Economic guidelines for oncology products: Adaptation of the Canadian Agency for Drugs and Technologies in Health (CADTH) technology assessment guidance document

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e17572-e17572
Author(s):  
N. Mittmann ◽  
W. K. Evans ◽  
A. Rocchi ◽  
C. J. Longo ◽  
H. J. Au ◽  
...  
Keyword(s):  
Working Group ◽  
Progression Free Survival ◽  
Target Population ◽  
Decision Makers ◽  
Phase Iii ◽  
Economic Evaluations ◽  
Guidance Document ◽  
Health Technologies ◽  
Intermediate Outcomes

e17572 Background: Economic evaluations (EE) are routinely used by decision-makers in Canada. CADTH's “Guidelines for the Economic Evaluation of Health Technologies: Canada” Third edition, 2006, provide guidance on the conduct of EEs for all therapeutic products. The consistency and quality of oncology EEs are variable and therapeutics in the cancer care environment presented unique challenges in decision making. Several chapters of the CADTH document adequately defined methods for the conduct of an oncology EE. However, some chapters required more specific guidance to improve the quality of oncology EEs. The goal was to provide direction on methods for the conduct of high quality EEs in oncology. Methods: The Working Group on Economic Analysis, NCIC CTG and CADTH jointly initiated this project and formed a working group (WG) of oncologists, health economists, decision makers and economic analysts. The WG identified CADTH chapters where oncology-specific guidance would be required. In-person and teleconference meetings provided content and structure for the document. Formal reviews by external academic experts, cancer agencies, patient groups and the pharmaceutical industry were conducted. Feedback was reviewed by the WG and incorporated as appropriate. Results: Chapters requiring guidance included: target population, comparators, perspective, effectiveness, modeling, type of evaluation, valuing health, time horizon, costs and resources, sensitivity analysis and equity. Guidance included clarity around CADTH methodology and recommendations for oncology products. For example for the effectiveness chapter, there was guidance around the use of intermediate outcomes (progression free survival vs. overall survival) and type of evidence (phase II vs. phase III). Overall recommendations for chapters will be presented. Conclusions: The oncology adapted economic guidelines provide specific guidance on the conduct of EEs for oncology products and will be published as an addendum to CADTH's third edition document. Their use should lead to more consistent application of EE methodologies for anti-cancer drugs and higher quality information for decision-makers at a national and perhaps international level. No significant financial relationships to disclose.

OP145 The Release Of The Fourth Edition Canadian Agency For Drugs And Technologies In Health (CADTH) Economic Guidelines – A Year In Review

2018 ◽  
Vol 34 (S1) ◽  
pp. 53-54
Author(s):  
Karen Lee ◽  
Doug Coyle
Keyword(s):  
Worked Examples ◽  
Target Population ◽  
Decision Makers ◽  
Economic Evaluations ◽  
Fourth Edition ◽  
Health Technologies ◽  
General Acceptance ◽  
Healthcare Decision Making ◽  
Patient Groups ◽  
Theoretical Foundations

Introduction:In March 2017, CADTH released the fourth edition of the Guidelines for the Economic Evaluations of Health Technologies. As part of the update a few notable changes were made to topics such as discount rate, target population, modeling, effectiveness, analysis, and the theoretical foundations for the Guidelines. In this presentation we will describe: the implementation of the Guidelines; approaches taken to facilitate the adoption of the Guideline statements; and the tools provided to assist users and doers in using cost-effectiveness information in healthcare decision making.Methods:Given some of the changes made to the Guidelines, CADTH identified the need to engage stakeholders early in preparation for the release of the fourth edition. Feedback on topics was sought from various stakeholders (researchers in the field, industry, patient groups, and decision makers) throughout the process. Also, suggestions for tools to support the understanding and implementation of the Guidelines were noted by CADTH. To further support use of the Guidelines, CADTH committed to undertake a number of activities including: workshops for decision makers and researchers; worked examples to illustrate the approaches; and development of tools to assist in the use of recommended methods. Updates to align drug submission guidance with the Guidelines are ongoing.Results:The final version of the Guidelines was greatly influenced by the stakeholder feedback received, with a focus on greater clarity. Whilst efforts to increase acceptance and adoption of the guidelines are ongoing, we present preliminary findings with respect to engagement with stakeholders and adoption of new guidance in drug submissions.Conclusions:The plan to engage stakeholders continues to be effective. As such, there has been general acceptance of the changes and an interest in education and tools to assist with implementation of the Guidelines.

