21 research outputs found
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A pragmatic patient-reported outcome strategy for rare disease clinical trials: application of the EORTC item library to myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia.
BackgroundNovel, pragmatic, patient-centered strategies are needed to ensure fit-for-purpose patient-reported outcomes (PRO) instruments in clinical trial research for rare diseases such as myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). The objective of the current study was to select supplemental items to add to the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life-Core 30 (QLQ-C30) to ensure content coverage of all important clinical concepts in patients with higher-risk (HR) MDS, low-blast count (LB) AML, and CMML, thus, improving the instrument's ability to detect clinically meaningful treatment benefit for this context of use.MethodsOur mixed methods approach comprised literature review, clinician consultation (n = 3), and qualitative and quantitative analysis of two stages of patient interview data (n = 14, n = 18) to select library bank items to supplement a generic cancer PRO, the EORTC QLQ-C30.ResultsUnique symptom (n = 54) and impact (n = 72) concepts were organized into conceptual frameworks of treatment benefit, compared with EORTC QLQ-C30 items and conceptual gaps identified. Supplemental items (n = 13) addressing those gaps were selected from the EORTC Item Library and tested with patients. Supplemental item endorsement frequencies met World Health Organization Quality of Life criteria, suggesting good targeting and relevance for this sample. However, three supplemental items were confirmed as problematic based upon cognitive debriefing results, and expert clinical consultations. Ultimately, 10 supplemental items (n = 7 symptom; n = 3 impact) were selected for the MDS/AML/CMML context.ConclusionSupplemental items were selected to enhance the conceptual coverage of the EORTC QLQ-C30 in the areas of fatigue, shortness of breath, and functioning
Budget impact analysis of rituximab biosimilar in Italy from the hospital and payer perspectives
Introduction: This article aims at investigating the 5-year budget impact of rituximab biosimilars in Italy. Methods: A budget impact analysis model was developed in accordance with the International Society For Pharmacoeconomics and Outcomes Research recommendations. Drug acquisition and drug administration costs were considered since the risk/benefit profile of biosimilars and the originator was assumed to be overlapping. The perspectives of hospitals and payers were used. Input data were retrieved from the literature and validated/integrated by an expert panel of seven clinicians from various Italian regions. A dynamic incidence-based approach was used. Results: From the hospital perspective, adopting a rituximab biosimilar would produce savings of €79.2 and €153.6 million over 3 and 5 years, respectively. The results are very similar if the payer perspective is considered, with a cumulated savings of about €153.4 million in 5 years. Lymphoma and chronic lymphocytic leukaemia would account for the most significant savings. Discussion: Despite its limitations, this study provides the first Italian evaluation of the financial impact of rituximab biosimilars and also incorporates the effects of biosimilars on the pricing strategies of the originator (dynamic impact). This dynamic effect is more relevant than the impact of the treatment shift from the originator to biosimilars. Our hope is that these savings will be used to cover new cost-effective drugs and not just for cost-cutting policies