Objectives: To incentivize innovation and investment, the US and EU have developed favorable regulations for orphan drugs. Given the low number of patients and the urgency to make treatments available, having robust data at launch is unlikely. As a result, markets such as Germany have revised HTA rules to provide increased leniency when reviewing orphan drugs for reimbursement decisions. Many approved orphan drugs have oncology indications. With this research, we wanted to determine if oncology orphan drugs can achieve higher prices with the same or lower quality of evidence as non-oncology orphan drugs. Methods: We built a database of all drugs launched in orphan indications in the US and Europe since 2012. The database tracks: the launch indication, follow-on indications, date of first approval, population size of each indication, the HTA assessment in France and Germany (as proxy for the quality of the evidence) and the price in France, Germany and the US. We conducted a data analysis by market using a linear regression model, with the prevalence-adjusted price (price per patient multiplied by expected population size) as a dependent variable and the quality of the evidence (measured via HTA ratings) as the independent variable. We controlled for all other variables. Results: From the analysis it emerges that in the FR and the UK, oncology orphan drugs have achieved similar prevalence-adjusted price points as nononcology orphan drugs despite less robust evidence or proven benefit at launch. In the US, the level of evidence at launch was not correlated with price. Conclusions: Markets that relay on evidence driven HTAs to negotiate price may be more lenient on orphan drugs approved for oncology indications. Non-oncology orphan drugs may need to generate more evidence at launch than oncology drugs to secure optimal price and market access in the EU.
Objectives: With KEYNOTE-189 and KEYNOTE-21G trials showing improved overall survival (OS) and progression free survival (PFS) using Pem+Pembro+Plat for initial treatment of patients with metastatic NSQ NSCLC with no EGFR or ALK aberrations, a cost-effectiveness analysis of Pem+Pembro+Plat is needed to enable value-driven decisions. Methods: An area under the curve model comprising four health states: responding, stable, progressed, and dead, was developed to evaluate cost-effectiveness of Pem+Pembro+Plat vs. pemetrexed plus platinum chemotherapy (Pem+Plat). The primary outcome measure was cost per life year (LY) incremental cost-effectiveness ratio (ICER). Clinical effectiveness was parameterized using the published KEYNOTE-189 OS, PFS, and response data. Four survival curves were fitted to the trial data: exponential (best fitting), Weibull, log-normal, and log-logistic. Modeled costs included acquisition, infusion, adverse event, monthly disease state, one-time progression, and death. One-way and probabilistic sensitivity analyses (PSA) were performed on survival curves and cost inputs. Cost-effectiveness of subgroups defined by programmed death-ligand 1 (PD-L1) tumor proportion score (TPS) was analyzed. Costs and LYs were discounted at 3% per annum.
Objectives: To incentivize innovation and investment, the US and EU have developed favorable regulations for orphan drugs. Given the low number of patients and the urgency to make treatments available, having robust data at launch is unlikely. As a result, markets such as Germany have revised HTA rules to provide increased leniency when reviewing orphan drugs for reimbursement decisions. Many approved orphan drugs have oncology indications. With this research, we wanted to determine if oncology orphan drugs can achieve higher prices with the same or lower quality of evidence as non-oncology orphan drugs. Methods: We built a database of all drugs launched in orphan indications in the US and Europe since 2012. The database tracks: the launch indication, follow-on indications, date of first approval, population size of each indication, the HTA assessment in France and Germany (as proxy for the quality of the evidence) and the price in France, Germany and the US. We conducted a data analysis by market using a linear regression model, with the prevalence-adjusted price (price per patient multiplied by expected population size) as a dependent variable and the quality of the evidence (measured via HTA ratings) as the independent variable. We controlled for all other variables. Results: From the analysis it emerges that in the FR and the UK, oncology orphan drugs have achieved similar prevalence-adjusted price points as nononcology orphan drugs despite less robust evidence or proven benefit at launch. In the US, the level of evidence at launch was not correlated with price. Conclusions: Markets that relay on evidence driven HTAs to negotiate price may be more lenient on orphan drugs approved for oncology indications. Non-oncology orphan drugs may need to generate more evidence at launch than oncology drugs to secure optimal price and market access in the EU.
Background: Neonatal growth measurements (weight, length, head circumference) are used to assess fetal growth, determine neonatal and surveillance needs, and as a baseline for future growth monitoring. Measurements are often inaccurate and unreliable. To develop interventions for promoting evidencebased practice (EBP), we need to understand the knowledge, attitudes, practice behaviors, and bases of practice knowledge for neonatal growth measurement. A valid and reliable instrument did not exist. Aims: To develop and test the psychometric properties of the Neonatal Growth Measurement Survey (NGMS), we aimed to determine if it was a valid and reliable survey of neonatal growth measurement knowledge, attitudes, practice behaviors, and bases of practice knowledge. Methods: The survey included knowledge, attitude, practice behavior, and bases of practice knowledge questions constructed from best evidence and an EBP questionnaire. Content validity was assessed by seven clinical experts. A revised survey was pilot tested with a convenience sample of 20 neonatal nurses each from two mother-baby units and two NICUs. The same online survey was distributed twice 4 weeks apart to assess reliability. Data were analyzed for test-retest reliability and internal consistency. Results: The NGMS was validated to adequately represent the characteristics and constructs of neonatal growth measurement knowledge, attitudes, practice behaviors, and bases of practice knowledge. Sixty-two nurses completed the survey twice. Knowledge: Mean 83.9% agreement between responses from both survey administrations. Pearson correlation for total correct knowledge scores between time 1 and time 2 was .64. Cronbach's alpha was .37 for time 1 and .32 for time 2, indicating the scale did not measure a single construct. Attitudes: Mean Spearman's correlation between times was .51. Cronbach's alpha was .89 for time 1 and .88 for time 2. Practice behaviors: Agreement between time 1 and time 2 responses for head circumference (n=49): instruments 87.75%, techniques 91.94-100%; weight (n=62): instruments 91.94-100%, techniques 75.81-100%; length (n=49): instruments 87.75%, techniques 69.39-100%. Bases of practice knowledge: Mean Spearman's correlation between times was .44. Cronbach's alpha was .83 for time 1 and .84 for time 2. Conclusions: The NGMS was validated by content experts. Overall reliability was adequate, and suggested areas for further refinement. Clinical Implications: A refined NGMS will be used for a national study to understand current practices and barriers to EBP in order to develop nursing interventions to improve neonatal growth measurement accuracy and reliability.
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.
hi@scite.ai
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.