Results 41 to 50 of about 1,709,620 (289)

Characteristics of Preapproval and Postapproval Studies for Drugs Granted Accelerated Approval by the US Food and Drug Administration. [PDF]

JAMA, 2017
Naci H, Smalley KR, Kesselheim AS.
europepmc   +2 more sources

The value of the accelerated approval pathway: real-world outcomes associated with reducing the time between innovations. [PDF]

Future Oncol
Wong W, Kowal S, To TM, Patel A, Veenstra D, Garrison L, Li M.   +6 more
europepmc   +2 more sources

Establishing a Public Resource for Acceptable Surrogate Endpoints to Support FDA Marketing Applications

Frontiers in Medicine, 2022
Following a comprehensive and coordinated effort between CBER and CDER, FDA established a table of acceptable surrogate endpoints (SEs) to support drug marketing applications.
Abena S. Agyeman, Jeffrey N. Siegel, Christopher Leptak   +2 more
doaj   +1 more source

FDA Accelerated Approval of Pembrolizumab for Recurrent Locally Advanced or Metastatic Merkel Cell Carcinoma. [PDF]

Oncologist, 2020
Bradford D, Demko S, Jin S, Mishra-Kalyani P, Beckles AR, Goldberg KB, Lemery S, Ward A, Keegan P, Pazdur R.   +9 more
europepmc   +3 more sources

A modified weighted log-rank test for confirmatory trials with a high proportion of treatment switching [PDF]

, 2020
In confirmatory cancer clinical trials, overall survival (OS) is normally a primary endpoint in the intention-to-treat (ITT) analysis under regulatory standards. After the tumor progresses, it is common that patients allocated to the control group switch
Bore, Alexander, Burman, Carl-Fredrik, Jiménez, José L., Niewczas, Julia   +3 more
core   +2 more sources

Accelerated approval drug labels often lack information for clinical decision‐making

Pharmacotherapy, 2023
We evaluated US Food and Drug Administration labels for drugs approved under the accelerated approval pathway and whether these labels contained in sufficient information regarding their accelerated approval.
Jeromie Ballreich, M. Socal, Charles L. Bennett, Andrew Xuan, A. Trujillo, G. Anderson   +5 more
semanticscholar   +1 more source

Development and Use of Gene Therapy Orphan Drugs—Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society

Frontiers in Medicine, 2020
Gene therapy orphan medicinal products constitute a unique group of new drugs which in case of hereditary diseases are usually administered only once at an early age, in the hope to provide sufficient gene product to last for the entire life of the ...
Sandor Kerpel-Fronius, Varvara Baroutsou, Sander Becker, Roberto Carlesi, Luis Collia, Brigitte Franke-Bray, Peter Kleist, Chieko Kurihara, Luis Filipe Laranjeira, Kotone Matsuyama, Shehla Naseem, Johanna Schenk, Honorio Silva   +12 more
doaj   +1 more source

FDA grants accelerated approval of mosunetuzumab for relapsed, refractory follicular lymphoma

Cancer, 2023
he final analysis of the phase 3 ALPINE trial shows ...
M. Nierengarten
semanticscholar   +1 more source

Timing and characteristics of cumulative evidence available on novel therapeutic agents receiving Food and Drug Administration accelerated approval [PDF]

, 2017
Context: Therapeutic agents treating serious conditions are eligible for Food and Drug Administration (FDA) accelerated approval. The clinical evidence accrued on agents receiving accelerated approval has not been systematically evaluated.
Gupta, Radhika, Ioannidis, John P. A., Naci, Huseyin, Wouters, Olivier J.   +3 more
core   +1 more source

A comparison of the Food and Drug Administration’s and Health Canada’s regulatory decisions about failed confirmatory trials for oncology drugs: an observational study

Journal of Pharmaceutical Policy and Practice, 2021
Background Oncology drugs are frequently approved on the basis of surrogate outcomes that require further trials to confirm the benefits, but at times these trials fail and regulators need to decide whether to withdraw approval for the indication and/or ...
Joel Lexchin
doaj   +1 more source