5 Areas Where CROs Are Making a Difference in Rare Disease Research

Rare disease research is becoming increasingly crucial for the biopharmaceutical industry. Several factors have contributed to this rise, including favorable government policies, extended exclusivity periods, and lower developmental costs. There’s also a huge unmet need. Consider that up to 95% of rare diseases don’t yet have an FDA-approved treatment

Despite all these incentives, rare disease research significantly lags research for more common diseases. But that’s changing, thanks in part, to Contract Research Organizations (CROs). These entities are uniquely equipped to provide expertise in areas that often face clinical trial-related obstacles. Here, we explore 5 areas where CROs are making a difference in rare disease research.

1. Specialized Expertise

More than 25 million Americans are living with a rare disease –– a group of about 7,000 disorders that affect fewer than 200,000 individuals. CROs bring specialized knowledge and expertise that accelerates rare disease research. For instance, these organizations:

• Know how to design and execute studies in small patient populations
• Are familiar with rare disease-specific endpoints and biomarkers
• Have strong relationships with advocacy groups that can improve study design and enhance patient recruitment efforts

Further, many CROs collaborate with full-time and fractional subject matter experts. Access to these specialists makes it easier to balance resources and tailor research to the specific patient population being served.  

2. Access to Global Patient Networks

Since rare diseases affect fewer than 200,000 people, many biopharmaceutical companies struggle to find and recruit diverse patient populations. Unfortunately, this lack of diversity hampers research. If clinical trials don’t represent the full spectrum of race and gender, results are more likely to be inaccurate and potentially harmful. Different populations metabolize drugs differently and may experience dissimilar side effects. 

CROs can address some of these issues by increasing access to diverse patient populations. Since many of these organizations have representatives around the globe, it’s easier for them to engage rare disease patients across multiple regions and gather more robust data. Many CROs also use proprietary tools and digital platforms, that make it easier to target and recruit ideal candidates.

3. Navigating Regulatory Challenges

Regulatory and legal hurdles make rare disease research particularly daunting. Without an experienced CRO in your corner, navigating this red tape can be costly and time-consuming. 

CROs specializing in rare disease research are familiar with orphan drug designation processes and regulatory incentives. They have expertise in drafting and preparing rare disease regulatory submissions and strong relationships with agencies that can help expedite approvals. 

4. Innovative Trial Design and Execution

CROs are much nimbler than in-house drug development teams. Their small size and ability to pivot quickly boost efficiency but also allow them to take liberties with trial design and execution. For instance, since the COVID-19 pandemic, many CROs have embraced hybrid and virtual clinical trials. These trial designs use wearable devices, remote monitoring, and patient-reported outcomes to optimize data collection and participant comfort. These creative approaches also save time and money, making it significantly easier to bring new drugs and therapeutics to market. 

5. Cost-Effective Solutions

Speaking of cost, let’s explore how a CRO can help your organization stay on budget. 

First, CROs have all the necessary resources in place, from project management to clinical patient care. Therefore, you don’t have to spend money building a trial framework alone. Second, CROs regularly work with limited patient populations. Since rare diseases only affect a small subset of the population, these organizations can confidently maximize quality data from a limited patient pool. Third, CROs are fueled by efficiency. They provide resources on a case-by-case basis, so you can pick and choose your needs a la carte.

The Bottom Line

As you can see, CROs are essential for advancing rare disease research, offering the specialized expertise, global reach, regulatory acumen, and innovative trial design that modern biopharmaceutical companies require. 

With the demand for rare disease treatments continuing to grow, partnering with a CRO (like Harbor Clinical) can increase efficiency and help you stay ahead of the competition. If you’re interested in learning more about how we can help you, request a proposal or inquire about specific services today.