Opinions and letters of support on the qualification of novel methodologies for medicine development

Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy
iBox Scoring System as a secondary efficacy endpoint in clinical trials investigating novel immunosuppressive medicines in kidney transplant patients
Use of Enroll-HD (a Huntington’s disease patient registry) as a data source and infrastructure support for post-authorisation monitoring of medical products
Prognostic Covariate Adjustment (PROCOVA™)
Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials
IMI PREFER
Multiple sclerosis clinical outcome assessment (MSCOA)
Treatment effect measures when using recurrent event endpoints
eSource Direct Data Capture (DDC)
Stride velocity 95th centile as a secondary endpoint in Duchenne Muscular Dystrophy measured by a valid and suitable wearable device
Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry
The European Cystic Fibrosis Society Patient Registry (ECFSPR) and CF Pharmaco-epidemiology Studies
Molecular neuroimaging of the dopamine transporter as biomarker to identify patients with early manifest Parkinsonism in Parkinson's disease
Plasma fibrinogen as a prognostic biomarker (drug development tool) for all-cause mortality and COPD exacerbations in COPD subjects
Proactive in COPD
Paediatric ulcerative colitis activity index (PUCAI)
Ingestible sensor system for medication adherence as biomarker for measuring patient adherence to medication in clinical trials
Total kidney volume (TKV) as a prognostic biomarker for use in clinical trials evaluating patients with autosomal dominant polycystic kidney disease (ADPKD)
Exacerbations of chronic pulmonary disease tool (EXACT), and EXACT-respiratory symptoms measure (E-RS) for evaluating treatment outcomes in clinical trials in COPD
In-vitro hollow fiber system model of tuberculosis (HFS-TB)
MCP-Mod as an efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty
A novel data-driven model of disease progression and trial evaluation in mild and moderate Alzheimer's disease
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
Low hippocampal volume (atrophy) by magnetic-resonance imaging for use in clinical trials for regulatory purpose in predementia stage of Alzheimer's disease
Novel methodologies in the predementia stage of Alzheimer's disease: cerebrospinal-fluid-related biomarkers for drugs affecting amyloid burden
Alzheimer's disease novel methodologies / biomarkers for BMS-708163
ILSI / HESI submission of novel renal biomarkers for toxicity
Final conclusions on the pilot joint European Medicines Agency / Food and Drug Administration VXDS experience on qualification of nephrotoxicity biomarkers
Letters of support
External links

The European Medicines Agency (EMA) publishes opinions on the qualification of innovative development methods and letters of support for novel methodologies that have been shown to be promising in the context of research and development into pharmaceuticals.

The opinions are given by EMA's Committee for Medicinal Products for Human Use (CHMP) on the basis of recommendations by the Scientific Advice Working Party.

For more information and guidance on the qualification of novel methodologies, see Qualification of novel methodologies for medicine development .

Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy

iBox Scoring System as a secondary efficacy endpoint in clinical trials investigating novel immunosuppressive medicines in kidney transplant patients

Use of Enroll-HD (a Huntington’s disease patient registry) as a data source and infrastructure support for post-authorisation monitoring of medical products

Prognostic Covariate Adjustment (PROCOVA™)

Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials

IMI PREFER

Multiple sclerosis clinical outcome assessment (MSCOA)

Treatment effect measures when using recurrent event endpoints

eSource Direct Data Capture (DDC)

Stride velocity 95th centile as a secondary endpoint in Duchenne Muscular Dystrophy measured by a valid and suitable wearable device

Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry

The European Cystic Fibrosis Society Patient Registry (ECFSPR) and CF Pharmaco-epidemiology Studies

Molecular neuroimaging of the dopamine transporter as biomarker to identify patients with early manifest Parkinsonism in Parkinson's disease

Plasma fibrinogen as a prognostic biomarker (drug development tool) for all-cause mortality and COPD exacerbations in COPD subjects

Proactive in COPD

Paediatric ulcerative colitis activity index (PUCAI)

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

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

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

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

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

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

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

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

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

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

List item

Qualification opinion of Alzheimer’s disease novel methodologies / biomarkers for BMS-708163 (PDF/183.55 KB)

Draft: consultation closed

First published: 10/02/2011
Last updated: 10/02/2011
Consultation dates: 10/02/2011 to 25/03/2011
EMA/CHMP/SAWP/102001/2011

