scholarly journals The Economic Impact of Compassionate Use of Medicines

2021 ◽  
Author(s):  
Claudio Jommi ◽  
Federico Pantellini ◽  
Lisa Stagi ◽  
Maria Verykiou ◽  
Marianna Cavazza
Keyword(s):  
Adverse Events ◽  
Economic Impact ◽  
Economic Benefit ◽  
Muscular Atrophy ◽  
Economic Value ◽  
Diagnostic Procedures ◽  
Compassionate Use ◽  
Cancer Treatments ◽  
Total Cost ◽  
Mean Cost

Abstract BACKGROUND: Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing earlier access to medicines to patients not recruited in trials. While the economic impact of clinical trials has been already investigated, no evidence on the net economic benefit of CUP exists. This research aims to fill the information gap by estimating the economic consequences of 11 CUP in Italy conducted between May 2015 and December 2020 from the perspective of health care payers. Eight programs concern cancer treatments, two refer to drugs for spinal muscular atrophy, and one is indicated for multiple sclerosis.METHODS: The net economic benefit includes the avoided costs from the Standard of Care (SoC) the patients would have received if they had not joined the CUP, and costs not covered by the pharmaceutical industry but instead sustained by payers, such as those associated to adverse events (only severe sides effects resulting in hospitalisation and attributed to CUP medicines), and 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: 2,712 patients were treated in the 11 CUP, where SoC was identified. The SoC mean cost per patient ranges 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 from €26.5 million to €50.6 million (€17.8 million - €42.0 million for cancer treatments). CONCLUSIONS: Despite research limitations, this paper illustrates for the first time the net economic impact of CUP in oncology patients from a 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.

scholarly journals The economic impact of compassionate use of medicines

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Claudio Jommi ◽  
Federico Pantellini ◽  
Lisa Stagi ◽  
Maria Verykiou ◽  
Marianna Cavazza
Keyword(s):  
Adverse Events ◽  
Economic Impact ◽  
Economic Benefit ◽  
Muscular Atrophy ◽  
Diagnostic Procedures ◽  
Public Health Care ◽  
Marketing Approval ◽  
Compassionate Use ◽  
Total Cost ◽  
Mean Cost

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.

scholarly journals Economic impact of compassionate use of medicines.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18840-e18840
Author(s):  
Claudio Jommi ◽  
Federico Pantellini ◽  
Lisa Stagi ◽  
Marianna Cavazza
Keyword(s):  
Economic Impact ◽  
Economic Value ◽  
Indirect Costs ◽  
Standard Of Care ◽  
Diagnostic Procedures ◽  
Cancer Drugs ◽  
Compassionate Use ◽  
Net Benefit ◽  
Total Cost ◽  
Mean Cost

e18840 Background: The economic impact of clinical trials in the perspective of trial sites has been already investigated. Instead, there is no evidence on the economic net benefit of compassionate use programs for medicines (CUP). This research aims to fill the information gap, investigating the economic consequences of 8 CUP in Italy carried out from May 2015 to December 2020 in the hospitals’ perspective. These programs concern five cancer medicines (alectinib, atezolizumab, cobimetinib, polatuzumab vedotin, trastuzumab emtansine TDM-1), for a total of 8 programs. Methods: Economic net benefit includes avoided costs for standard of care (SoC) the patient would have received if he/she has not joined the CUP and costs not covered by the pharmaceutical industry and sustained by the hospital hosting CUP. The latter include costs of adverse event (only severe sides effects generating hospitalisation and ascribed to medicines used in CUP), combination therapies and diagnostic procedures not covered by the sponsor. SoC costing relied on publicly available estimation. Adverse events and diagnostic procedures were retrieved from the CUP and monetized using the relevant fee for episode. Results: 1635 cancer patients were treated in eight CUPs. The SoC mean cost per patient ranges from €13355 to €28098 for all cancer drugs. The total cost of the SoC ranges from €21.8 - €45.9 million. The mean cost per patient covered by hospitals hosting CUP was equal to €2732 for cancer drugs, with a total cost of €4.5 million. The net economic benefit ranges from €17.4 million to €41.5 million for cancer treatments. Conclusions: Despite its limitations this paper illustrates for the first time the net economic impact of CUP in oncology patients in the perspective of payers. Additional evaluations are ongoing to better understand the overall effects of CUP implementation, i.e. the economic value of the comparative benefit profile of medicines used in CUP versus the SoC, including potential effects on indirect costs.

