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Recent Results in Cancer Research

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https://read.qxmd.com/read/30543014/prioritization-not-rationing-in-cancer-care
#1
Nikolaus Knoepffler, Jürgen Zerth, Martin O'Malley
Conditions of scarcity impact healthcare services for cancer patients. This is the unpleasant reality for nations, local governments, hospitals, and even individual doctors. This means that medical services judged by objective standards as potentially effective by medical professionals are limited because of financial or access scarcity. With this situation of scarcity as premise, one must raise the ethical question of how to deal with scarcity while respecting fundamental principles of human dignity and human rights...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543013/regulatory-and-evidence-requirements-and-the-changing-landscape-in-regulation-for-marketing-authorisation
#2
Francesco Pignatti, Elias Péan
In this chapter, we describe the changing landscape of the EU pharmaceutical legislation concerning regulation and evidence requirements for marketing authorisation. First, we describe the legal requirements for marketing authorisation and the development of EU pharmaceutical legislation and the concept of risk-benefit balance. Second, we describe special types of authorisation, such as conditional approval and approval under exceptional circumstances, and special provisions such as incentives for orphan medicinal products and paediatric investigational plans...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543012/drug-pricing-and-value-in-oncology
#3
Patricia M Danzon
This paper first reviews the evidence on price levels, price growth, and value for cancer drugs. The available evidence suggests that prices for originator (brand-name) drugs are rising significantly more rapidly than general inflation, but the available data are inadequate for robust comparisons between cancer and other categories of specialty drugs. We then examine the factors contributing to high and rising prices for cancer drugs. This analysis focuses mainly on the USA, which accounts for 46% of global expenditures on cancer drugs...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543011/recent-developments-in-health-economic-modelling-of-cancer-therapies
#4
William Green, Matthew Taylor
Arguably, the most common structure currently adopted for oncology modelling is the three-state partitioned survival model with the following states: stable disease, post-progression and dead. This design can, therefore, be adopted to capture the progressive nature of cancer. This chapter outlines the three-state model approach as well as introducing several other key aspects of economic modelling in oncology.
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543010/orphan-drugs-in-oncology
#5
Daria Korchagina, Szymon Jaroslawski, Guy Jadot, Mondher Toumi
Rare diseases represent a group of conditions affecting a very limited number of patients. Low profitability resulting from the small size of target population coupled with difficulties in conducting the research causes the lack of interest from the pharmaceutical industry. In order to promote research and development of medicines for rare diseases, a special 'orphan' legislation was introduced in a number of regions. These measures led to a significant increase in the number of approved orphan molecules. The high per patient cost of orphan drugs, as well the rapid growth of orphan drug sector, raised concerns regarding the sustainable funding of therapies for rare diseases...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543009/approaches-to-capturing-value-in-oncology
#6
Evelyn Walter
This article sets out to describe different value frameworks in the field of new developments in oncology. Since the costs of new oncological therapies follow a steep path, their implementation and financing demand a thorough assessment. This is an ambitious task due to the complex nature of oncological treatments within overall health policy. Five value frameworks were reviewed: European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, American Society of Clinical Oncology (ASCO) Value Framework (version 2...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543008/patient-reported-outcomes-in-health-economic-decision-making-a-changing-landscape-in-oncology
#7
Mandi Pratt-Chapman, Afsan Bhadelia
Cancer causes significant death and disability globally. However, costs of more personalized cancer care continue to climb, while access to basic cancer screening and treatment is not available to much of the world. This chapter provides an overview of the status of patient-reported outcomes (PROs) in cancer clinical care and research. PROs are valuable for health care and health economic decision-making at institutional, regional, national, and international levels. PRO data should be considered along with cost and survival data when approving new therapies...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543007/patient-reported-outcomes-in-oncology-beyond-randomized-controlled-trials
#8
