Supporting Patients with Rare Diseases: Aixial’s Role in Advancing Patient Journeys

Aixial’s Role in Advancing Patient Journeys

As a Clinical Research Organization (CRO) who specialize in the rare disease space at Aixial Group we understand that the complexities of rare disease research requires a nuanced understanding and a tailored approach. Having supported over 220 studies across all phases of clinical development in 90 indications we understand the challenges are multifaceted, whether that be due to limited patient populations, diverse genetic profiles, or the intricate nature of rare disease mechanisms all play a factor in complicating clinical trial design and execution in this field. Often these hurdles are further exacerbated by the global dispersion of patients which may necessitate multinational trials that involve multiple sites and varied regulatory frameworks. These risks can be mitigated by working with the right CRO partner, at Aixial Group we have over 20 years of in-depth global experience in this space.

Understanding the Patient Journey

At the core of successful rare disease studies is an in-depth understanding of the patient journey. Many individuals with rare diseases can face significant diagnostic delays and a lack of awareness about their condition. This can mean their journey is marked by frustration and isolation with many never meeting a same person with the same diagnosis. At Aixial Group we understand this and we work with our Sponsor partners to design and develop studies to bridge this gap ensuring the studies are not only scientifically robust but also empathetic to the unique needs of these patients and caregivers.

One way we do this is by ensuring we design protocols to fit around the needs of our patients; incorporating flexibility in visit schedules and assessments where possible. We also look at patient comfort measures and have strong collaborations with patient concierge, home health and reimbursement providers. In a recent study for a rare metabolic disorder, we introduced visit windows to accommodate patients’ daily responsibilities, such as work and family commitments. This approach ensures that participation in the study is manageable and less burdensome for patients, ultimately improving recruitment and retention rates, the case study can be found here.

Collaborative Recruitment Strategies

Recruitment in rare disease studies presents its own set of challenges due to the small and geographically dispersed populations. At Aixial Group our strategy involves a multi-pronged approach that includes broadening inclusion criteria where feasible, engaging with patient advocacy groups though our established relationships, and leveraging digital tools for patient identification and recruitment.

In a primary biliary cholangitis study, we successfully increased patient enrolment by revising restrictive eligibility criteria and enhancing site engagement through regular motivational visits. These visits not only provided updates on the study and product status but also strengthened relationships with Investigators and site staff, fostering a sense of partnership and commitment to the study’s success.

Harnessing Technological Innovations

As a technology-led CRO we appreciate and advocate that the integration of advanced technologies is essential in managing the complexities of rare disease trials and we build technological driven solutions into our trial execution strategies. We understand visibility and reportable metrics are important for all Sponsors, for all studies but none more so than in the rare disease space. Our systems allow for real-time data collection and analysis, providing immediate metrics on emerging trends and facilitating timely decision-making. This capability is particularly critical in rare disease studies where each patient’s data can significantly influence the overall study outcomes.

Moreover, decentralized clinical trial models have become increasingly important, especially in a post-COVID-19 world. By enabling remote data monitoring and virtual site visits, we ensure that studies continue uninterrupted, providing reassurance to patients and sites alike.

Regulatory Expertise and Fast-Track Approvals

Navigating the regulatory landscape for rare disease therapies requires specialized knowledge and experience. Orphan drugs often qualify for expedited review processes and may be eligible for approval under exceptional circumstances. However, these pathways come with their own set of challenges, including potential Risk Evaluation and Mitigation Strategies (REMS) requirements for FDA applications and preparation of orphan drug designation application for review by Committee for Orphan Medicinal Products (COMP) through the European Commission.

Rare disease therapies are often based on gene therapies and involve complex trial designs, such as the use of radiopharmaceuticals. It is crucial to work with a development partner, like Aixial Group, that understand the implications of these trials as, otherwise, the extensive regulatory pathway may lead to unforeseen complications and unnecessary delays which cost time and money.

Our regulatory teams are experienced with such submissions and are often involved at the pre-scientific advice stages of development to ensure the right questions are asked to best position the application for success. Our experts work closely with health authorities across different regions to streamline approval processes and find the best path to approval. We find that with our early engagement approach and maintaining open transparent lines of communication, we facilitate faster study start-ups and reduce any potential delays.

Building a Community of Care

We believe all stakeholders involved in clinical research have a responsibility to contribute beyond the confines of clinical trials. At Aixial Group we are proactive about this, collaborating with patient advocacy groups and supporting rare disease awareness initiatives, with these efforts we help build a community that extends support and resources to patients and their families. This community-focused approach not only aids in recruitment but also fosters long-term engagement with all stakeholders.

Our commitment to rare disease research is reflected in our corporate social responsibility initiatives. Many of our team members have personal connections to rare diseases, which fuels our dedication to making a meaningful impact. Through fundraising activities and partnerships with rare disease organizations, we actively contribute to advancing research and improving patient outcomes.

The journey of advancing rare disease research is fraught with challenges, but it is also immensely rewarding. As a CRO, we are uniquely positioned to navigate these challenges through our expertise, innovative strategies, and unwavering commitment to patient-led care. By fostering collaboration, embracing technology, and maintaining a deep understanding of the patient journey, we continue to drive progress in the rare disease space, bringing hope and new possibilities to patients worldwide.