As trilogue negotiations begin on the EU’s pharmaceutical package, there’s a renewed focus on access, innovation, and equity. But if there’s one thing I’ve learned from working at the intersection of policy, science, and lived experience, especially during my time running a think tank from 2010 to 2014 that brought together patients, researchers, and industry on equal footing, it’s this: the pace of change is rarely set by the most ambitious ideas. It’s set by the slowest stakeholder in the room.
And when it comes to conditions like sickle cell disease (SCD) and thalassemia, that pace has been far too slow.
A Legislative Moment: But Will It Deliver?
The EU’s pharmaceutical reform is the most significant overhaul in over two decades. It’s meant to modernise how we incentivise drug development, improve access across Member States, and make the system more responsive to unmet needs. But as trilogues get underway, the question is whether this package will actually shift the dial for the communities that have been waiting the longest.
SCD and thalassemia are among the most common rare genetic diseases in Europe. They affect tens of thousands of people; many from African, Mediterranean, Middle Eastern, and South Asian backgrounds. And yet, these conditions remain under-prioritised in both policy and practice.
“The pharmaceutical reform is Europe’s chance to shift the dial for communities that have been waiting far too long for action.”
Looking Back: A Fragmented Landscape
When I was leading a multi-stakeholder think tank in the early 2010s, rare diseases were only just beginning to gain traction in EU health policy. The 2009 Council Recommendation on rare diseases had laid some groundwork, but implementation across Member States was uneven. One of our pillars was rare diseases, and newborn screening was a topic we worked on extensively.
What we saw then was a patchwork: some countries had robust screening programmes, others had none. SCD and thalassemia were often left out of national panels, not because the science wasn’t there, but because the political will wasn’t. And that absence of urgency often came down to who the diseases affected.
“Sickle cell disease and thalassemia remain under-prioritised, not because the science isn’t there, but because the political will isn’t.”
Not Just a Black Disease: But Still Marginalised
“When diseases disproportionately affect racialised and migrant communities, their deprioritisation isn’t incidental. It’s systemic.”
Sickle cell is often framed as a “Black disease,” and while it disproportionately affects people of African descent, that framing can obscure the broader picture. Thalassemia, for example, is more prevalent in southern Europe and parts of Asia. What both conditions share is a history of being overlooked, underfunded, and misunderstood.
Even today, many patients still face delayed diagnoses, inadequate care pathways, and limited access to curative therapies. The fact that these diseases disproportionately affect racialised and migrant communities is not incidental. It’s part of why they’ve been deprioritised.
“Even today, many patients with sickle cell and thalassemia face delayed diagnoses, inadequate care pathways, and limited access to curative therapies.”
Why the Slowest Stakeholder Matters
“Discomfort with addressing race and equity in health has slowed progress, but it’s a conversation we can no longer afford to avoid.”
The pharmaceutical package could help. The Council’s latest position includes stronger access obligations and tighter rules on market exclusivity. But unless the slowest stakeholders, whether they’re regulators, national governments, or parts of the pharmaceutical industry, are brought along, the reforms won’t reach the people who need them most.
And let’s be honest: part of what slows things down is discomfort. Discomfort with talking about race and ethnicity in health. Discomfort with acknowledging that some communities have been systematically underserved. Discomfort with the idea that equity might require more than just equal treatment; it might require targeted investment and structural change.
What Patients Can (and Are) Doing
Patients aren’t waiting quietly. Groups like EURORDIS and the European Patients’ Forum are pushing hard to keep patient voices in the room; especially in EMA committees where decisions are made. Advocacy networks for SCD and thalassemia are growing stronger, more coordinated, and more visible.
But they need support. They need policymakers to listen not just during consultations, but during negotiations. They need incentives that reward companies for developing treatments for diseases that don’t always have the biggest markets. And they need systems, like universal newborn screening, that don’t leave their communities behind.
What Needs to Happen Now
As trilogues move forward, here’s what I hope negotiators keep in mind:
- Equity isn’t automatic. It has to be built into the system, through incentives, obligations, and accountability.
- Screening saves lives. Universal newborn screening for SCD and thalassemia should be a baseline, not a bonus.
- Patient voices matter. Not just as testimonials, but as experts in what works and what doesn’t.
- Speed matters too. Because every delay has a cost; and it’s not evenly distributed.
Final Thoughts
This is a moment of opportunity. The EU’s pharmaceutical package has the potential to reshape rare disease policy for the better. But it will only succeed if we confront the discomfort, engage the slowest stakeholders, and ensure that equity is at the heart of every decision.
We must remember that every delay has real-world consequences for patients and their families. By prioritising inclusivity, accountability, and speed, we can create a system that truly serves all rare disease communities.
Let’s not let the slowest stakeholder set the pace. Let’s move forward together, with urgency and purpose.