title: The FDA guidance for industry on PROs: the point of view of a pharmaceutical company.
abstract: The importance of the patients point of view on their health status is widely recognised. Patient-reported outcomes is a broad term encompassing a large variety of different health data reported by patients, as symptoms, functional status, Quality of Life and Health-Related Quality of Life. Measurements of Health-Related Quality of Life have been developed during many years of researches, and a lot of validated questionnaires exist. However, few attempts have been made to standardise the evaluation of instruments characteristics, no recommendations are made about interpretation on Health-Related Quality of Life results, especially regarding the clinical significance of a change leading a therapeutic approach. Moreover, the true value of Health-Related Quality of Life evaluations in clinical trials has not yet been completely defined. An important step towards a more structured and frequent use of Patient-Reported Outcomes in drug development is represented by the FDA Guidance, issued on February 2006. In our paper we aim to report some considerations on this Guidance. Our comments focus especially on the characteristics of instruments to use, the Minimal Important Difference, and the methods to calculate it. Furthermore, we present the advantages and opportunities of using the Patient-Reported Outcomes in drug development, as seen by a pharmaceutical company. The Patient-Reported Outcomes can provide additional data to make a drug more competitive than others of the same pharmacological class, and a well demonstrated positive impact on the patient' health status and daily life might allow a higher price and/or the inclusion in a reimbursement list. Applying extensively the FDA Guidance in the next trials could lead to a wider culture of subjective measurement, and to a greater consideration for the patient's opinions on his/her care. Moreover, prescribing doctors and payers could benefit from subjective information to better define the value of drugs.
authors.affiliation: Health Technology Assessment, Medical Department, GSK S.p.A. Verona, Italy. fabio.a.arpinelli@gsk.com
authors.forename: Fabio
authors.initials: F
authors.lastname: Arpinelli
authors.forename: Francesco
authors.initials: F
authors.lastname: Bamfi
mesh_terms: D001294:Attitude to Health; D002986:Clinical Trials as Topic / Q000592:standards*; D004345:Drug Industry; D017408:Guidelines as Topic / Q000592:standards*; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods*; D011788:Quality of Life; D012649:Self Concept; D018434:Sickness Impact Profile; D014481:United States; D014486:United States Food and Drug Administration