New Medicines, Novel Insights: Advancing Rare Disease Development

Novel therapies for rare diseases offer hope to thousands of patients and their families. Bringing these treatments to market swiftly is a primary goal for every biopharmaceutical company, including Parexel. However, speed and efficiency in drug development are valuable only when grounded in a thorough understanding of the illness from the perspectives of patients and caregivers. In this report, we share best practices illustrating how companies can embed patient centricity throughout the interconnected processes of drug development.

Achieving this is easier said than done. Despite the concept of patient centricity being over a decade old, the biopharmaceutical industry still lags behind other sectors in focusing product development on the people it serves. Why do many companies struggle to engage effectively with patients before designing trials and selecting endpoints? One reason is that multiple stakeholders often have divergent and even conflicting priorities. Additionally, it takes significant time and money to understand a rare disease and its progression. Many companies also find it daunting to communicate with patients, fearing that direct collaboration could raise legal or ethical issues.

While individual stakeholders can often recognize signs of improved quality of life for a given patient, collectively, they struggle to do so. Moreover, companies cannot make a drug commercially viable if it does not comply with technical standards, but this compliance is irrelevant if the product fails to address patient needs. Therefore, focusing on patients from the earliest stages of development makes practical sense.