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Shannon M Smith,
Anthony T Wang,
Nathaniel P Katz,
Michael P McDermott, Laurie B Burke,
Paul Coplan,
Ian Gilron,
Sharon H Hertz,
Allison H Lin,
Bob A Rappaport,
Michael C Rowbotham,
Cristina Sampaio,
Michael Sweeney,
Dennis C Turk,
Robert H Dworkin
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ABSTRACT: The development of valid and informative treatment risk-benefit profiles requires consistent and thorough information about adverse event (AE) assessment and participants' AEs during randomized controlled trials (RCTs). Despite a 2004 extension of the Consolidated Standards of Reporting Trials (CONSORT) statement recommending the specific AE information that investigators should report, there is little evidence that analgesic RCTs adequately adhere to these recommendations. This systematic review builds on prior recommendations by describing a comprehensive checklist for AE reporting developed to capture clinically important AE information. Using this checklist, we coded AE assessment methods and reporting in all 80 double-blind RCTs of noninvasive pharmacologic treatments published in the European Journal of Pain, Journal of Pain, and PAIN® from 2006 to 2011. Across all trials, reports of AEs were frequently incomplete, inconsistent across trials, and, in some cases, missing. For example, >40% of trials failed to report any information on serious adverse events. Trials of participants with acute or chronic pain conditions and industry-sponsored trials typically provided more and better-quality AE data than trials involving pain-free volunteers or trials that were not industry sponsored. The results of this review suggest that improved AE reporting is needed in analgesic RCTs. We developed an ACTTION (Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities, and Networks) AE reporting checklist that is intended to assist investigators in thoroughly and consistently capturing and reporting these critically important data in publications.
Pain 03/2013; · 5.78 Impact Factor
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Dennis C Turk,
Alec B O'Connor,
Robert H Dworkin,
Amina Chaudhry,
Nathaniel P Katz,
Edgar H Adams,
John S Brownstein,
Sandra D Comer,
Richard Dart,
Nabarun Dasgupta, [......],
Edward Michna,
Pamela Palmer,
Sarah Peirce-Sandner,
Jennifer S Potter,
Srinivasa N Raja,
Christine Rauschkolb,
Carl L Roland,
Lynn R Webster,
Roger D Weiss,
Kerry Wolf
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ABSTRACT: Opioids are essential to the management of pain in many patients, but they also are associated with potential risks for abuse, overdose, and diversion. A number of efforts have been devoted to the development of abuse-deterrent formulations of opioids to reduce these risks. This article summarizes a consensus meeting that was organized to propose recommendations for the types of clinical studies that can be used to assess the abuse deterrence of different opioid formulations. Because of the many types of individuals who may be exposed to opioids, an opioid formulation will need to be studied in several populations using various study designs to determine its abuse-deterrent capabilities. It is recommended that the research conducted to evaluate abuse deterrence should include studies assessing: (1) abuse liability, (2) the likelihood that opioid abusers will find methods to circumvent the deterrent properties of the formulation, (3) measures of misuse and abuse in randomized clinical trials involving pain patients with both low risk and high risk of abuse, and (4) postmarketing epidemiological studies.
Pain 07/2012; 153(10):1997-2008. · 5.78 Impact Factor
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Robert H Dworkin,
Dennis C Turk,
Sarah Peirce-Sandner, Laurie B Burke,
John T Farrar,
Ian Gilron,
Mark P Jensen,
Nathaniel P Katz,
Srinivasa N Raja,
Bob A Rappaport, [......],
Kenneth Sommerville,
Brett R Stacey,
Ilona Steigerwald,
Jeffrey Tobias,
Ann Marie Trentacosti,
Ajay D Wasan,
George A Wells,
Jim Williams,
James Witter,
Dan Ziegler
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ABSTRACT: A number of pharmacologic treatments examined in recent randomized clinical trials (RCTs) have failed to show statistically significant superiority to placebo in conditions in which their efficacy had previously been demonstrated. Assuming the validity of previous evidence of efficacy and the comparability of the patients and outcome measures in these studies, such results may be a consequence of limitations in the ability of these RCTs to demonstrate the benefits of efficacious analgesic treatments vs placebo ("assay sensitivity"). Efforts to improve the assay sensitivity of analgesic trials could reduce the rate of falsely negative trials of efficacious medications and improve the efficiency of analgesic drug development. Therefore, an Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials consensus meeting was convened in which the assay sensitivity of chronic pain trials was reviewed and discussed. On the basis of this meeting and subsequent discussions, the authors recommend consideration of a number of patient, study design, study site, and outcome measurement factors that have the potential to affect the assay sensitivity of RCTs of chronic pain treatments. Increased attention to and research on methodological aspects of clinical trials and their relationships with assay sensitivity have the potential to provide the foundation for an evidence-based approach to the design of analgesic clinical trials and expedite the identification of analgesic treatments with improved efficacy and safety.
