scholarly journals 2015 Behavioral clinical trials: Considerations for design and conduct using the new NIH study protocol template

2018 ◽  
Vol 2 (S1) ◽  
pp. 52-52
Author(s):  
Susan L. Murphy ◽  
Nancy Yovetich ◽  
Melissa Riddle
Keyword(s):  
Clinical Trials ◽  
Study Protocol ◽  
Research Community ◽  
Trial Protocol ◽  
Phase 2 ◽  
Conducting Phase ◽  
Study Population ◽  
Behavioral Trials ◽  
Supporting Materials

OBJECTIVES/SPECIFIC AIMS: (1) To discuss key differences of behavioral clinical trials from trials involving drugs, devices, and biologics and (2) to discuss NIH efforts to provide a study protocol template for use by investigators conducting behavioral clinical trials. METHODS/STUDY POPULATION: A working group was convened by NIH to refine the commonly used protocol template required for investigators conducting Phase 2 or 3 NIH-funded clinical trials. The committee met by phone regularly for 4 months to review, discuss, and refine each section of the template as needed to include aspects relevant to behavioral trials. RESULTS/ANTICIPATED RESULTS: The behavioral trial protocol template draft has been created and is being further modified by feedback from the research community. DISCUSSION/SIGNIFICANCE OF IMPACT: Use of the NIH behavioral trial protocol template is expected to enhance the quality of any behavioral study, because the template and supporting materials were developed with the unique aspects of behavioral research in mind.

Impact of study participation on survival of early-stage breast cancer patients: Results from the BRENDA study, a multicenter clinical cohort on quality of breast cancer care

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 521-521
Author(s):  
C. Kurzeder ◽  
J. König ◽  
A. Woeckel ◽  
G. Sauer ◽  
M. Wischnewsky ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Clinical Trials ◽  
Systemic Therapy ◽  
Early Stage ◽  
Trial Participation ◽  
Breast Cancer Patients ◽  
Study Participation ◽  
Neoadjuvant Systemic Therapy ◽  
Study Population

521 Background: Over the past two decades significant progress in treatment of early breast cancer has been accomplished through well designed, clinical trials (CTs). It has been hypothesized that trial participation could also be beneficial for the individual breast cancer patient. The BRENDA study group has been analysing quality of care provided by one university based and 16 regional breast cancer centers in south germany. In this study we investigated the impact of study participation on survival in an unselected clinical cohort of early stage breast cancer patients. Methods: The study population includes 5,966 patients who received primary therapy for early breast cancer between 1992 and 2005. Influence on survival by study participation was calculated by Cox proportional hazard analyses. Model adjustments include prognostic factors, type of treatment, age, risk group and time period. Guideline compliant treatment was assessed based on the St Gallen expert consensus recommendations and the German national S3 Guidelines. Results: A total of 738 patients (12%) received adjuvant (n = 552) or neoadjuvant (n = 186) systemic therapy within one out of 42 registered prospective multicenter phase II or III clinical trials. For patients not receiving neoadjuvant systemic therapy trial participation was associated with improved overall and disease free survival (hazard ratio [HR] = 0.75, 95% confidence interval [CI] 0.57–0.99, HR = 0.78, CI 0.63 - 0.97, respectively). The calculated effect was of similar magnitude after additional adjustment for co-existing morbidity of patients. Descriptional analysis revealed that guideline violations for locoregional or systemic treatment were more frequently found in patients receiving adjuvant treatment outside CTs. No significant effect on survival was calculated for 183 patients receiving neoadjuvant systemic therapy within CT protocols. Conclusions: In our study population participation in prospective clinical trials for adjuvant systemic therapy was associated with improved survival irrespective of treatment actually given. Intrinsic mechanisms within the framework of clinical trials can improve quality of breast cancer patient care. No significant financial relationships to disclose.

scholarly journals Review and Assessment of Patient-Reported Outcomes (PRO) in Patients with Chemotherapy-Induced Anemia (CIA)

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 5025-5025
Author(s):  
Antonia Morga ◽  
Chris Atzinger ◽  
Alexandra Barsdorf ◽  
Mary New ◽  
Ana Filipa Alexandre
Keyword(s):  
Clinical Trials ◽  
Conceptual Model ◽  
Cancer Patients ◽  
Patient Reported Outcomes ◽  
Signs And Symptoms ◽  
Phase 2 ◽  
Patient Reported ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

