Review
Biochemistry & Molecular Biology
Alexander C. Martins, Fernando Albericio, Beatriz G. de la Torre
Summary: The year 2022 saw the successful control of the COVID-19 pandemic in most countries through social and hygiene measures and vaccination campaigns. However, there was a decrease in total drug approvals by the FDA, particularly for small molecules. The Biologics class, on the other hand, maintained its figures with the authorization of 15 novel molecules.
Review
Pharmacology & Pharmacy
Michael S. Kinch, Zachary Kraft, Tyler Schwartz
Summary: The FDA approved a high number of novel molecular entities (NMEs) in 2020, with orphan drug approvals and priority reviews also increasing. Interestingly, the rate of consolidation in the pharmaceutical industry slowed down, leaving a significant number of clinical-stage pharmaceutical companies still active.
DRUG DISCOVERY TODAY
(2021)
Review
Pharmacology & Pharmacy
Michael S. Kinch, Zachary Kraft, Tyler Schwartz
Summary: In 2021, the FDA approved 60 new molecular entities (NMEs), reaching the highest level in the post-PDUFA era. Among these NMEs, 49 utilized the incentives of the Orphan Drug Act or obtained approval under Priority, Fast-Track, Accelerated, and/or Breakthrough designations. Although the number of registered clinical trials was lower than the 2020 peak, it remained within the 5-year running average. The rate of industry consolidation and turnover, however, raised concerns about the long-term sustainability of organizations involved in clinical development.
DRUG DISCOVERY TODAY
(2022)
Review
Rheumatology
Kathryn Henry, Natalie Anumolu, Michael Putman
Summary: The field of rheumatology has seen numerous new drug approvals in the past 25 years, but the regulatory mechanisms underlying these decisions are not well understood. In the USA, the FDA uses the New Drug Application (NDA) process to evaluate the safety and efficacy of novel drugs. In some cases, the FDA convenes Human Drug Advisory Committees to assess scientific or technical matters. A review of rheumatic disease drug applications granted FDA approval from 1996 to 2021 revealed 31 NDAs, out of which seven utilized an advisory committee. The reasons for using advisory committees and their impact on final approvals were unclear. Recommendations to enhance transparency and public trust in FDA decisions were provided.
Article
Public, Environmental & Occupational Health
Qingyang Xu, Elaheh Ahmadi, Alexander Amini, Daniela Rus, Andrew W. Lo
Summary: This study aims to address bias in drug approval predictions through debiasing and evaluate the impact of debiasing on financial value and drug safety. The results show that the debiased model performs better in predicting drug development outcomes and generates economic value for drug developers.
Review
Oncology
Chiara Corti, Gabriele Antonarelli, Carmine Valenza, Eleonora Nicolo, Hope Rugo, Javier Cortes, Nadia Harbeck, Lisa A. Carey, Carmen Criscitiello, Giuseppe Curigliano
Summary: This review discusses the challenges and opportunities of pan-histological expansion of ADCs in solid tumors. It covers technological and manufacturing advancements, current evidence from clinical trials, and the need for predictive biomarkers and methodological, statistical, and regulatory considerations in a biomarker-driven histology-agnostic approach.
EUROPEAN JOURNAL OF CANCER
(2022)
Editorial Material
Oncology
Steven Lemery, Richard Pazdur
Summary: In 2021, FDA Oncology focused on policy areas such as the Accelerated Approval program, expanding eligibility criteria, dose optimization, and patient-reported outcomes. The FDA approved new drugs and additional applications, including chimeric antigen receptor T cell products, antibody-drug conjugates, and targeted agents.
NATURE REVIEWS CLINICAL ONCOLOGY
(2022)
Editorial Material
Medicine, General & Internal
Julia A. Beaver, Richard Pazdur
Summary: In oncology, reevaluation of accelerated approvals for anti-PD-L1 antibodies revealed that for 10 indications, the required trials did not confirm benefit, yet their marketing authorization continued.
NEW ENGLAND JOURNAL OF MEDICINE
(2021)
Review
Biochemistry & Molecular Biology
Thais Cristina Mendonca Nogueira, Marcus Vinicius Nora de Souza
Summary: In 2020, FDA approved a total of 53 new drugs, with small-molecules accounting for the majority. Oncology was the dominant therapeutic area with 23 new approvals, including 13 new chemical entities.
BIOORGANIC & MEDICINAL CHEMISTRY
(2021)
Review
Chemistry, Medicinal
Shuo Yuan, Dan-Dan Shen, Rui Jia, Ju-Shan Sun, Jian Song, Hong-Min Liu
Summary: The U.S. FDA approved a total of 37 new drugs in 2022, including 20 chemical entities and 17 biologics. Among them, 17 small molecule drugs, 1 radiotherapy, and 2 diagnostic agents provide unique scaffolds, breakthrough clinical benefits, and new mechanisms of action for the discovery of more effective clinical candidates. Structure-based drug development and fragment-based drug development have always been important modules in drug discovery, allowing bypassing patent protection and enhancing biological activity. Therefore, we summarized valuable information on the clinical application, mechanism of action, and chemical synthesis of 17 newly approved small molecule drugs in 2022. We hope this timely and comprehensive review will inspire creative and elegant approaches in synthetic methodologies and mechanisms of action for the discovery of new drugs with novel chemical scaffolds and expanded clinical indications.
MEDICINAL RESEARCH REVIEWS
(2023)
Editorial Material
Biotechnology & Applied Microbiology
Asher Mullard
Summary: In 2022, the FDA approved 37 new drugs, the lowest number to pass regulatory scrutiny since 2016.
NATURE REVIEWS DRUG DISCOVERY
(2023)
Editorial Material
Biotechnology & Applied Microbiology
Asher Mullard
Summary: The FDA approved 50 novel drugs in 2021, including the first KRAS inhibitor for cancer and the first anti-amyloid antibody for Alzheimer's disease.
NATURE REVIEWS DRUG DISCOVERY
(2022)
Article
Oncology
Deepti Telaraja, Nicole Gormley, Richard Pazdur
Summary: In 2022, the FDA approved various new drugs and biologics, such as targeted small molecules, immunotherapeutics, gene therapy, and radiopharmaceuticals. The FDA Oncology Center of Excellence also focused on monitoring the Accelerated Approval program and optimizing drug doses.
NATURE REVIEWS CLINICAL ONCOLOGY
(2023)
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)
Article
Medicine, General & Internal
Sean T. Allen, Suzanne M. Grieb, Jennifer L. Glick, Rebecca Hamilton White, Tyler Puryear, Katherine C. Smith, Brian W. Weir, Susan G. Sherman
Summary: This study explores the applications of research evidence during the approval processes for syringe services programs (SSPs) in rural counties in Kentucky. The findings indicate that the use of research evidence varied, with instrumental applications being used to address concerns and support implementation. However, there were also instances of skepticism towards research evidence.
ANNALS OF MEDICINE
(2022)
