Editorial Material
Medicine, General & Internal
Daniel G. Aaron, I. Glenn Cohen, Eli Y. Adashi
Summary: This viewpoint discusses the controversy surrounding the FDA's efforts to withdraw Makena from the market and the broader implications for the accelerated approval pathway.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2022)
Letter
Medicine, General & Internal
Mahnum Shahzad, Huseyin Naci, Anita K. Wagner
Summary: This study investigates the relationship between the FDA's accelerated approval pathway and the speed of conversion to traditional approval or withdrawal for drugs without oncology indications.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2023)
Article
Oncology
Sakar Wahby, Lola Fashoyin-Aje, Christy L. Osgood, Joyce Cheng, Mallorie H. Fiero, Lijun Zhang, Shenghui Tang, Salaheldin S. Hamed, Pengfei Song, Rosane Charlab, Sarah E. Dorff, Tiffany K. Ricks, Kimberly Barnett-Ringgold, Jeannette Dinin, Kirsten B. Goldberg, Marc R. Theoret, Richard Pazdur, Laleh Amiri-Kordestani, Julia A. Beaver
Summary: FDA granted accelerated approval to Sacituzumab Govitecan-hziy for the treatment of metastatic triple-negative breast cancer. Data showed an objective response rate of 33.3% and a median duration of response of 7.7 months, supporting the approval.
CLINICAL CANCER RESEARCH
(2021)
Review
Health Care Sciences & Services
Tatiane Bomfim Ribeiro, Charles L. Bennett, Luis Enrique Colunga-Lozano, Ana Paula Vieira Araujo, Iztok Hozo, Benjamin Djulbegovic
Summary: This study aimed to analyze the characteristics of single-arm trials (SAT), evaluate the transparency in presenting historical control by the FDA, and assess confirmatory randomized controlled trials (RCTs). The findings showed that 47% of FDA-AA approvals were based on SAT, with 52% of treatments converting into regular approval and 46% based on confirmatory RCTs using overall survival (OS) as an outcome. Improved transparency in reporting historical controls is necessary.
JOURNAL OF CLINICAL EPIDEMIOLOGY
(2023)
Article
Oncology
Denise Casey, Suzanne Demko, Arup Sinha, Pallavi S. Mishra-Kalyani, Yuan-Li Shen, Sachia Khasar, M. Anwar Goheer, Whitney S. Helms, Lili Pan, Yuan Xu, Jianghong Fan, Ruby Leong, Jiang Liu, Yuching Yang, Katherine Windsor, Mei Ou, Olen Stephens, Byeongtaek Oh, Gregory H. Reaman, Abhilasha Nair, Stacy S. Shord, Vishal Bhatnagar, Selena R. Daniels, Sharon Sickafuse, Kirsten B. Goldberg, Marc R. Theoret, Richard Pazdur, Harpreet Singh
Summary: The FDA approved selumetinib for pediatric patients with neurofibromatosis type 1 who have inoperable plexiform neurofibromas. The approval was based on a single-arm multicenter trial showing clinical improvements and a durable overall response rate. The risks of selumetinib are consistent with MEK inhibitor class effects, with favorable benefit-risk assessment for patients with inoperable plexiform neurofibromas.
CLINICAL CANCER RESEARCH
(2021)
Article
Oncology
Cheryl Ho, Howard J. J. Lim, Dean A. A. Regier
Summary: This study evaluated the regulatory and funding decisions for FDA accelerated approved cancer therapies in Canada and found delayed access to promising new therapies in the country.
Article
Oncology
Deborah I. Bunin, Harold S. Javitz, Janet Gahagen, James Bakke, Joan H. Lane, Dina A. Andrews, Polly Y. Chang
Summary: The study evaluated the postirradiation survival and hematologic recovery of patients exposed to acute high doses of ionizing radiation following a single dose of Romiplostim with or without Pegfilgrastim. The results showed improved survival rates, reduced clinical signs, decreased incidence of thrombocytopenia and/or neutropenia, earlier hematologic recovery, and reduced morbidity from bacterial infection in treated animals compared to the control group.
