When developing drugs for a rare condition groups need to consider which evidence will be required by health technology assessment bodies to ensure a smooth pathway for patient access. While demonstrating that a treatment has a significant impact on outcome measures assessed by clinicians is key for the success of clinical trials, it has become increasingly important to demonstrate a real impact on the lives and wellbeing of the patients themselves to ensure positive reimbursement decisions and access.
Capturing such data is more easily said than done. A number of standard tools exist which try to measure and quantify an individual’s quality of life. These can go some way to capturing the impact of a new treatment, but most measure a small number of variables, and fail to capture the subtleties that matter to a given rare disease patient population. Subsequently, many groups are now trying to devise more disease specific quality of life measures – tools designed to more accurately measure change in the outcomes that matter to their specific rare disease.
The development and validation of these tools is a significant challenge, but when done right they can offer exceptional insight into the disease state and wellbeing of rare disease patients.
John Brazier, PhD – Professor of Health Economics – University of Sheffield
Jill Carlton, PhD – Senior Research Fellow – University of Sheffield
Yvette Venable – First VP of Patient Engagement – ICER
Mel Whittington – Associate Director of Health Economics — ICER
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