scholarly journals A 15 Year Review (2006-2020) of Patient-Reported Outcomes (PRO) in United States Oncology Product Labeling and Trends in Sponsor Size and Oncology Experience

2021 ◽  
Author(s):  
Lisa D Cooper ◽  
Emily Wo ◽  
Irene Lee
Keyword(s):  
Patient Reported Outcomes ◽  
Meaningful Change ◽  
Product Labeling ◽  
Fda Guidance ◽  
Marketing Application ◽  
Patient Reported ◽  
The Us ◽  
Approved Drugs ◽  
Pro Tools ◽  
Novel Drug

Abstract BackgroundDespite wide use of patient reported outcomes (PRO) tools in clinical development, resulting data is rarely incorporated into the US label. This study reviewed oncology product labels approved by the Food and Drug Administration (FDA) between 2006 and 2020 to determine if the rate of PRO inclusion in labeling has meaningfully changed. Sponsors were assessed to identify demographic trends in achieving PRO label success. MethodsFDA-approved drugs were searched utilizing the Drugs@FDA database by month from January 2006 to December 2020 for novel drug and biologic approvals. Labels and product summary basis of approval (SBA) were reviewed for inclusion of PRO data. Sponsor size and experience were determined for each product in the year of initial approval. Results155 oncology products received initial approval between 2006-2020, of which only 7 contained PRO data in the label. More than half (53.5%) of products had PRO data described in the SBA. Over time, PRO information increasingly been included in the product marketing application. Sponsors utilizing PRO data tend to be experienced in oncology development and larger in size. ConclusionsThere has been no meaningful change in inclusion of PRO data in oncology product labeling over the past 15 years. Recent FDA guidance and initiatives may provide additional clarification to support appropriate PRO tools to support label inclusion as well as another forum for distributing PRO data publicly.

Between a logic of disruption and a logic of continuation: Negotiating the legitimacy of algorithms used in automated clinical decision-making

2021 ◽  
pp. 136345932199674
Author(s):  
Rikke Torenholt ◽  
Henriette Langstrup
Keyword(s):  
Decision Making ◽  
Clinical Practice ◽  
Patient Reported Outcomes ◽  
Patient Involvement ◽  
Clinical Decision Making ◽  
Diagnostic Procedures ◽  
Clinical Decision ◽  
Patient Reported ◽  
Pro Tools

In both popular and academic discussions of the use of algorithms in clinical practice, narratives often draw on the decisive potentialities of algorithms and come with the belief that algorithms will substantially transform healthcare. We suggest that this approach is associated with a logic of disruption. However, we argue that in clinical practice alongside this logic, another and less recognised logic exists, namely that of continuation: here the use of algorithms constitutes part of an established practice. Applying these logics as our analytical framing, we set out to explore how algorithms for clinical decision-making are enacted by political stakeholders, healthcare professionals, and patients, and in doing so, study how the legitimacy of delegating to an algorithm is negotiated and obtained. Empirically we draw on ethnographic fieldwork carried out in relation to attempts in Denmark to develop and implement Patient Reported Outcomes (PRO) tools – involving algorithmic sorting – in clinical practice. We follow the work within two disease areas: heart rehabilitation and breast cancer follow-up care. We show how at the political level, algorithms constitute tools for disrupting inefficient work and unsystematic patient involvement, whereas closer to the clinical practice, algorithms constitute a continuation of standardised and evidence-based diagnostic procedures and a continuation of the physicians’ expertise and authority. We argue that the co-existence of the two logics have implications as both provide a push towards the use of algorithms and how a logic of continuation may divert attention away from new issues introduced with automated digital decision-support systems.

scholarly journals Linguistic validation of the simplified Chinese version of the US National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE™)

BMC Cancer ◽  
2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Cheng KKF ◽  
S. A. Mitchell ◽  
N. Chan ◽  
E. Ang ◽  
W. Tam ◽  
...  
Keyword(s):  
Adverse Events ◽  
Cancer Patients ◽  
Patient Reported Outcomes ◽  
A Priori ◽  
Cognitive Interviews ◽  
Simplified Chinese ◽  
Patient Reported ◽  
The Us ◽  
The Common ◽  
Response Choices

Abstract Background The aim of this study was to translate and linguistically validate the U.S. National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE™) into Simplified Chinese for use in Singapore. Methods All 124 items of the English source PRO-CTCAE item library were translated into Simplified Chinese using internationally established translation procedures. Two rounds of cognitive interviews were conducted with 96 cancer patients undergoing adjuvant treatment to determine if the translations adequately captured the PRO-CTCAE source concepts, and to evaluate comprehension, clarity and ease of judgement. Interview probes addressed the 78 PRO-CTCAE symptom terms (e.g. fatigue), as well as the attributes (e.g. severity), response choices, and phrasing of ‘at its worst’. Items that met the a priori threshold of ≥20% of participants with comprehension difficulties were considered for rephrasing and retesting. Items where < 20% of the sample experienced comprehension difficulties were also considered for rephrasing if better phrasing options were available. Results A majority of PRO-CTCAE-Simplified Chinese items were well comprehended by participants in Round 1. One item posed difficulties in ≥20% and was revised. Two items presented difficulties in < 20% but were revised as there were preferred alternative phrasings. Twenty-four items presented difficulties in < 10% of respondents. Of these, eleven items were revised to an alternative preferred phrasing, four items were revised to include synonyms. Revised items were tested in Round 2 and demonstrated satisfactory comprehension. Conclusions PRO-CTCAE-Simplified Chinese has been successfully developed and linguistically validated in a sample of cancer patients residing in Singapore.

