Overcoming the Challenges in Rare Disease Market Research

Navigating the complex landscape of rare diseases presents unique challenges and opportunities for pharmaceutical companies and market researchers. Understanding the nuanced needs and preferences of patients is not just beneficial — it is essential for driving innovation in pharmaceuticals and healthcare.

In this article, we highlight two key challenges with conducting market research in rare diseases, the implications of these challenges as well as solutions that pharmaceutical insights teams should consider when conducting market research in rare diseases to inform their brand strategy.

Challenge 1: Engaging with patient advocacy groups (PAGs) too late (or not at all!)

Background: PAGs are crucial allies, offering unparalleled insights and access to the rare disease community. Their early involvement can transform market research outcomes. However, too often PAGs are engaged late in the market research process for the purposes of recruitment.  

The implications of this are numerous:

• Limited access to patient networks: PAGs often have extensive networks within the rare disease community, including patients, caregivers, and healthcare professionals. Without their early involvement, researchers may struggle to identify and reach potential participants for their studies, leading to smaller sample sizes and less representative data.
• Lack of trust and credibility: PAGs play a crucial role in building trust between researchers and the rare disease community. Their endorsement of market research can enhance credibility and legitimacy, encouraging more active participation from patients. Without PAG involvement, researchers may face scepticism and reluctance from patients to engage in market research activities.
• Missed insights and perspectives: PAGs offer valuable insights into the lived experiences, challenges, and priorities of individuals affected by rare diseases. By collaborating with these groups, researchers can gain a deeper understanding of patient needs, preferences, and priorities, which are essential for developing patient-centred approaches in healthcare and pharmaceutical innovation. Without PAG input, researchers risk overlooking critical perspectives that could inform the development of more effective treatments and interventions.

Solution: Incorporate PAG collaboration from the project's outset, ensuring comprehensive insights and enhanced community trust.

Challenge 2: Trying to fit square pegs into round holes; the challenge of small sample sizes and plenty of variation.

Background: In hard-to-reach rare patient populations, patient journeys and experiences are often highly nuanced and individualised – this can make for challenging interviews to both moderate and analyse with significant variability.

Implication: Traditional research models may overlook the nuanced experiences of rare disease patients, risking valuable insights and innovations. Researchers may feel tempted to try to fit patients into neat and tidy boxes, at the same time overlooking the individual nuances that are strategically important to understand in rare diseases.

Solution: Utilise the PEACE model in qualitative interviews to prioritize patient narratives, ensuring a holistic understanding of their unique experiences. The PEACE model is a psychologically-founded interviewing method that allows the patient to 'drive' the discussion. This respects the individuality of patient experiences, and recognises the diverse and personal nature of their journeys. This approach is vital in understanding the complex realities of those living with rare diseases. At Sequentis Health, our skilled researchers are trained to utilise this model in patient research.

Contact info@sequentis-health.com to further discuss your rare disease market research!