An NIHR-funded research programme evaluating England’s Rare Diseases Action Plans, co-led by Realise Advocacy and Consilium Scientific.
Josie Godfrey leads the policy and research design as Co-Lead Investigator, while Lindsay Birrell leads 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.
We were invited to present the findings directly to NICE’s Technology Appraisal team and committee members. The report and subsequent discussions with NICE’s PPIE team informed work to improve support for patient organisations in access processes
