Regulatory assessment of approved rDNA-derived biotherapeutics, Annex 3, TRS No 999

Overview

This WHO guidance document considers the regulatory assessment needed to address situations where, for various reasons, biotherapeutic protein products prepared by recombinant deoxyribonucleic acid (DNA) technology (rDNAderived biotherapeutics) were licensed with data packages that do not follow current international regulatory standards for these biologicals. This includes, for example, biotherapeutic products licensed via a generic pathway or with limited analytical, nonclinical and/or clinical data. At its 2010 meeting in Singapore, the International Conference of Drug Regulatory Authorities (ICDRA) discussed such situations and requested WHO assistance in developing approaches for evaluating these already-licensed products in accordance with current WHO guidelines. In May 2014 the Sixty-seventh World Health Assembly adopted two relevant resolutions: one on promoting access to biotherapeutic products and ensuring their quality, safety and efficacy and the other on regulatory systems strengthening in which WHO was requested to provide guidance, especially on dealing with increasingly complex biological products.

Although primarily addressing rDNA-derived biotherapeutic protein products, some aspects of this document may also be relevant to other biotherapeutics.

Full version of the WHO Technical Report Series N° 999