Trial flexibility: Preventing rare disease patients exclusion from R&D

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Erica Cohen, chief experience officer, Greenphire, and Thomas Smith, life sciences public engagement specialist, explore the current issues that hinder rare disease clinical trials, and what solutions should be invested and incorporated to maximise flexibility for the trial patients.

Key insights:

  • Rare disease trials typically require more travel than non-rare diseases due to the limited accessibility of trial sites - emphasising the importance of adopting hybrid decentralised trials.
  • Trial-related expenses - such as distance to travel and time off work - discourages participants from trials which labours one of the biggest threats to a clinical trial; participant retention.
  • Sponsors and CROs need to provide sites with access to global services but also maintain visibility and cost predictability through their patient-centric suppliers and practices.

According to Rare Diseases Europe (EURORDIS) and its global partners, there are over 300 million people around the world living with rare diseases. Due to the lack of medical knowledge about these diseases, there is an urgent need for continuous research to be done. However, rare disease patients face many obstacles in being able to participate in studies, which in turn, limits the number of clinical trials taking place and the ability to develop new treatments.

The emotional and economical burdens that individuals with rare diseases face are immense. These patients often struggle for years attending countless doctor visits, undergoing unnecessary procedures, and receiving wrong diagnoses before ever receiving an official or accurate diagnosis, also referred to as the diagnostic odyssey.

During this time patients can begin to isolate themselves from society, which can lead to depression, and affect personal relationships with loved ones due to the added pressure of what lies ahead in the patient's journey.

Research has shown the average time for an accurate diagnosis is 4-5 years, and in some cases, takes over a decade. Meanwhile, the individual's physical and mental health declines and symptoms worsen. With wildly variable health literacy and standards of care across the globe, some people die without ever receiving a formal diagnosis.

While progress has been made in research and development for rare diseases, there are still many barriers and challenges the industry faces in conducting clinical trials for these individuals, due to the lack of data and overall knowledge of these diseases, their symptoms, and the effectiveness of treatment.

Participating in a clinical trial can be life-changing for these individuals, and the need to provide flexibility in order for people to be able to participate in research and achieve therapeutic innovation is more important than ever, especially given we now have mechanisms, products and services that help to this end. In short, there are increasingly fewer excuses.

Financial toxicity

In the clinical trial space, there continues to be a concern amongst participants on the cost of participating in a clinical trial.

In CISCRP’s 2021 Perceptions and Insights Study, individuals who have never participated in clinical trials cited potential costs and reimbursements as “very important” factors to consider when deciding to enrol in a trial.

In a report issued on the national economic burden of rare diseases, which analysed over 300 rare disease trials, the average monthly out-of-pocket expense per patient was nearly $2,300.This is compared to $1,000 per month for participants involved in early-stage oncology trials, according to the NIH. Despite the discrepancy between both of these figures, it is utterly unacceptable that economic status plays any part in a persons’ access to and interest in a clinical trial.

In most cases, these trial-related expenses are nearly impossible to endure, especially for those whose diseases keep them out of work, and require caregivers, leading many to question the benefit if there is no guarantee of treatment or that the treatment will work. It is coercive and actually encourages participants away from trials which labours one of the biggest threats to a clinical trial; participant retention. Not to mention that people with diseases and comorbidities are disproportionately economically hindered already – even if they are fit enough to work.

82% of clinical trial sites reported that delays in payments from study sponsors have a negative impact on their business operations. When site payments are delayed, participant payment delays follow.

With recent technological innovation, we now have the power to ensure participants receive their reimbursements in a timely manner – and even the ability to provide digital payments.

Without a financial barrier, clinical trials become more equitable which allows us to get more people in better health.

Clinical trial burdens

The cost of participation is just one obstacle but there are others. In a survey conducted with people who have participated in clinical trials, travel logistics was cited as the top burden.

Rare disease trials typically require even more travel than non-rare diseases due to the limited accessibility of trial sites, further emphasising the importance of hybrid decentralised trials. For example, in 2022, rare disease patients reported traveling an average of 135 miles to their clinical trial sites, compared with 67 miles traveled by non-rare disease trial participants.

Longer distance travel not only adds additional trial expenses, but also creates a heavy physical and emotional burden on these individuals – from accommodations to travel logistics, familial separation and time zones, and isolation from the things they actually want to do with their lives

Along with coordinating itineraries and flights, there are other accommodations that need to be made if the patient is traveling with medical devices, requires a passport, or will need transportation from the airport to hotels and trial sites. For patients that have not traveled before this can often cause stress and confusion, making it even more important to ensure they feel comfortable and understand what is needed for them to travel.

Like financial expenses, logistical elements are too challenging to overcome and the patient, caregivers, or families may decide it’s prohibitive. There are also considerations to be made to help people attend trial sites in the way that they see fit. Even if cost is not an obstacle, some patients can experience detrimental impacts on their physical and emotional well-being in getting to the site if they don’t have the resources. It does not need to be like this.

Importance of flexibility

With limited trial locations, financial burdens, and varying digital health literacy, the patient and caregivers can face unanswered questions and uncertainty.

Sponsors have an obligation toward the well-being of the patients they serve. Participants need the space to consider the best treatment options for them. Sponsors and sites need to focus on minimising concerns around trial locations. It is essential to provide services aimed at removing the burdens that come with orchestrating long-distance travel sometimes with specialist support needs.

Providing clinical trial sites with comprehensive options enabling patient travel allows the site staff with the opportunity for unwavering focus on patient care and trial conduct.

Sponsors and CROs need to provide sites with access to global services but also maintain visibility and cost predictability through their patient-centric suppliers and practices.

The evolution of technology within the clinical trial space has allowed the industry additional flexibility, catering to patients individually. Leveraging this technology ensures patient access to all available treatment innovations.

The continued development of effective treatments for millions of people and thousands of rare diseases around the globe, requires innovation to remove any barriers limiting the appeal of clinical trials.