marketing approval
Recently Published Documents


TOTAL DOCUMENTS

159
(FIVE YEARS 45)

H-INDEX

15
(FIVE YEARS 3)

2022 ◽  
Vol 8 ◽  
Author(s):  
Hideki Maeda

In Japan, a law called the Clinical Trials Act went into being effective on April 1, 2018, and clinical research on human subjects conducted in Japan has been undergone major changes. Those other than clinical trials for marketing approval of drugs or medical devices are broadly classified into “specific clinical trials” and others, and regulations have been tightened for each. As a result, clinical interventional study was drastically reduced, and observational clinical study increased. For the observational clinical study, the two previous ethical guidelines were merged into the “Ethical Guidelines for Medical and Biological Research Involving Human Subjects,” which was enacted in March 2021. The observational clinical study is now subjected to these ethical guidelines. In addition, changes are planned for the Act on the Protection of Personal Information, which greatly affects data collection in clinical research. Clinical research in Japan must be conducted appropriately while adapting to these various changes in the external environment and legal framework. Adapting to these changes is not an easy task, as it requires increased financial and human resources for all stakeholders.


Pharmaceutics ◽  
2021 ◽  
Vol 13 (12) ◽  
pp. 2192
Author(s):  
Alberto Reale ◽  
Arianna Calistri ◽  
Jennifer Altomonte

Oncolytic viruses (OVs) are an emerging class of therapeutics which combine multiple mechanisms of action, including direct cancer cell-killing, immunotherapy and gene therapy. A growing number of clinical trials have indicated that OVs have an excellent safety profile and provide some degree of efficacy, but to date only a single OV drug, HSV-1 talimogene laherparepvec (T-Vec), has achieved marketing approval in the US and Europe. An important issue to consider in order to accelerate the clinical advancement of OV agents is the development of an effective delivery system. Currently, the most commonly employed OV delivery route is intratumoral; however, to target metastatic diseases and tumors that cannot be directly accessed, it is of great interest to develop effective approaches for the systemic delivery of OVs, such as the use of carrier cells. In general, the ideal carrier cell should have a tropism towards the tumor microenvironment (TME), and it must be susceptible to OV infection but remain viable long enough to allow migration and finally release of the OV within the tumor bed. Mesenchymal stem cells (MSCs) have been heavily investigated as carrier cells due to their inherent tumor tropism, in spite of some disadvantages in biodistribution. This review focuses on the other promising candidate carrier cells under development and discusses their interaction with specific OVs and future research lines.


2021 ◽  
Author(s):  
Aishat Motolani ◽  
Matthew Martin ◽  
Steven Sun ◽  
Tao Lu

Drug discovery is an exciting yet highly costly endeavor. In the United States, developing a new prescription medicine that gains marketing approval takes near a decade and costs drugmakers for near 3 billion. More challengingly, the success rate of a compound entering phase I trials is just slightly under 10%. Because of these mounting hurdles, repurposing market approved drugs to new clinical indications has been a new trend on the rise. Another merit to this approach is the already confirmed toxicity profiles of the drugs and their possession of drug-like features. Thus, repurposed drugs can reach the market approved stage in a much faster, cheaper, and more efficient way. Notably, epigenetic enzymes play a critical role in the etiology and progression of different diseases. Researchers are now assessing the possibilities of using market approved drugs to target epigenetic enzymes as a novel strategy to curtail disease progression. Thus, in this book chapter, we will provide an outlook on repurposing market drugs to target epigenetic enzymes in various diseases. Consequently, this book chapter will not only provide the readers with current knowledge in this specific field, but also will shed light on the pathway forward for repurposing market drugs to target epigenetic enzymes in human diseases.


2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Claudio Jommi ◽  
Federico Pantellini ◽  
Lisa Stagi ◽  
Maria Verykiou ◽  
Marianna Cavazza

Abstract Background Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing access to medicines before marketing approval to patients not recruited in trials. The economic impact of clinical trials has previously been investigated. No evidence on the net economic benefit of CUP exists. This research aims to address this information gap by estimating the economic consequences of 11 CUP in Italy conducted between March 2015 and December 2020 from the perspective of public health care system in Italy (National Health Service). Eight programs concern cancer treatments, two refer to spinal muscular atrophy, and one is indicated for multiple sclerosis. Methods Since CUP medicines are covered by the industry, the net economic benefit includes: (i) avoided costs of the Standard of Care (SoC) the patients would have received had they not joined the CUP, (ii) costs not covered by the pharmaceutical industry sponsor, but instead sustained by payers, such as those associated with adverse events (only severe side effects resulting in hospitalisation and attributable to CUP medicines), and (iii) costs for combination therapies and diagnostic procedures not used with the SoC. The SoC costing relied on publicly available data. Information on adverse events and diagnostic procedures was retrieved from the CUP and monetized using the relevant fee for episode or service. One CUP was excluded since a SoC was not identified. Results 2,713 patients were treated in the 11 CUP where a SoC was identified. The SoC mean cost per patient ranged from €11,415 to €20,299. The total cost of the SoC ranged between €31.0 and €55.1 million. The mean cost per patient covered by hospitals hosting CUP was equal to €1,646, with a total cost of €4.5 million. The net economic benefit ranged €26.5 million - €50.6 million. Conclusions Despite research limitations, this paper illustrates for the first time the net economic impact of CUP from a public payer perspective. It is important to integrate these estimates with the prospective effects of CUP implementation, i.e., the economic value of the comparative benefit profile of medicines used in CUP versus the SoC, including effects from a societal perspective.


