Opinions and letters of support on the qualification of novel methodologies for medicine development
Table of contents
- GFR slope as a Validated Surrogate Endpoint for RCT in CKD
- 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 .
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DRAFT qualification opinion for GFR slope as a Surrogate Endpoint in RCT for CKD (PDF/283.21 KB)
Draft
First published: 06/09/2023
EMA/SA/00000104642 -
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Briefing document - GFR slope as a Surrogate Endpoint in RCT for CKD (PDF/5.73 MB)
First published: 06/09/2023 -
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Responses to list of issues - GFR slope as a Surrogate Endpoint in RCT for CKD (PDF/1.13 MB)
First published: 06/09/2023
Last updated: 28/09/2023 -
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Presentation - GFR slope as a Validated Surrogate Endpoint for CKD in RCT (PDF/2.25 MB)
First published: 06/09/2023
Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy
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Qualification Opinion for Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy studies (PDF/3.83 MB)
First published: 31/07/2023
EMADOC-1700519818-1127132 -
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Overview of comments on draft Qualification Opinion for Stride velocity 95th centile as a primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy (PDF/479.16 KB)
First published: 31/07/2023 -
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Draft Qualification Opinion for Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy (PDF/3.88 MB)
Draft: consultation closed
First published: 01/03/2023
Consultation dates: 28/02/2023 to 10/04/2023 -
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Responses to list of issues - Stride velocity 95th centile as primary endpoint in studies in ambulatory Duchenne Muscular Dystrophy (PDF/1.2 MB)
First published: 01/03/2023
iBox Scoring System as a secondary efficacy endpoint in clinical trials investigating novel immunosuppressive medicines in kidney transplant patients
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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)
First published: 16/12/2022
EMADOC-1700519818-946771 -
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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)
First published: 16/12/2022 -
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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)
First published: 06/10/2022
EMADOC-1700519818-946771 -
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Qualification opinion - Appendix for Final Draft Qualification Opinion (PDF/883.98 KB)
First published: 06/10/2022
EMADOC-360526170-1156137 -
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Qualification opinion - C-PATH response to iBox Scoring System (Composite Biomarker Panel) - Second List of Issues (PDF/978.7 KB)
First published: 06/10/2022 -
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Qualification opinion - C-PATH response to iBox Scoring System (Composite Biomarker Panel) - List of Issues (PDF/1.25 MB)
First published: 06/10/2022 -
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Qualification opinion - Applicant revised briefing book for iBox Scoring System (Composite Biomarker Panel) (PDF/4.9 MB)
First published: 06/10/2022
EMADOC-1700519818-946771
Use of Enroll-HD (a Huntington’s disease patient registry) as a data source and infrastructure support for post-authorisation monitoring of medical products
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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)
Adopted
First published: 28/07/2022
EMADOC-1700519818-828910 -
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Submission of comments on Enroll-HD: Registry for Huntington's Disease (PDF/250.47 KB)
First published: 28/07/2022 -
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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)
Draft: consultation closed
First published: 23/02/2022
Consultation dates: 23/02/2022 to 11/04/2022
EMA/SA/0000056662 -
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Enroll-HD Registry for Huntington's Disease - Briefing Package - 1 of 2 - questions and answers and context of use (PDF/288.25 KB)
First published: 23/02/2022 -
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Enroll-HD Registry for Huntington's Disease - Briefing package - 2 of 2 - Supplement (PDF/192 KB)
First published: 23/02/2022 -
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Qualification Advice List of Issues: Enroll-HD Response (PDF/2.13 MB)
First published: 23/02/2022
EMA/SA/0000056662
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Qualification opinion for Prognostic Covariate Adjustment (PROCOVA™) (PDF/518.38 KB)
First published: 20/09/2022
EMADOC-1700519818-907465 -
