Search Results (13)

Background: The continuous expansion of the National Reimbursement Drug List has led to an increasing cost disparity among alternative drugs for the same indications. Under the current proportional reimbursement mechanism, choosing higher-cost treatments often results in higher compensation. Given the lack of empirical evidence on whether income affects the medication choices of insured individuals in the Chinese context, this study aims to evaluate the impact of income levels on drug selection, providing a basis for optimizing the medical insurance reimbursement policy. Methods: This study extracts data from hospitalized patients enrolled in basic medical insurance from the China Health and Retirement Longitudinal Study (CHARLS) database and preprocesses it in Excel. Subsequently, SPSS is used to conduct descriptive statistics, difference analysis, correlation analysis, and regression analysis on the processed data to explore the impact of income levels on drug selection. Results: After controlling for length of hospitalization and hospitalization costs, the regression coefficient for urban employee basic medical insurance participants is β = 0.505 (p < 0.01), and the regression coefficient for new rural cooperative medical insurance participants is β = 0.195 (p < 0.01). This means that, regardless of whether participants are enrolled in urban employee basic medical insurance or new rural cooperative medical insurance, an increase in income will lead to higher hospitalization drug costs. Conclusions: Compared to low-income insured individuals, high-income participants in the basic medical insurance are more likely to choose higher-cost drugs among alternatives, which leads to unfair reimbursement under the current proportional reimbursement system. Full article

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. Full article

Objectives: The objective of this study is to quantify healthcare resource utilization, economic burden, and the multi-level medical security system for Spinal Muscular Atrophy (SMA) patients in Shaanxi Province, China, from a societal perspective using a survey. Methods: This observational study employed an online survey with a retrospective cross-sectional design in Shaanxi Province, China. The survey examined various aspects of SMA, including resource utilization, direct and indirect economic burdens, and co-payment mechanisms within a multi-level medical security system. Results: Following the inclusion of nusinersen in the National Reimbursement Drug List (NRDL) in 2022, the treatment rate for SMA patients increased significantly. After risdiplam was added to the NRDL in 2023, its use also saw a marked increase. Treatment costs varied by SMA type: Type 1 incurred the highest costs (RMB 300,000 or USD 41,000), followed by Type 2 (RMB 270,000 or USD 37,000), Type 3 (RMB 200,000 or USD 27,000), and Type 4 (RMB 80,000 or USD 11,000). The primary sources of costs were productivity losses due to primary caregivers (32.94%), nusinersen usage (29.29%), and risdiplam usage (17.33%). Out-of-pocket costs for SMA patients accounted for 29.29% of the total costs. In 2023, basic medical insurance covered 49% of direct costs and 32% of total costs. Patients still had to pay 25.73% of the total cost for the direct costs. Conclusions: Basic medical insurance is a critical foundation for patient security and plays a pivotal role in reimbursement. In contrast, commercial insurance has a relatively limited impact on covering the costs for SMA patients. These findings highlight the substantial healthcare burden faced by SMA patients under the current healthcare system in China. Full article

Introduction: Spinal muscular atrophy is a rare genetic disease. Nusinersen and Risdiplam, recognized as disease-modifying therapies, were included in the National Reimbursement Drug List in 2022 and 2023, respectively, in China. Policies have been implemented to enhance a multi-level medical security system, particularly for rare diseases. This study explores the self-perceived burden and offers policy suggestions to improve China’s social security for rare diseases. Methods: In our mixed study, we conducted 37 semi-structured online interviews and a quantitative survey with 3 adult SMA patients and 34 family members (primary caregivers) in collaboration with the Meier Advocacy and Support Center. The interviews explored self-perceived burdens in psychology, domestic relations, medical care, rehabilitation, and economy, analyzing mainly through thematic analysis and multiple linear regression. Results: Respondents reported significant psychological burdens mainly stemming from limited treatment access. The instability within these families was linked to inconsistent therapeutic schedules, the lack of development opportunities, and misunderstandings. Choices between institutional and home rehabilitation were influenced by economic conditions and symptom severity. After the inclusion of medications, six patients (16.2%) still had not received pharmacological treatment, and many of those who underwent treatment were dissatisfied with the outcomes. The high costs of rehabilitation, family labor loss, and an incomplete medical security system resulted in significant economic burdens. Respondents called for more effective medications and better patient support. Conclusion: Although the inclusion of medications in National Reimbursement Drug List has improved availability and affordability, families still experienced significant burdens across multiple domains. A broader focus on social security is needed to enhance the comprehensive development of patients with rare diseases. Full article

