Increasing adoption of digital trials and data-driven commercial strategies has laid the foundations for improved pinpointing of treatments and speedier diagnoses.
Though estimates show that there are now up to 30 million people in the European Union living with a rare disease, progress with diagnosis remains worryingly slow, with patients suffering as a result.1 Healthcare professionals (HCPs) must consider between 6,000 to 8,000 rare diseases when diagnosing a patient, with the result being that it is often common for them to fail to spot the symptoms within relatively small patient populations (five or fewer in 10,000). This means patients commonly wait years for a diagnosis or may never be diagnosed at all.
Dignosis Pharma companies trying to deliver effective treatments for rare diseases are searching for the proverbial needle in the haystack. During development, it’s a challenge to find, recruit, and retain patients for trials. Getting to a viable product requires seamless coordination of clinical data, document review, and regulatory submissions across multiple functions. In the commercial phase, medical science liaisons (MSLs) have to identify and engage the right experts to shape their medical strategy. After launch, many field teams struggle with access: 65% of accessible HCPs meet with three or fewer pharma companies in Europe.2 It’s unsurprising that 95% of rare diseases have no approved treatment.3
With the odds stacked against them, pharma companies of all sizes need to unlock the clinical and commercial value of their vital work swiftly. Here’s how some leaders are transforming their approach to data and technology to speed the delivery of new treatments to patients.
Boosting the Development Pipeline
To run successful clinical trials, CROs must be able to quickly and cost-effectively recruit participants with rare diseases across diverse locations. Fortunately, the industry has increasingly leveraged digital trials to find patients situated across the globe whilst minimising expenses. That being said, difficulty arises when it comes to retaining their involvement in a trial, especially if the trial is site-based and their health worsens.
To improve retention, patients deserve more choice over how to participate in studies from one day to the next, whether in person or through digital means, based on their daily health status and personal preferences. Wearable technology, electronic Patient-Reported Outcomes (ePRO), and remote monitoring devices can help track patients’ health status. But technology needs to keep pace with these scientific developments. Traditional electronic data capture (EDC) systems no longer give study teams the whole picture, and data managers lack the tools to manage non-traditional data at scale.
