Applicant

A pharmaceutical company or its agent that is submitting information in support of an application.

Applicant’s information

Regulatory information submitted by an applicant for, or to maintain, a marketing authorization that falls within the scope of this guidance document.

Cosmetic product

Substance to be brought into contact with the skin, hair, nails, lips, teeth, to clean them, perfume them, change their appearance, protect them, keep them in good condition or correct odors.

There is no marketing authorization for cosmetics, but manufacturers must ensure that their products pose no health hazard. Regulatory authorities often oversee the evaluation of the quality and safety of use of cosmetic products.

Firms constitute a technical dossier to keep available for regulatory authorities. This dossier must include the product formula, the conditions of manufacture and control, the evaluation of the safety for human health.

CP (Centralized Procedure)

One of the procedures by which a company can apply for a marketing authorization to the European Union. The application is made to the EMA. It is compulsory for biotechnology products, new active ingredients for cancer, neurodegenerative diseases, AIDS, Diabetes, and orphan drugs. The MA is automatically recognized and awarded by the member states.

See also MRP and DCP.

CTD (Common Technical Document)

Standardized format requested in Europe / USA / Japan for marketing authorization paper applications. It comprises 5 modules:

  • Module 1: Administrative information
  • Module 2: “Summary” of the last three modules
  • Module 3: Quality information: chemical properties (and / or biological) of the drug, its manufacture and control (stability, etc.).
  • Module 4: Preclinical information
  • Module 5: Clinical information

DCP (Decentralized Procedure)

One of the procedures by which a company can apply for a marketing authorization to the European Union. As the MRP, the DCP is based on recognition by national authorities of a first assessment performed by one Member State. The difference lies in that it applies to medicinal products which have not received a marketing authorization at the time of application.

An identical application for marketing authorization is submitted simultaneously to the competent authorities of the Reference Member State and of the Concerned Member States. See also MRP (Mutual Recognition Procedure), NP (National Procedure) and CP (Centralized Procedure).

Dossier

The Dossier contains all data submitted in support of the application for marketing authorization in accordance with the CTD (Common Technical Document) recommendations published by the European Commission.

Drug

A product is generally considered a drug in the following two cases (the definition can vary according to country specific regulation – check with your country regulation authority):

  • If it is presented as having properties for treating or preventing against human and animal diseases.
  • If it can be used in humans or animals for making a medical diagnosis or to restore, correct or modify physiological functions by exerting a pharmacological, immunological or metabolic action.

DTD (Document Type Definition)

Document that describes an XML document model. The DTD enables to automatically verify that the XML document is valid with respect to its formal definition provided by the DTD.

eCTD (electronic CTD)

Electronic submission in which the information submitted in support of the application complies with the eCTD structure and file formats. A submission is a sequence with a sequence number, which allows the management of changes in submission management. The eCTD dossier essentially consists of five elements:

  • A tree of folders respecting a standardized nomenclature and containing files mostly in PDF format
  • A table of contents called “backbone” containing:
    • an XML index for modules 2-5 of the CTD (generic modules)
    • a regional XML index of module 1 (Europe, USA and Japan)
  • A file to make an automatic verification of the dossier sequence integrity (MD5 hash file)
  • A folder named “util” which comprises a DTD file and a style sheet to verify that the XML file is syntactically correct, and to visualize it.

eCTD application

A collection of electronic documents compiled by a pharmaceutical company or its agent in compliance with European legislation and guidelines in order to seek a marketing authorization or any amendments thereof. An eCTD application may comprise a number of regulatory activities.

In the EU an eCTD application may comprise several dosage forms and strengths, all under one invented product name. This is understood to be equivalent to a Global Marketing Authorization according to Art. 6 para 2 Dir. 2001/83/EC as amended. Some review tools describe such a collection as a application (a term which will not be used in this document).

eCTD backbone files

These XML files (index.xml, us- regional.xml) are the main metadata files provided as part of an eCTD dossier, in addition to dossier documents. They contain for each document information such as title, subject (described by the title given in the table of contents for this document), relationship with other documents, version information , location, submission.

