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欧盟委员会澄清有关《罕见病药物管理规定》的问题

阅读: 2017年01月12日

欧盟委员会澄清有关《罕见病药物管理规定》的问题


在2015年11月进行公众咨询之后,欧盟委员会(EC)发布了一份新的通讯,旨在澄清有关欧盟《罕见病药物管理规定》的一些悬而未决的问题。

咨询旨在解决自《罕见病药物管理规定》于2000年1月生效以来出现的五大问题:

● 澄清“显著益处”的含义;
● 如何将《罕见病药物管理规定》应用于欧盟内部不存在的新出现的疾病,如埃博拉病毒;
● 在同时评估用于同一适应症的两个产品的情况下,是否应当赋予罕见病药物资格的灵活性;
● 如果申请人在首次上市许可后扩展其产品的用途,如何重新评估产品的罕见病药物资格;

● 澄清不同申请人之间转让罕见病药物资格的流程。


欧盟委员会澄清有关《罕见病药物管理规定》的问题

据欧洲药品管理局(EMA)的科学和监管管理部门负责人Jordi Llinares透露,该机构正在努力实施该通讯带来的变化。

显著益处


要获得罕见病药物资格,药物必须治疗、诊断或预防严重的或威胁生命的且在欧盟影响不到千分之五的人群的疾病。 

此外,针对该罕见疾病,在欧盟内部不得有任何“令人满意的”治疗方法,或者必须证明该药物相对于现有选择能够为患者带来显著益处。

根据该通讯,申请人必须基于临床相关的优势(例如与现有治疗相比改善的疗效或更好的安全性)或对患者护理的重大贡献(例如证明能够改善依从性的新药物形式)来证明显著益处。

但是,该通讯澄清,显著益处不应基于:

●  “由于现有授权产品或仅在一个或数量有限的会员国授权的现有产品的短缺而可能增加的供应/可用性。(如果申请人有患者伤害的证据,则可以例外);
● 根据相关的人用医药产品委员会(CHMP)指南对产品的药物质量的提高(这是所有上市许可持有人的义务);或者
● 新的药物形式、新的剂量规格或新的给药途径,除非对患者护理能够带来重大贡献;或者
●  一种替代的作用机制本身。然而,在特殊情况下,在授予罕见病药物资格时可以考虑这些发展。在以授予上市许可为目的进行标准审查时,必须将其转化为临床相关的优势或对病人护理的重大贡献。”

该通讯还规定,医院或药房根据指令2001/83/EC第3(7)条准备的药物“不应被视为诊断、预防或治疗某种疾病的令人满意的方法”,以便确立优于现有治疗方案的显著益处。

欧盟以外的流行率


该通讯涉及的另一个问题与如何处理不影响欧盟内部人群身体状况的罕见病药物资格有关。

这个问题是根据2014-2015年埃博拉疫情提出的,该疫情影响了西非数以万计的人,但没有在欧盟境内传播。

根据该通讯,用于这类疾病的药物有资格作为罕见病药物,因为欧盟以外的疾病流行率不是罕见病药物资格的考虑因素。

该通讯指出,“在符合所有其他标准以及《罕见病药物管理规定》的利益的情况下,根据流行率标准,旨在诊断、预防或治疗在某些非欧盟国家中影响大量人群但在欧盟中具有低流行率或流行率约为零的病症的药物可能有资格被指定为罕见病药物。”

然而,对于欧盟内零流行率的病症,委员会指出,申请人应考虑欧盟内部人群未来可能受到该疾病影响的风险。

用于同一罕见病适应症的两个产品的同时申请


该通讯还澄清了欧洲药品管理局(EMA)同时评估用于同一罕见病适应症的两个产品的情况下维持罕见病药物资格的问题。

“如果欧洲药品管理局同时收到针对同一病症的两份上市许可申请,可能不会并行。在这种情况下,由于可用的信息有限,与第一个产品相比,第二个产品可能难以显示出显著的益处,”委员会写道。

委员会指出,在这种情况下,第二个产品的申请人无需证明优于第一个产品的显著益处。

然而,委员会指出,到罕见病药物委员会(COMP)重新评估第二个产品的资格认定标准时,如果第一个产品的上市许可通告已经在《欧盟官方公报》上发布,那么第二个产品的申请人则需要证明优于第一个产品的显著益处,尽管委员会指出这可以使用间接比较来完成。

扩 展


委员会还澄清,如果某种罕见病药物的适应症发生变化(如:新的适应症、II型变化或上市许可延长),则必须重新评估该罕见病药物资格。

委员会指出,该验证将“确保罕见病药物上市许可条款的变化符合第7(3)条”,该条款规定,罕见病药物的上市许可只能涵盖满足罕见病药物标准的适应症。

不同申请人之间转让罕见病药物资格


虽然《罕见病药物管理规定》第5(11)条允许在不同申请人之间转让罕见病药物资格,但委员会澄清,“不能将罕见病药物资格转让给已经拥有相同药品上市许可的申请人。”


英语原文


European Commission Clears up Questions on Orphan Drug Regulation

Posted 05 January 2017 By Michael Mezher

Following a public consultation in November 2015, the European Commission (EC) has released a new communication intended to clarify some lingering questions about the EU's Orphan Regulation.