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Overview of comments received for Prognostic Covariate Adjustment (PROCOVA™) with responses (PDF/550.78 KB)
First published: 20/09/2022 -
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Draft qualification opinion for prognostic covariate adjustment (PROCOVA™) (PDF/502.62 KB)
Draft: consultation closed
First published: 22/03/2022
Consultation dates: 22/03/2022 to 02/05/2022
Case No.: EMA/SA/0000059571 -
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Briefing book (PDF/646.89 KB)
First published: 22/03/2022
Version 2.0 -
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PROCOVA™ handbook (PDF/276.87 KB)
First published: 22/03/2022 -
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Response to list of issues (PDF/280.04 KB)
First published: 22/03/2022 -
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Response to 2nd list of issues (PDF/315.15 KB)
First published: 22/03/2022
Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials
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Qualification Opinion of Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials (PDF/1.88 MB)
Adopted
First published: 31/03/2022
EMA/CHMP/SAWP/186420/2022 -
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Overview of comments received on Draft qualification opinion on Islet Autoantibodies (AAs) with answers (PDF/171.28 KB)
First published: 31/03/2022 -
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Draft qualification opinion of Islet Autoantibodies (AAs) as Enrichment Biomarkers for Type 1 Diabetes (T1D) Prevention Clinical Trials (PDF/1.87 MB)
Draft: consultation closed
First published: 03/11/2021
Consultation dates: 03/11/2021 to 14/12/2021
EMA/580542/2021 -
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Islet Autoantibodies (AAs) - Briefing document (PDF/1.97 MB)
First published: 03/11/2021 -
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Islet Autoantibodies (AAs) - Written response to the list of issues (PDF/585.28 KB)
First published: 03/11/2021 -
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Islet Autoantibodies (AAs) - Appendix H (PDF/8.45 MB)
First published: 03/11/2021
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Qualification opinion on IMI PREFER (PDF/349.74 KB)
Adopted
First published: 03/05/2022
EMADOC-1700519818-808373 -
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Overview of comments received on draft qualification opinion on IMI PREFER (PDF/548.86 KB)
First published: 06/05/2022 -
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Draft qualification opinion on IMI PREFER (PDF/356.51 KB)
Draft: consultation closed
First published: 15/10/2021
Consultation dates: 15/10/2021 to 25/11/2021
EMADOC-1700519818-732587 -
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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)
First published: 18/10/2021
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Qualification opinion on Multiple sclerosis clinical outcome assessment (MSCOA) (PDF/1.27 MB)
Adopted
First published: 02/03/2020
EMA/CHMP/SAWP/74371/2020 -
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Overview of comments received on 'Draft qualification opinion on Multiple Sclerosis Clinical Outcome Assessment (MSCOA) qualification opinion' (PDF/648.88 KB)
First published: 02/03/2020
EMA/550514/2019 -
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Draft qualification opinion of Multiple sclerosis clinical outcome assessment (MSCOA) (PDF/1.08 MB)
Draft: consultation closed
First published: 19/06/2019
Last updated: 02/07/2019
Consultation dates: 18/06/2019 to 20/09/2019
EMA/CHMP/SAWP/336445/2019 Corr.1
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Qualification opinion of clinically interpretable treatment effect measures based on recurrent event endpoints that allow for efficient statistical analyses (PDF/1.38 MB)
Adopted
First published: 14/04/2020
EMA/CHMP/SAWP/120610/2020 -
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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)
First published: 14/04/2020
EMA/365402/2019 -
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Draft qualification opinion of clinically interpretable treatment effect measures based on recurrent event endpoints that allow for efficient statistical analyses (PDF/820.73 KB)
Draft: consultation closed
First published: 19/06/2019
Consultation dates: 19/06/2019 to 09/10/2019
EMA/CHMP/SAWP/291384/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's submission (PDF/887.79 KB)
First published: 19/06/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - First list of issues (PDF/230.69 KB)
First published: 19/06/2019
EMA/CHMP/SAWP/179482/2018 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to first list of issues (PDF/926.41 KB)
First published: 19/06/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Second list of issues (PDF/635.97 KB)
