Events 2021 Rare Disease Day PRAH Scom Header v1r1 02

Key Highlights

PRA Health Sciences will host its annual Rare Disease Day celebration on February 26, 2021. This year’s event, held virtually, will focus on decentralized strategies and equitable access for rare disease research and in rare disease clinical studies.

Juliane K. Mills
Juliane K. Mills
Director Therapeutic Expertise, Center for Rare Diseases

Technology like wearable devices and health-monitoring apps are more mainstream and pervasive in our daily lives and comprise an important source of patient-generated health data. Examples of patient-generated health data (PGHD) include tracking daily weight or food consumption in a weight-loss app, wearing a heart-rate monitor during exercise, or tracking sleep or menstrual cycles. PGHD allows access to data that have historically been reported subjectively or not captured by healthcare-provided data. These data, when shared with a health care clinician, offer an objective insight into a patient’s health history that provides perspective and context for symptoms or results observed during an office visit. PGHD also allows for continuous data collection in a real-world setting, as opposed to discrete periodic assessments in a clinic at prescribed intervals. During the COVID-19 pandemic, there’s been an accelerated adoption by the healthcare system of PGHD and the related technologies to collect these data in lieu of actual face-to-face office visits and hands-on assessments.

Read more about the use of patient generated health data into pharmacoepidemiological research

PRA is committed to building upon the momentum of innovations stemming from the COVID-19 pandemic. We want to make these modern approaches part of a new paradigm in clinical research. These innovations include home health visits, training caregivers on patient assessments, as well as applying technologies such as virtual/telehealth visits, wearables, and electronic diaries.

PRA’s Center for Rare Diseases is excited to further these innovations by applying them to novel situations and, where possible, validating them for at-home use. As an example, the six-minute walk test is the gold standard for many clinical studies looking to assess change in cardiac, respiratory, or neuromuscular health over time. In this test, patients walk up and down a measured distance for a period of six consecutive minutes. To be used as an endpoint in a clinical trial, this test must be done in a standard way: the same hallway must be used by all patients, the same tester must perform each assessment. This standardization is very difficult to replicate in the at-home environment, especially on a global clinical trial in patients with a rare diseases. Instead, it may be possible to validate the use a wearable device in combination with video and other motion-capture technologies to assess changes in gait and balance for patients with neuromuscular disorders.

It is imperative to take a thoughtful approach to the application of technology in a context-appropriate setting. Patient age, demographics, socioeconomic status, culture, geography, education, and physical and cognitive ability are all important parameters that should be considered when evaluating the use of technology. Feedback from patients participating in the validation of technology is critical to understanding usability and behavior modification. While it may be possible to incorporate technology such as a wearables or an app into a clinical study, its function must be practical and as least disruptive as possible to how a patient manages their daily life and existing condition. Exit surveys and/or discussion groups with study volunteers are an effective means to solicit feedback on technology used in a clinical study, and do not add significant cost or time relative to the insights that can be generated.

PRA will help support your patient engagement strategy, whether for technology assessment or protocol design.

At PRA Health Sciences' Center for Rare Diseases, we are guided by patients who understand better than anyone else that rare diseases alter entire lives. We are transforming clinical research into meaningful healthcare options through patient partnerships, data analytics, and technology solutions. We want you to join in on the discussion!

Click here to sign-up for PRA’s annual Rare Disease Day event.

See what PRA’s Center for Rare Diseases is doing to fight rare disease

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