Patient engagement and patient centricity in clinical trials face numerous challenges that impact the research process and hinder the ability to have representative populations.
Misdiagnosis and delayed diagnosis
Some rare diseases lack clear biomarkers, while some have rare biomarkers. Both scenarios make accurate diagnosis challenging. With most rare diseases requiring genomic testing for assessing biomarkers, the inaccessibility of testing and regional variations lead to misdiagnosis and delayed diagnosis. Non-standard diagnostic tests and limited data on genetic mutations further complicate patient screening and recruitment.
Lack of awareness among HCPs and patients
There is limited understanding and awareness about the clinical trial opportunities for most rare diseases. HCPs who are unaware of available clinical trials for rare diseases may not refer eligible patients to these trials. They may not have the necessary knowledge or resources to identify appropriate trials or may simply not consider clinical trials as a treatment option. This can result in a smaller pool of potential participants for these trials.
Without information about ongoing trials, patients may not actively seek out these opportunities or understand the potential benefits they could receive from participating. Lack of patient awareness can therefore result in missed opportunities for patient advocacy groups or organizations to promote clinical trials to their communities.
Lack of trust in the medical system
Given the novelty of gene therapies, there is some apprehension in patient communities around their use. Patients have concerns and misconceptions about the safety, efficacy, and ethical implications of gene therapies. Patient acceptance and understanding of gene therapy mechanisms, as well as benefits versus risks, is an important milestone to ensuring successful engagement. There is also lack of knowledge and understanding among healthcare professionals (HCPs), contributing to a lack of trust between rare disease patients and HCPs. Sponsors typically face challenges in building collaboration and trust with patient communities, making it difficult to identify potential participants and address barriers to enrollment. Tailored communication approaches are therefore required to engage and involve patient communities effectively.
Patient Identification and recruitment
Small and hard-to-find patient populations create challenges in identifying and recruiting suitable participants. For some rare diseases, patients are required to have specific gene mutations or antigens for inclusion, resulting in limited sample sizes. Conducting statistically significant trials and drawing robust conclusions becomes challenging. Screening, testing, and diagnosis complexities also cause delays in patient recruitment.
Many of the rare diseases are under-diagnosed, and their true prevalence is not well understood. The resultant lack of reliable data hampers patient identification and recruitment for clinical trials.
Underserved communities
Patients in under-served communities face additional barriers to healthcare access and clinical trial participation. It is therefore important for you to understand the demographic disparities for your proposed indication and to account for these disparities in your trials. Addressing these disparities is crucial for promoting patient engagement and inclusivity in clinical trials.
Overcoming these challenges in patient engagement and patient centricity is crucial to advancing clinical research and improving healthcare outcomes. By raising disease awareness, enhancing trust and education, fostering collaboration with patient communities, and addressing barriers to access, we can strive towards a more all-encompassing and patient- centric approach in clinical trials. In the next section, we will dive into potential strategies to address this.