Patient Reported Outcomes: Looking beyond the Label Claim

Lynda C Doward; Ari Gnanasakthy; Mary G Baker


Health and Quality of Life Outcomes. 2010;8(82) 

In This Article

Abstract and Introduction


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.


In recent years, the pharmaceutical market has become characterized by more knowledgeable customers, growing cost pressure from private and public third party payers and increasing need for product differentiation in a highly competitive market. To ensure product success companies must generate value propositions that go beyond traditional safety and clinical efficacy messages. One route to achieving this is by generating evidence on the patient's perspective of treatment. Such evidence is commonly generated by using patient reported outcome (PRO) measures to assess patient views on product efficacy. PRO is an umbrella term used to describe outcomes collected directly from the patient without interpretation by clinicians or others.[1–3] PRO data are collected via standardised questionnaires designed to measure an explicit concept (construct) such as symptoms, functioning (activity limitations), health status/health related quality of life (HRQL) or quality of life (QoL).

Industry-sponsored PRO use centres predominantly around inclusion in marketing studies, patient registries and clinical trials. Although PRO endpoints are still used in a minority of clinical trials their use has grown in recent years, particularly in randomised Phase III trials. An analysis of clinical trials registered with shows that approximately 12% of the interventional trials registered by the pharma industry and over 15% of non-industry sponsored protocols now incorporate some form of PRO assessment.[4] Although for certain therapeutic areas (most notably, psychiatric disorders) PROs may be included in clinical trials as primary efficacy indicators, commercial use of PRO outcomes focuses predominantly on their employment as secondary endpoints designed to provide 'added value' data to support key biomedical endpoints. Such 'value' is viewed largely in terms of their potential for securing a labelling claim in the USA or inclusion in the summary of product characteristics (SmPC) in Europe and in providing supporting arguments for reimbursement. Indeed, since the publication of the US Food and Drug Administration's (FDA) Guidance on use of PROs to support potential claims in product labelling, discussion on PROs and label claims has received considerable attention both within the literature and at industry or professional society meetings.[5–7] Consequently PROs are routinely included in clinical trials with these objectives in mind and the data used solely for these purposes. However, regulators and payers are only two of the key stakeholders with an interest in the drug licensing and reimbursement process. Both clinicians and patients now play a key role in influencing the availability and use of pharmaceutical products. Indeed, it is the interaction between these interested bodies that can help or hinder a drug's progress to market and ultimately, its success as a product. The focus for the current study is to look at whether the pharmaceutical industry maximises the potential for generating PRO-based added value messages both in terms of the quality of the data collected and the relevance of those data for key stakeholders. Ultimately, this study aims to highlight the value in considering the use of PRO data beyond acquiring a label claim.