The guideline on anticancer medicinal products as revised in early 2010 (rev.3)1 included disease specific guidance which was later expanded (rev. 4 published January 2013) to constitute a separate appendix (Appendix 4). The Appendix 4 was recently revised (rev 2 published February 2016). This concept paper describes and discusses the basis for the revision to the existing Appendix 4 in relation to the use of minimal residual disease (MRD) as a clinical endpoint in multiple myeloma (MM) clinical studies.
