ISPOR PATIENT REPORTED OUTCOMES SPECIAL INTEREST GROUP (PRO)
PRO / QOL INFORMATION IN REGULATORY & HEALTH CARE
DECISIONS WG
Chair:
Patrick Marquis, MD, MBA
Managing Director - Global, Mapi Values
Background: The patients’ perspective has
never been more important in medical evaluations and
interventions. Clinical researchers are routinely incorporating
Patient Reported Outcomes (PRO) assessments in trial programs and
health-care providers are increasingly considering PRO information
for taking decisions on treatment and patient management.
Additionally, the FDA has asked for more meaningful endpoints that
accurately reflect patient needs and values. The creation of the
SEALD group and the development of the draft guidance on
“Patient-Reported Outcome Measures: Use in Medical Product
Development to Support Claims” illustrate the commitment of US
regulators toward PRO evaluation. The EMEA (CHMP) has developed a
draft “Reflection paper on the regulatory guidance for the use of
health-related quality of life measures in the evaluation of
medicinal products”.
Goal: The goal of the ISPOR PRO SIG
PRO/QOL Information in Regulatory & Health Care Decisions Working
Group is provide an update on the actual and envisioned role of
PRO from regulatory and health-care decision perspectives in USA,
Europe, and Japan. PRO information will be evaluated in different
regulatory and health-care decision settings such as PRO and drug
approval, PRO and label development, PRO and promotional claims,
PRO and risk management, PRO/utility and value messages for
pricing / reimbursement and formulary decisions, PRO and patient
screening, PRO and patient management, PRO and information to
prescribers and patients. Convergences and differences across
regulators and decision makers will be analyzed. The pragmatic
application of coming and existing guidance (FDA draft guidance
and EMEA reflection paper) is included.
Tasks:
To review the existing and future role of PRO information in
regulatory and health care decision (what type of PRO, when, how
and by whom?)
To determine the level of evidence required by the different
stake-holders for decision making
To identify “success-stories” from the decision-makers’
perspective
To determine barriers, limitations and needs for PRO
information
To develop recommendation for submitting PRO data (when and
how) to regulators and health-care decision-makers