First published: 19/06/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to second list of issues (PDF/1.49 MB)
First published: 19/06/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Third list of issues (PDF/61.41 KB)
First published: 19/06/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to third list of issues (PDF/1.4 MB)
First published: 19/06/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Fourth list of issues (PDF/60.18 KB)
First published: 19/06/2019 -
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Qualification opinion - Treatment effect measures when using recurrent event endpoints - Applicant's responses to fourth list of issues (PDF/603.62 KB)
First published: 19/06/2019
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Qualification opinion on eSource Direct Data Capture (DDC) (PDF/1.23 MB)
Adopted
First published: 19/09/2019
EMA/CHMP/SAWP/483349/2019 -
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Overview of comments on the draft qualification opinion on eSource Direct Data Capture (DDC) (PDF/785.42 KB)
First published: 19/09/2019
EMA/179292/2019 -
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Draft qualification opinion on eSource Direct Data Capture (DDC) (PDF/415.85 KB)
Draft: consultation closed
First published: 15/11/2018
Consultation dates: 15/11/2018 to 14/03/2019
EMA/282576/2018
Stride velocity 95th centile as a secondary endpoint in Duchenne Muscular Dystrophy measured by a valid and suitable wearable device
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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)
Adopted
First published: 29/05/2019
EMA/CHMP/SAWP/178058/2019 -
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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)
First published: 29/05/2019
EMA/178057/2019 -
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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)
Draft: consultation closed
First published: 21/09/2018
Last updated: 21/09/2018
Consultation dates: 21/09/2018 to 30/11/2018
EMA/CHMP/SAWP/527447/2018
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Qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry (PDF/584.25 KB)
Adopted
First published: 28/02/2019
EMA/CHMP/SAWP/792574/2018 -
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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)
First published: 28/02/2019
EMA/618737/2018 -
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Draft qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry (PDF/693.58 KB)
Draft: consultation closed
First published: 29/06/2018
Last updated: 29/06/2018
Consultation dates: 29/06/2018 to 21/08/2018
EMA/CHMP/SAWP/423488/2018
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Qualification Opinion on The European Cystic Fibrosis Society Patient Registry (ECFSPR) and CF Pharmaco-epidemiology Studies (PDF/1.1 MB)
Adopted
First published: 03/10/2018
EMA/CHMP/SAWP/622564/2018 -
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Review of comments submitted on the draft Qualification opinion - The European Cystic Fibrosis Society Patient Registry (ECFSPR) (PDF/195.58 KB)
First published: 03/10/2018
EMA/482816/2018 -
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Qualification opinion - The European Cystic Fibrosis Society Patient Registry (ECFSPR) (PDF/875.42 KB)
Draft: consultation closed
First published: 09/02/2018
Last updated: 09/02/2018
Consultation dates: 09/02/2018 to 09/04/2018
EMA/CHMP/SAWP/802259/2017
Molecular neuroimaging of the dopamine transporter as biomarker to identify patients with early manifest Parkinsonism in Parkinson's disease
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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)
Adopted
First published: 19/07/2018
Last updated: 19/07/2018
EMA/CHMP/SAWP/765041/2017 -
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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)
First published: 19/07/2018
Last updated: 19/07/2018
EMA/9764/2018 -
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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)
Draft: consultation closed
First published: 24/01/2018
Last updated: 24/01/2018
Consultation dates: 24/01/2018 to 07/03/2018
EMA/765041/2017
Plasma fibrinogen as a prognostic biomarker (drug development tool) for all-cause mortality and COPD exacerbations in COPD subjects
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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)
Adopted
First published: 02/05/2018
Last updated: 02/05/2018
EMA/CHMP/SAWP/264260/2018 -
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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)
First published: 02/05/2018
Last updated: 02/05/2018
EMA/264099/2018 -
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Draft qualification opinion on plasma fibrinogen as a prognostic biomarker (drug development tool) for all-cause mortality and COPD exacerbations in COPD subjects (PDF/330.04 KB)
