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Strategies for Maximizing Patient Engagement in Rare and Orphan Disease Clinical Trials

While it may seem obvious that clinical trials for rare and orphan disease groups are the most challenging for patient recruitment, the biggest hurdle lies in retaining those patients and the costs associated with bringing the patients to the study with all its complex procedures to collect data when results are not guaranteed. Beginning with the recruitment process, how do you target the right patients, especially when those patients are under or non-diagnosed?

We recently gathered with a group of clinical leaders in companies conducting challenging studies in rare diseases, and we have summarized below some of the key takeaways to consider when planning for these types of programs.

Recognize that KPIs should include high site and recruitment failure rates, allowing for significant time and cost devoted to both identification of patients and start up activities for centers 

When dealing with complex patients, factors normally used for screening more generic or “one size fits all” populations may be muddied by co-conditions or underlying conditions that create symptoms that correlate to an inaccurate diagnosis. It’s important to use distinctive key performance indicators (KPIs) that identify the correct biomarker. From a regulatory perspective, there are geographical distinctions between what is classified as a “rare disease” in each region of the world – something that can further complicate global or country to country recruiting.

While physician referrals may seem like an obvious route to recruiting, there is currently a strikingly small percentage of study participants that come from providers. Many physicians do not want to lose their patients,  and are therefore disincentivized to encourage enrollment in another treatment center. There is also a perception that clinical trials are an “11th hour” solution instead of an integrated part of patient care. In some cases, physicians are unable to even diagnose a patient, and this is instead initiated by Clinical Research Associates that infer diagnoses based on patient records. We must migrate towards a mindset of physicians acting as fearless advocates for their patients. We also need more physician education to enable identification of clinical research as a care option, and tools to facilitate diagnosis.

Social media has been facilitating patient identification and recruiting through the establishment of niche patient communities like those fostered through Inspire. They allow patients to communicate with others experiencing the same condition and provide a means by which a sponsor can access unique patient populations that they otherwise may not be able to identify without significant investments in recruitment activities.

In addition to this, understanding patient motivation for participation can also lend itself to retaining patients once they are identified.

Decreasing patient (and caregiver) burden through protocol design

Patient retention obviously varies for many reasons, but from a high level perspective, those that are diagnosed with life-threatening rare diseases are typically more likely to be adherent to their clinical trial protocol. Depending on the perceived risks vs. benefits of study participation for the patient and/or their caregiver (including time invested and inconvenience), a treatment that may provide a cure versus alleviating symptoms and improving the potential for a better long-term outcome are just a few of the factors that can dampen a patient’s willingness to participate.

While not appropriate for every study, utilizing digital tools, telemedicine, and remote monitoring of patients wherever possible are great enhancements in designing protocols to be more patient and caregiver friendly. If feasible, in-home nurse visits for procedures like blood draws and infusions to prevent the travel burden on behalf of the patient, telemedicine interim visits, and easily accessible check-in sites can all contribute to enhanced participation. When it comes to consent forms, utilizing iPads or visuals to support patient understanding of procedure and expectations can put the patient more “at ease” than a stack of forms, but are still not evolved to an optimized user friendly state at this time. It’s important to note that these aides must still be reviewed and approved by the Institutional Review Board before being used.

For patient touchpoints such as any opportunities to interface with the research site, interactions with clinical trial monitors and associates, and even transit, keeping faces familiar and consistent can foster a stronger sense of trust with the study site staff and increase overall comfort with the study experience. Avoiding outsourcing of services that may lead to gaps in continuity can change the patient and caregiver experience from a negative one to a positive one that yields quality data.

When budgeting for the trials, it’s prudent to include additional time or allocated funds to allow for extended or additional onsite visits with research staff to add a ‘white glove touch’ to the patient experience and enhance the relationships with site and patients.

Addressing cost-benefit gaps 

When budgeting for a challenging study, it is important to first consider realistic screen failure rates and communicate this to management so that expectations are set. After budgeting for initial recruiting, planning must also take into account the high costs of transit, lodging, and other miscellaneous expenses associated with getting the patient (and potential caregiver) to a study site. It is important to ask if the benefits of patient reporting outweighs the costs of executing the study. Additionally, it is critical to understand that many hospitals are not able to offer reimbursements for travel, making this a cost the sponsor must plan for and absorb. The costs for all of the additional patient and family support activities can potentially double the individual patient costs, but this is just an estimate. Care should be taken to consider the risks of prolonged delays as well as site replacement to justify the costs.

Accurately sharing success

When a study is yielding positive results, it is important to be clear, concise, and correct about how your campaign disseminates that information. Communicating success is a clear means of patient retention, as long as it is done well, and will provide the patients and families with the ‘white glove’ attention that must be factored in to build advocacy and patient community relationships with an eye towards future studies and  commercialization activities.

Clinical trials are already complicated and challenging as it is. We hope that by sharing a few of the insights gained through our community, your organization may be able to implement and efficiently execute in a way that puts the patient first to generate successful, reliable results. With smart strategies, we can relieve burdens at all touchpoints along the journey for clinical trial patients with rare and orphan diseases.

Questions about your clinical development and operations, contact us today.