Pain 04/2012; 153(6):1148-58. · 5.78 Impact Factor
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ABSTRACT: The importance of content validity in developing patient reported outcomes (PRO) instruments is stressed by both the US Food and Drug Administration and the European Medicines Agency. Content validity is the extent to which an instrument measures the important aspects of concepts that developers or users purport it to assess. A PRO instrument measures the concepts most significant and relevant to a patient's condition and its treatment. For PRO instruments, items and domains as reflected in the scores of an instrument should be important to the target population and comprehensive with respect to patient concerns. Documentation of target population input in item generation, as well as evaluation of patient understanding through cognitive interviewing, can provide the evidence for content validity. Developing content for, and assessing respondent understanding of, newly developed PRO instruments for medical product evaluation will be discussed in this two-part ISPOR PRO Good Research Practices Task Force Report. Topics include the methods for generating items, documenting item development, coding of qualitative data from item generation, cognitive interviewing, and tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies. Part 1 covers elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument. Part 2 covers the instrument development process, the assessment of patient understanding of the draft instrument using cognitive interviews and steps for instrument revision. The two parts are meant to be read together. They are intended to offer suggestions for good practices in planning, executing, and documenting qualitative studies that are used to support the content validity of PRO instruments to be used in medical product evaluation.
Value in Health 12/2011; 14(8):967-77. · 2.19 Impact Factor
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[show abstract]
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ABSTRACT: The importance of content validity in developing patient reported outcomes (PRO) instruments is stressed by both the US Food and Drug Administration and the European Medicines Agency. Content validity is the extent to which an instrument measures the important aspects of concepts developers or users purport it to assess. A PRO instrument measures the concepts most relevant and important to a patient's condition and its treatment. For PRO instruments, items and domains as reflected in the scores of an instrument should be important to the target population and comprehensive with respect to patient concerns. Documentation of target population input in item generation, as well as evaluation of patient understanding through cognitive interviewing, can provide the evidence for content validity. Part 1 of this task force report covers elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument. Building on qualitative interviews and focus groups used to elicit concepts, cognitive interviews help developers craft items that can be understood by respondents in the target population and can ultimately confirm that the final instrument is appropriate, comprehensive, and understandable in the target population. Part 2 details: 1) the methods for conducting cognitive interviews that address patient understanding of items, instructions, and response options; and 2) the methods for tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies. The task force report's two parts are meant to be read together. They are intended to offer suggestions for good practice in planning, executing, and documenting qualitative studies that are used to support the content validity of PRO instruments to be used in medical product evaluation.
Value in Health 12/2011; 14(8):978-88. · 2.19 Impact Factor
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ABSTRACT: Irritable bowel syndrome (IBS) involves a broad range of physiological and psychological alterations that may affect brain-gut dysregulation, gut function, visceral perception, and mucosal integrity and function. Despite advances in our understanding of basic neuroenteric mechanisms and the role of effectors and transmitters in the brain-gut axis, a reliable biologic marker of IBS has yet to be identified. IBS diagnosis and status depend entirely on an assessment of IBS signs and symptoms. This has made development of optimal end points and study design for evaluation of efficacy of IBS drugs a challenge. This article addresses three main topics: the evolution of primary end points for IBS clinical trials; a potential path forward for IBS end points in new clinical trials; and recommendations for the future development of patient-reported outcome (PRO) instruments for use in IBS clinical trials.
