Qualification of novel methodologies for medicine development

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The European Medicines Agency offers scientific advice to support the qualification of innovative development methods for a specific intended use in the context of research and development into pharmaceuticals.

The advice is given by the Committee for Medicinal Products for Human Use (CHMP) on the basis of recommendations by the Scientific Advice Working Party (SAWP). This qualification process leads to a CHMP qualification opinion or CHMP qualification advice.

CHMP qualification opinions

The CHMP can issue an opinion on the acceptability of a specific use of a method, such as the use of a novel methodology or an imaging method in the context of research and development. The method can apply to non-clinical or to clinical studies, such as the use of a novel biomarker.

The opinion is based on the assessment of data submitted to the Agency.

Before final adoption of qualification opinion, the CHMP makes its evaluation open for public consultation by the scientific community. This ensures that the CHMP shares information, as agreed with the applicant, and is open to scientific scrutiny and discussion.

CHMP qualification advice

The CHMP can issue advice on protocols and methods that are intended to develop a novel method with the aim of moving towards qualification.

The advice is based on the evaluation of the scientific rationale and on the preliminary data submitted to the Agency.

Update: Letter of intent

To facilitate parallel submissions of applications for drug biomarker qualification or clinical outcome assessment to EMA and to the United States Food and Drug Administration (FDA), the two agencies launched a joint letter of intent (LOI) in December 2014. 

The joint LOI allows the two agencies to share scientific perspectives and advice. The agencies are also able to provide the same response to submitters.

With the joint LOI, the agencies intend to reduce the time taken by applicants to prepare LOIs. However, applicants do not have to submit jointly to EMA and the FDA - they can send EMA or FDA-specific LOIs separately if they wish.

Some sections of the LOI are specific for EMA or the FDA. See the template for details.

Letters of support

Based on qualification advice, the Agency may propose a letter of support as an option, when the novel methodology under evaluation cannot yet be qualified but is shown to be promising based on preliminary data.

Letters of support aim to encourage data-sharing and to facilitate studies aimed at eventual qualification for the novel methodology under evaluation.

These letters include a high-level summary of the novel methodology, context of use, available data, and on-going and future investigations. The Agency publishes letters of support on this page, if the sponsors agree.

Table of contents


Guidance for applicants

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Paediatric ulcerative colitis activity index (PUCAI)

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Document(s) Language Status First published Last updated Effective Date
Overview of comments received on 'Qualification opinion on paediatric ulcerative colitis activity index (PUCAI)' (English only)   20/01/2016    
Qualification opinion on paediatric ulcerative colitis activity index (PUCAI) (English only) adopted 20/01/2016    
Draft qualification opinion on the paediatric ulcerative colitis activity index (PUCAI) (English only) draft: consultation closed 18/09/2015    

Ingestible sensor system for medication adherence as biomarker for measuring patient adherence to medication in clinical trials

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Total kidney volume (TKV) as a prognostic biomarker for use in clinical trials evaluating patients with autosomal dominant polycystic kidney disease (ADPKD)

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Document(s) Language Status First published Last updated Effective Date
Qualification opinion - Total Kidney Volume (TKV) as a prognostic biomarker for use in clinical trials evaluating patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) (English only) adopted 13/11/2015    
Overview of comments on 'Total kidney volume (TKV) as a prognostic biomarker for use in clinical trials evaluating patients with autosomal dominant polycystic kidney disease (ADPKD)' (English only)   13/11/2015    
Draft qualification opinion total kidney volume (TKV) as a prognostic biomarker for use in clinical trials evaluating patients with autosomal dominant polycystic kidney disease (ADPKD) (English only) draft: consultation closed 22/07/2015    
Presentation - PKD presentation of outcomes: Consortium / European Medicines Agency Scientific Advice Working Party teleconference (Third list of issues) (English only)   22/07/2015    
Final briefing book - Qualification of total kidney volume as a prognostic biomarker for use in clinical trials evaluating patients with autosomal dominant polycystic kidney disease (ADPKD) (English only)   22/07/2015    
PKDOC response to the European Medicines Agency third list of issues - Total kidney volume as a prognostic biomarker for use in clinical trials evaluating patients with autosomal dominant polycystic kidney disease (ADPKD) (English only)   22/07/2015    
Qualification opinion list of issues - Total kidney volume (TKV) as a prognostic biomarker for use in clinical trials evaluating patients with autosomal dominant polycystic kidney disease (ADPKD) (English only)   22/07/2015    

Qualification of exacerbations of chronic pulmonary disease tool (EXACT), and EXACT-respiratory symptoms measure (E-RS) for evaluating treatment outcomes in clinical trials in COPD

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In-vitro hollow fiber system model of tuberculosis (HFS-TB)

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MCP-Mod as an efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty

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Document(s) Language Status First published Last updated Effective Date
Qualification opinion of MCP-Mod as an efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty (English only) adopted 10/02/2014    
Overview of comments on the qualification opinion of MCP-Mod as an efficient statistical methodology for model-based design and analysis of Phase II dose finding studies under model uncertainty' (English only)   10/02/2014    
Request for CHMP Qualification Opinion - Annex 1 (English only)   10/02/2014    
Request for CHMP qualification opinion response to questions dated 11 June 2013 - Annex 2 (English only)   10/02/2014    
Discussion meeting for MCP-Mod qualification opinion request - Annex 3 (English only)   10/02/2014    
Draft qualification opinion of MCP-Mod as an efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty (English only) draft: consultation closed 15/10/2013    
Request for CHMP qualification opinion on efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty (English only)   15/10/2013    
Response to the questions raised by the qualification team - CHMP qualification opinion on efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty (English only)   15/10/2013    
Presentation for discussion meeting on efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty (English only)   15/10/2013    

