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5 Principles of Our Rare Disease Methodology That Drive Success for Our Sponsors

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills

When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. The team’s ability to apply insights and proven strategies from their experience in rare disease research brings concepts to reality, getting medications to patients faster.

As the strategists for Worldwide Clinical Trials’ rare disease sponsors, we design customized solutions that make a difference in achieving successful outcomes. Our approach is informed by our combined 30+ years in rare disease clinical development and our time in various stakeholder roles, such as scientists, CRAs, and project managers. Together, we have touched more than 200 studies per year and consolidated our knowledge into five principles that inform the methodology we use today:

1.The only solution is a custom solution.

At our core, we understand that the one-size-fits-all approach falls short. Each patient population requires a solution as distinct as the condition it addresses. This philosophy is encapsulated in our belief: “If you’ve seen one trial for a rare indication, you’ve seen one trial.”

It’s about understanding a trial’s individual components—the research objectives, the biology of the disease, the specific needs of the patient population, regional treatment options, the current competitive landscape—and then building a customized, informed solution.

2. Patients are at the core of everything we do.

We recognize that patients are not just participants in a study; they are its very foundation. A trial that doesn’t meet the patient’s needs is bound to lose direction or be time-consuming and costly. From recruitment to retention and beyond, ensuring the study provides value to the patient and their care circle is not just beneficial; it’s imperative to what drives successful outcomes for our sponsors. Patients navigating life with rare diseases are experts in their own right, and they offer a unique resource for CROs and pharmaceutical companies to help support study success.

3. Common goals empower change.

Driving the development of rare treatments is a joint effort between patients, sponsors, and advocacy groups, with often many overlapping goals that can be harnessed for mutual advancement. We foster enduring partnerships with more than 110+ advocacy groups, providing our support and resources to help them grow. We also cultivate transparent and collaborative relationships with our sponsors and focus on relationship-building over transactional delivery. Further, our relationships with sites are supportive and built to last—our commitment to relationship-building extends through every layer of the trial.

4. Engage stakeholders EARLY.

Our approach is to be proactive, not reactive. Early engagement with all stakeholders, including patients, caregivers, Key Opinion Leaders (KOLs), sites, vendors, and regulatory bodies, provides a strong foundation for a study. This intentional engagement paves the way for more informed trial designs, smoother execution, and, ultimately, more successful outcomes with each stakeholder.

5. Leverage innovative solutions.

As we craft custom solutions, we harness an array of resources that amplify our trials’ effectiveness. We’ve prioritized understanding these diseases, staying abreast of recent developments in the clinical landscape, locating patients, and creating access pathways by leveraging resources like registries and genetic databases. Moreover, our dedication to reducing patient burden is reflected in our use of enabling support, such as concierge resources and decentralized trial elements. To us, innovation does not end—it is a continuous part of our work to personalize solutions.

6. Every Patient Counts

In the realm of rare diseases, our focus is on unique and precious opportunities. With rare disease studies, we delve into orphan indications using specialized products tailored for these individual cases, often encountering a limited pool of patients. Ensuring patient retention throughout the entirety of the study is paramount to achieving statistical significance and unlocking breakthroughs. In addition, we believe protecting your endpoints will be a key to your success. Learn more about our clinical assessment services.

Discover Worldwide’s Methodology

While a depth of experience in rare disease research is undeniably valuable, it is the methodology—a company’s approach to each trial—that makes the difference. At Worldwide, our team applies a holistic, patient-centric methodology, fortified by strong relationships and a wealth of experience, to navigate the complexities of rare disease trials. It is this approach that sets us apart, ensuring that each trial we undertake is crafted with the precision, care, and empathy it deserves. We never forget that patients and families are waiting for these treatments now. It drives us every day in the work that we do.

We’d love to discuss how our approach can support your program. Please reach out to learn more and see what we can do for you. Contact us!