Next steps for highly specialised medicines, regulation and treatment
Morning, Thursday 19 May
This WHF event will discuss the future landscape, navigating changes in approach, and addressing challenges in orphan drug development.
Delegates will discuss the impact of changes to evaluation in improving patient access and engagement in the context of the Rare Diseases Action Plan.
They will also consider priorities for investment, collaboration and achieving value for money – as well as the latest thinking on effective managed access programmes, following the consultation held on the new Innovative Medicines Fund.
Overall, areas for discussion include:
• evaluation – the impact of the new regulatory approach
• research – support for innovation – investment priorities – positioning the UK an attractive place to launch new treatments
• orphan drug development – new ways of addressing the challenges
• value for money – key considerations in the provision of highly specialised treatments
• safety – regulation and keeping pace with innovation
• patients – use of real-world evidence within evaluations – delivering increased use
• managed access – assessing what works well
• genomics – taking forward potential opportunities
• the UK’s global role – international development and regulation of highly specialised medicines
There will be keynote sessions with: Dr Richard Scott, Chief Medical Officer, Genomics England; and Sheela Upadhyaya, Rare Diseases and Rapid-C19 Strategic Advisor, Centre for Health Technology Evaluation, NICE.
Find out information on registering here.