In order to give these patients access to such a medicine, the parties mentioned have devised a finance model tailored to the patient, whereby the manufacturer of the medicine and the health insurer share the financial risks: the manufacturer pays for the medicine in the study phase, and the health insurer then reimburses the patients for who it appears to work: a pay-for-performance model. A pilot is going to run in a clinical study involving more than 30 Dutch hospitals: the DRUP study, coordinated by the Center for Personalized Cancer Treatment.
Reason: existing medicines for rare tumour profiles
It is becoming increasingly clear that every tumour is biologically unique and that treatment must therefore increasingly be customized to the individual patient. For example, sometimes a tumour has a DNA profile that is very rare for most tumour types. There is sometimes medicine that may respond to this disorder, but that is not registered for all those tumour types.
A special clinical study was therefore started in the Netherlands in 2016: the DRUP study. DRUP stands for "Drug Rediscovery Protocol". In this study, small groups of patients, all with a rare molecular tumour profile, are given medicines that are already registered for another type of cancer but not theirs. There are no other treatment options for these patients.
The purpose of the DRUP study is to investigate whether the medicines are effective and safe for these patients too. This cannot be investigated in conventional large-scale medicine studies; the patient numbers are far too small for that.
Access to medicines for individual patients
Emile Voest, Medical Director of the Netherlands Cancer Institute and one of the research leaders of the DRUP study: "The question arose at the start of the DRUP study already: Suppose the medicine works for a patient, how do we ensure that the patient then also has access to it? After all, it is not registered for his or her indication."
The DRUP study has now shown that the immunotherapy drug nivolumab, which is used, among other things, for the treatment of melanoma, is also effective in 67% of 30 patients with cancer types other than those for which the medicine is indicated, but which, however, share a specific DNA defect. This is a promising result. But the study cohort is closed and the so-called off label use of nivolumab is not reimbursed outside the context of the study. This means that new patients with the same DNA abnormality would not have access to this treatment. What now?
Parties get together
The parties involved - oncologists, the manufacturer, the Healthcare Institute and health insurers - got together and devised a way to give these patients access to the medicine.
Manufacturer and health insurer share risk
An additional phase is being added to the DRUP study in which new patients with a similar tumour profile from Dutch hospitals can be included. The effectiveness and safety of the medicine will be assessed per patient. In this 16-week phase, the manufacturer of the product - in this case, nivolumab from the manufacturer Bristol-Myers Squibb - guarantees the medication costs.
If after 16 weeks the medicine appears to be effective and safe for these individual patients, the health insurer will reimburse their further treatment. The manufacturer and health insurance company thus jointly bear the financial risk.
Building up evidence
Large-scale comparative medicine studies, usually a condition for the approval of a medicine for a new indication, are not possible for these rare tumours. Because all information, such as treatment outcomes and a DNA profile, is systematically collected from the entire treatment in the DRUP study, researchers and hospitals nevertheless build up evidence. They systematically map the effectiveness and safety of this customized therapy. The care of the individual patient is thus substantiated and improved, also through the correct positioning in guidelines for treatment and reimbursement.
Pauline Evers, advocate for Medicines at the Dutch Federation of Cancer Patients, is very happy with this widely supported initiative:
"For cancer patients with a rare mutation in the DNA, a new application of an existing medicine in a research setting becomes available. The unique collaboration between researchers, the Healthcare Institute, health insurers, and the pharmaceutical industry ensures that this treatment becomes accessible in a financially responsible manner. The costs for treatment are shared. The future must show whether in specific cases this alternative development process can lead to a qualitatively and financially better-streamlined introduction of new applications of medicines."
More information about The DRUP Study