Qualification of novel methodologies for medicine development

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.

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 Microsoft Office document iconletter 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.

Guidance for applicants

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

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

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

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

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

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

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

Qualification opinion - 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

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

Qualification opinion - ILSI / HESI submission of novel renal biomarkers for toxicity

Letters of support

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