Patient reported outcomes: looking beyond the label claim

Abstract

The use of patient reported outcome scales in clinical trials conducted by the pharmaceutical industry has become more widespread in recent years. The use of such outcomes is particularly common for products developed to treat chronic, disabling conditions where the intention is not to cure but to ameliorate symptoms, facilitate functioning or, ultimately, to improve quality of life. In such cases, patient reported evidence is increasingly viewed as an essential complement to traditional clinical evidence for establishing a product's competitive advantage in the marketplace. In a commercial setting, the value of patient reported outcomes is viewed largely in terms of their potential for securing a labelling claim in the USA or inclusion in the summary of product characteristics in Europe. Although, the publication of the recent US Food and Drug Administration guidance makes it difficult for companies to make claims in the USA beyond symptom improvements, the value of these outcomes goes beyond satisfying requirements for a label claim. The European regulatory authorities, payers both in the US and Europe, clinicians and patients all play a part in determining both the availability and the pricing of medicinal products and all have an interest in patient-reported data that go beyond just symptoms. The purpose of the current paper is to highlight the potential added value of patient reported outcome data currently collected and held by the industry for these groups.

Patients' needs in hematology: whose perspectives?

“…PRO is an umbrella term encompassing a number of patient self-reported parameters related to a patient's health status and perception of treatment side effects. [1][2][3][4] As defined by the US Food and Drug Administration (FDA), PRO is "a measurement based on a report that comes directly from the patient about the status of a patient's condition without amendment or interpretation of the patient's response by a clinician or anyone else". 5,6 PRO assessments introduce the patients' perspective into the clinical process via standardized self-report instruments that are scored by the patient, not a clinician, or a researcher.…”

Patients engagemnet in clinical trial input

“…La scelta dei trattamenti sulla base di specifiche preferenze individuali, l'orientamento degli obiettivi e una stretta e attenta collaborazione tra paziente e medico curante sembrano essere la strafarmaci destinati al trattamento di condizioni croniche, invalidanti, non guaribili definitivamente, nelle quali si ricerca il miglioramento dei sintomi e della QoL. In tale contesto, infatti, i PRO sono visti come un complemento essenziale alle misure tradizionali di efficacia [22]. Figura 2.…”

Resultados comunicados por el paciente en las fichas técnicas de medicamentos aprobados en España durante el período 2000-2012

“…Una forma de conseguirlo es incorporar la perspectiva del paciente sobre el tratamiento que recibe. Se puede generar evidencia de la efectividad de un tratamiento desde la perspectiva del paciente utilizando medidas PRO (PROMs por sus siglas en inglés) 10 , debidamente validadas en la cultura en la que se usan, que permiten medir aspectos determinados, como síntomas, limitaciones en la actividad diaria, CVRS, etcétera.…”