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In the News
ICON experts frequently author or contribute to industry trade press.
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No patient left behind: The promise and challenges of N-of-1 studies for rare disease clinical development
Rare disease N of 1 studies offer patients the hope of finding a personalised, effective treatment. ICON experts discuss the challenges, solutions and promise of these unique trials in this PharmaTimes article.
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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 into patient engagement in preclinical, clinical and post-approval phases of development.
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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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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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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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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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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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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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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 to improve diversity in these trials
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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