INTERNATIONAL JOURNAL OF RADIATION ONCOLOGY BIOLOGY PHYSICS
(2023)
Article
Oncology
Elizabeth S. Duke, Amy K. Barone, Somak Chatterjee, Pallavi S. Mishra-Kalyani, Yuan-Li Shen, Emasenyie Isikwei, Hong Zhao, Youwei Bi, Jiang Liu, Nam Atiqur Rahman, Emily Wearne, John K. Leighton, Maritsa Stephenson, Idara Ojofeitimi, Barbara Scepura, Abhilasha Nair, Richard Pazdur, Julia A. Beaver, Harpreet Singh
Summary: On September 17, 2021, the FDA approved cabozantinib for the treatment of RAI-refractory locally advanced or metastatic differentiated thyroid cancer. The approval was based on data from an international trial, which showed that cabozantinib significantly prolonged progression-free survival compared to placebo.
CLINICAL CANCER RESEARCH
(2022)
Editorial Material
Medicine, General & Internal
Rachel E. Sachs, Julie M. Donohue, Stacie B. Dusetzina
Summary: The reauthorization of the Accelerated Approval User-fee creates new opportunities for the FDA to address issues in its accelerated-approval program.
NEW ENGLAND JOURNAL OF MEDICINE
(2022)
Editorial Material
Oncology
Gautam U. Mehta, R. Angelo de Claro, Richard Pazdur
Summary: This viewpoint compares the US FDA's accelerated approval pathway for drugs and biologics with similar programs developed by other international regulatory health authorities.
Review
Medicine, General & Internal
Jonathan J. Darrow, Jerry Avorn, Aaron S. Kesselheim
Summary: Medical device regulation in the US has become more complex over the past 45 years, with various regulatory pathways and evolving evidence requirements. Increased FDA support from industry and concerns about flexible authorization requirements highlight the ongoing tension between efficient access and ensuring patient safety.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2021)
Article
Oncology
Vaibhav Kumar, Leslie Doros, Margaret Thompson, Sirisha L. Mushti, Rosane Charlab, Elizabeth I. Spehalski, Hong Zhao, Matthew D. Thompson, Shenghui Tang, Richard Pazdur, Steven J. Lemery, Marc R. Theoret, Lola A. Fashoyin-Aje
Summary: On May 15, 2020, the FDA approved ripretinib for adult patients with advanced gastrointestinal stromal tumor who have received prior treatment with three or more kinase inhibitors, including imatinib. The approval was based on results from the INVICTUS trial, which showed a significant improvement in progression-free survival for patients in the ripretinib group compared to the placebo group. The median overall survival was also longer in the ripretinib group.
CLINICAL CANCER RESEARCH
(2023)
Article
Medicine, General & Internal
Jeromie Ballreich, Mariana Socal, Charles L. Bennett, Martin W. Schoen, Antonio Trujillo, Andrew Xuan, Gerard Anderson
Summary: Accelerated approved drugs without full approval can remain on the market for many years, causing uncertainty about their clinical benefits. In 2019, the U.S. Food and Drug Administration spent $1.2 billion on these drugs.
ANNALS OF INTERNAL MEDICINE
(2022)
Article
Oncology
Erica C. Nakajima, Nicole Drezner, Xiaoxue Li, Pallavi S. Mishra-Kalyani, Yajun Liu, Hong Zhao, Youwei Bi, Jiang Liu, Atiqur Rahman, Emily Wearne, Idara Ojofeitimi, Lauren Tesh Hotaki, Dianne Spillman, Richard Pazdur, Julia A. Beaver, Harpreet Singh
Summary: The FDA has granted accelerated approval to sotorasib for the treatment of advanced non-small cell lung cancer with a KRAS G12C mutation. The drug has shown significant overall response rate and duration of response in clinical trials, with common adverse reactions including diarrhea and musculoskeletal pain.
CLINICAL CANCER RESEARCH
(2022)
Article
Allergy
Katherine E. Clarridge, Stacy J. Chin, Kelly D. Stone
Summary: The U.S. FDA regulates various products to ensure their safety and effectiveness. The FDA-approved drug labeling is an important tool for conveying information about drug use. Providers should be familiar with the format of prescribing information and be cautious about off-label drug use.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE
(2022)