Rare cancers are defined by an annual incidence of fewer than 6 per 100,000. Bearing similarities to rare diseases, they are associated with substantial health inequalities due to diagnostic complexity and delayed access to innovative therapies. This situation is further aggravated in Southeastern European countries like Bulgaria, where limited public resources and expertise underscore the need for additional policy and translational research on rare cancers. This study aimed to explore the availability and access to orphan drugs for rare cancers in Bulgaria for the period of 2020–2023. We cross-compared data from both the European Union and national public sources to evaluate the number of available and accessible orphan drugs for rare cancers, the delay from market authorization to reimbursement, the dynamics of public expenditures, and regional disparities in access across the country. We juxtaposed the main characteristics of oncological and non-oncological orphan drugs as well. Only 15 out of 50 oncological orphan drugs that were authorized by the European Medicine Agency were accessible for rare cancer patients in Bulgaria. The median delay between market authorization and inclusion in the Bulgarian Positive Drug List was 760 days. The total expenditures for all orphan drugs for rare cancers amounted to EUR 74,353,493 from 2020 to 2023. The budgetary impact of this group rose from 0.24% to 3.77% of total public medicinal product expenditures for the study period. Rare cancer patients represent a vulnerable population that often faces limited to no access to treatment. We call for targeted European and national policies to address this major inequality. Full article

Gene therapies (GTs) have recently emerged as revolutionary personalized therapeutic options. Despite their promising potential, challenges such as uncertainty regarding long-term health benefits and safety, along with extreme price tags, pose significant obstacles to patient access. Within the EU, the European Medicines Agency plays a pivotal role with regards to GT market authorization. However, national authorities are responsible for pricing and reimbursement, which results in fragment patient access within the EU. This study aimed to provide an overview of the complex landscape of post-market authorization accessibility for GT products in Bulgaria, comparing it with neighboring EU countries. We applied a mixed-methods approach, including desk research, public data requests, and list price comparisons. As of 1 April 2023, 14 GTs had a valid market authorization at the EU level. In Bulgaria, Kymriah^® was the only GT included in the Positive Drug List (PDL), with an official list price of EUR 335,636.94. Similar results were found in Romania, whereas five GTs were included in Greece’s PDL. Additionally, Zolgensma^® was found accessible in Bulgaria through an alternative individual access scheme at an estimated price of EUR 1,945,000.00. In conclusion, this study emphasized targeted policy interventions to address health inequalities and to ensure timely access to GTs within the EU. Full article

Background and objectives: Multiple reforms aimed at improving the Chinese population’s health have been introduced in recent years, including several designed to improve access to innovative drugs. We sought to review current factors affecting access to innovative drugs in China and to anticipate future trends. Methods: Targeted reviews of published literature and statistics on the Chinese healthcare system, medical insurance and reimbursement processes were conducted, as well as interviews with five Chinese experts involved in the reimbursement of innovative drugs. Results: Drug reimbursement in China is becoming increasingly centralized due to the removal of provincial pathways, the establishment of the National Healthcare Security Administration and the implementation of the National Reimbursement Drug List (NRDL), which is now the main route for drug reimbursement in China. There is also an increasing number of other channels via which patients may access innovative treatments, including various types of commercial insurance and special access. Health technology assessment (HTA) and health economic evidence are becoming pivotal elements of the NRDL decision-making process. Alongside the optimization of HTA decision making, innovative risk-sharing agreements are anticipated to be increasingly leveraged in the future to optimize access to highly specialized technologies and encourage innovation while safeguarding limited healthcare funds. Conclusions: Drug public reimbursement in China continues to align more closely with approaches widely used in Europe in terms of HTA, health economics and pricing. Centralization of decision-making processes for public reimbursement of innovative drugs allows consistency in assessment and access, which optimizes the improvement of the Chinese population’s health. Full article