Draft: consultation closed
First published: 30/03/2017
Last updated: 30/03/2017
Consultation dates: 30/03/2017 to 03/05/2017
EMA/474186/2016
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Qualification opinion on Proactive in chronic obstructive pulmonary disease (COPD) (PDF/969.1 KB)
Adopted
First published: 19/04/2018
Last updated: 19/04/2018
EMA/CHMP/SAWP/226829/2018 -
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Overview of comments received on "Qualification opinion on Proactive in chronic obstructive pulmonary disease (COPD)" (PDF/80.41 KB)
First published: 19/04/2018
Last updated: 19/04/2018
EMA/225940/2018 -
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Draft qualification opinion on Proactive in chronic obstructive pulmonary disease (COPD) (PDF/1011.85 KB)
Draft: consultation closed
First published: 20/12/2017
Last updated: 20/12/2017
Consultation dates: 20/12/2017 to 29/01/2018
EMA/810227/2017
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Qualification opinion on paediatric ulcerative colitis activity index (PUCAI) (PDF/145.52 KB)
Adopted
First published: 20/01/2016
Last updated: 20/01/2016
EMA/CHMP/SAWP/801872/2015 -
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Overview of comments received on 'Qualification opinion on paediatric ulcerative colitis activity index (PUCAI)' (PDF/110.72 KB)
First published: 20/01/2016
Last updated: 20/01/2016
EMA/760013/2015 -
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Draft qualification opinion on the paediatric ulcerative colitis activity index (PUCAI) (PDF/149.37 KB)
Draft: consultation closed
First published: 18/09/2015
Last updated: 18/09/2015
Consultation dates: 18/09/2015 to 26/10/2015
EMA/CHMP/SAWP/485560/2015
Ingestible sensor system for medication adherence as biomarker for measuring patient adherence to medication in clinical trials
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Qualification opinion on ingestible sensor system for medication adherence as biomarker for measuring patient adherence to medication in clinical trials (PDF/1.08 MB)
Adopted
First published: 15/02/2016
Last updated: 15/02/2016
EMA/CHMP/SAWP/513571/2015 -
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Draft qualification opinion on the ingestible sensor system for medication adherence as biomarker for measuring patient adherence to medication in clinical trials (PDF/1.26 MB)
Draft: consultation closed
First published: 16/09/2015
Last updated: 16/09/2015
Consultation dates: 16/09/2015 to 26/10/2015
EMA/CHMP/SAWP/513571/2015
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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Qualification opinion - Total Kidney Volume (TKV) as a prognostic biomarker for use in clinical trials evaluating patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) (PDF/427.25 KB)
Adopted
First published: 13/11/2015
Last updated: 13/11/2015
EMA/CHMP/SAWP/473433/2015 -
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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)' (PDF/89.44 KB)
First published: 13/11/2015
Last updated: 13/11/2015
EMA/733775/2015 -
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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) (PDF/425.29 KB)
Draft: consultation closed
First published: 22/07/2015
Last updated: 22/07/2015
Consultation dates: 22/07/2015 to 02/10/2015
EMA/CHMP/SAWP/473433/2015 -
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Presentation - PKD presentation of outcomes: Consortium / European Medicines Agency Scientific Advice Working Party teleconference (Third list of issues) (PDF/576.73 KB)
First published: 22/07/2015
Last updated: 22/07/2015 -
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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) (PDF/4.5 MB)
First published: 22/07/2015
Last updated: 22/07/2015 -
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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... (PDF/904.91 KB)
First published: 22/07/2015
Last updated: 22/07/2015 -
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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) (PDF/210.16 KB)
First published: 22/07/2015
Last updated: 22/07/2015
EMA/CHMP/SAWP/298348/2014
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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Draft qualification opinion of qualification of exacerbations of chronic pulmonary disease tool (EXACT), and EXACT-respiratory symptoms measure (E-RS) for evaluating treatment outcomes in clinical trials in chronic pulmo... (PDF/739.37 KB)
Draft: consultation closed
First published: 13/04/2015
Last updated: 13/04/2015
Consultation dates: 13/04/2015 to 25/05/2015
EMA/CHMP/SAWP/178465/2015 -
