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Objectives: The aim of the study was to assess the reimbursement policy for orphan drugs (ODs) in selected European countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedures and reimbursement decision-making. Materials and Methods: A list of authorized ODs was extracted from a web-based registry of the European Medicines Agency, including information on active substance, Anatomical Therapeutic Chemical (ATC) classification code, and therapeutic area. A country-based questionnaire survey was conducted between September 2022 and September 2023 among selected experts from 12 European countries. A descriptive and statistical analysis was performed to identify correlations between country characteristic, HTA procedures, drug indication and positive recommendations or reimbursement decisions for ODs. Results: Safety assessment for ODs was mandatory in 10 countries, while it was optional in one country (Italy) and not required in one country (Iceland). Efficacy assessment for ODs was mandatory in 11 countries and not required in one country (Iceland). The impact of safety and efficacy assessment on reimbursement decisions was rated as high in 10 countries and as low in one country (Germany). Dedicated OD legislation and policies were reported in seven countries. In two countries (Belgium, Iceland), the HTA was not mandatory, and in one country (Germany), it only had an informative function. A positive recommendation (from an HTA agency or advisory body) guaranteed reimbursement in four countries, while a negative recommendation excluded reimbursement only in one country (Iceland). The proportion of reimbursed ODs ranged from 23.5% in Iceland to 86% in Germany (p < 0.001). ODs with ATC code L represented the largest group of medicines (n = 49). They were also very frequently reimbursed ODs in the countries studied, with a mean of 61.8% (p < 0.001). Conclusion: European countries differ in terms of the impact of clinical issues and additional clinical aspects on the reimbursement policy for ODs. Reimbursement decisions were affected by OD-specific legislation, policies, and EMA authorization status. HTA dossiers and procedures significantly influenced reimbursement decisions, although some ODs were reimbursed regardless of the positive or negative recommendations. ATC codes were significantly correlated with reimbursement status and positive recommendation. [ABSTRACT FROM AUTHOR]

With this quantitative research, we made an attempt to investigate physicians’ prescribing decision-making characteristics and generic medicines’ favorability. Specifically, we investigated physicians’ attitudes toward: (a) generic medicine companies brands, (b) generic medicines companies’ communications, (c) physician-prescribing criteria, (d) key success factors that may increase generic medicines’ prescriptions and (e) potential generic medicines’ deficits. Moreover, we searched for significant differences among the research participants’ responses, in order to categorize them into meaningful groups. To this end, we addressed to a random sample of 250 physicians, which yielded a 42.4% response rate. The study involved extensive literature review and augmented quantitative data analysis, comprising reliability assessment and cluster analysis, among others. Research findings indicated participants clustering into three segments, that is, Generic Advocates, Generic Slacks and Generic Skeptics, reflecting distinct physician prescribing decision-making archetypes, with distinct prescribing criteria, favorability on various companies’ communication policies and prescribing behavior. The implication is that generic pharmaceutical companies may use the proposed physician segmentation in order to match their marketing strategies with the respective physician’s archetypes, regarded as generics’ purchasing decision-making actors. The paper also discusses research limitations and directions for future research. [ABSTRACT FROM AUTHOR]