The EMA’s Reflection Paper published in September 2025 confirms an important shift. Patient experience data is no longer supporting material. It is core clinical evidence that informs regulatory and HTA decision making, including the EU Joint Clinical Assessment.
For rare-disease sponsors, this raises practical questions. Many teams collect insight from patients and carers, but the material varies in format and quality. The expectation now is structure, transparency and traceability.
What the EMA Reflection Paper sets out
The EMA asks companies to consider patient experience data throughout development. This covers early insight work, contribution to endpoint selection, preference studies, patient-reported outcomes and post-marketing evidence.
The Reflection Paper highlights the need for clarity on how the data was generated, who contributed, and how it shaped decisions. This is closely aligned with the Joint Clinical Assessment requirements, which ask sponsors to justify outcomes and PICOs in a way that reflects patient needs.
Why this matters for the Joint Clinical Assessment
The Joint Clinical Assessment is part of the EU HTA Regulation. It introduces a single EU-level clinical assessment that Member States will draw on for their pricing and reimbursement decisions. It applies today to oncology and ATMPs, and to orphan medicines from 2028.
This creates greater visibility of evidence gaps. If patient insight is unstructured or arrives late, it becomes difficult to show how it influenced outcomes, trial design or interpretation of benefit.
This challenge is more common in smaller and mid-sized companies. Many do not have dedicated patient engagement staff. Work often sits with Market Access, Medical or Regulatory, usually when timelines are already tight.
Where operational risk appears
When patient experience data is not organised early, issues tend to emerge:
- outcomes are harder to justify
- PICO definitions do not reflect what matters to patients
- affiliate insight arrives too late to shape planning
- dossiers appear incomplete
- timelines become more difficult to manage
These problems affect internal decision making and increase uncertainty as teams move toward the 2028 transition for orphan medicines.
A practical approach for sponsors
Most rare-disease programmes already collect meaningful patient and carer insight. The main issue is structure. Teams need a way to turn diverse insight into evidence that is clear, consistent and decision ready.
RAREready helps address this by:
- mapping patient experience data to outcomes and PICOs through a structured evidence grid
- maintaining a traceability log that links insight to decisions
- supporting affiliates to provide early, organised patient input
- using AI inside standard office tools to bring consistency to qualitative summaries
This gives sponsors earlier visibility on how their patient evidence may land in the JCA and where gaps may exist.
What teams can do now
Practical steps include:
- reviewing how patient insight is gathered today
- checking whether outputs are consistent and traceable
- identifying where insight arrives too late
- aligning early patient evidence with the expectations in the Reflection Paper
- preparing affiliates to contribute insight in a clear, structured way
For many teams this is not about adding new activities. It is about improving timing and structure.
Further reading
EMA Reflection Paper (September 2025):
https://lnkd.in/erjUDAXE
You can learn more about early evidence readiness at www.rareready.health.