May 17th, 2017

Roundtable On Rare Disease Clinical Trials Highlights Industry’s Unique Challenges


If rare disease clinical trials want to recruit patients more effectively, they need to embrace innovative digital technologies.

PharmaVoice recently published a great article on patient recruitment for rare disease clinical trials. In the piece, contributor Kim Ribbink sourced a wide variety of opinions and suggestions from thought leaders throughout the clinical research industry. Here are a few of the key takeaways, along with some potential solutions to the issues they raise.

Reaching a Narrow Audience

Right now, efforts are being made to bring effective treatments to market for the 7,000+ rare disease that currently afflict more than 350 million people across the globe. However, in order to secure approval for new drugs and therapies, sponsors must secure a large enough patient population (relative to the prevalence of the condition) to convince regulators that the treatment is, in fact, safe and effective.

Of course, this can be a difficult thing to do, especially when it comes to rare diseases. As Juliet Moritz, Executive Director for Rare Disease and General Medicine at Premier Research, points out, “Recruitment for rare disease trials requires a significant amount of commitment to the patient population – understanding the disease and what it means to the patient and to the family to live with that disease.

“The small size of rare disease populations makes ineffective some of the tactics used for recruitment in more common conditions.”

When it comes to rare disease recruitment, a thorough understanding of the patient population is key – that means a solid grasp on the disease’s epidemiology, including how many patients there are, their location, and how many trials for that particular condition are active at any given moment.

Once you’ve identified your target audience, you can leverage cutting-edge advertising platforms like Facebook and Google to reach prospective participants with the information they need to enroll. Demographic and location targeting tools make it possible to serve highly personalized ads to rare disease patients based on key qualifying information, such as age, zip code, gender, ethnic affinity, and interests.

Reducing the Burden for Participants

Patient retention can be a serious issue as well – dropouts cause costly delays, require more investment into recruitment, and have a negative impact on the quality of your trial data, according to Scott Gray, CEO of Clinicierge.

“Patient drop-outs also have a negative effect on trial cost and timelines, as they can lead to the need for trial extensions, bringing on new sites, and ultimately, delayed submissions and time to market.”

Patients often drop out of rare disease trials due to financial, logistical, or emotional reasons. As such, it’s crucial that sponsors and CROs adopt a patient-centric approach, providing the necessary resources to minimize the amount of burden involved with sticking with a study to its completion.

Some trials are taking proactive measures to address some of these issues, such as partnering with ridesharing services to make traveling to on-site appointments less of an issue, or leveraging mHealth and remote patient monitoring technologies to reduce the amount of travel overall. The net result is an expedited timeline and higher-quality data at the trial’s conclusion.

If sponsors and CROs want to see a real improvement in rare disease study outcomes, they need to start embracing the new tech-forward solutions that will ultimately make it possible. At the end of the day, outcomes for patients and sponsors are intrinsically linked – a faster time to approval means significant cost savings for pharmaceutical companies, which in turn means faster access to more affordable treatments for the people who need them most.