What are regulatory data and market exclusivity? Drug approval in Europe

by | Jun 3, 2024 | European Medicines Agency, Marketing Authorisation Application

Highlights

  • What is a reference medicinal product?
  • What is drug market protection?
  • What is regulatory data exclusivity?
  • Why data and market exclusivity matter for new medicines.
  • Market and data exclusivity for orphan medicines.
  • Exclusivity rewards for paediatric medicines.

Exclusivity—market and data—refers to the length of time an approved medicinal product will be protected from similar competitors.

What is a reference product?

Before discussing market and data exclusivity, we should explain the reference product to which these exclusivities apply.

A reference product is the first medicinal product of its kind to be approved by the European Commission or by an individual member state, and its approval is based on a full marketing authorisation application (MAA) dossier, which includes complete quality, non-clinical and clinical data in the submission. It is the medicinal product and MAA dossier that all generics, hybrid and biosimilar products will refer to.

Essentially, generic, hybrid and bio-similar products can reference the data the approved reference medicinal product collected, without having to submit a de novo MAA dossier required by Articles 8(3), 10a, 10b or 10c of Directive 2001/83/EC.1

To protect the reference medicinal product from biosimilars and generics and reap the commercial rewards, the reference medicinal product, along with all of its variations, extensions, strengths indications, and routes of administration, will receive market and data exclusivity from the date it was first granted marketing authorisation for a set time.

What is regulatory data exclusivity?

In line with Directive 2001/83/EC and Regulation (EC) No 726/2004,2 a company hoping to develop a hybrid, generic or bio-similar product cannot refer to or rely on the reference medicinal product’s MAA dossier to gain marketing authorisation for a set time.

This is known as ‘data exclusivity’ and is enforceable for 8 years from when the reference medicinal product was granted marketing authorisation.2

What is drug market exclusivity?

Alongside the 8-year data exclusivity period, the reference medicinal product is also protected from any hybrid, generic or bio-similar product being placed on the market, even if the medicine developer has submitted to and received a positive opinion from the European Medicines Agency (EMA) on their MAA.

Marketing exclusivity applies for 10 years from when the reference medicinal product was granted marketing authorisation.2

In short, and per Article 14(11) of Regulation (EC) No 726/2004:

“…medicinal products for human use which have been authorised in accordance with the provisions of this Regulation shall benefit from an eight-year period of data protection and a ten-year period of marketing protection, in which connection the latter period shall be extended to a maximum of 11 years if, during the first eight years of those ten years, the marketing authorisation holder obtains an authorisation for one or more new therapeutic indications which, during the scientific evaluation prior to their authorisation, are held to bring a significant clinical benefit in comparison with existing therapies.”

Extensions to market protection

Also, per Article 14(11) of Regulation (EC) No 726/2004, data exclusivity and market protection of a reference medicinal product may be extended by 1 year (11 years of market protection in total) if, in the first 8 years, the MAH receives authorisation for one or more new indications.2 Also, per Article 14(11) of Regulation (EC) No 726/2004, data exclusivity and market protection of a reference medicinal product may be extended by 1 year (11 years of market protection in total) if, in the first 8 years, the MAH receives authorisation for one or more new indications.2

Why are data and market exclusivity so beneficial for medicine developers, when the approved reference product would have been granted a 20-year patent? Because the drug development process sometimes spans decades, by the time the initial reference medicine is approved, there may be very little time left on the patent. This means the company would not have long to gain any return on investment before competitors begin referencing the dossier and marketing similar products.

line chart of MAA data exclusivity and market protection timelines

Market and data exclusivity for orphan medicines

There are further market and data exclusivity benefits for orphan medicines, including 10 years of market protection for every indication with orphan designation and 2 extra years of market protection for completing paediatric studies in an orphan condition.3

NOTE: To benefit from market exclusivity, your medicinal product must maintain its orphan designation at the time of marketing authorisation. Also, you cannot mix orphan-designated indications and non-orphan-designated indications in the same marketing authorisation.

black and white line chart of MAA data exclusivity and market protection timelines for orphan-designated products

How the new pharmaceutical legislation will affect orphan exclusivity

The European Commission (EC) published proposed revisions to the pharmaceutical legislation in the form of a new Directive and a new Regulation. Many changes are included, some of which will affect market exclusivity for orphan-designated medicinal products. The EC proposed that market exclusivity duration will vary depending on the type of orphan medicinal product. The variations include:4

  • 10 years exclusivity for products developed to address a high unmet medical need,
  • 9 years exclusivity for new active substances, and
  • 5 years exclusivity for well-established use applications.

