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Do sufferers worth enhancements in progression-free survival? – Healthcare Economist


Development-free survival (PFS) is a surrogate endpoint is outlined as follows (by ChatGPT):

In essence, PFS refers back to the size of time throughout a medical research or therapy routine the place a affected person’s illness doesn’t worsen or progress. For example, in most cancers analysis, it measures the time from the beginning of therapy till the most cancers exhibits indicators of progress, unfold, or relapse.
Understanding PFS is important as a result of it helps clinicians and researchers assess the effectiveness of a therapy in controlling the illness. An extended PFS means that the therapy is efficiently slowing down the development of the sickness. This metric is especially vital in medical trials when evaluating totally different therapy approaches or medication.

A key query is, do sufferers care about PFS? The reply could also be sure, as a result of higher PFS is most frequently (however not at all times) correlated with longer survival. Nevertheless, do sufferers worth PFS impartial of OS? Maybe so if their high quality of life is best (i.e., they’ve fewer signs) through the pre-progression part. Or, maybe they’ve decrease anxiousness in the event that they know their most cancers has not progressed.

What does the literature say?

A paper by Raphael et al. (2019) carried out a scientific literature assessment of research that consider whether or not sufferers with superior most cancers perceive and worth PFS. General, 17 research met their inclusion standards. Of those:

Ten research particularly introduced sufferers with the time period progression-free survival as an attribute alternative. Within the phrases used to outline the attribute of PFS, 6 research used the time period survival. 5 research clarified that PFS might not translate into higher general survival, and 5 research defined that enhancements in PFS might not mirror how nicely the affected person might really feel. No research clarified {that a} PFS occasion may symbolize both development or loss of life, and no research outlined for the affected person what constituted development. The research assessed herein underrepresented ethnic and racial minorities (imply share of white sufferers, 88%; vary, 77%-96%). Values and preferences might fluctuate throughout cultural backgrounds on condition that totally different relative preferences have been assigned to value and efficacy outcomes in North American vs Asian research, though only some research have been evaluated.

As you’ll be able to see from the desk beneath, there may be important heterogeneity when it comes to how PFS was introduced to the most cancers affected person respondents.

httpsjamanetworkcomjournalsjamaoncologyarticle abstract2751879

In among the research, PFS was an important attribute. In others, high quality of life components have been extra vital. General, nevertheless, it’s clear that extra analysis is required to completely perceive how sufferers understand the worth of PFS.

Do patients value improvements in progression-free survival?

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