Reimbursement Of Cancer Drugs Research Articles

Factors influencing the reimbursement of cancer drugs in Europe: A scoping review.

Reimbursement process of oncology drugs in Europe occurs within a complex decision-making process that varies between Member States. Distinctions between the States trigger societal debates since it is necessary to balance access to medicines and health systems sustainability. We aimed to review the evidence concerning factors associated with the reimbursement decision or Health Technology Agency recommendation of oncology drugs in Europe. A systematic literature search was performed in two databases from inception to august 2023. Screening and data extraction were performed by pairs. Thirteen articles were included and encompassed data from 11 nations. Seven articles showed that cost-effective (C-E) drugs and lower Incremental Cost-Effectiveness Ratios (ICERs) had higher likelihood of reimbursement. Disease severity might influence the reimbursement decision with financial agreements. Improvement in clinical outcomes, substantial clinical benefit (p < 0.01) or overall survival gains (p < 0.05) were positively associated. Orphan drug designation impact varies between countries but positive decisions are usually achieved under specific conditions. Clinical and C-E uncertainty frequently led to reimbursement with financial agreements or outcomes-based conditions. Sociodemographic factors as: social health insurance system, higher Gross Domestic Product and larger elderly population were positively associated with reimbursement (p < 0.01). There is a need for further research into key determinants of reimbursement decisions in Europe and the development of drug access models that can effectively address and overcome costs and effectiveness uncertainties.

Disagreement on cancer drug decisions in Europe.

Despite the efforts of the European Union (EU) to promote voluntary cooperation among Health Technology Assessment (HTA) agencies, different reimbursement decisions for the same drug are made across European countries. The aim of this paper is to compare the agreement of cancer drug reimbursement decisions using inter-rater reliability measures. This study is based on primary data on 161 cancer drug reimbursement decisions from nine European countries from 2002 to 2014. To achieve our goal, we use two measures to analyze agreement, in other words, congruency: (i) percentage of agreement and (ii) the κ score. One main conclusion can be drawn from the analysis. There is a weak to medium agreement among cancer drug decisions in the European countries analyzed (based on the percentage of agreement and the κ score). England and Scotland show the highest consistency between the two measures, showing a medium agreement. These results are in line with previous literature on the congruency of HTA decisions. This paper contributes to the HTA literature, by highlighting the extent of weak to medium agreement among cancer decisions in Europe.

Examining the relationship between the clinical benefit of oncology drug indications and the time from pan-Canadian Oncology Drug Review (pCODR) recommendation to public reimbursement.

e19360 Background: We examined if publicly reimbursed oncology drug indications with evidence of high clinical benefit, as measured by the American Society of Clinical Oncology Value Framework v2 (ASCO-VF), and European Society for Medical Oncology Magnitude of Clinical Benefit Scale v1.1 (ESMO-MCBS), received reimbursement status faster than those with lower clinical benefit from the time of pCODR recommendation. Methods: Oncology drug indications that received pCODR recommendations between Jan 2012 and July 2018 were identified. Indications that did not receive provincial reimbursement, without notice of compliance, or received a negative pCODR recommendation were excluded. The relationship between clinical benefit, as measured by ASCO-VF and ESMO-MCBS, and the time to reimbursement was evaluated using Spearman correlation coefficient, univariable, and multivariable linear regression analyses. Results: Overall, 84 indications met inclusion criteria yielding 80 ASCO-VF and 66 ESMO-MCBS scores. The mean ASCO-VF and ESMO-MCBS scores were 38.8 (SD = 23.8) and 3.0 (SD = 1.1) respectively. Higher ASCO-VF and ESMO-MCBS scores had low correlation with shorter time to provincial funding, (rho = -0.15, 95%CI -0.24, -0.06) and (rho = -0.25, 95%CI -0.34, -0.16) respectively. Univariable analyses showed that manufacturer reported incremental cost effectiveness ratio (ICER) values, year of pCODR recommendation, province and cancer type were associated with time to public reimbursement (all p &lt; 0.0001). After adjusting for potential confounders in the respective multivariable analysis, ASCO-VF (p = 0.29) and ESMO-MCBS (p = 0.15) scores were not significantly associated with time to public reimbursement. Year of pCODR recommendation remained associated with time to public reimbursement (p &lt; 0.001). Earlier years (2012-2014) had a shorter time to reimbursement (mean = 10.4 months) than later years (2015-2018) (mean = 14.5 months). Other factors that were associated with time to reimbursement in multivariable analysis were province (p &lt; 0.001) and cancer type (p &lt; 0.001). Conclusions: Currently, oncology drug indication with evidence of high clinical benefit do not appear to be funded faster than those with low clinical benefit. This suggests the need to prioritize cancer drug indications based on clinical benefit in order to allow for timely public reimbursement of cancer drugs with higher clinical benefit to patients.

Health-related quality of life in oncology drug reimbursement submissions in Canada: A review of submissions to the pan-Canadian Oncology Drug Review.