scholarly journals Cisplatin-Based First-Line Treatment of Elderly Patients With Advanced Non–Small-Cell Lung Cancer: Joint Analysis of MILES-3 and MILES-4 Phase III Trials

2018 ◽  
Vol 36 (25) ◽  
pp. 2585-2592 ◽  
Author(s):  
Cesare Gridelli ◽  
Alessandro Morabito ◽  
Luigi Cavanna ◽  
Andrea Luciani ◽  
Paolo Maione ◽  
...  
Keyword(s):  
Elderly Patients ◽  
Advanced Nsclc ◽  
Performance Status ◽  
Progression Free Survival ◽  
Phase Iii ◽  
First Line ◽  
Free Survival ◽  
Phase Iii Trials ◽  
Health Status Score

Purpose To test the efficacy of adding cisplatin to first-line treatment for elderly patients with advanced non–small-cell lung cancer (NSCLC) within a combined analysis of two parallel phase III trials, MILES-3 and MILES-4. Patients and Methods Patients with advanced NSCLC who were older than age 70 years with Eastern Cooperative Oncology Group performance status 0 to 1 were randomly assigned to gemcitabine or pemetrexed, without or with cisplatin. In each trial, 382 events were required to detect a hazard ratio (HR) of death of 0.75, with 80% power and two-tailed α of .05. Trials were closed prematurely because of slow accrual, but the joint database allowed us to analyze the efficacy of cisplatin on the basis of intention-to-treat and adjusted by trial, histotype, non-platinum companion drug, stage, performance status, sex, age, and size of the study center. Results From March 2011 to August 2016, 531 patients (MILES-3, 299; MILES-4, 232) were assigned to gemcitabine or pemetrexed without (n = 268) or with cisplatin (n = 263). Median age was 75 years, 79% were male, and 70% had nonsquamous histology. At a median 2-year follow-up, 384 deaths and 448 progression-free survival events were recorded. Overall survival was not significantly prolonged with cisplatin (HR, 0.86; 95% CI, 0.70 to 1.05; P = .14) and global health status score of quality of life was not improved, whereas progression-free survival (HR, 0.76; 95% CI, 0.63 to 0.92; P = .005) and objective response rate (15.5% v 8.5%; P = .02) were significantly better. Significantly more severe hematologic toxicity, fatigue, and anorexia were found with cisplatin. Conclusion The addition of cisplatin to single-agent chemotherapy does not significantly prolong overall survival, and it does not improve global health status score of quality of life in elderly patients with advanced NSCLC.

Randomized Phase II Trial of Concurrent Cisplatin-Radiotherapy With or Without Neoadjuvant Docetaxel and Cisplatin in Advanced Nasopharyngeal Carcinoma

2009 ◽  
Vol 27 (2) ◽  
pp. 242-249 ◽  
Author(s):  
Edwin P. Hui ◽  
Brigette B. Ma ◽  
Sing F. Leung ◽  
Ann D. King ◽  
Frankie Mo ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Neoadjuvant Chemotherapy ◽  
Positive Impact ◽  
Nasopharyngeal Cancer ◽  
Progression Free Survival ◽  
Hazard Ratio ◽  
High Rate ◽  
Phase Iii ◽  
Tumor Control

