Achieving Breakthroughs in Rare Disease Trials: Key Factors & Exclusive Webinar on Strategies for Cross-Border Participation

Despite the name “Rare Disease”, these conditions have a substantial global impact, affecting 3.5-5.9% of the total population—approximately 300 million people worldwide. As Rare Disease Day approaches on February 28th, Medpace reflects on the significant opportunity within the drug development industry to make a meaningful impact on the lives of those affected by rare diseases, many of whom have no effective treatment options available. 

Conducting clinical trials for rare diseases comes with its own set of challenges, including complex trial design, regulatory hurdles, site selection, and project management. The limited patient population adds further considerations, particularly around recruitment and retention. To navigate these challenges and accelerate rare disease clinical development, it’s essential to partner with a highly experienced CRO that can provide innovative strategies and tailored solutions.  

Continue reading to explore 4 factors for conducting successful rare disease clinical trials. 

1. Minimizing Patient Burden Through a Patient Centric Approach 

Patient recruitment and retention remain among the most significant challenges in clinical development, particularly for rare diseases. Given the daily obstacles faced by patients with rare conditions, it is essential to minimize patient burden through a patient-centric study design while ensuring the recruitment of a committed and engaged group of patients. Rare disease studies often involve pediatric patients, requiring careful attention to operational, regulatory, and ethical considerations specific to pediatric populations, including concerns of both the investigator and the caregiver. Once patients are enrolled, ongoing support becomes paramount to address the needs and concerns of the patient and caregiver, which helps to enhance patient retention and ensures the collection of high-quality data.  

2. Considerations for Complex Trial Designs  

Awareness of rare diseases is growing, and with a deeper understanding of their pathophysiology, innovative treatment options—such as gene therapies—are emerging, offering the potential for long-term symptom relief or even a definitive cure. While these breakthroughs provide new hope, they also introduce additional complexities that must be addressed. A study protocol must be scientifically robust, feasible, and provide clear guidance to investigators. Early and ongoing engagement with regulators is critical for effective planning. Additionally, choosing the appropriate patients to assess the proposed endpoints is paramount and depends heavily on the rare disease studied and the goal of the therapy. Study participants and their caregivers need to be well informed about the clinical trial process and the potential benefits and risks. Finally, a comprehensive feasibility assessment is essential for successful trial execution, particularly as study designs become increasingly complex. 

3. Strong Collaboration Between Key Stakeholders 

Fostering a collaborative approach between the Sponsor, CRO, sites, and advocacy groups is crucial. This partnership ensures that patient safety is prioritized from the start, while close operational and medical collaboration facilitates proactive planning and quick issue resolution through streamlined communication. Engagement with patient advocacy groups can enhance study awareness and understanding, which can lead to increased site and patient interest, faster recruitment, and improved retention. This collaborative approach among all key stakeholders in rare disease research has led to substantial advancements in treatment options for patients with rare diseases. 

4. Cross-Border Patient Enrollment and Retention 

Cross-border patient participation in rare disease trials enables access to a broader patient pool. Key considerations for cross-border participation include site selection, patient transportation, language and cultural barriers, specific regulatory requirements in each country, managing patient care with research participation, and patient education – each of which is critical for the success of rare disease clinical trials.  

Upcoming Rare Disease Webinar – Register Now  

To learn more about strategies for cross-border patient participation, register for this upcoming webinar on Rare Disease Day 2025. In this webinar, Medpace experts will share key cross-border enrollment and retention factors for conducting rare disease trials, covering regulatory intelligence, operational considerations, and methods for enhancing patient experience. 

Register Now> 

Your Next Rare Disease Breakthrough Starts Here 

Medpace understands the unique challenges faced by rare disease clinical trials, having conducted global trials across Phase I-IV for a broad range of rare disease indications. Drive patient recruitment and site selection for difficult-to-recruit studies with guidance from our experienced team of cross-functional experts, innovative study approaches, and strong site relationships. 

We invite you to contact our experts to learn how we can help accelerate your path to approval.  

References:

1. What is a rare disease?. Rare Disease Day. (2024, October 29). https://www.rarediseaseday.org/what-is-a-rare-disease/