Stelvio for life

If they benefit from it, (cancer)patients are reimbursed for ‘rediscovered’ medicines.

Sometimes cancer drugs against more types of cancer than they were developed and registered for help. However, they are only reimbursed if they have been approved for the treatment of a certain type of cancer. If this is not the case, patients will not have access to a drug that would be of use to them. Doctors, pharmacists, health insurance companies and the Care Institute have now come up with a solution to this problem.

In a sixteen-week study, 130 patients with all kinds of cancer were included. The similarity between them is that their tumours all have the same genetic trait, namely an MSI profile. The research builds on a successful pilot.

The patients are treated with the immunotherapy nivolumab. This drug is intended to boost the immune system of the patient. The pharmacists pay for the expensive medicine in this research phase. After that, the health insurers will pay the costs of the treatment for all patients who, after sixteen weeks, show that they benefit from it. Regardless of whether the drug used is officially intended for the cancer they have.

Pilot study
In a small pilot study among 24 patients, two out of three cancer patients with such a rare tumour proved to respond well to the drug. It did not matter whether it was an MSI tumour in the large or small intestine or, for example, an MSI tumour in the endometrium.

MSI profiles occur in all types of tumours, but most frequently in the various types of colon cancer. Approximately 5 percent of all bowel cancer patients have an MSI tumour. For all cancer patients the share is 2 percent.

The fine research results were harshly confronted by the scientists with the question: what to do now? Because nivolumab is not registered for the treatment of colon cancer or mucosal membrane cancer, it is not reimbursed for the treatment of these diseases.

“That is why we have sat down with all those involved”, says Emile Voest, chairman of the board of the Antoni van Leeuwenhoek hospital in Amsterdam. “To see how we can arrange for patients who benefit from a medicine to have access to such a medicine. In the pilot study, which will continue for a few years, producer Bristol-Myers Squibb will pay for the nivolumab.

Small patient groups
Voest says that we will increasingly have to deal with very small groups of patients for whom a tailor-made treatment has to be sought.

“These patient groups are too small to properly examine the efficacy of a drug, but we do not want to deprive people of a working drug either. With this new approach, the pharmacist pays for the medicine in the first four months. After that period, we check whether it works for the patient who receives it. If that is the case, the health insurance company will pay for it.”

In the meantime, patients continue to participate in the research. “In this way, we can collect more and more data. You can use this data to determine how to proceed. As well as extensive DNA analyses to predict who the drug will work for. We are now going to expand this pilot study with nivolumab to include a follow-up study with 130 patients in 30 hospitals. If it gives the same results, then you can be sure with 90 percent who the drug works for.”

A good response
Soraya den Boogert has bowel cancer. She signed up just in time for the pilot study with nivolumab. After her, no new patients were included in the study. She responded well to the treatment. “After three months, my tumours were stable,” she says.

“After six months they had shrunk. Then it stopped for a while. It was a bit of a step-by-step process. In the meantime they are so much smaller that two tumours can no longer be seen on the scan. It only picks up half a millimetre-long tumours. And the third, the largest, has shrunk by half.”

Den Boogert will participate in the study for a total of five years. “If my tumours remain stable, I would be able to stop using the nivolumab at the end of the year. But I don’t have to. And I don’t know if I will. I’m a little afraid of that. Fortunately, I don’t have to decide yet.”

Larger studies
The pilot study with nivolumab is part of a much larger study with more than 400 participating patients. In this DRUP-study (Drug Rediscovery Protocol) 26 existing drugs are tested for their effect against other cancers than those for which they are actually intended.

Watch the (Dutch) interview with Emile Voest -Medical Director of the Antoni van Leeuwenhoek-Dutch Cancer Institute.