The correlation between clinical benefit and financial cost of cancer drugs.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 7071-7071
Author(s):  
Aaron Philip Mitchell ◽  
Sara Tabatabai ◽  
Pranammya Dey ◽  
Jennifer Ohn ◽  
Michael A. Curry ◽  
...  
Keyword(s):  
Clinical Benefit ◽  
Treatment Cost ◽  
Treatment Costs ◽  
Value Assessment ◽  
Cancer Drugs ◽  
Cancer Treatments ◽  
Quality Of Evidence ◽  
Treatment Indications ◽  
The Cost

7071 Background: The cost of many cancer drugs is very high, but it is unclear if these costs are associated with commensurate improvement in outcomes. We aimed to assess the association between the cost of cancer treatments and their clinical benefit, using the NCCN Evidence Blocks value assessment framework. Methods: The NCCN Evidence Blocks include 4 measures of clinical benefit: Efficacy, Safety, Quality of Evidence, and Consistency of Evidence. The NCCN assigns scores on each measure ranging from 1 (least favorable) to 5 (most favorable). We obtained the NCCN Evidence Blocks scores as of December 31, 2018 for all recommended cancer treatments for the 30 most prevalent cancers in the US. For each treatment, we calculated total treatment costs (including drugs, administration fees, and supportive care medications) using Medicare reimbursement rates. We categorized treatments as either “time-limited” or “time-unlimited” according to whether their costs are best reflected as per full treatment course (often, adjuvant/neoadjuvant treatments) (time-limited) or per month of therapy (often, treatments for advanced disease) (time-unlimited). We used generalized estimating equations, with clustering within treatment indications, to estimate the association between Evidence Blocks scores and treatment costs, modeling the expected change in cost associated with a one-unit increase in the score on an Evidence Blocks measure. Results: There were 541 time-unlimited and 845 time-limited treatments. Among time-unlimited treatments, monthly treatment cost ranged from $4 to $64,630. Monthly treatment cost was positively associated with Efficacy ($3,036, 95%CI: $1,782, $4,289) and Quality of Evidence ($1,509, 95%CI: $171, $2,847) but negatively associated with Safety (-$1,470, 95%CI: -$2,790, -$151) and Consistency of Evidence (-$2,003, 95%CI -$3,420, -$586). Among time-limited treatments, cost per course of therapy ranged from $0 to $775,559, and no measure was significantly associated with cost. Evidence Blocks scores accounted for little of the variation in treatment cost (linear model R-squared = 0.10 for time-unlimited, and < 0.01 for time-unlimited). Conclusions: The association between NCCN Evidence Blocks measures and treatment cost was inconsistent, and accounted for little of the cost variation among treatments for the same indication. The clinical benefit of cancer treatments does not appear to be a primary determinant of treatment cost, suggesting that current pricing models may be inadequate to incentivize the development and utilization of high-value treatments.

scholarly journals Association Between Clinical Value and Financial Cost of Cancer Treatments: A Cross-Sectional Analysis

2020 ◽  
Vol 18 (10) ◽  
pp. 1349-1353
Author(s):  
Aaron P. Mitchell ◽  
Sara M. Tabatabai ◽  
Pranammya Dey ◽  
Jennifer A. Ohn ◽  
Michael A. Curry ◽  
...  
Keyword(s):  
Cancer Treatment ◽  
Treatment Cost ◽  
Drug Acquisition ◽  
The United States ◽  
Clinical Value ◽  
Cancer Treatments ◽  
Cross Sectional ◽  
Quality Of Evidence ◽  
The Cost

