罕见疾病

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.