scholarly journals Multidisciplinary molecular tumour board: a tool to improve clinical practice and selection accrual for clinical trials in patients with cancer

ESMO Open ◽  
2018 ◽  
Vol 3 (5) ◽  
pp. e000398 ◽  
Author(s):  
Christian Rolfo ◽  
Paolo Manca ◽  
Roberto Salgado ◽  
Peter Van Dam ◽  
Amelie Dendooven ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Liquid Biopsy ◽  
New Technologies ◽  
Treatment Options ◽  
Personalised Medicine ◽  
Point Mutations ◽  
Tumour Tissue ◽  
Expanded Access Programme ◽  
Access Programme

BackgroundThe complexity of delivering precision medicine to oncology patients has led to the creation of molecular tumourboards (MTBs) for patient selection and assessment of treatment options. New technologies like the liquid biopsy are augmenting available therapeutic opportunities. This report aims to analyse the experience of our MTB in the implementation of personalised medicine in a cancer network.Materials and methodsPatients diagnosed with solid tumours progressing to standard treatments were referred to our Phase I unit. They underwent comprehensive next generation sequencing (NGS) of either tumour tissue or cell-free circulating tumour DNA (ctDNA) or both. The MTB expressed either a positive or negative opinion for the treatment of the patients with discovered druggable alterations inside a clinical trial, in an expanded access programme, with a compassionate use. Afterwards, discovered alterations were matched with OncoKB levels of evidence for the choice of alteration-specific treatments in order to compare MTB outcomes with a standardised set of recommendations.ResultsNGS was performed either on ctDNA or tumour tissue or in both of them in 204 patients. The MTB evaluated 173 of these cases. Overall, the MTB proposed alteration-specific targeted therapy to 72 patients (41.6%). 49 patients (28.3% of the total evaluated) were indicated to enter a clinical trial. In 29 patients with matched liquid biopsy NGS (lbNGS), tumour tissue NGS (ttNGS) and MTB evaluation, the MTB changed the treatment strategy coming from standardised recommendations based on lbNGS and ttNGS alone in 10 patients (34.5%), thanks to the evaluation of other clinical parameters. In our cohort, lbNGS was more likely, compared with ttNGS, to detect point mutations (OR 11, 95% CI 2.9 to 24.1, p<0.001) and all-type alterations (OR 13.6, 95% CI 5.5 to 43.2, p<0.001) from the same genes of matched patients.ConclusionsOur MTB allows patients with refractory cancer to be included in clinical trials and improves the precision of clinical decisions compared with a standardised set of mutation-driven recommendations.

The brain metastasis journey: Experience from patient, caregiver, and physician surveys on diagnosis and treatment of brain metastases.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e14004-e14004
Author(s):  
Albert Eusik Kim ◽  
GI-Ming WANG ◽  
Kristin A Waite ◽  
Scott Elder ◽  
Avery Fine ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Brain Metastases ◽  
Checkpoint Inhibitors ◽  
Treatment Options ◽  
Whole Brain Radiotherapy ◽  
Well Being ◽  
Cross Sectional Survey ◽  
Cross Sectional ◽  
Brain Radiotherapy

e14004 Background: Brain metastases (BM) is one of the most feared complications of cancer due to substantial neurologic sequalae, neuro-cognitive morbidity and grim prognosis. In the past decade, targeted therapies and checkpoint inhibitors have resulted in meaningfully improved overall survival for a minority of these patients. Accordingly, there is a growing need to identify issues surrounding patient survivorship and to standardize physician practice patterns for these patients. To date, there has not been a well-conducted formal study to specifically explore these questions of survivorship and practice standardization for BM patients. Methods: Here, we present results from a cross-sectional survey in which we analyzed responses from 237 BM patients, 209 caregivers, and 239 physicians. Surveys contained questions about BM symptoms, discussion of BM diagnosis by the clinician, psychosocial concerns, available treatment options for BM, BM patient advocacy resources, and BM-specific clinical trials. Results: Our survey revealed compelling findings about current care of BM patients. There were discrepancies in the perceived discussion of the implications of the diagnosis of BM, from the patient/caregiver and physician perspective. Important topics, such as prognosis and worrisome symptoms, were felt to have been discussed more frequently by physicians than by patients or caregivers. In our physician survey, private practice physicians, compared to academic physicians, were significantly more likely to recommend whole brain radiotherapy (61.1 vs 39.7%; p = 0.009). Participation in a clinical trial was one of the least recommended treatment options. Many physicians (59.1% private; 71.9% academic) stated that BM patients in their care are denied participation in a clinical trial, specifically due to the presence of BM. The consensus among physicians, patients and caregivers was that the highest yield area for federal assistance is increased treatment and research funding for BM. Conclusions: Our hope is that these findings will serve as a basis for future quality improvement measures to enhance patient-physician communication and patient well-being, continuing medical education activities detailing latest advances in BM for oncologists, and lobbying efforts to the federal government in prioritizing BM research, clinical trials, and patient survivorship.