Background: The cost of cancer treatment has increased significantly in recent decades, but it is unclear whether these costs have been associated with commensurate improvement in clinical value. This study aimed to assess the association between the cost of cancer treatment and 4 of the 5 NCCN Evidence Blocks (EB) measures of clinical value: efficacy of regimen/agent, safety of regimen/agent, quality of evidence, and consistency of evidence. Methods: This is a cross-sectional, observational study. We obtained NCCN EB ratings for all recommended, first-line, and/or maintenance treatments for the 30 most prevalent cancers in the United States and calculated direct pharmacologic treatment costs (drug acquisition, administration fees, guideline-concordant supportive care medications) using Medicare reimbursement rates in January 2019. We used generalized estimating equations to estimate the association between NCCN EB measures and treatment cost with clustering at the level of the treatment indication. Results: A total of 1,386 treatments were included. Among time-unlimited treatments (those administered on an ongoing basis without a predetermined stopping point), monthly cost was positively associated with efficacy ($3,036; 95% CI, $1,782 to $4,289) and quality of evidence ($1,509; 95% CI, $171 to $2,847) but negatively associated with safety (–$1,470; 95% CI, –$2,790 to –$151) and consistency of evidence (–$2,003; 95% CI, –$3,420 to –$586). Among time-limited treatments (those administered for a predetermined interval or number of cycles), no NCCN EB measure was significantly associated with treatment cost. Conclusions: An association between NCCN EB measures and treatment cost was inconsistent, and the magnitude of the association was small compared with the degree of cost variation among treatments with the same EB scores. The clinical value of cancer treatments does not seem to be a primary determinant of treatment cost.

A weighted criterion-based approach to value assessment of oncology drugs.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 6627-6627
Author(s):  
Doreen Anuli Ezeife ◽  
Ellen R. Cusano ◽  
Aline Fusco Fares ◽  
Mike Sung ◽  
Francois Dionne ◽  
...  
Keyword(s):  
Clinical Benefit ◽  
Unmet Need ◽  
Progression Free Survival ◽  
Well Being ◽  
Sensitivity Analyses ◽  
Supporting Evidence ◽  
Value Assessment ◽  
Cancer Treatments ◽  
Novel Approach

6627 Background: The rising cost of anti-cancer therapy has motivated recent efforts to quantify the overall value of new cancer treatments. Multi-criteria decision analysis offers a novel approach to establish an explicit framework to evaluate new cancer treatments. Methods: We recruited a diverse multi-stakeholder group who identified and weighted key criteria to establish the Drug Assessment Framework (DAF). Strength of evidence (SOE) modifiers deducted points for lower quality evidence. Through one-on-one meetings with stakeholders, face and content validity of the DAF were established in an iterative process. Construct validity assessed the degree to which DAF scores were associated with the pan-Canadian oncology drug review (pCODR) funding decisions and European Society for Medical Oncology Magnitude of Clinical Benefit score (ESMO-MCBS, version 1.1). Sensitivity analyses were performed on the final results. Results: The final validated DAF includes ten criteria: overall survival, progression-free survival, response rate, quality-of-life, toxicity, unmet need, equity, feasibility, disease severity and caregiver well-being. The first five clinical benefit criteria represent 64% of the total weight. DAF scores range from 0 to 300, reflecting both the expected impact of the drug and the quality of the supporting evidence. When the DAF was retrospectively applied to the last 60 drugs (in blinded fashion) reviewed by pCODR (2015-2018), the mean total DAF score was 94 (range, 18-179). Drugs with positive pCODR funding recommendation had higher DAF scores than drugs not recommended for reimbursement (103 vs. 63, t-test p = 0.0007). Funded drugs had fewer SOE points deducted than those that were not funded (median 0 vs. 24 points deducted, Wilcoxon p = 0.03). The correlation coefficient for DAF and ESMO-MCBS was 0.37 (95% CI, 0.10 to 0.59). Sensitivity analyses that varied the subjective criteria either positively or negatively did not change the results. Conclusions: Using a structured and explicit approach, a criterion-based valuation framework was designed and validated. The DAF can provide a transparent and consistent method to value and prioritize cancer drugs, in order to facilitate the delivery of affordable cancer care.

