{
  "abstract": "Background and Importance Therapeutic innovation is continuously expanding leading to a growing number of new active substances (NAS) and indication extensions of prexisting medicines (IEPM) evaluated by the European Medicines Agency (EMA). The need for new therapeutic options has driven many approvals to occur under accelerated procedures or based on preliminary evidence. This raises questions regarding the maturity and robustness of clinical data supporting them. It’s crucial to assess the quality of evidence behind these authorisations ensuring informed decision-making by emphasising hospital pharmacists’ role in evidence appraisal and real-world assessment.Aim and Objectives Characterise and assess the level of clinical evidence underlying all positive opinions issued by the Committee for Medicinal Products for Human Use (CHMP) in 2024 for NAS and IEPMMaterial and Methods Observational, descriptive, retrospective study including all NAS and IEPM receiving a positive CHMP opinion in 2024 [age-based extensions excluded.] Data were retrieved from the European Public Assessment Reports (EPARs). Collected variables included: active substance, therapeutic area, mechanism of action, regulatory classification (ORPHAN, PRIME), special conditions (conditional approval [CA], accelerated assessment [AA], exceptional circumstances [EC], additional monitoring [AM]), evaluated indication, study design characteristics, primary outcome, hazard ratio, ‘first-in-class’ status, ESMO-MCBS score (when relevant), EPAR conclusions, study limitations, and study identifier.Results A total of 100 authorisations were included: 42 NAS (46 indications and 35.7% first-in-class) and 54 IEPM across 15 therapeutic areas and 23% held an orphan designation. The majority were supported by phase III (86%), randomised (85%), active-controlled (55%), open-label (49%). Additionally, 28,3% of the NAS were approved under special conditions (CA/AA/EC). Most frequent therapeutic areas were Oncology (45%), Haematology (16%) and Cardiovascular (6%). Among oncology therapies, 44% achieved an ESMO-MCBS score ≥4 or A, indicating relevant clinical benefit, although 55,5% relied on surrogate endpoints and/or with immature overall survival data.Conclusion and Relevance Most 2024 approvals were supported by phase III, randomised, active-controlled trials, however, a considerable proportion relied on preliminary data, particularly within oncology. Accelerated and conditional pathways highlight the need for systematic post-authorisation re-evaluation supported by real-world evidence to confirm clinical benefit and ensure rational use.References and/or Acknowledgements 1. European Medicines Agency. Human medicines in 2024. EMA; 2024.2. Martínez-Barros H, et al. Farmacia Hospitalaria. 2024;48(6):272–7.Conflict of Interest No conflict of interest",
  "authors": [
    {
      "affiliations": [
        "Hospital Garcia De Orta- Almada Seixal Local Health Unit, Pharmaceutical Services, Almada, Portugal"
      ],
      "name": "MDS Lourenço"
    },
    {
      "affiliations": [
        "Hospital Garcia De Orta- Almada Seixal Local Health Unit, Pharmaceutical Services, Almada, Portugal"
      ],
      "name": "B Resende"
    },
    {
      "affiliations": [
        "Hospital Garcia De Orta- Almada Seixal Local Health Unit, Pharmaceutical Services, Almada, Portugal"
      ],
      "name": "S Bastos"
    },
    {
      "affiliations": [
        "Hospital Garcia De Orta- Almada Seixal Local Health Unit, Pharmaceutical Services, Almada, Portugal"
      ],
      "name": "T Ferreira"
    },
    {
      "affiliations": [
        "Hospital Garcia De Orta- Almada Seixal Local Health Unit, Pharmaceutical Services, Almada, Portugal"
      ],
      "name": "A Soares"
    },
    {
      "affiliations": [
        "Hospital Garcia De Orta- Almada Seixal Local Health Unit, Pharmaceutical Services, Almada, Portugal"
      ],
      "name": "A Alcobia"
    }
  ],
  "title": "4CPS-208 How certain are we of innovation? A review of clinical evidence of ema approved new indications in 2024",
  "uid": "cf0296a4-1824-5930-b8a6-eab699eff5fa"
}