Navigating the Rare Disease Experience: A Three-Part Series

PART TWO

Living with a rare disease is an experience that few can comprehend. Clinical researchers can’t expect to succeed with a rare disease trial unless they go the extra mile to understand the patient population in question reports that “Living with any disease can be a trial, but patients with rare conditions face a host of uniquely different challenges. Simply getting an accurate diagnosis can be an enormous task, and many with poorly understood illnesses struggle with isolation and loneliness.”[1]  When preparing for a rare disease trial, partnering with the patient population to develop your study is the most effective route you can take.

Rare diseases affect patients’ physical, mental, and emotional health. It is incredibly important to understand each of these facets of a patient’s experience in order to best serve them with a clinical trial opportunity. When developing your study, there are several key questions that you should keep in mind, including:

  • What are the patient population’s general thoughts and feelings toward healthcare systems?
  • What are their thoughts, feelings, and knowledge of clinical research itself?
  • Has this community been involved in clinical research before?
  • How does this disease affect the patients’ work life? Social life? Family life?
  • What do these patients consider to be the most important end points? (ease of symptoms, a cure, improved mobility, etc.)

Your patient population may be generally frustrated with the healthcare system after enduring months, or maybe even years of appointments with various specialists who failed to diagnose them. They may have attempted clinical trials before only to have been excluded from the process. Or, clinical research may never before have been conducted for their particular patient population. All of this could illicit myriad  feelings including anxiety, fear, as well as curiosity and optimism. Discussing perceptions and concerns with your patients is important.

You should also understand how living with a rare disease affects a patient population’s ability to work. Do they often have to take sick days because their condition is debilitating? What about their social life? Do they frequently cancel plans or omit themselves from social outings because their disease limits them? Understanding the patient population beyond their physical condition will best serve them throughout the clinical trial process.

You should be engaging with the patient population before there is even a study on paper.

  • Identify different patient networks that may be accessible to you. It is valuable to reach out to medical specialists, disease support groups, and even caregivers to get feedback on what the particular experience of a given rare disease includes.
  • Conduct patient panels and surveys to ask patients directly – whether in person or digitally – what it is like to live with their disease and what outcomes that would like to achieve if they were to participate in a trial.
  • Coordinate social listening strategies  by reviewing  what patients post on social media. Look at Facebook groups and hashtags on Twitter and Instagram to get an authentic picture of what is bothering them most, what unique vocabulary they may use to describe their symptoms or experience, and what goals they have.

When creating your study protocol, patients can provide you with valuable insight on what is possible for the population in question. How many site visits is reasonable to include? How far can you ask a patient to travel to your site and still expect them to participate? What sort of study would they be most interested in participating in? What solutions do they want most?

 

How can you partner with rare disease patients when it comes to recruitment efforts? Part 3 of this 3-part series reviews approaches.

 

[1] https://www.nytimes.com/interactive/2018/03/27/well/patient-voices-rare.html

 

 

 

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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