Overall survival, quality of life and magnitude of clinical benefit of breast cancer drugs over the last 25 years.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 6571-6571
Author(s):  
J. Carlos Tapia ◽  
Aida Bujosa Rodríguez ◽  
Consolacion Molto ◽  
Arnoud J. Templeton ◽  
Daniel Shepshelovich ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Clinical Benefit ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Curative Intent ◽  
Substantial Clinical Benefit ◽  
Breast Cancer Drugs ◽  
Palliative Setting ◽  
Over Time

6571 Background: The American Society of Clinical Oncology Cancer Research Committee (ASCO-CRC), the ASCO Value Framework Net Health Benefit score version 2 (ASCO-VF), and the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS) are validated tools quantifying the clinical benefit for cancer drugs. Here, we assess the overall survival (OS), quality of life (QoL) and magnitude of clinical benefit of trials supporting breast cancer drug approval by the US Food and Drug Administration (FDA) in the last 25 years. Methods: We searched Drugs@FDA website for all breast cancer drug approvals from January 1, 1995 to December 30, 2020. Drug labels and reports of registration trials were reviewed. We collected data on trial characteristics, efficacy, toxicity and QoL. When more than one study supported a single indication, we preferred efficacy-oriented endpoints (typically OS) to QoL. We excluded trials supporting accelerated-approval indications if these were converted to regular approval during the study period. We scored clinical benefit using the ASCO-CRC in palliative setting, and ASCO-VF and ESMO-MCBS in both curative and palliative setting. Substantial clinical benefit was defined as: OS gains ≥2.5 months and progression-free survival gains ≥ 3 months for ASCO-CRC criteria; ASCO-VF scores ≥ 45 and grade of A or B for trials of curative intent and 4 or 5 for those of non-curative intent using ESMO-MCBS. Trends over time were assessed using Chi-squared test for trend. Results: We identified 51 trials supporting the approval of 32 individual drugs for 51 indications; 12 (24%) were in the curative setting and 39 (76%) in the palliative setting. At the time of approval, 8 (16%) trials reported significant improvement in OS. QoL was reported in 22 trials (43%). Among these, 8 (36%) showed improvement in QoL. For curative intent, we applied ASCO-VF and ESMO-MCBS score to 11 (92%) trials, finding clinical benefit in 10 (91%) and 2 (18%) trials, respectively. In the palliative setting, we used ASCO-CRC, ASCO-VF and ESMO-MCBS scores to rate 32 (82%), 33 (85%) and 38 (97%) trials. Substantial clinical benefit was observed in 20 (63%), 12 (36%) and 7 (19%) trials, respectively. Over time, there has been a decrease in the number of trials supporting approval based on OS (1996-2003 50% vs 2004-12 38% vs 2013-20 13%, P trend = 0.033). There were no statistically significant changes over time in QoL, ASCO-CRC, ASCO-VF and ESMO-MCBS scores. Conclusions: For palliative intent, most trials supporting FDA approval of breast cancer drugs do not meet the ASCO-VF or ESMO-MCBS criteria for substantial clinical benefit. There is substantial inter-framework variability in the assessment of clinical benefit in the curative setting. Over time, there has been a substantial shift towards use of surrogate endpoints as the basis for approval without a clear improvement in substantial clinical benefit.

The Cost of HIT-Safe Anticoagulant Use at a Tertiary Care, Adult, Academic Hospital.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 1293-1293
Author(s):  
Natasha Nanwa ◽  
Nicole Mittmann ◽  
Sandra Knowles ◽  
Claudia Bucci ◽  
Rita Selby ◽  
...  
Keyword(s):  
Optical Density ◽  
Treatment Cost ◽  
Treatment Costs ◽  
Thrombin Inhibitors ◽  
Management Policy ◽  
Enzyme Linked Immunosorbent Assay ◽  
Academic Hospital ◽  
Average Treatment ◽  
Medication Costs ◽  
The Cost

