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

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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

Treatment effect measures when using recurrent event endpoints

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

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

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

ILSI / HESI submission of novel renal biomarkers for toxicity

Letters of support


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