The low prevalence of rare diseases makes it difficult to recruit and retain a sufficient number of participants for robust clinical studies. This can lead to inadequate sample sizes, reducing the statistical power of the research. Through early consultation, a biometrics CRO can advise on study designs that may reduce the number of subjects needed, reducing the challenges of recruitment and retention, and ensure an overall more efficient and optimal design of a study.

Many rare diseases lack comprehensive natural history studies, which provide crucial baseline data about the progression of a disease without treatment. This absence can complicate the design and interpretation of clinical trials. Biometric CROs can assist in gathering and analyzing existing data from various sources to construct a pseudo-natural history of the disease, providing a valuable context for new clinical trials.

Patients with rare diseases often experience significant delays in diagnosis or are misdiagnosed. This issue can impact the identification of eligible study participants and influence the study outcomes due to varying stages of disease progression. Leveraging advanced diagnostic tools and partnerships with diagnostic labs, a biometric CRO can facilitate more accurate and timely diagnosis, ensuring appropriate patient enrolment.

For many rare diseases, the underlying pathophysiology is not well understood. This lack of understanding can hinder the development of effective treatments and appropriate study endpoints. Biometric CROs often have expertise in advanced biostatistical modelling and can collaborate with academic researchers to deepen the understanding of disease mechanisms, influencing trial design and endpoint selection.

Regulatory pathways for rare disease research can be complex and varied, depending on the region. Navigating these pathways requires specialized knowledge and can pose significant challenges. With their experience in navigating regulatory landscapes, biometric CROs can guide sponsors through complex approval processes, ensuring compliance with varying regional regulations.

Rare diseases often attract less funding compared to more common conditions, which can limit the scope and scale of research studies. Biometric CROs can provide swift reporting of results and well-designed analyses to help in making research proposals more attractive to potential funders.

Ethical concerns, such as ensuring informed consent and balancing risk-benefit ratios, can be more pronounced in rare disease research due to the severity and unmet medical need of many rare conditions. These organizations have expertise in ethical guidelines and can ensure that studies are designed and conducted in compliance with ethical standards, prioritizing patient safety and informed consent.

The absence of established and standardised outcome measures for many rare diseases can complicate the assessment of treatment efficacy and safety. Biometric CROs can aid in the development or selection of appropriate, validated outcome measures and can help standardize these across studies for better comparability.

Rare diseases often exhibit a wide range of symptoms and disease severities, even among patients with the same diagnosis. This variability can complicate clinical trial design and data interpretation. Utilizing sophisticated statistical methods and adaptive trial designs, biometric CROs can account for patient variability, ensuring that study designs are robust and capable of detecting treatment effects across diverse patient populations.

Conducting research on rare diseases often requires international collaboration and data sharing to gather sufficient data. However, differences in regulatory standards, data privacy laws, and logistical issues can impede these collaborations. With their global networks and experience in multinational studies, biometric CROs can facilitate international collaboration, manage logistical challenges, and navigate data sharing agreements in compliance with local laws and regulations.