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Abstract
Decision making around funding and access of rare disease products is a well-known challenge for healthcare systems worldwide. Generation of clinical evidence is not straightforward, due to inability to perform randomised controlled trials, lack of comparators and/or use of surrogate endpoints. From an economic perspective, high “price density” with high one-off upfront costs adds to the existing challenge. This research aims to explore potential solutions that can remove barriers to fund and make rare disease drugs available to patients.
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