Research and development

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The European Medicines Agency (EMA) provides guidance and support to medicine developers. This includes scientific and regulatory information on how to design and run clinical trials, compliance standards, and obligations and incentives for developers of specialised medicines.

United Kingdom’s (UK) withdrawal from the European Union (EU)

EMA has published important information to help users of the centralised procedure prepare for the expected consequences of 'Brexit', including their obligations related to establishment within the European Economic Area (EEA). For more information, see UK’s withdrawal from the EU.

In this section

 

Early development advice services

EMA has developed a consolidated list of available guidance and opportunities for interaction in the development phase of a medicinal product.

EMA offers medicine developers several opportunities for early dialogue and consultation before submitting a marketing authorisation application. This is intended to provide regulatory and scientific support to facilitate the preparation of applications and enable a smooth validation and assessment procedure. 

In addition to making use of these services, medicine developers can also:

Main procedural advice and guidance for medicine development

AreaGuidanceKey points
Scientific advice for human medicinesScientific Advice general informationScientific advice is advice to a medicine developer on the appropriate tests and studies in the development of a medicine
Guidance for applicants seeking scientific advice and protocol assistanceProtocol assistance is a special form of scientific advice, reserved for medicines with an orphan designation
How to submit a requestProcedural advice for requestin scientific advice and protocol assistance
Parallel EMA-United States (US) Food and Drug Administration (FDA) scientific adviceParallel EMA-FDA scientific advice should focus primarily on important breakthrough drugs or important safety issues
Parallel consultation with regulators and health technology assessment bodiesProvides feedback from regulators and HTA bodies at the same time, at any point in the developmental lifecycle of medicines.
Paediatric developmentGeneral informationDefinitions, links to submission templates and deadline, and further information on paediatric procedures
European Commission Guideline on PIP/waiver applicationsGuideline from the European Commission on the format and content of applications for agreement or modification of a paediatric investigation plan (PIP)/waiver/modification
Questions and answersProcedural advice for PIP/waiver/modification submissions
Orphan drug designationGeneral information and guidanceHow to apply, prevalence considerations, medical plausibility and significant benefit guidance
European Commission Guideline on orphan drug designation applicationsGuideline on the format and content of applications for designation as orphan product
Innovation task force (ITF)General informationIncludes the ITF mandate, guidance on how to apply for an ITF briefing meeting for human or veterinary development, and information on advanced therapy classification
PRIME scheme (PRIority MEdicines)PRIME general informationPRIME provides enhanced support for the development of medicines that target an unmet medical need, that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options
GuidanceEMA guidance on the scope and features of PRIME
Qualification of novel methodologiesGeneral informationScientific advice to support the qualification of innovative development methods (rather than a specific product) for a specific intended use in the context of research and development into pharmaceuticals
GuidanceProcedural advice

 

SME support

 

SME supportRegulatory and administrative assistance by phone, email or teleconference, including SME office briefing meetings to discuss the regulatory strategy of a human or veterinary medicinal product and navigate the range of procedures and incentives. Quarterly newsletters, info days and training
User Guide for SMEsProvides an overview of procedures to support research and development activities and regulatory requirements to obtain a marketing authorisation
Applying for SME statusIncludes the SME declaration form and guidance for enterprises applying for SME status before requesting financial or administrative assistance from the Agency

 

Opportunities to interact with EMA during the development of a medicine

AreaType of interactionKey points
Scientific advice for human medicinesPresubmission meeting for scientific advice or protocol assistanceAim: to facilitate validation and discuss the proposed questions. Submit a letter of intent to sa_submissions@ema.europa.eu approximately seven weeks in advance before the intended submission date
Paediatric developmentEarly paediatric interaction meetingAim: to discuss paediatric strategy very early in development. Send a request using the template, together with a letter of intent, at least two months in advance to paediatrics@ema.europa.eu. See also: regulatory Q&A
Presubmission meetingAim: to ensure smooth validation. Request a presubmission meeting with letter of intent to paediatrics@ema.europa.eu, at least two months prior to PIP/waiver submission. See also: regulatory Q&A
Clarification of the PDCO requests for modificationAim: to clarify any details of the PDCO's requests for modification, during clock-stop. Request to paediatrics@ema.europa.eu. See also: regulatory Q&A
Orphan drug designationPresubmission meetingAim: to ensure smooth validation. Request a presubmission meeting with an email to orphandrugs@ema.europa.eu, at least two months prior to submission of the orphan designation application 
ITFITF briefing meetingSend a request form to itfsecretariat@ema.europa.eu for human medicines (or vet.applications@ema.europa.eu for veterinary medicines)
PRIME schemeQueriesPresubmission meetings are not held for PRIME. Queries prior to submission of PRIME eligibility requests can be addressed to prime@ema.europa.eu
Qualification of novel methodologiesPreparatory meetingRequest a preparatory meeting sending the EMA letter of intent template or the joint EMA-FDA letter of intent template to qualification@ema.europa.eu

 

SME support

 

SME office briefing meeting   

Aim: to discuss the regulatory strategy of a product and navigate the range of procedures and incentives available to support development and authorisation. Contact the SME office on +44 (0)20 3660 8787 or sme@ema.europa.eu

 

 

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