Abstract OBJECTIVES: Chemotherapy-induced anemia (CIA) is a common adverse event of cancer treatment and is associated with several symptoms that negatively impact health-related quality of life (HRQoL). Due to the nature of CIA signs and symptoms, the use of fit-for-purpose PRO measures reflecting patients' experience of the disease and its treatment is an important step in drug development and evaluation. In this project, we aimed to review and assess the most commonly used PRO measures to best capture the experiences of patients with non-myeloid malignancies. METHODS: A targeted literature review (TLR), including searches of the OVID database, US FDA/European Medicines Agency (EMA), clinical trial databases, regulatory labelling data and health technology assessment (HTA) reports/payer landscape recommendations, was conducted to identify PRO measures used to capture symptoms and HRQoL outcomes in non-myeloid cancer patients with CIA (2008-2021). PRO measures were identified from published studies, phase 2-4 clinical trials, and categorized according to the concept(s) measured and cancer type. Study outcomes were used to determine the optimal PRO strategy to capture the CIA symptoms of patients with non-myeloid cancer. The concepts captured from PROs identified in the TLR were used to develop a preliminary 'conceptual model': a graphical representation of signs and symptoms, and the perceived impact on HRQoL. Selected PRO measures, most relevant to the study objectives, were evaluated for their reliability, validity, responsiveness, and extent to which they cover HRQoL domains of interest and, in relation to the current regulatory environment, industry standards and guidance (e.g., FDA Guidance For Industry: Patient-Reported Outcome Measures, 2009). Reviews of the HTA guidelines and reports were conducted to provide insights into the applicability of PRO data from an HTA perspective. RESULTS: The conceptual model captured fatigue as the most important disease-related symptom experienced by non-myeloid cancer patients with CIA (Figure). The OVID search identified 17 PRO measures, which were then categorized by the key CIA-related concepts they assessed, including: sleep quality; HRQoL; anemia-related symptoms; depression/anxiety; and work productivity/activity impairment. The most frequently used PRO was the Functional Assessment of Cancer Therapy-Fatigue (FACT-F, N=11), followed by the FACT-Anemia (FACT-An, N=6). Ten PRO measures were identified from Phase 2-4 ongoing/completed US clinical trials and 6 PROs from EU clinical trials, with FACT-F (N=13) being the most common instrument used . FDA and EMA HRQoL-related product labelling information included the FACT-An, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) and Cancer Linear Analogue Scale (CLAS). From the TLR results, 9 PRO measures were selected for further review: FACT-An (An, Anemia); FACT-F; FACT-G (G, General); Functional Assessment of Chronic Illness Therapy-F; Patient-reported Outcomes Measurement Information System-F; Brief Fatigue Inventory; Visual Analogue Scale-F; Fatigue Symptom Inventory; and EORTC QLQ-C30. Of these, FACT-An was the only PRO measure specifically developed to measure fatigue and HRQoL in anemic cancer patients. FACT-An includes four HRQoL domains (assessing physical, social/family, emotional and functional wellbeing) from the FACT-G, plus an anemia subscale that comprises a fatigue subscale (13 items) and 7 anemia-specific questions. Results from the HTA/payer landscape review also indicated that the majority of recent HTA submissions reported findings from EORTC QLQ-C30 alongside the EORTC disease-specific adaptations for oncology-related indications and the EQ-5D. Many of the recent HTAs reported data from 2 or more different PRO measures. CONCLUSIONS: CIA impacts various aspects of patients' HRQoL; however, current PRO measures may only partially capture concepts of interest to patients. Multiple measures may be needed to adequately reflect all domains affected by CIA. Future studies may consider conducting concept elicitation interviews with patients and clinicians, to inform future updates to the conceptual model, and validation work to estimate meaningful change thresholds for these measures if not available. Figure 1 Figure 1. Disclosures Morga: Astellas Pharma Europe Ltd.: Current Employment. Atzinger: Astellas Pharma Inc.: Current Employment. Barsdorf: Clinical Outcomes Solutions: Current Employment. New: IQVIA: Current Employment. Alexandre: Astellas Pharma Europe B.V.: Current Employment.

Response to Salvage Therapies and Outcome of Patients with Multiple Myeloma Relapsing After Pomalidomide Therapy

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1965-1965
Author(s):  
Shirshendu Sinha ◽  
Shaji Kumar ◽  
Suzanne Hayman ◽  
Francis Buadi ◽  
Kristen Detweiler Short ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Multiple Myeloma ◽  
Clinical Trials ◽  
Survival Data ◽  
Research Funding ◽  
Phase 2 ◽  
Treatment Regimens ◽  
Phase 2 Clinical Trial ◽  
Study Population ◽  
Ongoing Phase

