罕见疾病

了解罕见疾病行业面临的独特挑战是 Costello Medical 跨学科团队致力于罕见疾病的专业基础。我们在罕见疾病领域积累了丰富的工作经验,具备在临床或卫生经济证据不足、对疾病认知理解不足的情况下自如应对各种挑战的能力和策略,能够协助客户加速审批流程,帮助患者快速获取延长生命的新疗法

罕见疾病

我们在罕见疾病领域的经验包括:

多种孤儿药和超级孤儿药适应证,包括罕见肿瘤、儿科疾病和遗传疾病

为制药行业、公共卫生和慈善部门的众多客户提供科学支持

在整个临床开发生命周期中提供高质量的项目支持

早期证据生成与合成
  • 疾病负担综述
  • 早期定价模型
  • 差距分析
  • 科学建议
医学沟通
  • 专家咨询会
  • 多方利益相关者研讨会
  • 出版计划、学术论文和会议支持
  • 患者材料
统计分析
  • 注册研究和试验数据
  • 患者报告结局数据
  • 调查分析
真实世界证据
  • 专家共识讨论会 (Delphi panels)
  • 成本和资源使用研究
  • 问卷设计
市场准入
  • 战略建议
  • 证据生成
  • 价值定位、筹备价值文档
药品报销
  • 卫生经济建模
  • 系统性文献综述
  • 全球卫生技术评估,包括 NICE 高度专业化技术评估

请查看 服务&专长 页面以了解更多信息

哪些特质让我们脱颖而出?

  • 为您配备一支了解您临床及卫生经济需求的项目团队
  • 清晰透明的项目预算
  • 高品质和卓越的客户服务
  • 致力于在罕见疾病领域开展研究和公益项目
Rare Diseases

如果您想了解项目报价或更多详情,请联系 Ma Qian 马茜

案例展示

为 NICE 高度专业化技术评估 (HST) 申报准备资料

In seeking reimbursement in the UK for a novel treatment in a rare, paediatric, neurodegenerative condition, we developed a suite of materials in support of a submission to the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies (HST) programme. These materials included clinical and economic systematic literature reviews, a cost-effectiveness model, and statistical analyses of patient-reported outcome (PRO) data.

Our dedicated rare diseases team drew upon expertise from across our Evidence Development, Health Economics and Statistics divisions to combine the relevant technical skills required to deliver this project. By working on both clinical and health economic components, we were able to efficiently collate and critically evaluate all available evidence to ensure seamless and well-coordinated completion of high-quality deliverables. We worked closely with our client to find solutions to the numerous challenges associated with developing a health economic model in a rare disease, including innovative methods for sourcing utility values and running Delphi panels to obtain inputs from leading global experts in the therapy area.

在血友病领域组织医学教育活动、准备培训材料

We have a broad range of experience working in the area of haemophilia, in particular in delivering project types that fall within our Medical Affairs services offerings. We have organised numerous medical education events and advisory boards, where support is provided across both logistical aspects, as well as the close collaboration with key clinical experts for the development of scientific programmes and content. In addition, we have developed interactive training materials for use by internal client teams, coupling creativity with scientifically rigorous content to maximise learning.

Through these collaborations, we have gained extensive experience in a competitive, high-profile rare disease, in which there is a rapidly changing treatment landscape. Therefore, we are perfectly placed to apply our experience and knowledge to the full spectrum of rare diseases, and confidently tackle the range of challenges posed by each indication and treatment.

为药物重定位提供经济学证据

We have a long-term collaboration with the rare disease charity Findacure, a UK charity that builds the rare diseases community to drive research and develop treatments. Our ongoing work with Findacure has included health economic models, design materials and publications on a pro bono and paid basis.

As part of our work with Findacure we have developed three cost of illness/budget impact models in congenital hyperinsulinism, Wolfram syndrome and Friedreich’s ataxia, which provide valuable and novel economic evidence for drug repurposing in rare diseases in support of Findacure’s innovative social impact bond project. We have subsequently supported Findacure in the presentation and communication of the models at a number of conferences, including the annual ISPOR European Congress and DPharm Europe, and been actively involved in discussions with NHS England to make this concept a reality for patients.