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.
- 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
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.
__________________
| 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 | |
| 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 | ||
| Paediatric development |
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. | ||
| 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 |
Protocol assistance is a special form of scientific advice, reserved for medicines with an orphan designation | ||
| How to submit a request | Procedural advice for requesting 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
|
| 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 |
| 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 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 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
|
| 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
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
|