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User manual E-RS (EXACT-respiratory symptoms): Applicant submission version 3.0 (PDF/1.17 MB)
First published: 13/04/2015
Last updated: 13/04/2015 -
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User manual (EXACT): Applicant submission version 7.0 (PDF/2 MB)
First published: 13/04/2015
Last updated: 13/04/2015
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Qualification opinion on in-vitro hollow-fibre-system model of tuberculosis (HFS-TB) (PDF/301.42 KB)
Adopted
First published: 06/02/2015
Last updated: 09/02/2015
EMA/CHMP/SAWP/47290/2015 -
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Draft qualification opinion on in vitro hollow-fibre-system model of tuberculosis (HFS-TB) (PDF/315.39 KB)
Draft: consultation closed
First published: 18/11/2014
Last updated: 18/11/2014
Consultation dates: 18/11/2014 to 09/01/2015
EMA/CHMP/SAWP/381716/2014 -
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Response to the European Medicines Agency list of issues - In vitro hollow-fibre-system model of tuberculosis (HFS-TB) (PDF/344.83 KB)
First published: 18/11/2014
Last updated: 18/11/2014 -
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Dossier submitted for qualification opinion - In vitro hollow-fibre-system model of tuberculosis (HFS-TB) (PDF/757.04 KB)
First published: 18/11/2014
Last updated: 18/11/2014
Consultation dates: 18/11/2014 to 09/01/2015 -
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Presentation for Drug Regimens Consortium (CPTR) scientific-advice meeting with the European Medicines Agency: Hollow-fiber system for tuberculosis (HFS-TB) (PDF/887.43 KB)
First published: 18/11/2014
Last updated: 18/11/2014
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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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 (PDF/228.82 KB)
Adopted
First published: 10/02/2014
Last updated: 10/02/2014
EMA/CHMP/SAWP/757052/2013 -
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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' (PDF/334.49 KB)
First published: 10/02/2014
Last updated: 10/02/2014
EMA/617236/2013 -
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Request for CHMP Qualification Opinion - Annex 1 (PDF/1.06 MB)
First published: 10/02/2014
Last updated: 10/02/2014 -
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Request for CHMP qualification opinion response to questions dated 11 June 2013 - Annex 2 (PDF/404.71 KB)
First published: 10/02/2014
Last updated: 10/02/2014 -
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Discussion meeting for MCP-Mod qualification opinion request - Annex 3 (PDF/745.39 KB)
First published: 10/02/2014
Last updated: 10/02/2014 -
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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 (PDF/400.47 KB)
Draft: consultation closed
First published: 15/10/2013
Last updated: 15/10/2013
Consultation dates: 15/10/2013 to 24/11/2013
EMA/CHMP/SAWP/592378/2013 -
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Request for CHMP qualification opinion on efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty (PDF/1.06 MB)
First published: 15/10/2013
Last updated: 15/10/2013 -
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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 (PDF/404.71 KB)
First published: 15/10/2013
Last updated: 15/10/2013 -
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Presentation for discussion meeting on efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty (PDF/745.39 KB)
First published: 15/10/2013
Last updated: 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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Qualification opinion of a novel data-driven model of disease progression and trial evaluation in mild and moderate Alzheimer’s disease (PDF/1.57 MB)
Adopted
First published: 03/10/2013
Last updated: 03/10/2013
EMA/CHMP/SAWP/567188/2013 -
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Overview of comments on 'qualification opinion of a novel data-driven model of disease progression and trial evaluation in mild and moderate Alzheimer’s disease' (PDF/79.55 KB)
First published: 03/10/2013
Last updated: 03/10/2013
EMA/471475/2013 -
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Draft qualification opinion of a novel data-driven model of disease progression and trial evaluation in mild and moderate Alzheimer’s disease (PDF/1.6 MB)
Draft: consultation closed
First published: 19/07/2013
Last updated: 19/07/2013
Consultation dates: 19/07/2013 to 27/08/2013
EMA/CHMP/SAWP/420174/2013
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
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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 ... (PDF/565.94 KB)
Adopted
First published: 04/04/2012
Last updated: 04/04/2012
EMA/CHMP/SAWP/893622/2011 -
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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... (PDF/134.24 KB)