Drug pricing methods vary extensively across countries. Japan calculates drug prices using cost accounting and based on the efficacy of similar drugs. This study investigated the relationship between drug prices and their clinical efficacy and usefulness using public information on anticancer drugs reimbursed by the National Health Insurance price listing between January 2009 and March 2020. We investigated drug characteristics, prices, and clinical benefits based on overall survival (OS) and progression-free survival (PFS). Eighty anticancer drugs were approved in Japan during the study period. The largest number (28 drugs, 35.0%) was approved based on PFS, 18 (22.5%) were approved based on OS, and 13 (16.3%) based on the response rate. The mean (±SD) drug price was JPY 88,416.2 (±148,974.7), while the median drug price (with quartiles) was JPY 21,694 (JPY 4855.0–JPY 93,396.8). Drug prices were significantly higher for PFS than for OS, while cost index—the drug price to extend PFS or OS by one day—did not differ significantly between PFS and OS. The relationship between the 46 drugs approved based on OS or PFS and their prices was examined. A correlation was found between drug prices and their clinical usefulness in terms of OS but not PFS. Full article

Background: The overexpression of the human epidermal growth factor receptor-2 (HER2) gene is present in 20~25% of breast cancer (BC) patients, contributing to an inferior prognosis. Recent clinical trials showed that pyrotinib has promising antitumor activities and acceptable tolerability for those patients (ClinicalTrials.gov, NCT03080805 and NCT02422199). Therefore, this study aims to assess the cost-effectiveness of pyrotinib plus capecitabine versus lapatinib plus capecitabine for patients with HER2-positive metastatic BC after prior trastuzumab. Methods: A lifetime-partitioned survival model was established to evaluate health and economic outcomes with different treatment strategies. The primary outcome was the incremental cost-effectiveness ratio (ICER). Data were derived from the published literature, clinical trials, expert opinions, and other local charges. Sensitivity analyses were performed to assess the robustness of the findings. Scenario analyses were developed to make further evaluations. Results: The pyrotinib regimen had significant advantages over the lapatinib regimen after enrolling in the National Reimbursement Drug List (NRDL), with cost savings of USD 15,599.27 and a gain of 0.53 QALYs. Meanwhile, before enrolling in NRDL, the pyrotinib regimen afforded the same QALYs at a higher incremental cost of USD 45,400.64 versus the lapatinib regimen, producing an ICER of USD 85,944.79 per QALY. Scenario analyses yielded similar results. Sensitivity analyses suggested stability in the cost-effectiveness findings. Conclusions: Compared to lapatinib plus capecitabine, the pyrotinib plus capecitabine enrolled in NRDL is a cost-effective alternative second-line treatment for patients with HER2-positive metastatic BC in China. Full article

The Canadian system for approval of new cancer drugs is complex with multiple steps. Health Canada grants a license for a drug to be marketed and prescribed. The Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d’excellence en santé et services sociaux (INESSS) make recommendations by way of health technology assessments (HTA). If positive, the latter then lead to confidential price negotiations at the pan-Canadian pharmaceutical alliance (pCPA), after which individual provinces and territories make a listing decision. Delays can occur at each stage, but post-HTA delays can be lengthy and unpredictable, denying or impeding access to an effective drug with the potential for devastating clinical outcomes. Conditional funding models have been adopted in a number of European countries with the goal of providing timely access to new medications in areas of unmet need, in advance of further steps in the reimbursement process. This manuscript discusses different stakeholder perspectives on conditional funding agreements—including a recent successful example of such a process in the UK—based on a panel discussion at the 2021 Canadian Association of Population Therapeutics (CAPT) Conference. Full article