scholarly journals Responder Definition of a Patient-Reported Outcome Instrument for Laryngopharyngeal Reflux Based on the US FDA Guidance

2015 ◽  
Vol 18 (4) ◽  
pp. 396-403 ◽  
Author(s):  
Han-Chung Lien ◽  
Chen-Chi Wang ◽  
Shou-Wu Lee ◽  
Jeng-Yuan Hsu ◽  
Hong-Zen Yeh ◽  
...  
Keyword(s):  
Laryngopharyngeal Reflux ◽  
Patient Reported Outcome ◽  
Fda Guidance ◽  
Outcome Instrument ◽  
Patient Reported ◽  
The Us ◽  
Us Fda ◽  
Definition Of

scholarly journals PROMIS and FAAM Minimal Clinically Important Differences in Foot and Ankle Orthopedics

2018 ◽  
Vol 40 (1) ◽  
pp. 65-73 ◽  
Author(s):  
Man Hung ◽  
Judith F. Baumhauer ◽  
Frank W. Licari ◽  
Maren W. Voss ◽  
Jerry Bounsanga ◽  
...  
Keyword(s):  
Patient Reported Outcomes ◽  
Minimum Clinically Important Difference ◽  
Measurement Information ◽  
Diagnostic Study ◽  
Foot And Ankle ◽  
Meaningful Change ◽  
Level Of Evidence ◽  
Patient Reported ◽  
Level I

Background: Establishing score points that reflect meaningful change from the patient perspective is important for interpreting patient-reported outcomes. This study estimated the minimum clinically important difference (MCID) values of 2 Patient-Reported Outcomes Measurement Information System (PROMIS) instruments and the Foot and Ankle Ability Measure (FAAM) Sports subscale within a foot and ankle orthopedic population. Methods: Patients seen for foot and ankle conditions at an orthopedic clinic were administered the PROMIS Physical Function (PF) v1.2, the PROMIS Pain Interference (PI) v1.1, and the FAAM Sports at baseline and all follow-up visits. MCID estimation was conducted using anchor-based and distribution-based methods. Results: A total of 3069 patients, mean age of 51 years (range = 18-94), were included. The MCIDs for the PROMIS PF ranged from approximately 3 to 30 points (median = 11.3) depending on the methods being used. The MCIDs ranged from 3 to 25 points (median = 8.9) for the PROMIS PI, and from 9 to 77 points (median = 32.5) for the FAAM Sports. Conclusions: This study established a range of MCIDs in the PROMIS PF, PROMIS PI, and FAAM Sports indicating meaningful change in patient condition. MCID values were consistent across follow-up periods, but were different across methods. Values below the 25th percentile of MCIDs may be useful for low-risk clinical decisions. Midrange values (eg, near the median) should be used for high stakes decisions in clinical practice (ie, surgery referrals). The MCID values within the interquartile range should be utilized for most decision making. Level of Evidence: Level I, diagnostic study, testing of previously developed diagnostic measure on consecutive patients with reference standard applied.

Unveiling the real-world outcomes of breast cancer (BC) patients with taxanes-induced neuropathy using a digital patient-powered network (PPN).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18248-e18248
Author(s):  
Raanan Berger ◽  
Lior Hasid ◽  
Irad Deutsch ◽  
Eliran Malki ◽  
Maytal Bivas-Benita
Keyword(s):  
Cancer Patients ◽  
Real World ◽  
Healthcare Costs ◽  
Patient Reported Outcomes ◽  
Early Stage ◽  
Treatment Decisions ◽  
Real World Evidence ◽  
Patient Reported ◽  
The Us ◽  
Disease Stages

e18248 Background: Taxanes-induced neuropathy is common in BC patients receiving taxanes, forcing dose reductions and treatment delays and posing serious challenges for the long-term patient QoL. Discovering neuropathy predictors in patients could guide better treatment decisions, improved QoL and reduce healthcare costs. Belong digital PPN is a social network for cancer patients and caregivers that supports disease management. In this study we used our artificial intelligence (AI) engine to classify the prevalence, characteristics and taxanes-induced neuropathy status of BC patients. Methods: We analyzed real-world patient-reported outcomes provided voluntarily and anonymously from users on the Belong PPN. Data from BC patients reporting treatment with taxanes was extracted and additional analysis segmented the data to those who experienced neuropathy and those who did not. Further validation of the data was performed by our research team to assure accuracy. Results: We evaluated 169 BC cancer patients from the US treated with taxanes. In the cohort 72% were Paclitaxel-treated and 28% Docetaxel-treated at various disease stages: 68% at early stage BC (0-2) and 32% at the advanced/metastatic stages (3-4). 83% of Paclitaxel-treated patients and 67% of Docetaxel-treated patients reported experiencing neuropathy in the Belong platform. These real-world reports indicated significantly higher incidence of taxane-induced neuropathy in comparison to literature summarizing data from clinical trials, suggesting neuropathy incidence of 27% for paclitaxel and 16% for docetaxel (grades 2-4). Conclusions: Real-world patient-reported outcomes from the Belong PPN captured the prevalence of taxanes-induced neuropathy in BC patients and correlated it to the specific drug in use. Evidence for higher incidence of taxanes-induced neuropathy may lead to lower patient QoL and higher healthcare costs and should stimulate better treatment decisions. Further exploration of the gap between controlled clinical studies and real-world evidence is urgently needed to understand the true patient outcomes and optimize healthcare accordingly.

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