Regulations of Advanced Therapies (ATMPs)

Study Notes

ATMPs include gene therapy medicinal products (GTMP), somatic cell therapy medicinal products (sCTMP), tissue-engineered products (TEP), and combined ATMPs that combine advanced therapy with a medical device.
The ATMP regulation (1394/2007) ensures that principles of existing legislation on medicines apply to advanced therapies, including marketing authorization, demonstration of quality, safety, and efficacy, GMP, GCP, post-authorization vigilance, and RMP.

Ensures principles of existing legislation on medicines apply to advanced therapies
Imposes a new committee for advanced therapies (CAT) with 2 members, responsible for preparing a draft opinion on each ATMP application submitted to the EMA
Simultaneous national scientific advice allows multiple regulatory agencies to provide a joint evaluation to the applicant, instead of separate meetings with each authority
Marketing exclusivity provides 10-year market exclusivity to prevent other companies from submitting similar products unless they demonstrate significant superiority

The PRIME scheme supports innovative medicines addressing unmet medical needs and bringing major therapeutic advantages to patients
PRIME designation grants shorter overall MAA assessment time (8-12 months instead of 12-29 months)

HTA critical aspects:
- Targeting rare and severe diseases
- Uncertainty about long-term effects (efficacy, safety, durability)
- Lack of comparators
- Importance of prescribing centers
Reimbursement critical aspects:
- High costs per patient (return on investment critical)
- High costs of management (payment of services in addition to the drug)
- Risk management agreements or payment by results to spread costs over multiple years

France, Italy, Spain, and UK have similar P&R procedures to drugs
Germany has a preliminary assessment by G-BA, with drugs in the AMNOG system and advanced therapies following a specific pathway (PEI evaluation)

Fall within the field of medical devices
Manufacturers must register devices to obtain CE certification and commercialize them
Devices that are not certified are defined as RUO (research use only)
Devices require:
- Risk management plan for good vigilance, safety, and complaint collection
- Adherence to all necessary guarantees
Medical devices are classified into 3 classes based on complexity and risk, with higher classifications indicating greater risks:
- Class 1: Low-risk devices that require minimal evaluation and can be self-certified by the manufacturer
- Class 2A, 2B, and 3: Higher-risk devices that require more extensive evaluation and certification