Introduction: Real-world data indicate disparities in biologic access across Europe. Objectives: To describe the national structure of PsA care in Poland, with a particular focus on the population of inadequate responders (IRs) and difficulties associated with biologic therapy access. Methods: A pool of rheumatologic and dermatologic care centers was created based on National Health Fund contract lists (n = 841), from which 29 rheumatologic and 10 dermatologic centers were sampled randomly and successfully met the inclusion criterium. Additionally, 33 tertiary care centers were recruited. For successful center recruitment, one provider had to recruit at least one patient that met the criteria for one of the four pre-defined clinical subgroups, in which all patients had to have active PsA and IR status to at least 2 conventional synthetic disease-modifying drugs (csDMARDs). Self-assessment questionnaires were distributed among physicians and their patients. Results: Barriers to biologic DMARD (bDMARD) treatment are complex and include stringency of reimbursement criteria, health care system, logistic/organizational, and personal choice factors. For patients who are currently bDMARD users, the median waiting time from the visit, at which the reimbursement procedure was initiated, to the first day of bDMARD admission was 9 weeks (range 2–212; 32% < 4 weeks, 29% 5–12 weeks, 26% 13–28 weeks, 13% with >28 weeks delay). Out of all inadequate responder groups, bDMARD users are the only group with “good” therapeutic situation and satisfaction with therapy. Patient satisfaction with therapy is not always concordant with physician assessment of therapeutic status. Conclusions: Despite the fact that over a decade has passed since the introduction of biologic agents, in medium welfare countries such as Poland, considerable healthcare system barriers to biologic access are present. Out of different IR populations, patient satisfaction with treatment is often discordant with physician assessment of disease status. Full article

This study reviews and evaluates the national drug formulary system used to improve patient access to new drugs by making reimbursement decisions for new drugs as part of the South Korean national health insurance system. The national health insurance utilizes three methods for improving patient access to costly drugs: risk-sharing agreements, designation of essential drugs, and a waiver of cost-effectiveness analysis. Patients want reimbursement for new drugs to be processed quickly to improve their access to these drugs, whereas payers are careful about listing them given the associated financial burden and the uncertainty in cost-effectiveness. However, pharmaceutical companies are advocating for drug prices above certain thresholds to maintain global pricing strategies, cover the costs of drug development, and fund future investments into research and development. The South Korean government is expected to develop policies that will improve patient access to drugs with unmet needs for broadening health insurance coverage. Simultaneously, the designing of post-listing management methods is warranted for effectively managing the financial resources of the national health insurance system. Full article

Background: Pharmaceutical companies design clinical development programs to generate the data that they believe will support reimbursement for the experimental compound. Objective: The objective of the study was to present a process for using multicriteria decision analysis (MCDA) by a pharmaceutical company to estimate the probability of a positive recommendation for reimbursement for a new drug given drug and environmental attributes. Methods: The MCDA process included 1) selection of decisions makers who were representative of those making reimbursement decisions in a specific country; 2) two pre-workshop questionnaires to identify the most important attributes and their relative importance for a positive recommendation for a new drug; 3) a 1-day workshop during which participants undertook three tasks: i) they agreed on a final list of decision attributes and their importance weights, ii) they developed level descriptions for these attributes and mapped each attribute level to a value function, and iii) they developed profiles for hypothetical products ‘just likely to be reimbursed’; and 4) use of the data from the workshop to develop a prediction algorithm based on a logistic regression analysis. The MCDA process is illustrated using case studies for three countries, the United Kingdom, Germany, and Spain. The extent to which the prediction algorithms for each country captured the decision processes for the workshop participants in our case studies was tested using a post-meeting questionnaire that asked the participants to make recommendations for a set of hypothetical products. Results: The data collected in the case study workshops resulted in a prediction algorithm: 1) for the United Kingdom, the probability of a positive recommendation for different ranges of cost-effectiveness ratios; 2) for Spain, the probability of a positive recommendation at the national and regional levels; and 3) for Germany, the probability of a determination of clinical benefit. The results from the post-meeting questionnaire revealed a high predictive value for the algorithm developed using MCDA. Conclusions: Prediction algorithms developed using MCDA could be used by pharmaceutical companies when designing their clinical development programs to estimate the likelihood of a favourable reimbursement recommendation for different product profiles and for different positions in the treatment pathway. Full article