EU Committee of Experts on Rare Diseases (EUCERD), Global Public Policy & Government Relations, SOBI - Swedish Orphan Biovitrum, Tomtebodvägen, 23a, Solna, Sweden.
Orphanet J Rare Dis. 2012 Sep 26;7:74. doi: 10.1186/1750-1172-7-74.
Since its enactment in 2000, the European Orphan Medicinal Products Regulation has allowed the review and approval of approaching 70 treatments for some 55 different conditions in Europe. Success does not come without a price, however. Many of these so-called "orphan drugs" have higher price points than treatments for more common diseases. This has been raising debate as to whether the treatments are worth it, which, in turn risks blocking patient access to treatment. To date, orphan drugs have only accounted for a small percentage of the overall drug budget. It would appear that, with increasing numbers of orphan drugs, governments are concerned about the future budget impact and their cost-effectiveness in comparison with other healthcare interventions. Orphan drugs are under the spotlight, something that is likely to continue as the economic crisis in Europe takes hold and governments respond with austerity measures that include cuts to healthcare expenditures. Formally and informally, governments are looking at how they are going to handle orphan drugs in the future. Collaborative proposals between EU governments to better understand the value of orphan drugs are under consideration. In recent years there has been increasing criticism of behaviours in the orphan drug field, mainly centring on two key perceptions of the system: the high prices of orphan drugs and their inability to meet standard cost-effectiveness thresholds; and the construct of the system itself, which allows companies to gain the benefits that accrue from being badged as an orphan drug. The authors hypothesise that, by examining these criticisms individually, one might be able to turn these different "behaviours" into criteria for the creation of a system to evaluate new orphan drugs coming onto the market. It has been acknowledged that standard methodologies for Health Technology Assessments (HTA) will need to be tailored to take into account the specificities of orphan drugs given that the higher price-points claimed by orphan drugs are unlikely to meet current cost-effectiveness thresholds. The authors propose the development of a new assessment system based on several evaluation criteria, which would serve as a tool for Member State governments to evaluate each new orphan drug at the time of pricing and reimbursement. These should include rarity, disease severity, the availability of other alternatives (level of unmet medical need), the level of impact on the condition that the new treatment offers, whether the product can be used in one or more indications, the level of research undertaken by the developer, together with other factors, such as manufacturing complexity and follow-up measures required by regulatory or other authorities. This will allow governments to value an orphan drug that fulfilled all the criteria very differently from one that only met some of them. An individual country could determine the (monetary) value that it places on each of the different criteria, according to societal preferences, the national healthcare system and the resources at its disposal - each individual government deciding on the weighting attributed to each of the criteria in question, based on what each individual society values most. Such a systematic and transparent system will help frame a more structured dialogue between manufacturers and payers, with the involvement of the treating physicians and the patients; and foster a more certain environment to stimulate continued investment in the field. A new approach could also offer pricing and reimbursement decision-makers a tool to handle the different characteristics amongst new orphan drugs and to redistribute the national budgets in accordance with the outcome of a differentiated assessment. The authors believe that this could, therefore, facilitate the approach for all stakeholders.
自 2000 年颁布以来，欧洲罕见病药品法规允许在欧洲范围内对近 70 种治疗方法进行评估和批准，这些治疗方法适用于约 55 种不同的疾病。然而，成功并非没有代价。这些所谓的“孤儿药”中有许多比治疗更常见疾病的药物价格更高。这引发了人们对于这些药物是否物有所值的争论，而这反过来又有可能阻碍患者获得治疗。迄今为止，孤儿药仅占整体药物预算的一小部分。似乎随着孤儿药数量的增加，政府越来越担心未来的预算影响以及它们与其他医疗干预措施相比的成本效益。孤儿药成为关注焦点，随着欧洲经济危机的加剧以及政府采取包括削减医疗支出在内的紧缩措施，这种情况可能会继续下去。政府正在正式和非正式地考虑未来如何处理孤儿药。欧盟政府之间提出了一些合作提案，以更好地了解孤儿药的价值，目前正在审议之中。近年来，孤儿药领域的行为受到了越来越多的批评，主要集中在该系统的两个关键观点上：孤儿药的高价及其无法达到标准的成本效益阈值；以及该系统本身的构建，这使得公司能够从被贴上孤儿药的标签中获得好处。作者假设，通过单独检查这些批评，人们或许能够将这些不同的“行为”转化为评估新上市孤儿药的标准。人们已经认识到，需要针对卫生技术评估（HTA）的标准方法进行调整，以考虑到孤儿药的特殊性，因为孤儿药声称的较高价格不太可能达到当前的成本效益阈值。作者提出了基于几个评估标准的新评估系统的开发，该系统将作为成员国政府在定价和报销时评估每个新孤儿药的工具。这些标准应包括罕见性、疾病严重程度、其他替代方法的可用性（未满足的医疗需求程度）、新药对疾病的影响程度、产品是否可用于一种或多种适应症、开发者开展的研究水平，以及其他因素，例如监管或其他机构要求的制造复杂性和后续措施。这将使政府能够非常不同地评估符合所有标准的孤儿药，与仅符合部分标准的孤儿药相比。一个国家可以根据社会偏好、国家医疗体系和可支配资源，确定其对每个不同标准的（货币）价值——每个政府根据每个社会最看重的标准，决定对每个相关标准的权重。这样一个系统和透明的系统将有助于在制造商和支付方之间建立一个更有条理的对话，同时让治疗医生和患者参与其中，并为鼓励继续在该领域投资创造一个更确定的环境。一种新的方法还可以为定价和报销决策者提供一种工具，以处理新孤儿药之间的不同特点，并根据差异化评估的结果重新分配国家预算。作者认为，这因此可以为所有利益相关者提供便利。
