ATMPs: Applicable regulatory pathways

Once a medicine has been qualified as an Advanced Therapy Medicinal Product-ATMP (a CAT scientific recommendation on classification is highly recommended), the main regulatory pathway for ATMPs to access the entire European Union market is the centralised procedure of marketing authorisation as regulated by the general EU pharmaceutical legislation (Directive 2001/83/EC and Regulation 726/2004) and the ATMP regulation. See “Market access for ATMPs”

However, several other main European Union legislations may be applicable:

• If the ATMP is based on human biological elements, the EU directive on tissues and cells or the EU directive on blood is applicable to the donation, collection/procurement and testing of these biological elements, along the development of the ATMP. See “Human body elements for human applications”
• If the ATMP involved genetically modified cells, the EU directive on the deliberate release into the environment of genetically modified organisms or/and the EU directive on the contained use of genetically modified micro-organisms is/are applicable.
• If the ATMP incorporate one or more medical devices, and is qualified as a combined ATMP, the EU regulation on medical devices is applicable to the medical device part of the product.
• If the ATMP is used with a companion diagnostic in order to define patients’ eligibility to the ATMP, the EU regulation on in vitro medical devices is applicable to the medical device.
• If the ATMP is also qualified as an orphan medicine, the EU orphan regulation is applicable. See “ATMPs and Orphan medicinal products".
• The EU regulation on paediatrics is applicable. 

Moreover, patients may access ATMPs through other regulatory pathways:

• If the ATMP is investigational (i.e. experimental), patients are legally research participants, and may access the investigational ATMP in the context of clinical trials. (See “Clinical trials”) as a general derogation to the mandatory marketing authorisation of medicines in accordance with article 3.3 of Directive 2001/83/EC.
• If the ATMP is “prepared on a non-routine basis according to specific quality standards, and used within the same Member State in a hospital under the exclusive professional responsibility of a medical practitioner, in order to comply with an individual medical prescription for a custom-made product for an individual patient”, patients may access the ATMP under the so-called “hospital exemption” pathways which are ATMPs-specific derogations to the centralised procedure of marketing authorisation of ATMPs in accordance with article 3.7 of Directive 2001/83/EC.
• Patients may also access ATMPs through “compassionate use” or “named-patients basis” pathways which are general derogations to the mandatory marketing authorisation of medicines in accordance with article 5.1 of Directive 2001/83/EC or article 83 of Regulation 726/2004.
• Finally, and although this is unlikely given the complexity of ATMPs, pathways exist for pharmacies to prepare medicines for patients (the so-called “magistral formula” and “officinal formula”). These are general derogations to the mandatory marketing authorisation of medicines in accordance with articles 3.1 and 3.2 of Directive 2001/83/EC respectively.)