A489 Objectives: As the emphasis on value of oncology agents grows, ASCO has released a framework proposing to evaluate the net health benefit of such therapies. This study aims to evaluate the application of the ASCO framework for all recently approved oncology therapies, including barriers and challenges that may be encountered. MethOds: Oncology drugs approved from January 2013 to May 2015 (N= 19) were obtained from the FDA website. Palliative therapies were excluded. Registrational trials (N= 31) for the corresponding drugs were identified from peer-reviewed publications. The Net Health Benefit (NHB) score worksheet was completed for each clinical trial as outlined in the ASCO advanced disease framework using Overall Survival (OS), Progression-Free Survival (PFS), Response Rate (RR), Toxicity, Palliation, and Treatment-Free Interval. Issues encountered were catalogued for review. Results: 1 of 31 clinical trials was excluded due to lack of publication in peer-reviewed journals. An additional 9 trials were excluded due to non-randomized trial design, resulting in 22 trials corresponding to 16 drugs. OS data was used for scoring in 59% of the remaining trials. PFS was used in 27% of the trials, and RR in 14% of the trials. Toxicity data was scored in 95% of the trials, palliation data in 77% of trials, and treatment-free interval data in 95% of the trials. The most common scoring issues encountered included median OS or PFS not reached (8/22) and incomplete palliation or treatment-free interval data (5/22). cOnclusiOns: Lack of peer-reviewed publications, non-randomized trial designs, and requisite clinical data resulted in methodological challenges that deterred application of the ASCO framework for recent oncology drugs. Lack of appropriate data may result in lower scores than could be recognized from the available evidence at time of market authorization.
regression analyses were conducted using Stata 14. Results: The FDA approved 203 orphan drugs for chronic use (41.7% of 487 FDA-approved orphan drugs) in the study period. Most orphan drugs for chronic use were new drug applications (NDAs) (n= 166; 81.8% of the approvals), approved using the priority review process (107; 52.7%), and oral formulations (112; 55.2%). By December 31, 2014, 21.7% (n= 44) of the drugs had generic competition and 11.8% (n= 24) were discontinued from the market. The inflation-adjusted median cost per year at market entry increased from $1,573 in 1983-1984 to $100,555 in 2010-2014, representing an increase of 63.9 times the inflation-adjusted median cost during the study period. Biologic License Application (BLAs) approval (p< 0.0030), non-oral route formulation (p< 0.0000), FDA priority review (p< 0.0000), and year of approval (p< 0.0000) were significantly associated with orphan drug prices at market entry. ConClusions: An increasing number of orphan drugs have been approved by the FDA since the enactment of the Orphan Drug Act in 1983. Median prices at market entry of orphan drugs for chronic use have doubled every five years. FDA application type, review process, approval year, and route of administration were the main factors associated with the price at market entry of orphan drugs for chronic use.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
customersupport@researchsolutions.com
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.