Unique challenges and complexities of rare and orphan disease clinical trials explored.
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
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.
Rare disease clinical trials have several challenges that make them more dif¿cult 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 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