There is a broad agreement that patient-reported outcome (PRO) assessment in health care should proceed from a strong conceptual basis, with rationales clearly articulated in advance concerning what is to be measured and how this is to be accomplished. The representation of the patient's perspective has been part of clinical trials for some time; but the formalization of, and broader emphasis on PROs has become increasingly important with the release of the draft guidance for industry on patient-reported outcomes. In response, we address the challenges in constructing the conceptual foundations for PRO assessment to support drug product labeling claims submitted to regulatory agencies worldwide. After discussing what constitutes a PRO concept and an adequate basis for framing a PRO assessment, we examine the consequences of choosing PRO instruments without reference to a well-established conceptual framework for measurement. Then we illustrate through a hypothetical example the important interplay between the sponsor's proposed product claim, the corresponding conceptual model that depicts hypotheses involving the PRO concept(s) in the claim, and the resulting conceptual framework(s) for measurement to guide instrument selection and psychometric analyses. We discuss how these conceptual issues may vary or evolve over time depending on the phase of product development. As the science of PRO measurement continues to develop and experience accumulates, a consensus may emerge on how best to articulate the conceptual basis of PRO measurement for purposes of product labeling and regulation. In the meantime, one point is imminently clear: in regulatory decisions expected to affect not only the quantity but the quality of life, it is imperative to incorporate the patient's own perspective on the illness experience and the effects of therapy.
This qualitative approach confirmed past research while identifying novel concepts related to maintaining well-being and a sense of purpose despite caregiving hardships. Some aspects of caregiver suffering were identified. Caregiver self-regulation strategies revealed by this work suggest ideas for psychosocial interventions caregivers could use to protect themselves and the care recipient from impending distress.
Patient-reported outcomes (PROs) have become increasingly prevalent in clinical research and practice. On February 2, 2006, the Food and Drug Administration (FDA) released a draft guidance document with respect to incorporating PROs into clinical research endeavors which include FDA involvement. Researchers at the Mayo Clinic worked with FDA personnel and experts from academia, industry, clinical research, and clinical practice to facilitate discussion, dissemination, and operationalization of the FDA guidance document. This article introduces a manuscript series that resulted from this collective effort. Basic terms are defined and a précis of each article in the manuscript series is given. The ultimate conclusion to be drawn from this series is that, while the goals of assessing and analyzing PRO elements of clinical practice and research are challenging, there now exists a scientific foundation that makes achieving these goals feasible and the results credible. This is vitally important because after all, at the heart of all healthcare endeavors is the patient.
BACKGROUNDMedicare and third‐party payers may be reluctant to pay for investigational (protocol) therapy for patients with cancer on the premise that such treatment is more expensive than standard therapy. However, prior studies that have attempted to compare protocol therapy with standard therapy have been difficult to interpret because of the assortment of malignancies studied and the lack of suitable control groups of patients who received standard therapy.METHODSIn the current study, the authors conducted a retrospective review of the financial charges associated with protocol or nonprotocol (standard) chemotherapy in patients with a single malignancy, newly diagnosed acute myelogenous leukemia (AML), who received their initial course of chemotherapy (“induction”) at the Memorial Sloan‐Kettering Cancer Center (MSKCC) between 1996 and 1999. Protocol and nonprotocol groups were analyzed according to clinical characteristics and standard prognostic features to determine whether the two groups were comparable. Median charges for all patients were determined using a database that linked clinical information, financial data, and clinical outcomes.RESULTSA total of 353 patients with newly diagnosed AML were registered at MSKCC during the time period studied; of these, 79 patients (22%) received all of their care at the institution. Thirty patients (38%) received treatment on an investigational protocol. Forty‐nine patients (62%) did not receive protocol therapy for the following reasons: 10 patients (20%) did not meet eligibility criteria, 4 patients (8%) were eligible for protocol therapy but declined, and 35 patients (71%) met protocol criteria but were not offered protocol therapy based on the judgment of their primary oncologist. The groups were not comparable because patients treated with standard therapy were older and had a poorer initial Eastern Cooperative Oncology Group (ECOG) performance status. Overall median charges for patients in the nonprotocol group were higher than for patients treated on a protocol although charges were not related to age, initial ECOG performance status, or cytogenetic risk group.CONCLUSIONSAlthough charges for the nonprotocol group were higher, specific factors responsible for this difference were not identified. This study emphasizes the problems inherent in assembling suitable groups of patients for comparison. Cancer 2002;95:1064–70. © 2002 American Cancer Society.DOI 10.1002/cncr.10805
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