Pilot Prequalification of Biotherapeutic Products

In recent years, a great number of biotherapeutic products (BTPs) have demonstrated success in treating many life-threatening chronic diseases. In May 2014, the World Health Assembly (WHA) adopted Resolution WHA67.21 on "Access to biotherapeutic products, including similar biotherapeutic products, and ensuring their quality, safety and efficacy".

BTPs, including their corresponding similar biotherapeutic product (SBPs), are highly complex biological medicines and the regulatory assessment of those products according to internationally acceptable guidelines and standards can be challenging in some countries. In order to explore options to facilitate access to safe, effective and quality assured BTPs and SBPs, WHO is launching a pilot project to prequalify selected BTPs and SBPs as a step forward to support national and global efforts to increase access to and the affordability of BTPs and their corresponding SBPs.

WHO has developed a pilot procedure for prequalification of BTPs. Currently, the procedure is limited to rituximab (used principally to treat non-Hodgkin's lymphoma and chronic lymphocytic leukemia), trastuzumab (for treating breast cancer) and human insulin (for treating diabetes).

General principles

The WHO pilot procedure  is based on the following principles and conditions:

  • The invitations for a submission of interest for prequalification of rituximab or trastuzumab BTPs or their corresponding SBPs, or for prequalification of human insulin BTPs or their corresponding SBPs are published on the WHO website.
  • It is the applicant's responsibility to monitor and investigate product complaints and promptly report these to WHO, as necessary or required.
  • It is the applicant's responsibility to promptly communicate to WHO any safety concerns arising from its product.
  • WHO monitors complaints and safety concerns notified by United Nations agencies and WHO Member States.
  • WHO has a general understanding of the applicant's production and quality control activities.
  • It is the applicant's responsibility to provide a WHO prequalification-specific addendum to the risk management plan (RMP) and to undertake pharmacovigilance and related activities according to the agreed RMP.
  • It is the applicant's responsibility to submit, following each marketing authorization, a summary on how the applicant has addressed, after product prequalification, any potential differences in healthcare settings, compared to stringent regulatory authorities (if applicable), that have required a revision of the adequacy of the safety concerns, pharmacovigilance activities, risk minimisation measures and/or traceability of the product.
  • WHO’s inspection of finished drug product (DP) and drug substance (DS) manufacturing site(s) for compliance with current good manufacturing practices (cGMPs) and current good distribution practices (cGDPs).
  • WHO’s inspection of clinical testing units or contract research organizations performing clinical trials for compliance with current good clinical practice (cGCP), current good laboratory practices (cGLPs).
  • It is the applicant’s responsibility to provide random sampling and testing of DS and DP if requested by WHO.