1. OLGA: A nice niche designed to meet your evolving needs Pennifer Erickson, Ph.D. Presented at the Spring meeting of the Special Library Association’s Pharmaceutical & Health Division’s annual meeting, Napa, California March 22-24, 2009

2. Patient Reported Outcome (PRO) measurement: Initial steps and major initiatives in an evolving field Policy Major Research Developments Present19801990 Initial steps Based on: Patrick and Erickson, Health Status and Health Policy: Quality of Life in Health Care Evaluation and Resource Allocation, 1993 Croog et al. QoL article NEJM ‘86 CHI, NCHS 2000 Harmonizat’n Committee EMEA Medical Outcomes Study SIP for clinical practice Disease- specific measures, Guyatt et al. Responses to FDA PRO Guidance Satisfaction and Adherence NICE Translations Major initiatives Content validity & qualitative anal. FDA QWB PRO Guidance

3. After the 1986 NEJM article touting QoL benefits: the genie was out of the bottle Statements started appearing in labeling: Advair, as an example:... use of an instrument called the Asthma Quality of Life Questionnaire... patients... had clinically meaningful improvements in overall asthma-specific quality of life as defined by... and were used for promotional claims. The importance of having the patient’s voice heard in describing treatment benefit became clear to both industry and regulators. Clarity and standards were needed.

4. http://www.fda.gov/cder/guidance/5460dft.pdf Guidance states FDA’s approach to evaluating PRO instruments. Draft published Feb 2006. Final version currently going through clearance process…. No major policy revisions expected.

5. PRO: FDA Guidance definition “... any aspect of a patient’s health status that comes directly from the patient (i.e., without the interpretation of the patient’s responses by a physician or anyone else). In clinical trials, a PRO instrument can be used to measure the impact of an intervention on one or more aspects of patients’ health status,... ranging from the purely symptomatic (response to headache) to more complex concepts (e.g., ability to carry out activities of daily living), to extremely complex concepts such as quality of life.” That is, claims of treatment benefit are based on data from purposefully developed questionnaires and rating scales.

6. OLGA: A web-based service defined by PRO tools OLGA’s basic components: PRO tools that are used in clinical research, especially for labeling and reimbursement decisions Empirical studies that identify and support the use of outcomes measures for labeling and reimbursement Query-based software: OLGA-specific software that incorporates research-based file querying

7. OLGA: A niche service that focuses on PRO assessments and their uses Assessments Empirical Studies All PRO/HRQoL measures PRO/HRQoLs for clinical&policy PRO/HRQoLs in OLGA All studies Studies with 1+ PRO measure Studies in OLGA

8. Why OLGA? An evolving field needs a specialized information center to: identify available tools OLGA has over 20 years of cumulated tools and their uses OLGA constantly looks for new developments published literature key meetings provide contacts, copyright and other conditions of use find obscure tools, e.g., those identified only by initials keep up with regulatory and reimbursement PRO demands

9. OLGA answers tool-specific questions, such as: Tool development: Why -- e.g., for patients with diabetes, arthritis, etc. What -- items, content How -- patient input, types of patients, validity Tool availability Who to contact for permission to use, copyright, etc. Which translations/adaptations have been done How have they been done, and Where to obtain translations/adaptations

10. OLGA also: provides review versions or sample questions/items of tools indexed in OLGA identifies PROs used in labeling evaluates translation methods used by consultancies specific instrument systems, e.g., EORTC keeps track of methodologies related to FDA’s latest concerns, e.g., PRO-related qualitative methods

11. OLGA’s query-based software is designed to respond to compound inquiries: Query: Is there a valid QoL instrument available in Portuguese for use in a clinical trial of patients with osteoarthritis (OA)? Parsing the question to develop a response: 1. OA-specific instrument is preferred 2. Documentation on instrument validity, both qualitative and quantitative, as determined by the developer needs to be available 3. Use by other investigators further supports validity and informs about instrument generalizability 4. Instrument needs to be able to detect treatment change for use in a clinical trial 5. Portuguese -- if not the original language, then information on translation/adaptation process and results is needed

12. PRO measurement is a dynamic field new concerns and issues are regularly raised by regulators, e.g., FDA, and other policy makers, e.g., NICE these concerns increase the informational needs of Outcomes Researchers many of these concerns are too specific to be covered by more general databases OLGA is the only service that is solely dedicated to tracking the development and use of PRO instruments, especially with regard to regulatory and policy settings.

13. How OLGA can work for you: web-based OLGA can answer 75-80% of the questions from your patrons Because of our experience we can anticipate most questions and assemble the information so it’s ready when its needed -- prospective consulting. person-based OLGA can answer the remaining queries Call or e-mail with specific information needs.

14. Contact OLGA -- Value Added Interaction with users provides valuable information about new features to add to our services, e.g., satisfaction clients’ areas of research to note in our content-selection process topics to include in our user group distributions An important part of OLGA services is to provide personal responses to user queries on an as-needed basis.

15. For more information, contact Pennifer Erickson, Ph.D. 1316 Deerfield Drive State College, PA 16803 (814) 237-3281 Penny@OLGA-QoL.com Or, see me after the session for a demonstration.