Letter
Medicine, General & Internal
Guneet S. Janda, Joshua D. Wallach, Meera M. Dhodapkar, Reshma Ramachandran, Joseph S. Ross
Summary: This study evaluates the clinical trial support for supplemental new drug applications and supplemental biologics license applications from 2017 to 2019.
JAMA INTERNAL MEDICINE
(2023)
Review
Oncology
Xue Lin, Shiowjen Lee, Poornima Sharma, Bindu George, John Scott
Summary: The approval of autologous CAR T-cell therapies in 2017 has been an important milestone in the field of oncology. These therapies have shown breakthroughs in treating previously incurable diseases, and their unique manufacturing process for each patient presents challenges in study design and statistical analyses. This article shares experiences in statistical review and provides considerations for CAR T-cell trial design. The adoption of the estimand framework in clinical trials is also discussed for addressing nuanced issues in CAR T-cell trials.
JOURNAL OF CLINICAL ONCOLOGY
(2022)
News Item
Oncology
Mary Beth Nierengarten
Summary: This news section provides timely information for cancer readers, covering events, public policy analysis, topical issues, and personalities. This issue explores the use of single-arm studies for accelerated drug approvals, the impact of evidence-based surgical metrics on early-stage lung cancer patients, and the cost-effectiveness of lung cancer screening based on personal risk factors.
Article
Medicine, General & Internal
Daniel Feldman, Jerry Avorn, Aaron S. Kesselheim
Summary: This study found that extrapolation from pivotal trial data to FDA-approved indications is common, with extrapolation based on disease severity, disease subtype, and concomitant medication use. Such extrapolation may limit the generalizability of indications to specific prescribing decisions, highlighting the importance of postapproval monitoring.
Article
Medicine, General & Internal
Daniel S. Budnitz, Nadine Shehab, Maribeth C. Lovegrove, Andrew Geller, Jennifer N. Lind, Daniel A. Pollock
Summary: Based on data from 60 nationally representative US emergency departments from 2017 to 2019, visits attributed to medication harms were frequent, with variation in medication type, intended use, and patient age.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2021)
Article
Medicine, General & Internal
Mayookha Mitra-Majumdar, Simon J. Gunter, Aaron S. Kesselheim, Beatrice L. Brown, Krysten W. Joyce, Murray Ross, Catherine Pham, Jerry Avorn, Jonathan J. Darrow
Summary: This study aimed to assess the regulatory context, pivotal design characteristics, and postmarket requirements of novel drugs approved in 2020. The findings showed that drug approvals were often based on fewer, smaller, and less rigorous pivotal trials, raising concerns about their efficacy and clinical value.
Article
Dermatology
Sophia Z. Shalhout, Romi Bloom, Lynn Drake, David M. Miller
Summary: The study evaluated the fragility of clinical trial data used to support FDA approval of therapies for psoriasis and found that pivotal trials in psoriasis appear quite robust to changes in outcomes compared with randomized controlled trials for FDA approval across various diseases.
JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
(2021)
Review
Biochemistry & Molecular Biology
Beatriz G. G. de la Torre, Fernando Albericio
Summary: While the world was battling with the COVID-19 Omicron wave at the end of 2021, except for China, most countries ended 2022 with COVID-19 being treated like the flu. As a result, the U.S. Food and Drug Administration (FDA) only authorized 37 new drugs this year, compared to an average of 52 in the previous four years. This marks the second lowest number of drug approvals in the last six years, even though it may change in the future. The decrease in FDA approvals mainly applies to small molecule drugs, while biologics and TIDES have remained relatively constant.
Article
Oncology
Viktoria Gloy, Andreas M. Schmitt, Pascal Dueblin, Julian Hirt, Cathrin Axfors, Hanna Kuk, Tiago V. Pereira, Clara Locher, Laura Caquelin, Martin Walter-Claudi, Mark P. Lythgoe, Amanda Herbrand, Benjamin Kasenda, Lars G. Hemkens
Summary: We analyzed all cancer drugs approved by the FDA between 2000 and 2020 and found that 49% of drugs were approved without randomized controlled trials, and 82% had only one clinical trial. Although some drugs showed benefits in overall survival, there were limited improvements in quality of life.
INTERNATIONAL JOURNAL OF CANCER
(2023)
Letter
Medicine, General & Internal
Mark P. Lythgoe, Paul Middleton
Summary: This qualitative improvement study examines COVID-19 vaccine approvals by medicine regulatory agencies in the US, EU, and Canada, comparing regulatory review times and analyzing clinical evidence supporting authorization.
Review
Oncology
Esin Aysel Kandemir
Summary: This article summarizes the most recent clinical trial results of targeted cancer drugs approved by the FDA in 2021, reflecting the changing treatment landscape of solid malignancies. Targeted cancer treatments are becoming increasingly important in the treatment of malignant diseases.
JOURNAL OF ONCOLOGY PHARMACY PRACTICE
(2023)
Article
Medicine, General & Internal
So-Yeon Kang, Angela Liu, Gerard Anderson, G. Caleb Alexander
Summary: This study examined the use of manufacturer-sponsored coupons by patients during a treatment episode for a chronic condition. It found that coupon use mainly occurred within the first four prescription fills and was associated with market competition rather than patients' out-of-pocket costs.
Letter
Medicine, General & Internal
Jeremy Puthumana, Alexander C. Egilman, Audrey D. Zhang, Jason L. Schwartz, Joseph S. Ross
Summary: This study evaluates all novel vaccines approved by the US FDA over the past decade.
