EMA Guideline on the clinical evaluation of Factor VIII products published

Recommendation

14/15 December 2023

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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.