EU – RWD/RWE is Embedded into the New EU Medicines Regulations

RWE 201 – EU – RWD/RWE is Embedded into the New EU Medicines Regulations


Coming Soon…New EU Medicines Regulations:

In 2023, the European Commission undertook an ambitious overhaul of its pharmaceutical regulations. This revision addresses foundational pharmaceutical legislation, specifically Regulation 726/2004, Directive 2001/83/EC, and rules for medicines tailored for children and rare diseases, namely Regulation 1901/2006 and Regulation 141/2000/EC.

Primary Aims:

– Ensure all EU patients access safe, effective, and affordable medicines promptly and fairly.

– Bolster medicine supply security across the EU.

– Propagate an innovation-centric environment for medicine R&D in Europe.

– Pivot towards environmentally sustainable medicines.

– Confront antimicrobial resistance and environmental pharmaceutical contamination through a holistic One Health approach.

Key Points of the Revision:

  1. Individual Patient Data: Regulators can now request structured individual patient data from clinical studies, promoting data-driven benefit-risk assessments for medicines throughout their life cycle (Recital 63 of MP-R).
  2. Transparency of Public Support: There’s now a mandate to disclose any direct financial backing received from public authorities for medicine R&D, fostering accountability and transparency (Recital 131 of MP-D).
  3. Patient Representation: The CHMP (Committee for Medicinal Products for Human Use) and PRAC (Pharmacovigilance Risk Assessment Committee) now include patient representatives, enriching patient voices in decisions.
  4. Real World Data (RWD): The revision endorses the use of health data, particularly RWD, for regulatory decision-making. Through systems like the DARWIN and European Health Data Space infrastructure, the agency can harness supercomputing, AI, and big data without jeopardizing privacy (Recital 60 of MP-R).
  5. Regulatory Sandbox: This introduces a controlled setting wherein innovative regulatory solutions can be tested, cultivated, and authenticated under scrutiny (Articles 2(12), 113-155 of MP-R).
  6. Compassionate Use Programs: Provisions for these programs, which offer early medicinal product access, have been strengthened. It’s vital to collect data from these programs to evaluate the benefit-risk ratio of medicines (Recital 57 & Article 26(4) of MP-R).
  7. Comparative Effectiveness: By repurposing medicines and leveraging comparative trial data, patients will have expedited access to novel treatments. Such data assists authorities in ascertaining a medicine’s cost-effectiveness.
  8. Relative Effectiveness: The EU has devised a scientific, evidence-based methodology to gauge the relative effectiveness of medicinal products. This focuses on a medicine’s added value against other health technologies but doesn’t extend to its marketing authorization (Recital 130 of MP-R).

In essence, this legislative revamp by the European Commission fosters a more patient-centric, transparent, and data-driven approach in the EU pharmaceutical landscape.

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