Rare diseases present unique challenges for health technology assessment (HTA), a critical tool for evaluating the value of new treatments. Traditional HTA methods often fail to capture the full impact of treatments on patients and caregivers, particularly in rare diseases where data is limited and outcomes are highly individualized. A patient-centered approach is essential to ensuring assessments reflect what matters most to those living with these conditions.
A recent commentary published in The American Journal of Managed Care (AJMC), by Michelle Cheng, the Center’s Research Manager, explores why early and continuous patient engagement is key to improving HTA frameworks for rare diseases.
Read the full article here.