Mondher Toumi, Szymon Jarosławski, Christos Chouhaid, Bruno Fallissard, P Auquier
The goal of the treatment of a disease has moved from treating organs and diseases through symptoms, biological parameters and imaging towards treating a human being as a whole. The treatments should deliver benefits that patients can personally perceive. However, the patient's perspective does not always match the one of those surrounding them. Illustratively, patients' symptom assessments are more predictable for daily health status, whereas clinicians' symptom measurements are more related to clinical outcomes...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543006/heterogeneous-recommendations-for-oncology-products-among-different-hta-systems-a-comparative-assessment
#9
Szymon Jarosławski, Eve Hanna, Monique Dabbous, Lylia Chachoua, Mondher Toumi
Rising budget constraints and demands for healthcare services create additional complexity within the decision process for resource allocation. Innovations and scientific progress have been shown to be key drivers of the increase in healthcare expenditures (1). In the context of rising medical care costs and limited resources, Health Technology Assessment (HTA) was developed as a tool to inform decision-making and to provide the rationalization behind these decisions driving resource allocation and spending for health technology products...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543005/oncology-from-an-hta-and-health-economic-perspective
#10
Clement Francois, Junwen Zhou, Michał Pochopien, Leila Achour, Mondher Toumi
In this chapter, we will present and discuss the challenges of assessing oncology products from a health economic perspective. We will provide a brief introduction on the need for economic evaluation in health care and focus on cost-effectiveness and comparative aspects of the evaluation of oncology products, which are of paramount interest to HTA decision-making bodies using economic evaluation in their decision-making framework. As the burden of oncology is well-documented, we do not discuss it in detail here...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543004/cost-of-cancer-healthcare-expenditures-and-economic-impact
#11
Bengt Jönsson
Healthcare expenditures for cancer account for a low share of total healthcare expenditures, compared to the relative burden of the disease. The share has also not changed very much over the last decades. Cost for cancer drugs has increased as a share of total expenditures, but this has been offset by a reduction of inpatient hospital care for cancer. Accounting for the cost of cancer should not be limited to healthcare expenditures. Resources are also used for public and private care of cancer patients outside the healthcare sector, for example for palliative care...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30543003/introduction-and-overview
#12
Evelyn Walter
The overall aim of this book is to set out the main changes needed in the field of economic and regulatory conditions as a consequence of these rapid developments in oncology. The traditional approaches of health economics, like health economic evaluation, health technology assessment (HTA), modeling methods, assessing value, pricing techniques, are bound to be altered in the contributions to this book. It is understandable that with the life-threatening diagnosis of cancer the new treatment options need to be accompanied by the best available health economic tools...
2019: Recent Results in Cancer Research
https://read.qxmd.com/read/30069773/osimertinib
#13
REVIEW
Umberto Malapelle, Biagio Ricciuti, Sara Baglivo, Francesco Pepe, Pasquale Pisapia, Paola Anastasi, Marco Tazza, Angelo Sidoni, Anna M Liberati, Guido Bellezza, Rita Chiari, Giulio Metro
Epidermal growth factor receptor (EGFR)-mutated (exons 18-21) advanced non-small cell lung cancers (NSCLCs) are generally characterized by exquisite sensitivity to treatment with an EGFR-tyrosine kinase inhibitor (-TKI). First-generation or reversible EGFR-TKIs include gefitinib and erlotinib, while, more recently, second-generation or irreversible EGFR-TKIs have been developed, namely afatinib and dacomitinib, with the aim of overcoming/delaying acquired resistance to treatment. Nevertheless, clinical trials have shown that resistance eventually emerges after a median time of slightly less than one year, regardless of whether first- or second-generation EGFR-TKIs are used...
2018: Recent Results in Cancer Research
https://read.qxmd.com/read/30069772/alectinib
#14
REVIEW
M Herden, Cornelius F Waller
Alectinib is an ATP-competitive small molecule and a second-generation inhibitor of ALK. EML4-ALK rearrangement is found in 3-5% of patients with NSCLC. The first-generation inhibitor crizotinib has changed the treatment dramatically, though most of the patients show disease progression within one year. Extra-thoracic progress, i.e., CNS metastases is common. The second-generation inhibitor alectinib has shown significant improvement in PFS and a remarkable prolongation of time to CNS progression. Alectinib has received approval as first-line therapy as well as second-line therapy after crizotinib failure...
