title: Impact of Measuring Patient-Reported Outcomes in Dermatology Drug Development.
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.
authors.affiliation: RTI Health Solutions, 3005 Boardwalk St., Suite 105, Ann Arbor, MI, 48108, USA. kcmerriman@rti.org.
authors.forename: Catherine
authors.initials: C
authors.lastname: Copley-Merriman
authors.affiliation: GlaxoSmithKline, Research Triangle Park, NC, USA.
authors.forename: Susan
authors.initials: S
authors.lastname: Zelt
authors.affiliation: RTI Health Solutions, 3005 Boardwalk St., Suite 105, Ann Arbor, MI, 48108, USA.
authors.forename: Marci
authors.initials: M
authors.lastname: Clark
authors.affiliation: RTI Health Solutions, Research Triangle Park, NC, USA.
authors.forename: Ari
authors.initials: A
authors.lastname: Gnanasakthy
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D001623:Betamethasone / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002117:Calcitriol / Q000031:analogs & derivatives* / Q000627:therapeutic use; D003879:Dermatologic Agents / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D000071066:Patient Reported Outcome Measures; D017060:Patient Satisfaction / Q000706:statistics & numerical data*; D011788:Quality of Life; D057566:Self Report; D012871:Skin Diseases / Q000188:drug therapy*; D011795:Surveys and Questionnaires
chemical_list: D000893:Anti-Inflammatory Agents; D003879:Dermatologic Agents; C055085:calcipotriene; C011175:betamethasone-17,21-dipropionate; D001623:Betamethasone; D002117:Calcitriol