Results for 'Clinical benefits'
Tom Caravela
Promotability: How to Land Your Next Promotion
professional visibility, career advancement, promotion tips, career progression, networking strategies, mentorship benefits, resume upgrades, servant leadership, problem-solving skills, personal branding
“Promotability” may be defined as one’s suitability for being promoted. Is it your goal to obtain a promotion this year? If so, how suitable or promotable are you for that next step? Job-seekers and professionals often ask me what steps are necessary for advancement. I have broken …
May 5th • 10 mins read
B. Gyawali, E. G. E. de Vries, U. Dafni, T. Amaral, J. Barriuso, J. Bogaerts, A. Calles, G. Curigliano, C. Gomez-Roca, B. Kiesewetter, S. Oosting, A. Passaro, G. Pentheroudakis, M. Piccart, F. Roitberg, J. Tabernero, N. Tarazona, D. Trapani, R. Wester, G.
Biases in study design, implementation, and data analysis that distort the appraisal of clinical benefit and ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) scoring
ESMO-MCBS, bias, clinical trial design, clinical trial implementation, clinical trial reporting, clinical trial analysis
The ESMO-MCBS is a tool used for scoring the clinical benefit of cancer medicines as reported in clinical trials, assuming valid research methodologies and quality implementation. The tool's effectiveness is compromised by studies with flawed design, implementation, or data analysis, w…
Apr 20th • 8 mins read
Jayson L. Parker, Sebnem S. Kuzulugil, Kirill Pereverzev, Stephen Mac, Gilberto Lopes, Zain Shah, Ashini Weerasinghe, Daniel Rubinger, Adam Falconi, Ayse Bener, Bora Caglayan, Rohan Tangri, Nicholas Mitsakakis
Does biomarker use in oncology improve clinical trial failure risk? A large-scale analysis
biomarker, clinical trial, failure, analysis, NSCLC, oncology
Data from clinical trials across four oncology indications (breast cancer, NSCLC, melanoma, and colorectal cancer) from 1998 to 2017 were analyzed to assess drug approval likelihood based on biomarker status. Using multi-state Markov models, which describe stochastic processes, the study…
Feb 23rd • 8 mins read
Fei Liang, Sheng Zhang, Qin Wang, Wenfeng Li
Clinical benefit of immune checkpoint inhibitors approved by US Food and Drug Administration
Randomized trials, Clinical benefits, Immune checkpoint inhibitors, Cancer, Food and drug administration agency
The study evaluates the clinical benefits of immune checkpoint inhibitors using the ESMO-MCBS and ASCO VF scales. 18 indications for these inhibitors were approved based on RCTs conducted between 2011 and 2018. All indications meet the ESMO-MCBS 1.1 threshold for meaningful bene…
Aug 31st • 16 mins read
Consolación Molto MD, Thomas J. Hwang AB, Maria Borrell MD, Marta Andres MD, Ignasi Gich MD, PhD, Agustí Barnadas MD, PhD, Eitan Amir MD, PhD, Aaron S. Kesselheim MD, JD, MPH, Ariadna Tibau MD, PhD
Clinical benefit and cost of breakthrough cancer drugs approved by the US Food and Drug Administration
USFDA, ESMO-MCBS, NCCN, ASCO-CRC, clinical, drug aroval
The study evaluates the clinical benefit and pricing of breakthrough-designated versus non-breakthrough-designated cancer drugs. The analysis covers approvals from July 2012 to December 2017, using frameworks like ASCO-VF, ASCO-CRC, ESMO-MCBS, and NCCN Evidence Blocks. High clinical benef…
Jul 22nd • 12 mins read
Nicole Grossmann, Martin Robausch, Eleen Rothschedl, Claudia Wild, Judit Simon
Publicly accessible evidence of health-related quality of life benefits associated with cancer drugs approved by the European Medicines Agency between 2009 and 2015
Antineoplastic agents, Health-related quality of life, Clinical efficacy, Drug approvals, Patient-relevant outcomes
The study investigates cancer drugs approved by the European Medicines Agency (EMA) that initially lack Health-related Quality of Life (HRQoL) information. Data was collected for cancer indications approved between January 2009 and October 2015, using sources like the EMA website, clinical…
Feb 23rd • 12 mins read
Barbara Kiesewetter , Nathan I Cherny, Nicolas Boissel, Francesco Cerisoli, Urania Dafni, Elisabeth G E de Vries, Paolo Ghia, Nicola Gökbuget, Verónica González-Calle, Brian Huntly, Ulrich Jäger, Nicola Jane Latino, Jean-Yves Douillard, Luca Malcovati, Ma
EHA evaluation of the ESMO-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for hematological malignancies
ESMO, hematological malignancies, Clinical benefit scale, EHA
The ESMO-MCBS v1.1 has not been previously validated for haematological malignancies, but it is being explored to avoid duplication of efforts. A feasibility test was conducted using 80 studies related to various haematological malignancies such as acute and chronic leukaemia, lymphoma, myel…
Jan 20th • 20 mins read
Consolacion Molto, Ariadna Tibau, Aida Bujosa, Jose Carlos Tapia, Abhenil Mittal, Faris Tamimi & Eitan Amir
Association between control group therapy and magnitude of clinical benefit of cancer drugs
control group therapy, clinical benefit scales, ESMO-MCBS, ASCO-VF, randomized trials
The study investigated the impact of control group therapy on various clinical benefit scales like ASCO-VF, ESMO-MCBS, NCCN Evidence Blocks, and ASCO-CRC. Researchers analyzed cancer drugs approved between 2012 and 2021 using data from randomized controlled trials (RCTs) listed on Drugs@FDA. Sig…
Dec 9th • 20 mins read
Envision Pharma Group
Patient involvement: A must-have in medicine development, but is it being overlooked in a cost-constrained environment?
