Cell and gene therapy development does not end at regulatory approval. If the evidence you generate does not answer the questions that payers and HTA bodies will later ask, therapies may never reach patients. In this conversation, Thomas of hollandbio reflects on the policy and administrative realities that shape access, manufacturing, and reimbursement across the Dutch and European ecosystem.
A Sector View of Development Challenges
Hollandbio’s role is not to direct the strategy of any one company, but to understand where barriers emerge across the ecosystem. With hundreds of members, Thomas explains that their focus stays on patterns that affect the whole sector rather than individual commercial priorities.
“We always try to keep a sector perspective.”
That means paying attention to the hurdles faced first by the “front runners,” the companies pushing into new regulatory or manufacturing territory. If the environment works for them, it becomes easier for everyone who follows.
For early-stage companies, financing is often the dominant challenge. But Broekhoff points out that policy and regulatory readiness matter from the beginning as well, especially for cell and gene therapies, which are not only regulated as medicines, but are also classified as GMO, requiring environmental permits to make the step to clinical translation.
Policy questions can arise at multiple points along the development chain, from preclinical work to clinical studies and eventually market access. HollandBio often hears from member companies when these questions appear, helping them navigate the broader regulatory environment or pointing them toward the right expertise. Those interactions also provide insight into where systemic challenges exist, allowing the organisation to advocate for improvements at the policy level.
About Thomas Broekhoff
Thomas Broekhoff is Programme Manager Medicines at hollandbio, with a background in biomedical sciences and epidemiology, specialising in HTA and health economics. For over five years, he has supported biotech policy across the development pathway, from preclinical research to market access.
“Take reimbursement and access into account in the early phase of your development process.”
Regulation, Permits, and Manufacturing Reality
For cell and gene therapy developers, production is not only a technical scaling exercise. In the Netherlands, commercial-scale manufacturing brings an additional layer of environmental regulation.
Thomas highlights a recent change that reduces administrative burden for companies working with GMOs on industrial scale. Previously, companies were automatically assigned to the strictest risk regime and had to prove they qualified for a lower-risk classification with changes of protocol. Now, it has become easier to adjust an existing permit rather than submit an entirely new application.
This kind of policy shift is not abstract. If you are building manufacturing capacity, the difference between a full new application and an adjusted registration can translate into months of delay, added cost, and uncertainty.
The broader point is that regulatory systems shape development timelines as much as scientific decisions do. For therapies already complex in production and oversight, reducing unnecessary administrative friction can be one of the most practical forms of support.
The Evidence Gap Between EMA and HTA
One of the most difficult structural issues for European developers is that marketing authorization and reimbursement assessment operate with different questions.
EMA evaluates efficacy and safety: does the therapy work, and is it acceptably safe? HTA bodies, by contrast, ask how it compares to existing standards of care within a specific national context.
“The biggest question and the challenge here is that the EMA looks at efficacy… HTA bodies, they look at how does this work in comparison to the current care we have,”.
If you are building a trial strategy, this creates a moving target. Standards of care differ between countries, and smaller companies may not have the resources to generate comparative evidence across multiple jurisdictions.
Thomas’ point is not that developers can solve this alone, but that anticipating these questions early can prevent painful surprises later, such as needing additional studies after approval just to secure reimbursement.
The Netherlands as a Hub, and the Translation Gap
Thomas describes the Netherlands as having a strong life sciences infrastructure, with closely connected university medical centers and a high-quality academic base. He also notes the presence of multiple therapy production sites, reflecting the country’s growing ecosystem.
At the same time, he sees tension in government posture. On one side, ministries promote biotechnology as a national priority. On the other hand, reimbursement decisions remain stringent, with hard negotiations on price and endpoints.
The result, he suggests, is a system pressing forward and holding back simultaneously. The Netherlands may be strong in research, but less effective in translating that research into therapies that patients can receive.
About Hollandbio
Hollandbio is the industry association representing the biotech sector in the Netherlands. Within its personalized health programme, the organization works on the policy environment around medicines, including how new therapies can move through development and reach patients efficiently.
Hollandbio
2014
The Hague, Netherlands
Dutch biotech industry association representing innovators
Why Thomas’ Moderation Matters
Thomas brings a policy-grounded view of cell and gene therapy development that many scientific teams only confront late in the process. His work highlights that innovation does not move through laboratories alone, but through regulatory classifications, permit systems, reimbursement frameworks, and national decision-making.
For developers, the takeaway is not to become policy experts, but to recognise that access is built alongside evidence. The questions asked early, in trial design, manufacturing planning, and regulatory engagement, shape whether therapies can move beyond approval into real clinical practice.