Economic Impact of Adverse Events in Chronic Myelogenous Leukemia: A Cost of Treatment Analysis of Dasatinib.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3309-3309 ◽  
Author(s):  
Sarah Y. Liou ◽  
Kimbach T. Tran ◽  
Jennifer M. Stephens ◽  
Nneka C. Onwudiwe ◽  
Marc F. Botteman
Keyword(s):  
Adverse Events ◽  
Economic Impact ◽  
Chronic Myelogenous Leukemia ◽  
Package Insert ◽  
Myelogenous Leukemia ◽  
Cost Of Treatment ◽  
Blast Phase ◽  
Total Cost ◽  
Grade 3

Abstract OBJECTIVES: With a greater focus on costs in today’s health care markets, it is important to evaluate overall costs and quality of care associated with new therapies in cost-effectiveness analyses. Adverse events (AEs) associated with cancer therapies often result in substantial economic costs and negative impacts on patient quality of life. AEs may also affect adherence to therapy and result in suboptimal treatment outcomes. Important AEs of dasatinib in chronic myelogenous leukemia (CML) patients who are resistant to imatinib include neutropenia, thrombocytopenia, anemia, gastrointestinal hemorrhage, central nervous system hemorrhage, and fluid retention leading to effusions and heart dysfunction. This study evaluated the total cost of treatment with dasatinib in patients with chronic, accelerated, and myeloid blast phase CML, taking into account both drug and AE-associated costs. METHODS: A literature-based economic model was developed to examine the short-term potential impact of AEs in patients with CML treated with dasatinib. The incidence rates of Grade 3/4 AEs were obtained from the SPRYCEL™ (dasatinib) package insert. The AEs were assumed to occur within the first 6 months of treatment. The costs associated with the AEs were obtained from published medical literature and the 2003 Healthcare Cost and Utilization Project database of mean hospital charges by principal diagnosis, converting to costs using a Medicare cost-to-charge ratio of 0.55. Dasatinib dosing assumptions were based on the FDA briefing documents of the SPRYCEL™ filing submission presented at the June 2, 2006 Oncology Drug Advisory Committee meeting. The initial dose for dasatinib was 70 mg twice daily based on the package insert. Patients were assumed to remain on treatment for 6 months. However, dose interruption of 7–14 days during the first 3 months of treatment and dose reduction to 50 mg twice daily in the second 3 months of treatment were assumed in 46–55% and 9–10% of patients, respectively. Drug costs were based on wholesale acquisition cost. Univariate and multivariate sensitivity analyses were conducted. All costs were adjusted to 2006 U.S. dollars using the medical care component of the consumer price index. RESULTS: In the base case analysis, the expected 6-month per-patient cost of treatment for dasatinib was $31,528 for chronic phase, $44,340 for accelerated phase, and $48,666 for myeloid blast phase. Six-month drug cost alone was $22,284 per patient in each phase. Grade 3/4 AEs accounted for approximately 29%, 50%, and 54% of the total cost of care for chronic, accelerated, and myeloid blast phases, respectively. Sensitivity analysis showed that the 6-month cost of Grade 3/4 AEs may range from $5,235–$10,696 for chronic, $16,109–$23,723 for accelerated, and $12,971–$27,551 for myeloid blast phases. Primary cost drivers were the hematologic AEs. CONCLUSIONS: This study provides an estimate for the total cost of treatment with dasatinib that considers treatment costs for severe AEs associated with therapy. These results highlight the importance of including treatment-related AEs in addition to drug costs when assessing the economic value of treatment options for CML. Future studies should expand the time horizon of the analysis and include recurring AEs to more comprehensively evaluate the overall economic impact of AEs with dasatinib.

