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欧盟在2008年正式从全欧洲的战略层面开展罕见病保障专项工作，已经形成的罕见病相关工作经验，非常值得中国借鉴。通过总结欧盟罕见病概况及孤儿药目录、罕见病组织结构与战略计划、罕见病筹资支付体系和服务提供等情况，分析欧盟罕见病保障体系的优缺点，提出我国开展罕见病保障工作的实施建议，如统一罕见病的合理定义、编码与目录；建立罕见病保障工作的组织架构，制定罕见病国家（地区）计划或政策，优化罕见病服务提供，建立可持续性的罕见病筹资体系，注重罕见病工作的综合全面性和地区差异性，为提高我国罕见病防治工作的保障力度和可持续性提供参考。 相似文献
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Pascale A. Allotey Caitlin D. Allotey-Reidpath Daniel D. Reidpath 《Critical public health》2018,28(2):248-252
A resilient and responsive health system providing universal health coverage is one that is able to cope with both the commonplace conditions faced by the majority as well as rare conditions, particularly when experienced by more marginalised groups. This is critical to ensure that under the sustainable Development Goals agenda, no one is left behind. Low- and middle-income economies are in the process of refining their health systems to respond to the epidemiological and demographic transition. However, with economic development comes the requirement for an ethical transition; the need to justify, with some transparency, the allocation of resources for the less common, but often more expensive conditions. Drawing on a case study of a rare genetic condition, this paper highlights the various pathways in the system that support or hinder access to care, to identify the policy directions for rare diseases in resource constrained settings. 相似文献
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Background
The number of authorized orphan and non-orphan medicines for rare diseases has increased in Europe. Patient access to these medicines is affected by high costs, weak efficacy/safety evidence, and societal value. European health care systems must determine whether paying for expensive treatments for only a few patients is sustainable.Objectives
This study aimed to evaluate patient access to orphan and non-orphan medicines for rare diseases in 22 European countries during 2005 to 2014.Methods
Medicines for rare diseases from the Orphanet list, authorized during 2005 to 2014, were searched for in the IMS MIDAS Quarterly Sales Data, January 2005 – December 2014 (IQVIA, Danbury, CT). The following three measures were determined for each country: number of available medicines, median time to continuous use, and medicine expenditure. A medicine was considered available if uninterrupted sales within a 1-year period were detected.Results
From 2005 to 2014, 125 medicines were authorized and 112 were found in the search. Of those, between 70 (63%) and 102 (91%) were available in Germany, the United Kingdom, Italy, France, and the Scandinavian countries. These countries were also the fastest to enable continuous use (3–9 mo). Only 27% to 38% of authorized medicines were available in Greece, Ireland, Bulgaria, Romania, and Croatia, which took 1 to 2.6 years to begin continuous use. A country’s expenditure on medicines for rare diseases in 2014 ranged between €0.2 and €31.9/inhabitant.Conclusions
Patient access to medicines for rare diseases varies largely across Europe. Patients in Germany, Scandinavian countries, Switzerland, France, and the United Kingdom can access larger numbers of medicines in shorter time. 相似文献6.
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Objectives
Reimbursement recommendations on (orphan) drugs are usually made at a national level and this can lead to variation in patient access to the same drug in different countries. We compared differences in patient access to ultra-orphan drugs between countries. Furthermore, we describe how reimbursed and non-reimbursed orphan drugs differ with respect to pharmacoeconomic properties.Methods
We studied patient access to eight high-priced inpatient ultra-orphan drugs in nine countries. In addition, we determined whether differences with respect to cost per patient, budget impact and cost-effectiveness existed between orphan drugs with a positive and negative reimbursement status.Results
Reimbursement status was available for 78 orphan drugs, of which 56 (72%) were positive. Large differences were observed between countries; while two countries had a positive status for two out of nine ultra-orphan drugs, four countries had positive status for all drugs it assessed. A number of drugs were reimbursed only after price negotiations and/or through specific orphan drug policies. The average cost per patient, budget impact and incremental cost-effectiveness ratios were lower for ultra-orphan drugs with a positive reimbursement status than for those with a negative status, although only cost-effectiveness ratios were statistically significant.Conclusions
Large differences in patient access to ultra-orphan drugs were observed between countries. Future research should examine if similar findings can be seen in other countries and with other orphan drugs, and it should also determine which other factors play a role in reimbursement status of orphan drugs. 相似文献8.
