SUBJECT

Measurement of patient reported outcomes

lecture

master

Faculty of Social Sciences

Health Policy, Planning and Financing (Specialization in Health Economics) MSc (part time)

4

Semester 3

Autumn semester

• Students need to be aware of the recent paradigm shift in medicine resulted in the increasing relevance of measuring health outcomes based on the opinion of the patient/individual
• Students are required to be familiar with the basic concept of measuring patient reported outcomes (PROs), including international guidances
• Students are introduced the concept of measuring quality of life (QoL), including measurement in special populations, especially in childhood
• Students are required to be familiar with concept, steps and key terms of development, local adaptation and validation of PRO/QoL measures/instruments
• Students are required to be aware classification, strength and weakness of generic and specific measures/instruments, they need to be able to select appropriate measures with respect to study question
• Students are introduced the relevance of measuring PROs in economic evaluation and decision making in health care, they need to be aware of the most widely used utility measurement tools, as well as their strength and limitations to select appropriate utility measures
• Students are required to be familiar with the concept of quality adjusted life year (QALY), disability adjusted life year (DALY), their potential strength and limitations, the literature of their critics including ethical and methodological issues
• Students will be aware of the key issues of the transferability of QALYs across different countries
• Students are introduced to the concept of mapping measures to utilities
• Students are introduced the relevance and key terms of measuring medication adherence
• Students are introduced the key terms and measurement methods of patient satisfaction
• Students needs to be aware of the principles of designing non-interventional trials

Development of PRO instruments

• FDA. Guidance for industry. Patient-reported outcome measures: use in medical product development to support labeling claims. FDA, USA, 2009.
• Patrick DL, Burke LB, Gwaltney CJ, Leidy NK, Martin ML, Molsen E, Ring L. (2011) Content validity--establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: part 1--eliciting concepts for a new PRO instrument. Value Health 14 (8):967-977.
• Patrick DL, Burke LB, Gwaltney CJ, Leidy NK, Martin ML, Molsen E, Ring L. (2011) Content validity--establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: part 2--assessing respondent understanding. Value Health 14 (8):978-988.

Adaptation of QoL measures

• Lovas K, Kaló Z. et al. Establishing a standard for patient-completed instrument adaptations in Eastern Europe: experience with the Nottingham Health Profile in Hungary. Health Policy, 2003, 63, 49-61.

Measuring QoL in children

• Matza LS, Swensen AR. Assessment of Health-Related Quality of Life in Children: A Review of Conceptual, Methodological, and Regulatory Issues. Value in Health 2004, 7 (1), 79-82.

Utility measurement

• Torrance GW. (1986) Measurement of health state utilities for economic appraisal. J Health Econ, 5: 1-30.

QALY and DALY

• Murray CLJ. (1994) Quantifying the burden of disease: the technical basis for disability-adjusted life years. Bulletin of the World Health Organisation, 72 (3): 429-445.

Critique of QALY and DALY

• Lyttkens CH. (2003) Time to disable DALYs? On the use of disability-adjusted life-years in health policy. European Journal of Health Economics, 4(3): 195-202.
• Wiliams, A. (1996) QALYS and ethics: a health economist's perspective. Soc sci med, 43(12): 1795-1804.
• Harris J. (1987) QALYfying the value of life. J Med Ethics, 13: 117-123

Burden of disease studies

• Inotai A, Ágh T, Mészáros Á. (2012) Quality of life, utility and health burden in asthma, chronic obstructive pulmonary disease and rheumatoid arthritis. International Journal of Person Centered Medicine, 2: 505-510.

Transferability of QALY

• Paul Kind. A revised protocol for the valuation of health states defined by the EQ-5D-3L classification system. Learning the lessons from the MVH study

Medication adherence

• Cramer JA, Roy A, Burrell A, Fairchild CJ, Fuldeore MJ, Ollendorf DA, Wong PK. Medication compliance and persistence: terminology and definitions. Value Health. 2008 Jan-Feb;11(1):44-7.