Abstract Objective: Heparin-induced thrombocytopenia (HIT) is a potential adverse outcome of heparin therapy. At Sunnybrook Health Sciences Centre (SHSC), a patient suspected of HIT has heparin discontinued, a HIT enzyme-linked immunosorbent assay (ELISA) ordered, and is then treated with a HIT-safe anticoagulant. Few studies have assessed the cost of treating suspected (negative and confirmed) HIT. The objective of our study was to quantify the direct costs associated with treating HIT from a Canadian hospital perspective. Methods: A cost analysis was conducted at SHSC, a 701-bed academic hospital with cardiac surgery. Suspected HIT included all patients who had a HIT ELISA ordered. A confirmed HIT case had one of the following: positive serotonin release assay (SRA), positive HIT ELISA (optical density: 0.4 to 1.0) plus high clinical probability for HIT, or strongly positive HIT ELISA (optical density &gt; 1.0). A negative HIT case had a negative HIT ELISA or SRA. Costs associated with using HIT-safe anticoagulants included: drug acquisition, preparation time, monitoring tests, and the management of bleeding (e.g., blood transfusions). The average treatment cost (2007 Canadian dollars) per case of confirmed HIT, confirmed HIT with thrombosis (HITT), and negative HIT was calculated. Cost data was obtained from the hospital human resources, pharmacy database, and laboratory. Results: There were 108 suspected HIT cases in 2005. Thirty-one of the suspected HIT cases were treated: 12 out of 88 negative HIT cases, 7 out of 8 confirmed HIT cases, and all 12 confirmed HITT cases (Table). Six cases were treated with more than one HIT-safe anticoagulant (one negative HIT case and five confirmed HITT cases). Lepirudin accounted for 64% of the total HIT-safe medication costs in 2005. Overall, the direct thrombin inhibitors (DTIs) accounted for 96% of the treatment costs. Seventeen patients were treated with fondaparinux, eight of whom were negative HIT cases. Cases with confirmed HITT had greater treatment costs per patient than those with confirmed HIT. The average treatment cost of a negative HIT case was $64 ($0–$3,987). Conclusions: The treatment costs presented appear to be lower than costs presented in other studies. This may, in part, be the result of a hospital HIT management policy and a comprehensive TE Service that manages all cases of HIT. The total costs and the cost per patient were greatest for the DTIs. Table: HIT-safe anticoagulant use. Negative HIT Confirmed HIT Confirmed HITT Total cost of use NA - not applicable, SD - standard deviation, 1One patient received both lepirudin and fondaparinux, 2 One patient died before receiving any treatment, 3 Five patients received more than one HIT-safe anticoagulant Average argatroban cost ± SD (range) $3,987&#x2028; n = 1 $0&#x2028; n = 0 $4,023 ± $4,211 ($693 – $8,757)&#x2028; n = 3 $16,056 Average bivalirudin cost ± SD (range) $0&#x2028; n = 0 $9,302&#x2028; n = 1 $0&#x2028; n = 0 $9,302 Average lepirudin cost ± SD (range) $221&#x2028; n = 1 $3,562 ± $23 ($3,545 – $3,578)&#x2028; n = 2 $4,793 ± $4,842 ($1,099 – $16,744)&#x2028; n = 9 $50,482 Average danaparoid cost ± SD (range) $228 ± $135 ($90 – $359)&#x2028; n = 3 $0&#x2028; n = 0 $54&#x2028; n = 1 $738 Average fondaparinux cost ± SD (range) $91 ± $58 ($28 – $196)&#x2028; n = 8 $81 ± $67 ($42 – $182)&#x2028; n = 4 $263 ± $100 ($140 – $365)&#x2028; n = 5 $2,367 Total number treated 131 72 183 NA Average HIT-safe anticoagulant cost ± SD (range) $64 ± $428 ($0 – $3,987)&#x2028; Median = $0&#x2028; n = 88 $2,094 ± $3,314 ($0 – $9,302)&#x2028; Median = $119&#x2028; n = 8 $4,715 ± $4,776 ($252 – $16,744)&#x2028; Median = $3,156&#x2028; n = 12 $78,945

scholarly journals What Happens when the Cost of Cancer Care Becomes Unsustainable?

2017 ◽  
Vol 13 (02) ◽  
pp. 108 ◽  
Author(s):  
Steven Simoens ◽  
Wim van Harten ◽  
Gilberto Lopes ◽  
Arnold Vulto ◽  
Klaus Meier ◽  
...  
Keyword(s):  
Cancer Care ◽  
Decision Makers ◽  
Incremental Innovation ◽  
Cancer Drugs ◽  
Cancer Treatments ◽  
Access To Treatment ◽  
Scarce Resources ◽  
Patients With Cancer ◽  
Heavy Burden ◽  
The Cost

