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Three Ways Patients and Advocates Can Inform Rare Disease Trials

A survey led by PPD, Thermo Fisher Scientific’s clinical research business, revealed that rare diseases are a top therapeutic area in drug developers’ pipelines. Learn how relationships can be built and leveraged to combat the unique challenges rare disease trials face, including a limited and geographically dispersed patient pool.

Pediatric doctor visiting young girl in hospital dad with dad smiling in the background

In an industry survey led by PPD, Thermo Fisher Scientific’s clinical research business, rare diseases emerged as a top therapeutic area in drug developers’ pipelines, with 39% of global biotech and biopharma leaders pursuing therapies in the broad rare disease space.

The strong industry focus on rare diseases emerges as drug developers are contending with an already limited patient pool, requiring larger geographies to access these populations and ongoing challenges in patient recruitment. In our survey, patient recruitment and retention remain significant concerns cited by clinical trial sponsors across all therapies.

This is especially salient in the rare disease space, where patient populations are limited and, as such, may be significantly dispersed geographically. Research by Global Data finds that more than one out of four rare disease clinical trials terminate due to low patient accrual rates.

While rare disease trials present obstacles in patient recruitment and retention, biotech and biopharma organizations that form partnerships with patients and advocates – and work with clinical research partners skilled at doing so – can develop and execute more successful clinical trials. Patient centricity in the rare disease space, including early adoption of platforms and services that reduce clinical trial burdens, has taken on a more crucial role.

Read on to learn three ways clinical trial sponsors can strategically engage rare disease patients and advocates to make studies more advantageous for everyone involved.

1. Involve patients and caregivers early in the planning process.

Sponsors that seek patient input throughout the planning process and build their programs with this input in mind are more likely to build trials that work for patients and their caregivers, maximizing recruitment and retention success. Patients and their families are often their own best advocates, especially in the cases of rare diseases, and provide valuable insight into the experience of living with a rare condition.

Roughly 75% of rare diseases are exclusively pediatric in onset, so the logistics of study participation are dependent on parents and caregivers rather than patients alone. When trials are designed with the patient and family experiences in mind, when appropriate, it ensures that participation is feasible and positive — and makes participants more likely to start and to stick with the trial from start to finish. Sponsors must evaluate study demands closely for patient feasibility.

Similarly, studies should contain outcome measures that are meaningful to the patient, sensitive to measurable patient change and appropriately tailored to the disease of interest. The incorporation of patient-reported outcome measures in addition to clinical evaluation scales can offer a more relevant and holistic picture of patient improvement and better capture the nuances of a rare disease.

2. Expand access with decentralized clinical trials (DCTs).

Among respondents whose organizations currently use DCTs, usage in the next two years is expected to grow by 24 percentage points, according to our industry trends survey. Those not currently using DCTs anticipated that more than a quarter of their trials would be at least partially decentralized by 2024.

In the rare disease space, decentralization is a foundational step toward reaching a sparse patient population and reducing the burdens of trail participation for both the patient and family. Most rare diseases have few dedicated research and treatment facilities worldwide, making conventional in-person, on-site study formats challenging for patients and families.

Decentralization allows sponsors access to more participants without geographic constraints and removes burdens and barriers to participation for patients. This is especially valuable for critically ill patients or those with significant limitations in mobility, who may be unable to safely travel for study participation. By incorporating tools like telehealth visits, eConsent, ePRO platforms and in-home health care into a trial design, sponsors make participation feasible within an already limited study population.

3. Leverage community connections to recruit trial candidates.

New recruitment methods, such as relationships with patient advocacy groups and active patient communities, shouldn’t be overlooked as a recruitment strategy for rare disease trials.

For many rare disease patients and caregivers, connections through online communities or dedicated advocacy organizations are important sources of support, knowledge and understanding. Small but widespread patient populations can be united in one place, making them much easier to reach.

By engaging with these groups, trial sponsors can spread awareness and information about clinical research opportunities and potentially recruit patients. Importantly, these connections accomplish more than just study recruitment. Working with reputable groups can build patients’ trust in clinical research, assuage hesitations and misconceptions, and help patients to appreciate their valued role in developing therapies for their rare condition.

Make rare disease clinical trials work better for every stakeholder.

Rare disease trials present many distinct obstacles, requiring drug developers to have a profound understanding of the diagnostic odyssey patients and families face, the specific care landscape and the overall experience interacting with institutions and providers. Knowing this information will allow developers to leverage innovative research tools and methods and to develop long-standing partnerships in the specific rare disease community.

Biotech and biopharma organizations that select an expert contract research organization (CRO) partner will have an edge in designing more accessible trials, recruiting and retaining study participants, and meeting the evidentiary needs of regulatory bodies.

We design and conduct rare disease studies that solve the challenges that patients, advocates and drug developers face. Learn how our global rare disease study expertise allows us to create an unmatched approach for each client.

Check out the rest of PPD’s findings about the state of the drug development industry, and learn how our experts can partner with you to simplify your next drug development project.