Exclusivity for paediatric medicines

To incentivise pharma and biotech companies, and other medicine developers to develop very much-needed medicines for children, the EMA offers several benefits for medicine developers completing agreed paeditric investigation plans (PIPs)including:3

  • 6-month extension to the supplementary protection certificate (patent exclusivity).
  • 10 years of market protection for authorised medicines developed specifically for children, and who aren’t protected by a patent or supplementary protection and are eligible for a paediatric use marketing authorisation (PUMA).

The idea of the 6-month SPC3 – similar to the 2-year extension to orphan exclusivity – is to compensate for the added paediatric clinical trials the EMA deem necessary.

black and white line chart of MAA data exclusivity and market protection timelines for paediatric medicines

Summary

Market authorisations in Europe benefit from 8 years of data exclusivity and 10 years of market protection if the approved medicine is classed as a reference medicinal product. A reference medicinal product comes from a full marketing authorisation application dossier under Articles 8(3), 10a, 10b or 10c of Directive 2001/83/EC.

Approved new indications extend marketing protection for 1 year making the total marketing protection from the date a product was approved, 11 years.

Approved MAs for orphan-designated products benefit from the same data exclusivity and market protection as non-orphan-designated products, and each new orphan-designated indication further benefits from a separate 10 years of market protection.

MAs with completed paediatric trials in line with agreed PIPs benefit from 6 months of SPC, and MAs for orphan-designated products are awarded an extra 2 years of market protection for completed paediatric trials in line with agreed PIPs.

How Somerville Development Partners can help

We can support every aspect of the filing, including the following:

  • Developing a Common Technical Document (CTD) table of contents for the submission and conducting a gap analysis to ensure that all submission components have been identified and planned.
  • Preparing key messaging and the company core data sheet.
  • Preparing regional labelling such as the Summary of Product Characteristics and US Prescribing Information.
  • Planning the data presentation, analysis and discussion in the dossier.
  • Conducting detailed reviews of the data (tables, figures and listings) shells to ensure the required analysis and outputs are planned.
  • Writing Modules 1–5 of the electronic CTD.
  • Publishing and submitting the completed submission.
  • Responding to questions from the agency during the evaluation.
  • We frequently, work as part of the global filing team for parallel submission to the US, EU and additional authorities. If possible, it is worthwhile writing a submission with common US and EU Module 2–5 components, especially the Module 2 summaries, so that multiple consistent submissions can be made to streamline regulatory agency interactions.
  • We can also provide peer review of existing projects, to stress-test the work that has been done and recommend solutions to address any gaps. Peer review may be especially useful to prepare for the MAA submission, where an additional perspective may help to highlight any potential risks.

Get in touch

We welcome the opportunity to discuss scientific advice and regulatory strategy with you!

Nicole

Author

Nicole Brooks, Regulatory Consultant / Copywriter
Nicole@somerville-partners.com

References

1. 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. Official Journal of the European Union. L 311, 28/11/2001 P. 0067 – 0128. Available at: https://eur-lex.europa.eu/legal-content/en/TXT/?uri=CELEX%3A32001L0083

2. Commission Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. Official Journal of the European Union. L 136, 30/04/2004 P. 0001 – 0033. Available at:https://eur-lex.europa.eu/eli/reg/2004/726/oj

3. European Medicines Agency. (2018). Data exclusivity, market protection, orphan and paediatric rewards. SME info day: Regulatory toolbox for medicines and combined devices developers. Available at: https://www.ema.europa.eu/system/files/documents/presentation/data_exclusivity_market_protection_orphan_and_paediatric_rewards-en.pdf

4. Proposal for a REGULATION OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006. COM/2023/193 final. Document 52023PC0193. Available at: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX%3A52023PC0193