The Path to Rare Disease Clinical Trial Innovation
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For decades, the pharmaceutical industry has faced the same recurring problems with clinical development: the struggle to fully recruit and retain enough patients, meet target timelines, and have trials conclude on time.
Certainly, the industry does overestimate its ability to recruit, but a bigger issue is that study designs and protocol development seemingly fail to truly reflect patients’ lives, or account for the reality in the clinic. In fact, data shows the probability of success for any clinical development effort is 6.2% for orphan drug trials, compared with 13.8% overall, which translates to a 93.8% failure rate for orphan drug development efforts.