E-Book: Your Strategy for Patient-Forward Rare Disease Research

5 Steps to Designing and Operationalizing Meaningful Rare Disease Drug Trials

Michael Murphy, MD, PhD

A successful, patient-forward program goes beyond patient feedback and gathers input from all stakeholders surrounding the patient in order to produce actionable insights to direct your study to success. This e-book shows you how to:
• Utilize patients as SMEs to inform your program
• Develop fit-for-purpose endpoints, guided by your stakeholders
• Solve statistical challenges threatening your study’s success
• Design feasible study plans for patients (and sites!)
• Increase your chances of market success

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Michael Murphy, MD, PhD
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Michael Murphy, MD, PhD

Chief Medical and Scientific Officer
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