'No Cure, No Pay' Pilot for Expensive Cancer Drugs

Marc van Impe

July 15, 2019

In the Netherlands, a new project is underway: the "no cure, no pay" model, in which certain expensive cancer drugs will be paid for if they turn out to be effective after 16 weeks. Drug companies will not receive payment if these drugs do not work.

The hope is that this model will allow expensive medicines to remain available in this way, and that doctors will be able to prescribe them to patients who have exhausted other therapeutic options, and who may stand to benefit from them. The new project was developed by Dutch oncologists, researchers at the Netherlands Care Institute, and health insurers, and is being supported by Bristol-Myers Squibb.  

There are currently three such experiments in progress in the Netherlands. One pilot project was completed in January. The results are now being evaluated.

The agreements stem from the so-called DRUP studies, conducted at 30 Dutch hospitals, in which patients with rare tumor subtypes who have not responded to standard treatment received a cancer drug that was actually intended for another tumor type. The idea is that there are already drugs on the market that can help with certain rare types of cancer, but which have never been studied for them because the number of patients is far too small for large-scale clinical studies.

For example, there is a sub-study with 30 patients with a very rare MSI-H tumor, which showed that two thirds of patients had a response to the immunotherapy nivolumab (Opdivo, Bristol-Myers Squibb). These patients had several cancer types, including renal cell carcinoma; non-small cell lung cancer; recurrent or refractory classic Hodgkin lymphoma after autologous stem cell transplantation and treatment with brentuximab vedotin; squamous cell carcinoma of the head-neck area, urothelial carcinoma, and colon cancer.

The Dutch researchers are now adding a new phase to the DRUP study: 135 patients with MSI-H tumors will receive nivolumab for 16 weeks, paid for by Bristol-Myers Squibb. For every patient who has tumor shrinkage or no disease progression, there will be an opportunity to claim reimbursement from the basic health insurance.

Model a Possible "Game-Changer"

The new reimbursement model is a game-changer, say observers, and is being closely followed by the European Union (EU). If the model is successful, the EU Commission will likely do everything in its power to ensure that it is adopted throughout the Union.

"This is the type of agreement we are looking for," said Linda van Saase, manager of oncology at the Dutch National Health Care Institute, which monitors what resources are flowing into the basic health insurance.

According to Gerard Schouw, director of the Vereniging Innovatieve Geneesmiddelen (Industry Association for the Innovation of Medicines), agreements of this kind "can develop into a game changer in the debate on the affordability of medicines." It is easier for pharmacists to justify high drug prices if they only apply to the patients who benefit from them, he added.

The model was conceived by oncologist Emile Voest, MD, PhD, medical director of Antoni van Leeuwenhoek Hospital and chairman of the Netherlands Cancer Institute. He calls this a "first building block" to look at the problem of extremely expensive medicines for treatment tailored to the patient in a different way. As he explains, one could ask, "Why are those medicines expensive?" or one could also say, "How sure are we that these medicines can benefit patients?"

Edwin Ket, market access director at Bristol-Myers Squibb Nederland, calls the collaboration "an example for the future." The new model is in line with changing medicine, in which care becomes tailor-made. Central to this is the DNA profile of the patient. By looking at DNA profiles and other data in detail, researchers are finding more and more associations between patients and the reasons why they do or don't respond to certain medicines.

After all, every patient is unique, every tumor is different, every DNA profile has its own peculiarities, said Ket. Pharmacists and research institutes are therefore investing heavily in reading DNA profiles from patients in order to find indications as to which medicines may or may not be of use to them.

"And when more and more care is tailor-made and more medicines are tailor-made, it is not surprising that payment also becomes tailor-made," said Anke Pisters-van Roy, a medical advisor at the health insurance company CZ.

Systematic Tracking Needed

Voest believes that this could be taken a step further. If a drug is so expensive that the government has to negotiate the price with the pharmaceutical company, then a requirement should be that all data of new patients should be recorded and compared very systematically. So he suggests there should be fixed assessments, DNA mapping, and regular scans. Only in this way will it be possible to use a treatment more specifically and effectively.

However, there is also a downside. This model does not address the question of whether the drugs could be cheaper. And it also raises the possibility that drug manufacturers will not use this "no cure, no pay" model in order "to keep the price super high," said Pauline Evers, patient advocate at the NFK , the Dutch federation of cancer organizations. After all, a discount is easier to negotiate if a medicine does not work for a large number of users than if it has been established in advance that every patient for whom payment is made will benefit from the medicine.

This is also the reason why some governments are sometimes not in favor of pay-for-performance agreements. In the case of lumacaftor/ivacaftor (Orkambi, Parexel International), which is used in the treatment of cystic fibrosis (CF), the Dutch government preferred to agree on the price and volume: the more often a drug is dispensed, the higher the discount. The advantage is less risk, because the government knows in advance how much drug will be needed and paid for. It is often more difficult to predict how many patients will benefit from the drug in practice.

And last, in some cases it may be unclear when a drug works. "Many pay-for-performance agreements ultimately run aground due to bureaucracy and the question of what the performance actually is," Ket noted.

However, cancer drugs are relatively easy to make agreements about, experts from the Cancer Fund say: "A tumor can be measured. If it doesn't get smaller, then the drug won't work. You can actually see that right away."

The situation is different for chronic diseases, in which a patient's condition slowly deteriorates. How long is a reasonable time to wait before the drug should be effective? There is also the question of how long the patient should receive the medicine. 

This article was originally published in French and Dutch by MediQuality, a sister site of Medscape.

For more from Medscape Oncology, join us on Twitter and Facebook


Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.
Post as: