Unique challenges and complexities of rare and orphan disease clinical trials explored.
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.Read the briefing
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.Read the whitepaper
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.Read the whitepaper
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.Read the whitepaper
ICON’s experts present best practices for sponsors and patient advocacy organisations interested in working together to design and implement patient registries.Read the whitepaper
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.Read the whitepaper
Rare disease drug development is challenging due to limited info on patient distribution, change in disease progression and relevant outcomes to define treatment benefits.Read the whitepaper
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.Read the whitepaper
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.Learn more
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.Read the whitepaper
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.Read the whitepaper
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.
This blog details initiatives that are being taken by the Government of Canada and various organizations like Canadian Organization for Rare Disorders to promote a health care system that works for those with rare disorders.
Rare disease registries: practical ways to build trust and collaboration with patient advocacy groups.
Navigating advanced therapy development for rare diseases is challenging from many perspectives and requires a customised development strategy.
Advanced therapies are a class of products based on genes, tissues or cells used to treat or deliver treatment to patients.
Learn how patients and advocates are key to driving legislative changes in rare disease research.
With 350 million people affected worldwide, rare diseases represent a major unmet medical need.
There are over 7,000 rare diseases which affect a small number of patients worldwide.
Orphan drugs, by nature, remain commercially underdeveloped, and as demand in the market increases, patients in clinical trials need to be considered.
Emerging research approaches and real-world evidence are fulfilling critical patient needs faster.
Developing drugs for rare diseases involves complexities beyond those typically seen in large trials for more common conditions.
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.
In this article, Patricia Murphy discusses how the industry needs to make rare disease clinical trials more human.
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.
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.
The following article will explore the advantages of using HCs, as well as several considerations for their effective use, including scientific methodology and regulatory guidance.
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 registries.
Devra Densmore discusses the importance of DEI in rare disease clinical trials and shares actionable points for a more inclusive approach.
Will Maier outlines the importance of utilising real world evidence from rare diseases in accelerating product approval and development.
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.
Will Maier, VP of Rare Diseases, shares how a more patient-focused approach to trials is leading to alternatives for randomisation.
Will Maier offers expert advice on planning and executing clinical studies with the unique needs of rare disease patients in mind.
William C Maier and Maggie Adamski consider how RWE can support those suffering with rare diseases around the world.
Benoit Arnould, Director of Patient Centred Outcomes, discusses how putting the patient first in clinical research is essential to discovering new therapies for orphan diseases.
Rare disease clinical trials have several challenges that make them more difficult to conduct than clinical trials for more common diseases. (PDF)
Examine the key clinical, regulatory, and commercial challenges associated with the development of therapies for the treatment of rare diseases. (PDF)
Rare diseases are usually genetic and therefore chronic, and the lack of natural history data poses several key challenges in the development of new therapies.
Orphan drugs, by nature, remain commercially underdeveloped, and as demand in the market increases, patients in clinical trials need to be considered. (PDF)
Project teams need to be challenged constantly to justify the study procedures and eligibility criteria they are proposing.
Real-world data studies play a key role in orphan and rare disease research. (PDF)
Precision medicine can have an immense impact for patient outcomes in rare diseases and other therapeutic areas. (PDF)
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.
Learn how the freely available Patient-Centric Trial Development Toolkit for rare diseases provides a framework for systematically and iteratively incorporating patient perspective from earliest stage of development and throughout the drug development lifecycle for any rare disease clinical program.
Receive guidance on the design and conduct of rare disease natural history studies including practical strategies for patient identification, recruitment and retention.
The challenges and opportunities for rare disease drug development.
Address the major challenges of patient engagement and retention during the study.
Real world evidence is expected to play a major role in the approval of future new medicines.
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