First published: 04/04/2012
Last updated: 04/04/2012
EMA/922/2012 -
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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 / neg... (PDF/521.1 KB)
Draft: consultation closed
First published: 02/12/2011
Last updated: 02/12/2011
Consultation dates: 17/11/2011 to 22/12/2011
EMA/CHMP/SAWP/893622/2011 -
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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... (PDF/369.23 KB)
Adopted
First published: 04/04/2012
Last updated: 04/04/2012
EMA/CHMP/SAWP/892998/2011 -
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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 us... (PDF/142.14 KB)
First published: 04/04/2012
Last updated: 04/04/2012
EMA/991202/2011 -
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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... (PDF/281.54 KB)
Draft: consultation closed
First published: 02/12/2011
Last updated: 02/12/2011
Consultation dates: 17/11/2011 to 22/12/2011
EMA/CHMP/SAWP/892998/2011
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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Qualification opinion of low hippocampal volume (atrophy) by magnetic-resonance imaging for use in clinical trials for regulatory purpose in predementia stage of Alzheimer’s disease (PDF/664.89 KB)
Adopted
First published: 09/12/2011
Last updated: 09/12/2011
Legal effective date: 17/11/2011
EMA/CHMP/SAWP/809208/2011 -
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Overview of comments on 'qualification opinion of low hippocampal volume (atrophy) by magnetic-resonance imaging for use in clinical trials for regulatory purpose in predementia stage of Alzheimer’s disease' (PDF/164.88 KB)
First published: 09/12/2011
Last updated: 09/12/2011
EMA/879297/2011 -
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Qualification opinion of low hippocampal volume (atrophy) by magnetic-resonance imaging for use in regulatory clinical trials in predementia stage of Alzheimer’s disease (PDF/700.84 KB)
Draft: consultation closed
First published: 12/10/2011
Last updated: 12/10/2011
Consultation dates: 22/09/2011 to 01/11/2011
EMA/CHMP/SAWP/102001/2011
Novel methodologies in the predementia stage of Alzheimer's disease: cerebrospinal-fluid-related biomarkers for drugs affecting amyloid burden
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Qualification opinion of novel methodologies in the predementia stage of Alzheimer’s disease: cerebrospinal-fluid-related biomarkers for drugs affecting amyloid burden (PDF/418.09 KB)
Adopted
First published: 16/05/2011
Last updated: 16/05/2011
Legal effective date: 14/04/2011
EMA/CHMP/SAWP/102001/2011 -
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Overview of comments received on 'qualification opinion of novel methodologies in the predementia stage of Alzheimer’s disease: cerebrospinal-fluid-related biomarkers for drugs affecting amyloid burden' (PDF/351.91 KB)
First published: 16/05/2011
Last updated: 16/05/2011
EMA/252172/2011
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Qualification opinion ILSI / HESI submission of novel renal biomarkers for toxicity (PDF/1.25 MB)
Adopted
First published: 26/11/2010
Last updated: 26/11/2010
Legal effective date: 21/10/2010
EMA/CHMP/SAWP/283298/2010 -
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Overview of comments on qualification opinion ILSI / HESI submission of novel renal biomarkers for toxicity (PDF/149.75 KB)
First published: 26/11/2010
Last updated: 26/11/2010
Legal effective date: 21/10/2010
EMA/CHMP/SAWP/627644/2010 -
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Consultation on qualification opinion ILSI / HESI submission of novel renal biomarkers for toxicity (PDF/709.29 KB)
Draft: consultation closed
First published: 11/05/2010
Last updated: 07/06/2010
Consultation dates: 18/03/2010 to 31/07/2010
EMA/283298/2010
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Letter of Support for World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR) (PDF/338.11 KB)
First published: 16/10/2023
EMADOC-1700519818-1173599 -
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Letter of Support for Forskolin-Induced Swelling (FIS) Assay: Biomarker for Cystic Fibrosis Transmembrane Conductance Regulator Protein (CFTR) (PDF/230.56 KB)
First published: 23/06/2023
EMADOC-1700519818-1037383 -
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Letter of Support for TUMMY-UC – a Patient- and Observer-Reported Outcome for Paediatric Ulcerative Colitis (UC) (PDF/216.35 KB)
First published: 12/06/2023
EMADOC-1700519818-1033762 -
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Letter of support for the development of the PAH-SYMPACT Instrument for use in Pulmonary Hypertension clinical trials (PDF/147.76 KB)
First published: 03/04/2023
