Three evidence planning signals for rare diseases in 2026

Several signals from policy bodies and industry commentary suggest evidence expectations are evolving in 2026.  For rare diseases, where small patient populations and limitations in traditional clinical data are inherent, these developments reinforce the value of thoughtful, well contextualised evidence planning rather than simply larger datasets or more complex models.

Below we discuss three key areas of change and their implications for rare disease evidence strategies.

1. Patient experience data continues to gain structured attention

In late 2025, the European Medicines Agency (EMA) published a draft Reflection Paper on Patient Experience Data (PED), now open for public consultation until January 31, 2026 [1]. This guidance does not create new regulatory requirements but encourages medicine developers to consider PED throughout the product lifecycle, from pre-authorisation to post marketing.

The paper emphasises that PED can include a range of patient or caregiver reported information, such as symptoms, health related quality of life, treatment preferences, and treatment impacts.  It also recognised multiple data sources including clinical trials, real world data, and digital or patient generated data streams, and stressed the importance of transparent reporting of methods and context [1].

In rare diseases, where clinical evidence is often constrained by small samples and heterogeneous presentations, structured inclusion of patient experience can add essential context. It enables sponsors to articulate outcomes that matter to patients and carers in settings where traditional endpoints or large trial datasets may not exist. The framework remains deliberately principle based, prioritising clarity of purpose and proportionality over prescriptive methodological standards.

Implication for 2026: Early engagement on PED strategies, with well-defined objectives, realistic scope, and clear reporting, can strengthen evidence packages for regulators and HTA bodies without overstating their role.

2. Real world evidence and analytics are increasingly central to value narratives

Recent industry commentary on HEOR and market access trends for 2026 suggests a shift in how real-world evidence (RWE) is viewed, moving from a supportive input to a core component of value narratives in certain contexts [3]. This is particularly relevant for rare diseases, where clinical trial data alone may not fully capture treatment use, outcomes, or longer-term experience in routine care.

Several themes recur across recent discussions:

• RWE is increasingly considered alongside clinical and economic evidence in access and reimbursement discussions

• Advanced analytics, including AI enabled approaches, are being used to support interpretation of complex or multi source datasets, particularly where data are sparse

• Pricing and access discussions are evolving in parallel, with increased scrutiny of budget impact, uncertainty, and outcomes-based considerations in some therapeutic areas [2,3]

  
  

These trends do not imply that RWE replaces traditional evidence, instead that, increasingly, it is expected to be well integrated and clearly contextualised.

Implication for 2026: For rare disease programmes, RWE strategies that explicitly acknowledge limitations while explaining relevance to payer and policy questions are likely to be more credible than approaches that rely on scale or complexity alone.

3. Evidence planning is increasingly cross functional and occurs earlier in the lifecycle

A further theme emerging from recent HEOR and market access outlooks is the importance of earlier, more integrated planning across clinical development, HEOR, access, and policy functions [3]. Rather than sequential evidence generation, there is growing emphasis on aligning evidence objectives and outputs across disciplines from an early stage.

For rare diseases, this approach is particularly important. Small patient populations often necessitate multiple complementary evidence streams, and misalignment between disciplines can lead to gaps, duplication, or evidence that does not fully address decision-maker needs.

Implication for 2026: Cross-functional planning can help ensure that PED, RWE, and economic analyses are designed to work together, producing clearer and more coherent narratives for regulators and HTA bodies.

Practical considerations for rare disease evidence planning in 2026

Taken together, these developments suggest several pragmatic considerations for sponsors planning evidence generation in rare diseases in 2026:

• Define evidence objectives early and link them to specific regulatory or access decisions

• Integrate patient experience, RWE, and clinical data in a way that is proportionate and transparent

• Be explicit about methodological choices, constraints, and uncertainty

• Engage cross-functional teams early to support coherent evidence packages

  
  

Rather than signalling abrupt changes in requirements, recent guidance and commentary point towards a more explicit expectation that evidence in rare diseases is carefully reasoned, clearly contextualised, and aligned with decision-making needs. For evidence, HEOR, and market access teams working in rare diseases, these signals have direct implications for 2026 planning.

References

[1] European Medicines Agency. Reflection Paper on Patient Experience Data. Consultation open until 31 January 2026.[2] ReMap Consulting. Market Access Trends 2026.[3] Xtalks. What’s Driving HEOR and Market Access in 2026? Four Trends to Watch.