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Well-established drugs – the procedure  of well established use

Publication date: January 21, 2026

Every drug introduced to the EU market must obtain a permit from the competent authority. In Poland, this is the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products. EU regulations included in Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use, implemented into Polish law by the Act of 6 September 2001 – Pharmaceutical Law provide for several procedures enabling drug registration, one of which is the so-called well-established medicine procedure (WEU). This is a special procedure for obtaining marketing authorization for medicines containing active substances with well-established medical use, i.e., medicinal substances known and systematically used in the EU for at least 10 years. The most important difference with this procedure is that there is no need to submit new clinical and pre-clinical studies, and the application can be based on available scientific studies that confirm the effectiveness and safety of the substance.

Most often, this procedure applies to herbal medicines and medicines containing commonly used substances that were already in use before the introduction of the drug registration requirement – examples include medicines containing paracetamol or aspirin.

Administrative and documentation requirements

Article 10 paragraph 1 of the Pharmaceutical Law governs the general requirements of an application for marketing authorization for a medicinal product, which should include:

  1. name and address of the responsible entity, manufacturer or importer;
  2. name of the medicinal product;
  3. detailed quantitative and qualitative data of the active substance or active substances and other substances relating to the medicinal product and their common names, and in their absence – chemical names;
  4. the pharmaceutical form, strength and route of administration and shelf life of the medicinal product, as well as environmental data related to the destruction of the medicinal product, if necessary and resulting from the properties of the product.

Pursuant to paragraph 2, the application shall be accompanied by:

  1. description of the manufacture of the medicinal product;
  2. description of the control methods used in the manufacturing process;
  3. written confirmation by the manufacturer of the medicinal product that it has verified, through an audit, compliance with the requirements of Good Manufacturing Practice by the manufacturer of the active substance at the place where it conducts its manufacturing activities;
  4. information on special requirements for the storage of the medicinal product, its dispensing to patients and the disposal of the expired product, together with an assessment of the environmental risk associated with the medicinal product and a description of methods to reduce this risk;
  5. results, summaries and research reports:
    1. pharmaceutical: physicochemical, biological or microbiological,
    1. non-clinical: pharmacological and toxicological,
    1. clinical

– together with a quality overall summary, a non-clinical overview and a non-clinical summary, and a clinical overview and a clinical summary;

  • a summary description of the pharmacovigilance system used by the marketing authorisation holder, including:
    • a declaration by the marketing authorisation holder (submitted under penalty of perjury) that the marketing authorisation holder has the services of a qualified person responsible for monitoring the safety of medicinal products,
    • a list of the Member States of the European Union or the EFTA Member States in which the person referred to in point a resides and performs his or her duties,
    • a declaration by the marketing authorisation holder that it has the necessary resources to fulfil its obligations regarding the pharmacovigilance of medicinal products,
    • contact details of the person referred to in point a,
    • an indication of the place where the full description of the pharmacovigilance system for medicinal products referred to in Article 36g paragraph 1 point 4 is available for inspection;
  • a risk management plan for the use of a medicinal product;
  • in the case of clinical trials conducted outside the territory of the European Union Member States or EFTA Member States, a statement that the trials meet the ethical requirements set out in Regulation 536/2014;
  • declarations signed by the experts preparing the overall quality summary, the non-clinical review and non-clinical data summary, and the clinical review and clinical summary that they possess the necessary technical or professional qualifications, as described in the attached curriculum vitae;
  • Summary of Product Characteristics;
  • templates of primary and secondary packaging presented in descriptive and graphic form, as well as a leaflet, along with a report on its readability;
  • copies of all authorisations issued by the relevant authorities in the European Union Member States or EFTA Member States or third countries, Summaries of Product Characteristics, summaries of safety data, including data from periodic safety update reports and adverse reaction reports, where available, and copies of package leaflets, where applicable, and copies of all decisions refusing to grant an authorisation and the reasons for such decisions;
  • a list of the European Union Member States and EFTA Member States where an application for a permit is being considered and, where applicable, details of any refusal to grant a permit in any country;
  • a copy of the authorization to manufacture the medicinal product in the country of manufacture.

As can be seen, any entity wishing to market a medicinal product must have extensive documentation to ensure its safe use after marketing authorization. The template for applications submitted to the Polish Office was published in the Regulation of the Minister of Health of January 10, 2014, regarding the template for applications for marketing authorization for medicinal products (consolidated text: Journal of Laws of 2018, item 593).

With regard to the application submitted under the WEU procedure, significant differences are introduced by Article 16 of the Act, according to which the marketing authorisation holder is not obliged to present the results of non-clinical or clinical trials if:

  • the active substance or substances of the medicinal product have a well-established medical use;
  • in the territory of a Member State of the European Union or an EFTA Member State;
  • for a period of at least 10 years, counting from the first systematic and documented use of this substance in a medicinal product and recognized efficacy and an acceptable level of safety.

In this case, the results of non-clinical or clinical studies are replaced or supplemented publications from the scientific literature, and the application must be accompanied by a justification prepared by an expert regarding the use of scientific literature in accordance with the requirements set out in Annex I.

The publications to be submitted must include the full scope of non-clinical and clinical trial results required for new medicinal products. Furthermore, the wording of Article 16 of the Act contradicts Article 10a of the Directive, which only refers to the replacement of scientific studies with publications, not to their replacement or supplementation.

Criteria for granting a permit

The above-mentioned conditions for applying the WEU procedure leave considerable room for interpretation and discretion in the issuance of marketing authorizations for medicinal products. Consequently, EU and national authorities have issued numerous guidelines on this topic.