Cancer places a heavy burden on healthcare systems. The cost of cancer drugs is increasing, driven largely by the introduction of new, ever more innovative cancer treatments. This raises questions about value for money and the future sustainability of cancer care, and presents significant challenges for decision-makers in providing all patients with access to treatments and effective new cancer medicines. The aims of this article are to provide an understanding of how sustainability in cancer care is defined, what signs indicate that the limits of sustainability are being reached, and what potential impact this may have on patients with cancer within Europe. Each country is faced with making difficult decisions about the allocation of healthcare resources to cancer care, to best meet the health needs of their patients. Determining the value of individual cancer drugs can help to inform these decisions, because premium pricing for incremental innovation is no longer sustainable. When the cost of cancer care becomes unsustainable, countries may be forced to restrict health expenditure by limiting demand, cutting spending and reducing investment. This can lead to restricted access to treatment. New, innovative cancer treatments must provide greater value than current options, and measures are needed to contain and reduce expenditure and make best use of scarce resources, without impeding access to effective and safe treatments for all patients.

UPDATE: Should Hydroxychloroquine (HCQ) or Chloroquine (CQ) be used in the treatment of COVID-19?

2020 ◽  
Vol 54 ◽  
Author(s):  
Lia M. Palileo-Villanueva ◽  
Elenore Judy B. Uy
Keyword(s):  
Clinical Benefit ◽  
Common Adverse Event ◽  
Hospitalized Patients ◽  
Efficacy And Safety ◽  
Quality Of Evidence ◽  
Randomized Controlled ◽  
The Us ◽  
Us Fda ◽  
Meta Analyses

KEY FINDINGS There is insufficient evidence to support the routine use of HCQ or CQ for the treatment of COVID-19. Results from interim analyses of 2 large RCTs, the Recovery and the Solidarity trials, reportedly showed no clinical benefit from HCQ for hospitalized patients with COVID-19. There are 3 randomized controlled trials that investigated the efficacy and safety of HCQ compared to standard therapy. Overall quality of evidence was very low. Meta-analyses from the “COVID-19 Living Data” project suggests that the use of HCQ may increase the incidence of adverse events at day 14 to day 28 (RR 2.49, 95% confidence interval: 1.04 to 5.98, moderate quality of evidence); the most common adverse event across the two trials is diarrhea (n=8). In a statement dated June 5, 2020, the investigators of the Recovery trial announced their decision to halt further enrollment to the HCQ arm of the trial because an interim analysis showed no clinical benefit from the use of HCQ in hospitalized patients with COVID. On June 15, 2020, the US FDA revoked the emergency use authorization for HCQ and CQ as treatment for COVID-19. On June 18, 2020, the WHO announced that recruitment to the HCQ arm of the Solidarity trial has been halted.

Development of an MCDA framework for evaluation and positioning of oncological treatments in clinical practice.

2019 ◽  
Vol 37 (27_suppl) ◽  
pp. 95-95
Author(s):  
Carlos Camps ◽  
Xavier Badia ◽  
Divya Chugani ◽  
Rosario García Campelo ◽  
Jesus Garcia-Foncillas ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Decision Making ◽  
Clinical Practice ◽  
Treatment Cost ◽  
Unmet Needs ◽  
Cost Comparison ◽  
Expert Group ◽  
Response Scale ◽  
Quality Of Evidence

95 Background: Ensuring that effective innovations are accessible in a timely and affordable manner to all cancer patients is a challenge that stakeholders face today. Several oncology frameworks (ASCO, ESMO, ICER, NCCN) have been developed to define and quantify the value of oncological therapies to support clinicians and patients at the time of selection and as a basis for decision-making. However, current frameworks only define treatment value in terms of clinical benefit, creating a need for one that allows holistic evaluation of treatments and supports decision-making in clinical practice. The ECO Foundation led this study to develop a reflective multi-criteria decision analysis (MCDA) based framework for evaluation and positioning of new oncological drugs from the clinical oncology perspective. Methods: The framework was developed following EVIDEM methodology. Systematic literature review was performed to identify most relevant criteria used for evaluation of innovative treatments to complement the EVIDEM V4.0 framework. The criteria compendium was presented to a group of clinical oncologists from ECO. The Expert Group assessed each criterion for its inclusion in the framework and suggested modifications in their definition and/or response scale. This framework was then tested with two case studies (Abemaciclib for metastatic HER-/HR+ breast cancer and TAS for metastatic colorectal cancer). The objective was to validate the criteria selected alongside their definitions and response scale with practical examples. A reflective discussion based on the score assigned to each criterion was also carried out. Results: Out of 15 criteria presented to the Expert Group, 8 were included in the final framework, and definition and/or response scale of 7 of these criteria were modified: Disease severity, Unmet needs, Efficacy comparison, Safety/tolerability comparison, Treatment intention, Treatment cost comparison, Comparison of other medical costs and Quality of evidence. Conclusions: A reflective MCDA framework has been developed and validated, based on the value of the treatment, for assessment and positioning of oncological therapies in the context of clinical practice in Spain.

