Key Laboratory of Pulp and Paper Science & Technology of Ministry of Education, State Key Laboratory of Biobased Material and Green Papermaking, Faculty of Light Industry, Qi Lu University of Technology (Shandong Academy of Sciences), Jinan 250353, PR China.
Key Laboratory of Pulp and Paper Science & Technology of Ministry of Education, State Key Laboratory of Biobased Material and Green Papermaking, Faculty of Light Industry, Qi Lu University of Technology (Shandong Academy of Sciences), Jinan 250353, PR China.
Key Laboratory of Pulp and Paper Science & Technology of Ministry of Education, State Key Laboratory of Biobased Material and Green Papermaking, Faculty of Light Industry, Qi Lu University of Technology (Shandong Academy of Sciences), Jinan 250353, PR China.
Key Laboratory of Pulp and Paper Science & Technology of Ministry of Education, State Key Laboratory of Biobased Material and Green Papermaking, Faculty of Light Industry, Qi Lu University of Technology (Shandong Academy of Sciences), Jinan 250353, PR China.
Key Laboratory of Pulp and Paper Science & Technology of Ministry of Education, State Key Laboratory of Biobased Material and Green Papermaking, Faculty of Light Industry, Qi Lu University of Technology (Shandong Academy of Sciences), Jinan 250353, PR China. Electronic address: wjy@qlu.edu.cn.
Institute of Optical Materials and Chemical Biology, Guangxi Key Laboratory of Electrochemical Energy Materials, School of Chemistry and Chemical Engineering, Guangxi University, Nanning, Guangxi, China.
Institute of Optical Materials and Chemical Biology, Guangxi Key Laboratory of Electrochemical Energy Materials, School of Chemistry and Chemical Engineering, Guangxi University, Nanning, Guangxi, China.
Institute of Optical Materials and Chemical Biology, Guangxi Key Laboratory of Electrochemical Energy Materials, School of Chemistry and Chemical Engineering, Guangxi University, Nanning, Guangxi, China.
In 2022, the US Food and Drug Administration (FDA) approved 37 novel drugs. Twenty-four of the 37 (65%) novel drug approvals were reviewed and approved through an expedited review pathway and 20 of th...
Conditional approval pathways facilitate accelerated marketing authorisation based on immature clinical evidence for drugs that address an unmet medical need in a life-threatening or chronically debil...
The objective of this study is to assess whether conditionally approved drugs face higher probabilities of HTA rejection or delays in HTA approval relative to drugs with standard marketing authorisati...
This paper adopts a mixed-methods approach to provide a meta-analysis of HTA outcomes across 80 drug-indication pairs in France, England, Scotland and Canada. Differences in the characteristics (i.e. ...
Relative to standard approval drugs, conditionally approved drugs are less likely to include phase III trial designs, less likely to include clinical endpoints and less likely to include an active com...
Overall, conditionally approved drugs likely face increased barriers at the HTA level....
The U.S. Food and Drug Administration provides accelerated approval to drugs on the basis of surrogate end points deemed to be "reasonably likely" to predict clinical benefit. To receive full approval...
To estimate fee-for-service Medicare payments on accelerated approved drugs without full approvals....
Cross-sectional analysis....
Fee-for-service Medicare Part B and Part D drug claims in 2019....
Beneficiaries enrolled in Medicare Part B and Part D plans....
Medicare spending for drugs treating accelerated approved indications without full approval, beneficiary spending, and drug characteristics....
In 2019, 45 drugs associated with 69 accelerated approved indications lacked full approval. Of those, the fee-for-service Medicare program spent $1.2 billion on 36 drugs across 55 indications. Medicar...
The study drugs may have clinical benefit and may come to receive full approval after this analysis. The algorithm used to identify accelerated approved indications is novel. Generalizability to other...
In 2019, fee-for-service Medicare spent $1.2 billion on accelerated approved drugs without full approval. Medicare should adjust incentives to encourage sponsors to complete confirmatory trials as soo...
Laura and John Arnold Foundation....
Drugs addressing unmet medical needs can change the lives of millions. Developing and validating new drugs can, however, take many years. To streamline the assessment of new drugs, regulatory agencies...
The development of orphan drugs (ODs) is challenging from both development and business perspectives because of their small patient populations. To overcome such business challenges, lifecycle managem...
The 203 ODs consisting of 173 active ingredients were approved in Japan between 2004 and 2019. Sixty-eight (39%) of the 173 active ingredients have additional indications, of which 57 have at least on...
Pharmaceutical companies were actively adding non-OD indications and were emphasizing the use of patent rights by registering extensions of substance or use patents for exclusive marketing periods. Th...
The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) offer expedited regulatory approval programs for drugs with high potential patient value applicable at different stage...
50 New drugs including 36 chemical entities and 14 biologics were approved by the U.S. Food and Drug Administration during 2021. Among the marketed drugs, 31 new small molecule agents (29 small molecu...
New formulations of prescription drugs can improve convenience and tolerability for patients, but they also constitute manufacturer strategies to extend brand-name drug market exclusivity periods....
To examine whether new formulations of brand-name novel drugs were associated with novel drugs' sales and/or therapeutic value, as well as characterize first new formulations' approval timing relative...
This cross-sectional study used the Drugs@FDA database to identify all novel tablet and capsule drugs approved by the US Food and Drug Administration (FDA) between 1995 and 2010 and followed through D...
Novel drugs' blockbuster status, defined as annual sales of $1 billion or greater, and therapeutic value, measured by (1) accelerated approval status, (2) World Health Organization Model Lists of Esse...
Approval of a new formulation and timing relative to a novel drug's first generic's approval....
Among the 206 novel drugs in tablet or capsule form approved by the FDA from 1995 to 2010, 81 (39.3%) were followed by an FDA-approved new formulation, and 167 (81.1%) had a generic version as of Dece...
In this cross-sectional study of novel drugs in tablet or capsule form approved by the FDA between 1995 and 2010, manufacturers pursued new formulations of best-selling brand-name drugs and those gran...