The consultation sought to address five issues that have arisen since the Orphan Regulation came into effect in January 2000:

● Clarifying the meaning of "significant benefit"
● How to apply the Orphan Regulation to emerging diseases, such as Ebola, that are not present within the EU
● Whether flexibility should be applied in granting orphan designation in cases where two products for the same indication are assessed in parallel
● How to reassess a product's orphan designation when a sponsor extends the use of its product after the initial marketing authorization
● Clarifying the process of transferring orphan designations between sponsors

According to Jordi Llinares, head of the department for scientific and regulatory management at the European Medicines Agency (EMA), the agency is working to implement the changes brought on by the communication.

Significant Benefit

To qualify for orphan designation, a medicine must treat, diagnose or prevent a serious or life threatening condition that affects fewer than 5 in 10,000 people in the European Union. 

Additionally, there must not be any "satisfactory" treatment for the condition within the EU, or the medicine must be shown to demonstrate significant benefit to patients over existing options.

According to the communication, sponsors must demonstrate significant benefit based on /html/list_1677.htmlly relevant advantages such as improved efficacy or a better safety profile compared to existing treatments, or a major contribution to patient care, such as a new pharmaceutical form that is demonstrated to improve adherence.

However, the communication clarifies that significant benefit should not be based on:

● "Possible increased supply/availability due to shortages of existing authorised products or to existing products being authorised in only one or a limited number of Member States. (Exceptions may be made if the sponsor has evidence of patient harm);
● enhancement of the pharmaceutical quality of a product in compliance with relevant Committee for Medicinal Products for Human Use (CHMP) guidelines – this is an obligation for all marketing authorisation holders; or
● a new pharmaceutical form, a new strength or a new route of administration, unless it brings a major contribution to patient care; or
● an alternative mechanism of action per se. However, in exceptional cases consideration may be given to those developments at the time the designation is granted. At the time the criteria are reviewed for the purposes of granting the marketing authorisation, this must translate into a /html/list_1677.htmlly relevant advantage or a major contribution to patient care."

The communication also establishes that drugs prepared by hospitals or pharmacies under Article 3(7) of Directive 2001/83/EC "should not be considered a satisfactory method of diagnosis, prevention or treatment of a condition" for the purposes of establishing significant benefit over existing treatment options.

Prevalence Outside the EU

Another question addressed by the communication has to do with how the orphan designations for conditions that do not affect people within the EU are handled.

The question was raised in light of the 2014-2015 Ebola outbreak, which affected tens of thousands of people in West Africa, but was not transmitted within the EU.

According to the communication, such diseases do qualify for consideration as orphan products as the prevalence of the disease outside of the EU is not a consideration for orphan designation.

"A medicinal product intended to diagnose, prevent or treat a condition which affects a large number of people in certain non-EU countries, but which has a low prevalence or a prevalence of approximately zero in the EU, may be eligible for designation as an orphan medicinal product with respect to the prevalence criterion, and if all other criteria are met, eligible for the benefits set out in the Regulation," the communication states.

However, for conditions with zero prevalence within the EU, the Commission notes that sponsors should account for the risk that people within the EU may be affected by the disease in the future.

Parallel Orphan Applications

The communication also clarifies questions about maintaining orphan designation in cases where two products for the same orphan indication are being assessed by EMA at the same time.

"Where two applications for marketing authorisation for the same condition have been received by the European Medicines Agency at the same time, they might not remain in parallel. In such cases, it may be difficult for the second product to show significant benefit as compared with the first product due to the limited information available," the Commission writes.

In such cases, the Commission says the sponsor of the second product will not be required to demonstrate significant benefit over the first product.

However, the Commission says that the sponsor of the second product will need to demonstrate significant benefit over the first product if the notification of marketing authorization for the first product has already been published in the Official Journal of the European Union by the time the Committee for Orphan Medicinal Products (COMP) re-evaluates the second product's designation criteria, though the Commission notes that this may be done using an indirect comparison.

Extensions

The Commission has also clarified that an orphan product's designation must be reassessed when there is a change to its indication, such as a new indication, type-II variation or extension of the marketing authorization.

This verification, the Commission says, will be done "to ensure that the variation of the terms of the orphan marketing authorisation complies with Article 7(3)," which establishes that the marketing authorisation for an orphan product may only cover indications that meet the criteria for an orphan product.

Transfer of Orphan Designation Between Sponsors

While Article 5(11) of the Orphan Regulation allows an orphan designation to be transferred between sponsors, the Commission has clarified that "it is not possible to transfer an orphan designation to a sponsor who already has a marketing authorisation for the same medicinal product and condition."




内容来自:RAPS

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