The American Journal of Gastroenterology 04/2010; 105(4):731-5. · 7.28 Impact Factor
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Robert H Dworkin,
Dennis C Turk,
Sarah Peirce-Sandner,
Ralf Baron,
Nicholas Bellamy, Laurie B Burke,
Amy Chappell,
Kevin Chartier,
Charles S Cleeland,
Ann Costello, [......],
Christine Rauschkolb,
Bryce B Reeve,
Thomas Rhodes,
Cristina Sampaio,
David M Simpson,
Joseph W Stauffer,
Gerold Stucki,
Jeffrey Tobias,
Richard E White,
James Witter
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ABSTRACT: There has been an increase in the number of chronic pain clinical trials in which the treatments being evaluated did not differ significantly from placebo in the primary efficacy analyses despite previous research suggesting that efficacy could be expected. These findings could reflect a true lack of efficacy or methodological and other aspects of these trials that compromise the demonstration of efficacy. There is substantial variability among chronic pain clinical trials with respect to important research design considerations, and identifying and addressing any methodological weaknesses would enhance the likelihood of demonstrating the analgesic effects of new interventions. An IMMPACT consensus meeting was therefore convened to identify the critical research design considerations for confirmatory chronic pain trials and to make recommendations for their conduct. We present recommendations for the major components of confirmatory chronic pain clinical trials, including participant selection, trial phases and duration, treatment groups and dosing regimens, and types of trials. Increased attention to and research on the methodological aspects of confirmatory chronic pain clinical trials has the potential to enhance their assay sensitivity and ultimately provide more meaningful evaluations of treatments for chronic pain.
Pain 03/2010; 149(2):177-93. · 5.78 Impact Factor
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Robert H Dworkin,
Dennis C Turk,
Michael P McDermott,
Sarah Peirce-Sandner, Laurie B Burke,
Penney Cowan,
John T Farrar,
Sharon Hertz,
Srinivasa N Raja,
Bob A Rappaport,
Christine Rauschkolb,
Cristina Sampaio
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ABSTRACT: An essential component of the interpretation of results of randomized clinical trials of treatments for chronic pain involves the determination of their clinical importance or meaningfulness. This involves two distinct processes--interpreting the clinical importance of individual patient improvements and the clinical importance of group differences--which are frequently misunderstood. In this article, we first describe the essential differences between the interpretation of the clinical importance of patient improvements and of group differences. We then discuss the factors to consider when evaluating the clinical importance of group differences, which include the results of responder analyses of the primary outcome measure, the treatment effect size compared to available therapies, analyses of secondary efficacy endpoints, the safety and tolerability of treatment, the rapidity of onset and durability of the treatment benefit, convenience, cost, limitations of existing treatments, and other factors. The clinical importance of individual patient improvements can be determined by assessing what patients themselves consider meaningful improvement using well-described methods. In contrast, the clinical meaningfulness of group differences must be determined by a multi-factorial evaluation of the benefits and risks of the treatment and of other available treatments for the condition in light of the primary goals of therapy. Such determinations must be conducted on a case-by-case basis, and are ideally informed by patients and their significant others, clinicians, researchers, statisticians, and representatives of society at large.
Pain 12/2009; 146(3):238-44. · 5.78 Impact Factor
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Robert H Dworkin,
Dennis C Turk,
Dennis A Revicki,
Gale Harding,
Karin S Coyne,
Sarah Peirce-Sandner,
Dileep Bhagwat,
Dennis Everton, Laurie B Burke,
Penney Cowan,
John T Farrar,
Sharon Hertz,
Mitchell B Max,
Bob A Rappaport,
Ronald Melzack
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ABSTRACT: The objective of the present research was to develop a single measure of the major symptoms of both neuropathic and non-neuropathic pain that can be used in studies of epidemiology, natural history, pathophysiologic mechanisms, and treatment response. We expanded and revised the Short-form McGill Pain Questionnaire (SF-MPQ) pain descriptors by adding symptoms relevant to neuropathic pain and by modifying the response format to a 0-10 numerical rating scale to provide increased responsiveness in longitudinal studies and clinical trials. The reliability, validity, and subscale structure of the revised SF-MPQ (SF-MPQ-2) were examined in responses from 882 individuals with diverse chronic pain syndromes and in 226 patients with painful diabetic peripheral neuropathy who participated in a randomized clinical trial. The data suggest that the SF-MPQ-2 has excellent reliability and validity, and the results of both exploratory and confirmatory factor analyses provided support for four readily interpretable subscales-continuous pain, intermittent pain, predominantly neuropathic pain, and affective descriptors. These results provide a basis for use of the SF-MPQ-2 in future clinical research, including clinical trials of treatments for neuropathic and non-neuropathic pain conditions.
