Creating a DCT Roadmap for Clinical Research in Rare Diseases
Researchers estimate that the effects of rare diseases impact over a billion patients and caregivers worldwide. However, less than 10% of rare diseases have an effective treatment. Decentralized clinical trial (DCT) tools can make rare disease research feasible for patients and trial teams, helping increase available therapies. This article covers:??
Differences between rare disease and traditional clinical trials
Recruiting enough patients to generate significant results
Rare diseases often affect small populations spread over wide geographic areas. For example, some patients diagnosed with a rare genetic disorder may live in the western US, whereas others live in southern India. The widely dispersed populations complicate finding central sites for rare disease trials.??
Patient demographics?
Many rare diseases occur in pediatric populations, so researchers must plan for the complexities of including legally authorized representatives, such as parents, in the trial workflow. Where many traditional clinical trials recruit adult patients to simplify elements like informed consent, rare disease trials must adapt the consent process for pediatric populations.??
Choosing suitable clinical endpoints
Traditional trials typically have established clinical endpoints to choose from. For example, the Kansas City Cardiomyopathy Questionnaire is a commonly used tool for measuring outcomes in quality of life for patients experiencing cardiac conditions. These types of widely accepted tools are not necessarily available for rare diseases. Because rare diseases often lack existing literature on disease progression or previous studies, researchers must put extra effort into finding reliable measurements of treatment efficacy.??
Extended trial timelines
Recruitment for rare disease clinical trials can take longer than recruitment for traditional trials because of small patient populations. Researchers may need to reach out to patients individually or by partnering with advocacy groups instead of relying on broad efforts like billboards or advertisements.
Also, study teams may want to lengthen the time they spend collecting data to learn as much as possible about the rare disease. Both of these factors can extend trial timelines for rare disease research.?
How DCT technology meets rare disease research challenges
Decentralized clinical trial tools like electronic consent (eConsent), electronic patient-reported outcomes (ePRO), electronic clinician-reported outcome assessments (eCOA), and integrations like real-world data (RWD) can help meet the challenges rare disease trials pose for patients, their caregivers, and researchers.
Let's illustrate how technology could improve trial workflow and patient experience using a hypothetical trial of a treatment for Schnitzler's syndrome, a rare autoinflammatory condition.?
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Simplifying recruitment, enrollment, and follow-up with eConsent?
Fewer than 1,000 people in the US are diagnosed with Schnitzler's syndrome. Researchers can use simple and engaging technology to meet their enrollment goals despite the small pool of potential participants. Researchers can use the eConsent to:???
Like many rare diseases, much about Schnitzler's syndrome is unknown. Researchers may need to adjust the protocol mid-study to accommodate new developments. If changes require reconsent, researchers can use the eConsent tool to automatically send patients the most recent version of the informed consent form (ICF) to sign.
Researchers would likely want to follow patients after trial completion to collect more data and improve the collective knowledge of the disease. Reconsenting for follow-up through registries or new studies with the same eConsent tool used for enrollment simplifies the patient experience.
Improving the participation experience with ePRO and EDC
Patients with Schnitzler's syndrome often experience debilitating symptoms, such as joint and bone pain, hives, and fatigue. Traveling to a research site is a huge burden for patients struggling with symptoms, especially those living far from sites. Researchers can limit travel using remote data collection tools like ePRO and wearables to track vitals. Data from wearables flow directly into the trial's electronic data capture (EDC) system via an application programming interface (API).??????
Researchers can use the data stored securely in the EDC system to make real-time decisions to adjust the trial design. This flexibility is especially significant for rare disease clinical trials, where the lack of knowledge about the natural history of the illness can limit initial designs.
Here's a summary of where to incorporate decentralized clinical trial tools in your rare disease research.
Overcoming challenges in adopting DCT tools
Understanding where to use DCT technology in your rare disease research is a critical first step to adoption. Many rare disease studies benefit from a hybrid approach—using a mix of traditional and remote tools. Including digital tools in the initial study design improves adoption, although many decentralized research tools can scale as the study progresses.
Involving sites, regulatory agencies, sponsors, patient representatives, and caregiver input in the planning process can also increase the adoption of DCT tools. Site and patient feedback help ensure the tools make workflow simpler, not more burdensome. Representatives from ethics committees can also help streamline the approval process for eConsent and other digital tools.?
Wondering how to empower your rare disease research with DCT? Contact us to learn how Castor’s modular solutions can support the specific needs of your trial.???