Purpose To compare the toxicities, tumor control, survival, and quality of life of nasopharyngeal cancer (NPC) patients treated with sequential neoadjuvant chemotherapy followed by concurrent cisplatin-radiotherapy (CRT) or CRT alone. Patients and Methods Previously untreated stage III to IVB NPC were randomly assigned to (1) neoadjuvant docetaxel 75 mg/m2 and cisplatin 75 mg/m2 every 3 weeks for two cycles, followed by cisplatin 40 mg/m2/wk concurrent with radiotherapy, or (2) CRT alone. Planned accrual was 30 patients per arm to detect 20% difference of toxicities based on 95% CIs. Results From November 2002 to November 2004, 65 eligible patients were randomly assigned to neoadjuvant chemotherapy followed by CRT (n = 34) or CRT alone (n = 31). There was a high rate of grade 3/4 neutropenia (97%) but not neutropenic fever (12%) during neoadjuvant chemotherapy. No significant differences in rates of acute toxicities were observed between the two arms during CRT. Dose intensities of concurrent cisplatin, late RT toxicities and quality of life scores were comparable in both arms. The 3-year progression-free survival for neoadjuvant versus control arm was 88.2% and 59.5% (hazard ratio = 0.49; 95% CI, 0.20 to 1.19; P = .12). The 3-year overall survival for neoadjuvant versus control arm was 94.1% and 67.7% (hazard ratio = 0.24; 95% CI, 0.078 to 0.73; P = .012). Conclusion Neoadjuvant docetaxel-cisplatin followed by CRT was well tolerated with a manageable toxicity profile that allowed subsequent delivery of full-dose CRT. Preliminary results suggested a positive impact on survival. A phase III study to definitively test this neoadjuvant-concurrent strategy is warranted.

Metiv-HCC: A phase III clinical trial evaluating tivantinib (ARQ 197), a MET inhibitor, versus placebo as second-line in patients (pts) with MET-high inoperable hepatocellular carcinoma (HCC).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. TPS4159-TPS4159 ◽  
Author(s):  
Armando Santoro ◽  
Camillo Porta ◽  
Lorenza Rimassa ◽  
Ivan Borbath ◽  
Bruno Daniele ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Overall Survival ◽  
Progression Free Survival ◽  
Target Population ◽  
Phase Iii ◽  
Tyrosine Kinase Receptor ◽  
Second Line ◽  
Phase Iii Clinical Trial ◽  
Arq 197 ◽  
Liver And Kidney

TPS4159 Background: Tivantinibis a selective, non-ATP competitive, oral inhibitor of MET, the tyrosine kinase receptor for hepatocyte growth factor (HGF). MET over-expression is associated with poor prognosis in HCC patients. A phase Ib study (Santoro et al, Br J Cancer, 2013) with tivantinib 360mg BID revealed no worsening of liver function in cirrhotic HCC pts. A randomized, placebo-controlled phase 2 study identified HCC patients with high tumor MET expression at immunohistochemistry (IHC) as the target population for tivantinib in second line (overall survival: 7.2 months on tivantinib, 3.8 months on placebo, HR: 0.38, p=0.01), and selected 240mg BID as the appropriate dose for HCC patients (Santoro et al, Lancet Oncol, 2013). Methods: Enrollment forthis phase III clinical trial (ARQ 197-A-U303, NCT01755767) has begun.Eligible pts must present with Child Pugh A; ECOG performance score <1; inoperable RECIST 1.1 measurable disease; adequate bone marrow, liver and kidney functions; no prior liver transplant. Pts must have progressed after or not tolerated one prior line of systemic therapy including sorafenib and their tumor samples must be deemed MET-High by IHC at a central laboratory to be eligible. Approximately 303 pts are randomized 2:1 to receive tivantinib 240mg PO twice daily or placebo. Pts are stratified by vascular invasion, metastases, and alphafetoprotein level, and they are evaluated by CT or MRI scan at 8-week intervals. The primary endpoint is overall survival (OS). Secondary endpoints include progression-free survival and safety. Treatment continues until confirmed disease progression or unacceptable toxicity. Pts discontinued from study treatment will be followed for survival. Participating centers are located in Europe, Australia, New Zealand, and the Americas. This trial is expected to complete enrollment by mid-2015, and an interim analysis is planned when approximately 60% of OS events are reached. Clinical trial information: NCT01755767.

scholarly journals PREFERRED REPORTING ITEMS FOR STUDIES MAPPING ONTO PREFERENCE-BASED OUTCOME MEASURES: THE MAPS STATEMENT

2015 ◽  
Vol 31 (4) ◽  
pp. 230-235 ◽  
Author(s):  
Stavros Petrou ◽  
Oliver Rivero-Arias ◽  
Helen Dakin ◽  
Louise Longworth ◽  
Mark Oppe ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Working Group ◽  
De Novo ◽  
Economic Evaluations ◽  
Biomedical Journal ◽  
Modified Delphi ◽  
Journal Editorial ◽  
Guidance Documents ◽  
Completeness Of Reporting

