Rare Disease Week 2023: Exploring Accessibility in Rare Disease Clinical Research (4-Day Webinar Series)

Discussions around diversity, equity, and inclusion (DEI) in clinical trials have become increasingly central in drug development. Despite this evolution, there remain gaps in understanding about where accessibility fits within DEI strategies and initiatives.

These gaps present a significant challenge to research in general, but particularly in rare diseases where patient populations are much smaller and margins for error narrow. With these constraints in mind, inclusive trial design is essential, and accessibility can often become a critical success factor.

In this series, attendees will learn about:

• Myriad types of accessibility considerations for rare disease clinical research
• How accessibility considerations fit into DEI plans
• Best practices in accessible clinical trial design and implementation

Each panel, whether watched individually or in totality with the other panels, will provide insights into the value of accessibility in rare disease clinical research and how sponsors, patient advocates, researchers and regulators might move from discussion to action.

Monday, March 27, 2023

10am – 11am EDT: Is Disability Part of Your DEI Planning and Engagement in Rare Disease?

As trial sponsors lean into incorporating the US Food and Drug Administration (FDA)’s guidance on diversity, equity and inclusion (DEI) in their rare disease clinical research, disability should be part of those plans and initiatives. This panel will explore the intersectionality of people who live with a rare disease, disability and other important lived experiences. Discussions will also explore how patient advocates, CROs, pharma companies, researchers and other key stakeholders can better engage, include and incorporate insights from people who live within that intersectionality of experiences into improved clinical trial research and development.

11am – 12pm EDT: Unique Considerations Around Accessibility for Global Clinical Trials

Join patient advocacy, CRO and trial sponsor leadership as they explore key considerations for accessible clinical trials in different geographic regions. Perspectives from Europe, Mexico, India and the United States will be featured to demonstrate both the unique and universal needs rare disease patients may have when participating in global clinical trials.

Tuesday, March 28, 2023

10am – 11am EDT: Not All Measures Matter: How to Identify Outcomes Measures Appropriate for Your Rare Disease Patient Populations

Join leaders in the research, patient advocacy and clinical design fields as they discuss how sponsors can leverage or develop outcomes measures that demonstrate value in rare disease clinical research. The importance of clinical data, electronic medical records, registrational data and patient-reported outcomes will be explored, including the intersectionality between what matters to patients and to regulatory bodies.

Wednesday, March 29, 2023

10am – 11am EDT: Best Practices in Accessible Clinical Trial Design

Join advocate, research and industry leaders as they discuss lessons learned in rare disease clinical research design when examining the process through an accessibility lens. Best practices and real examples of successful accessible clinical research in rare disease will be shared.

11am – 12pm EDT: Accessibility in Rare Disease Paediatric Clinical Trials

This fireside chat between rare disease parents and paediatrician will delve into personal insights around accessibility for study participants who have a unique set of needs based on age and development. Speakers will explore nuanced issues, including what barriers they see in clinical trial accessibility for paediatric rare disease patients and how those barriers might be addressed, mitigated or solved.

Thursday, March 30, 2023

10am – 11am EDT: Where do Expanded Access Programs fit into Trial Accessibility?

In rare disease, compassionate use and Expanded Access Programs (EAPs) are becoming increasingly more common. As sponsors grapple with the unique needs of rare disease patients who may not have access to an approved therapy, impact of Expanded Access Programs and how or where they may fit within the larger context of accessible clinical trial landscape becomes more pressing. Experts within patient advocacy, CROs and industry will provide insights into important considerations for all these stakeholders as they develop and implement EAPs.

11am – 12pm EDT: Meeting Trial Participants Where They’re at: Where DCTs can help Achieve Greater Accessibility

Experts from CROs, patient advocacy and the pharmaceutical industry will share insights on the role of decentralised clinical trials (DCTs) as a potential option to make clinical research more accessible to participants who may not otherwise be able to participate. The panel will discuss important topics, like:

• Stakeholder engagement and resources needed to determine if a DCT is an optimal option to improve trial accessibility
• What capabilities are needed to successfully execute a DCT through the lens of accessibility

Accessibility Considerations

The following accessibility considerations have been provided.