Rare and orphan diseases
Unique challenges and complexities of rare and orphan disease clinical trials explored.
Future trends in Rare Disease drug development
Explore this on-demand webinar where industry experts share their thoughts on innovations, improving patient experiences, advancing therapies, the role of PAOs and what the future might hold.
No patient left behind
N-of-1 trial challenges and solutions in rare disease clinical development
In this on-demand webinar, we explore N-of-1 trial designs and their unique ability to provide valuable insights into the efficacy of interventions, leading to more personalised approaches to managing and treating rare diseases.
Therapeutic spotlight: Precision medicine considerations in rare diseases
In our latest therapeutic spotlight, ICON’s experts explore the evolving therapeutic landscape of precision medicine with a special focus on the unique application in ALS. We outline key considerations for developing patient-centric, streamlined precision medicine programs that promote patient engagement, improve protocol adherence and reduce dropout rates – ultimately accelerating the delivery of meaningful therapies to patients.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Rare and orphan diseases blogs
Pushing boundaries in idiopathic pulmonary fibrosis clinical research
The landscape of IPF research is evolving and early-stage biopharmaceutical teams need a new roadmap to success. We offer strategies to meet the challenges to develop new treatments.
Cross-border initiative to empower rare disease researchers
ICON joins new EU initiative to boost cross-border collaboration.
Rare disease trials require effective participation support strategies to succeed
Every day, thousands of colleagues at ICON work tirelessly to support the success of rare disease clinical research - because patients can't wait.
Strategies for de-risking rare disease programmes during research and development
ICON experts share how biotechs can maximise funding for rare disease therapeutic development in our blog.
Patient-centric best practices for improved diversity
In this blog, we share best practices for a more patient-centric approach to trials as discussed in our webinar to improve accessibility, inclusion and diversity.
Filling the gaps for rare and orphan diseases
Read the blog to learn more about the benefits and challenges of Rare Disease natural history studies.
Clinical development challenges faced by advanced therapies for rare disease
Advanced therapies are a class of products based on genes, tissues or cells used to treat or deliver treatment to patients.
Advanced therapies for rare disease - a regulatory roadmap
Navigating advanced therapy development for rare diseases is challenging from many perspectives and requires a customised development strategy.
Rare and orphan diseases media contributions
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Media article: Strategies for building greater DE&I in Rare Disease trials
This article describes some of the systemic factors that produce barriers at various stages of the rare disease drug development process, as well as identifies strategies
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Media article: Historical Controls in Rare Disease drug development: Using RWE to overcome key challenges
The following article will explore the advantages of using HCs, as well as several considerations for their effective use, including scientific methodology and regulatory
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Media article: The power of partnership: Collaborating with patient advocacy organisations for better clinical research
Laura Iliescu explores the powerful potential of collaboration with PAOs for clinical trials, particularly how PAOs can inform trial design and the creation of patient re
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Media article: Inclusive approaches to diversity and inclusion
Devra Densmore, Senior Lead of Patient Advocacy Strategy at the Centre for Rare Diseases at ICON, discusses the importance of DEI in rare disease clinical trials.
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Media article: Rare disease drug development
In this article, Will Maier outlines the importance of utilising real world evidence from rare diseases in accelerating product approval and development.
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Media article: Using RWE in rare disease drug development: effective innovations with historical controls
With a growing number of therapies under development for rare diseases, William Maier discusses how real-world evidence can be effectively used as a historical control, overcoming challenges presented in clinical development.
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Media article: How real world evidence is impacting rare disease drug development
Will Maier, VP of Rare Diseases, shares how a more patient-focused approach to trials is leading to alternatives for randomisation.
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Media article: Real world evidence and the patient experience: Involving rare disease patients for successful trials
ICON's William C Maier and Maggie Adamski discuss case studies from their line of work, and how real world evidence can support those suffering with rare diseases around the world.
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Rare and orphan diseases on-demand webinars
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.
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.