According to DHSC, the purpose of this update is to review progress over the past year in terms of rare disease strategy and to also take a forward look on actions for 2019-20.
The update covers five main areas:
- empowering those affected by rare diseases
- identifying and preventing rare diseases
- diagnosis and early intervention
- coordination of care
- the role of research
MAP has examined the strategy plan and highlighted points that may be of interest to MAP Online members. In regard to reviewing the past year, the policy paper noted increased engagement with the Early Access Medicines Scheme.
“Engagement has increased through work with the Early Access Medicines Scheme, participation in National Institute for Health and Care Excellence (NICE) scoping exercises and ad hoc round table discussions.”
In addition, the paper looked ahead to the forthcoming year in terms of the Accelerated Access Pathway:
“Continue to be involved in working group discussions between all partners (including MHRA, NICE, NHS England/I and NIHR) on the development of the Accelerated Access Pathway (lead by the Office of Life Sciences)”
For further information relating to rare disease medicines, read MAP’s recent report Access to Orphan Medicines: A Case for Change