Clinical Trial Design and Statistics

2018 ◽  
Author(s):  
Julie Ann Sosa
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Data Analysis ◽  
Trial Design ◽  
New Technologies ◽  
Drug Application ◽  
Clinical Trial Design ◽  
Double Blind Study ◽  
Type I ◽  
End Points

A clinical trial is a planned experiment designed to prospectively measure the efficacy or effectiveness of an intervention by comparing outcomes in a group of subjects treated with the test intervention with those observed in one or more comparable group(s) of subjects receiving another intervention.  Historically, the gold standard for a clinical trial has been a prospective, randomized, double-blind study, but it is sometimes impractical or unethical to conduct such in clinical medicine and surgery. Conventional outcomes have traditionally been clinical end points; with the rise of new technologies, however, they are increasingly being supplemented and/or replaced by surrogate end points, such as serum biomarkers. Because patients are involved, safety considerations and ethical principles must be incorporated into all phases of clinical trial design, conduct, data analysis, and presentation. This review covers the history of clinical trials, clinical trial phases, ethical issues, implementing the study, basic biostatistics for data analysis, and other resources. Figures show drug development and clinical trial process, and type I and II error. Tables list Food and Drug Administration new drug application types, and types of missing data in clinical trials. This review contains 2 highly rendered figures, 2 tables, and 38 references

Factors affecting the offering and enrollment of patients on clinical trials in radiation oncology.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. e17571-e17571
Author(s):  
Shayna Eliana Rich ◽  
Nancy Price Mendenhall
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
General Population ◽  
Proton Therapy ◽  
New Technologies ◽  
Patient Acceptance ◽  
Proton Radiation ◽  
Available N ◽  
Treatment Approaches ◽  
Factors Affecting

e17571 Background: Improvements in cancer treatment require significant patient involvement in research, which may be particularly limited for new technologies such as proton radiation therapy. Studies with biased referrals or enrollment may not be generalizable to a general population. This study examines the reasons why patients were not offered or refused enrollment in clinical trials at the University of Florida Proton Therapy Institute (UFPTI). Methods: All patients seen at UFPTI between April-October 2012 for proton therapy for tumor sites with a clinical trial available (N=463) had information collected prospectively regarding whether they were offered enrollment and consented for clinical trials, and the reasons for each decision. The majority of patients had already secured funding for proton therapy. Results: Seven percent (34/463) of patients were ineligible for an available clinical trial, due to study exclusion criteria, concerns for patient safety based on comorbidity, or concerns for data integrity (e.g., other non-skin cancer within five years). Only 3% (9/275) of eligible patients were not offered a clinical trial. Forty-four percent (99/226) of patients offered a clinical trial refused. The most common reasons for refusal included: discomfort with lack of mature data, dislike of protocol, fear that protocol is not best option for disease control, and fear of side effects. Although UFPTI treats a variety of malignancies, the overwhelming majority of those who refused consent were prostate adenocarcinoma patients, who often self-referred for proton therapy. Conclusions: Despite near universal availability of clinical trials at UFPTI, less than half of patients enroll in clinical trials. The greatest factor for non-enrollment appears to be patient acceptance. Despite the availability of non-randomized trials with fairly standard treatment approaches, 44% refused to enroll on clinical trials, suggesting discomfort for less well documented treatment approaches. Further studies should examine whether findings are similar among all US cancer patients, as patients seeking proton therapy may not resemble the general population.

