Qualification of novel methodologies for medicine development: Letters of support

The European Medicines Agency (EMA) issues letters of support for promising novel methodologies that require further evidence before qualification.

EMA may propose a letter of support when it cannot yet qualify a novel methodology that addresses a relevant need in evidence generation and shows promise based on preliminary data. 

The letter of support encourages: 

• Further evidence generation

• Data-sharing

• Collaboration towards a possible future qualification

It is based on the CHMP's assessment of the methodology as part of a qualification advice request.

Find all letters of support in the expandable panels below, grouped into six categories.

For more information, see:

• Qualification of novel methodologies for medicine development

(Bio)markers

Select the expandable panels below to find letters of support on methodologies qualifying biological, imaging or digital markers.

Letters of support: (Bio)markers

Letter of Support for intermediate Age-Related Macular Degeneration (AMD) biomarker and novel clinical endpoint development

Reference Number: EMADOC-1700519818-1790091

Letter of Support for Braintale platforms

Reference Number: EMADOC-1700519818-982447

Letter of support for Neurofilament light in childhood neurological diseases

Reference Number: EMADOC-1700519818-959805

Letter of support for Mobilise-D digital mobility outcomes as monitoring biomarkers - Follow-up

Reference Number: EMA/CHMP/SAWP/277641/2021

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

Reference Number: EMA/508140/2020

Letter of support for Islet autoantibodies as enrichment biomarkers for type 1 diabetes prevention studies, through a quantitative disease progression model

Reference Number: EMA/625951/2019

Letter of support for Corrected T1 (cT1)

Reference Number: EMA/274962/2019

Letter of support for Magnetic Resonance Imaging Proton Density Fat Fraction (MRI-PDFF)

Reference Number: EMA/274960/2019

Letter of support for molecular imaging of the dopamine transporter biomarker as an enrichment biomarker for clinical trials for early Parkinson's disease

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

Reference Number: EMA/794518/2015 Summary:

On 14 September 2015 the Applicant EU-AIMS Consortium (IMI) requested follow-up qualification advice for methodology EEG to be used to stratify populations of people with Autism Spectrum Disorder (ASD) pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council.

Letter of support to explore MRI methodology to be used to stratify populations of people with autism spectrum disorder (ASD)

Reference Number: EMA/794542/2015 Summary:

On 14 September 2015 the Applicant EU-AIMS Consortium (IMI) requested follow-up qualification advice for MRI methodologies to be used to stratify populations of people with Autism Spectrum Disorder (ASD) pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council.

Letter of support for eye tracking to be used to stratify populations of people with autism spectrum disorder (ASD)

Reference Number: EMA/794527/2015 Summary:

On 14 September 2015 the Applicant EU-AIMS Consortium (IMI) requested follow-up qualification advice for Eye-tracking methodologies to be used to stratify populations of people with Autism Spectrum Disorder (ASD) pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council.

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

Reference Number: EMA/794534/2015 Summary:

On 14 September 2015 the Applicant EU-AIMS Consortium (IMI) requested follow-up qualification advice 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 pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council.

Letter of support for micro-aneurysm formation rate (MAFR) biomarker

Reference Number: EMA/775397/2014 Summary:

On 07 June 2013 the applicant Critical Health S.A. requested qualification of micro-aneurysm formation rate (MAFR) measured with a validated automated method, as an enrichment biomarker for studies of clinically significant macular oedema (CSMO) pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council.

Letter of support for drug-induced renal tubular injury biomarker(s)

Reference Number: EMA/715025/2016 Summary:

On 12 August 2016 the Applicant Firalis SAS (on behalf of Safer and Faster Evidence-based Translation (SAFE-T) consortium) requested follow up qualification advice for novel biomarkers to assess DIKI pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council. The procedure started during the SAWP meeting held on 30 August – 02 September 2016 and the advice was given in the framework of a joint EMA-FDA parallel advice procedure.

Letter of support for glutamate dehydrogenase, a biomarker of hepatocellular liver injury

Reference Number: EMA/666090/20177

Letter of support for skeletal muscle injury biomarkers

Reference Number: EMA/116308/2015

Letter of support for Model-based CT enrichment tool for CTs in aMCI

Reference Number: EMA/693969/2018

Outcome measures

Select the expandable panels below to find letters of support on methodologies qualifying instruments and endpoints that measure how a patient feels, functions or survives.

Letters of support: Outcome measures

Letter of support for Measurable Residual Disease (MRD) as a Surrogate Efficacy Endpoint in Clinical Studies with Acute Myeloid Leukemia

Reference Number: EMADOC-1700519818-1285629

Letter of Support for PRE score

Reference Number: EMADOC-1700519818-1207253

Letter of support for a composite endpoint method for acceptability evaluation of oral drug formulations in the paediatric population

Reference Number: EMADOC-1700519818-1181874

Letter of Support for TUMMY-UC – a Patient- and Observer-Reported Outcome for Paediatric Ulcerative Colitis (UC)

Reference Number: EMADOC-1700519818-1033762

Letter of support for the development of the PAH-SYMPACT Instrument for use in Pulmonary Hypertension clinical trials

Reference Number: EMADOC-1700519818-1054252

Letter of Support for an Acceptability Score Test in relative acceptability testing for oral medicines in children under 12 years of age

Reference Number: EMADOC-1700519818-1066960

Letter of support for Minimal Disease Activity Score (MDA) as primary outcome instrument for clinical studies in psoriatic arthritis (PsA)

