An NIHR-funded research programme evaluating England’s Rare Diseases Action Plans, co-led by Realise Advocacy and Consilium Scientific.
Josie Godfrey served as co-lead investigator on research design, while Lindsay Birrell led the Patient and Public Involvement and Engagement (PPIE) and peer-researcher programme.
The work has developed a practical approach to evaluating complex health policy and generated recommendations for the Department of Health and Social Care.
Commissioned by a specialist pharmaceutical company, we developed a practical toolkit to help European rare disease patient organisations understand and navigate HTA and access processes.
The toolkit included step-by-step guidance, terminology, evidence generation checklists and practical tools to support engagement with decision makers.
We worked with the board of a rare disease charity to help prepare the organisation for emerging drug development and access processes.
Beginning with an audit of skills, capacity and the treatment pipeline, we delivered interactive workshops and developed an action plan covering community engagement, lived-experience evidence gathering and effective participation in HTA.
Sponsored by the BioIndustry Association (BIA), we interviewed patient advocacy leaders to understand the organisational impact of participating in HTA.
The resulting report was presented to NICE and highlighted the need for independent capacity building and practical support for patient organisations engaging in access processes.
