Rare Diseases - When Market Research Reaches Its Limits

Sometimes market research must push boundaries. This is particularly true when it comes to rare diseases. When the incidence rate is exceptionally low, yet the burden is high, it requires creative recruitment professionals and researchers with expertise and a delicate touch. Read here to find out how it can be done.

How do we succeed in attracting a very small and specific target group for our research? What challenges and emotional states do patients with rare diseases experience on their journey to diagnosis and therapy? How can we make the patient journey more efficient and improve the quality of life for those affected?

Many pharmaceutical companies specializing in rare diseases face these and similar questions. This is because people with rare diseases often endure years of suffering, marked by numerous incorrect diagnoses and unsuccessful treatment attempts. Without sufficient knowledge of the patient journey, pharmaceutical companies are also unable to address the specific needs of those affected.

As part of the project, patients with a rare and difficult-to-diagnose disease were to be recruited for the study. Estimates suggest that the disease has been diagnosed in only about 300 people in Germany. This low incidence and the specific recruitment requirements made a traditional approach impossible.

Our team therefore strategically leveraged networks and patient organizations to realize the project. Through close cooperation and jointly developed trust-building campaigns, we succeeded in gaining access to the target group.

Initially, the response was muted. However, we soon experienced a positive turn: After the first interviews were successfully conducted, participants shared their positive experiences in patient forums and networks, thereby promoting the project. This led to a rapid increase in registration numbers on the specially set-up online platform within a short time.

Crucial to reaching our targeted sample was the continuous trust-building communication towards the target group. At every stage, clear and transparent information was provided about the study goals and process, highlighting the benefits of the project for all involved.

The interviews also aimed to create a trusting atmosphere and consider the unique situations of the respondents. The patient journey for rare diseases is not merely a series of medical appointments and diagnostic steps but also a deeply emotional process where phases of hope and disappointment alternate, often culminating in resignation.

In our study, it was crucial not only to document the physical and medical milestones on the path to diagnosis but also to explore the emotional state of the patients. Consequently, researchers faced the unique challenge of sensitively and accurately capturing these shifting emotional states to paint a comprehensive picture. This demanding task required experienced qualitative researchers who have the ability to explore sensitive health topics in interviews and provide interviewees with the space to openly discuss their fears and desires.
 
For the respondents, the interview meant more than just a "research survey" – they found it particularly relieving to express their experiences and all associated emotions in conversation. As a result, the interviews became not only a valuable source for the clients but also a supportive experience for the respondents, who could openly share their stories and feelings.

Encouraged by the positive experiences from the initial survey phase, the client decided to further refine the project results using a collaborative approach and to develop actions. Moderated by our research professionals and design thinking coaches, employees, medical specialists, and patients discussed and further developed the patient journey.

Together, they succeeded in identifying the critical Moments of Truth – those key moments where the patient's path to the correct diagnosis and therapy can be influenced. These insights allow for the design of information and communication strategies that provide guidance to both doctors and patients, increasing the chances that those affected find the appropriate support at an early stage.