Value-based estimate of market size and opportunity for economic benefit through innovative pancreatic cancer (PC) therapies.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e16790-e16790
Author(s):  
Vincent J. Picozzi ◽  
Victoria G. Manax ◽  
Semmie Kim ◽  
Zachary Wintrob ◽  
Margaret T. Mandelson ◽  
...  
Keyword(s):  
Economic Benefit ◽  
Economic Value ◽  
Cost Of Care ◽  
List Price ◽  
Total Cost ◽  
Average Value ◽  
Incidence Data ◽  
Treatment Prevalence ◽  
Life Years ◽  
Commercial Opportunity

e16790 Background: Over the past 20 years, cancer drugs have contributed to increased life expectancy, reduced mortality, decreased hospitalization and decreased use of medical services. The economic value of these improvements is about as large as the value of the increase in the US gross domestic product during that time period. Recently, a health economic study presented at ASCO GI 2020 cited that every $1 (adjusted for inflation) spent on innovative PC treatments reduced non-drug expenditures by $9, thereby lowering the total cost of care for PC patients. Accordingly, the commercial opportunity of a new therapy should be measured by some combination of the clinical, economic and social value generated. We demonstrate the value of a novel PC drug from this perspective. Methods: Analysis of SEER survival and incidence data between 2008 and 2016 shows the introduction of new medicines for PC of all stages was associated with a cumulative increase of 26,456 life years, or 2.52 life years per patient. It was also associated with quality of life improvements, measured by a decline in hospitalizations rates and emergency room visits that can also lead to more days at work, at school and with family. Several studies have suggested the average value of an additional year of life, for the age of a typical patient diagnosed with PC, is at least $250,000. Using this figure, the value of 26,456 life years gained from 2008-2016 is $6.61 billion (26,456*$250,000) to patients, the healthcare system and society, as a result of advancing medical innovation for patients with PC. Results: The median annual list price of a life-enhancing cancer therapy is $150,000 per patient. Using the NCI treatment prevalence estimator (holding incidence constant), we estimate that between 2020-2025, there will be an additional 10,728 advanced PC patients requiring treatment who could benefit from innovative drugs. The total cost of these drugs for these patients would be $1.61 billion. However, the economic value of the life years saved would be $6.76 billion (10,728*2.52 life years*$250,000 = $6.76 billion). A review of cancer medicine payor coverage suggests a new PC therapy that produces such value would be able to obtain coverage from US payors given this value-based price. Conclusions: A value-based approach to estimating the opportunity for clinical and economic benefit reveals significant potential for new PC medicines.

Economic Impact of Sugarcane Variety CO-86032 on Farmers Economy in Maharashtra

2020 ◽  
pp. 354-359
Keyword(s):  
Economic Impact ◽  
Production Technology ◽  
Economic Benefit ◽  
Extreme Rainfall ◽  
Sugar Recovery ◽  
Sugarcane Variety ◽  
Public Research ◽  
Productivity Improvement ◽  
Farming Community ◽  
The Government

The area under sugarcane in Maharashtra state was found to be more stable and consistent rather than production and productivity. It may be due to the F & RP of sugarcane. In the year 1996, MPKV, Rahuri released a promising variety of sugarcane viz., Co-86032 which is very famous in farming community due to its hardiness, sugar recovery (percent) and resistance to the extreme rainfall as well as deficit rainfall. The total economic worthiness of university released sugarcane variety Co-86032(production technology) over other competing varieties of sugarcane in the Maharashtra was `51449.14per ha. The sugarcane growers in Maharashtra state earned net economic benefit of `11059.40 crores from improved sugarcane variety Co-86032. Therefore, it is suggested that the Government should allocate substantial funds to public research in sugarcane for productivity improvement.

EM2 Economic IMPACT of Compassionate Use of Medicines

2021 ◽  
Vol 24 ◽  
pp. S7
Author(s):  
C. Jommi ◽  
F. Pantellini ◽  
L. Stagi ◽  
M. Cavazza
Keyword(s):  
Economic Impact ◽  
Compassionate Use

PMU41 Clinical Pharmacy Provider Dashboards May Prevent Avoidable Adverse Events and Reduce Total Cost of Care

2021 ◽  
Vol 24 ◽  
pp. S151-S152
Author(s):  
E. Lucas ◽  
Snow LM Tyndall ◽  
B. Mohundro ◽  
W.E. Seggerman ◽  
M. Carby ◽  
...  
Keyword(s):  
Adverse Events ◽  
Clinical Pharmacy ◽  
Cost Of Care ◽  
Total Cost

scholarly journals Potato Late Blight in the Columbia Basin: An Economic Analysis of the 1995 Epidemic

