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Rapid Responses: Rapid Responses published:
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Time and payment issue excluded
- Jens M Lauritsen (13 January 2009)
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Selecting appropriate patient reported outcomes measures
- Jose M Valderas, Jordi Alonso (16 January 2009)
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The role of patient reported outcomes in the regulatory process needs to be better defined
- Promote group Patient-Reported Outcomes MOving Toward Evidence (4 February 2009)
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Jens M Lauritsen, MD, consultant Denmark, Odense
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The editoral points to a highly important issue of standardised usage of patient reported outcome. The editorial suggests SF36 as a choice, but an important issue is lacking - the demand for payments to actually use the scaling algoritms. Another measure mentioned in the referenced litterature from the editorial is EuroQol EQ-5d, which from last year has the principle of payment to the organisation if a given trial has more than 5000 participants. If the CONSORT principles were to be extended with a list of suggested "patient reported outcome measures" it would in my opinion be highly appropriate to ensure, that the use - at least for publicly financed non-commercial research - could take place without payment of neither a registration fee nor a pay per patient. Or alternatively at least that a review was made with payment schemes for the various measures. Obviously e.g. the limit of 5000 for EuroQol EQ-5d poses no problems to most studies in contrast to demands for a registration or per patient fee. In such a review also the time to complete the form or control contents should be included. Competing interests: Currently in favor of using EuroQol EQ-5d in clinical studies. |
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Jose M Valderas, Clinical Lecturer in Primary Care University of Manchester. Oxford Road. Williamson Building. 5th floor, Suite 5 M13 9PL, Jordi Alonso
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A recent editorial (1) has summarized current challenges in the use of patient reported outcomes. Some issues mentioned in the editorial deserve some comment.
First, the readers of the journal may be interested in knowing that several of the research lines suggested in the editorial are already producing fruitful results. The editorial identifies as a priority the comparison of available instruments. A tool for the standardized assessment of PRO measures has been developed (EMPRO) based on the solid criteria as elicited by the Scientific Advisory Committee of the Medical Outcomes Trust (2). It has been used for comparing well known instrument (including SF-36 Health Survey, and EuroQol-5D) (3), and it is currently being used in a European wide initiative that aims to assess and compare available instruments for the measurement of PRO in heart failure. The editorial also suggests that funding bodies should ask for documentation supporting the choice of patient reported outcomes. Similar guidance has been already proposed in the United States by the FDA in relation to the regulation of claims made about a drug or medical product in its labeling (4). In particular, the guidance requests further clarification about methods for determining the responsiveness and for interpreting clinically meaningful differences (e.g., minimal important difference), as also supported by the COSMIN (5) and EMPRO initiatives (3). Finally, the advocacy of the SF-36 as a measure of choice without further specification does not take into consideration that it cannot meet the needs of every trial without further consideration of the population being assessed or the nature of the evaluations being undertaken. Valid alternatives would include shorter measures (SF-12), measures including graphical support for populations with low literacy levels (COOP charts) or, most importantly preference-based measures, such as the SF-6D or EuroQol-5D that would allow for cost-utility analyses while also summarizing measurements in a single numerical value. 1. Garratt A. Patient reported outcome measures in trials. BMJ 2009;338:a2597 2. Scientific Advisory Committee of the Medical Outcomes Trust. Assessing health status and quality-of life instruments: attributes and review criteria. Qual Life Res 2002;11:193–205 3. Valderas JM, FerrerM, Mendivil J, Garin O, Rajmil L, Herdman M, et al. Development of EMPRO: A tool for the standardized assessment of patient reported outcome measures. Value in Health, 2008;11(4):700–708. 4. Revicki DA; Regulatory Issues and Patient-Reported Outcomes Task Force for the International Society for Quality of Life Research. FDA draft guidance and health-outcomes research. Lancet. 2007 Feb 17;369(9561):540-2. 5. Mokkink LB, Terwee CB, Knol DL, Stratford PW, Alonso J, Patrick DL, et al. Protocol of the COSMIN study: consensus-based standards for the selection of health measurement instruments. BMC Med Res Methodol 2006;6:2 Competing interests: None declared |
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Promote group Patient-Reported Outcomes MOving Toward Evidence, coordinate by Istituto Superiore di Sanità Istituto Superiore di Sanità, Viale Regina elena 299, 00161, Roma
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PROmote* group, Rome, Italy
We read the editorial with great interest and we strongly support its conclusions on the necessity to standardise the patients reported outcomes (PROs) measures and stimulate their appropriate use. The growing amount of applications in clinical research of these outcomes has stimulated the regulatory agencies, in the United States and Europe, to take into consideration PROs in the regulatory process and to produce documents where this topic is addressed. In 2004 the European Medicines Agency’s (EMEA) Committee for Medical
Products in Human Use published a “Reflection Paper on the Regulatory
Guidance for the Use of Health-Related Quality of Life Measures in the
Evaluation of Medicinal Products” 1. In 2006 a draft guidance for
industry, the “Patient-Reported Outcome Measures: Use in Medical Product
Development to Support Claims”, was published by the Food and Drug
Administration (FDA)2. The two documents have some agreement points and
several different aspects.
In 2005, a review analysing the use of PROs measures for the approval of new pharmaceutical products was published3 by the EMEA. According to the authors, the regulatory decision-makers should develop and update harmonized procedures to adequately use PROs information in the regulatory decision processes. They concluded that the reflection paper released by EMEA1 on HRQoL research is a promising step towards achieving this aim. As a multidisciplinary group of health care researchers and physicians (PROmote group), coordinated by the Italian National Institute of Health (Istituto Superiore di Sanità), it is our opinion that the EMEA reflection paper is based on preliminary assumptions but needs a urgent update. We strongly encourage both the International and National regulatory authorities to align their documents with the current thinking that all PROs, including HRQoL, should have a significant role in the evaluation of efficacy and safety of medicines. ___________________________________________________ References 1. European Medicine Agency. Reflection paper on the regulatory guidance for the use of health-related quality of life (HRQL) measures in the evaluation of medicinal products; EMEA/CHMP/EWP139391/2004. London, 2004 www.emea.eu.int 2 U.S. Department of Health and Human Service Food and Drug Administration. Guidance for Industry – Patients-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims; February 2006. www.fda.gov/cder/guidance/5460dft.pdf 3. Szende A, Leidy NK, Revicki D. Health-Related Quality of Life and Other Patient-Reported Outcomes in the European Centralized Drug Regulatory Process: A Review of Guidance Documents and Performed Authorizations of Medicinal Products 1995 to 2003. Value in Health, 2005; 8(5): 534-548 __________________________________________ PROmote group Damiano Abeni Istituto Dermopatico dell'Immacolata - I.R.C.C.S.,
Roma.
Competing interests: None declared |
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