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:
- Patient advocacy in action: Why we are doing this – setting the scene.
- PNH basics: Biology, clinical mechanisms, treatment landscape, and unmet patient needs.
- How medicines development works and the importance of patient engagement, with a focus on PNH drug development.
- Quality of Life and Patient-Reported Outcomes (PROs).
- Key elements of clinical trial protocols, including study endpoints.
- Effective online communication and tools.
- Health technology assessment
- Community Advisory Boards
The in-person training sessions in Madrid, held before the European Hematology Association (EHA) Congress 2024 (June 12-16), covered:
- Collaborating with industry.
- Evidence-based patient advocacy.
- Case study: Review of a PNH clinical trial protocol and informed consent form (ICF).
- Statistics and their relevance to advocacy.
- 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.