Designation of orphan conditions in Europe: regulatory observations and considerations after implementation of regulation 141/2000

ABSTRACT

Introduction

In the first 18 years of the implementation of the orphan medicinal products regulation, it has been noted that every year a steady number of orphan designations refer to new conditions, not designated previously. This is important because it offers documented evidence that research and development is ongoing for many areas of rare diseases. These newly designated rare conditions reflect drug development opportunities in areas of limited regulatory knowledge. Authors carried out a literature search via pubmed and Google as well as refering to previous articles they have been involved in as authors.

Areas covered

The aim of this paper is to review the outcomes associated with new rare conditions designation by the COMP. With over 2000 designations made since its creation data specific to conditions designation collected by the European Medicines Agency (EMA) after each monthly plenary session is presented here. The data is observational and has been grouped into therapeutic criteria based on ATC codes.

Expert opinion

Regulators should continue to engage in constructive dialogue with stakeholders so that the regulatory requirements are less of a hurdle and more of an opportunity to speed up drug development in areas of unmet medical need. The designation of new conditions further supports the utility, need, and meaning of the orphan regulation as a catalyst of drug development.

KEYWORDS:

• Orphan designation
• regulatory
• condition
• clustering
• drug development

Article highlights

• Many rare diseases exist in the world and many are still to be identified and better defined.

• Since the implementation of the orphan medicinal products regulation, every year a steady number of orphan designations refer to new conditions, not designated before.

• Orphan designation helps understand the gathering of science to meet the aims of the regulation.

• The documented evidence that research is ongoing for many rare diseases through orphan designation and these newly defined rare conditions reflect drug development opportunities in areas of limited regulatory knowledge.

• The designation of new conditions further supports the utility, need and meaning of the orphan regulation as a catalyst of drug development.

This box summarizes key points contained in the article.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.