AJMC November 7, 2024
Patient engagement is a necessity when performing health technology assessment to help better assess and identify the outcomes of a rare disease.
Introduction: Challenges of Conducting Health Technology Assessment in Rare Diseases
Rare diseases present distinct challenges to health technology assessment (HTA), also known as value assessment. HTA helps determine the value of new health care treatments, such as drugs or medical devices, by evaluating how well the treatment works and its net cost. Decision-makers use this information to help guide treatment decisions and determine how treatments should be covered. However, for rare diseases, traditional HTA methods often fail to capture the outcomes that matter most to patients. Limited patient populations, scarce data, and diverse clinical manifestations make it challenging...
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