Reference Number: EMADOC-1700519818-782278

Letter of support for Sjögren’s Tool for Assessing Response (STAR)

Reference Number: EMADOC-1700519818-746443

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

Reference Number: EMA/548136/2020

Letter of support for the development of Patient-Reported Outcomes tools for use as an endpoint in Inflammatory Bowel Disease (IBD) clinical trials

Reference Number: EMA/262838/2018

Letter of support for the development of a needs-based quality of life Patient Reported Outcome (PRO) measure specific to adults with plexiform neurofibromas

Reference Number: EMA/716127/2017 Summary:

On 1 March 2017 the Applicant Galen Research Ltd requested qualification opinion for a plexiform neurofibromas quality of life scale (the PlexiQoL), as biomarker for use as a secondary endpoint in clinical studies pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council.

Letter of support for reading speed and functional reading independence (FRI) index in geographic atrophy

Reference Number: EMA/14494/2016 Summary:

On 16 February 2015 the applicant Roche Registration Ltd requested scientific advice for use of reading speed and functional reading independence (FRI) index pursuant to Article 57(1)(n) of regulation (EC) 726/2004 of the European Parliament and of the Council.

Letter of support for Leuven Postprandial Distress Scale (LPDS) as PRO in Postprandial Distress Syndrome (PDS)

Reference Number: EMA/794574/2015

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

Reference Number: EMA/794518/2015 Summary:

On 14 September 2015 the Applicant EU-AIMS Consortium (IMI) requested follow-up qualification advice for methodology EEG to be used to stratify populations of people with Autism Spectrum Disorder (ASD) pursuant to Article 57(1)(n) of Regulation (EC) 726/2004 of the European Parliament and of the Council.

 

 

Data sources

Select the expandable panels below to find letters of support on methodologies qualifying real-world and study data infrastructures that are used to generate regulatory-grade evidence.

Letters of support: Data sources

Letter of Support for World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR)

Reference Number: EMADOC-1700519818-1173599

Letter of support for TREAT-NMD Core Dataset for Spinal Muscular Atrophy (SMA)

Reference Number: EMADOC-1700519818-958775

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)

Reference Number: EMADOC-1700519818-759902

Letter of support for International Niemann-Pick Disease Registry (INPDR)

Reference Number: EMA/206704/2021

Letter of support for Patient Data Platform for capturing patient-reported outcome measures for Dravet syndrome

Reference Number: EMA/327846/2016 Summary:

On 09 December 2015 the applicant Dravet Syndrome Foundation Spain requested qualification opinion for Patient Data Platform as an electronic tool for capturing patient reported outcomes in paediatric epilepsies, pursuant to article 57(1)(n) of regulation (EC) 726/2004 of the European Parliament and of the Council.

 

Methodologies for data analysis and decision support

Select the expandable panels below to find letters of support on methodologies for data analysis and decision support. These include approaches based on artificial intelligence (AI) and machine learning.

Letters of support: Methodologies for data analysis and decision support

Letter of support for Universal Immune System Simulator – Tuberculosis disease model (UISS-TB-DR)

Reference Number: EMADOC-1700519818-1207681

Letter of Support for the statistical adjustment on deep learning prognosis covariates obtained from histological slides

Reference Number: EMADOC-1700519818-1064889

Letter of support for a Model-based Clinical Trial Simulation Platform to Optimize Design of Efficacy Evaluation Studies in Parkinson’s Disease

Reference Number: EMADOC-1700519818-955717

Non-clinical methodologies

Select the expandable panels below to find the letters of support on the qualification of non-clinical methodologies. These include in-vitro and animal models.

Letters of support: Non-clinical methodologies

Letter of support for drug-induced vascular injury (DIVI) biomarker

Summary:

We are issuing this Letter of Support to SAFE”T and PSTC to encourage the further study of soluble biomarkers of endothelial cell injury and inflammation, and potential soluble markers of vascular smooth muscle cell injury, to monitor for drug”induced vascular injury (DIVI) in early clinical drug development.

Letter of Support for Forskolin-Induced Swelling (FIS) Assay: Biomarker for Cystic Fibrosis Transmembrane Conductance Regulator Protein (CFTR)

Reference Number: EMADOC-1700519818-1037383

Letter of support for Predictive Safety Testing Consortium translational drug-induced kidney injury biomarkers

Reference Number: EMA/651243/2014

Other letters of support

Select the expandable panels below to find letters of support on methodologies outside the categories listed above.

Other letters of support

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)

Reference Number: EMADOC-1700519818-746444

Letter of support for Master Protocol for Type 1 diabetes prevention studies in INNODIA

Reference Number: EMADOC-1700519818-703684

Retraction of the “Letter of support for drug -induced liver injury (DILI) biomarker” (EMA/423870/2016)

Reference Number: EMA/188045/20199

Page update history

An update log is available to show the date and summary of changes to this webpage. It does not include updates to linked documents or minor edits like typos or broken link fixes.

The tracking of updates begins in October 2026.

See update history

13 October 2026

• New page with letters of support of novel methodologies for medicine development
• The content on this page was transferred from the page 'Opinions and letters of support on the qualification of novel methodologies for medicine development' and organised per categories
• CHMP qualification opinions available in Qualification of novel methodologies for medicine development: CHMP qualification opinions