JAMA INTERNAL MEDICINE
(2021)
Article
Medicine, General & Internal
Patrick C. DeMartino, Milos D. Miljkovic, Vinay Prasad
Summary: This study aims to estimate the number of eligible cancer patients for newly approved drug-indication pairs and project the potential spending and use in the US. The findings suggest that if all eligible patients use the 2018 FDA-approved oncology drugs, there would be a $39.5 billion increase in cancer drug spending, while using the drugs in fewer than 20% of eligible patients is consistent with industry forecasts for drug spending.
JAMA INTERNAL MEDICINE
(2021)
Review
Pharmacology & Pharmacy
Derek Z. Yang, Ali Alhadab, Kourosh Parivar, Diane D. Wang, Mohamed Elmeliegy
Summary: Cancer patients with advanced hepatic impairment are often excluded from clinical trials and there is limited dose guidance for this patient population. This review examined FDA-approved oncology drugs to identify strategies and regulatory expectations for assessing the impact of hepatic impairment on drug pharmacokinetics, and proposed a decision tree for dose recommendations.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2022)
Letter
Medicine, General & Internal
Alexander C. Egilman, Audrey D. Zhang, Joshua D. Wallach, Joseph S. Ross
ANNALS OF INTERNAL MEDICINE
(2019)
Article
Ethics
Audrey D. Zhang, Joseph S. Ross
JOURNAL OF LAW MEDICINE & ETHICS
(2019)
Article
Medicine, General & Internal
Joshua D. Wallach, Kun Wang, Audrey D. Zhang, Deanna Cheng, Holly K. Grossetta Nardini, Haiqun Lin, Michael B. Bracken, Mayur Desai, Harlan M. Krumholz, Joseph S. Ross
BMJ-BRITISH MEDICAL JOURNAL
(2020)
Article
Medicine, General & Internal
Audrey D. Zhang, Jeremy Puthumana, Nicholas S. Downing, Nilay D. Shah, Harlan M. Krumholz, Joseph S. Ross
Letter
Medicine, General & Internal
Andrea MacGregor, Audrey D. Zhang, Joshua D. Wallach, Joseph S. Ross, Matthew Herder
Letter
Health Care Sciences & Services
Audrey D. Zhang, Jeremy Puthumana, Alexander C. Egilman, Jason L. Schwartz, Joseph S. Ross
JOURNAL OF GENERAL INTERNAL MEDICINE
(2022)
Article
Medicine, Research & Experimental
Joshua J. Skydel, Audrey D. Zhang, Sanket S. Dhruva, Joseph S. Ross, Joshua D. Wallach
Summary: The US Food and Drug Administration requires most new therapeutics to conduct at least one postmarketing clinical study, focusing on safety and efficacy for both approved and unapproved indications. The median number of clinical studies outlined per therapeutic has remained constant over the past decade. Increasing emphasis on faster approval and lifecycle evaluation may necessitate more postmarketing requirements and commitments to address gaps in clinical evidence for therapeutics at approval.
Letter
Medicine, General & Internal
Jeremy Puthumana, Alexander C. Egilman, Audrey D. Zhang, Jason L. Schwartz, Joseph S. Ross
Summary: This study evaluates all novel vaccines approved by the US FDA over the past decade.
JAMA INTERNAL MEDICINE
(2021)
Article
Medicine, General & Internal
Tanvee Varma, Joshua D. Wallach, Jennifer E. Miller, Dominic Schnabel, Joshua J. Skydel, Audrey D. Zhang, Michaela A. Dinan, Joseph S. Ross, Cary P. Gross
Summary: This study found that older adults and Black patients were underrepresented in postmarketing studies of novel cancer therapeutics to a similar degree that they were underrepresented in premarketing studies, suggesting that postmarketing studies are not associated with improvements to demographic representation gaps present at the time of FDA approval.
Article
Medicine, General & Internal
Aaron S. Long, Audrey D. Zhang, Caitlin E. Meyer, Alexander C. Egilman, Joseph S. Ross, Joshua D. Wallach
Summary: Most newly marketed FDA-approved single-enantiomer drugs are infrequently directly compared with their racemic precursors, and when compared, they are uncommonly found to provide improved efficacy or safety.
Letter
Health Care Sciences & Services
Alexander Egilman, Joshua D. Wallach, Jeremy Puthumana, Audrey D. Zhang, Jason L. Schwartz, Joseph S. Ross
JOURNAL OF GENERAL INTERNAL MEDICINE
(2021)
Letter
Medicine, General & Internal
Joshua D. Wallach, Audrey D. Zhang, Joshua J. Skydel, Victoria L. Bartlett, Sanket S. Dhruva, Nilay D. Shah, Joseph S. Ross
Summary: This study aims to evaluate the feasibility of using real-world data to emulate confirmatory clinical trials for new therapeutic agents that received accelerated approval between 2009 and 2018.
Letter
Medicine, General & Internal
Audrey D. Zhang, Timothy S. Anderson
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2022)
Article
Medicine, General & Internal
Anisa Rowhani-Farid, Kyungwan Hong, Mikas Grewal, Jesse Reynolds, Audrey D. Zhang, Joshua D. Wallach, Joseph S. Ross
Summary: This study examined the consistency of reporting study design, end points, and results of clinical trials presented at major international medical conferences in conference abstracts, published article abstracts, and press releases. The results showed that primary efficacy endpoint definitions were consistently reported, while reporting consistency rates were lower for sample sizes, follow-up periods, and effect size estimates.
BMJ EVIDENCE-BASED MEDICINE
(2023)