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6ER-003 Assessment of overall survival and safety in newly approved oncohaematologic drugs
  1. AB Pousada Fonseca1,
  2. H Martínez Barros2,
  3. J Pedreira Bouzas3
  1. 1Hospital Universitario De Móstoles, Hospital Pharmacy, Móstoles, Spain
  2. 2Hospital Universitario Ramón Y Cajal, Hospital Pharmacy, Madrid, Spain
  3. 3Hospital Universitario De Fuenlabrada, Hospital Pharmacy, Fuenlabrada, Spain


Background and Importance To be included on the World Health Organization (WHO) Model List of Essential Medicines, cancer drugs should increase overall survival (OS) by at least 4–6 months.

Aim and Objectives To evaluate OS benefit and safety of oncohaematological drugs approved by the European Medicines Agency (EMA) from 2017 to 2020.

Material and Methods This retrospective observational study identified the first indication of new oncohaematological drugs approved by the EMA between 2017 and 2020. The following variables were collected using the European Public Assessment Reports: drug, primary endpoint, Hazard Ratio (HR) of OS with confidence intervals, OS benefit in months (if medians were reached) and total grade 3 or 4 adverse events (AE) for both drug and comparator. A Student’s t-test was conducted to compare AE.

Results A total of 49 indications were identified. The primary endpoint used was surrogate in 41 indications (83.7%): Response rate in 20 (40.8%); progression-free survival in 15 (30.6%); disease-free survival in two (4.1%); metastasis-free survival in two (4.1%); and invasive disease-free survival in one indication (2.0%). One drug (2.0%) was approved with a pharmacokinetic equivalence outcome.

In the remaining eight indications (16.3%), the primary endpoint was OS with a median HR of 0.71 [IQI 0.59–0.77] and a median interval width (upper minus lower interval) of 0.36 [IQI 0.29–0.42]. In four additional indications (8.2%), there was benefit for OS as a secondary endpoint.

Globally, OS benefits were reported in 12 indications (24.5%) (8 as primary and four as secondary endpoint), but median OS had not been reached in two. Median benefit was 4.1 months [IQI 3.6–16.7], with six indications (6/10) demonstrating benefits equal to or exceeding 4 months.

Regarding safety, the mean of serious AE (≥ grade 3) in the 49 indications was 63.6% in the experimental groups and 52.2% in the control groups, with a difference of 11.4% (95% CI: 0.74–22.1).

Conclusion and Relevance OS was the primary endpoint in 1 in 6 approved indications. While HR values were acceptable, considerable interval widths were noted.

Approximately one-quarter of indications demonstrated OS benefit and six approved indications met the lower limit for inclusion in the WHO Model List of Essential Medicines.

Despite modest OS outcomes, statistically significant increases in AE were observed.

Conflict of Interest No conflict of interest.

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