Wim Goettsch appointed professor of Health Technology Assessment of Pharmaceuticals

Utrecht University has appointed epidemiologist Wim Goettsch as professor of Health Technology Assessment of Pharmaceuticals. As professor, Goettsch will contribute to methods that balance drug innovation and sustainability of our healthcare system. The chair is part of a collaboration between the Dutch National Health Care Institute, Erasmus University Rotterdam and Utrecht University.

We all want the best possible healthcare for all citizens, but not at any price. This raises the question for treatments, such as medicines, on how to balance the added value and additional costs. In other words: are they worth the money?

Epidemiologist Wim Goettsch will contribute to methods that address these questions, as Professor of Health Technology Assessment of Pharmaceuticals. His field of expertise evaluates information about medical, social, economic and ethical issues surrounding the use of medicines, medical devices and other forms of healthcare.

Assuring drug effectiveness

Knowledge gained from this type of research is used by hospitals, for example, to assess if they should offer certain treatments to their patients. Governments and health insurers are also interested in these results, because they want assurance that a health care intervention, such a medicines, works well enough to pay for it. In the Netherlands, many medicines can only be included in the standard health insurance after a positive evaluation by the Dutch Health Care Institute.

Keeping healthcare affordable

"If we want to keep healthcare affordable, we have to make choices," says Goettsch. "Certainly, we aim to make innovative, new medicines available to the patients who need them. However, we simply can't afford to include every single medicine in the basic healthcare package. Some treatments are extremely expensive. Gene therapies, for example, can cost up to two million euros per patient."

If we want to keep healthcare affordable, we have to make choices

As a professor, Goettsch focuses on three areas that he believes are important to enable appropriate care with innovative new medicines. One example is looking into new ways of paying for medicines. "For instance, can we link effectiveness of the medicine to the actual price tag? By that I mean that we don't want to pay the total sum up front in one go, but instead pay in segments over several years. Additionally, this payment depends on how the medicines effectively work in the patient."

This may seem like a simple plan, but it is not, says Goettsch. "It happens very little in practice yet. We may want it, but setting up such payment models is complicated and our healthcare funding systems are not ready for it at the moment."

How well do medicines work in practice?

To make the right choices, Goettsch says it is important to better assess how well a medicines works in practice. Until now, medicine evaluators primarily focus on outcomes of clinical trials, in which the medicine's effect has been studied on patients in a highly controlled environment. But when a medicine is being used in clinical practice, it may turn out to be less effective than expected, or only work for a specific group of patients. In such an example, you might renegotiate the price of the treatment, says Goettsch, or offer the medicine only to those patients in whom it really works.

When medicines turn out to be less effective than expected in clinical practice, you might renegotiate the price of the treatment

A prerequisite is that reviewers can evaluate effectiveness of medicines with greater certainty in real-world settings. This requires new statistical methods and mathematical models, which Goettsch aims to contribute to with his research team. One solution may be to develop advanced methods for combining data from clinical studies with data from clinical practice.

Smaller studies, less data

Another reason to include more data from clinical practice is that many new medicines are intended for relatively small groups of patients, often with rare diseases. The clinical trials designed to demonstrate the efficacy of those medicines are therefore small and provide limited data. That makes it more difficult for reviewers to determine, for example, whether the medicine is truly effective in clinical practice.

Often medicines are very unique. In clinical trials, there are no similar medicines available to compare them to.

"Often these medicines are very unique," Goettsch says. "In clinical trials there are no similar medicines available to compare it to. Assessors then need to find other ways to compare the effectiveness of those medicines to patients who only receive supportive care. This way, they can advise whether or not the medicine is good enough to be included in the basic healthcare package."

Coordinated assessment in Europe

As professor, Goettsch will also focus on how assessments for drug reimbursement can be better aligned within the European Union. The European Medicines Agency (EMA) assesses whether a drug should be allowed to enter the European market. But subsequently, the EU member states themselves decide whether to include that drug in their basic healthcare package. "All EU countries have similar questions regarding how to decide on reimbursements for medicines," says Goettsch. "Our research has demonstrated that the current national approaches can lead to big differences between EU countries regarding recommendations to reimburse medicines."

Searching for common ground

This could perhaps be done more efficiently, for example, by doing some assessments jointly. The European Commission recently passed legislation to support joint clinical assessments that can be used as part of the national reimbursement recommendations. But the question remains which parts of an assessment are suitable for this and what elements should be included. Searching for that common ground is very complex, Goettsch said.

How should you interpret results from medicine effectivity research? Several EU countries maintain different views on how to answer that question.

"Sometimes the assessment seems very simple. For example, if you evaluate a new cancer drug, you could look at whether patients receiving the drug have a longer survival. But often data on survival are unavailable or inadequate. Instead, researchers may look at how well the medicine inhibits tumour growth. But the subsequent question is: Does that outcome predict whether the patient lives longer, or whether the quality of life is better for that patient? Several EU countries maintain different views on how to answer that question."

Advising on medicines in healthcare insurance

In the Netherlands, the government determines what is included in the basic healthcare package. The Dutch National Health Care Institute (ZIN) advises the Dutch Minister of Health on whether to include certain medicines. In addition to his appointment at Utrecht University, Goettsch also works for this institute, as special advisor Health Technology Assessment.

Goettsch's professorship is part of the Academic Research Network (HTA), a partnership between Dutch National Health Care Institute, Erasmus University Rotterdam and Utrecht University.