We are entering an exciting time for orphan drugs, with many of them reaching the end stage in their development and set to come to market in the near future. Research, development and the approval of orphan drugs for both rare oncological and non-oncological diseases are predicted to increase over the next few years, with orphan drugs accounting for a fifth of prescription drug sales by 2024.
As of 2019, 14 gene therapy, cell therapy and tissue engineering products (including Kymriah®, LUXTURNA® and Zynteglo®) were granted marketing authorisation in Europe for the treatment of different orphan diseases.
With the growth of orphan drugs and their approval, effective communication of rare disease and the associated drugs is vital to ensure patients and healthcare providers (HCPs) can make informed decisions about their use.
We know that patients with rare diseases present unique challenges that can affect the success of important communications to them, including a lack of disease awareness and knowledge from HCPs, inadequate diagnosis, small patient populations, limited clinical data and poorly defined patient pathways.
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