Selecting the right Contract Research Organization (CRO) for rare disease programs can be daunting for sponsors. Rare diseases come with unique challenges, from small patient populations to complex regulatory landscapes. To navigate these hurdles, it’s essential to find a CRO that not only understands the intricacies of rare disease research but also aligns with your program’s specific needs. Based on insights shared in Ergomed’s recent webinar, we’ve outlined five critical factors to consider when choosing a CRO partner for your rare disease study.
1. Prior Experience with Rare Diseases Matters — But It’s Not All About Specific Indications
While looking for a CRO with experience in your exact indication is tempting, the sheer diversity of rare diseases means this may not always be feasible. There are over 7,000 recognized rare diseases globally, affecting an estimated 400 million people. Most CROs won’t have experience in every rare indication. Instead, focus on whether the CRO has broader expertise in the rare disease space.
As highlighted by Juliette Moritz, Senior Vice President of Strategic Solutions and Patient Centricity at Ergomed, the real value lies in the CRO’s familiarity with the methodologies, challenges, and regulatory processes specific to rare diseases. Whether it’s lysosomal storage disorders, pediatric rare conditions, or rare oncology indications, CROs with a track record in related therapeutic areas can often transfer their expertise to your program.
Key takeaway: Look beyond exact indication experience. Assess a CRO’s capability to manage rare disease programs holistically, leveraging their experience in related conditions and patient populations.
2. Regulatory Knowledge is Crucial for Rare Disease Success
Rare disease trials often involve navigating a complex and varied regulatory landscape. From orphan drug designations to accelerated pathways, having a CRO with deep regulatory expertise is vital. Benjamin Rossano, Director of Rare Disease Strategy at Ergomed, emphasized the importance of choosing a CRO that understands the nuances of different regional regulatory requirements.
For instance, rare disease definitions and approval processes vary significantly between the U.S., EU, China, and Japan. A CRO with global regulatory experience can help streamline submissions, avoid delays, and optimize the pathway to market approval.
Key takeaway: Ensure your CRO has comprehensive regulatory expertise across different regions to help your rare disease program reach its milestones efficiently.
3. Robust Patient Recruitment Strategies are Non-Negotiable
Patient recruitment is often the most significant bottleneck in rare disease trials. With patient populations being geographically dispersed and often limited to a few dozen individuals globally, a strategic approach to recruitment is critical. Ergomed’s experts highlighted that successful CROs don’t rely solely on big data for feasibility studies but engage directly with investigators, patient advocacy groups, and specialized sites.
Effective recruitment also requires patient-centric trial designs that minimize the burden on participants and their families. For rare diseases, where patients may have already endured years of diagnostic delays and numerous clinical visits, reducing the burden can significantly enhance recruitment and retention.
Key takeaway: Opt for a CRO with a strong feasibility process and patient engagement strategy to maximize your trial’s success.
4. A Patient-Centric Approach is Essential
Rare disease patients and caregivers often face long diagnostic journeys and complex treatment regimens. The CRO you choose should demonstrate a patient-centric approach, not just in rhetoric but in practice. This includes designing trials that minimize patient inconvenience, offering travel support, and incorporating flexible protocols that accommodate patients’ needs.
Juliette Moritz stressed that placing patients at the center of trial designs is crucial to improving patient engagement and data quality. Patient-centric CROs are more likely to anticipate challenges related to patient recruitment and retention, ensuring that trials are both efficient and empathetic.
Key takeaway: Prioritize CROs that go the extra mile in reducing patient burden, thereby increasing enrollment rates and improving data integrity.
5. Leverage Networks and Conferences to Identify Potential CRO Partners
Finding the right CRO goes beyond looking at directories and online databases. As Ergomed’s experts suggested, attending conferences like the World Orphan Drug Congress or disease-specific symposia can provide valuable opportunities to engage with CROs directly. Networking with key opinion leaders, site coordinators, and other sponsors can also offer insights into which CROs have a strong reputation in the rare disease space.
Additionally, utilizing industry benchmarking reports and client satisfaction surveys can provide an objective perspective on a CRO’s performance. These resources can help narrow down your options and identify partners who are not just competent but also collaborative and patient-focused.
Key takeaway: Use a mix of industry networks, conferences, and benchmarking data to vet CROs and find a partner who aligns with your rare disease program’s goals.
Conclusion: Aligning with the Right CRO is Key to Rare Disease Trial Success
Selecting the right CRO for your rare disease program can be the difference between a successful trial and one fraught with delays and challenges. By focusing on the five key factors outlined above — broad rare disease experience, regulatory expertise, patient recruitment strategies, patient-centricity, and leveraging industry networks — you can better position your program for success.
To dive deeper into these considerations, watch Ergomed’s webinar for expert insights and best practices.