The Regional Health Authorities in Norway has recently decided to reimburse patients who are included in the first expansion cohort in IMPRESS-Norway according to the pay-for-performance model described below. IMPRESS-Norway is a precision medicine trial that tests approved drugs on new indications based on genetic changes in the patient’s tumor. The study started in April last year, and so far more than 50 patients have been offered treatment (identified in the National Molecular Tumor Board among 250 patients who have undergone extended molecular profiling).
Many new drugs are targeting specific characteristics of the cancer, such as a mutation or more complex molecular changes (precision medicine). Through extended molecular diagnostics, such characteristics can be identified in cancers where the drug is not yet approved, but still expected to have good effect. Through IMPRESS-Norway, several such drugs are available to patients in Norway (currently IMPRESS-Norway is supported by Roche, Novartis, Eli Lilly & Company and most recently also Incyte).
In the first phase, up to twenty-four patients can be included in a treatment cohort defined by specific biomarker (molecular changes), drug and tumor diagnosis. If five or more patients of these first twenty-four have clinical benefit from treatment (not progression) after sixteen weeks of treatment, then the effect of the treatment needs to be further investigated in a larger group of patients, – a so-called expansion cohort. A proposed model for expansion cohorts is a phase 2 study in which the pharmaceutical companies give the drug to patients for the first sixteen weeks, and then the public takes over the cost of the drug for the patients who have clinical benefit (pay for performance). It is this model that the Regional Health Authorities have
approved for Norway.
IMPRESS-Norway recently agreed to share data with similar precision medicine studies in the Nordic countries and the Netherlands (DRUP) in order to more quickly collect efficacy data on small patient groups with specific mutations. In the wake of this, IMPRESS-Norway has now been invited by the Dutch DRUP study to participate in an expansion cohort for the treatment of patients with inactivation of both alleles of the genes encoding BRCA1 and / or BRCA2 (bi-allelic) with PARP inhibitors (olaparib). This expansion cohort will open based on results from twenty-four patients who have been treated in the DRUP study. The Regional Health Authorities has granted a group exemption for patients who will be included in this expansion cohort in IMPRESS-Norway if it is initiated in Norway. This means that the public sector takes over the cost of the drug for those patients who have clinical benefit after sixteen weeks of treatment in this expansion cohort.
The patient groups in precision medicine studies are very small and in Norway we estimate to be able to include approx. 5 patients per year in this specific cohort (estimated based on how often these changes are observed). Therefore, the collaboration with the other precision medicine studies is very important in order to gain knowledge about the effect of the treatment on the patient group.
This is an example of early introduction of precision medicine in the health service while collecting additional effect data for cost / benefit calculations. It has also been decided that the solution with group exemptions and a distribution of study costs between the pharmaceutical companies and the public sector will also be relevant for the continuation of other cohorts in IMPRESS if findings from the first twenty-four patients indicate the effect of the treatment.
The opening of the first expansion cohort in IMPRESS is an example of how important public-public-private partnerships are for precision cancer medicine. The topic of expansion cohorts has been discussed in the established partnership for precision medicine called CONNECT, where representatives from the hospitals, the industrial partners and the authorities participate. Through this collaboration all stakeholders collaborate in order to test new models for implementation of precision cancer medicine.