Background/Objectives: Orphan medicinal products offer essential treatments for rare diseases, but patient access varies across European Union countries despite a common regulatory framework. In Bulgaria, access is primarily through inclusion in the positive drug list following health technology assessment or via individual access schemes under Ordinance No. 2/2019, which allows for ad hoc reimbursement. This study evaluates the timeliness and extent of Bulgarian patient access to orphan-designated drugs authorized by the European Medicines Agency. Methods: We analyzed European Medicines Agency-authorized orphan drugs between July 2006 and September 2023 using data from the European Medicines Agency, Bulgarian health technology assessment bodies, positive drug list records, and individual access scheme reports. Medians, interquartile ranges, stratified analyses, and permutation/bootstrapping methods were applied. Results: Of the 142 European Medicines Agency-approved orphan drugs, only 41 (28.9%) were included in the Bulgarian positive drug list. The median time to positive drug list inclusion was 828 days, with pre-health technology assessment delays (median 570 days) as the main bottleneck. Health technology assessment evaluations had a median duration of 204 days. Cancer and accelerated-assessment drugs reached health technology assessment faster, while conditional approvals faced longer delays. Twenty-four drugs were accessed through individual schemes; twenty remained outside the positive drug list. Overall, 43.0% of orphan drugs reached Bulgarian patients via either mechanism. Conclusions: Access to orphan drugs in Bulgaria is limited and delayed, mainly due to pre-health technology assessment lags. In light of the forthcoming European Union health technology assessment regulation, Bulgaria must ensure that national processes are capable of rapidly translating centralized assessments into meaningful patient access.

Background/Objectives: Despite major advances in precision medicine, equitable and timely access to innovative oncology therapies remains challenging because of differences in reimbursement systems, health technology assessment (HTA), and healthcare organization. Physicians are well positioned to identify barriers affecting the implementation of these therapies in routine practice. This study evaluated Bulgarian physicians’ perceptions of innovative oncology therapies, assessed patient access, identified barriers to timely availability, and examined factors perceived as associated with these perceptions. Methods: A cross-sectional web-based survey was conducted between March and May 2025 among physicians involved in oncology care in Bulgaria. The questionnaire comprised six domains addressing professional characteristics, self-perceived knowledge, clinical implementation, perceptions of patient access, barriers to implementation, and policy priorities. Descriptive statistics, Pearson’s chi-square tests, Cramér’s V, and multivariable ordinal logistic regression were used to analyze the data. Results: A total of 114 physicians participated. Respondents generally reported moderate-to-high levels of self-perceived knowledge regarding innovative oncology therapies. Self-perceived knowledge was higher among medical oncologists (adjusted p < 0.001) and physicians treating at least 100 patients with cancer per month (adjusted p = 0.003). Overall, 57.9% of respondents (95% CI: 48.7–66.6%) rated the perceived timeliness of access as good or very good, while 68.4% (95% CI: 59.4–76.2%) reported encountering patients who sought treatment abroad because innovative therapies were perceived as unavailable or insufficiently accessible in Bulgaria. Professional experience (aOR = 0.68, 95% CI: 0.50–0.93; p = 0.014) and higher monthly patient volume (aOR = 0.45, 95% CI: 0.21–0.96; p = 0.039) were independently associated with more favorable perceptions of access. Financial constraints, regulatory and HTA-related delays, and perceived delays in pharmaceutical-company submissions were the principal perceived barriers. Conclusions: Bulgarian oncology specialists demonstrate high self-reported professional readiness for innovative oncology therapies; however, the perceived timely patient access remains constrained primarily by financial, organizational, and reimbursement-related factors. Although, these findings reflect physicians’ perceptions and do not establish causal determinants of access, strengthening reimbursement pathways, HTA implementation, molecular diagnostic capacity, and clinical research infrastructure may improve equitable access to innovative oncology therapies within the evolving European HTA framework.

Background: Budget Impact Analysis is an integral part of the Health Technology Assessment in Bulgaria, informing reimbursement decisions of the National Health Insurance Fund. Inaccurate projections risk both fiscal unsustainability and restricted patient access to innovation. Yet projection accuracy methods remains uncertain, particularly given limited epidemiologic data and evolving clinical use. Objectives: This study aimed to assess the empirical validity of Health Technology Assessment budget-impact projections for medicines approved in 2019 by comparing projected patient volumes and expenditures with real-world National Health Insurance Fund reimbursements through 2025, and to identify drivers of divergence across therapeutic areas and reimbursement channels. Methods: We conducted a retrospective cohort analysis linking 2019 Health Technology Assessment submissions with monthly National Health Insurance Fund claims for both hospital and outpatient reimbursement channels. Actual utilization was calculated as the annualized median number of treated patients per month. Projected costs were derived by multiplying HTA-projected patient volumes by the observed unit cost per patient-month. We quantified deviations using observed-to-projected ratios and absolute gaps and assessed the relationship between projected and actual expenditures using a log–log regression model. Results: By September 2025, realized volumes typically exceeded projections (median ratio 1.6; range 0.02–21.3). Large overshoots were observed for Avelumab, Risankizumab, and Guselkumab; Cobimetinib and Abemaciclib remained below forecast. Expenditure deviations were driven predominantly by volume: immunology (+€17.4 million) and oncology (+€5.0 million) accounted for the largest absolute gaps. Elasticity was near proportional overall (β = 1.002; standard error = 0.24; R² = 0.50), lower in hospitals (β = 0.79; p = 0.055) and higher in outpatient care (β = 1.30; p = 0.003). Conclusions: Health Technology Assessment Budget Impact Analyses captured broad cost scaling but systematically missed product-specific uptake, with deviations largely volume-driven. Strengthening national registries and real-world data pipelines, and adopting dynamic, indication-responsive contracting and forecasting, could materially improve budget predictability while preserving access to innovation.