Personalised Medicine
EMA Biomarker Qualification procedure. This formal procedure not only offers companies a route for gain- ing regulatory acceptance of their biomarker, but also encompasses a broader scope for obtaining regulatory advice on additional novel methodologies, including imaging methods or other drug development tools. A Qualification Opinion can be gained on the acceptability of a biomarker in a research and development context (non-clinical or clinical stud- ies), based on the data package submitted. If the amount of data is not sufficient to achieve the for- mal Opinion, a Qualification Advice based on the evaluation of the scientific rationale and prelimi- nary data submitted, identifying gaps that need to be filled to gain the formal Opinion is provided. The specialised group (Qualification team) appointed by the EMA is composed of selected key opinion leaders from the EMA experts’ network. The procedure is highly interactive and involves several opportunities for both teleconferences and face-to-face meetings throughout the procedure. Prior to adoption of the Qualification Opinion a public consultation step is implemented for a set time period of six weeks to allow the scientific com- munity to give their views. It should be noted that the content for release to the public domain will be agreed with the company prior to publication. The Qualification Advice and Opinion route can take approximately 100 and 190 days, respective- ly. As more companies go through this procedure, since its introduction in January 2009, the outlined timetable may be modified depending on the data packages submitted.
Guidance on the structure and content of the data package required is outlined in the document EMEA/CHMP/SAWP/72894/2008. ICH E16 also provides some useful guidance and structure appli- cable to the generation of biomarker data intended to support qualification. In brief, the following keys pieces of information are needed:
lDisease setting associated with your biomarker(s) l Intended use of biomarker, its need and impact, how it will be integrated into drug development and regulatory review l Relevance and adequacy to extrapolate the pre- clinical models to clinical setting l Details on study design, critical analysis of results, assay validation, statistical plans l Inclusion of as much supportive data to strengthen the package, such as systematic litera- ture reviews, meta-analysis, study reports l Gaps (if any, should you only wish to opt for Qualification Advice) that remain and how these will be addressed in future plans/studies.
Drug Discovery World Summer 2010
Prior to entering these Qualification procedures, there are other opportunities to engage with the European regulatory authorities. Advice and input from such groups as the Innovation Task Force and/or the Pharmacogenomic Working Group is often recommended and indeed encouraged prior to entering the EMA Qualification procedures. The FDA also has a biomarker qualification mechanism, although currently it is still at the pilot stage and is less structured compared to the EMA procedure. The procedure is termed a Voluntary Exploratory Data Submission (VXDS) and involves the submission of an information dossier to the Interdisciplinary Pharmacogenomics Review Group (IPRG) which forms a tailored Biomarker Qualification Review Team (BQRT). VXDSs can be submitted as a stand-alone submission in which case a new pre-IND number is issued or they can be associated with a pre-existing IND in which case no new number is issued and the status of the current IND is not affected.
In principle a VXDS is similar to the EMA qualifi- cation procedure in that a dossier containing studies supporting the use of the biomarker are submitted to the regulatory agency which then provides an opin- ion and/or advice. The key differences are that the FDA procedure does not have formal timelines and currently there is no scope for public consultation.
Figure 5: Summary steps involved in the EMA Biomarker Qualification Procedure
Dossier submission,
review and questions to the Applicant
Discussion of draft Qualification Opinion
Meeting with Applicant and finalisation of report
Public Consultation
Review and adoption of Qualification Advice
Adoption of Qualification Opinion
Source: ERA Consulting
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