Cumulatively, there are nearly 7000 rare diseases currently affecting between 25 to 30 million Americans today. A condition or disorder is classified as rare when it affects fewer than 200,000 individuals; these may include certain cancers, genetic disorders like Duchenne Muscular Dystrophy, and individual lysosomal storage disorders. Given the complexity of studying diseases with so few patients on record, sponsors often need to rely on the expertise and resources of a contract research organization (CRO) to achieve success. The benefit of CROs, especially those with rare disease specialties, is that they come equipped with experienced trial teams, offer tailored patient-centric technology, and often have an existing global support network. Here we present the five most common challenges of rare disease clinical trials and how sponsors can overcome them by leveraging a strong CRO partnership.
1 | Quality Site Selection
One of the most pressing challenges for sponsors in any clinical trial is identifying the right clinical sites early to prepare for success from the start. The difficulty rare disease clinical research presents is that many sites may not have the required specialty experience or facilities. Enlisting the help of a CRO’s existing network of qualified sites presents an easier solution for pharmaceutical companies. Resources like Vial’s Preferred Site Network can match sponsors with top-performing sites with a proven track record of conducting complex studies with efficiency and skill. Some CROs will also offer support teams to guide sites through rigorous training processes and even patient concierge groups to better coordinate communication with patients throughout a trial.
2 | Lack of Precedence
Unlike conditions like diabetes or hypertension, rare diseases are labeled as such because there is a limited understanding of their pathophysiology and natural history. This makes drug discovery infinitely more challenging because the lack of drug development precedence introduces ambiguity and guesswork in choosing a rare disease trial’s endpoints. A specialized rare disease CRO’s input can help guide sponsors with their biomarker development and use of validated clinical assessments to ensure the study’s endpoints are appropriately chosen. For example, they may already have experience leveraging large observational patient registries to better understand disease progression and expected clinical outcomes. This is crucial for improving the chances of reaching market approval because the protocol design will be optimized to collect higher-quality clinical data.
3 | Small Patient Populations in Rare Disease Clinical Trials
It’s well-known already that patient recruitment is the rate-limiting factor to account for when attempting to avoid delays in a clinical trial’s timeline. This is especially true for rare disease clinical trials, when certain conditions are so uncommon that few cases have even been captured on record. As with any other study, CROs are a valuable resource for sponsors to boost enrollment because many have developed their own digital tools to support electronic health record data mining, multi-channeling marketing, and online referrals to connect sites with more distant patients. An experienced niche CRO may also leverage their familiarity with local and national patient advocacy groups to strengthen the study’s credibility and present clinical trial options to individuals early after a diagnosis.
4 | Study Startup Delays
When approaching a new study, any number of issues can arise because clinical trials are complex with multiple moving parts for a sponsor to oversee, especially those for rare diseases. For one, by outsourcing to their CRO’s experienced project team, sponsors can save time and money in training their own individuals to manage study startup activities. Combined with beginning site selection early, this partnership streamlines the process of training coordinators and investigators on complex protocols associated with rare diseases prior to activation. Given how sparsely spread these populations can be, leaning on a CRO during startup is especially beneficial because there is more time to accommodate the challenges of recruitment.
5 | Specialized Rare Disease CRO Expertise
Rather than spending time looking for individuals, technologies, and vendors who can meet their needs, sponsors can instead outsource it through a harmonious collaboration with globally active CROs like PPD, Vial, and IQVIA. A rare disease CRO’s specialized expertise is especially beneficial when considering how today’s clinical trials must be mindful of a protocol design’s impact on patient participation burden. For example, these companies are increasingly introducing more digital variety to streamline data and patient monitoring, and reduce the need for excessive on-site patient visits. A seasoned CRO’s resources can also connect sponsors with preferred vendors who provide patients and sites with access to specialty equipment often needed to study orphan diseases.
The Vial Rare Disease CRO
Vial is a full-service CRO that recognizes the central role of technology in advancing our understanding of rare diseases and is paving the way for modernized clinical research through digital innovation. Trusted by leading sponsors, our specialized teams deliver shorter study timelines, quality affordable services, and a clinical trial experience that puts you first. Contact a team member today to discover how we can help!
