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Making rare disease clinical trials more human: patient-centric best practices for improved diversity

Improving patient population diversity in clinical trials has been an ongoing priority for the industry, but it is additionally important within Rare Disease where diverse patient populations are still underrepresented and clinical research faces compounding barriers to achieving diversity in trials. Creating more accessible, inclusive clinical trials will improve diversity and create an opportunity for more patients to access potentially meaningful therapies.

In recent discussions with sponsor, CRO, consultancy and patient advocacy organisation representatives in our Rare Disease Week webinar series, we explored the ways in which a patient-centric approach to clinical research can improve diversity. Heretofore, sponsors and CROs have shaped clinical trials – it is time we make them more human. When we consider accessibility for clinical trials, it is important to start from the patient’s perspective.

Below, we share best practices for a more patient-centric approach to trials as discussed in our webinar to improve accessibility, inclusion and diversity. 

Language services and accessible information

Language and translation services are key to accessible trial design. Patients must be able to access, understand and digest the information they need. Patients rely on sponsors and sites to communicate critical information to them regarding the investigational treatment being studied, the requirements of the clinical trial and how it will fit into their healthcare options. This information must be accessible in terms of:

  • Language and dialect – consider the vast differentiation between regional dialects and the way meaning is ascribed to certain words or phrases
  • Cultural translation – ensure conceptual equivalency that simple language-to-language translations don’t
  • Health literacy – the relative degree of complexity should accommodate a determined community baseline of health literacy
  • Mediums for transmission – the ways in which someone can receive this information and their ability to share it with their families and support systems
  • Different ability – for example, low-vision or hearing considerations

Providing patients with the information they need, including inclusion/exclusion criteria, enables their independent decision-making and affords a greater sense of agency in their journey. It also allows individuals to take information home to their families to discuss the nuances of participation and how they can factor the trial into their daily lives. This may be especially important for underserved and underrepresented communities with a historic distrust of the medical system that will want to consult with their loved ones.

The onus for accessible communication rests on sponsors and CRO partners to adapt to patient needs and go beyond the minimum requirements. CROs and sponsors can assist sites to overcome the barriers that would limit their ability to engage, inform and build trust in a clinical trial among patient populations.

Building communities

Making clinical research more human and patient-centric begins before a patient joins a trial; it begins with relationship-building. As clinical researchers, our patients provide invaluable data which leads to potential breakthroughs and new therapies. We can do more to support patients by intentionally developing stronger community-level relationships and then committing to more supportive patient-level relationships during trials.

In collaboration with patient advocacy organisations (PAOs) and CROs, sponsors can identify the appropriate communities to approach, then provide information about the clinical trial and the disease itself, by sponsoring local organisations and events, attending those events as public health communicators, thereby fostering relationships and giving as well as receiving information. This is important for maintaining relationships, encouraging trust and leaving communities better off after each trial by working with PAOs to continue support. It would be a mutually beneficial, constructive initiative to examine how to best communicate with and engage patients, via PAOs, during the data interpretation stage of clinical trials.

Humanising data

Traditionally, clinical trials have been restrictively homogeneous in the pursuit of good, clean data. This perspective on objective data discouraged inclusion of diverse populations and often excluded the sickest patients that need those therapies the most.

We are rewriting the definition of good data to be more diverse and include elements such as ethnicity, socioeconomic status, sexual orientation and gender identity and levels of ability that allow us to account for social determinants of health, comorbidities and concordance of disease.

More meaningful outcome measures

Traditional outcome measures supported by regulators are site-centric, which exacerbates geographical barriers to achieving diverse trial populations. As an industry, we rely on restrictive inclusion/exclusion criteria to compensate for measures that are not designed for sensitivity to the rare disease being studied.

However, developing bespoke outcome measures is possible with input from patient communities to help define the ways diseases manifest within specific communities. Novel endpoints require robust disease natural history data and entail significant investments and risks for sponsors who bring them to regulators for review and potential validation. Although there is risk in defining new outcomes and endpoints, there is also opportunity to develop measures that matter more to the patients and are more relevant to the trial.

Toward a more human clinical trial, together

Working together with stakeholders across the clinical research spectrum, we are shifting our perspectives and approach to bring meaningful therapies to the diverse communities affected by rare disease. We are engaging in these discussions across the industry and sharing tools like the RARE-X DE&I metrics toolkit for PAOs or the “What to ask when you’re interested in a clinical trial: A guide for rare disease patients and families” that we developed with our Rare Disease Advisory Council.

There is no one right way to achieve diversity in clinical trials, and best practices are still being pressure-tested. But it is imperative that we continue to innovate and improve our approaches from a patient-centric perspective to ensure the patients at the heart of our efforts benefit from accessible, inclusive trials.

Contact us to discover insights into the patient journey and build patient-centric rare disease clinical trials.

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