Rare and orphan diseases
Unique challenges and complexities of rare and orphan disease clinical trials explored.
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.
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Rare disease patients and advocates drive change in public policy
Learn how patient advocacy organisations and rare disease communities are driving change in US public policy to make safe and effective treatment options available to all patients.
Four Challenges to Successful Rare Disease Drug Development
Overcoming these challenges can greatly improve chances for a successful rare or orphan drug trial.
The Rare Disease Revolution
Emerging research approaches and real-world evidence are fulfilling the needs of rare disease patients with increased speed.
The importance of the patient voice in the rare disease market
Orphan drugs, by nature, remain commercially underdeveloped, and as demand in the market increases, patients in clinical trials need to be considered.
Rare and orphan diseases media contributions
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Media article: The Pivotal Role of Patient Advocacy Organisations in Driving Rare Disease Therapeutic Development
ICON’s Dr Neena Nizar and Dr Jana Benesh discuss the ways effective engagement with PAOs is shaping rare disease research and contributing to better therapeutic outcomes. Read the article in IPI for insights.
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Media article: Therapeutic Focus - Pushing Boundaries in Idiopathic Pulmonary Fibrosis Clinical Research
With more than 170 early stage IPF clinical programs, IPF is one of the most dynamic rare diseases spaces for development.
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Media article: Engaging minority communities: collaborative practises and long-term success
Rare diseases can disproportionately affect minorities, yet these communities are often underrepresented in clinical studies. ICON’s experts suggest ways to improve engagement and diversity.
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Media article: External controls can augment low populations in rare disease trials
Advantages and disadvantages of using historical controls, and how data can be analysed using Bayesian statistics to develop a ‘predictive understanding’ of a drug.
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Media article: Accessibility in rare disease paediatric clinical trials
In this International Pharmaceutical Industry article, ICON experts Kirsten Sherman Cervati and William C. Maier discuss strategies to make clinical trials more accessible for Paediatric patients and their families.
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Media article: The human condition
In this article, Patricia Murphy discusses how the industry needs to make rare disease clinical trials more human.
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Media article: Making accessibility strategy part of your rare disease trial planning
In this article, Laura Iliescu, director of patient advocacy strategy at ICON Plc, discusses strategies to improve inclusion of people with disabilities in rare disease trials.
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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
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.