EMA Guideline on the clinical evaluation of Factor VIII products published

Recommendation

9/10 May 2023
Heidelberg, Germany

Annex 2 & Co. - GMP Compliance for Biopharmaceuticals
Regulatory Requirements and Practical Implementation

On 28 January, the committee for medicinal products for human use (CHMP) adopted the Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products. It will come into effect on 1 May 2016.

In this document, the applicant as well as the regulator can find advice, which information should be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor VIII products is made for use in treatment and prevention of bleeding in patients with haemophilia A. The current document covers clinical investigations to be conducted pre- and post-marketing authorisation. It also covers authorised products where a significant change in the manufacturing process has been made. Amongst a brief summary and the scope, the guideline document includes the following content:

3. Legal basis
4. Efficacy: General aspects
5. Safety: General aspects 
5.1. Adverse events
5.2. Safety with respect to viruses and other transmissible agents
5.3. Immunogenicity
6. Application for marketing authorisation: "New products"
6.1. General aspects on clinical trials
6.1.1. Potency measurements
6.2. Efficacy in PTPs ?12 years
6.3. Clinical investigation in PTPs ?12 years
6.4. Clinical investigation in children <12 years
6.5. Clinical investigation in PUPs
6.6. Post-marketing investigation
7. Change in the manufacturing process
7.1. General aspects on clinical trials
7.2. Efficacy
8. Risk management plan
References
Annex I - Overview on clinical trial concept18
Annex II - Clinical trials with factor VIII products: new products
Annex III - Post-marketing investigation

"This guidance introduces general principles on efficacy and safety in chapters 4 and 5. Information on the clinical development concept is included in subsequent chapters regarding “new products” and significant changes of the manufacturing process. Detailed “at a glance” requirements for clinical trials for factor VIII products are found in Annexes I to III."

Read more details in the Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products.