Previous

1. Advanced therapy medicines (ATMPs)

1.2. Incentives

The ATMP Regulation also introduced a number of incentives for manufacturers of advanced therapies, particularly small and medium-sized enterprises (SME). The aim is foremost to provide regulatory certainty to various questions relating to R&D of ATMPs in pursuing a marketing authorisation. These include:

  • Scientific recommendation on advanced therapy classification (“ATMP classification”);
    • Companies can consult the EMA on a voluntary basis to determine whether a medicine they are developing is an ATMP.
    • ‘What guidelines are applicable to my product?’
    • 60-day procedure (often shorter),
    • NNo fee
    • All classification outcomes are published (summary)
    • For early developments (no expectation that the product already has  substantial non-clinical or clinical data available);
  • Certification of (early) quality and (early) non-clinical data for SMEs only
    • Questions on Quality, Non-clinical and clinical development. According to Article 18 of Regulation (EC) No 1394/20071 which provides that SMEs developing an ATMP may submit to the EMA all relevant quality and, where available, non-clinical data
    • ‘Is my product development so far on track for a future Marketing Authorisation Application?’
    • 90-day procedure
    • Applicant will always receive the evaluation report and List of issues for future consideration
    • If positive evaluation: Certificate by EMA;
  • Scientific advice:
    • A fee reduction of 90% for SMEs and 65% for other applicants on any scientific advice given with respect to ATMPs;
  • Scientific advice on the design and conduct of Pharmacovigilance and risk management systems;
  • Reduction of fees for marketing authorization (50%) for hospitals and SMEs if a public health interest in the ATMP can be proven; furthermore, the 50% fee reduction is also granted for post-authorisation activities in the first year following the granting of the product’s marketing authorisation.

Of note: in case where the reference medicinal product is a biologic (or ATMP), the general criteria for generic MAAs do not apply; rather, follow-on biologicals that are similar to the reference product must still provide a certain amount of preclinical and clinical data in support of its MAA. For further details on biosimilars see EMA-Biosimilars.

Of note: The ATMP Regulation, in line with Directive 2001/83/EC, applies to ATMPs manufactured by industrial methods and intended to be marketed in EU Member States. Under the so-called hospital exemption, certain ATMPs are excluded from its provisions (Article 3(7) of Directive 2001/83/EC 2001), if they are:

  • prepared on a non-routine basis according to specific quality standards,
  • used within the same Member State in a hospital under the exclusive professional responsibility of a medical practitioner,
  • to be compliant with an individual medical prescription for a custom-made product for an individual patient.

ATMPs falling under the above exemptions do not need to apply for a MA, but can rather be authorised for use by the concerned Member State if they apply with national requirements for quality, traceability and pharmacovigilance, equivalent to those for ATMPs with a centralised MA. This non-routine application of ATMPs was intended to facilitate the research and development of advanced therapies by academic groups, hospitals or not-for-profit organisations.

Legal basis:

Regulation 1394/2007/EC on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 – i.e. the provisions of the ATMP Regulation are now also reflected in those acts.

Guidelines with specific ATMP relevance

GMP for ATMPs
EC adopted Guidelines on Good Manufacturing Practice (GMP) specific for ATMPs (November 2017). The Guidelines provide a specific GMP framework that is adapted to the specific characteristics of ATMPs. Guidelines on Good Manufacturing Practices (GMP)

GCP for ATMPs
EC adopted Guidelines on Good Clinical Practice (GCP) specific for ATMPs (October 2019). Guidelines on Good Clinical Practice (GCP) specific for Advanced Therapy Medicinal Products (ATMP)

GMO (Genetically Modified Organism) requirements for investigational products Questions & Answers document

Further reading: European Commission-DG Health and Food Safety and European Medicines Agency Action Plan on ATMPs (europa.eu)

Previous