Pain 05/2009; 144(1-2):35-42. · 5.78 Impact Factor
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Dennis C Turk,
Robert H Dworkin,
Michael P McDermott,
Nicholas Bellamy, Laurie B Burke,
Julie M Chandler,
Charles S Cleeland,
Penney Cowan,
Rozalina Dimitrova,
John T Farrar, [......],
Patrick McGrath,
Henry J McQuay,
Steve Quessy,
Bob A Rappaport,
Dennis A Revicki,
Margaret Rothman,
Joseph W Stauffer,
Ola Svensson,
Richard E White,
James Witter
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ABSTRACT: The increasing complexity of randomized clinical trials and the practice of obtaining a wide variety of measurements from study participants have made the consideration of multiple endpoints a critically important issue in the design, analysis, and interpretation of clinical trials. Failure to consider important outcomes can limit the validity and utility of clinical trials; specifying multiple endpoints for the evaluation of treatment efficacy, however, can increase the rate of false positive conclusions about the efficacy of a treatment. We describe the use of multiple endpoints in the design, analysis, and interpretation of pain clinical trials, and review available strategies and methods for addressing multiplicity. To decrease the probability of a Type I error (i.e., the likelihood of obtaining statistically significant results by chance) in pain clinical trials, the use of gatekeeping procedures and other methods that correct for multiple analyses is recommended when a single primary endpoint does not adequately reflect the overall benefits of treatment. We emphasize the importance of specifying in advance the outcomes and clinical decision rule that will serve as the basis for determining that a treatment is efficacious and the methods that will be used to control the overall Type I error rate.
Pain 09/2008; 139(3):485-93. · 5.78 Impact Factor
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Dennis C Turk,
Robert H Dworkin,
Dennis Revicki,
Gale Harding, Laurie B Burke,
David Cella,
Charles S Cleeland,
Penney Cowan,
John T Farrar,
Sharon Hertz,
Mitchell B Max,
Bob A Rappaport
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ABSTRACT: This two-phase study was conducted to identify relevant domains of patient-reported outcomes from the perspective of people who experience chronic pain. In Phase 1, focus groups were conducted to generate a pool of patient outcome-related domains and their components. The results of the focus groups identified 19 aspects of their lives that were significantly impacted by the presence of their symptoms and for which improvements were important criteria they would use in evaluating the effectiveness of any treatment. Phase 2 was conducted to examine the importance and relevance of domains identified from a much larger and diverse sample of people with chronic pain. A survey was developed and posted on the American Chronic Pain Association website. Participants were asked to rate the importance of each item or domain identified by the focus groups on a scale of 0 to10 (i.e., 0="not at all important" and 10="extremely important"). The survey was completed by 959 individuals. The results indicate that all 19 aspects of daily life derived from the focus groups were considered important with a majority of respondents indicating a score of 8 or greater. In addition to pain reduction, the most important aspects were enjoyment of life, emotional well-being, fatigue, weakness, and sleep-related problems. Chronic pain clearly impacts health-related quality of life. The results of the two phases of the study indicate that people with chronic pain consider functioning and well-being as important areas affected by the presence of symptoms and as appropriate targets of treatment. These multiple outcomes should be considered when evaluating the efficacy and effectiveness of chronic pain treatments.
Pain 08/2008; 137(2):276-85. · 5.78 Impact Factor
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Robert H Dworkin,
Dennis C Turk,
Kathleen W Wyrwich,
Dorcas Beaton,
Charles S Cleeland,
John T Farrar,
Jennifer A Haythornthwaite,
Mark P Jensen,
Robert D Kerns,
Deborah N Ader, [......],
Christine Rauschkolb,
Dennis A Revicki,
Margaret Rothman,
Kenneth E Schmader,
Brett R Stacey,
Joseph W Stauffer,
Thorsten von Stein,
Richard E White,
James Witter,
Stojan Zavisic
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ABSTRACT: A consensus meeting was convened by the Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials (IMMPACT) to provide recommendations for interpreting clinical importance of treatment outcomes in clinical trials of the efficacy and effectiveness of chronic pain treatments. A group of 40 participants from universities, governmental agencies, a patient self-help organization, and the pharmaceutical industry considered methodologic issues and research results relevant to determining the clinical importance of changes in the specific outcome measures previously recommended by IMMPACT for 4 core chronic pain outcome domains: (1) Pain intensity, assessed by a 0 to 10 numerical rating scale; (2) physical functioning, assessed by the Multidimensional Pain Inventory and Brief Pain Inventory interference scales; (3) emotional functioning, assessed by the Beck Depression Inventory and Profile of Mood States; and (4) participant ratings of overall improvement, assessed by the Patient Global Impression of Change scale. It is recommended that 2 or more different methods be used to evaluate the clinical importance of improvement or worsening for chronic pain clinical trial outcome measures. Provisional benchmarks for identifying clinically important changes in specific outcome measures that can be used for outcome studies of treatments for chronic pain are proposed. PERSPECTIVE: Systematically collecting and reporting the recommended information needed to evaluate the clinical importance of treatment outcomes of chronic pain clinical trials will allow additional validation of proposed benchmarks and provide more meaningful comparisons of chronic pain treatments.
