4. Reading: Reflection paper
Reading: Reflection paper
Reflection paper on the Regulatory Guidance
For The Use Of Health -Related Quality Of Life (HRQoL) Measures
In The Evaluation Of Medicinal Products
Note some concerns expressed by the regulator in assessing HRQoL. First, there is a concern about double-counting. We have seen in previous topics that the domains and items used to measure any PRO require validation and should not overlap. However, the regulator is more concerned here with scenarios where a new therapy both improves HRQoL and improves an outcome that is part of the HRQoL assessment (like reduction of pain).
They also suggest that companies who would like to claim improvements in HRQoL should show improvement across ‘all or most of’ several key domains.
The last part of the paper devotes a considerable amount of attention to the proper use of these instruments in clinical trials. The authors advise those developing new medicines to have HRQoL instruments validated prior to conducting experiments. This avoids a situation of questionable data collection. We can imagine a situation where several different HRQoL instruments are developed and only one records an observable response to therapy. Is the response a property of the medicine or does it have something to do with the instrument? Evaluators are left with such questions if the validation of the instrument is not conducted prior to using it.
The paper also points out several other factors that may make interpretation of the findings from HRQoL measurement difficult. One is the situation where patients know they are receiving therapy (an open label trial). Patients who know they are receiving therapy have been shown to be more positive when providing subjective information. It can be difficult for the evaluators to distinguish the effect of the medicine versus the satisfaction that the patient has from having access to something new (and believing it might be working).
A last point made is that although HRQoL is important, short-term measures do not really provide us with insights into a person’s overall well-being. Rather they tell us how patients are doing from day to day without providing information on how they might be over a longer and more meaningful period of time.