Plant Disease ◽  
1997 ◽  
Vol 81 (1) ◽  
pp. 103-106 ◽  
Author(s):  
D. A. Johnson ◽  
T. F. Cummings ◽  
P. B. Hamm ◽  
R. C. Rowe ◽  
J. S. Miller ◽  
...  
Keyword(s):  
Late Blight ◽  
Management Practice ◽  
Columbia Basin ◽  
Copper Hydroxide ◽  
Total Cost ◽  
Late Season ◽  
The Mean ◽  
The Cost ◽  
Mean Cost ◽  
First Time

The cost of managing late blight in potatoes during a severe epidemic caused by new, aggressive strains of Phytophthora infestans in the Columbia Basin of Washington and Oregon in 1995 was documented. The mean number of fungicide applications per field varied from 5.1 to 6.3 for early- and midseason potatoes, and from 8.2 to 12.3 for late-season potatoes in the northern and southern Columbia Basin, respectively. In 1994, a year when late blight was not severe, the mean number of fungicide applications per field made to early- and midseason potatoes was 2.0; whereas late-season potatoes received a mean of 2.5 applications. The mean per acre cost of individual fungicides applied varied from $4.90 for copper hydroxide to $36.00 for propamocarb + chlorothalonil. Total per acre expenses (application costs plus fungicide material) for protecting the crop from late blight during 1995 ranged from $106.77 to $110.08 for early and midseason potatoes in different regions of the Columbia Basin and from $149.30 to $226.75 for lateseason potatoes in the northern and southern Columbia Basin, respectively. Approximately 28% of the crop was chemically desiccated before harvest as a disease management practice for the first time in 1995, resulting in an additional mean cost of $34.48/acre or $1.3 million for the region. Harvested yields were 4 to 6% less than in 1994. The total cost of managing late blight in the Columbia Basin in 1995 is estimated to have approached $30 million.

scholarly journals Rheumatological Adverse Events Following Immunotherapy for Cancer

Medicina ◽  
2022 ◽  
Vol 58 (1) ◽  
pp. 94
Author(s):  
Ioana Cretu ◽  
Bogdan Cretu ◽  
Catalin Cirstoiu ◽  
Adrian Cursaru ◽  
Mihaela Milicescu ◽  
...  
Keyword(s):  
Side Effects ◽  
Adverse Events ◽  
Adverse Reactions ◽  
Cancer Treatments ◽  
Treatment Conditions ◽  
Clinical Presentations ◽  
Rheumatic Manifestations ◽  
Grade 3 ◽  
Dose Dependent ◽  
Rheumatological Manifestations

Background and Objectives: The occurrence of rheumatological side effects in a patient after receiving immunotherapy for cancer is becoming increasingly common. Oncologists often fail to diagnose and refer affected patients to rheumatologists. This paper presents the various rheumatological adverse events that occur after immunotherapy in patients as well as their treatment and evolution. Materials and Methods: A total of 36 patients were monitored between November 2018 and March 2020. The oncologist monitoring the immunotherapy-treated patients identified the occurrence of musculoskeletal side effects. The grading of toxicities was performed by both the oncologist and the rheumatologist using common terminology criteria for adverse events (CTCAE). Rheumatological treatment was administered, and for some patients, immunotherapy was discontinued. Results: The clinical presentations of the patients varied. Mild side effects (grade 1–2) were reported in a higher proportion than severe side effects (grade 3–5). Therefore, thirty-one patients had mild-to-moderate side effects, and five patients had severe side effects. Adverse reactions occurred, on average, 10 weeks after the initiation of immunotherapy; this indicated that the severity of the toxicity was dose dependent. Patients were treated with NSAIDs or prednisone, depending on the severity of the side effects, and for patients with severe manifestations, immunotherapy was discontinued. The remission of rheumatic manifestations varied depending on the grade of the manifestations. Conclusions: The clinical, biological, and ultrasound presentations of the patients with adverse events followed by cancer treatments differed from classic rheumatological manifestations. Thorough examinations of these patients by both oncologists and rheumatologists are needed in order to correctly diagnose and treat rheumatological adverse events. Multiple studies that include a larger number of participants are needed in order to better understand the pathogenesis and clinical evolution of these patients under different treatment conditions.

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