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Laurie B. Burke - One of the best experts on this subject based on the ideXlab platform.
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FDA Oncology Center of Excellence Project Renewal: Engaging the Oncology Community to Update Product Labeling for Older Oncology Drugs.
Clinical cancer research : an official journal of the American Association for Cancer Research, 2020Co-Authors: Paul G. Kluetz, Patricia Keegan, George D. Demetri, Katherine A Thornton, Joohee Sul, Janice Kim, Harvey Katzen, Laurie B. Burke, R. Donald Harvey, Elleni AlebachewAbstract:The U.S. Food and Drug Administration (FDA) conducts independent reviews of scientific data obtained with investigational drug Products to ensure that they are safe and effective. As a result of this process, FDA-approved Product Labeling is generated that is considered one of the most trusted sources of information for use of an approved drug. But FDA approval is only the beginning of the life cycle of a new drug; the first oncology drugs now have more than 7 decades of clinical experience in the post-marketing setting. Due in part to lack of incentives, some companies may not seek inclusion of new data, other than new safety information, in FDA-approved Product Labeling. Ensuring that Product Labeling provides adequate directions for use is important for all drugs, including older therapies that may form the backbone of many standard combination regimens for pediatric and adult cancers. Project Renewal is an FDA Oncology Center of Excellence pilot program that leverages expertise from the clinical and scientific oncology communities to review published literature and generate a drug-specific Product report summarizing data that may support updates to FDA-approved Product Labeling. This article provides a broad overview of Project Renewal9s collaborative pilot process for identifying and assessing literature supporting potential Labeling updates, while engaging the oncology community to increase awareness of FDA9s evidentiary standards and deliberative processes used when considering the addition of new indications and dosing regimens to Product Labeling.
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a concept taxonomy and an instrument hierarchy tools for establishing and evaluating the conceptual framework of a patient reported outcome pro instrument as applied to Product Labeling claims
Value in Health, 2009Co-Authors: Pennifer Erickson, Richard J Willke, Laurie B. BurkeAbstract:ABSTRACT Objective To facilitate development and evaluation of a PRO instrument conceptual framework, we propose two tools—a PRO concept taxonomy and a PRO instrument hierarchy. FDA's draft guidance on patient reported outcome (PRO) measures states that a clear description of the conceptual framework of an instrument is useful for evaluating its adequacy to support a treatment benefit claim for use in Product Labeling the draft guidance, however does not propose tools for establishing or evaluationg a PRO instrument's conceptual framework. Methods We draw from our review of PRO concepts and instruments that appear in prescription drug Labeling approved in the United States from 1997 to 2007. Results We propose taxonomy terms that define relationships between PRO concepts, including "family," "compound concept," and "singular concept." Based on the range of complexity represented by the concepts, as defined by the taxonomy, we propose nine instrument orders for PRO measurement. The nine orders range from individual event counts to multiitem, multiscale instruments. Conclusion This analysis of PRO concepts and instruments illustrates that the taxonomy and hierarchy are applicable to PRO concepts across a wide range of therapeutic areas and provide a basis for defining the instrument conceptual framework complexity. Although the utility of these tools in the drug development, review, and approval processes has not yet been demonstrated, these tools could be useful to improve communication and enhance efficiency in the instrument development and review process.
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Patient-Reported Outcomes to Support Medical Product Labeling Claims: FDA Perspective
Value in Health, 2007Co-Authors: Donald L. Patrick, Laurie B. Burke, John H. Powers, Jane Scott, Edwin P. Rock, Sahar Dawisha, Robert T. O'neill, Dianne L. KennedyAbstract: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 andevaluated 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.
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Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved Product labels.