Electronic Submission

Submission in which the CTD information provided in support of the application is submitted in electronic form in accordance with the harmonized European recommendations for file formats and tables of contents.

EMA (European Medicines Agency)

This authority delivers marketing authorizations (MA) for medicines and health products for the European territory, for centralized procedures.

Envelope

Specific to the eCTD format, this dossier contains around ten pieces of structured information specific to the product and the application (full name, applicant, regulatory activity, type of application, procedure number, etc.).

EU NeeS (European Union Non eCTD electronic Submission )

Electronic submission in which the information provided in support of the application complies with the structure and file formats defined by EU NeeS. This type of application may be made only under certain conditions and otherwise must be made in eCTD format.

FDA (Food and Drug Administration)

The FDA is an agency of the United States Department of Health and Human Services. It is responsible for regulating food safety, tobacco products, dietary supplements, pharmaceutical drugs, vaccines, biopharmaceuticals, blood transfusions, medical devices and veterinary products. MAAs for medical devices and drugs for marketing in the United States are submitted to the FDA.

Finished Product

Product ready for the market.

ICH (International Conference on Harmonization … )

International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use. ICH International Conference aims to define the standards to be applied to the development of new drugs. It was created in 1990 by the regulatory authorities and pharmaceutical companies of the United States, the European Union and Japan. It has published numerous guidelines to facilitate the harmonization of marketing authorization procedures for medicines.

IND Application (Investigational New Drug Application)

It is made to apply to the FDA for approval to conduct clinical trials of a new drug. The FDA has 30 days to review the application. The IND may be for commercial purposes or purely research. It contains information about:

  • Preclinical studies (toxicological and pharmacological) made on the animal, and any studies that have been done on humans
  • Manufacturing information (composition, manufacturer, stability, quality control)
  • Clinical protocols, investigator qualifications and a series of commitments to comply with regulations

There are three types of INDs:

  • Investigator IND: submitted by a physician who both initiates and conducts the investigation, as well as directly conducts the administration of the drug
  • Emergency use IND: allows to use a drug in an emergency situations for which there is not enough time to submit the drug for a MAA
  • Treatment IND: allows the use of promising drugs with terminally ill patients or patients whose life is immediately threatened

Linked Modifications

When a main modification entails one or more other modifications, they are deemed to be linked. All these modifications put together form a single regulatory activity and the modifications are submitted in one dossier. The applicant should justify that the modifications are linked.

MAA (Market Authorization Application)

Submitting a Market Authorization Application is a mandatory prerequisite for the commercialization of a pharmaceutical product. In the USA, the license is issued by the FDA.

MD (Medical Device)

Any instrument, apparatus, appliance, material or other article, whether used alone or in combination, including the software necessary for its proper application intended by the manufacturer to be used for human beings for the purpose of:

  • Diagnosis, prevention, monitoring, treatment or alleviation of disease
  • Diagnosis, monitoring, treatment, alleviation of or compensation for an injury or handicap
  • Investigation, replacement or modification of the anatomy or of a physiological process
  • Control of conception

and which does not achieve its principal intended action in or on the human body by pharmacological, immunological or metabolic means, but which may be assisted in its function by such means (European Council Directive 93/42/EEC).

MRP (Mutual Recognition Procedure)

One of the procedures by which a company can apply for a marketing authorization to the European Union. This procedure is mandatory for any drug already having an MAA and to be marketed in more than one Member State. The principle is the recognition by the concerned member states (CMS) of the marketing authorization granted by the reference member state (RMS) chosen by the firm. See also DCP (Decentralized Procedure), NP (National procedure) and CP (Centralized Procedure).

NDA (New Drug Application )

A term used by the FDA to describe applications to market new drugs.

NeeS (Non eCTD electronic Submission)

Electronic submission for marketing authorization for which the data submitted meet the eCTD format, but for which the requirements are eased compared to the eCTD. In particular, the format does not include XML table of contents, but instead a table of contents as a PDF file.

