Accelerating Drug Development for Patients with Rare Diseases

There are over 7,000 rare diseases which affect a small number of patients worldwide, many of whom are physically and/or mentally disabled, and 50% of which are children.

7,000
Rare diseases
50%
Of patients are children

Given the geographically disparate nature of these patients as well as the family and patient burden of travel to specialist sites, finding, engaging and enrolling patients who qualify for a particular study can be very difficult.

To address this issue, CROs and Sponsors have begun to identify the feasibility of “place-shifting” rare disease trials from research trial sites to patients’ homes using device, sensors and home care teams. This has resulted in the development of a variety of wearables and sensors that can measure critical physiological indicators such as activity levels, sleep, blood pressure, heart rate, EEG and ECG, which can be combined and deployed in a non-clinical home setting, and tailored to specific therapeutic areas.

In addition to medical devices, there are a number of other options that should be considered to create more patient-centric clinical trials for rare disease patients. These include in-home visits by nurses or phlebotomists that can administer study medication or collect samples as well as utilise ePRO to collect patient-reported data. Also, where a patient does have to visit a specialist site for a physical exam by a physician, the use of a “travel concierge” service to arrange patient travel reduces both the site and patient burden and can remove barriers (including financial) that may otherwise hinder clinical trial enrolment or result in a drop-out altogether.

By taking a visit-by-visit approach to study design and using the maximum number of in-home or virtual visits as well as remote data collection, a truly patient-centric study can be delivered that orchestrates the patient experience for minimum disruption to life whilst providing maximum opportunity to partake in research.

CROs can help to create more patient-centric trials placing the patients’ needs and day to day life (including their disease) as the highest priority in protocol and operational design.

For further information on this topic, please contact us.

This blog is an edited version of "How to accelerate drug develoment for patients with rare diseases" which appeared in PharmaFile Autumn 2018.To view the full article, please visit PharmaFile.com (page 32).

Rare and orphan diseases insights

ICON's Rare and Orphan Diseases team provides analysis including whitepapers, blogs and contributions to media and industry conversations relating to all aspects of rare and orphan diseases in clinical trials.

Accelerating Drug Development for Patients with Rare Diseases

There are over 7,000 rare diseases which affect a small number of patients worldwide, many of whom are physically and/or mentally disabled, and 50% of which are children.

Rare and orphan diseases insights

In this section

Connect with us

Accelerating Drug Development for Patients with Rare Diseases

There are over 7,000 rare diseases which affect a small number of patients worldwide, many of whom are physically and/or mentally disabled, and 50% of which are children.

Rare and orphan diseases insights

In this section

Connect with us

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