What we do

Specific objectives of the PNH Global Alliance include the following:

  • maximising access and availability of medicines for all PNH patients
  • promoting research and development for medicines and procedures to treat and cure PNH
  • advocating for PNH patients to be actively involved in the research and development process of medicines from as early as possible in order that these medicines
    1. better reflect patient unmet needs
    2. reflect clinical outcome measures relevant to patients
    3. measure patient experience of specific medicines (preferably with a PNH relevant Quality of Life measure)
    4. are acceptable to patients including in their delivery method
  • being involved in Healthcare Technology Assessment (HTA) agency processes (approval for payment) in order that the appropriate submissions from the patient perspective are made
  • 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 Adminstration (FDA) in order that the appropriate submissions from the patient perspective are made
  • 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 ourselves on the latest developments in research for dissemination to our communities
  • raising awareness among healthcare professionals of the symptoms of PNH in order to promote timely diagnosis and appropriate treatment
  • collecting and spreading best practice of care, treatment and cure for PNH