2018: Recent Results in Cancer Research
https://read.qxmd.com/read/30069771/gefitinib
#15
REVIEW
Justyna Rawluk, Cornelius F Waller
Gefitinib is an orally active selective inhibitor epidermal growth factor receptor (EGFR). The large randomised phase III IPASS study (gefitinib 250 mg, daily vs carboplatin and paclitaxel) showed a beneficial effect on progression-free survival (PFS) and quality of life in selected patient populations under the treatment with gefitinib (HR for TKI 0.74; 95% CI: 0.65-0.85). In the subgroup of patients with EGFR mutation the effect of gefitinib on PFS was notably, PFS HR 0.48; 95% CI: 0.36-0.64, p < 0.001) and the objective response rate (RR) was 71...
2018: Recent Results in Cancer Research
https://read.qxmd.com/read/30069770/olaparib
#16
REVIEW
Sylvia Bochum, Stephanie Berger, Uwe M Martens
Olaparib (Lynparza [AstraZeneca, Cambridge, UK], formerly referred to as AZD2281 or KU0059436) is an oral poly(ADP-ribose) polymerase (PARP) inhibitor. It is rationally designed to act as a competitive inhibitor of NAD+ at the catalytic site of PARP1 and PARP2, both members of the PARP family of enzymes that are central to the repair of DNA single-strand breaks (SSBs) mediated via the base excision repair (BER) pathway. Inhibition of the BER pathway by olaparib leads to the accumulation of unrepaired SSBs, which leads to the formation of deleterious double-strand breaks (DSBs)...
2018: Recent Results in Cancer Research
https://read.qxmd.com/read/30069769/afatinib
#17
REVIEW
Helga Wecker, Cornelius F Waller
Afatinib (BIBW 2992, US: GilotrifTM , other countries: Giotrif©) is an irreversible blocker of the ErbB family, acting at the tyrosine kinases of these proteins. In 2013, it was approved by the FDA and the EMA for the treatment of adults with advanced, EGFR mutation-positive non-small-cell lung cancer. Further investigations for the treatment of many other tumors with afatinib, e.g., HNSCC and breast cancer, are ongoing.
2018: Recent Results in Cancer Research
https://read.qxmd.com/read/30069768/lenvantinib-a-tyrosine-kinase-inhibitor-of-vegfr-1-3-fgfr-1-4-pdgfr%C3%AE-kit-and-ret
#18
REVIEW
Stefanie Zschäbitz, Carsten Grüllich
Lenvatinib is an oral receptor tyrosine kinase inhibitor (TKI) with activity against vascular endothelial growth factor (VEGF) receptors 1-3, fibroblast growth factor receptors (FGFR) 1-4, platelet-derived growth factor receptor-alpha (PDGFRα), and RET and KIT proto-oncogenes. Lenvatinib is approved for the treatment of radioiodine-refractory differentiated thyroid cancer and in combination with everolimus for the treatment of advanced renal cell carcinoma following anti-VEGF treatment. In hepatocellular carcinoma lenvatinib was non inferior to sorafenib in first line with an improved progression-free survival and approval in this indication is expected...
2018: Recent Results in Cancer Research
https://read.qxmd.com/read/30069767/cobimetinib-gdc-0973-xl518
#19
REVIEW
Hana Andrlová, Robert Zeiser, Frank Meiss
The mitogen-activated protein kinase cascade (MAPK/ERK pathway) is a signaling pathway activated as a cellular response to various stimuli and for regulating the proliferation and survival of several types of eukaryotic cells, among others a wide variety of tumor cells. Mutations of the proteins involved in this pathway have been discovered in several tumor entities, indicating their inhibition as a potential therapeutic target. BRAF inhibitors have been in the clinical use since 2011. Several MEK inhibitors have been studied for metastatic cancer treatment in the recent past...
2018: Recent Results in Cancer Research
https://read.qxmd.com/read/30069766/palbociclib-the-first-of-a-new-class-of-cell-cycle-inhibitors
#20
REVIEW
Marcus Schmidt, Martin Sebastian
During the last decades, much has been learned about with cyclin-dependent kinases (CDK) playing a pivotal role in the cell cycle regulation. CDK4/6 is the key regulator of the G1-S transition. Palbociclib (PD 0332991, Ibrance®) is the first oral CDK4/6 inhibitor showing a substantially improved median progression-free survival (PFS) in advanced estrogen receptor (ER) positive and human epidermal growth factor receptor 2 (HER2) negative breast cancer. This PFS prolongation was seen both with letrozole as first-line therapy (24...
2018: Recent Results in Cancer Research
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