Studying rare diseases in real-world evidence often comes with challenges, such as lack of disease-specific diagnosis codes and complex definition of relevant endpoints. Over the years, Quantify has faced these challenges on several occasions together with our clients – a demanding but also rewarding puzzle, as the need to generate evidence for the benefit of patients with rare diseases is high.
We are therefore especially excited to have attended last month’s 𝗡𝗼𝗿𝗱𝗶𝗰 𝗥𝗮𝗿𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲 𝗦𝘂𝗺𝗺𝗶𝘁 to learn more about initiatives that are underway to facilitate the study of rare diseases in RWE. Rare diseases share many scientific challenges and the emergence of rare disease quality of care registers, and broader use of ORPHA codes are steps in the right direction.
Quantify aims to be an active partner in the development of RWE for rare diseases, whether in collaboration with industry, patient organizations, data holders or academia. Let’s connect along the way and always feel free to reach out to us!