The bar for patient evidence in EU health technology assessment (HTA) is rising fast.
As the Joint Clinical Assessment (JCA) becomes mandatory under Regulation (EU) 2021/2282, sponsors in the rare disease space are under increasing pressure to deliver earlier, clearer, and more structured patient insights.
Yet many organisations still operate on outdated assumptions, beliefs that may have been true in a pre-JCA world, but which now risk slowing access and weakening value dossiers.
Here are five of the most common myths we see and why they no longer hold up.
Myth 1: “Patient evidence isn’t needed until after approval.”
Reality:
HTA bodies expect meaningful patient input much earlier, often before your clinical narrative is finalised.
In particular, insights linked to Modules 3, 5, and 7 can strengthen the relevance of your value story, and help you anticipate questions from national assessors.
Myth 2: “We’ll figure out patient needs once pricing is sorted.”
Reality:
By then, you’ve missed the moment.
Burden of disease and unmet need are key components of Module 3 and they directly influence access outcomes, especially in ultra-rare or high-uncertainty conditions.
Early input isn’t just helpful. It’s strategic.
Myth 3: “Our PROs cover everything.”
Reality:
Patient-reported outcomes (PROs) are essential, but they’re not enough on their own.
You also need to show why those outcomes matter to patients, and how they reflect real-world impact. That means linking qualitative input, lived experience, and even advocacy insights to your PRO narrative.
Myth 4: “Real-world insight? That’s for the post-launch team.”
Reality:
This mindset causes unnecessary gaps.
Real-world insight including early access data from schemes like EAMS or ATU, can be instrumental in shaping your JCA narrative. These data points demonstrate how the treatment works outside the trial setting, and how patients perceive benefit in practice.
Myth 5: “Advocacy is for awareness, not evidence.”
Reality:
Structured advocacy input can strengthen your evidence base if it’s traceable, scoped, and transparent.
More and more, HTA bodies want to know how you involved patients and how their views shaped the submission. Advocacy isn’t peripheral. It’s part of the package.
Conclusion: What This Means for Sponsors
These five myths don’t just create blind spots — they cost time, trust, and impact.
To meet the new expectations of EU JCA, sponsors must move beyond tokenistic or late-stage patient input. Instead, they need integrated, strategic patient evidence that’s mapped to HTA needs from the start.
At RAREready, we help sponsors build this capability, combining advocacy engagement, behavioural insight, and AI-enhanced evidence workflows to accelerate EU readiness.
Curious how your patient strategy holds up under EU JCA?