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

• Adaptive pathways
• Advanced therapy medicines
• Clinical trials
• Compassionate use
• Compliance
• Data on medicines (ISO IDMP standards)
• Ethical use of animals in medicine testing
• Innovation in medicines
• Medicines for older people
• Non-pharmaceutical products
• Orphan designation: research and development
• Paediatric medicines: research and development
• Pharmacovigilance
• PRIME
• Quality by design
• Scientific advice and protocol assistance
• Scientific guidelines
• Supporting SMEs
• Support for early access

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, which is updated regularly.

User guide for micro, small and medium-sized enterprises

Adopted

Main procedural advice and guidance for medicine development

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

  For more information, see:

  • Scientific advice and protocol assistance
• How to submit a request:

  Procedural advice for requesting scientific advice and protocol assistance.

  For more information, see:

  • Requesting scientific advice or protocol assistance from EMA
• Parallel EMA-FDA scientific advice:

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

  For more information, see:

  General principles: European Medicines Agency-Food and Drug Administration parallel scientific advice
• Joint consultation:

  Parallel consultationwith 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.

  For more information, see:

  Guidance on parallel EMA / EUnetHTA 21 Joint Scientific Consultation

Paediatric development

• General information and guidance:

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

  For more information, see:

  • Paediatric investigation plans
• 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.

  For more information, see:

  • Paediatric investigation plans: questions and answers

Orphan drug designation

• General information and guidance:

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

  For more information, see:

  • Applying for orphan designation
• European Commission Guideline on orphan drug designation applications:

  For more information, see:

  • Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another

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.

  For more information, see:

  • PRIME: priority medicines
• Guidance:

  EMA guidance on the scope and features of PRIME.

  For more information, see:

  European Medicines Agency Guidance for applicants seeking access to PRIME scheme

Innovation task force (ITF)

• General information:

  Guidance on how to apply for an ITF briefing meeting for human or veterinary development, and information on advance therapy classification.

  For more information, see:

  Mandate of the European Medicines Agency Innovation Task Force (ITF)

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.

  For more information, see:

  • Opinions and letters of support on the qualification of novel methodologies for medicine development
• Guidance:

  Procedural advice.

  For more information, see:

  Qualification of novel methodologies for drug development: guidance to applicants

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.

  For more information, see:

  • Support to SMEs
• User guide for SMEs:

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

  For more information, see:

  User guide for micro, small and medium-sized enterprises
• 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.

  For more information, see:

  • Applying for SME status

Opportunities to interact with EMA during the development of a medicine

Scientific advice for human medicines

• Presubmission meeting for scientific advice or protocol assistance:

  Aim: to facilitate validation and discuss the proposed questions. Submit a meeting request to [e-mail] 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:

  • Paediatric investigation plans: questions and answers
• 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.

  For more information, see:

  • Paediatric investigation plans: questions and answers

Orphan drug designation

• Presubmission meeting:

  Aim: to ensure smooth validation. Request a pre-submission meeting via the IRIS portal, at least two months prior to submission of the orphan designation application

Innovation task force (ITF)

• ITF briefing meeting:

  Send a request form to [e-mail] for human medicines (or [e-mail] for veterinary medicines)

  For more information, see:

  Innovation Task Force (ITF) briefing meeting request form

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.

  For more information, see:

  • PRIME: priority medicines
• Pre-submission support and queries:

  EMA offers pre-submission support to any applicant planning a PRIME application, including a virtual pre-submission meeting to discuss PRIME eligibility. Applicants can submit these requests via IRIS.

  Queries prior to submission of PRIME eligibility requests can be sent to [e-mail].

Qualification of novel methodologies

• Preparatory meeting:

  Request a preparatory meeting sending the joint EMA-FDA letter of intent template to [e-mail]

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 [phone] or [e-mail]