Navigating patient experience data in rare disease

Patient experience data (PED) has become increasingly important in drug development, and especially so for rare disease, offering critical insight into how people live with their condition, what outcomes matter most to them and how they experience participation in research.

Regulators and HTA bodies expect to see PED alongside clinical evidence in submissions. Yet collecting patient insights is only part of the story. Across the pharmaceutical industry, dedicated teams work to bring patient perspectives into development for the right reasons, yet they frequently face the challenge of translating these efforts into a tangible reflection of the patients’ true priorities and realities.

A key objective of capturing PED is to identify and help address unmet patient needs.
 
However, at Sprout we recognise that pharmaceutical and biotech companies operate within commercial, regulatory and operational constraints, and drug development is as much a business endeavour as it is a scientific one. Decisions must balance budgets, timelines, evidence requirements and stakeholder expectations. While these competing demands can create tension, our specialists know they can also be navigated transparently, with a continued focus on what matters most to patients.

Maintaining this focus on patient priorities is particularly important in rare disease, where traditional measures of success may not always reflect what matters most to patients. As rare disease patient advocate and cystic fibrosis survivor, Caleigh Haber observed: “A statistical win is not always a patient win.” This highlights the challenge of ensuring that success metrics remain connected to outcomes that patients themselves value. Meaningful engagement is not defined solely by trial outcomes, but by whether patients feel heard, informed and involved throughout the development process. Keeping this perspective at the centre of decision-making helps ensure that patient needs remain visible, even as priorities and constraints evolve.

Patient communities in rare disease are often small, highly engaged and deeply invested in advancing research. This places a responsibility on sponsors to be transparent, set realistic expectations and demonstrate how patient insights inform decision-making. These principles become particularly important when a trial or study is stopped. Such moments can expose a gap between sponsors’ intentions and patients’ experiences, but they also reveal what patients value most in their interactions with research.

Patients understand that drug development is inherently uncertain and that success cannot be guaranteed. What often shapes their experience is not simply the outcome itself, but how decisions are communicated, whether expectations have been managed honestly and whether their contributions have been acknowledged and respected. While transparency cannot eliminate disappointment, it can help preserve trust and reinforce the value of continued partnership.

Understanding how patients experience these moments is essential if sponsors are to learn from them and improve future engagement. At Sprout, we advise clients to view these moments not as the end of a conversation, but as a critical opportunity to learn.

In one recent rare disease programme, we worked alongside both patient communities and the sponsor following the decision to discontinue development. By capturing patient perspectives before, during and after the announcement, we helped identify what information patients needed, how communication could be improved and which aspects of the experience had the greatest impact on trust. These insights informed both the immediate engagement strategy and future development programmes, ensuring that lessons from the study were not lost alongside the asset itself.

Experiences such as these demonstrate that the value of PED extends beyond supporting successful development programmes. It helps sponsors understand the consequences of difficult decisions, improve future engagement and strengthen the relationship between patients and industry. That relationship should be reciprocal: patients share their experiences, priorities and unmet needs to help shape better research, while industry is transparent about its remit, constraints and decisions. The balance is not a fixed point, but an ongoing dialogue grounded in openness, humility and respect for the fact that only patients themselves can judge what is meaningful in their own lives.

Learn more at Sprout-hs.com