In Canada, the Canadian Agency for Drugs and Technologies in Health (CADTH) evaluates and makes recommendations for the reimbursement of cancer drugs. One component of its recommendation is based on an economic evaluation, which typically takes the form of a cost-utility analysis. A cost-utility analysis measures the effects of competing therapies with quality-adjusted life-years (QALYs). The data for this calculation typically come from generic, preference-based measures of health-related quality of life (HRQOL). The objective of this review is to determine the frequency at which HRQOL data are collected alongside cancer drug trials and used in the cost-utility analysis submitted to theCADTH pan-Canadian Oncology Drug Review (pCODR). Submissions between 2015 and 2018 to pCODR, the group charged with evaluating cancer drug submissions at CADTH, were reviewed. All pCODR submissions, either in progress or completed, were publicly available online. The search was restricted to completed evaluations. Forty-three submissions met the inclusion criteria. The incremental gain in QALYs in most submissions from the new technology was small (median incremental gain, 0.86; interquartile range, 0.6-1.39). More than half of the submissions (56%) did not include original data on HRQOL, with most relying on previous studies of variable relevance and quality. Re-analyses by pCODR based on concerns over HRQOL data used in the submitted model were common (52%). Drug manufacturers do not consistently collect data on HRQOL alongside clinical trials and instead rely on evidence generated in previous studies to inform cost-utility analyses. These findings should induce manufacturers to collect original HRQOL data that are simultaneously relevant to patients and decision makers.

Impact of a novel prioritization framework on clinician-led oncology drug submissions.

In Canada, requests for public reimbursement of cancer drugs are predominately initiated by pharmaceutical manufacturers. Clinician-led submissions provide a mechanism to initiate the drug funding process when industry does not submit a request for funding consideration. Although such requests are resource-intensive to produce, Cancer Care Ontario (cco) has the capacity to facilitate clinician-led submissions. In 2014, cco began developing a cancer drug prioritization framework that allocates resources to systematically address a growing number of clinician-identified funding gaps with clinician-led submissions. Cancer site-specific drug advisory committees established by cco consist of health care practitioners whose roles include identifying and prioritizing funding gaps. The committees submit their identified gaps to a cross-cancer-site prioritization exercise in which the requests are ranked based on a set of guiding principles derived from health technology assessment. The requests are then sequentially allocated the resources needed to meet submission requirements. Whether the funding gap is of provincial or pan-Canadian relevance determines where the submission is filed for assessment. Since its inception, the cco framework has identified 17 funding gaps in 9 cancer sites. In 4 prioritizations, the framework supported 6 submissions. As of June 2018, the framework had contributed to the eventual funding of more than 9 new drug-indication pairs, with more awaiting funding consideration. The cco prioritization framework has enabled clinicians to effectively and systematically identify, prioritize, and fill funding gaps not addressed by industry. Ultimately, the framework helps to ensure that patients can access evidence-informed and cost-effective therapies. The framework will continue to evolve as it encounters new challenges, including funding requests for rare indications.

Contrasting evidence to reimbursement reality for off-label use (OLU) of drug treatments in cancer care: rationale and design of the CEIT-OLU project

BackgroundOff-label use (OLU) of a drug reflects a perceived unmet medical need, which is common in oncology. Cancer drugs are often highly expensive and their reimbursement is a challenge for many healthcare systems. OLU is frequently regulated by reimbursement restrictions. For evidence-based healthcare, treatment ought to be reimbursed if there is sufficient clinical evidence for treatment benefit independently of patient factors not related to the treatment indication. However, little is known about the reality of OLU reimbursement and its association with the underlying clinical evidence. Here, we aim to investigate the relationship of reimbursement decisions with the underlying clinical evidence.Methods/ designWe will extract patient characteristics and details on treatment and reimbursement of cancer drugs from over 3000 patients treated in three Swiss hospitals. We will systematically search for clinical trial evidence on benefits associated with OLU in the most common indications. We will describe the prevalence of OLU in Switzerland and its reimbursement in cancer care, and use multivariable logistic regression techniques to investigate the association of approval/rejection of a reimbursement requests to the evidence on treatment effects and to further factors, including type of drug, molecular predictive markers and the health insurer.DiscussionOur study will provide a systematic overview and assessment of OLU and its reimbursement reality in Switzerland. We may provide a better understanding of the access to cancer care that is regulated by health insurers and we hope to identify factors that determine the level of evidence-based cancer care in a highly diverse western healthcare system.

