Advanced therapy drugs – legislative requirements and availability

Medical Review (Med. pregled), 2024, 60(1), 28-33.

J. Alexandrova, P. Milushewa, G. Petrova

Faculty of Pharmacy, Medical University – Sofia

Abstract. Advanced therapy medicinal products (ATMPs) are a group of modern medicinal products developed through gene therapy, cell therapy, and tissue engineering, aiming to modify physiological functions using alterations in them. Due to their more complex nature and susceptibility to adverse reactions compared to conventional chemically derived drugs, they are subject to additional requirements. Still, their implementation is associated with other resources which hinders their accessibility. The current study analyzes the key aspects of legislative provisions regarding ATMPs, the challenges in meeting the necessary standards, ways to overcome them, and their impact on patients. АТМРs represent an innovative approach to the treatment of patients, for whom usually no suitable alternative is available. European legislation provides a number of regulatory regimes to ensure their rapid access to the market and 25 LPMTs have been authorized to date. Collaboration between all stakeholders is essential to ensure that patients across the EU have equal access and can benefit from the long-term benefits that innovative therapies bring.

Key words: advanced therapy medicinal products, regulatory requirements, market entry challenges

Address for correspondence: Joanna Alexandrova, е-mail: 105233@students.mu-sofia.bg