EMADOC-1700519818-1054252 -
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Letter of Support for the statistical adjustment on deep learning prognosis covariates obtained from histological slides (PDF/157.01 KB)
First published: 11/05/2023
EMADOC-1700519818-1064889 -
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Letter of support for Minimal Disease Activity Score (MDA) as primary outcome instrument for clinical studies in psoriatic arthritis (PsA) (PDF/254.36 KB)
First published: 09/03/2022
EMADOC-1700519818-782278 -
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Letter of Support for an Acceptability Score Test in relative acceptability testing for oral medicines in children under 12 years of age (PDF/147.59 KB)
First published: 03/05/2023
EMADOC-1700519818-1066960 -
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Letter of Support for Braintale platforms (PDF/138.68 KB)
First published: 26/01/2023
EMADOC-1700519818-982447 -
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Letter of Support of model-based clinical trial simulation platform (CTSP) for Duchenne Muscular Dystrophy (PDF/194.04 KB)
First published: 16/12/2022
EMADOC-1700519818-955047 -
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Letter of support for TREAT-NMD Core Dataset for Spinal Muscular Atrophy (SMA) (PDF/105.44 KB)
First published: 08/12/2022
EMADOC-1700519818-958775 -
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Letter of support for Neurofilament light in childhood neurological diseases (PDF/142.56 KB)
First published: 29/11/2022
EMADOC-1700519818-959805 -
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Letter of support for a Model-based Clinical Trial Simulation Platform to Optimize Design of Efficacy Evaluation Studies in Parkinson’s Disease (PDF/187.02 KB)
First published: 29/11/2022
EMADOC-1700519818-955717 -
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Letter of support for the Global Platform Study of Novel Medicines in Paediatric and Adolescent Relapsed and Refractory B-cell Non-Hodgkin Lymphoma (Glo-BNHL platform) (PDF/376.19 KB)
First published: 05/04/2022
EMADOC-1700519818-746444 -
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Letter of support for Sjögren’s Tool for Assessing Response (STAR) (PDF/176.18 KB)
First published: 17/03/2022
EMADOC-1700519818-746443 -
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Letter of support for intermediate Age-Related Macular Degeneration (AMD) biomarker and novel clinical endpoint development (PDF/126.38 KB)
First published: 22/02/2022
EMADOC-1700519818-776978 -
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Letter of Support for performing registry-based post authorisation safety studies (PASS) in Multiple Sclerosis (MS) using data of the Big MS Data Network (BMSD) (PDF/143.32 KB)
First published: 20/01/2022
EMADOC-1700519818-759902 -
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Letter of support for Master Protocol for Type 1 diabetes prevention studies in INNODIA (PDF/188.65 KB)
First published: 20/10/2021
EMADOC-1700519818-703684 -
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Letter of support for International Niemann-Pick Disease Registry (INPDR) (PDF/102.32 KB)
First published: 12/10/2021
EMA/206704/2021 -
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Letter of support for Mobilise-D digital mobility outcomes as monitoring biomarkers - Follow-up (PDF/167.5 KB)
First published: 11/06/2021
EMA/CHMP/SAWP/277641/2021 -
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Letter of support for Mobilise-D digital mobility outcomes as monitoring biomarkers (PDF/127.95 KB)
First published: 11/11/2020
EMA/234828/2020 -
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Letter of support for N170 ERP as a prognostic biomarker for adaptive social functioning and its potential to stratify study populations in people with Autism spectrum disorders (ASD) without intellectual disability (PDF/185.49 KB)
First published: 20/11/2020
EMA/508140/2020 -
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Letter of support for the VABS-II Adaptive Behavior Composite (VABS-II-ABC) score as measure of adaptive social functioning in people with Autism Spectrum Disorders (ASD) without intellectual disability (PDF/182.88 KB)
First published: 20/11/2020
EMA/548136/2020 -
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Letter of support for drug-induced renal tubular injury biomarker(s) (PDF/131.9 KB)
First published: 12/01/2017
Last updated: 12/01/2017
EMA/715025/2016 -
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Letter of support for Islet autoantibodies as enrichment biomarkers for type 1 diabetes prevention studies, through a quantitative disease progression model (PDF/186.28 KB)
First published: 30/03/2020
EMA/625951/2019 -
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Letter of support for Corrected T1 (cT1) (PDF/124.38 KB)
