Published Jun 2017
Citation
Benjamin K, Vernon MK, Patrick DL, Perfetto E, Nestler-Parr S, Burke L. Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials - an ISPOR COA Emerging Good Practices Task Force Report. Value Health. 2017;20(7):838-855.
Abstract
Background: Rare diseases (RDs) affect a small number of people
within a population. About 5000 to 8000 distinct RDs have been
identified, with an estimated 6% to 8% of people worldwide suffering
from an RD. Approximately 75% of RDs affect children. Frequently,
these conditions are heterogeneous; many are progressive. Regulatory
incentives have increased orphan drug designations and approvals.
Objective: To develop emerging good practices for RD outcomes
research addressing the challenges inherent in identifying, selecting,
developing, adapting, and implementing patient-reported outcome
(PRO) and observer-reported outcome (ObsRO) assessments for use in
RD clinical trials.
Good Practices for Outcomes Research: This report
outlines the challenges and potential solutions in determining clinical
outcomes for RD trials. It follows the US Food and Drug Administration
Roadmap to Patient-Focused Outcome Measurement in Clinical
Trials. The Roadmap consists of three columns: 1) Understanding the
Disease or Condition, 2) Conceptualizing Treatment Benefit, and 3)
Selecting/Developing the Outcome Measure.
Challenges in column 1
include factors such as incomplete natural history data and heterogeneity
of disease presentation and patient experience. Solutions
include using several information sources, for example, clinical
experts and patient advocacy groups, to construct the condition’s
natural history and understand treatment patterns. Challenges in
column 2 include understanding and measuring treatment benefit
from the patient’s perspective, especially given challenges in defining
the context of use such as variations in age or disease severity/
progression. Solutions include focusing on common symptoms across
patient subgroups, identifying short-term outcomes, and using multiple
types of COA instruments to measure the same constructs. Challenges
in column 3 center around the small patient population and heterogeneity
of the condition or study sample. Few disease-specific instruments
for RDs exist. Strategies include adapting existing instruments
developed for a similar condition or that contain symptoms of importance
to the RD patient population, or using a generic instrument
validated for the context of use.
Conclusions: This report provides
state-of-the-art solutions to patient-reported outcome (PRO) and
observer-reported outcome (ObsRO) assessments challenges in clinical
trials of patients with RDs. These recommended solutions are both
pragmatic and creative and posed with clear recognition of the global
regulatory context used in RD clinical development programs.
Keywords: rare diseases, clinical outcomes assessment, instrument
development, clinical trials.
Copyright © 2017, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc.
Full Content
Log In to View ReportRelated Content
Questions?
For any questions about this report please contact us.