A novel data-driven model of disease progression and trial evaluation in mild and moderate Alzheimer’s disease

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Alzheimer’s disease novel methodologies / biomarkers for the use of cerebrospinal-fluid amyloid beta 1-42 and t-tau and / or positron-emission-tomography amyloid imaging (positive / negative) as biomarkers for enrichment, for use in regulatory clinical trials in mild and moderate Alzheimer’s disease

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Document(s) Language Status First published Last updated Effective Date
Qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for the use of cerebrospinal-fluid amyloid beta 1-42 and t-tau and / or positron-emission-tomography amyloid imaging (positive / negative) as biomarkers for enrichment, for use in regulatory clinical trials in mild and moderate Alzheimer’s disease (English only) adopted 04/04/2012    
Overview of comments received on 'qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for the use of cerebrospinal-fluid amyloid beta 1-42 and t-tau and / or positron-emission-tomography amyloid imaging (positive / negative) as biomarkers for enrichment, for use in regulatory clinical trials in mild and moderate Alzheimer’s disease’ (English only)   04/04/2012    
Qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for the use of cerebrospinal-fluid amyloid beta 1-42 and t-tau signature and / or positron-emission-tomography amyloid imaging (positive / negative) as a biomarkers for enrichment for use in regulatory clinical trials in mild and moderate Alzheimer’s disease (English only) draft: consultation closed 02/12/2011    
Qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for positron-emission-tomography amyloid imaging (positive / negative) as a biomarker for enrichment, for use in regulatory clinical trials in predementia Alzheimer’s disease (English only) adopted 04/04/2012    
Overview of comments received on 'qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for positron-emission-tomography amyloid imaging (positive / negative) as a biomarker for enrichment, for use in regulatory clinical trials in predementia Alzheimer’s disease’ (English only)   04/04/2012    
Qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for positron-emission-tomograpy amyloid imaging (positive / negative) as a biomarker for enrichment for use in predementia Alzheimer's disease clinical trials (English only) draft: consultation closed 02/12/2011    

Low hippocampal volume (atrophy) by magnetic-resonance imaging for use in clinical trials for regulatory purpose in predementia stage of Alzheimer’s disease

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Novel methodologies in the predementia stage of Alzheimer’s disease: cerebrospinal-fluid-related biomarkers for drugs affecting amyloid burden

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Alzheimer’s disease novel methodologies / biomarkers for BMS-708163

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Document(s) Language Status First published Last updated Effective Date
Qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for BMS-708163 (English only) draft: consultation closed 10/02/2011    

Final conclusions on the pilot joint European Medicines Agency / Food and Drug Administration VXDS experience on qualification of nephrotoxicity biomarkers

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Document(s) Language Status First published Last updated Effective Date
Final conclusions on the pilot joint European Medicines Agency / Food and Drug Administration VXDS experience on qualification of nephrotoxicity biomarkers (English only)   22/01/2009    

ILSI / HESI submission of novel renal biomarkers for toxicity

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Document(s) Language Status First published Last updated Effective Date
Qualification opinion ILSI / HESI submission of novel renal biomarkers for toxicity (English only) adopted 26/11/2010   21/10/2010
Overview of comments on qualification opinion ILSI / HESI submission of novel renal biomarkers for toxicity (English only)   26/11/2010   21/10/2010
Consultation on qualification opinion ILSI / HESI submission of novel renal biomarkers for toxicity (English only) draft: consultation closed 11/05/2010 07/06/2010  

Letters of support

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Document(s) Language Status First published Last updated Effective Date
Letter of support for Patient Data Platform for capturing patient-reported outcome measures for Dravet syndrome (English only)   19/05/2016    
Letter of support for reading speed and functional reading independence (FRI) index in geographic atrophy (English only)   28/01/2016    
Letter of support for Leuven Postprandial Distress Scale (LPDS) as PRO in Postprandial Distress Syndrome (PDS) (English only)   15/12/2015    
Letter of support to explore EEG utility to measure deficits in social recognition in people with autism spectrum disorders (ASD) and its potential to stratify patient groups (English only)   09/12/2015    
Letter of support to explore MRI methodology to be used to stratify populations of people with autism spectrum disorder (ASD) (English only)   09/12/2015    
Letter of support for eye tracking to be used to stratify populations of people with autism spectrum disorder (ASD) (English only)   09/12/2015    
Letter of support for measures of executive function and basic emotions to be used to stratify populations of people with autism spectrum disorder (ASD) and predict clinical outcome (English only)   09/12/2015    
Letter of support to explore clinical outcomes assessments utility to measure clinical symptoms in people with autism spectrum disorders (ASD) (English only)   09/12/2015    
Letter of support for skeletal muscle injury biomarkers (English only)   19/03/2015    
Letter of support for micro-aneurysm formation rate (MAFR) biomarker (English only)   21/01/2015    
Letter of support for Predictive Safety Testing Consortium translational drug-induced kidney injury biomarkers (English only)   07/11/2014