Supporting structured, independent patient input in the EU JCA era
The European Joint Clinical Assessment (JCA) is reshaping how medicines are assessed across the EU. For rare diseases—where patient perspectives are often crucial to understanding clinical relevance—this shift offers new opportunities to influence outcomes. But it also creates new responsibilities for all stakeholders involved.
Patient advocacy groups, particularly in rare diseases, bring unique insights rooted in lived experience, daily management, and unmet needs. Their contribution can help ensure that assessments focus on what truly matters to people affected. However, to influence the JCA process meaningfully, patient input must align with the technical frameworks used by HTA bodies.
Central to this is the PICO model: Population, Intervention, Comparator, Outcome. While familiar to researchers and regulators, applying PICO in rare disease contexts is rarely straightforward. Advocacy groups must be supported in navigating its complexity—without being directed or influenced.
Understanding the Role of PICO in Rare Disease Contexts
Each element of the PICO framework presents distinct challenges in rare conditions:
- Population: Defining the target population is complicated by clinical diversity, diagnostic uncertainty, and variable prevalence across EU countries. Accurate, inclusive definitions are essential to reflect the full range of patient experience.
- Intervention & Comparator: For many rare conditions, trials are limited, and appropriate comparators may not exist. Patient groups often provide crucial context on treatment relevance and realistic alternatives based on real-world care.
- Outcome: Clinical trials may overlook outcomes that matter most—such as independence, fatigue, cognitive function, or caregiver impact. Advocacy groups can help align outcome measures with the lived priorities of patients and families.
Patient insight is not an “add-on.” It fills evidence gaps, sharpens relevance, and strengthens legitimacy. But for this input to be effective within the JCA process, it must be clearly structured, timely, and aligned with PICO terminology.
Building Capacity Without Steering Input
To participate meaningfully in the JCA process, rare disease communities need opportunities to strengthen their understanding of how HTA frameworks work. This includes knowing how their insights can be translated into the language of evidence and incorporated effectively.
This isn’t about influencing advocacy—it’s about creating space for structured independence. Effective support should:
- Be designed with and for rare disease advocates
- Reflect the realities of limited data, cross-border variability, and evolving evidence needs
- Provide practical tools and guidance that are relevant, accessible, and non-directive
Managing Conflict of Interest Transparently
In rare diseases, the lines between stakeholders can easily blur. Patient groups often rely on external funding to operate and engage in policy processes. This makes transparency around collaboration essential—especially in the context of HTA.
Any capacity-building initiative must address potential or perceived conflicts of interest openly and proactively. This includes:
- Clarity of Roles: Training must preserve the independence of patient voices, with no attempt to steer advocacy positions or preferences.
- Transparent Funding: Financial support and facilitators should be disclosed clearly and consistently.
- Credibility of Content: Training should be shaped by multi-stakeholder input, ideally involving both HTA experts and experienced patient advocates.
Handled properly, transparency builds trust—not only between patients and companies, but with HTA bodies and decision-makers who assess the credibility of submitted evidence.
A Shared Responsibility in the JCA Era
As the JCA becomes operational, its success in rare diseases will depend not only on methodology, but on inclusivity. The goal should be to ensure that structured patient contributions are not only encouraged but enabled—so they hold real weight in HTA decision-making.
This is a shared responsibility. Regulators, industry, and patient communities all have a role to play in building the infrastructure that supports independent, relevant input.
How We Can Help
At swii.ch health, we deliver independent, ethically grounded PICO training designed specifically for rare disease advocacy groups engaging in EU JCA. Our programs are:
- Co-created with HTA experts and patient leaders, ensuring relevance and credibility
- Focused on translation, helping patient groups structure lived experience into PICO-aligned contributions
- Fully transparent, with clear disclosure of funding and facilitation to safeguard trust and neutrality
We don’t speak for patient groups—but we help them speak the language that ensures they are heard.
To explore how we can support your patient partners in building credible, independent input for JCA, get in touch with our team.