Results for 'Surrogate End Points'
Idine Mousavi, BA; Timothée Olivier, MD; Vinay Prasad, MD, MPH
Cost per Event Averted in Cancer Trials in the Adjuvant Setting From 2018 to 2022
anticancer drugs, adjuvant therapy, FDA approvals, cost per event averted, surrogate endpoints, oncology drug costs
Importance: Evaluating the cost-effectiveness of adjuvant therapies based on the cost per event averted. Objective: To assess the costs per event averted for anticancer drugs approved by the FDA between January 2018 and March 2022. Design, Setting, and Participants: Cross-sectional study of F…
Jun 10th • 30 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
Rachel E. Sachs, JD, MPH, Kyle A. Gavulic, BA, Julie M. Donohue, PhD
Recent Trends in Medicaid Spending and Use of Drugs With US Food and Drug Administration Accelerated Approval
FDA accelerated approval, Medicaid spending, prescription drug costs, surrogate endpoints, cancer drugs, drug approval pathway
The study examines the impact of drugs approved through the FDA’s accelerated approval program on state Medicaid spending. From 1992 to 2020, 216 drug-indication pairs were granted accelerated approval, with a significant increase in cancer drug approvals in recent years. Although drugs wi…
Oct 8th • 25 mins read
Anushka Walia, Alyson Haslam, Vinay Prasad
FDA validation of surrogate endpoints in oncology: 2005–2022
FDA drug approval, surrogate endpoints, oncology drugs, overall survival correlation, clinical trials analysis, drug approval guidelines
ere are summary bullets based on the provided text: The FDA has increasingly approved oncologic drugs based on surrogate endpoints, which often lack a demonstrated correlation with clinically meaningful outcomes like overall survival. A review of FDA analyses from 2005 to 2022 examined the valid…
Dec 1st • 20 mins read
Mahnum Shahzad, BA, Huseyin Naci, MHS, PhD, Anita K. Wagner, PharmD, MPH, DrPH
Estimated Medicare Spending on Cancer Drug Indications With a Confirmed Lack of Clinical Benefit After US Food and Drug Administration Accelerated
medicare spending, drug indications, AA, FDA, medicare, ODAC
Medicare Parts B and D spent at least $569 million between 2017 and 2019 on 10 cancer drug indications without confirmed overall survival (OS) benefit after accelerated approval (AA). Approximately $224 million was spent on drug indications that were either voluntarily withdrawn or recommended fo…
Oct 18th • 5 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
Meena N Murugappan, PharmD, MPH, PhD(c), Bellinda L King-Kallimanis, PhD, Gregory H Reaman, MD, Vishal Bhatnagar, MD, Erica G Horodniceanu, MPH, Najat Bouchkouj, MD, Paul G Kluetz, MD
Patient-Reported Outcomes in Pediatric Cancer Registration Trials: A US Food and Drug Administration Perspective
cancer, pediatrics, product labeling, united states food and drug administration, childhood cancer, denosumab, surrogate endpoints, adverse event, patient self-report, pediatric oncology, selumetinib, benefit-risk assessment, tisagenlecleucel, statistica
PROs have been feasible to collect from patients as young as 7 years old and were included in trials from 2013 onward. PROs were used as exploratory endpoints in four product applications but not included in product labeling. To include PRO data in FDA labeling, early interaction with the FDA, c…
Apr 30th • 12 mins read
Emerson Y. Chen, MD, Alyson Haslam, PhD, Vinay Prasad, MD
FDA Acceptance of Surrogate End Points for Cancer Drug Approval: 1992-2019
FDA, Surrogate End Points, Cancer drug approval
194 approvals were analyzed, with 64 (32.9%) relying on surrogate endpoints for the first time in a specific cancer type. Surrogate endpoints often have weak or unknown correlations to overall survival (OS). Many approvals lack strong scientific or regulatory justification, indicating a higher t…
Mar 9th • 4 mins read
Jiaxi Zhou, Jonathon Vallejo, Paul Kluetz, Richard Pazdur, Tamy Kim, Patricia Keegan, Ann Farrell, Julia A Beaver, Rajeshwari Sridhara
Overview of Oncology and Hematology Drug Approvals at US Food and Drug Administration Between 2008 and 2016
drug approval, medical oncology, united states food and drug administration, hematology, surrogate endpoints, marketing, accelerated approval
Background: An overview was conducted of oncology products reviewed by the Office of Oncology Drug Products (OODP) at the FDA for marketing approval between July 2005 and December 2007. The study aims to understand the impact of post-2007 changes in laws, regulations, and organizational structure …