Abstract Abstract 1965 Background: Pomalidomide is an immunomodulatory compound to be used for heavily pre-treated patients with relapse and refractory multiple myeloma and has shown considerable efficacy in recent clinical trials. It is not clear how patients may respond to other therapies, once the disease becomes refractory to pomalidomide. We examined this question among a cohort of patients receiving pomalidomide therapy in phase 2 clinical trials. Methods: Patients enrolled in an ongoing phase 2 clinical trial of pomalidomide (2-4 mg daily) along with dexamethasone (40 mg weekly), in relapsed myeloma, form the study population. Several cohorts of patients (>= 3 prior therapies not specified, lenalidomide refractory, and lenalidomide and bortezomib refractory patients were enrolled sequentially in this trial (Lacy, M.Q., et al., J Clin Oncol, 2009. 27(30): p.5008–14). Only those patients who have gone off study for disease progression were included in the current analysis. Details of subsequent therapies and survival data were obtained from the medical records. Results: Forty-seven patients from among 142 patients enrolled on the clinical trial between November, 2007 and April, 2010 were included in the study. The median (range) duration of pomalidomide therapy was 4 (0.6-16) months. 15 (32%) patients were lenalidomide refractory and 11 (23%) were refractory to both lenalidomide and bortezomib at the time of study entry. The best confirmed response to pomalidomide was MR (Minimal Response) or better in 23 patients (52%). Various regimens were employed following relapse on pomalidomide. The response to the first regimen following relapse in the 34 patients where salvage therapy was initiated is as shown in Table 1. Overall, 81 regimens were employed across 34 patients; median (range) number of regimens per patients was 1 (0-8). The response rates to the different regimens were as shown in the Table 1. The median overall survival (OS) from the time of progression on pomalidomide was 14.7 months (95% CI; 4, NR). The OS was similar between those patients who had a response to pomalidomide (MR or better) and those who did not. However the OS was shorter for patients who were refractory to lenalidomide and bortezomib (2.1 months) compared to those who were lenalidomide refractory only (6.5 months), and the non-refractory group (NR), P=0.06. Conclusions: This study confirms poor outcome of the patients relapsing after all available therapies. It also gives interesting insights into the activity of different regimens among patients who have relapsed after pomalidomide. The findings once again highlight the incurable nature of the disease and warrant further investigation to develop newer effective treatment regimens for this group of patients who presently do not have effective therapeutic options especially in the relapsed setting. Regimens: ASCT: Autologous Stem Cell Transplantation, Bz: Bortezomib, Mel=Melphalan, Dex: Dexamethasone, CRD: Cyclophosphamide, Revlimid (Lenalidomide), Dexamethasone, CTX; Cyclophosphamide, Len; Lenalidomide, Sor: Sorafenib, VDT-PACE: Velcade (Bortezomib), Dexamethasone, Thalidomide, Cisplatin, Adriamycin, Cyclophosphamide, Etoposide. Response: SD; Stable Disease; Prog; Progression MR: Minimal Response; PR: Partial Response. Disclosures: Kumar: Celgene: Consultancy, Research Funding; Millennium: Research Funding; Merck: Consultancy, Research Funding; Novartis: Research Funding; Genzyme: Consultancy, Research Funding; Cephalon: Research Funding. Mikhael:Celgene: Research Funding; Onyx: Research Funding; Novartis: Research Funding. Dispenzieri:Celgene: Honoraria, Research Funding; Binding Site: Honoraria.

scholarly journals QOL-36. QUALITY OF SURVIVAL IN SIOPE BRAIN TUMOUR CLINICAL TRIALS FOR CHILDREN AGED LESS THAN 5 YEARS: DEVELOPMENT OF A CLINICAL TRIAL PROTOCOL

2018 ◽  
Vol 20 (suppl_2) ◽  
pp. i164-i165
Author(s):  
Jenny Limond ◽  
Sophie Thomas ◽  
Kim Bull ◽  
Jurgen Lemiere ◽  
Thomas Traunwieser ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Brain Tumour ◽  
Trial Protocol ◽  
Clinical Trial Protocol

PENGARUH KUALITAS LAYANAN DAN NILAI PELANGGAN TERHADAP KEPUASAN PELANGGAN TOKO SERBA ADA MATAHARI DI PONDOK GEDE PLAZA KOTA BEKASI

2017 ◽  
Vol 5 (3) ◽  
Author(s):  
Drs. Iwan Kurniawan Subagja, SE., MM. ◽  
Adista Fitriani
Keyword(s):  
Regression Analysis ◽  
Customer Satisfaction ◽  
Service Quality ◽  
Customer Value ◽  
Research Study ◽  
Linear Regression Analysis ◽  
Multiple Linear Regression Analysis ◽  
Analysis Technique ◽  
Study Population

Business competition is currently increasingly stringent requires that marketers develop marketing strategy that is telling. Companies that win business competition can solidify the company's position to survive in the future. This research study aims to analyze the effect of service quality and customer value on customer satisfaction of Multipurpose Shop Matahari in Pondok Gede Plaza simultaneously and partially. The study population is all customers of multipurpose store Matahari. This research uses multiple linear regression analysis technique, with survey data and questionnaire to 130 respondents. The results of this study indicate that the quality of service and customer value positively and significantly affect the customer satisfaction, service quality and customer value positively and partially significant to customer satisfaction.