NP (National procedure)

Procedure by which a company seeks a marketing authorization directly to the authorities of a member state of the EU. This mainly concerns known active ingredients. See also MRP (Mutual Recognition Procedure), DP (Decentralized Procedure) and CP (Centralized Procedure).

Paper submission

Submission in which the CTD information provided in support of the application is submitted in the form of paper documents.

PDF (Portable Document Format)

PDF is a document description language created by Adobe Systems. It helps preserve the formatting of the pages, regardless of the system used to view or print.

The PDF document format is used in particular for:

  • Printing by printing companies (ensuring WYSIWYG)
  • Archiving (using specific versions of the format)
  • Electronic submissions

Procedure

A Community registration procedure for the authorization of medicinal products in the European Community. There are 4 types of procedure that operate within the EC – Centralized, Decentralized, Mutual Recognition and National.

Regulatory activity

Object of the submission for a dossier, particularly a new marketing authorization application, type IA, IB or II variation, periodic safety update report (PSUR), renewal, or follow-up measure (as part of drug safety assessment after the marketing authorization is granted).

SaaS (Software as a Service)

Software as a Service is the concept of offering a software subscription rather than buying a license. This concept is also often associated (but not always) to software that is accessed remotely and online, generally using a Web browser. ASP (Application Service Provider) is a concept that is almost synonymous with SaaS.

Sequence

A single set of information and / or electronic documents submitted at one particular time by the applicant as a part of, or the complete, Application. Any collection of content assembled in accordance with the eCTD specification (ICH and EU) will be described using metadata as defined by the EU envelope.

This eCTD notion corresponds to a submission where the numbering is defined as follows: 0000 first submission 0001 second submission, etc. . Generally the 0000 sequence corresponds to the first MAA and the subsequent sequences to variation applications, PSURs renewal… Nevertheless, it is possible to adopt the eCTD format during the product life cycle, for example upon a major variation.

SPC (Summary of Product Characteristics)

It states the following information:

  • Drug name
  • Qualitative & quantitative composition
  • Pharmaceutical form
  • Clinical data: indications, dosage, cons-indications, precautions, interactions, adverse effects, overdose
  • Pharmacological properties: pharmacodynamics, pharmacokinetics, preclinical data security
  • Pharmaceutical data: excipients, incompatibility, conservation, packaging, instructions for use, handling and disposal
  • Holder of the authorization to use
  • Presentation and administrative ID
  • Date of first authorization / renewal of authorization
  • Text update date

STF (Study Tagging File)

This XML metadata file is a compulsory part of an eCTD submission dossier. It includes in particular information on the topics covered by some documents of the dossier, especially report files, which do not appear in the eCTD backbone submission files.

Submission

A submission corresponds to all (simultaneous) application(s) submitted by a company. The submission may concern several products.  The documentation common to all these applications is then submitted within the framework of a single submission.

Submission Type

The submission type describes the regulatory activity to which the content will be submitted.

Submission Unit

The submission unit element of the envelope metadata set describes the content at a lower level (a “sub-activity”) which is submitted in relation to a defined regulatory activity such as the initial submission, the applicant response to validation issues or list of questions or any other additional information.

Validation Report

The validation report is a document generated by the validator. It is one of the requested documents that must be attached to the electronic submission.

Validator

Tool for checking the conformity of the electronic submission with the criteria specified in the European guidelines in terms of structure, naming conventions, format and version of PDF file used.

vNeeS (veterinary Non eCTD electronic Submission)

Format of application for market authorization for veterinary products, close to the NeeS format. This format complies with the recommendations published by the TIGes-Vet.

XML (eXtensible Markup Language)

XML is a document description computer language. The goal of XML is to facilitate and standardize:

  • The representation of documents of all types
  • The automated exchange of content between computers

XML is an essential element of the interoperability of software and computer systems, even heterogeneous.

It uses tags delimited by chevrons, such as the <XML> tag. The XML syntax is extensible because it is possible to define new tags. An XML file is a hierarchical structure. The structure of an XML document can be verified using a DTD or more completely by using an XML schema.

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