PNH Advocate Development Programme

Launched in December 2023, the PNH Advocate Development Program (ADP) is the first disease-specific training initiative for patient advocates in Paroxysmal Nocturnal Hemoglobinuria (PNH). This hybrid program offers a comprehensive education on PNH-specific topics, such as disease understanding, current treatments, and regulatory pathways, alongside cross-disease topics like clinical research and patient organization management.

The webinars have covered the following topics:

  1. Patient advocacy in action: Why we are doing this – setting the scene.
  2. PNH basics: Biology, clinical mechanisms, treatment landscape, and unmet patient needs.
  3. How medicines development works and the importance of patient engagement, with a focus on PNH drug development.
  4. Quality of Life and Patient-Reported Outcomes (PROs).
  5. Key elements of clinical trial protocols, including study endpoints.
  6. Effective online communication and tools.
  7. Health technology assessment
  8. Community Advisory Boards

 

The in-person training sessions in Madrid, held before the European Hematology Association (EHA) Congress 2024 (June 12-16), covered:

  1. Collaborating with industry.
  2. Evidence-based patient advocacy.
  3. Case study: Review of a PNH clinical trial protocol and informed consent form (ICF).
  4. Statistics and their relevance to advocacy.
  5. Reading scientific papers and posters.

Participants have access to an online classroom with recordings, training materials, tests, and recommended resources. This combination of virtual and in-person learning equips advocates with the knowledge and skills to engage both locally and globally.