Clinical Drug Development for the Treatment of Pulmonary Arterial Hypertension: Collaboration With the Pharmaceutical Industry and Regulatory Agencies

2010 ◽  
Vol 9 (4) ◽  
pp. 214-219
Author(s):  
Robyn J. Barst
Keyword(s):  
Clinical Trials ◽  
Pulmonary Arterial Hypertension ◽  
Drug Development ◽  
Phase 1 ◽  
Preclinical Studies ◽  
Phase 2 ◽  
Phase 3 ◽  
Preclinical Testing ◽  
New Drug ◽  
Pulmonary Arterial

Drug development is the entire process of introducing a new drug to the market. It involves drug discovery, screening, preclinical testing, an Investigational New Drug (IND) application in the US or a Clinical Trial Application (CTA) in the EU, phase 1–3 clinical trials, a New Drug Application (NDA), Food and Drug Administration (FDA) review and approval, and postapproval studies required for continuing safety evaluation. Preclinical testing assesses safety and biologic activity, phase 1 determines safety and dosage, phase 2 evaluates efficacy and side effects, and phase 3 confirms efficacy and monitors adverse effects in a larger number of patients. Postapproval studies provide additional postmarketing data. On average, it takes 15 years from preclinical studies to regulatory approval by the FDA: about 3.5–6.5 years for preclinical, 1–1.5 years for phase 1, 2 years for phase 2, 3–3.5 years for phase 3, and 1.5–2.5 years for filing the NDA and completing the FDA review process. Of approximately 5000 compounds evaluated in preclinical studies, about 5 compounds enter clinical trials, and 1 compound is approved (Tufts Center for the Study of Drug Development, 2011). Most drug development programs include approximately 35–40 phase 1 studies, 15 phase 2 studies, and 3–5 pivotal trials with more than 5000 patients enrolled. Thus, to produce safe and effective drugs in a regulated environment is a highly complex process. Against this backdrop, what is the best way to develop drugs for pulmonary arterial hypertension (PAH), an orphan disease often rapidly fatal within several years of diagnosis and in which spontaneous regression does not occur?

Prerequisites to support high-quality clinical trials in children and young people

2020 ◽  
pp. archdischild-2019-318677
Author(s):  
Steven Hirschfeld ◽  
Florian B Lagler ◽  
Jenny M Kindblom
Keyword(s):  
Clinical Trials ◽  
Healthcare Delivery ◽  
Paediatric Population ◽  
Ecosystem Functions ◽  
Healthcare Delivery System ◽  
High Quality ◽  
Children And Young People ◽  
The Right ◽  
Support Decision Making

Children have the right to treatment based on the same quality of information that guides treatment in adults. Without the proper evaluation of medicinal products and devices in paediatric clinical trials that are designed to meet the rigorous standards of the competent authorities, children are discriminated from advances in medicine. There are regulatory, scientific and ethical incentives to address the knowledge gap regarding efficacy and safety of medicines in the paediatric population. High-quality clinical trials involving children of all ages can generate data that will ultimately close the knowledge gaps and support decision making.For clinical trials that enrol children, the needs are specialised and often resource intensive. Prerequisites for successful paediatric clinical trials are personnel with training in both paediatrics and neonatology and expertise in clinical trials in these populations. Moreover, national and international networks for efficient collaboration, dissemination of information, and sharing of resources and expertise are also needed, together with competent, efficient and high-quality local infrastructure with effective processes. Monitoring and oversight bodies with the relevant competence, including expertise in paediatrics, is also an important prerequisite for paediatric clinical trials. Compromise in any of these components will compromise the downstream results.This paper discusses the structures and competences needed in order to perform effective, high-quality paediatric clinical trials with the ultimate goal of better medicines and treatments for children. We propose a model of examining the process as a series of components that each has to be optimised, then all the components are actively optimised to function together as an ecosystem, and the resulting ecosystem functions well with the general research system and the healthcare delivery system.

Getting out of the Field

2017 ◽  
Author(s):  
Jeasik Cho
Keyword(s):  
Qualitative Research ◽  
Academic Research ◽  
Scientific Research ◽  
Research Community

This chapter provides a review of the book, which explores how to conceptually understand and practically evaluate the quality of qualitative research. Despite the fact that there are few scholarly pieces regarding qualitative research, the depth and creativity that the pioneering researchers have demonstrated are profound, and the extent to which they cover not only the broad quality of qualitative research but also most of the specific qualities expected by many different kinds of qualitative research is incredible. This chapter summarizes the major topics of this book. Final remarks on this exciting, creative, but difficult topic are preceded by the following summary: Fortunately, There are commonly agreed, bold standards for evaluating the goodness of qualitative research in the academic research community. These standards are a part of what is generally called “scientific research.”

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