Controlled clinical trials, 2004Co-Authors: Richard J Willke, Laurie B. Burke, Pennifer EricksonAbstract:Abstract Context The term “patient-reported outcomes” (PROs) has evolved to include any endpoint derived from patient reports, whether collected in the clinic, in a diary, or by other means, including single-item outcome measures, event logs, symptom reports, formal instruments to measure health-related quality of life (HRQL), health status, adherence, and satisfaction with treatment. This term coincides with the explicit interest from drug development researchers and regulatory authorities in the appropriate utilization and reporting of treatment impact measures. Objective To determine the level and nature of use of PROs compared to other types of effectiveness endpoints in approved Product Labeling for new drugs recently approved in the United States. Design and sources Review and analysis of effectiveness endpoints as reported in clinical study descriptions in approved Product Labeling of new molecular entities (NMEs) approved in the United States from 1997 through 2002. Main outcome measures Effectiveness study endpoints reported in approved Product Labeling that fall into the following categories of measurement: PROs, clinician-reported outcomes (CROs), and laboratory test/device measurement endpoints. Results PROs were reported in 64 (30%) of the 215 Product labels reviewed. Clinician-reported outcomes were reported most frequently (62%) followed by laboratory/device endpoints (50%). PROs were the only type of endpoint used in the FDA-approved label for 23 Products. Formal multiitem PRO scales were cited 22 times. Use of PROs is most common in antiinflammatory, CNS, gastrointestinal, respiratory, allergic conjunctivitis, and urologic therapy areas. The frequency of reported PRO use over this period did not change. Conclusion PROs, although quite variable as a class of study endpoints, were found to have a significant role in the development and evaluation of new medicines. More formal guidance from the FDA about use of such measures along with continued collaboration by PRO researchers to develop and disseminate standards will enhance the appropriate use of PROs in future drug development and Labeling.
Donald L. Patrick - One of the best experts on this subject based on the ideXlab platform.
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pediatric patient reported outcome instruments for research to support medical Product Labeling report of the ispor pro good research practices for the assessment of children and adolescents task force
Value in Health, 2013Co-Authors: Louis S Matza, Donald L. Patrick, Anne W Riley, John J Alexander, Luis Rajmil, A Pleil, Monika BullingerAbstract:Abstract Background Patient-reported outcome (PRO) instruments for children and adolescents are often included in clinical trials with the intention of collecting data to support claims in a medical Product label. Objective The purpose of the current task force report is to recommend good practices for pediatric PRO research that is conducted to inform regulatory decision making and support claims made in medical Product Labeling. The recommendations are based on the consensus of an interdisciplinary group of researchers who were assembled for a task force associated with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In those areas in which supporting evidence is limited or in which general principles may not apply to every situation, this task force report identifies factors to consider when making decisions about the design and use of pediatric PRO instruments, while highlighting issues that require further research. Good Research Practices Five good research practices are discussed: 1) Consider developmental differences and determine age-based criteria for PRO administration: Four age groups are discussed on the basis of previous research ( Conclusions Additional research is needed to provide methodological guidance for future studies, especially for studies involving young children and parents’ observational reports. As PRO data are increasingly used to support pediatric Labeling claims, there will be more information regarding the standards by which these instruments will be judged. The use of PRO instruments in clinical trials and regulatory submissions will help ensure that children’s experience of disease and treatment are accurately represented and considered in regulatory decisions.
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Patient-Reported Outcomes to Support Medical Product Labeling Claims: FDA Perspective
Value in Health, 2007Co-Authors: Donald L. Patrick, Laurie B. Burke, John H. Powers, Jane Scott, Edwin P. Rock, Sahar Dawisha, Robert T. O'neill, Dianne L. KennedyAbstract: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 andevaluated 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.
Monika Bullinger - One of the best experts on this subject based on the ideXlab platform.
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pediatric patient reported outcome instruments for research to support medical Product Labeling report of the ispor pro good research practices for the assessment of children and adolescents task force
Value in Health, 2013Co-Authors: Louis S Matza, Donald L. Patrick, Anne W Riley, John J Alexander, Luis Rajmil, A Pleil, Monika BullingerAbstract:Abstract Background Patient-reported outcome (PRO) instruments for children and adolescents are often included in clinical trials with the intention of collecting data to support claims in a medical Product label. Objective The purpose of the current task force report is to recommend good practices for pediatric PRO research that is conducted to inform regulatory decision making and support claims made in medical Product Labeling. The recommendations are based on the consensus of an interdisciplinary group of researchers who were assembled for a task force associated with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In those areas in which supporting evidence is limited or in which general principles may not apply to every situation, this task force report identifies factors to consider when making decisions about the design and use of pediatric PRO instruments, while highlighting issues that require further research. Good Research Practices Five good research practices are discussed: 1) Consider developmental differences and determine age-based criteria for PRO administration: Four age groups are discussed on the basis of previous research ( Conclusions Additional research is needed to provide methodological guidance for future studies, especially for studies involving young children and parents’ observational reports. As PRO data are increasingly used to support pediatric Labeling claims, there will be more information regarding the standards by which these instruments will be judged. The use of PRO instruments in clinical trials and regulatory submissions will help ensure that children’s experience of disease and treatment are accurately represented and considered in regulatory decisions.
