Research and development

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.

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 information

Scientific advice is advice to a medicine developer on the appropriate tests and studies in the development of a medicine

PDF icon Guidance for applicants seeking scientific advice and protocol assistance

Protocol assistance is a special form of scientific advice, reserved for medicines with an orphan designation

How to submit a request

Procedural advice for requestin scientific advice and protocol assistance

PDF icon Parallel EMA-United States (US) Food and Drug Administration (FDA) scientific advice

Parallel EMA-FDA scientific advice should focus primarily on important breakthrough drugs or important safety issues

PDF icon Parallel consultation with regulators and health technology assessment bodies

Provides feedback from regulators and HTA bodies at the same time, at any point in the developmental lifecycle of medicines.

Paediatric developmentGeneral information

Definitions, links to submission templates and deadline, and further information on paediatric procedures

European Commission Guideline on PIP/waiver applications

Guideline 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 answers

Procedural advice for PIP/waiver/modification submissions

Orphan drug designationGeneral information and guidance

How to apply, prevalence considerations, medical plausibility and significant benefit guidance

European Commission Guideline on orphan drug designation applications

Guideline on the format and content of applications for designation as orphan product

Innovation task force (ITF)General information

Includes the PDF icon 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 information

PRIME 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

PDF icon Guidance

EMA guidance on the scope and features of PRIME

Qualification of novel methodologiesGeneral information

Scientific 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

PDF icon Guidance Procedural advice

 

SME support

 

SME support

Regulatory 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

PDF icon User Guide for SMEs

Provides an overview of procedures to support research and development activities and regulatory requirements to obtain a marketing authorisation

Applying for SME status

Includes 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 meeting request to sa_submissions@ema.europa.eu approximately seven weeks in advance before the intended submission date
Paediatric development
Presubmission meeting

Aim: to ensure smooth validation. Submit your regulatory/administrative questions at least two months prior to PIP/waiver submission via the eSubmission Gateway. See also point 1.10 in: regulatory Q&A

Clarification of the PDCO requests for modification

Aim: to clarify any details of the PDCO’s requests for modification during clock-stop, contact the assigned Paediatric Coordinator. See also point 1.10 in: regulatory Q&A

Orphan drug designationPresubmission meeting

Aim: 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 meeting

Send a File request form to itfsecretariat@ema.europa.eu for human medicines (or vet.applications@ema.europa.eu for veterinary medicines)

PRIME schemeQueries

Presubmission 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 Microsoft Office document icon 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 +31 (0)88 781 8787 or sme@ema.europa.eu

 

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