Evolving HTA frameworks to accelerate innovation

Innovation in the cancer treatment space offers great promise, but even when approved by the European Medicines Agency (EMA) and other national regulatory bodies, patients can still face protracted delays in accessing new treatments.    Uncertainties in the evidence base for pioneering therapies can lead to delays from health technology assessment (HTA) agencies and payers in securing reimbursement.    To accelerate progress here, HTA frameworks must evolve, says Clare Hague, Therapy Area Market Access Lead, Hematology at Janssen EMEA, speaking ahead of the upcoming eyeforpharma Barcelona 2020 conference. “We need to look beyond overall survival (OS) when assessing the efficacy of innovative cancer medicines,” says Hague. “This is because the demonstration of a survival benefit may not be feasible or practical in situations where we see step-changes in the clinical benefit of our medicines and patients are living for a very long time. This is especially the case when the aim of treatment is moving towards disease modification, interception and, in earlier stage disease, the promise of cure or long-term durable remission.  “Collectively, we must consider alternative endpoints and shift the focus away from static decision-making on the basis of data observed at a single point in time, towards iterative decision-making based on evolving information and knowledge over time.”  “To achieve this, trus...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news

Related Links:

Authors: Pablo A, Breaud AR, Clarke W Abstract Bone marrow transplantation is used to treat particular types of cancers such as lymphoma, leukemia, and multiple myeloma. Appropriate dosing of busulfan during the preparative phase is critical for a successful allograft; if blood concentrations get too high significant liver toxicity can occur, if blood concentrations are too low, then graft-versus-host disease (GVHD) can develop. Busulfan monitoring in blood allows hospitals with the opportunity to provide individualized medicine to patients and improve overall patient outcome. Liquid Chromatography-Tandem Mass Spec...
Source: Current Protocols in Toxicology - Category: Toxicology Tags: Curr Protoc Toxicol Source Type: research
In an analysis of the outcome of pediatric acute myeloid leukemia (AML) with FMS-like tyrosine kinase 3 (FLT3) internal tandem duplication (ITD) mutation over 10 years at Children ’s Cancer Hospital Egypt, we found that FLT3-ITD mutation was associated with poor outcomes, and survival of patients who experienced relapse was extremely poor. Allogeneic bone marrow transplantation while the patient is experiencing first remission was the best treatment option. FLT3 inhibitors are needed to improve the outcome in AML in developing countries.
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Tags: Original Study Source Type: research
In this study, we investigated the link between AF and senescence markers through the assessment of protein expression in the tissue lysates of human appendages from patients in AF, including paroxysmal (PAF) or permanent AF (PmAF), and in sinus rhythm (SR). The major findings of the study indicated that the progression of AF is strongly related to the human atrial senescence burden as determined by p53 and p16 expression. The stepwise increase of senescence (p53, p16), prothrombotic (TF), and proremodeling (MMP-9) markers observed in the right atrial appendages of patients in SR, PAF, and PmAF points toward multiple inter...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
ConclusionsThese data demonstrate that a transplantation protocol involving only selective tumor-reactive donor T cell families is an effective immunotherapy and results in long-term survival in a mouse model of human MM. The results highlight the need to develop similar ATCT strategies for MM patients that result in enhanced survival without symptoms of GvHD.
Source: Journal for Immunotherapy of Cancer - Category: Cancer & Oncology Source Type: research
Three researchers at the  Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the state’s stem cell agency. The recipients areDr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;  Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; andDr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.Deng ’s four-year, $10.3 million award ...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news
Conclusions MDSC are major players in the immunosuppressive scenario in cancer, thanks to their phenotype heterogeneity and critical interaction with several innate immune cells, thus representing a crucial target in oncology. Here we reviewed the interactions of MDSCs with NK cells. The contribution of key cytokines, chemokines and mediators active in this process have been discussed. We also described the contribution of MDSC on angiogenesis directly or indirectly through interactions with NK and immunosuppressive activities. A parallel of the cancer associated to the decidual counterpart of these cells is discussed, a...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Discussion In this section, we discuss the mechanisms responsible for lymphomagenesis in the various inborn errors of immunity and provide an overview of the treatment. Defects in Immune Responses That Predispose to Lymphomagenesis in PIDDs The complex immune mechanisms and their interplay that predisposes to neoplastic transformation of B or T cells and development of lymphomas in PIDD patients has not been fully elucidated. However, it is expected that the etiology in most cases is multifactorial and related to a dynamic regulation of immune response and environmental triggers (Figure 3). An underlying intrinsic susce...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Conclusions: CAR T cell therapies have demonstrated the clinical benefits of harnessing our body's own defenses to combat tumor cells. Similar research is being conducted on lesser known modifications and gene-modified immune cells, which we highlight in this review. Introduction Chimeric antigen receptors and engineered T cell receptors (based on previously identified high affinity T cell receptors) function by redirecting T cells to a predefined tumor-specific (or tumor-associated) target. Most of these modifications use retroviral or lentiviral vectors to integrate the construct, and most of the receptors ...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
Conclusions:Intestinal microbiota containing several butyrate-producing anaerobes appear to be associated with MRD-negativity in patients with myeloma, with higher relative abundance of Eubacterium hallii and Faecalibacterium prausnitzii in MRD(-) patients compared with MRD(+) patients. Butyrate and other short-chain fatty acids are biologically active metabolites formed during microbial fermentation of dietary or host-derived carbohydrates, which supply the host with energy and also modulate immunity, including exerting anti-inflammatory functions. Microbes of the genus Eubacterium have been associated with reduced risk o...
Source: Blood - Category: Hematology Authors: Tags: 651. Myeloma: Biology and Pathophysiology, excluding Therapy: Poster II Source Type: research
This study analyzes patient and hospital characteristics of patients undergoing BMT, in addition the study describes resource utilization and outcomes of BMT among various hematological cancers.Methods: We performed a retrospective cohort analysis of the National Inpatient Sample 2014 Database (HCUP-NIS). Patients were included in the study if they had a procedure diagnosis of BMT and a medical diagnosis of acute or chronic leukemia (ALL, AML, CLL, CML), lymphoma (HL and NHL), MM, MDS or HLH. HSCT or BMT includes both Allogeneic and Autologous Stem Cell Transplant however we did not differentiate one from the other since B...
Source: Blood - Category: Hematology Authors: Tags: 902. Health Services Research-Malignant Diseases: Poster III Source Type: research
More News: Bone Marrow Transplant | Budgets | Cancer | Cancer & Oncology | Clinical Trials | Conferences | European Medicines Agency (EMA) | Hematology | Hospitals | Marketing | Myeloma | Partnerships | Pharmaceuticals | Study | Transplants