The CataloniaBio & HealthTech Regulatory Affairs Workgroup devoted its ninth Hard Reg Café session to the regulatory challenges facing advanced therapy medicinal products (ATMP), on 28 April.

ATMP are highly innovative drugs that are needed to treat diseases in the 21st century. In the European Union, 17 were authorised between 2009 and 2020. Oncology is the main area, followed by neurology, infectious diseases and endocrinology.

“ATMP based on the patient’s own genes, cells or tissues are revolutionising healthcare, but the regulatory process for them isn’t easy,” noted Lluís Chico, managing partner of NEOS Surgery and first vice-president of CataloniaBio & HealthTech, in his introduction to the session. Chico coordinates this Workgroup with Lídia Cánovas, general manager for Regulatory Affairs at Asphalion, and Melqui Calzado, secretary general of CataloniaBio & HealthTech.

Some forty professionals from Catalan companies followed the chat online with experts who shared the point of view of advisers, companies, hospitals and regulatory agencies.

Christopher Mann, Scientific and Regulatory Affairs director at Asphalion, explained that it is important to understand what is considered an ATMP to guide decision-making and regulatory paths. Currently, the main EU regulations in this arena are Directive 2001/83/EC (medicinal products for human use), Regulation 1394/2007/EC (advanced therapy medicinal products) and Regulation 726/2004/EC (community procedures for the authorisation and supervision of medicinal products). Mann said, “the indication and availability of treatments are very important” in facilitating the path from research to market.

Jürgen Bednarz, head of Regulatory Affairs at TETEC (the BBraun Group tissue engineering company), noted the importance of complying with Good Manufacturing Practices (GMP), the processes being as simple as possible and focusing on standardisation.

This year, Hospital Clinic Barcelona has been granted the first exceptional authorisation for hospital use of a CAR-T (Chimeric Antigen Rector T-Cell) from the Spanish Agency of Medicines (AEMPS). CAR-T is a type of cell and gene therapy in which the patient is their own donor. Manel Juan, head of Immunology at the Hospital Clinic Biomedical Diagnostic Centre (CDB) and head of the joint platforms of Hospital Sant Joan de Déu and the Blood and Tissue Bank, said, “It is a very important milestone. We’ve managed to get a highly innovative treatment developed at a hospital with a team of 175 professionals to patients with lymphoblastic leukaemia resistant to conventional treatments.”

The main tip Sol Ruiz, head of Biologics, Biotechnology and Advanced Therapies at AEMPS, had for biomedical companies was "to contact the innovation agency at the country’s regulatory agency as early as possible to identify the best path and move through clinical development quickly.” Ruiz assured that “in the very near future, we’ll see important ATMP reaching patients.”

Members who couldn’t attend the Hard Reg Café session and are interested in watching the recording can contact secretari@cataloniabioht.org.