ILSI / HESI submission of novel renal biomarkers for toxicity

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

List item

Final conclusions on the pilot joint European Medicines Agency / Food and Drug Administration VXDS experience on qualification of nephrotoxicity biomarkers (PDF/169.77 KB)

First published: 22/01/2009
Last updated: 22/01/2009
EMEA/679719/2008 Rev. 1

Letters of support

External links

Topics

Opinions and letters of support on the qualification of novel methodologies for medicine development

Table of contents

Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy

Draft Qualification Opinion for Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy (PDF/3.88 MB)

Responses to list of issues - Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy (PDF/1.2 MB)

iBox Scoring System as a secondary efficacy endpoint in clinical trials investigating novel immunosuppressive medicines in kidney transplant patients

Qualification opinion for the iBox Scoring System as a secondary efficacy endpoint in clinical trials investigating novel immunosuppressive medicines in kidney transplant patients (PDF/267.77 KB)

Overview of comments received on Draft Qualification Opinion of the iBox Scoring System as a secondary efficacy endpoint in clinical trials investigating novel immunosuppressive medicines in kidney transplant patients (PDF/92.13 KB)

DRAFT Qualification opinion for the iBox Scoring System as a secondary efficacy endpoint in clinical trials investigating novel immunosuppressive medicines in kidney transplant patients (PDF/395.33 KB)

Qualification opinion - Appendix for Final Draft Qualification Opinion (PDF/883.98 KB)

Qualification opinion - C-PATH response to iBox Scoring System (Composite Biomarker Panel) - Second List of Issues (PDF/978.7 KB)

Qualification opinion - C-PATH response to iBox Scoring System (Composite Biomarker Panel) - List of Issues (PDF/1.25 MB)

Qualification opinion - Applicant revised briefing book for iBox Scoring System (Composite Biomarker Panel) (PDF/4.9 MB)

Use of Enroll-HD (a Huntington’s disease patient registry) as a data source and infrastructure support for post-authorisation monitoring of medical products

Qualification opinion of the use of Enroll-HD (a Huntington’s disease patient registry) as a data source and infrastructure support for post-authorisation monitoring of medical products (PDF/451.18 KB)

Submission of comments on Enroll-HD: Registry for Huntington's Disease (PDF/250.47 KB)

Draft qualification opinion for the use of Enroll-HD (a Huntington’s disease patient registry) as a data source and infrastructure support for post-authorisation monitoring of medical products (PDF/357.31 KB)

Enroll-HD Registry for Huntington's Disease - Briefing Package - 1 of 2 - questions and answers and context of use (PDF/288.25 KB)

Enroll-HD Registry for Huntington's Disease - Briefing package - 2 of 2 - Supplement (PDF/192 KB)

Qualification Advice List of Issues: Enroll-HD Response (PDF/2.13 MB)

Prognostic Covariate Adjustment (PROCOVA™)

Qualification opinion for Prognostic Covariate Adjustment (PROCOVA™) (PDF/518.38 KB)

Overview of comments received for Prognostic Covariate Adjustment (PROCOVA™) with responses (PDF/550.78 KB)

Draft qualification opinion for prognostic covariate adjustment (PROCOVA™) (PDF/502.62 KB)

Briefing book (PDF/646.89 KB)

PROCOVA™ handbook (PDF/276.87 KB)

Response to list of issues (PDF/280.04 KB)

Response to 2nd list of issues (PDF/315.15 KB)

Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials

Qualification Opinion of Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials (PDF/1.88 MB)

Overview of comments received on Draft qualification opinion on Islet Autoantibodies (AAs) with answers (PDF/171.28 KB)

Draft qualification opinion of Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials (PDF/1.87 MB)

Islet Autoantibodies (AAs) - Briefing document (PDF/1.97 MB)

Islet Autoantibodies (AAs) - Written response to the list of issues (PDF/585.28 KB)

Islet Autoantibodies (AAs) - Appendix H (PDF/8.45 MB)

IMI PREFER

Qualification opinion on IMI PREFER (PDF/349.74 KB)

Overview of comments received on draft qualification opinion on IMI PREFER (PDF/548.86 KB)

Draft qualification opinion on IMI PREFER (PDF/356.51 KB)

CHMP & EUnetHTA parallel Scientific Advice: Qualification of a Framework & Points to consider for method selection with methods for performing patient preference studies to inform regulatory & HTAbody medical product decision-making: Briefing document (PDF/3.37 MB)

Multiple sclerosis clinical outcome assessment (MSCOA)