The DISCO App: A pilot test of an electronic patient intervention to reduce the financial burden of cancer through improved cost communication.

2020 ◽  
Vol 38 (29_suppl) ◽  
pp. 1-1
Author(s):  
Lauren M. Hamel ◽  
David W. Dougherty ◽  
Theresa A. Hastert ◽  
Erlene Kuizon Seymour ◽  
Seongho Kim ◽  
...  
Keyword(s):  
Self Efficacy ◽  
Treatment Cost ◽  
Financial Burden ◽  
Controlled Trial ◽  
Treatment Costs ◽  
Patient Acceptance ◽  
Financial Toxicity ◽  
Post Intervention ◽  
Intervention Measures ◽  
The Cost

1 Background: Financial toxicity, the burden of treatment cost, affects 30-50% of people with cancer in the US. Although experts recommend patients and oncologists discuss treatment cost to identify patients who need assistance, cost discussions occur in fewer than half of cancer treatment discussions. We pilot-tested the feasibility and efficacy of the Discussions of Cost (DISCO) App, a patient communication intervention designed to improve cost discussions and other financial toxicity-related outcomes during and following oncology treatment consultations. The DISCO App provides an individualized list of cost-related questions patients can ask their oncologist, specific to a patient’s economic situation. Methods: While waiting to see their oncologist, newly diagnosed patients with breast or lung cancer (n=32) used the DISCO App on an iPad. Clinic visits were videorecorded and patients completed pre- and post-intervention measures of self-efficacy for managing treatment costs, self-efficacy for interacting with oncologists, cost-related distress, and perceptions of the DISCO App. A trained coder observed the recordings to determine the presence of a cost discussion, the cost-related topic, and any emergent factors. Results: Findings showed increases in patients’ self-efficacy for managing treatment costs (p=.02) and interacting with oncologists (p=.001). Cost-related distress decreased but not significantly (p=.20). Patients reported the DISCO App was understandable (M=4.5 out of 5), useful as they talked with their oncologist (M=4.0), and 84% of patients reported needing less than 15 minutes to use the DISCO App. Most (94%) interactions were videorecorded (in two cases technical difficulties prevented videos from being collected); all (100%) of the videorecorded interactions included a cost discussion. The most frequently discussed topics were: insurance, time off from work, and financial navigation. Frequently, the oncologist asked the patient for his/her question list and discussed/answered the questions. Conclusions: Findings suggest the DISCO App is feasible to implement in the clinic and effective in improving patient-oncologist cost discussions and financial toxicity-related outcomes. Patient acceptance of the DISCO App and oncologist engagement suggested the intervention prompted cost discussions. Next steps include conducting a longitudinal randomized controlled trial to determine the effectiveness of the DISCO App on financial toxicity, and other outcomes. Clinical trial information: NCT03676920 .

scholarly journals Bénéfice clinique et coût des traitements anticancéreux

2020 ◽  
Vol 36 (11) ◽  
pp. 1095-1097
Author(s):  
Bertrand Jordan
Keyword(s):  
Detailed Analysis ◽  
Clinical Benefit ◽  
Treatment Costs ◽  
European Countries ◽  
Cancer Drugs ◽  
Weekly Treatment ◽  
Assessment Systems ◽  
The Usa ◽  
Traitements Anticancéreux

A detailed analysis of the clinical benefit for 47 approved cancer drugs, using two internationally recognized assessment systems, shows essentially no correlation between clinical benefit and weekly treatment costs. This is true both in the USA and in four European countries, although prices are dramatically lower in Europe.

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