Assess and address the lack of good quality clinical and health economic evidence using state of the art evidence generation methodologies
Provide a clear, engaging and strategic approach to communications with a wide range of stakeholders to address the poor awareness and understanding of rare conditions in the wider community
Effectively communicate the value of orphan and ultra orphan products to address the higher degree of uncertainty inherent to their evidence bases
120+
Abstracts
55+
Advisory boards/small meetings
45+
Congress-related activities (exhibition, internal, on-site, scientific coverage)
10+
Delphi Panels
100+
Design-related activities
165+
Education/Training Materials
125+
Manuscripts
10+
Oral presentations
125+
Poster presentations
15+
Lay summaries
15+
Strategic Publication Planning Support
10+
Virtual meeting/events
80+
Literature reviews [incl. critical appraisals and gap analyses]
120+
Economic Model Development and Validation
70+
HTA submissions
15+
Health Policy Projects
65+
Value Materials
25+
Statistical analyses
30+
RWE Projects
The National Institute for Health and Care Excellence (NICE) is revising its criteria for the routing of technologies to the Highly Specialised Technologies (HST) Programme. In this article, we explore NICE’s proposed changes to the criteria and provide our thoughts on what the revisions may mean for manufacturers of ultra-rare products.
Our Rare Diseases team have recently published recommendations on collecting clinical expert judgement to support the reimbursement of new rare disease therapies.
The manuscript provides actionable recommendations on collecting clinical expert judgement, and is the first to provide specific guidance to overcome the challenges commonly faced in the rare disease space.
Our Rare Diseases team are committed to actively contributing research to overcome the challenges faced by industry and the patient community. Our research contributions include peer-reviewed manuscripts, panels and posters, to high impact journals and international conferences such as ISPOR and ISMPP.
