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Impact of Measuring Patient-Reported Outcomes in Dermatology Drug Development

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Abstract

Although some symptoms of dermatologic diseases, such as pruritus and pain, can be subjectively assessed only by patients, the most commonly used endpoints in dermatology drug research traditionally have been clinician-reported outcomes. Research has found that patient-reported outcomes (PROs) were included in only one-quarter of 125 trials conducted between 1994 and 2001. Our objective was to characterize the impact of PROs in dermatology drug development from the patient, prescriber, regulator, payer, and manufacturer perspectives using a case study approach. We conducted a structured literature review for pivotal clinical trials using PROs for six dermatologic products (MAS063DP, onabotulinumtoxinA, calcipotriene hydrate plus betamethasone dipropionate, pimecrolimus, tacrolimus, and ustekinumab). We also searched regulatory websites to identify product labeling and the UK National Institute for Health and Care Excellence website to identify submissions for the products of interest. A total of 32 articles illustrating the various perspectives were selected for inclusion. Clinical trials that include PROs allow patients to differentiate among treatments based on the experience of other patients participating in trials and enable prescribers to understand the benefit–risk profile of new treatments. The inclusion of PROs enables regulators to evaluate product benefits with a patient-centered perspective; five of the products of interest obtained eight total product labeling statements. PRO data supported manufacturers’ dissemination of product benefits in the form of publications and PRO labeling for the product. For payers, PRO data were used in an analysis of cost effectiveness of new treatments. Inclusion of PROs in dermatology drug development programs benefits patients, prescribers, regulators, manufacturers, and payers.

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Acknowledgments

Kate Lothman of RTI Health Solutions provided medical writing assistance.

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Authors and Affiliations

  1. RTI Health Solutions, 3005 Boardwalk St., Suite 105, Ann Arbor, MI, 48108, USA

    Catherine Copley-Merriman & Marci Clark

  2. GlaxoSmithKline, Research Triangle Park, NC, USA

    Susan Zelt

  3. RTI Health Solutions, Research Triangle Park, NC, USA

    Ari Gnanasakthy

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  1. Catherine Copley-Merriman
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Correspondence to Catherine Copley-Merriman.

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Author contributions

Catherine Copley-Merriman and Marci Clark were involved in all aspects of this research and development of the manuscript. Ari Gnanasakthy and Susan Zelt reviewed the research report and manuscript.

Conflicts of interest

Catherine Copley-Merriman, Marci Clark, and Ari Gnanasakthy are employees of RTI Health Solutions. Susan Zelt is an employee of GlaxoSmithKline.

Funding

This research was performed under a contract between RTI Health Solutions and GlaxoSmithKline and was sponsored by GlaxoSmithKline. RTI Health Solutions supported the preparation of this publication. RTI Health Solutions develops PRO instruments, among other research projects.

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Copley-Merriman, C., Zelt, S., Clark, M. et al. Impact of Measuring Patient-Reported Outcomes in Dermatology Drug Development. Patient 10, 203–213 (2017). https://doi.org/10.1007/s40271-016-0196-6

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  • DOI: https://doi.org/10.1007/s40271-016-0196-6

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