scholarly journals Clinical Trial Information As a Measure of Quality Cancer Care

2010 ◽  
Vol 6 (3) ◽  
pp. 170-171 ◽  
Author(s):  
Wei Chua ◽  
Stephen J. Clarke
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Cancer Care ◽  
Treatment Options ◽  
Small Cell ◽  
Small Cell Lung ◽  
Quality Cancer Care ◽  
Improved Outcomes ◽  
New Treatment ◽  
Clinical Trial Information

Participation in clinical trials enables patients to access new treatment options. Evidence shows improved outcomes in participants compared with nonparticipants in non–small-cell, lung, breast, colorectal, and testicular cancers.

scholarly journals INTELLECTUAL PROPERTY ON DATA OF COVID-19 OFF-LABEL TREATMENT AND DATA OF COMPASSIONATE USE OF MEDICINES AND ACCESS TO TREATMENT IN A PANDEMIC

2020 ◽  
Author(s):  
Оксана Кашинцева ◽  
Микита Трохименко
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Intellectual Property ◽  
Treatment Options ◽  
Legal Protection ◽  
New Drugs ◽  
European Medicines Agency ◽  
Compassionate Use ◽  
Access To Treatment ◽  
Off Label

The article concerns the issues of legal protection of data obtained as a result of off-label drugs therapy of COVID-19 and of data obtained of the compassionate use of medicines in treatment of COVID-19. The authorsargue that neither data on the use of off-label or on the basis of a compassionate use in the treatment of coronavirus (data obtained in solidarity clinical trials) are not determined as the information that should be protected from unfair commercial use.Regarding to the use of off-label for a new purpose of drag it is not be considered as a «new chemical substance», because the drug is already registered, and therefore known, and in the case of obtaining data in compassionate use, this information is removed from the trade secret regime by the WHO and EMA opinion. Such information is opened to use from the beginning. Therefore, in both of the above cases, it could notbe considered as an «unfair use» in the meaning of Art. 39 TRIPS Agreements.The WHO apply to the world community and informed about the «Solidarity» clinical trial for COVID-19 treatments». Solidified clinical trials of COVID-19 treatment compare four treatment options to evaluate their efficacy in COVID-19 therapy. Solidarity-based clinical trials aim to quickly identify which of the drugs tested slows disease progression or improves survival. New drugs can be added to solidarity studiesbased on new data.On April 3, 2020, the Committee for Medicinal Products for Human Use of European Medicines Agency (EMA) issued recommendations for compassionate use for remdesivir as the most promising treatment for COVID-19. The EMA explicitly states that the compassionate use is not part of the clinical trial in its usual meanings.IFLA (International Federation of Library Associations and Institutions) also wrote an open letter to WIPO urging WIPO to use all available flexible intellectual property mechanisms to maximize worldwide access to information (research data) on COVID-19 treatment.Thus, the legal regime of «Solidarity clinical trials and the WHO and EMA declarations lead us to conclude that all data obtained in the Solidarity clinical trials should not be monopolized by intellectual property, either as objects of patenting for a new scope / new purpose of treatment, or like an object of data exclusivity protection.

Clinical Trial Design and Statistics

2016 ◽  
Author(s):  
Julie Ann Sosa ◽  
Samantha M. Thomas ◽  
April K.S. Salama
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Data Analysis ◽  
Trial Design ◽  
New Technologies ◽  
Drug Application ◽  
Clinical Trial Design ◽  
Double Blind Study ◽  
Type I ◽  
End Points

