Inconsistencies in Measurement of Health-Related Quality of Life (HRQoL)/ Patient-Reported Outcomes (PROs) in Rare Skin Diseases (RSDs)

Author(s)

Thakur D1, Igbelina CD1, Yu J2, Zhou M3
1Cytel Inc., Waltham, MA, USA, 2Cytel Inc., Toronto, ON, Canada, 3Cytel Inc., Mississauga, ON, Canada

Presentation Documents

OBJECTIVES: Skin conditions affect different aspects of the patients’ HRQoL. PROs are increasingly being considered by Health Technology Agencies (HTAs) in evaluation of new treatments. The objective of this review was to assess the frequency of use of HRQOL/PROs in HTA submissions for RSDs, and how different HTA agencies evaluate and assess appropriateness of PRO measures.

METHODS: HTA websites of NICE, SMC, HAS, IQWiG, and CADTH, were reviewed for submissions from last 10 years in RSDs. Data regarding type of PROs, inclusion of PROs in the economic model, and HTA comments regarding appropriateness of the PRO measures were collected.

RESULTS: A total of 33 submissions across 8 RSDs were identified. HRQoL/PRO measures were reported in 17 submissions across 6 indications (mastocytosis: 4, Merkel cell carcinoma (MCC): 3, mycosis fungoides: 5, neurofibromatosis type 1: 2, pemphigus vulgaris: 1, scleroderma: 2). Only 7 submissions received HTA approval (HAS:5, CADTH: 2). The EQ-5D questionnaire was the most used measure (7 submissions). Other PRO measures included Skindex-29, FACT-M, Likert scale & the Itchy Quality of Life questionnaire, SF-12, PedsQL, NRS-11, PII scale, DLQI-14, Clinical Global Impression, VAS, SGRQ, TMSAS. EQ-5D was considered appropriate in capturing changes in HRQoL related to adverse events by multiple HTAs. FACT-M was considered appropriate to assess HRQoL in individuals with MCC. Other measures including the SF-12, and Skindex-29 were critiqued for the difficulty in drawing reliable conclusions, for containing subjective improvements, and for not having validated thresholds to determine minimal important difference (MID).

CONCLUSIONS: Our study identified a variety of PRO tools being used to assess HRQoL in RSD HTA submissions, even within same indications. Generic tools are often used despite not capturing dermatologic issues. Thus, true impact of RSDs on HRQoL may be underestimated. Further research is warranted to validate MID thresholds and to improve the reliability of PRO tools in RSDs.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Value in Health, Volume 25, Issue 12S (December 2022)

Code

HTA159

Topic

Health Technology Assessment, Patient-Centered Research

Topic Subcategory

Decision & Deliberative Processes, Patient-reported Outcomes & Quality of Life Outcomes

Disease

SDC: Sensory System Disorders (Ear, Eye, Dental, Skin)

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