Journal of Pain 03/2008; 9(2):105-21. · 4.93 Impact Factor
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Robert C Bast,
J Tate Thigpen,
Susan G Arbuck,
Karen Basen-Engquist, Laurie B Burke,
Ralph Freedman,
Sandra J Horning,
Robert Ozols,
Gordon J Rustin,
David Spriggs,
Lari B Wenzel,
Richard Pazdur
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ABSTRACT: The unique characteristics of cancer, particularly issues involving the use of surrogate endpoints in clinical trials, present special challenges in the development of cancer drugs. In response, the U.S. Food and Drug Administration (FDA) has partnered with the American Society of Clinical Oncology, the American Association for Cancer Research, and the American Society of Hematology to conduct public workshops evaluating potential endpoints for drug approvals for the most common tumor types.
A workshop evaluating potential endpoints in ovarian cancer drug research was held in Bethesda, Maryland, in April 2006. Invited experts presented research findings and discussed endpoints in trials of drugs for treatment of Stage III and IV ovarian cancer.
The panel responded to specific questions from FDA, discussing use of progression-free survival as a surrogate for overall survival and use of CA-125 levels as an indicator of response. Panel members also addressed endpoints in first-line therapy, second-line and subsequent therapy, and maintenance therapy.
Expert commentary provided by panel members will inform FDA's draft guidance on clinical endpoints for cancer drug approvals and will be discussed at meetings of the FDA's Oncologic Drugs Advisory Committee. FDA intends to develop a set of principles that can be used to define efficacy standards for drugs used to treat ovarian and other cancers.
Gynecologic Oncology 12/2007; 107(2):173-6. · 3.89 Impact Factor
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Joseph Lipscomb,
Bryce B Reeve,
Steven B Clauser,
Jeffrey S Abrams,
Deborah Watkins Bruner, Laurie B Burke,
Andrea M Denicoff,
Patricia A Ganz,
Kathleen Gondek,
Lori M Minasian,
Ann M O'Mara,
Dennis A Revicki,
Edwin P Rock,
Julia H Rowland,
Maria Sgambati,
Edward L Trimble
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ABSTRACT: To evaluate and improve the use of cancer trial end points that reflect the patient's own perspective, the National Cancer Institute organized an international conference, Patient-Reported Outcomes Assessment in Cancer Trials (PROACT), in 2006. The 13 preceding articles in this special issue of the Journal were commissioned in preparation for or in response to the PROACT conference, which was cosponsored by the American Cancer Society. Drawing from these articles and also commentary from the conference itself, this concluding report takes stock of what has been learned to date about the successes and challenges in patient-reported outcome (PRO) assessment in phase III, phase II, and symptom management trials in cancer and identifies ways to improve the scientific soundness, feasibility, and policy relevance of PROs in trials. Building on this synthesis of lessons learned, this article discusses specific administrative policies and management procedures to improve PRO data collection, analysis, and dissemination of findings; opportunities afforded by recent methodologic and technologic advances in PRO data collection and analysis to enhance the scientific soundness and cost efficiency of PRO use in trials; and the importance of better understanding the usefulness of PRO data to the full spectrum of cancer decision makers, including patients and families, health providers, public and private payers, regulatory agencies, and standards-setting organizations.
Journal of Clinical Oncology 12/2007; 25(32):5133-40. · 18.37 Impact Factor
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ABSTRACT: In 2006, the US Food and Drug Administration (FDA) published draft guidance to provide recommendations for development, validation, implementation, and interpretation of patient-reported outcome (PRO) measures that can support treatment benefit claims in product labeling. Here, we summarize and discuss FDA approvals of anticancer products in the context of the draft guidance. We identified anticancer product approvals having efficacy claim(s) based at least in part on a PRO. In addition, we collated limitations of PRO instruments commonly submitted for regulatory review over the period from October 1, 2004 to September 30, 2006. From 1995 onward, nine indications were approved for seven anticancer products based at least in part on a PRO. In eight of nine approvals, PRO data supplemented other evidence of clinical benefit. In seven approvals, the PRO measured a single symptom or functional domain that was directly attributable to the treatment benefit observed in the disease. The FDA's draft PRO guidance describes principles that have been used in anticancer product approvals for more than a decade. PRO end points typically support treatment benefit claims that refer to a patient's symptoms or ability to function. Single-item PROs may be acceptable. PRO data should be both internally consistent and aligned with other evidence of clinical benefit. The FDA encourages sponsors to consult with the FDA early in the process of PRO development.
