One year after Pia: what’s the story with expensive medicine?


Baby Pia, there can’t be anyone in Flanders who doesn’t know her. Suffering from a rare muscular disease, she was in the eye of the storm during the fall of 2019. A medicine that could save her life came with a dizzying price tag of almost 2 million euro. The debate surrounding too expensive medicines caught fire. But did it change anything?

It did for Pia. Her parents started a campaign to collect money that would allow them to pay for the Zolgensma treatment, an innovative gene therapy. Since then, she has greatly improved. On top of that, the medicine has been officially approved for the European market in May and RIZIV refunds the treatment.

The discussion doesn’t end here. Even though the treatment is available for everybody, the cost of the actual medicine remains very high. There are many very expensive medicines. “Pia’s medicine is a precedent”, professor Lieven Annemans, a health economist at the UGent, suggests. “It is about time we had a general policy, as we are expecting dozens of similar cases.”

The long journey of medicine

When the dust of the initial outrage had settled, the question of why a particular medicine was so expensive lingered. “There are various reasons”, Professor Catherine Van Der Straten posits. The development process for starters. “It just takes so long to produce a medicine because there are many steps that must be undertaken before it can be released to the market. We must be absolutely certain that the product is safe.” Catherine is the president of the Board of Directors of Sciensano and the head of the department at the Health, Innovation and Research Institute of UZ Gent. Her department focuses on research and innovation. They support high-quality clinical research and help develop innovative projects, all with affordable care in mind.

She is supported by Dr Dominic De Groote, business development manager at IOF (Industrieel OnderzoeksFonds) of UGent. He manages research groups and platforms, like CRIG (Cancer Research Institute Ghent), for internal development trajectories for new medicine and collaborations with companies. As part of the IOF, he screens research results that could interest companies in developing medicine. “During discussions at the time, I noticed that people often don’t realize how complex and time-consuming it is to develop medicine. The first step in that long journey is generally university research. This is followed by a very committed collaboration with companies, should they be interested to begin with. Together we map out the feasibility and potential of the medicine. If these results are positive, we — as a university — grant a commercial license to the company. This way they can further develop the product. It then takes an average of 10 to 12 years before the medicine is effectively brought to market.


Limit profit

Catherine also appreciates that there is a commercial side: “Many people think that any money primarily goes to the pharmaceutical industry, especially after the Pia story. That is partially the case, as these companies want to make a profit. They do marketing, which costs money. But that is on top of the expensive development and production costs. Fortunately, profits aren’t as high as they used to be.”

“It is the government’s job to supervise these profit margins”, Lieven adds. “There should be a clear limit as to what we want to pay for medicine, one defined by the government. A maximum profit amount for health, or else a pharmaceutical company could establish any price point they wanted to. They are thinking about this already. The discussion around Pia has accelerated this — but we are not there yet. There is no unambiguous policy. We are looking at it on a case by case basis.”

But how do you define that limit? Lieven: “That is a difficult balancing exercise. You can’t just halve the production cost. Something that is more efficient and adds many qualitative years of life, like Pia’s medicine, can cost more. This way, you stimulate the industry to continue innovating. But it can’t be at whatever cost. Healthcare is a fundamental right and the payment capacity of governments is not unlimited.”

Belgium is too small

Defining pricing naturally means negotiating with pharmaceutical companies. And that’s where there is a problem. “Belgium as a market is too small for negotiations. Governments in Europe must group together. The first steps had already happened when the discussion around baby Pia erupted, but it has sped up the whole thing”, says Dominic. Catherine feels that it can be kicked into a higher gear still more: “why do medicine prices still differ per country? The government can intervene so that a specific treatment costs the same everywhere. Or at least within the EU.”

Working together to reduce cost

Baby Pia’s story has not only resulted in awareness, but also led to steps being taken. Catherine sees an evolution in the right direction coming from the pharmaceutical industry. “They are making more efforts to make medicine accessible, by collaborating with the academic world, for instance. We must create a system where there are more partnerships, so that the academic world can work closely with companies that are producing on a larger scale. Only by doing that can we reduce the development and production cost.”

The UZ Gent is leading by example: it will produce CAR-T, a hugely expensive but vital immune therapy, in-house. Catherine: “We recently started collaborating on a project to have CAR-T therapy happen here, perhaps ultimately in collaboration with the pharmaceutical industry. This is a cell therapy whereby living cells are used to attack cancer cells. Don’t be misled … the therapy remains expensive. We need a high-tech lab, specialized staff, and so on. But we normally send these living cells all the way to the United States for treatment, only to then bring them back here. This project will ensure we save important time and also save on transport costs too. That will make a huge difference.”

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