EU pharmacovigilance legislation has recently undergone a major review that led to the adoption of new legislation in 2010, namely Directive 2010/84/EU and Regulation (EU) No 1235/2010. While the main focus of pharmacovigilance is the safety of the medicinal product, any new information received or new pharmacovigilance signals detected may have a potential impact on overall product assessment and, more particularly, on its benefit-risk balance.
To streamline and clarify the regulatory tools of competent authorities for imposing certain obligations on marketing authorisation holders, Directive 2001/83/EC and Regulation (EC) No 726/2004 summarise the conditions, restrictions and obligations
- under which a marketing authorisation may be granted or
- which may be requested following the granting of the initial marketing authorisation.
In this context, the new pharmacovigilance legislation refers to the possibility of requesting a marketing authorisation holder to conduct post-authorisation efficacy studies (PAESs), complementing efficacy data available at the time of the initial authorisation5. To determine situations in which such studies may be required, the Commission is authorised to adopt, by means of a delegated act, measures supplementing the provisions of Directive 2001/83/EC and Regulation (EC) No 726/2004.
Post-authorisation efficacy studies are not a new feature in the EU’s regulatory framework for medicinal products for human use. They have been required for quite some time, especially in the context of conditional marketing authorisations and marketing authorisations under exceptional circumstances7. Likewise, Regulation (EC) No 1901/2006 on paediatric medicines and Regulation (EC) No 1394/2007 on advanced therapy medicinal products9 refer to the possibility of making the performance of specific post-marketing studies a condition of the marketing authorisation.
The added value that the 2010 pharmacovigilance legislation brings is a more global and systematic approach to post-authorisation efficacy studies. Instead of referring to post-marketing studies in different pieces of legislation or hinting at their existence in several provisions, the new rules bring the instrument to the forefront, listing it together with other conditions that may apply to a marketing authorisation.
According to the legal provisions the Commission enjoys discretionary powers in deciding whether to adopt a delegated act. That said, Directive 2001/83/EC and Regulation (EC) No 726/2004 require that any post-authorisation efficacy study imposed as an obligation on the marketing authorisation holder ‘shall be based on the delegated act’. This implies that a delegated act is a pre-condition for imposing post-authorisation efficacy studies. For the application of the new concept, it therefore seems necessary to adopt a delegated act.
Moreover, the delegated act will contribute to predictability and transparency regarding situations in which post-authorisation studies may be required.
The delegated act will be complemented by scientific guidance on post-authorisation efficacy studies that will be drawn up by the European Medicines Agency in accordance with Article 108a of Directive 2001/83/EC.
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