Less than 5% of rare diseases have an approved treatment, highlighting the urgent need for improved patient identification and engagement in clinical trials. Survey data reveals that adults may wait up to five years for a diagnosis, with nearly half experiencing misdiagnosis, underscoring the emotional toll on patients.
Yet less than 5% of rare diseases ... approved treatment.3 Even when effective therapies and drugs exist, reaching the right patients who need them is difficult. Survey data show a long and emotional journey for those awaiting a diagnosis. It can take up to five years for adults, and nearly half will receive a misdiagnosis.4 · Rare diseases challenge the standard ways of operating. They require the industry to better identify target patient populations for trials and keep them ... Yet less than 5% of rare diseases have at least one approved treatment.3 Even when effective therapies and drugs exist, reaching the right patients who need them is difficult. Survey data show a long and emotional journey for those awaiting a diagnosis. It can take up to five years for adults, and nearly half will receive a misdiagnosis.4 · Rare diseases challenge the standard ways of operating. They require the industry to better identify target patient populations for trials and keep them engaged throughout the study across geographies or time zones. Life sciences leaders are overcoming challenges to speed the development, launch, and education of novel medicines for rare diseases. The impact could include greater patient retention during rare disease studies, an improvement that can increase access to life-enhancing new treatments. Reflecting on her own experiences as a rare disease patient, Helen Shaw, co-founder of the virtual site VCTC, says, “I see how hard it is to take part in a clinical trial. Once a medicine is approved, it is critical to seamlessly transition from trial to treatment, given the smaller patient cohort, as the physicians who conduct the trials often become prescribers. The good news is that the industry is making significant progress in how medicines for rare diseases are developed and brought to market.
Original title: “Accelerating Time to Treatment in Rare Disease | Applied Clinical Trials Online”