Rare and orphan diseases

Unique challenges and complexities of rare and orphan disease clinical trials explored.

ICON supports World Rare Disease Day 2024

ICON is joining other healthcare & patient organisations to raise awareness of rare diseases on World Rare Disease Day.

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Pushing boundaries in idiopathic pulmonary fibrosis clinical research

Explore the evolving idiopathic pulmonary fibrosis (IPf) treatment landscape and how early-stage biopharmaceutical teams can meet emerging development challenges and chart a new roadmap to success in our whitepaper.

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Using data from externally controlled trials in rare disease clinical development

External controls are most frequently leveraged in situations where conducting prospective randomized, controlled studies is not feasible; examples include products approved for rare, life-threatening, or severely debilitating conditions, in some cases slowly progressing, with no/inadequate available therapy. This briefing document describes external control arms and provides concise information on the FDA’s regulatory precedent regarding their acceptable use.

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Beyond buzzwords: Diversity, equity and inclusion in rare disease clinical trials

The rarity of each individual rare disease introduces many unique challenges and complexity in clinical research including barriers to achieving representative trial populations and equitable access to trials.

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Navigating orphan drug development from early phase to marketing authorisation

In this white paper, ICON’s experts discuss the intricacies of orphan drug development and the challenges that both the US and EU experience, including the EU concept of significant benefit criterion. 

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Crossing the finish line: Why effective participation support strategy is critical to trial efficiency and success in rare diseases

Rare disease clinical trials face unique challenges, such as the low prevalence of disease and few qualified investigators, resulting in higher risks to clinical development programmes for sponsors.

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The key to remarkable rare disease registries

ICON’s experts present best practices for sponsors and patient advocacy organisations interested in working together to design and implement patient registries.

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Cross-border enrollment of rare disease patients

The patient pool for rare disease clinical trials is often small and widely dispersed. Likewise, clinical sites with specialised experience are rare. These issues can be mitigated by enrolling patients who are not residents of the country where the trial is being conducted.

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Natural history studies to improve drug development in rare diseases

Rare disease drug development is challenging due to limited info on patient distribution, change in disease progression and relevant outcomes to define treatment benefits.

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Unlocking the potential of advanced therapies developed for rare diseases

Development of advanced therapies for rare diseases requires a customised regulatory strategy. Discover an approach and interaction plan that sponsors can follow across all phases of development to accelerate time to market.

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Leveraging historical data for use in rare disease trials

Rare disease clinical trials have several challenges that make them more difficult to conduct than clinical trials for more common diseases. Small patient populations, pediatric populations and the desire by patients and their caregivers to receive active therapy while on a clinical trial make it much more likely that these trials will need to incorporate historical data into the design.

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Patient centricity in orphan drug development

ICON analysed data on the use of patient-reported outcomes measures (PROMs) in orphan drug labeling claims and performed an extensive literature review on the use of PROMs in all phases of clinical research, observational/registry studies, and instrument development and validation. The findings and recommendations can be found in this whitepaper.

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Participant-focused tools designed to improve study success in rare disease clinical trials

There is a recognised a gap in the availability of structured tools to help sponsors operationalise patient-centricity and identify and mitigate risks in rare disease clinical development.

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Rare & Orphan Diseases media contributions

Receive more insights on rare and orphan diseases from ICON

Please visit ICON's Preference Centre and select 'rare diseases' under 'Therapeutic Areas of Interest' to receive new insights on rare and orphans diseases.

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Rare and orphan diseases videos

Rare and orphan diseases services

ICON is an experienced partner who can assist sponsors in navigating the many challenges of orphan drug development. ICON can help sponsors optimise the whole continuum of the clinical trial lifecycle from patient enrolment to payer reimbursement with its powerful solutions and proven strategies. ICON’s highly experienced clinical and therapeutic teams have conducted numerous rare disease trials across a wide range of therapeutic areas.

Read more about ICON's services in rare diseases