Aug 4th • 15 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
Emerson Y. Chen, MD, Sunil K. Joshi, BA, Audrey Tran, BA
Estimation of Study Time Reduction Using Surrogate End Points Rather Than Overall Survival in Oncology Clinical Trials
bevacizumab, metastatic breast cancer, RR, PFS, FDA, oncology clinical trials
The use of Response Rate (RR), Progression-Free Survival (PFS), and Overall Survival (OS) in clinical trials leading to FDA approval is associated with different study durations: RR: Median study duration of 25 months (range, 11-54 months). PFS: Median study duration of 31 months (range, 10-…
Apr 1st • 10 mins read
Shubham Sharma, J Connor Wells, Wilma M Hopman, Joseph C Del Paggio, Bishal Gyawali, Nazik Hammad, Annette E Hay, Christopher M Booth
Cancer, Clinical Trials, and Canada: Our Contribution to Worldwide Randomized Controlled Trials
cancer, clinical trials, research funding, Canada, high-income countries
This document reviews Canadian involvement in oncology randomized controlled trials (RCTs) from 2014-2017, comparing them to those from other high-income countries (HICs). Canada contributed to 155 (24%) of 636 HIC-led RCTs. Canadian RCTs focused more on palliative care (72%) compared to 62% in …
Apr 13th • 10 mins read
Avi Cherla, MSc; Huseyin Naci, MHS, PhD; Aaron S. Kesselheim, MD, JD, MPH; Bishal Gyawali, MD, PhD; Elias Mossialos, MD, PhD
Assessment of Coverage in England of Cancer Drugs Qualifying for US Food and Drug Administration Accelerated Approval
FDA, NHS, accelerated approval, NICE, clinical trial findings
The study evaluates the acceptance and coverage of FDA-accelerated approved cancer drugs by the National Health Service (NHS) in England, focusing on decisions made by the National Institute for Health and Care Excellence (NICE). A total of 93 cancer drug indications received accelerated approval…
Feb 22nd • 10 mins read
Vanessa Arciero, BS, Seanthel Delos Santos, Liza Koshy
Assessment of Food and Drug Administration- and European Medicines Agency-Approved Systemic Oncology Therapies and Clinically Meaningful Improvements in Quality of Life: A Systematic Review
ESMO, MCBS, FDA, ASCO-VF, EMA, QOL
Recent oncology therapies approved by the FDA and EMA often lack evidence of clinically meaningful improvements in quality of life (QOL). Only 40% of FDA-approved and 58% of EMA-approved oncology therapies had published QOL evidence. Clinically meaningful QOL improvements beyond minimal dif…
Feb 11th • 4 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
Audrey D. Zhang, Jeremy Puthumana, Nicholas S. Downing, MD
Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017
clinical trials, FDA, Novel therapeutic agents, biologics
The number of special regulatory programs for drug and biologic approvals by the FDA has increased since the introduction of the Fast Track designation in 1988. This study analyzed 273 new drugs and biologics approved by the FDA for 339 indications across three periods: 1995-1997, 2005-2007,…
Apr 21st • 20 mins read
Miloš D. Miljković, MD, MSc, Jordan E. Tuia, BA, Timothée Olivier, MD
Association Between US Drug Price and Measures of Efficacy for Oncology Drugs Approved by the US Food and Drug Administration From 2015 to 2020
Cancer drug pricing, Cancer care costs, Cancer drug efficacy, Progression-free survival, Value-based pricing in oncology, FDA anticancer approvals
The US has worse cancer-related outcomes compared to other high-income countries and has the highest cost of cancer care globally. High costs may be attributed to the improved efficacy of expensive new cancer drugs, though the relationship between cost and benefit is debated. A study found a lin…
Oct 31st • 10 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
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
Bishal Gyawali, Benjamin N Rome, Aaron S Kesselheim
Report from American Society of Clinical Oncology Symposium 2020 and American Society of Clinical Oncology Gastrointestinal Cancer Symposium 2021
ASCO, TNT, Gastrointestinal Cancer Symposium
Six out of 18 cancer drugs with accelerated approval still have indications on labeling and are recommended in guidelines despite not showing improvement in primary endpoints in post-approval trials. This issue highlights the imbalance between speed and evidence in the accelerated approval pathwa…
Aug 4th • 13 mins read