With 350 million people affected worldwide, rare diseases represent a major unmet medical need.
However, the timely recruitment of eligible participants is a challenge for any rare disease clinical trial. Although many patients with rare diseases are desperate for a treatment, strong engagement is critical, as patients and their families have been perpetually desperate and get burned out over time.
It is important to effectively recruit participants through a partnership with patient organisations, patient contact registries, and clinician education to increase disease recognition and decrease time to diagnosis.
ICON uses a variety of strategies to find patients such as partnering with rare disease patient groups and employing a digital engagement strategy, which allows recruitment for clinical trials at specific points in time when patients are searching for information online – such as at the point of diagnosis. Additionally, ICON works with ongoing registries to help identify patients for clinical trials and to locate historical control data.
Consistent trial participation is contingent upon the patient’s ability to get to and from site appointments. For success in a rare disease trial, the sponsor and other stakeholders must ensure there are no obstacles blocking patient compliance. It is imperative that contingencies to assist patients with transportation and accommodation costs be included in a company’s trial plan.
ICON provides trial participants with services to support every aspect of their lifestyle in relation to the trial including travel, lodging, and periodic home health nurse visits, and ensures that those arrangements are made so that patients don’t have to. It is important to give patients the resources they need to continue in the study.
ICON’s experienced team of clinical and therapeutic experts have conducted trials in over 60 disease indications across a range of therapeutic areas. To speak to one of our experts, please contact us.
This blog is an edited version of “An overview on rare disease research” which appeared in the Journal for Clinical Studies on 18th June 2020. To view the full article, please visit https://www.jforcs.com/.
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.
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Digital Disruption
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Clinical trial data anonymisation and data sharing
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Clinical Trial Tokenisation
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Closing the evidence gap: The value of digital health technologies in supporting drug reimbursement decisions
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Digital disruption in biopharma
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Disruptive Innovation
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Personalising Digital Health
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The triad of trust: Navigating real-world healthcare data integration
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Patient Centricity
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Agile Clinical Monitoring
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Capturing the voice of the patient in clinical trials
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Charting the Managed Access Program Landscape
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Developing Nurse-Centric Medical Communications
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Exploring the patient perspective from different angles
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Patient safety and pharmacovigilance
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A guide to safety data migrations
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Taking safety reporting to the next level with automation
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Outsourced Pharmacovigilance Affiliate Solution
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The evolution of the Pharmacovigilance System Master File: Benefits, challenges, and opportunities
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Sponsor and CRO pharmacovigilance and safety alliances
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Understanding the Periodic Benefit-Risk Evaluation Report
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A guide to safety data migrations
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Patient voice survey
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Patient Voice Survey - Decentralised and Hybrid Trials
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Reimagining Patient-Centricity with the Internet of Medical Things (IoMT)
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Using longitudinal qualitative research to capture the patient voice
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Agile Clinical Monitoring
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Regulatory Intelligence
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An innovative approach to rare disease clinical development
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Using innovative tools and lean writing processes to accelerate regulatory document writing
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Current overview of data sharing within clinical trial transparency
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Global Agency Meetings: A collaborative approach to drug development
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Keeping the end in mind: key considerations for creating plain language summaries
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Navigating orphan drug development from early phase to marketing authorisation
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Procedural and regulatory know-how for China biotechs in the EU
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RACE for Children Act
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Early engagement and regulatory considerations for biotech
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Regulatory Intelligence Newsletter
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Requirements & strategy considerations within clinical trial transparency
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Spotlight on regulatory reforms in China
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Transfer of marketing authorisation
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An innovative approach to rare disease clinical development
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Glycomics
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Rare and orphan diseases
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Advanced therapies for rare diseases
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Cross-border enrollment of rare disease patients
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Crossing the finish line: Why effective participation support strategy is critical to trial efficiency and success in rare diseases
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Diversity, equity and inclusion in rare disease clinical trials
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Identify and mitigate risks to rare disease clinical programmes
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Leveraging historical data for use in rare disease trials
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Natural history studies to improve drug development in rare diseases
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Patient Centricity in Orphan Drug Development
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The key to remarkable rare disease registries
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Advanced therapies for rare diseases
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Transforming Trials
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Ensuring the validity of clinical outcomes assessment (COA) data: The value of rater training
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Linguistic validation of Clinical Outcomes Assessments
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Decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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Decentralised and Hybrid clinical trials
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Practical considerations in transitioning to hybrid or decentralised clinical trials
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Navigating the regulatory labyrinth of technology in decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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eCOA implementation
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Implications of COVID-19 on statistical design and analyses of clinical studies
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Improving pharma R&D efficiency
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Increasing Complexity and Declining ROI in Drug Development
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Innovation in Clinical Trial Methodologies
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Risk Based Quality Management
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Transforming the R&D Model to Sustain Growth
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Accelerating biotech innovation from discovery to commercialisation
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Strategies for commercialising oncology treatments for young adults
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Accelerated early clinical manufacturing
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Cardiovascular Medical Devices
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COVID-19 navigating global market access
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Evidence Synthesis: A solution to sparse evidence, heterogeneous studies, and disconnected networks
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Global Outcomes Benchmarking
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ICER’s impact on payer decision making
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Medical communications in early phase product development
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Navigating the Challenges and Opportunities of Value Based Healthcare
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Payer Reliance on ICER and Perceptions on Value Based Pricing
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Payers Perspectives on Digital Therapeutics
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Precision Medicine
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RWE Generation Cross Sectional Studies and Medical Chart Review
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Survey results: How to engage healthcare decision-makers
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The affordability hurdle for gene therapies
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The Role of ICER as an HTA Organisation
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Strategies for commercialising oncology treatments for young adults
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