Background:“Mapping” onto generic preference-based outcome measures is increasingly being used as a means of generating health utilities for use within health economic evaluations. Despite publication of technical guides for the conduct of mapping research, guidance for thereportingof mapping studies is currently lacking. The MAPS (MApping onto Preference-based measures reporting Standards) statement is a new checklist, which aims to promote complete and transparent reporting of mapping studies.Methods:In the absence of previously published reporting checklists or reporting guidance documents, ade novolist of reporting items was created by a working group comprised of six health economists and one Delphi methodologist. A two-round, modified Delphi survey with representatives from academia, consultancy, health technology assessment agencies, and the biomedical journal editorial community was used to identify a list of essential reporting items from this larger list.Results:From the initialde novolist of twenty-nine candidate items, a set of twenty-three essential reporting items was developed. The items are presented numerically and categorized within six sections, namely: (i) title and abstract, (ii) introduction, (iii) methods, (iv) results, (v) discussion, and (vi) other. The MAPS statement is best applied in conjunction with the accompanying MAPS explanation and elaboration document.Conclusions:It is anticipated that the MAPS statement will improve the clarity, transparency. and completeness of reporting of mapping studies. To facilitate dissemination and uptake, the MAPS statement is being co-published by seven health economics and quality of life journals, and broader endorsement is encouraged. The MAPS working group plans to assess the need for an update of the reporting checklist in five years’ time.

DISINVESTING FROM INEFFECTIVE TECHNOLOGIES: LESSONS LEARNED FROM CURRENT PROGRAMS

2015 ◽  
Vol 31 (6) ◽  
pp. 355-362 ◽  
Author(s):  
Julia Mayer ◽  
Anna Nachtnebel
Keyword(s):  
Health Care ◽  
Stakeholder Involvement ◽  
Lessons Learned ◽  
Decision Makers ◽  
Target Groups ◽  
Hand Search ◽  
Health Technologies ◽  
Active Identification ◽  
International Experts

Objectives: Many of the currently used health technologies have never been systematically assessed or are misused, overused or superseded. Therefore, they may be ineffective. Active identification of ineffectiveness in health care is gaining importance to facilitate best care for patients and optimal use of limited resources. The present research analyzed processes and experiences of programs for identifying ineffective health technologies. The goal of this study was to elucidate factors that facilitate implementation.Methods: Based on an overview article, a systematic literature search and unsystematic hand-search were conducted. Further information was gained from international experts.Results: Seven programs were identified that include identification, prioritization and assessment of ineffective health technologies and dissemination of recommendations. The programs are quite similar regarding their goals, target groups and criteria for identification and prioritization. Outputs, mainly HTA reports or lists, are mostly disseminated by means of the internet. Top–down and bottom–up programs both have benefits in terms of implementation of recommendations, either as binding guidelines and decisions or as nonbinding information for physicians and other stakeholders. Crucial facilitators of implementation are political will, transparent processes and broad stakeholder involvement focusing on physicians.Conclusions: All programs can improve the quality of health care and enable cost reduction in supportive surrounding conditions. Physicians and patients must be continuously involved in the process of evaluating health technologies. Additionally, decision makers must support programs and translate recommendations into concrete actions.

A phase III study comparing amrubicin and cisplatin (AP) with irinotecan and cisplatin (IP) for the treatment of extended-stage small cell lung cancer (ED-SCLC): JCOG0509.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. 7003-7003 ◽  
Author(s):  
Yoshikazu Kotani ◽  
Miyako Satouchi ◽  
Masahiko Ando ◽  
Kazuhiko Nakagawa ◽  
Nobuyuki Yamamoto ◽  
...  
Keyword(s):  
Standard Treatment ◽  
Progression Free Survival ◽  
Phase Iii ◽  
Eligibility Criteria ◽  
Severe Diarrhea ◽  
Secondary Endpoints ◽  
Phase Iii Study ◽  
Ecog Ps ◽  
To Receive

