Patient reported outcomes: looking beyond the label claim
Open accessAbstract
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.
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Patient-reported outcomes (PROs) are centrally important in clinical trials.
“The central importance of PROs in clinical trials has been emphasized by both international health regulatory agencies and patients [216, 217]”
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“Primary outcomes: HADS, fb‐EEG The central importance of PROs in clinical trials has been emphasized by both international health regulatory agencies and patients [216, 217], therefore, the primary outcome of interest will be the HADS [218, 219].”
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- Authors
- Doward, L. C., Gnanasakthy, A., & Baker, M. G.
- Affiliations
- Galen Research (United Kingdom) (GB)
- Novartis (United States) (US)
- European Brain Council (BE)
- Corresponding author
- Doward, Lynda C
- Venue
- Health and Quality of Life Outcomes
- Published
- 2010
- Volume
- 8
- Issue
- 1
- Pages
- 89
- ISSN
- 1477-7525 (ISSN-L)
- PMC
- PMC2936442
- OpenAlex
- W2108535474
- Cited by
- 162
- References
- 70
- Citations by year
- 2026: 5 · 2025: 14 · 2024: 9 · 2023: 8 · 2022: 11 · 2021: 10 · 2020: 13 · 2019: 10 · 2018: 10 · 2017: 12 · 2016: 15 · 2015: 10 · 2014: 6 · 2013: 14 · 2012: 8
- FWCI
- 21.88
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- cc-by
- Locations
- Health and Quality of Life Outcomes (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Topics
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Health Systems, Economic Evaluations, Quality of Life
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
- Keywords
- patient-reported outcomes, patient-reported outcome measures, pharmaceutical industry, FDA guidance, regulatory approval, quality of life, symptom improvement, drug pricing, payers, clinical trials
- MeSH terms
- Humans, Quality of Life, Surveys and Questionnaires, Patient Satisfaction, Outcome Assessment, Health Care, Quality Indicators, Health Care, Self Report
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