By: Juliane Mills, Therapeutic Strategy Lead, Rare Disease
More and more sponsors are stepping into the rare disease research landscape for the first time. With the various complexities and unique requirements rare disease indications demand, venturing into rare disease research can be daunting. Although these indications may be a new clinical landscape for you, at Worldwide Clinical Trials (Worldwide), our seasoned team of rare disease experts offers more than 30 years of combined experience in the space, working with sponsors like you to drive efficient, effective rare disease trials.
Understanding the Nuances of Rare Disease Research
Rare disease research requires a unique approach, one centered on specialized patient-optimized solutions. Every patient and data point is crucial in a clinical trial, especially in small populations, and trials should be designed to ease participation while optimizing enrollment and retention. Rare disease patients expect to be equal stakeholders with drug developers and clinical researchers to partner with their communities.
As a whole, rare disease communities have worked tirelessly for a seat at the research table and have much to offer clinical development. From perspectives that can increase your trial’s enrollment success to registries that can provide a head start in endpoint selection, working in a mutually beneficial way with these communities will advance your trial.
To guarantee your study is making the most of every patient and data point provided, we partner with patient advocacy organizations and patient communities to leverage your population’s insights and data. These collaborations help ensure patient perspectives are incorporated into our strategy and study design.
Rare disease research also often encompasses complicated logistics. Due to the small patient populations, potential study participants are typically geographically dispersed, requiring complex solutions like cross-border enrollment. While every indication is unique and every study requires a personalized approach, rare disease trials require strategists capable of applying lessons learned from previous trials and implementing a “solutioneering” approach. At Worldwide, it’s our methodology that drives successful rare disease programs.
Worldwide’s Methodology: Our Proven Approach and Innovative Solutions
A CRO’s methodology will be critical to the success of your rare disease trial. At Worldwide, our strategists have worked on more than 210 studies and have developed a robust methodology for impactful and successful research. Here are some of the primary factors that contribute to our rare disease methodology:
The Importance of a Customized Solution
In rare disease research, a one-size-fits-all approach is highly ineffective. Our guiding philosophy at Worldwide is, “If you’ve seen one trial for a rare indication, you’ve seen one trial.” This mindset ensures that every aspect of the trial, from study design to execution, is tailored to meet its distinct requirements.
Engagement with Patients and Advocacy Groups
As we mentioned, patients are at the core of everything we do, and understanding their needs and perspectives is crucial for the success of any rare disease trial. Our collaboration extends to more than 110 patient advocacy groups, fostering partnerships that align our efforts with the goals of all stakeholders involved.
Early Stakeholder Engagement
Proactive engagement with stakeholders is vital for rare disease trials. From patients and caregivers to Key Opinion Leaders (KOLs) and sites, early involvement leads to more informed trial designs and smoother execution. This collaborative approach helps identify potential challenges early, paving the way for successful outcomes across all parties.
Resource Utilization to Reduce Patient Burden
At Worldwide, we leverage several resources, including registries and genetic databases, to amplify our trials’ effectiveness. By reducing patient burden through resources and decentralized trial elements, we ensure participation is as seamless and accessible as possible.
Make Your Move into Rare Disease with Confidence
Our team of rare disease experts has activated over 6,100 sites, enrolled 29,500+ patients, and conducted 210+ projects across more than 60 countries. Our global experience positions us to handle the intricate details of rare disease research effectively. With our expertise and proven methodology, we can optimize the chances of your drug development program, making the transition into rare disease research smooth and efficient. From advocacy engagement and clinical development plans to regulatory guidance and clinical operations, learn more about our extensive rare disease services.