Until March 30, 2007, detailed requirements for the WEU procedure were specified in the Regulation of the Minister of Health of January 16, 2003, regarding documentation of medicinal product test results, including veterinary medicinal products, and expert reports, which was repealed with the amendment to the Pharmaceutical Law. Currently, Polish regulations do not provide a binding interpretation of the concept of a substance with a well-established medical use.

The criteria for applying this concept can be found in Annex I to the Directive , according to which the following principles apply to demonstrate well-established medicinal use:

a/ The factors that should be taken into account in order to recognize the well-established medical use of the ingredients of medicinal products are as follows:

– how long a given substance has been used,

– the quantitative aspect of the use of a given substance,

– the degree of scientific interest in a given substance (as reflected in the published scientific literature) and

– consistency of scientific assessments.

b/ The documentation submitted by the applicant should cover all aspects of the safety and/or efficacy assessment and must include or refer to a review of the relevant literature, including pre- and post-marketing studies and published scientific literature on experience gained, in the form of epidemiological studies, and in particular comparative epidemiological studies. All documentation, both favorable and unfavorable, must be provided. In accordance with the provisions on “well-established medical use,” it is particularly necessary to clarify that “bibliographic references” to other sources of evidence (post-marketing studies, epidemiological studies, etc.), and not just data relating to tests and trials, can serve as valid evidence of the product’s safety and efficacy if the application satisfactorily explains and justifies the use of these sources of information.

c/ Particular attention must be paid to any missing information and an explanation must be provided as to why demonstration of an acceptable level of safety and/or efficacy can be supported despite the absence of some studies.

d/ Non-clinical and/or clinical reviews must clarify the relevance of any data submitted that relate to a product other than the product intended for marketing (so-called bridging data). A decision must be made as to whether the investigational product can be considered similar to the product for which marketing authorization has been applied for, despite existing differences.

e/ Post-marketing experience with other products containing the same ingredients is particularly important and the applicant should place special emphasis on this issue.

Furthermore, in July 2019, the European Commission issued guidance for applicants for marketing authorizations (Volume 2A, Chapter 1), which also addresses this procedure. The following indications follow from this guidance.

The Commission stressed that applications submitted under Article 10a of the Directive should not lower the level of safety and efficacy that a product with a well-established substance must also meet.

With respect to advanced therapy medicinal products (ATMPs), the Commission has determined that applications under this procedure are only permissible to the extent that the published scientific literature is relevant and sufficient to demonstrate the safety and efficacy profile of the medicinal product. Due to the specific nature of the ATMP manufacturing process, an application under this procedure will be admissible only exceptionally, provided that the manufacturing process for the product covered by the application is identical to the manufacturing process already described in the literature.

It is also worth noting that the term “medical use” does not necessarily mean use as an approved medical product, but evidence of the medicinal activity of a given substance must be provided. The 10-year period referred to in the regulations, in the case of a substance used in products authorised for marketing, does not have to cover the entire period during which a given country was a member of the EU or EFTA – this period also includes the years in which the product was authorised before the country joined the organisation in question.

The term “well-established use” refers to the use of a substance for a specific therapeutic purpose – if a well-known substance is to be used for a completely new therapeutic purpose for which no well-established use can be applied, it will not be possible to use this procedure.

It is the applicant’s responsibility to provide a detailed description of the strategy used to search for published literature and the rationale behind the selections made. The term “published” literature means that the text must be freely available in the public domain and published by a reputable, preferably peer-reviewed, source. Full texts of the research studies must be included in the application, along with any necessary translations.

European Medicines Agency (EMA) has also issued guidelines on this topic.

It is the applicant’s responsibility to demonstrate that the active substance contained in the medicinal product for which the application is submitted is identical to the substance described in the literature and to provide appropriate bridging data. Any studies submitted must support the efficacy and safety of the active substance.

The Agency stated that the applicant should submit a tabulated summary of data to support the assessment, including at least the following details:

  • Comprehensive information on specific products (formulas that have been used in efficacy and safety studies reported in the literature)
  • Justification for the studies and literature included to support the judgement that the product studied in the literature can be considered similar to the product for which marketing authorisation is being sought.
  • Which of the attached literature is considered crucial to demonstrate the effectiveness and safety of a given substance for a specific therapeutic purpose?

For specific types of substances and products, EMA has also issued detailed guidance on the requirements for bridging data used to establish the well-established use of a specific substance.

Comparison of active substances in given products should be based on one or more of the following criteria:

  • comparison of the qualitative and quantitative composition of the product;
  • comparison of critical quality attributes;
  • comparison of dissolution data;
  • comparison of physicochemical properties;
  • comparative in vitro release or permeation studies for a locally acting product;
  • comparative bioavailability (PK) studies;
  • clinical justification for the lack of comparative bioavailability studies.

Finally, it is worth emphasizing that if a medicinal product is authorized for the market under the WEU procedure, it may constitute a reference product when submitting applications for authorization to market equivalent products (as stated by the CJEU in its judgment of 23 October 2014 in case C-104/13).

Sources:

  1. Act of 6 September 2001 – Pharmaceutical Law (consolidated text: Journal of Laws of 2025, item 750, as amended).
  2. Pharmaceutical law. Commentary, 2nd edition, ed. M. Kondrat, Warsaw 2016.
  3. Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use (OJ L 311, 2001, p. 67, as amended).
  4. Annex I to the above-mentioned Directive
  5. European Commission Guidelines for Applicants (Volume 2A: Marketing Authorisation Procedures. Chapter 1: Marketing Authorisations)
  6. https://www.ema.europa.eu/en/human-regulatory-overview/research-development/scientific-guidelines/clinical-pharmacology-pharmacokinetics/clinical-pharmacology-pharmacokinetics-questions-answers#section-9-well-established-use-78013
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