Core Outcome Measures in Effectiveness Trials

Defining a Minimum Set of Standardized Patient-centered Outcome Measures for Macular Degeneration

General Information

Abstract:
Purpose

To define a minimum set of outcome measures for tracking, comparing, and improving macular degeneration care.

Design

Recommendations from a working group of international experts in macular degeneration outcomes registry development and patient advocates, facilitated by the International Consortium for Health Outcomes Measurement (ICHOM).

Methods

Modified Delphi technique, supported by structured teleconferences, followed by online surveys to drive consensus decisions. Potential outcomes were identified through literature review of outcomes collected in existing registries and reported in major clinical trials. Outcomes were refined by the working group and selected based on impact on patients, relationship to good clinical care, and feasibility of measurement in routine clinical practice.

Results

Standardized measurement of the following outcomes is recommended: visual functioning and quality of life (distance visual acuity, mobility and independence, emotional well-being, reading and accessing information); number of treatments; complications of treatment; and disease control. Proposed data collection sources include administrative data, clinical data during routine clinical visits, and patient-reported sources annually. Recording the following clinical characteristics is recommended to enable risk adjustment: age; sex; ethnicity; smoking status; baseline visual acuity in both eyes; type of macular degeneration; presence of geographic atrophy, subretinal fibrosis, or pigment epithelial detachment; previous macular degeneration treatment; ocular comorbidities.

Conclusions

The recommended minimum outcomes and pragmatic reporting standards should enable standardized, meaningful assessments and comparisons of macular degeneration treatment outcomes. Adoption could accelerate global improvements in standardized data gathering and reporting of patient-centered outcomes. This can facilitate informed decisions by patients and health care providers, plus allow long-term monitoring of aggregate data, ultimately improving understanding of disease progression and treatment responses.

Aim:
To define a minimum set of outcome measures for tracking, comparing, and improving macular degeneration care.

Authors:
Ian A. Rodrigues, Sara M. Sprinkhuizen, Daniel Barthelmes, Mark Blumenkranz, Gemmy Cheung, Julia Halle, Robert Johnston, Ramasamy Kim, Caroline Klaver, Martin McKibbin, Nor Fariza Ngah, Suzann Pershing, Dato Shankar, Hiroshi Tamura, Adnan Tufail, Christina Y. Weng, Inger Westborg, Catherine Yelf, Nagahisa Yoshimura, Mark C. Gillies

Publication

Journal:
American Journal of Opthmology
Volume:
168
Issue:
Pages:
1 - 12
Year:
2016
DOI:
Further Study Information

Date:
Funding source(s):
Sponsorship to support this project was received from charitable organizations including the Macula Foundation USA; Macular Society UK; Retina Society USA; Save Sight Institute, Sydney, Australia; St Eric Eye Hospital, Stockholm, Sweden; Wills Eye Hospital, Philadelphia, Pennsylvania, USA; and Retina Suisse, Switzerland.

Health Area

Disease Category
Eyes & vision

Disease Name
Macular degeneration

Target Population

Age Range
-

Sex
Unknown


Nature / type of Intervention
Unknown

Method(s)

Delphi process
Literature review
Survey
Teleconferences

The ICHOM approach to defining health outcomes for conditions involves forming an international working group that follows a structured approach to compile the key outcomes and clinical characteristics that are recommended to be measured in routine clinical practice, called the “Standard Set.” The work was supported by sponsorship from a number of charitable health care organizations, but has no financial support from any pharmaceutical or health care technology organizations.


Stakeholders Involved

Unknown

Study Type

COS for practice

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