First published: 23/09/2019
EMA/274962/2019 -
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Letter of support for Magnetic Resonance Imaging Proton Density Fat Fraction (MRI-PDFF) (PDF/160.86 KB)
First published: 23/09/2019
EMA/274960/2019 -
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Retraction of the “Letter of support for drug -induced liver injury (DILI) biomarker” (EMA/423870/2016) (PDF/59.56 KB)
First published: 17/04/2019
EMA/188045/20199 -
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Letter of support for the development of Patient-Reported Outcomes tools for use as an endpoint in Inflammatory Bowel Disease (IBD) clinical trials (PDF/126.57 KB)
First published: 12/04/2019
EMA/262838/2018 -
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Letter of support for Model-based CT enrichment tool for CTs in aMCI (PDF/87.51 KB)
First published: 30/10/2018
EMA/693969/2018 -
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Letter of support for intermediate age related macular degeneration (AMD) biomarker and novel clinical endpoint development (PDF/129.15 KB)
First published: 21/02/2018
Last updated: 21/02/2018
EMA/72511/2018 -
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Letter of support for the development of a needs-based quality of life Patient Reported Outcome (PRO) measure specific to adults with plexiform neurofibromas (PDF/81.94 KB)
First published: 15/01/2018
Last updated: 15/01/2018
EMA/716127/2017 -
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Letter of support for glutamate dehydrogenase, a biomarker of hepatocellular liver injury (PDF/80.97 KB)
First published: 28/11/2017
Last updated: 28/11/2017
EMA/666090/20177 -
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Letter of support for drug-induced vascular injury (DIVI) biomarker (PDF/171.82 KB)
First published: 09/11/2017
Last updated: 09/11/2017 -
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Letter of support for molecular imaging of the dopamine transporter biomarker as an enrichment biomarker for clinical trials for early Parkinson's disease (PDF/79.84 KB)
First published: 07/10/2016
Last updated: 07/10/2016 -
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Letter of support for Patient Data Platform for capturing patient-reported outcome measures for Dravet syndrome (PDF/79.73 KB)
First published: 19/05/2016
Last updated: 19/05/2016
EMA/327846/2016 -
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Letter of support for reading speed and functional reading independence (FRI) index in geographic atrophy (PDF/84.7 KB)
First published: 28/01/2016
Last updated: 28/01/2016
EMA/14494/2016 -
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Letter of support for Leuven Postprandial Distress Scale (LPDS) as PRO in Postprandial Distress Syndrome (PDS) (PDF/72.61 KB)
First published: 15/12/2015
Last updated: 15/12/2015
EMA/794574/2015 -
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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 (PDF/97.41 KB)
First published: 09/12/2015
Last updated: 09/12/2015
EMA/794518/2015 -
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Letter of support to explore MRI methodology to be used to stratify populations of people with autism spectrum disorder (ASD) (PDF/82.87 KB)
First published: 09/12/2015
Last updated: 09/12/2015
EMA/794542/2015 -
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Letter of support for eye tracking to be used to stratify populations of people with autism spectrum disorder (ASD) (PDF/98.87 KB)
First published: 09/12/2015
Last updated: 09/12/2015
EMA/794527/2015 -
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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 (PDF/86.97 KB)
First published: 09/12/2015
Last updated: 09/12/2015
EMA/794534/2015 -
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Letter of support to explore clinical outcomes assessments utility to measure clinical symptoms in people with autism spectrum disorders (ASD) (PDF/98.82 KB)
First published: 09/12/2015
Last updated: 09/12/2015
EMA/794457/2015 -
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Letter of support for skeletal muscle injury biomarkers (PDF/77.24 KB)
First published: 19/03/2015
Last updated: 19/03/2015
EMA/116308/2015 -
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Letter of support for micro-aneurysm formation rate (MAFR) biomarker (PDF/68.87 KB)
First published: 21/01/2015
Last updated: 21/01/2015
EMA/775397/2014 -
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Letter of support for Predictive Safety Testing Consortium translational drug-induced kidney injury biomarkers (PDF/69.46 KB)
First published: 07/11/2014
Last updated: 07/11/2014
EMA/651243/2014
- Innovative Medicines Initiative (IMI) projects and regulators
src="/sites/default/files/youtube_logo.gif"> IMI webinar on regulatory acceptance mechanisms