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.

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:

• submit questions of a general nature using EMA's online enquiry form;
• consult a general overview of the main aspects of medicinal product legislation and of the European regulatory system in the User guide for micro-, small and medium-sized enterprises (SMEs ), which is updated regularly.

Main procedural advice and guidance for medicine development

Area	Guidance	Key points
Scientific advice for human medicines	Scientific Advice general information	Scientific 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 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
	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
	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 development	General 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 designation	General 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 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
	Guidance	EMA guidance on the scope and features of PRIME
Qualification of novel methodologies	General 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
	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
	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

Area	Type of interaction	Key points
Scientific advice for human medicines	Presubmission meeting for scientific advice or protocol assistance	Aim: to facilitate validation and discuss the proposed questions. Submit a 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. 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 modification	Aim: 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 designation	Presubmission 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
ITF	ITF briefing meeting	Send a request form to itfsecretariat@ema.europa.eu for human medicines (or vet.applications@ema.europa.eu for veterinary medicines)
PRIME scheme	Queries	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 methodologies	Preparatory meeting	Request a preparatory meeting sending the 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 +31 (0)88 781 8787 or sme@ema.europa.eu