A clinical trial is a planned experiment designed to prospectively measure the efficacy or effectiveness of an intervention by comparing outcomes in a group of subjects treated with the test intervention with those observed in one or more comparable group(s) of subjects receiving another intervention.  Historically, the gold standard for a clinical trial has been a prospective, randomized, double-blind study, but it is sometimes impractical or unethical to conduct such in clinical medicine and surgery. Conventional outcomes have traditionally been clinical end points; with the rise of new technologies, however, they are increasingly being supplemented and/or replaced by surrogate end points, such as serum biomarkers. Because patients are involved, safety considerations and ethical principles must be incorporated into all phases of clinical trial design, conduct, data analysis, and presentation. This review covers the history of clinical trials, clinical trial phases, ethical issues, implementing the study, basic biostatistics for data analysis, and other resources. Figures show drug development and clinical trial process, and type I and II error. Tables list Food and Drug Administration new drug application types, and types of missing data in clinical trials. This review contains 2 highly rendered figures, 2 tables, and 38 references

The impact of the COVID-19 pandemic on oncology clinical trial recruitment in an Irish cancer center.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18756-e18756
Author(s):  
Ronan Andrew McLaughlin ◽  
Valerie Madigan ◽  
Maureen O'Grady ◽  
Thamir Andrew Mahgoub ◽  
Roshni Andrew Kalachand ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Informed Consent ◽  
Treatment Options ◽  
Cancer Clinical Trial ◽  
University Hospital ◽  
Cancer Center ◽  
Cancer Clinical Trials ◽  
Number Of Patients ◽  
The Impact

e18756 Background: The COVID-19 pandemic has created unprecedented disruptions to cancer clinical trial research across the world due to a temporary global suspension of patients’ recruitment to cancer clinical trials. Access to clinical trials permits better treatment options and best clinical practice standards for patients with cancer. We present the impact of the COVID-19 pandemic on cancer clinical trial activity at the Cancer Clinical Trials Unit (CCTU) at the Mid-Western Cancer Centre, University Hospital Limerick (UHL). Over the last 4 years 28 clinical trials, both interventional and translational, have opened here, across a variety of primary disease sites, with 5 trials opened in 2017, 11 in 2018, 7 in 2019 but only 2 in the first 10 months of 2020 until 3 further trials were opened in December. Methods: CCTU records were reviewed to identify the number of patients screened and consented to participate in cancer clinical trials at UHL in 2020, which were compared directly with corresponding numbers for 2019. Results: In 2019, 17 clinical trials were open and recruiting at the CCTU, UHL. During 2020, 19 trials were recruiting although during the 1st surge of the COVID-19 pandemic recruitment was essentially suspended and CCTU staff were redeployed throughout the hospital. 1st Six months 2020 vs 2019 In the six months from January 2020 until the end of June 2020, 99 patients were screened and only 15 (15.2%) signed informed consent to participate in a cancer clinical trial. When these figures are directly compared with the first six months of 2019, there is a 33% reduction in patients screened for participation (147 vs 99) and a 60% reduction in patients consented (37 vs 15) to clinical trials. 12 Months 2020 vs 2019 In total during 2019, 376 patients were screened for inclusion to participate and 49 (13%) patients signed informed consent to participate in a clinical trial within CCTU at UHL. In 2020, 914 patients were screened for participation with 51 patients consented to participate (5.6%). The majority (45/51 (88%)) of patients consented to cancer clinical trials in 2020 at the CCTU, UHL were recruited to translational based studies and only 6 (12%) consented to interventional studies compared with 2019 when 30/49 (61%) consented to translational and 30/49 (39%) to interventional studies. Conclusions: During the COVID-19 pandemic, the percentage of patients consented to participation in a clinical trial reduced significantly, as compared to the previous year (5.6% vs 13%). Fewer interventional studies have recruited patients during 2020. As we enter the third surge of COVID-19 infections in Ireland, we must continue to monitor and identify effective strategies to navigate the ever-changing situation for cancer clinical trials, in an attempt to maintain access to high quality cancer clinical trial opportunities for our patients.

