Zinnige Zorg room for improvement report - Appropriate use of expensive medicines in cases of metastatic renal cell carcinoma

Within the framework of the Zinnige Zorg Programme, Zorginstituut Nederland systematically assesses the Dutch minimal and mandatory package of health care that all Dutch health care insurers must provide. There are four phases to this systematic assessment: screening, in-depth assessment, implementation and monitoring. In 2015 we published a screening report: ‘Systematic analysis of neoplasms. This Room for Improvement Report is part of the in-depth phase. During the screening phase, one of the topics mentioned by the parties for in-depth assessment was ‘appropriate use of expensive oncolytics’. At the time we asked the parties whether they could cite an example of a recommendation by an authoritative organisation that suspected that expensive oncolytics were not being implemented correctly. The Dutch Urological Association stated that an analysis of the guidelines dating from 2011/2012 revealed that more than half of the patients with metastatic renal cell carcinoma received different advice for primary care treatment from that advised in the guidelines. The question was whether this was a case of inappropriate use. Realising appropriate use is essential, not only to realise greater health gains for cancer patients, but also to guarantee accessibility by avoiding unnecessary costs where possible.

Room for Improvement Report

Despite the fact that the study did not fully answer the question posed, we were able to formulate three possibilities for improvement based on the study outcome. We regard trying to realise the most appropriate use possible, based in part on the improvements suggested here, as a crucial step in the treatment of not only renal cell cancer, but also other types of tumours where similar developments are taking place.

1. Guideline development

We conclude that the guidelines need to be updated, with special attention to criteria – based on patient characteristics and disease characteristics – for starting and ending treatment, or giving no treatment at all. We feel that the treatment guidelines are not keeping pace with developments, and that this could potentially result in care being deployed less appropriately. This Room for Improvement Report can also apply to other types of tumours where the treatment landscape is continually changing. For example, prostate cancer, melanoma and non-small cell lung carcinoma.

2. Registration at source

We are unable to draw any firm conclusions about the appropriate use of pharmaceutical products in a case of renal cell carcinoma. There are no indications for suspecting that registration was inadequate from the perspective of treatment, but the current analysis makes it clear that registration focussed insufficiently on the systematic collection of feedback information. It is important to actually register the patient characteristics and disease characteristics that in practice may be used to determine what treatment will be given so that these data can subsequently be used to draw relevant conclusions. To date, this has not taken place sufficiently. Registries are increasingly used for providing feedback information on appropriate use. In general, we can state that if the objective of registers is to provide insight into the appropriate use of pharmaceutical products, then data must be collected prospectively with registration at source.

3. Use of surrogate outcome measures

In cases of renal cell carcinoma a surrogate outcome measure – progression-free survival – is used to measure the effectiveness of some of the pharmaceutical products used in second line treatment and subsequent care. We feel it is necessary to discuss the clinical relevance of pharmaceutical products whose effectiveness is mainly determined based on a surrogate outcome measure. Carrying out concrete improvement activities in relation to this demands an international investigation, which goes beyond the scope of this report. The Zorginstituut does want to discuss the matter within its Dutch context with the parties in the field.

Zinnige Zorg (Appropriate Care)

Zorginstituut Nederland’s motto is "Taking care of good health care: no more and no less than necessary". Every citizen must be able to count on receiving good health care. No more and no less than is necessary, while also avoiding unnecessary costs.
As a public organisation, the Zorginstituut assesses health care systematically. We assess whether diagnostics and (therapeutic) interventions are being deployed in a patient-oriented, effective and cost-effective manner. We do these analysis within the framework of the 'Zinnige Zorg' (Appropriate Care) programme of Zorginstituut Nederland.

This report is a summary of recommendations by Zorginstituut Nederland. The original text is in Dutch.