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Clinical Development

Patient-Focused Clinical Trial Design for Rare Diseases – Part 1

  • June 13, 2018
Rare Disease Concept

A few months ago, Medpace and the Cincinnati Children’s Hospital Medical Center presented a webinar on the importance of patient-centric clinical research. The webinar, which can be viewed in its entirety here, was presented on the nationally recognized Rare Disease Day and specifically focused on how patient-focused clinical trial design and collaborative approaches to site enrollment can accelerate clinical research.

Over the next several weeks, we will build off each topic that was addressed during the presentation. In this post, we take a look at what patient-centric clinical trial design looks like for these studies.

Patient-Centric Trial Design

Input from key stakeholders on trial design is a must for making rare disease studies more patient-centric. Endpoints should be chosen not only on the basis of which ones will best assess the efficacy of a therapy, but also on what would be most meaningful to the patients in terms of improving quality of life. The presenters suggested engaging a number of key groups to help inform trial design decisions that will ultimately affect patients participating in the study. This emphasizes the importance of patient advocacy groups and attendance at both local and international patient meetings for the rare disease.

But it’s not enough to engage with patients only when making study design and protocol decisions. It’s imperative that participants – and the rare disease patient community as a whole – be regularly updated on study findings and other important information by way of emails, press releases and webinars. Providing a layperson’s summary of results can be helpful in making sure everyone feels included.

Once enrolled, retaining patients can be challenging in many rare disease trials. Participant burden can be reduced in a number of ways, including having flexible visit windows to allow patients who work during the day to complete their study visit on nights and weekends, and making use of home healthcare services which could eliminate some of these visits altogether. The family and caregivers of the participant should also be taken into account when establishing support systems, particularly in pediatric trials where parents may have other small children to care for. Alternative trial designs can also reduce patient burden and improve retention without compromising the quality of clinical data collected during the course of a study. For example, applying a crossover trial design can give all patients access to the investigational therapy, while providing the sponsor with the placebo/standard of care data necessary to accurately compare treatments.

With rare disease there are often complex endpoints which also reduce the number of centers that are able to participate in the clinical trial. This means further travel and increased burden on the patients and caregivers. To address this, the presenters suggest sites consider a “hub and spoke” trial design which would still require patients to travel to the main study site for major procedures but allow them to visit local sites for minor procedures. Alternatively, if a study protocol requires collection of patient data at frequent time points, participants could be alternately assigned to only a few of those time points to reduce sample collection burden.

In Part 2 of this series, we’ll look at rare disease trials from the patient and caregiver perspective.