages for clinical trials here.

Would you like to shape the future with gene and cell therapeutics? Our experienced GMP experts and scientists will be happy to support you during your clinical trial.

In addition to consulting services, we also offer a comprehensive service in investigational medicinal product management. Get in touch with us now. Together we will bring your therapy to the patient.