patient engagement in pharma, patient-centric drug development, life sciences industry innovation, patient insights in medicine, regulatory compliance in patient engagement, patient involvement in clinical trials, benefits of patient advocacy, patient-cen
The life sciences industry is in a constant state of advancement, bringing more and more groundbreaking medicines, cutting-edge technologies, and innovative solutions to market. Amidst these rapid changes, patients remain at the heart of these scientific developments. In recent decades, the l…
May 9th • 5 mins read
Ravi B. Parikh, MD, MPP Rebecca A. Hubbard, PhD Erkuan Wang, MA Trevor J. Royce, MD, MPH Aaron B. Cohen, MD, MSCE Amy S. Clark, MD, MSCE Ronac Mamtani, MD, MSCE
Exposure to US Cancer Drugs With Lack of Confirmed Benefit After US Food and Drug Administration Accelerated Approval
US, FDA, approval, drugs, benefits, cancer
Among 5 oncology indications, 26.1% of eligible treatment initiations involved an Accelerated Approval (AA) indication that was later withdrawn due to lack of benefit. There is an inherent trade-off between expediting access to promising cancer drugs and the potential withdrawal of some indicatio…
Dec 8th • 2 mins read
Robbe Saesen, Mieke Van Hemelrijck, Jan Bogaerts, Christopher M. Booth, Jan J. Cornelissen, Andre Dekker, Elizabeth A. Eisenhauer, André Freitas, Alessandro Gronchi, Miguel A. Hernán, Frank Hulstaert, Piet Ost, Petr Szturz, Helena M. Verkooijen, Michael W
Defining the role of real-world data in cancer clinical research: The position of the
real-world data, real-world evidence, randomised, pragmatic trials, cancer, clinical research, add-on cohorts
Real-World Data (RWD) are increasingly used in cancer clinical research as an alternative evidence source. RWD studies primarily involve observational data, which offer insights into real-world conditions but may have quality limitations. Some research questions necessitate Randomized Controlled…
Mar 20th • 10 mins read
Bishal Gyawali, Benjamin N Rome, Aaron S Kesselheim
Regulatory and clinical consequences of negative confirmatory trials of accelerated approval cancer drugs: retrospective observational study
clinical guidelines, FDA, accelerated approval cancer drugs, surrogate measures, NCCN, EMA
Six of 18 cancer drugs that initially received accelerated approval have indications that remain on the labeling and are recommended in clinical guidelines despite no improvement in the primary endpoint in post-approval trials. These findings reflect the lack of fulfillment of the compromise between…
Aug 4th • 12 mins read
Aviv Ladanie, PhD, Andreas M. Schmitt, MD, Benjamin Speich, PhD, Florian Naudet, PhD, Arnav Agarwal, MD, Tiago V. Pereira, PhD, Francesco Sclafani, PhD, Amanda K. Herbrand, MD, Matthias Briel, MD, MSc, Juan Martin-Liberal, PhD, Thomas Schmid, MD, Hannah E
Clinical Trial Evidence Supporting US Food and Drug Administration Approval of Novel Cancer Therapies Between 2000 and 2016
cancer treatment outcomes, outcomes, drug approval, clinical data, FDA
Data from 17 years of studies on novel cancer drugs show limited information available at market entry. Randomized Controlled Trials (RCTs) data are available for only half of the drug indications. Lack of data is particularly problematic for patients with hematological cancers. Novel therapies…
Nov 10th • 6 mins read
Pan Pantziarka, Ciska Verbaanderd & Lydie Meheus
Biased by design? Clinical trials and patient benefit in oncology
clinical trial design, drug development, drug registration, patient benefit, risks of bias
The study by Naci et al. raises numerous questions relating to the design characteristics, risks of bias and reporting of pivotal clinical trials in oncology. These findings add to the existing body of evidence that suggests drug development in oncology is not delivering the clinical benef…
Nov 27th • 3 mins read
Dominic H. Moon, Ronald C. Chen
Defining a Clinically Meaningful Benefit in Cancer Clinical Trials: From the Perspectives of the Clinical Trialist, Patient, and Society