Clinical Trial Design and Statistics

2018 ◽  
Author(s):  
Julie Ann Sosa ◽  
Samantha M. Thomas ◽  
April K.S. Salama
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Data Analysis ◽  
Trial Design ◽  
New Technologies ◽  
Drug Application ◽  
Clinical Trial Design ◽  
Double Blind Study ◽  
Type I ◽  
End Points

A clinical trial is a planned experiment designed to prospectively measure the efficacy or effectiveness of an intervention by comparing outcomes in a group of subjects treated with the test intervention with those observed in one or more comparable group(s) of subjects receiving another intervention.  Historically, the gold standard for a clinical trial has been a prospective, randomized, double-blind study, but it is sometimes impractical or unethical to conduct such in clinical medicine and surgery. Conventional outcomes have traditionally been clinical end points; with the rise of new technologies, however, they are increasingly being supplemented and/or replaced by surrogate end points, such as serum biomarkers. Because patients are involved, safety considerations and ethical principles must be incorporated into all phases of clinical trial design, conduct, data analysis, and presentation. This review covers the history of clinical trials, clinical trial phases, ethical issues, implementing the study, basic biostatistics for data analysis, and other resources. Figures show drug development and clinical trial process, and type I and II error. Tables list Food and Drug Administration new drug application types, and types of missing data in clinical trials. This review contains 2 highly rendered figures, 2 tables, and 38 references

How we treat higher-risk myelodysplastic syndromes

Blood ◽  
2014 ◽  
Vol 123 (6) ◽  
pp. 829-836 ◽  
Author(s):  
Mikkael A. Sekeres ◽  
Corey Cutler
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Myelodysplastic Syndromes ◽  
Risk Group ◽  
Treatment Options ◽  
International Prognostic Scoring System ◽  
Hypomethylating Agents ◽  
Hematopoietic Stem ◽  
Patient Goals ◽  
Interim Period

Abstract Higher-risk myelodysplastic syndromes (MDS) are defined by patients who fall into higher-risk group categories in the original or revised International Prognostic Scoring System. Survival for these patients is dismal, and treatment should be initiated rapidly. Standard therapies include the hypomethylating agents azacitidine and decitabine, which should be administered for a minimum of 6 cycles, and continued for as long as a patient is responding. Once a drug fails in one of these patients, further treatment options are limited, median survival is <6 months, and consideration should be given to clinical trials. Higher-risk eligible patients should be offered consultation to discuss hematopoietic stem cell transplantation close to the time of diagnosis, depending on patient goals of therapy, with consideration given to proceeding to transplantation soon after an optimal donor is located. In the interim period before transplantation, hypomethylating agent therapy, induction chemotherapy, or enrollment in a clinical trial should be considered to prevent disease progression, although the optimal pretransplantation therapy is unknown.

scholarly journals Advance of Novel Coronavirus Registration Clinical Trial

2020 ◽  
Author(s):  
Gao Song ◽  
Meng Qun Cheng ◽  
Xian Wen Wei
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Treatment Options ◽  
Descriptive Analysis ◽  
Clinical Trial Registry ◽  
Chi Square ◽  
Plasma Therapy ◽  
Study Duration ◽  
Design Characteristics ◽  
Novel Coronavirus

AbstractBackgroundTo analyze the characteristics and heterogeneity of clinical trials of Novel Coronavirus(COVID-19) registered in the China Clinical Trial Registry (ChiCTR), and provide data bases and information references for clinical treatmentMethodsStatistics of COVID-19 clinical trials registered with ChiCTR as of February 24, 2020 were collected. Descriptive analysis of registration characteristics. The chi-square test is used to compare statistical differences between different study types, intervention methods, study stage, and Primary sponsor.Results232 COVID-19 studies registered at the ChiCTR were collected. The overall number of COVID-19 registrations was increased. Hubei Province, China has the largest number of registrations. There were significant differences between the number of participants(P=0.000), study duration(P=0.008), study assignment(P=0.000), and blind method(P=0.000) for different study types. Significant differences could be seen in the dimensions of multicenter study(P=0.022), of participants numbe(P=0.000), study duration(P=0.000) and study assignment(P=0.001) for the four intervention methods. There were significant differences in study assignment(P=0.043) between the early and late studies. CMT drugs with high research frequency are chloroquine, lopinavir / ritonavir, and I-IFN; BI was Cell therapy, plasma therapy, Thymosin, and M/P-AB.ConclusionsDifferent study design characteristics have led to significant differences in some aspects of the COVID-19 clinical trial. Timely summary analysis can provide more treatment options and evidence for clinical practice.

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