“Our aim is to collaborate in order to advocate in the best interests of PNH patients worldwide”
specific objectives
Medicines
Maximising access and availability of medicines for all PNH patients & promoting research and development for medicines and procedures to treat and cure PNH
Advocating
To be actively involved in the research and development process of medicines from as early as possible in order that these medicines:
- better reflect patient unmet needs
- reflect clinical outcome measures relevant to patients
- measure patient experience of specific medicines (preferably with a PNH relevant Quality of Life measure)
- are acceptable to patients including in their delivery method
assessments
Being involved in assessments by regulatory authorities including the European Medicines Agency (EMA), Medicines Healthcare Regulatory Authority (MHRA), Health Canada and the US Food and Drug Administration (FDA) in order that the appropriate submissions from the patient perspective are made
events
Being represented at groups and events relevant to the PNH community including the International PNH Interest Group (IPIG) in order that the appropriate submissions from the patient perspective are made
educating
Educating ourselves on the latest developments in research for dissemination to our communities
awareness
Raising awareness among healthcare professionals of the symptoms of PNH in order to promote timely diagnosis and appropriate treatment
Treatment & cure
Collecting and spreading best practice of care, treatment and cure for PNH