Pennifer Erickson - One of the best experts on this subject based on the ideXlab platform.
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a concept taxonomy and an instrument hierarchy tools for establishing and evaluating the conceptual framework of a patient reported outcome pro instrument as applied to Product Labeling claims
Value in Health, 2009Co-Authors: Pennifer Erickson, Richard J Willke, Laurie B. BurkeAbstract:ABSTRACT Objective To facilitate development and evaluation of a PRO instrument conceptual framework, we propose two tools—a PRO concept taxonomy and a PRO instrument hierarchy. FDA's draft guidance on patient reported outcome (PRO) measures states that a clear description of the conceptual framework of an instrument is useful for evaluating its adequacy to support a treatment benefit claim for use in Product Labeling the draft guidance, however does not propose tools for establishing or evaluationg a PRO instrument's conceptual framework. Methods We draw from our review of PRO concepts and instruments that appear in prescription drug Labeling approved in the United States from 1997 to 2007. Results We propose taxonomy terms that define relationships between PRO concepts, including "family," "compound concept," and "singular concept." Based on the range of complexity represented by the concepts, as defined by the taxonomy, we propose nine instrument orders for PRO measurement. The nine orders range from individual event counts to multiitem, multiscale instruments. Conclusion This analysis of PRO concepts and instruments illustrates that the taxonomy and hierarchy are applicable to PRO concepts across a wide range of therapeutic areas and provide a basis for defining the instrument conceptual framework complexity. Although the utility of these tools in the drug development, review, and approval processes has not yet been demonstrated, these tools could be useful to improve communication and enhance efficiency in the instrument development and review process.
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Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved Product labels.
Controlled clinical trials, 2004Co-Authors: Richard J Willke, Laurie B. Burke, Pennifer EricksonAbstract:Abstract Context The term “patient-reported outcomes” (PROs) has evolved to include any endpoint derived from patient reports, whether collected in the clinic, in a diary, or by other means, including single-item outcome measures, event logs, symptom reports, formal instruments to measure health-related quality of life (HRQL), health status, adherence, and satisfaction with treatment. This term coincides with the explicit interest from drug development researchers and regulatory authorities in the appropriate utilization and reporting of treatment impact measures. Objective To determine the level and nature of use of PROs compared to other types of effectiveness endpoints in approved Product Labeling for new drugs recently approved in the United States. Design and sources Review and analysis of effectiveness endpoints as reported in clinical study descriptions in approved Product Labeling of new molecular entities (NMEs) approved in the United States from 1997 through 2002. Main outcome measures Effectiveness study endpoints reported in approved Product Labeling that fall into the following categories of measurement: PROs, clinician-reported outcomes (CROs), and laboratory test/device measurement endpoints. Results PROs were reported in 64 (30%) of the 215 Product labels reviewed. Clinician-reported outcomes were reported most frequently (62%) followed by laboratory/device endpoints (50%). PROs were the only type of endpoint used in the FDA-approved label for 23 Products. Formal multiitem PRO scales were cited 22 times. Use of PROs is most common in antiinflammatory, CNS, gastrointestinal, respiratory, allergic conjunctivitis, and urologic therapy areas. The frequency of reported PRO use over this period did not change. Conclusion PROs, although quite variable as a class of study endpoints, were found to have a significant role in the development and evaluation of new medicines. More formal guidance from the FDA about use of such measures along with continued collaboration by PRO researchers to develop and disseminate standards will enhance the appropriate use of PROs in future drug development and Labeling.
Dianne L. Kennedy - One of the best experts on this subject based on the ideXlab platform.
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Patient-Reported Outcomes to Support Medical Product Labeling Claims: FDA Perspective
Value in Health, 2007Co-Authors: Donald L. Patrick, Laurie B. Burke, John H. Powers, Jane Scott, Edwin P. Rock, Sahar Dawisha, Robert T. O'neill, Dianne L. KennedyAbstract: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 andevaluated 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.