Katy Benjamin Margaret K. Vernon Donald L. Patrick Eleanor Perfetto Sandra Nestler-Parr Laurie Burke 《Value in health》2017,20(7):838-855
Background
Rare diseases (RDs) affect a small number of people within a population. About 5000 to 8000 distinct RDs have been identified, with an estimated 6% to 8% of people worldwide suffering from an RD. Approximately 75% of RDs affect children. Frequently, these conditions are heterogeneous; many are progressive. Regulatory incentives have increased orphan drug designations and approvals.Objective
To develop emerging good practices for RD outcomes research addressing the challenges inherent in identifying, selecting, developing, adapting, and implementing patient-reported outcome (PRO) and observer-reported outcome (ObsRO) assessments for use in RD clinical trials.Good Practices for Outcomes Research
This report outlines the challenges and potential solutions in determining clinical outcomes for RD trials. It follows the US Food and Drug Administration Roadmap to Patient-Focused Outcome Measurement in Clinical Trials. The Roadmap consists of three columns: 1) Understanding the Disease or Condition, 2) Conceptualizing Treatment Benefit, and 3) Selecting/Developing the Outcome Measure. Challenges in column 1 include factors such as incomplete natural history data and heterogeneity of disease presentation and patient experience. Solutions include using several information sources, for example, clinical experts and patient advocacy groups, to construct the condition’s natural history and understand treatment patterns. Challenges in column 2 include understanding and measuring treatment benefit from the patient’s perspective, especially given challenges in defining the context of use such as variations in age or disease severity/progression. Solutions include focusing on common symptoms across patient subgroups, identifying short-term outcomes, and using multiple types of COA instruments to measure the same constructs. Challenges in column 3 center around the small patient population and heterogeneity of the condition or study sample. Few disease-specific instruments for RDs exist. Strategies include adapting existing instruments developed for a similar condition or that contain symptoms of importance to the RD patient population, or using a generic instrument validated for the context of use.Conclusions
This report provides state-of-the-art solutions to patient-reported outcome (PRO) and observer-reported outcome (ObsRO) assessments challenges in clinical trials of patients with RDs. These recommended solutions are both pragmatic and creative and posed with clear recognition of the global regulatory context used in RD clinical development programs. 相似文献9.
Objectives
To determine US societal burden of illness, including direct and indirect costs and annual bleed rate (ABR), for persons with hemophilia B (HB), a rare and debilitating genetic disorder, and to examine associations of hemophilia severity and treatment regimens with costs and ABR.Methods
From 2009 to 2014, the Hemophilia Utilization Group Studies Part Vb collected prospective data from 10 US hemophilia treatment centers. Participants with HB completed initial surveys on sociodemographic characteristics, clinical characteristics, and treatment patterns. During the 2-year follow-up, participants reported bleeding episodes, work absenteeism, and caregiver time quarterly. These data were used to calculate ABR and indirect costs. Direct costs were calculated using 1-year clinical chart records and 2-year dispensing records.Results
Of the 148 participants, 112 with complete medical records and one or more follow-up survey were included. Total mean annual per-person costs were $85,852 (median $20,160) for mild/moderate HB, $198,733 (median $147,891) for severe HB, and $140,240 (median $63,617) for all participants without inhibitors (P < 0.0001). Mean ABR for participants with severe HB on prophylaxis (5.5 ± 7.9 bleeds/y) was almost half that of those treated episodically. Clotting factor and indirect costs accounted for 85% and 9% of total costs, respectively. Compared with episodic treatment, prophylaxis use was associated with 2.5-fold higher clotting factor costs (P < 0.01), low but significantly more missed parental workdays (P < 0.0001) and clinician (P < 0.001) or nursing visits (P < 0.0001), less part-time employment and unemployment, and lower hospitalizations costs (P = 0.17) and ABR (P < 0.0001).Conclusions
HB is associated with high economic burden, primarily because of clotting factor costs. Nevertheless, prophylaxis treatment leads to clinical benefits and may reduce other nonfactor costs. 相似文献10.
Kay Hogan Smith 《Journal of consumer health on the Internet》2017,21(2):216-225
This column profiles three important online resources for rare disorder information. The National Organization for Rare Disorders (NORD) Rare Disease Database set the standard for thorough, detailed monographs on rare diseases starting in the 1990s. The NORD Database is complemented by the National Institutes of Health (NIH) Genetic and Rare Diseases Information Center online and the European resource, Orphanet, both of which provide much broader coverage, if less in depth than NORD’s. Reading level for all three information sources is high, and there are occasional issues with currency. However, consumer health librarians should find these organizations’ web-based databases to be reliable sources of information on health conditions that can be perplexing for both patient and health care providers alike. 相似文献