Making accessibility strategy part of your rare disease trial planning

Over the past several years, diversity, equity, and inclusion (DEI) has become a central subject of conversation in clinical trials. In addition to the ethical obligation to engage participants who are representative of a patient population, legal imperatives are increasingly being introduced, such as the recent US Food and Drug Administration (FDA) legislation requiring patient diversity action plans.

One important element for increasing diversity in clinical trials is planning for the inclusion of people with disabilities. Disability spans age, gender, and race to impact people from every walk of life. In fact, a 2018 statistic from the Centers for Disease Control and Prevention states that one in four Americans has a disability that impacts major life activities.¹ With such a large number of people affected by disability, rare disease trials - which historically have a limited participant pool from which to draw - cannot afford to exclude individuals living with disabilities. 

To address the question of making DEI – and, in particular, disability – a part of rare disease trials, we held a conversation with healthcare and patient advocacy leaders to seek their perspective on strategies to better include people with disabilities in clinical trials. By incorporating these strategies, CROs, pharmaceutical companies, and researchers can better improve and optimise these trials.

Understand patient point of view

One of the first steps to including people with disabilities in rare disease trials is to understand their perspective. Though researchers may be very familiar with the concerns particular to their therapeutic area of study and are prepared to address them, disabilities come with their own barriers to participation. Working to recognise how elements of a clinical trial will impact persons with disabilities better enables the removal or mitigation of barriers.

Communication is one area in which this becomes very important. Traditional avenues of recruiting participants for a clinical trial will not necessarily be the best way to convey messages to people with disabilities. For example, in the case of someone with vision loss, print advertisements likely will not be an effective option. Further, emails and other communication will have to be constructed with accessibility in mind. 

Additionally, it will likely be important for prospective participants to know, upfront, whether a clinical trial will provide accommodations. Communicating this clearly from the start can provide  individuals with assurances that this is a trial in which they can potentially participate at a practical level, making engagement more likely.

The need for accommodations within the clinical trial also requires an understanding of patient needs. Everything from transportation and patient materials to the physical requirements of the trial may be impacted, necessitating consideration at every step of a patient’s journey through the trial. Resources, such as concierge services, which are designed to help connect with patients and increase patient engagement, can support patients and sponsors in navigating some of these challenges. In the case of patients for whom travel or mobility is difficult, in-home services that bring mobile healthcare professionals directly to the patient are another helpful option.

Make people with disabilities part of the conversation

Engaging people with disabilities behind the scenes is another worthwhile approach to incorporating DEI in rare disease clinical trials. Through hiring qualified individuals with disabilities to be part of an organisation’s staff - whether as consultants, an advisory board, or core personnel - they become part of the conversation in the creation of a trial. These employees can offer invaluable insights and unique perspectives with regard to accessibility.

Another important approach is “co-creation” alongside patients with disabilities. By asking for input on study protocol, endpoint strategy, conduct planning, and study materials, a sponsor is in a better position to meet their needs and is more likely to productively engage with them. Listening and incorporating feedback is key to this approach, and is most effective when sponsors approach the process with an open mind - the optimal solution may not be what the sponsor originally expected. 

Invest time and energy outside of clinical trials

Making an effort to learn about communities of people with disabilities outside the context of clinical trials can be invaluable to a company’s DEI initiatives. Internal education within a sponsor organisation has the potential to be a powerful resource to improve its understanding of, and connection with, different groups. Such education and enlightenment can come from guest speakers, employee resource groups, or specific employer initiatives. Regardless of the source, it is important that the culture of inclusion be encouraged throughout the organisation and endorsed by leadership.

Engaging with advocacy groups on a regular basis is also important. Supporting these groups through methods such as participation in conferences, volunteering, or regular calls with advocacy group leaders builds stronger relationships. In addition to increasing the likelihood of a particular advocacy group supporting future clinical trials, such efforts open the door to learning more about people with disabilities, and provides a better understanding of accessibility challenges and barriers that will only improve clinical trials.

Take action

Disability impacts a large percentage of people across the world and has no single appearance or set of needs. The process of improving DEI in rare disease clinical trials takes continual listening and learning across the industry, not only within the clinical trials themselves, but also within the organisations that lead them. Taking action to better understand the needs of people with disabilities - and working to meet those needs - is the only way forward in conducting truly inclusive, representative clinical trials.

About the author

Laura Iliescu, director of patient advocacy strategy at ICON Plc, has 20 years of experience in development and commercialisation of therapies, and patient-centred healthcare services. She has held strategic development roles in leading pharmaceutical, biotechnology, CRO, and clinical homecare organisations throughout every phase of the product lifecycle, from pre-clinical to beyond patent expiry. She has deep experience in rare metabolic, neuromuscular, and respiratory indications, among others. Within ICON’s Center for Rare Diseases, Iliescu advises clinical-stage rare disease focused companies on patient-centred clinical strategy to optimise both study efficiency and the experience of patients and caregivers. She holds a Master of Human Factors and Ergonomics from the University of Nottingham.