Intended for healthcare professionals

Letters FDA on PROMs

Two important points

BMJ 2010; 341 doi: (Published 05 October 2010) Cite this as: BMJ 2010;341:c5454
  1. D Rowen, research fellow ,
  2. J Carlton, research associate,
  3. J E Brazier, professor of health economics,
  4. B Mulhern, research associate,
  5. S Palfreyman, research nurse/honorary lecturer,
  6. K Stevens, research fellow,
  7. A Tsuchiya, reader in economics and health economics
  1. 1Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Sheffield S1 4DA, UK
  1. d.rowen{at}

The new Food and Drug Administration (FDA) guidance on patient reported outcome measures (PROMs)1 2 will affect the use of PROMs in clinical trials and thus the evidence available to licensing and reimbursement authorities worldwide.

We have two comments. Firstly, the guidance conflicts with recommendations from other bodies. For example, generic measures, as favoured by the National Institute for Health and Clinical Excellence (NICE), will rarely be appropriate for submissions to the FDA.3

Secondly, the FDA guidance recommends scoring each PROM item and change in response choice equally. This assumes that all items, and the difference between all response choices, are equally important. This could result in misleading or difficult to interpret results. Furthermore, changes in scores may not reflect changes in quality of life that patients themselves value.4

In contrast, economic evaluation scores health status using preferences, whereby items and response choices are weighted according to the impact respondents believe they will have on their quality of life. Such a system would allow patients to indicate what matters to them, not what happens to them. This is more consistent with the earlier stages of PROM development recommended by the FDA that emphasises patient involvement and rigorous techniques in the development of PROMs.


Cite this as: BMJ 2010;341:c5454


  • Competing interests: None declared.