Navigating the Rare Disease Experience: A Three-Part Series

PART THREE

To be most successful in engaging rare disease patients, you must rely on the only true experts – the patients themselves. No one understands the experience of living with a rare disease better than the individuals who suffer from them. A rare disease affects a patient’s physical, mental, and emotional health. The experience of living with a disease for which there is most often no treatment or cure is grueling and debilitating. In a previous post [link to BLOG #2] we discussed the ways in which patient insights on their experience can inform study development. It is helpful to incorporate patient feedback into your recruitment strategy when enrolling patients in a rare disease study. observe that “Engagement of patients and other stakeholders in clinical research may help to ensure that research efforts in rare diseases address relevant clinical questions and patient-centered health outcomes…Engagement may promote research that evaluates health outcomes that are both relevant to patients with rare diseases and useful for decision making.”[1] Patient contributions may take several forms.

Patients can help you brand advertisements and educational collateral for your study. They know what sort of information will catch the eye of your target patient population, and they will also know the most sensitive and effective ways to present the information that you need to convey in your materials.

Patients can work with you to create highly-targeted national and global campaigns. These are cost-effective recruiting methods for most studies, but for a rare disease trial, you are trying to reach a “needle in a haystack” patient. Feedback from the exact group you are trying to reach will help you refine your campaign.

Many patient populations suffering from rare diseases are part of networks and communities (on the ground and online) where they share information about treatments they have tried or treatments that may be available. Refer-a-friend programs are an effective recruiting technique to employ for a rare disease trial due to the direct access you can have to these niche populations.

Effective engagement with rare disease patients for clinical trials lies in customization around the target patient population. Imagine you are running a clinical trial for scleroderma. Scleroderma is a rare disease that affects primarily females. Symptoms include tightening of the skin of hands, fingers, and mouth; joint pain; trouble breathing and swallowing; sexual dysfunction; and an exaggerated response to cold. The symptoms present themselves in varying amounts and intensities form patient to patient, and can be very frustrating when moving through daily life. These patients also tend to struggle socially. If you work with the patient population to recruit for this study, here are some things that might come up:

  • Branding of study collateral should lean toward the feminine; colors, fonts, language used
  • Messaging for the study should focus on how the disease affects aspects of daily life and lowers quality of life
  • Advertising and outreach should focus on reaching patients and their communities directly
    • Support groups on Facebook
    • Advocacy group walks & events
    • Physician education initiatives

If you know all of this insider information before beginning to recruit for your study, you will be ahead of your enrollment goals before you even begin.

Patients should be treated as partners in the study development process, from protocol review to engagement in a collaborative recruitment strategy. Remember – a rare disease study is a mutually beneficial partnership between clinical researchers and the patient community. Our patients need us just as much as we need them. If you keep the ultimate goal of a successful treatment in mind throughout the clinical study process, you will be serving your target patient population well.

The ClinEdge Rare Disease program utilizes defined methods to compassionately engage, partner and empower rare disease patients and their families. Our global strategies improve the clinical trial process by using the voice of the patient to build and support clinical trial programs, using their own journey and story to increase patient engagement and retention. about how we can drive your study success with a patient-centric focus.

 

[1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4124116/

Melissa Daley

Post by Melissa Daley -

Melissa develops social media and digital marketing content strategies and produces a variety of collateral with creative, effective messaging. Melissa has served as an educator in higher education for close to two decades.

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