Clinical trials face many obstacles regardless of which disease they aim to investigate. However, rare disease trials face additional problems that don’t typically impact studies for more common conditions. Small patient populations, minimal funding, and low awareness are just a few of these issues. Because of this, researchers need to be innovative and intelligent when designing rare disease trials.

Outsourcing-Pharma interviewed two leaders from ICON in an effort to understand what we can do to improve rare disease clinical trials.

Interview with Scott Schliebner and Will Maier

Scott Schliebner and Will Maier are two leaders from ICON, a clinical research organization. They answered a few questions from Outsourcing-Pharma in an effort to explain how to design and run successful clinical trials.

Trends in Rare Disease Trials

The two leaders pointed out that rare disease clinical trials often operate under different restrictions due to a lack of available treatments or severe unmet medical needs. Some of these differences include smaller groups of participants, lower costs, and a higher chance of market authorization.

One trend that is becoming increasingly popular is grassroots advocacy and funding. Patient organizations have created natural history studies, patient registries, rare disease communities, and fundraisers. These efforts help to overcome some of the common obstacles that stand in the way of rare disease research.

Patient Voices in Rare Disease Trials

Patient voices must be listened to when it comes to rare disease research. Maier and Schliebner point out that patients are a trial’s “ultimate stakeholders,” and listening to them is absolutely essential for a successful study. Their voices should be sought out and taken into consideration when designing and running a trial.

A few benefits of truly listening to patients are:

• Faster clinical development
• Rapid enrollment
• Less screen fails
• Lowers the cost of drug development
• Lowers the number of patients who drop out mid-trial

ICON has actually developed a tool to facilitate and aid patient/researcher interactions. Additionally, it allows sponsors to really look into and understand the potential burden their trial could place on patients. In the end, it increases access and aids the clinical trial process. Titled the Patient-Centric Trial Development Toolkit, it is the first of its kind.

Incorporating Rare Disease Patients’ Voices

The two ICON leaders immediately point out the variability that exists within the rare disease sphere, whether that refers to the standard of care, disease course, or other factors. This variability places more obstacles in place of drug development and research.

They also pointed out other issues that exist when it comes to soliciting the opinions and voices of rare disease patients. Many rare diseases impact children, meaning that parents or other caregivers must be involved in the trial process. This already complicates things. Additionally, patients are often few in numbers and spread across the globe, which makes it hard to reach and gather them. Lastly, understandings of rare diseases are often lacking, making it difficult to design and evaluate trials.

How do Researchers Fall Short on Listening to Patients?

Time can be a major hurdle when it comes to soliciting and understanding patients’ voices. Researchers often fear that including these voices and opinions will slow the entire trial down, if they even have enough time to consider it. Another hurdle is minimal understanding of how to even approach and interact with patients. Lastly, Schliebner and Maier point out that it can be difficult to reach a consensus on trial protocols, just as it is difficult to include patients in a process that is otherwise completely medical and scientific.

Data Sources and Informatics to Improve Rare Disease Trials

There are a number of things that can be used to better comprehend the patient journey, such as pharmacy claims data, diagnostic claims data, and procedural claims data. They also named SES factors, demographics, healthcare utilization levels, site performance data, and historical enrollment rate data as helpful data that should be utilized when designing clinical trials.

Decentralized Rare Disease Clinical Trials

Decentralized clinical trials (DCTs) do not require the patients to travel to a trial site. Instead, they bring the trial to the patient. A few of the tools that help to decentralize a study are mobile health platforms, home healthcare nursing, telehealth direct-to-patient drug shipping, eSource direct EHM/EMH data collection, and Bluetooth-enabled devices to remotely capture data.

Weighing Treatment Impact and Disease Burden

Endpoints are extremely important in a trial, and they should work for both patients and regulators. Researchers should make sure to avoid selection bias, alongside considering patient motivation to participate and unmet medical needs within that community.

ICON’s Rare Disease Advisory Committee

ICON has an external Rare Disease Advisory Committee (RDAC) that is filled with patients, parents, caregivers, and advocates. These people come together to provide a holistic view of the rare disease landscape, guiding the study process so that it is compatible with patients.

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