Regulations of Advanced Therapies (ATMPs)

Advanced Therapies (ATMPs)

• 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.

ATMP Regulation Key Principles

• 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

EMA Support for ATMPs

• 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)

Critical Aspects of ATMPs

• 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

P&R in EU Countries

• 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)

Digital Therapeutics (DTx)

• 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

Learn about the regulations and principles governing Advanced Therapies Medicinal Products (ATMPs), including gene therapy, somatic cell therapy, and tissue-engineered products.