cancer clinical trials, benefit of clinical trials, clinically meaningful, ASCO
In value-based medicine, clinical trials must show both statistically significant and clinically meaningful improvements, such as ASCO's recommendation of a 20% relative improvement in overall survival for metastatic solid tumors. Lawrence et al. found that while many phase III trials met statistica…
Jun 27th • 2 mins read
Ariadna Tibau, Consolación Molto, Alberto Ocana, Arnoud J Templeton, Luis P Del Carpio, Joseph C Del Paggio, Agustí Barnadas, Christopher M Booth, Eitan Amir
Magnitude of Clinical Benefit of Cancer Drugs Approved by the US Food and Drug Administration
antineoplastic agents, immunologic adjuvants, pharmaceutical adjuvants, phase 3 clinical trials, drug approval, drug labeling, medical oncology, united states food and drug administration, diagnosis, palliative care, surrogate endpoints, weight measureme
Regulatory agencies assess drug safety and efficacy, but thresholds may differ from those accepted by clinicians . Only 43.8% of RCTs for FDA-approved drugs meet the ESMO-MCBS threshold for meaningful benefit, reflecting potential softening of FDA standards. Encouraging trends include an increas…
Dec 13th • 7 mins read
Louis Everest, BSc; Monica Shah, BSc; Kelvin K.W. Chan, MD, MSc, PhD
Comparison of Long-term Survival Benefits in Trials of Immune Checkpoint Inhibitor vs Non-Immune Checkpoint Inhibitor Anticancer Agents Using ASCO Value Framework and ESMO Magnitude of Clinical Benefit Scale
immunotherapy-triggered LTP, RCTs, FDA, ICI
Importance: Anticancer agents, especially immune checkpoint inhibitors (ICIs), have shown potential for long-term durable survival in some patients. However, traditional clinical benefit measures may not accurately capture this, leading to proposed amendments in valuation frameworks. Objectives: …
Jul 10th • 12 mins read
Yichen Zhang, MSc; Huseyin Naci, PhD; Anita K. Wagner, PharmD, DrPH
Overall Survival Benefits of Cancer Drugs Approved in China From 2005 to 2020
cancer drug therapy, overall survival, FDA approvals, surrogate endpoints, EMA approvals, quality of life
The study evaluates the overall survival (OS) benefit of cancer drugs approved in China between 2005 and 2020. Out of 141 cancer drug indications approved, 68 showed a documented overall survival benefit, while 34 did not prolong life. Recent regulatory reforms in China aim to improve the availa…
Aug 10th • 30 mins read
S.F. Oosting, J. Barriuso, A. Bottomley, M. Galotti, B. Gyawali, B. Kiesewetter, N.J. Latino, F. Martinelli, M. Pe, G. Pentheroudakis, F. Roitberg, H. Vachon, E.G.E. de Vries, M. Piccart, N.I. Cherny
Methodological and reporting standards for quality-of-life data eligible for European Society for Medical Oncology-Magnitude of Clinical Benefit Scale
Benefit Scale, MCBS, Quality of Life, Health Assessment, Oncology Scale, Cancer Guidelines, Treatment Evaluation, Medicine Scoring
The ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) was introduced in 2015 to standardize the assessment of clinical benefits from cancer treatments. ESMO-MCBS helps in health-technology assessment, especially given the increasing number of treatment options and rising cancer care costs. Ne…
Apr 1st • 30 mins read
Shen Lin, Yaping Huang, Liangliang Dong, Meiyue Li, Yahong Wang, Dian Gu, Wei Wu, Dongni Nian, Shaohong Luo, Xiaoting Huang, Xiongwei Xu and Xiuhua Weng
The correlation between the costs and clinical benefits of PD-1/PD-L1 inhibitors in malignant tumors: An evaluation based on ASCO and ESMO frameworks
PD-1/PD-L1 inhibitors, malignant tumors, ASCO, ESMO, Immune Checkpoint Inhibitors, Gastrointestinal Cancers
Cancer drug innovation has significantly accelerated in the 21st century, with novel drug approvals and expenditures increasing notably. Assessment frameworks ASCO-VF and ESMO-MCBS were used to evaluate the clinical benefit of PD-1/PD-L1 inhibitors, finding that nearly half of the trials met "mea…
Feb 23rd • 9 mins read