How Can We Have More Successful Rare Disease Trials?

Rare Disease Day ribbon

By: Reverba Clinical Partnerships Team

More than 300 million people around the world live with a rare disease—that is, a condition that affects fewer than 1 in 2,000. Although each of these estimated 5,000 to 7,000 or more rare diseases affects a relatively small number of individuals, the impact on those patients’ lives is significant.

The vast majority of rare diseases—95 percent—have no approved treatments.

As we mark #RareDiseaseDay on February 28, 2023, we recognize the challenges that people who live with rare disease face, like misdiagnoses, isolation and treatment inequality. We also acknowledge the important contributions these individuals can make to rare disease clinical programs.

Clinical trials in rare diseases face unique challenges.

Randomized, double-blind, controlled clinical trials are the gold standard—but they are more difficult in the rare disease space. For a given condition, the potential number of trial participants is small—and often made smaller by strict enrollment criteria. What’s more, that small number of eligible patients is often spread out over wide geographic areas. Aspects of the condition may make it difficult to travel to trial sites. That makes both design and conduct of trials more difficult. Heterogeneity is another complicating factor. Many of rare diseases are inherited. As we dive into the genetics, we are starting to see that sometimes what we have viewed as a single condition is actually many different diseases with similar symptoms. How do we pick endpoints that matter if we don’t understand the disease—or what matters to the patient? Further, when patients perceive an investigative therapy as their only hope, many are reluctant to risk being assigned to the placebo arm. And in small communities that share their experiences, unintentional unblinding is a possibility.

What impacts rare disease trial success?

GlobalData looked at 736 rare disease trials between 2016 and 2020 and found that 26 percent ended early due to low accrual, making it the most common reason for termination. Lack of efficacy was cited in 12 percent of trial terminations. A successful trial needs to enroll sufficient patients and minimize dropout. A successful therapy must make a difference in patients’ lives.

The problem often lies in trials that don’t reflect the perspectives and realities of those living with a rare disease. Sometimes enrollment criteria—like age or disease stage—exclude too many potential participants. Often the logistics—too many visits at a distant trial site—make participation impossible. Sometimes individuals feel that the risks of trial participation, or invasive procedures that might be involved outweigh the potential benefits.

How are sponsors to know? In an article based on interviews with members of Europe’s ASTERIX Project Patient Think Tank, the authors noted, “No one can assess the clinical relevance of an outcome measure better than those who live with the consequences of a disease on a day to day basis.”

In research funded by the US Food and Drug Administration and the Clinical Trials Transformation Initiative, the authors found that “A combination of empirical data and subjective parameter estimates shows that engagement activities with the potential to avoid protocol amendments and/or improve enrollment, adherence, and retention may add considerable financial value.”

How can an organization engage and recruit rare disease patients?

So many patients, so many rare diseases… so few therapies. Clearly, there is need—and getting patients involved early in study design and expected outcomes can help overcome some of the barriers to trial participation. So how to go about it? How can an organization increase patient engagement in rare disease programs?

At Reverba, we bring the patient’s journey, disease specifics and the patient’s voice to the forefront of the strategy. We quickly align stakeholders, reducing time to launch and improving quality. We’ve been serving the biopharmaceutical industry since 2002, so we’re not just experts in patient engagement – we helped define it.

Based on our experience with more than 50 rare disease programs, we understand what organizers must do to show these unique patient populations that they value them as true partners—from conveying empathy to offering payment to sharing trial results. We put that knowledge to work to make our clients’ programs successful.

Reverba’s patient engagement solutions improve trial design by collecting valuable feedback directly from patients before, during, and after trial participation. We offer agile research design, rapid deployment, in-depth analysis, and actionable reporting. We also help to boost recruitment by reviewing your trial through a strategic lens, selectively opening recruitment channels based on the unique population and circumstances of your rare disease program. Reverba’s platform tracks progress towards agreed-upon goals, altering or iterating tactics with a nimble, growth-oriented mindset. We work collaboratively with our sponsors, CROs, and other key stakeholders to enable a model designed to drive trial success.

If you work in the rare disease space, increasing patient engagement can benefit both patients and your program. Reverba has the relationships, processes and proprietary platform to help you connect compliantly, so you can identify endpoints that matter, proactively address barriers to enrollment and—most important—make a positive impact on the lives of those experiencing rare diseases. We welcome an opportunity to tell you more.

Where to go from here.

Understanding how people think about clinical trials is the first step towards improving the experience you offer participants. Help achieve your 2023 goals by integrating the patient’s voice in your clinical strategy. Download our Clinical Trial Engagement Guide for more insights on how you can make more people aware of your trial, establish trust faster, and boost recruitment.

Contact us to schedule a personalized consultation focused on addressing your clinical challenges and how to optimize your trial to reach goals faster.