Due to legislation and incentives to drug developers, the rare disease market is experiencing unprecedented growth and the current return on investment now makes it more favorable for both small and large biopharmaceutical companies to invest into the space. Due to the small numbers of patients available for rare disease clinical trials, recruitment and retention remain critical challenges to successful rare disease drug development.
Listening to what patients with rare disease say about their health-related needs and preferences can speed drug development and decrease the burden on patients. Most regulators today expect patient-led initiatives, and companies that step up are seeing strong return-on-engagement. To accelerate rare (and ultra-rare) disease drug development, the patient voice must be incorporated into every aspect of the process.
Download the paper below to learn how integrating the patient voice in rare disease drug development impacts not only the patient, but also the regulators and payers--during and after commercialization.