2021 ◽  
Author(s):  
Stephen Lockhart ◽  
William Gruber

Vaccine Clinical Development is an integrated part of the overall process of vaccine development, which involves clinical trials in which study participants are usually prospectively and randomly allocated to receive the vaccine candidate or a control vaccine. Participants are then actively monitored to generate information on safety, and often immune responses and/or cases of the target disease. Clinical development starts with small phase 1 studies in tens or hundreds of volunteers. In phase 2, volunteers in the target population are studied for safety and evidence of a desired effect, often an immune response. In phase 3, pivotal studies will generally examine prevention of the target disease with at least 3000 participants receiving the vaccine candidate. The key outputs are reports for regulatory agencies, policy makers and health care workers to support marketing approval and appropriate ways to use the vaccine. Clinical trial designs and results are made public through registries and peer-reviewed publications.


2021 ◽  
Vol 3 ◽  
Author(s):  
Jane Nielsen ◽  
Lisa Eckstein ◽  
Dianne Nicol ◽  
Cameron Stewart

Public participation, transparency and accountability are three of the pillars of good governance. These pillars become particularly important for innovative, personalised health technologies, because of the tendency of these technologies to raise distinct scientific, ethical, legal and social issues. Genome editing is perhaps the most personal of all innovative health technologies, involving precise modifications to an individual’s genome. This article focuses on the adequacy of current requirements for public participation, transparency and accountability in the governance of the market authorisation for genome edited products. Although clinical trials for genome edited products are only just underway, lessons can be drawn from the marketing approvals pathways for related gene therapy products. This article provides a broad overview of the regulatory pathways that have been adopted by the US Food and Drugs Administration, the European Medicines Authority, and the Australian Therapeutic Goods Administration for reviewing gene therapy products for marketing approval. This analysis focuses on the extent to which public participation processes and transparency and accountability of review pathways are incorporated into marketing approval policy and practice. Following this review, the article proposes the application of Sheila Jasanoff’s “technologies of humility” as a foundation for meaningfully incorporating these pillars of good governance into regulatory processes for the review of products of genome editing. We conclude by articulating clear mechanisms for operationalising technologies of humility in the context of public participation, transparency and accountability, providing a blueprint for future policy development.


2021 ◽  
Vol 39 (28_suppl) ◽  
pp. 306-306
Author(s):  
Jennifer Leigh Lund ◽  
Michael Webster-Clark ◽  
Emily W. Bratton

306 Background: Randomized controlled trials are the gold standard for assessing the efficacy of new cancer therapies and are required for marketing approval. However, participants enrolled into trials are often not representative of the more diverse populations in whom treatment will ultimately be delivered. Real-world data can be used to potentially bridge this gap by evaluating the effectiveness and safety of therapies in more generalizable populations. In this study, we describe differences in demographic and clinical characteristics between participants enrolled in a phase III trial of adjuvant chemotherapy for colon cancer and a contemporary comparator cohort using structured oncology electronic medical records (EMR) data. Methods: We drew upon publications from the Multicenter International Study of Oxaliplatin/5 Fluorouracil/Leucovorin in the Adjuvant Treatment of Colon Cancer (MOSAIC) recruiting patients primarily from Western Europe with stage II or III colon cancer from October 1998-January 2001. This trial established FOLFOX (oxaliplatin + 5-FU) as the standard of care over 5FU alone in treating stage III colon cancer and led to US marketing approval. For comparison, we identified patients diagnosed with stage II or III colon cancer (September 2014-2020) who received FOLFOX in the IQVIA US Oncology EMR database via E360, IQVIA’s Real World Discovery and Analytics platform. In the two populations, we described distributions of prognostic factors including age, sex, stage of disease, body mass index (BMI), and Eastern Cooperative Group (ECOG) performance status. Results: The MOSAIC trial included 2,246 participants; we identified 4,566 patients starting FOLFOX in the EMR comparator. Median age was similar: 61 versus 62 years, but the range differed (19-75 years versus 18-85 years) in the trial and EMR comparator, respectively. Female sex was similar at 44% and 48%, while the proportion of patients with stage III disease differed considerably at 60% and 84% for trial and EMR comparator, respectively. Half (51%) of all EMR patients were missing ECOG status; among those with a reported ECOG status, 96% had a score of 0-1, while 87% of trial participants had an ECOG of 0-1. BMI was missing in 34% of patients in the EMR comparator; among those with a reported BMI, 28% had a BMI < 25, while 54% of trial participants had a BMI < 25. Conclusions: Colon cancer patients receiving FOLFOX in an EMR comparator had a similar age and sex distribution as MOSAIC trial participants, but were more likely to have stage III disease. Missing ECOG and BMI data were common in the EMR comparator, which limited comparisons. Structured oncology EMR data represent an important resource for generating real-world evidence; future data enrichment efforts focused on critical patient-level variables via unstructured EMR extraction may improve data completeness and quality.