Qualification opinion on Multiple sclerosis clinical outcome assessment (MSCOA) (PDF/1.27 MB)

Overview of comments received on 'Draft qualification opinion on Multiple Sclerosis Clinical Outcome Assessment (MSCOA) qualification opinion' (PDF/648.88 KB)

Draft qualification opinion of Multiple sclerosis clinical outcome assessment (MSCOA) (PDF/1.08 MB)

Treatment effect measures when using recurrent event endpoints

Qualification opinion of clinically interpretable treatment effect measures based on recurrent event endpoints that allow for efficient statistical analyses (PDF/1.38 MB)

Overview of comments on the draft qualification opinion of clinically interpretable treatment effect measures based on recurrent event endpoints that allow for efficient statistical analyses (PDF/579.82 KB)

Draft qualification opinion of clinically interpretable treatment effect measures based on recurrent event endpoints that allow for efficient statistical analyses (PDF/820.73 KB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's submission (PDF/887.79 KB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - First list of issues (PDF/230.69 KB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to first list of issues (PDF/926.41 KB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Second list of issues (PDF/635.97 KB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to second list of issues (PDF/1.49 MB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Third list of issues (PDF/61.41 KB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to third list of issues (PDF/1.4 MB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Fourth list of issues (PDF/60.18 KB)

Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to fourth list of issues (PDF/603.62 KB)

eSource Direct Data Capture (DDC)

Qualification opinion on eSource Direct Data Capture (DDC) (PDF/1.23 MB)

Overview of comments on the draft qualification opinion on eSource Direct Data Capture (DDC) (PDF/785.42 KB)

Draft qualification opinion on eSource Direct Data Capture (DDC) (PDF/415.85 KB)

Stride velocity 95th centile as a secondary endpoint in Duchenne Muscular Dystrophy measured by a valid and suitable wearable device

Qualification opinion on stride velocity 95th centile as a secondary endpoint in Duchenne Muscular Dystrophy measured by a valid and suitable wearable device (PDF/2.72 MB)

Overview of comments on 'Stride velocity 95th centile as a secondary endpoint in Duchenne Muscular Dystrophy measured by a valid and suitable wearable device' (EMA/532515/2018) (PDF/316.92 KB)

Draft qualification opinion on stride velocity 95th centile as a secondary endpoint in Duchenne Muscular Dystrophy measured by a valid and suitable wearable device (PDF/2.49 MB)

Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry

Qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry (PDF/584.25 KB)

Overview of comments received on 'Draft qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry' (EMA/CHMP/SAWP/423488/2018) (PDF/498.42 KB)

Draft qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry (PDF/693.58 KB)

The European Cystic Fibrosis Society Patient Registry (ECFSPR) and CF Pharmaco-epidemiology Studies

Qualification Opinion on The European Cystic Fibrosis Society Patient Registry (ECFSPR) and CF Pharmaco-epidemiology Studies (PDF/1.1 MB)

Review of comments submitted on the draft Qualification opinion - The European Cystic Fibrosis Society Patient Registry (ECFSPR) (PDF/195.58 KB)

Qualification opinion - The European Cystic Fibrosis Society Patient Registry (ECFSPR) (PDF/875.42 KB)

Molecular neuroimaging of the dopamine transporter as biomarker to identify patients with early manifest Parkinsonism in Parkinson's disease

Qualification opinion on dopamine transporter imaging as an enrichment biomarker for Parkinson’s disease clinical trials in patients with early Parkinsonian symptoms (PDF/762.14 KB)

Overview of comments on 'Draft qualification opinion on molecular neuroimaging of the dopamine transporter as biomarker to identify patients with early manifest Parkinsonism in Parkinson’s disease' (PDF/297.59 KB)

Draft qualification opinion on molecular neuroimaging of the dopamine transporter as biomarker to identify patients with early manifest Parkinsonism in Parkinson’s disease (PDF/723.8 KB)

Plasma fibrinogen as a prognostic biomarker (drug development tool) for all-cause mortality and COPD exacerbations in COPD subjects

Qualification opinion on plasma fibrinogen as a prognostic biomarker (drug development tool) for all-cause mortality and COPD exacerbations in COPD subjects (PDF/293.22 KB)

Overview of comments on 'Draft qualification opinion on plasma fibrinogen as a prognostic biomarker (drug development tool) for all-cause mortality and COPD exacerbations in COPD subjects' (PDF/76.34 KB)