Objective:

The European Medicines Agency's marketing authorisation criteria for drugs are reflected in the European Public Assessment Reports. The objective is to describe the expectations and preferences of our oncohematological outpatients with respect to their oral treatments, and to evaluate the concordance with the results of European Public Assessment Reports.

Method:

A survey of onco-hematological patients' expectations and preferences about overall survival and quality of life was developed, with three items: expectations on treatment, preferences of benefit and willingness to receive novel treatments with non-definitive results. European Public Assessment Reports of the indicated drugs were reviewed. Kappa index (κ) was used to assess the agreement between patients' expectations and preferences respect to the benefit in overall survival and quality of life described in the corresponding European Public Assessment Report. Concordance between willingness of patients to receive novel treatments and European Public Assessment Reports results was evaluated by absolute agreement (Ao).

Results:

There were 29 participants, and 19 different European Public Assessment Reports were consulted. Patients' expectations about their treatment: 82.1% expected improvement in overall survival and quality of life; the κ value between expectations and results of European Public Assessment Reports was 0.091 (confidence interval 95%: –0.025 to 0.207). Patients' preferences about benefit of their treatment: 92.6% preferred quality of life; the κ value was 0.016 (confidence interval 95%: –0.127 to 0.160). Willingness to receive novel treatments: 82.1% participants demanded benefit in overall survival or quality of life; exigences were met in Ao = 53.6% of patients.

Conclusions:

Little agreement was observed between expectations and preferences of our onco-hematological patients and European Public Assessment Reports, according to overall survival and quality of life. Most patients preferred an improvement in quality of life, but also expected an increase in overall survival with their treatment. Almost half of patients would not meet their requirements to receive their drug when it was authorized.

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