Author(s):  
Aida Bujosa ◽  
Consolación Moltó ◽  
Thomas J. Hwang ◽  
José Carlos Tapia ◽  
Kerstin N. Vokinger ◽  
...  

Background: Most anticancer drugs are approved by regulatory agencies based on surrogate measures. This article explores the variables associated with overall survival (OS), quality of life (QoL), and substantial clinical benefit among anticancer drugs at the time of approval and in the postmarketing period. Methods: Anticancer drugs approved by the FDA between January 2006 and December 2015 and with postmarketing follow-up until April 2019 were identified. We evaluated trial-level data supporting approval and any updated OS and/or QoL data. We applied the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) and the ASCO Value Framework (ASCO-VF) to initial and follow-up studies. Results: We found that 58 drugs were approved for 96 indications based on 96 trials. At registration, approval was based on improved OS in 39 trials (41%) and improved QoL in 16 of 45 indications (36%). Postmarketing data showed an improvement in OS for 28 of 59 trials (47%) and in QoL for 22 of 48 indications (46%). At the time of approval, 25 of 94 (27%) and 26 of 80 scorable trials (33%) met substantial benefit thresholds using the ESMO-MCBS and ASCO-VF, respectively. In the postmarketing period, 37 of 69 (54%) and 35 of 65 (54%) trials met the substantial benefit thresholds. Drugs with companion diagnostics and immune checkpoint inhibitors were associated significantly with substantial clinical benefit. Conclusions: Compared with the time of approval, more anticancer drugs showed improved OS and QoL and met the ESMO-MCBS or ASCO-VF thresholds for substantial benefit over the course of postmarketing time. However, only approximately half of the trials met the threshold for substantial benefit. Companion diagnostic drugs and immunotherapy seemed to be associated with greater clinical benefit.


Author(s):  
Anshi Mehra ◽  
Hemanreet Kaur ◽  
Anoor Fatima ◽  
Aman Noor ◽  
Khushi Gupta ◽  
...  

A biosimilar is the new emerging drug product of the sector of Biologics that comes under speedily evolving area of pharmaceutical industry. It is the generic substitute for original research-based drugs. Since the Biologics are produced by a variety of products more commonly including cells extracted from humans, animals, microorganisms which varies phenotypically therefore they are “similar but not same” to the original product. Being a new product, it produces several challenges in the market including its regulation guidelines, production efficiency, quality management, and safety factors etc. The quality of these products along with its effectiveness and reduced costs are making them more and more popular. Different research is still being going on for enhancing the efficacy of biosimilars due to its more and more usage in the market. Several countries have developed their separate norms for the production and clinical usage of these biosimilars constituting Canada, Japan, Korea, and United States of America etc. The more biologics grab the attention of the eminent scientists for better innovations, the less marketing approval it gets. To enhance such a situation U S Congress passed the Biologics Price Competition and Innovation act 2009 and US FDA allowed “abbreviated pathway” for their approval and India being the most suited manufacturing area has also prepared certain guidelines stated as “Draft Guidelines on Similar Biologics” which were announced in June 2012, by Department of Biotechnology at Boston bio. The regulatory environment for biosimilars continues to evolve, both in recognition of advances in technology/ analytical methods and the availability of new targets for biosimilar development. With the advent of such efforts biosimilars are trying to surround the market. The demanding sector of biosimilars reveals the challenging gateway towards the nanomedicines also which shows that with the advancing biotechnology after these biosimilars, we are heading towards other newly biologically derived products.           


PLoS ONE ◽  
2021 ◽  
Vol 16 (7) ◽  
pp. e0255093
Author(s):  
Joyce M. Hoek ◽  
Sarahanne M. Field ◽  
Ymkje Anna de Vries ◽  
Maximilian Linde ◽  
Merle-Marie Pittelkow ◽  
...  

Background Following testing in clinical trials, the use of remdesivir for treatment of COVID-19 has been authorized for use in parts of the world, including the USA and Europe. Early authorizations were largely based on results from two clinical trials. A third study published by Wang et al. was underpowered and deemed inconclusive. Although regulators have shown an interest in interpreting the Wang et al. study, under a frequentist framework it is difficult to determine if the non-significant finding was caused by a lack of power or by the absence of an effect. Bayesian hypothesis testing does allow for quantification of evidence in favor of the absence of an effect. Findings Results of our Bayesian reanalysis of the three trials show ambiguous evidence for the primary outcome of clinical improvement and moderate evidence against the secondary outcome of decreased mortality rate. Additional analyses of three studies published after initial marketing approval support these findings. Conclusions We recommend that regulatory bodies take all available evidence into account for endorsement decisions. A Bayesian approach can be beneficial, in particular in case of statistically non-significant results. This is especially pressing when limited clinical efficacy data is available.


Sign in / Sign up

Export Citation Format

Share Document