{
  "abstract": "Background and Importance The evaluation of avoided cost (AC) in investigational medicines addresses the economic dimension of clinical trials, an objectively quantifiable factor. Although sponsors are legally required to provide study drugs free of charge, this aspect remains under-reported in our setting. Quantifying AC is essential to assess the real economic impact of clinical trials, especially in diseases with high pharmaceutical expenditure such as transthyretin amyloidosis.Aim and Objectives The aim of this study was to quantify the avoided drug cost associated with the participation of patients in clinical trials on transthyretin amyloidosis, in order to assess their real economic impact on the healthcare system.Material and Methods A retrospective observational study was conducted in a tertiary hospital including clinical trials on transthyretin amyloidosis from 2019 to 2024. Trials were included if they were active during this period, investigated medicines already marketed by March 2025, and provided study drugs free of charge by the sponsor. Trials with no patients enrolled were excluded. Data were obtained from the institutional clinical trial management software and the pharmacy department database. For each trial, information was collected on protocol code, phase, scope, sponsor, investigational drug, number of enrolled patients, doses dispensed per patient, and drug cost (ex-factory price + VAT – applied discounts). The primary outcome was the total avoided cost over the period 2019–2024, while the secondary outcome was the mean avoided cost per patient. AC was calculated assuming patients would have received the same treatment, dose and regimen outside the clinical trial.Results During 2019–2024, two clinical trials on transthyretin amyloidosis were conducted; only one met the inclusion criteria. This was an international, multicentre, industry-sponsored phase III trial evaluating tafamidis in both hereditary and wild-type forms of the disease. The total avoided cost reached €9,710,268 over the study period, with a mean avoided cost per patient of €134,865, corresponding to an approximate annual saving of €1.9 million.Conclusion and Relevance The participation of hospitals and patients in clinical trials contributes significantly to reducing direct drug costs associated with transthyretin amyloidosis. Beyond this economic impact, clinical trials also reinforce the sustainability of healthcare systems, foster scientific progress and facilitate early patient access to innovative therapies.Conflict of Interest No conflict of interest",
  "authors": [
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "A Torres-Pérez"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "E Gómez-Costa"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "MT Rabuñal-Álvarez"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "S Jimeno-Aguado"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "P Sánchez-Carballo"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "E Sánchez-Mira"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "C Fernandez-Diz"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "L Caeiro-Martinez"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "M Calvin-Lamas"
    },
    {
      "affiliations": [
        "A Coruña University Hospital Complex Sergas, Pharmacy, A Coruña, Spain"
      ],
      "name": "MJ Mauriz-Montero"
    },
    {
      "affiliations": [
        "Research Group of Hospital Pharmacy. Biomedical Research Institute A Coruña Inibic., Pharmacy, A Coruña, Spain"
      ],
      "name": "L Margusino-Framiñan"
    }
  ],
  "title": "4CPS-216 Avoided drug cost derived from participation in clinical trials on transthyretin amyloidosis",
  "uid": "ef2195ac-9058-5275-b599-4cb5f29201e8"
}