7003 Background: IP is the standard treatment for ED-SCLC, however often cause severe diarrhea. AP have shown promising activity in SCLC with fewer diarrhea. We conducted a phase III trial comparing AP with IP. Methods: Eligibility criteria included patients (pts) with chemotherapy-naïve, ED-SCLC, aged 20 to 70, and ECOG PS 0-1. Pts were randomized to receive either IP or AP, balancing for site, sex, and PS. IP comprised administration of I (60 mg/m2) iv on days 1, 8, and 15, and P (60 mg/m2) iv on day1,every 4 weeks. AP comprised administration of A (40 mg/m2) iv on day 1-3, and P (60 mg/m2) iv on day1, every 3 weeks. The planned sample size was 141 pts in each arm with a one-sided alpha of 5% and power of 70% and a non-inferiority margin of hazard ratio (HR) as 1.31. The primary endpoint was overall survival (OS). The secondary endpoints were response rate (RR), progression-free survival (PFS), adverse events (AEs), and quality of life (QOL). We evaluated pts’ QOL twice: at the baseline and after completion of the second course. Results: 284 pts were randomized to IP (n=142) and AP (n=142). Median age was 63, 84% were male, and 56% had PS 0. When 191pts enrolled, more febrile neutropenia (FN) was observed in AP than anticipated, and the initial dose of A was decreased from 40 mg/m2 to 35 mg/m2. At the second interim analysis conducted after the completion of patient accrual, the median OS of AP (15.0 m) was much worse than that of IP (18.3 m) and the HR (1.41; 96.3% CI, 1.03-1.93) exceeds even the non-inferiority margin, so the Data and Safety Monitoring Committee recommended early publication of the results. Median PFS was 5.7 (IP) vs. 5.2 months (AP) (HR 1.43, 95% CI, 1.13-1.82). RR was 69.5% (IP) vs. 77.9% (AP) (p=0.14). AEs in IP and AP arm were Grade 4 neutropenia (22.5% vs. 78.6%), G3-4 FN (10.7% vs. 32.1%), and G3-4 diarrhea (7.1% vs.1.4%). Proportion of improvement in physical status of QOL was 37.1%(IP) vs. 31.7%(AP), (odds ratio 0.72; 95%CI, 0.43-1.22; P=0.227). Conclusions: AP showed more bone marrow suppression than expected although it caused less diarrhea. The non-inferiority of AP to IP was not demonstrated and IP remains the standard treatment for ED-SCLC.

Effects of regorafenib therapy on health-related quality of life (HRQoL) in patients with metastatic colorectal cancer (mCRC) in the phase III CONCUR trial.

2015 ◽  
Vol 33 (3_suppl) ◽  
pp. 697-697 ◽  
Author(s):  
Shukui Qin ◽  
Tae Won Kim ◽  
Thomas Cheung Yau ◽  
Brigette Ma ◽  
Hongming Pan ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Progression Free Survival ◽  
Phase Iii ◽  
Life Questionnaire ◽  
Multikinase Inhibitor ◽  
Entire Treatment Period ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
Mean Time

697 Background: The CONCUR trial (NCT01584830) showed that the multikinase inhibitor regorafenib (REG) significantly improved overall survival and progression-free survival vsplacebo (PBO) in Asian patients with mCRC who had progressed on approved standard therapies. HRQoL was an exploratory endpoint. Methods: In this international multicenter trial conducted in Asia, patients were randomized 2:1 to receive REG 160 mg (n=136) or PBO (n=68) once daily for the first 3 weeks of each 4-week cycle. Prespecified QoL analyses were conducted on all 204 patients using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) and the EuroQol-five dimension questionnaire (EQ-5D). HRQoL outcomes were expressed as time-adjusted area under the curve (AUC) to allow descriptive evaluations of QoL in the REG and PBO groups across the entire treatment period. Individual domains were also compared using descriptive statistics. Results: Overall, changes in HRQoL were similar in the REG and PBO groups. Difference in least-squares (LS) mean time-adjusted AUC of the EORTC QLQ-C30 global health status/QoL (GH) score: −0.40 (95% CI: −3.53 to 2.72); differences in LS mean time-adjusted AUC for EQ-5D index and visual analog scale (VAS) scores: −0.03 (95% CI: −0.08 to 0.01) and −1.18 (95% CI: −4.01 to 1.66), respectively. The PBO group had <5 patients after cycle 3, so results should be interpreted with caution. Changes from baseline scores in cycles 2 and 3 did not appear to differ between REG and PBO on the 15 domains of the EORTC QLQ-C30, the EQ-5D index, and the VAS. Conclusions: No substantial differences in overall changes in HRQoL were seen between patients treated with REG and PBO in the CONCUR trial. Clinical trial information: NCT01584830. [Table: see text]

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