Conventional examples demonstrate the feasibility of the 15 percent threshold redefined by IQWiG for accepting respondent analyzes in early drug benefit assessments.
To demonstrate the clinical appropriateness of differentiating between two treatment options, in recent years, respondent analyzes have included relevant patient outcomes in manufacturer doses included in early benefit assessments of new drugs.
In such analyzes, it is examined whether the proportion of patients receiving a marked change in the individual outcome differs between the two treatment groups in a study.
This includes information on health-related quality of life or on individual symptoms such as pain or itch, which patients recorded with the help of scales in questionnaires.
But what difference does change make to the individual? That is, to what extent can the patient respond to an intervention, so that, for example, the difference in response groups of two groups can be used as an effective measure for early welfare assessments?
When is a difference relevant?
To answer this question, the methodological debate has long shifted around so-called so-called (MID) differences. This approach is based on the idea that thresholds can be identified for the individual questionnaires that represent the smallest relevant change for patients.
However, the procedural difficulties of this approach have emerged recently. For example, it has been demonstrated that MID is not a fixed value for a questionnaire, but a variable. It depends, for example, on the type and severity of a disease, the direction of change (improvement or decline), or the methods used to diagnose it.
Furthermore, it has become apparent that many scientific studies on MID validation do not meet modern methodological standards or that the methodology used is not defined. enough in the scientific publications.
In this scenario, IQWiG has developed a new approach that makes it possible to determine thresholds that reduce relevant range with sufficient certainty. The aim of this specification was also to create clarity for manufacturers and to make non-regulatory analyzes of respondents based on unambiguous non-attractive respondent definitions.
Concerns were unfounded
From an evaluation of new scientific reviews on the subject, last year, IQWiG, therefore, identified the value of 15% of the range of individual blades as a plausible threshold for change that is relatively small, but certainly certain.
In its Common Approaches 6.0 published in November 2020, the Institute then stated that specifically in future assessments, it would use respondent analyzes from a threshold of at least 15% of scale range. the measuring instrument used.
The tight tightening of the 15% threshold for acceptance of respondent analyzes in IQWiG assessments prompted conversations in the period up to the Standard Methods 6.0.
Among other things, some manufacturers feared that this approach would not apply to all scales and that it would be more difficult to prove the added benefit of a new drug.
At the time of the addenda published today on early benefit evaluations of the drugs secukinumab (for psoriatic arthritis), ivacaftor (for cystic fibrosis) and alpelisib (for breast cancer), Katrin Nink from the IQWiG Drug Assessment Department confirms: ” The concerns were unfounded. We will see that the new threshold is suitable for use in practice. “
In these assessments, the manufacturers submitted the requested data so that the Institute could use the respondents’ analyzes for its assessments.
This applies to several questionnaires on both the burden of disease symptoms and on health-related quality of life in very different symptoms such as cystic fibrosis in children, psoriatic arthritis, or breast cancer.
Based on analyzes with the new response thresholds, we were able to reveal the advantages and disadvantages of treatments, such as the benefit of ivacaftor for the quality of life of children with cystic fibrosis. So the new gender criterion works. “
Katrin Nink, IQWiG’s Department of Drug Assessment
Source:
Institute for Quality and Efficiency in Healthcare