Making sense of medicine access

On Public Day in Brussels, the World Bladder Cancer Patient Forum 2025 hosted “Making sense of medicine access,” a practical, multi-stakeholder conversation on how promising science becomes real treatment for real people. Moderated by Lauren Pretorius (Campaigning for Cancer), the session brought together Mihai Rotaru (EFPIA) and Robert Sauermann (Austrian Federation of Social Insurances) to unpack where and why access stalls, and how to fix it.

Innovation only matters if patients receive it. Opening the session, Lauren reminded the room that breakthroughs mean little if they never leave the shelf. Access is a human-rights issue, she argued, and understanding the full route from approval to reimbursement and uptake is essential.

What the data says (industry view). Mihai shared EFPIA findings showing wide variation in the availability and speed of access to new cancer medicines across Europe. He highlighted a growing “grey zone” of limited availability (narrow indications, individual schemes, private-market routes) that helps some but fails to deliver equity. Most delay, he noted, stems from lengthy national pricing and reimbursement processes rather than companies simply “not launching.” Practical levers include stronger, HTA-ready evidence packages, earlier dialogue with payers, and broader use of outcome-based agreements and coverage-with-evidence-development.

How payers decide (payer view). Robert Sauermann described Austria’s approach: ensure everyone gets needed therapy while protecting system sustainability. With many approvals based on immature or single-arm data, uncertainty is real, so Austria leans on managed entry agreements, staged pricing, data collection, and periodic reassessment as real-world evidence accrues. He welcomed direct input from patient groups on what outcomes truly matter and how services work on the ground, while stressing the value of transparent, independent advocacy.

Policy knots to untangle. The room debated external reference pricing useful for budgets but a source of cascading delays if countries “wait for the neighbours,” and a risk of affordability distortions in lower-income settings. The shared path forward: align trials to patient-relevant endpoints, co-design pragmatic data plans, pilot more joint assessments (and, where feasible, joint negotiations), and be honest about everyone’s incentives commercial, budgetary, societal while anchoring decisions in patient benefit.

A call to action for advocates. Lauren closed by urging patient organisations to learn their country’s access pathway, show up in HTA and pricing consultations, bring lived experience and real-world data, and keep the human lens in the room. Real progress will come when patients, payers, policymakers, and companies reduce uncertainty responsibly, share risk fairly, and turn genuine innovation into timely, equitable care.