ISPOR Releases Recommendations for Outcome Assessment in Rare Disease Clinical Trials

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Task Force Report Seeks to Improve Bringing the Patient Voice Into Rare Disease Clinical Trials

Princeton, NJ—July 25, 2017—The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) announced today the publication of a new Task Force Report, Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: Report of the ISPOR Clinical Outcome Assessment Emerging Good Practices Task Force, in the July/August 2017 issue of Value in Health. This new report fills a long-standing gap in guidance recommendations for patient-centered outcomes measures for use in rare disease clinical trials.

Approximately 5000 to 8000 rare diseases have been identified to date. These conditions affect an estimated 6% to 8% of the population worldwide, and roughly 75% of rare diseases affect children. Regulatory incentives have increased the number of orphan drug designations and approvals. However, outcomes research assessments of technologies to treat rare diseases has been hampered by challenges inherent in the small, heterogeneous, and widely dispersed patient populations that are typical of these diseases.

This ISPOR Task Force Report provides state-of-the-art solutions to the challenges in measuring patient-reported and observer-reported outcomes in clinical trials involving patients with rare diseases. The report’s recommendations fill a long-standing gap in guidance for identifying, adapting, and implementing these types of clinical outcome assessments.

Beyond better outcomes measurement in rare disease trials, we hope that these guideline recommendations will improve the overall drug development process for rare diseases,” said lead author Katy Benjamin, PhD, SM of ICON, plc, Gaithersburg, MD, USA. “The explosion of new treatments for rare diseases provides clinicians and patients with many more viable options that can extend life and decrease suffering. Understanding what outcomes are important to the patient will help payers, providers, and patients themselves make better medical care decisions.”

Stressing the importance of defining patient-relevant treatment benefits, co-author Laurie Burke, RPh, MPH of the University of Maryland, Baltimore, MD, USA and former US Food and Drug Administration Director of Study Endpoints and Labeling Development added, “It is crucial during medical product development to incorporate the patient perspective to provide the rare disease community with valid, useful information that will ultimately help other  patients, caregivers, and clinicians make informed treatment decisions. Clinical outcome assessments are an important component of clinical trials and medical product development moving forward.”

ISPOR has published 11 Good Practices for Outcomes Research Task Force Reports that provide guidance recommendations on clinical outcomes assessments based on the FDA’s 2009 Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. These Task Force Reports are freely accessible here.

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ABOUT ISPOR
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that promotes health economics and outcomes research excellence to improve decision making for health globally.
Web: www.ispor.org LinkedIn: http://bit.ly/ISPOR-LIn Twitter: http://bit.ly/ISPOR-T (@ISPORorg) YouTube: http://bit.ly/ISPOR-YT Facebook: http://bit.ly/ISPOR-FB

ABOUT VALUE IN HEALTH
Value in Health (ISSN 1098-3015) is an international, indexed journal that publishes original research and health policy articles that advance the field of health economics and outcomes research to help health care leaders make evidence-based decisions. The journal’s 2016 impact factor score is 4.235. Value in Health is ranked 3rd out of 77 journals in health policy and services, 7th out of 347 journals in economics, and 9th out of 90 journals in health care sciences and services. Value in Health publishes 10 issues a year and circulates to more than 10,000 readers around the world.
Web: www.ispor.org/valueinhealth_index.asp Twitter: http://bit.ly/ISPORjournals-T (@ISPORjournals)

ABOUT ISPOR GOOD PRACTICES FOR OUTCOMES RESEARCH TASK FORCE REPORTS
ISPOR has earned an international reputation for research excellence based, in part, on its Good Practices for Outcomes Research Task Force Reports. These highly cited reports are expert consensus guideline recommendations on good practice standards for outcomes research (clinical, economic, and patient-reported outcomes) and on the use of this research in health care decision making. ISPOR Task Forces are comprised of subject matter experts representing different stakeholders from diverse work environments (i.e., regulators, payers, manufacturers, technology assessors, etc. from research, government, academic, and industry sectors around the world). All ISPOR Good Practices for Outcomes Research Task Force Reports are published in the Society’s scientific journal, Value in Health, and are made freely available as open access articles as part of the Society’s mission.

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