Studies and clinical trials in the rare disease space are complex and have novel designs or new endpoints. They are often unique in design, cost, and revenue structures. When you factor in how inherently different each patient’s disease state, age, and geographic location are, finding an experienced research site can be difficult. While these studies may present some research challenges, structuring them with the right technology can get clinical teams over the study design hurdles and help improve patient recruitment and retention by decreasing trial burden.
On Tuesday, May 8, our David Turner will be joining Scott Schliebner in a free webinar to discuss Easing the Rare Disease Patient's Burden in Clinical Trials with Innovative Technology. Here, David explains:
How much do you really know about technology in rare disease trials? Take the quiz and find out!
What does the future hold for research in the rare disease space?
Over the past few years there has been a shift in the way rare disease research is being conducted. The idea that the finish line was getting a drug approved is no longer the case. With more and more requirements, such as post-marketing commitments, patient required outcome reporting, and pediatric investigational plans, the finish line is moving. As the requirements for drug developers to keep gathering data well after the drug is approved increase, advancements in technology to help source the data is growing too. The future of clinical research in the rare disease space is exciting. From innovation in gene therapy to the potential of virtual trials, to the collaboration and data sharing will help to bring therapies to market faster.
How do you see the role of technology changing as it relates to clinical research?
The future of research in rare diseases will become much more virtual. Designing and executing clinical trials to further development of therapies. Rare disease research is different than research in other areas due to the low prevalence of the diseases, the geographic disbursement of patients, and research site locations. Leveraging innovative technology to better connect patients to clinical trials, and to make data collection throughout the study easier through virtual platforms is a win-win, benefiting both the patients, their families and clinical research. The ability to move to more virtual clinical trials – eliminating the need for travel to a clinical site – will make it easier for rare disease patients and their families to participate.
What do all these advancements mean for patients participating in clinical trials?
Due to the complex structure of rare disease studies and how inherently different each patient’s disease state, age and geographic location – patient recruitment and retention can be difficult. Thanks to technology we have seen new and innovative ways of gathering rare disease study data. Using mobile and connected devices to gather real-time patient data helps alleviate some of the patient’s burden. Using mobile and connected devices makes it easier for patients and their families by cutting down on clinic visits – but it also speeds up research through real-time, device-based data collection.
Imagine a Gene Therapy that requires a 20 year follow-up. Then imagine enrolling 50 patients over the course of 5 years wherever they show up in the world speaking whatever language they speak, all going to a single site to receive the treatment. How do you follow those patients throughout the first few decades of their lives?
To successfully complete the program, the Clinical6 solution had to ensure that the technology deployed would be multilingual, engage the patient’s parents/caregivers as well as local healthcare providers and be able to evolve over the 15-year span of the study without becoming irrelevant or burdensome for the patients and their parents/caregivers.
The study required a unified global mobile clinical platform for patient Enrollment, Engagement and Data Collection that cold deploy a BYOD (bring your own device) patient app within 90 days in a new language once a new patient entered the registry.
The patients enter the registry between 6 months to 2 years of age and will be followed throughout their life in pediatrics, adolescence and early adulthood, thus patients are expected to change healthcare providers a few times as they transition from grade school to University and into adulthood.
The solution was expected to allow patients/parents/caregivers to invite the new healthcare providers to participate and complete annual case report (CRFs), adverse events (AEs), admissions, etc. for the registry. To accommodate all of the needed solution functionality as well as the regulatory hurdles for such a global study, there was significant design, development and workflow challenges that had to be overcome by the Clinical6 team to successfully deliver a custom solution to support all of the Sponsor needs.
Language translations were handled via video consults between patient caregivers, primary care physicians, and the global PI. Elegant patient, caregiver, primary care physician, and clinical staff apps were deployed automatically setting the language settings to the language of the handset allowing the appropriate persons to easily digitally consent the patients, and securely capture information from the patients and primary care physicians over time. It was a geographically dispersed and completely connected multi-language rare disease configuration of Clinical6.
RARE DISEASES NEWSLETTER Volume 15, December 2019
RARE DISEASES NEWSLETTER Volume 5, January 2017
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.