tag rare-disease-framework 
Pharmaceutical Technology
FEBRUARY 10, 2023
This is not the first treatment to come with a high price tag. However, more highly innovative/high-cost treatments, within infectious disease and neurology space, are likely to enter the market for rare disorders, increasing the number of non-oncology RSAs.
XTalks
FEBRUARY 27, 2024
Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy.
Pharmaceutical Technology
JANUARY 27, 2023
On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. A refund up to 12 inpatient doses will also be offered for patients whose disease worsens while receiving treatment.
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