Journal of Clinical Oncology 12/2007; 25(32):5094-9. · 18.37 Impact Factor
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ABSTRACT: The U.S. Food and Drug Administration (FDA) approves labeling claims of drug efficacy based on substantial evidence of clinical benefit demonstrated in adequate and well-controlled investigations. Patient-reported outcomes (PROs) may support marketing claims of clinical benefit, either alone or with other study endpoints. Health-related quality of life (HRQL) is a PRO that comprehensively measures patients' reported health status. We present an overview of why HRQL-based efficacy claims have not to date been accepted by the FDA for inclusion in anticancer product labels. Persistent challenges to allowance of such claims include shortcomings in randomization and blinding of clinical trials, missing data, statistical multiplicity, and unclear intrinsic meaning of selected HRQL findings.
JNCI Monographs 02/2007;
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Bryce B Reeve, Laurie B Burke,
Yen-pin Chiang,
Steven B Clauser,
Lisa J Colpe,
Jeffrey W Elias,
John Fleishman,
Ann A Hohmann,
Wendy L Johnson-Taylor,
William Lawrence,
Claudia S Moy,
Louis A Quatrano,
William T Riley,
Barbara A Smothers,
Ellen M Werner
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ABSTRACT: Many of the Institutes, Agencies and Centers that make up the US Department of Health and Human Services (DHHS) have recognized the need for better instrumentation in health outcomes research, and provide support, both internally and externally, for research utilizing advances in measurement theory and computer technology (informatics). In this paper, representatives from several DHHS agencies and institutes will discuss their need for better instruments within their discipline and describe current or future initiatives for exploring the benefits of these technologies. Together, the perspectives underscore the importance of developing valid, precise, and efficient measures to capture the full burden of disease and treatment on patients. Initiatives, like the Patient-Reported Outcomes Measurement Information System (PROMIS) to create health-related quality of life item banks, represent a trans-DHHS effort to develop a standard set of measures for informing decision making in clinical research, practice, and health policy.
Quality of Life Research 02/2007; 16 Suppl 1:175-86. · 2.30 Impact Factor
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Dennis C Turk,
Robert H Dworkin, Laurie B Burke,
Richard Gershon,
Margaret Rothman,
Jane Scott,
Robert R Allen,
J Hampton Atkinson,
Julie Chandler,
Charles Cleeland, [......],
Joseph W Stauffer,
Gerold Stucki,
Jane Tollett,
Thorsten von Stein,
Mark S Wallace,
Joachim Wernicke,
Richard E White,
Amanda C Williams,
James Witter,
Kathleen W Wyrwich
Pain 01/2007; 125(3):208-15. · 5.78 Impact Factor
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ABSTRACT: This article concerns development and use of patient-reported outcomes (PROs) in clinical trials to evaluate medical products. A PRO is any report coming directly from patients, without interpretation by physicians or others, about how they function or feel in relation to a health condition and its therapy. PRO instruments are used to measure these patient reports. PROs provide a unique perspective on medical therapy, because some effects of a health condition and its therapy are known only to patients. Properly developed and evaluated PRO instruments also have the potential to provide more sensitive and specific measurements of the effects of medical therapies, thereby increasing the efficiency of clinical trials that attempt to measure the meaningful treatment benefits of those therapies. Poorly developed and evaluated instruments may provide misleading conclusions or data that cannot be used to support product labeling claims. We review selected major challenges from Food and Drug Administration's perspective in using PRO instruments, measures, and end points to support treatment benefit claims in product labeling. These challenges highlight the need for sponsors to formulate desired labeling claim(s) prospectively, to acquire and document information needed to support these claim(s), and to identify existing instruments or develop new and more appropriate PRO instruments for evaluating treatment benefit in the defined population in which they will seek claims.
Value in Health 10 Suppl 2:S125-37. · 2.19 Impact Factor