What is driving HTA decision-making? Evidence from cancer drug reimbursement decisions from 6 European countries

BackgroundDecisions on the reimbursement of the same cancer drugs are different across European countries, but empirical work on the reasons behind these differences has been scarce. The main objective of this paper is to make a methodological contribution to existing research, specifically by outlining the systematic process of analysis to address such questions and determining the factors that might lead to different drug reimbursement decisions, and to explore its application in the field of oncology. MethodsReimbursement decisions on cancer drugs in six European countries (Belgium, England, Poland, Portugal, Scotland, and Sweden) between 2006 and 2014 were included in the study. A taxonomy was developed, comprising two groups of variables (system-level and product-specific) and an econometric model was specified (multilevel mixed-effects ordered probit). ResultsOnly one in six evaluations in the sample reach the same reimbursement recommendation. Most health system variables were not determinants of a higher or lower probability of a positive reimbursement recommendation. However, the probability of reimbursement was higher when a drug was considered cost-effective by NICE/SMC and when there was a financial Managed Entry Agreement. This work also demonstrated a possible econometric approach for analysing differences in reimbursement decisions and contributes a structured approach for collecting and preparing data for such analyses. ConclusionsDrug reimbursement decisions can be analysed in detail along a set of factors that are related to each decision. This information is essential, not only for understanding why a particular drug is accepted in one country and not in another but also when trying to implement a new HTA system or reform an existing one. This analysis provides policy makers and stakeholders with a model that enables a better understanding of the factors that drive HTA decisions and is adaptable to answer similar questions. Moreover, the data collection limitations encountered and described in this work shed light on the need for greater accessibility and transparency in HTA systems and regarding HTA outcomes.

PCN98 - The Limited Accessibility of New Cancer Drugs in Taiwan National Health Insurance

The objective of this study is to describe the accessibility of new cancer drugs under Taiwan National Health Insurance (NHI) in the past five years. This study reviewed all new drug applications to Taiwan NHI from January, 2013 to December, 2017. The new drugs were categorized into cancer and non-cancer drugs. The approval rate, time to approval, and reimbursement price were calculated and compared between cancer drugs and non-cancer drugs. The price was expressed as a ratio of Taiwan NHI reimbursement price to the median public price of 10 developed countries that Taiwan NHI sets its benchmarks (Germany, the US, the UK, France, Japan, Switzerland, Canada, Australia, Belgium and Sweden). In total, 42 cancer drugs and 186 non-cancer drugs were reviewed by Taiwan NHI during the study period. The approval rate of cancer drugs was lower than non-cancer drugs (approval rate of cancer drugs, 55%; non-cancer drugs, 60%). Among the approved cases, the median time to approval was longer in cancer drugs (18.77 months), compared with non-cancer drugs (9.67 months). The average price of cancer drugs was 65% to the median price of the benchmarked countries, higher than non-cancer drugs (58%). The study indicated the median time to approval of cancer drugs was double of non-cancer drugs in Taiwan NHI. Further studies are required to examine barriers in delayed reimbursement of cancer drugs.

PCP25 - Cancer Drug Reimbursement Decision-Making in the Republic of Ireland: Can the Process Be Made More Transparent and Systematic?

PCP25 - Cancer Drug Reimbursement Decision-Making in the Republic of Ireland: Can the Process Be Made More Transparent and Systematic?

The Relative Importance of Clinical, Economic, Patient Values and Feasibility Criteria in Cancer Drug Reimbursement in Canada: A Revealed Preferences Analysis of Recommendations of the Pan-Canadian Oncology Drug Review 2011-2017.

BackgroundMost Canadian provinces and territories rely on the pan-Canadian Oncology Drug Review (pCODR) to provide recommendations regarding public reimbursement of cancer drugs. The pCODR review process considers four dimensions of value—clinical benefit, economic evaluation, patient-based values and adoption feasibility—but they do not define weights for individual decision criteria or an acceptable threshold for any of the criteria. Given this implicit review process, it is of interest to understand which factors appear to carry the most weight in pCODR recommendations using a revealed preferences approach.MethodsUsing publicly available decision summaries (n = 91) describing submissions and resulting recommendations 2011–2017, we extracted ten attributes that characterized each submission. Using logistic regression, we identified statistically significant attributes and estimated their relative impact in final recommendations.ResultsClinical aspects appear to carry the greatest weight in the decision to reject or not reject, along with aspects of patient value (treatments with no alternatives were less likely to be rejected). Cost effectiveness does not appear to play a role in the initial decision to reject or not reject but is critical in full versus conditional approvals. There is evidence of a maximum acceptable threshold of around $Can140,000 per quality-adjusted life-year (QALY) gained.ConclusionA set of factors driving pCODR recommendations is identifiable, supporting the consistency of the review process. However, the implicit nature of the review process and the difficulty of extracting and interpreting some of the attribute levels used in the analysis suggests that the process may still lack full transparency.Electronic supplementary materialThe online version of this article (10.1007/s40273-018-0610-0) contains supplementary material, which is available to authorized users.

Decision Making and Priority Setting: The Evolving Path Towards Universal Health Coverage.

Health technology assessment (HTA) is widely viewed as an essential component in good universal health coverage (UHC) decision-making in any country. Various HTA tools and metrics have been developed and refined over the years, including systematic literature reviews (Cochrane), economic modelling, and cost-effectiveness ratios and acceptability curves. However, while the cost-effectiveness ratio is faithfully reported in most full economic evaluations, it is viewed by many as an insufficient basis for reimbursement decisions. Emotional debates about the reimbursement of cancer drugs, orphan drugs, and end-of-life treatments have revealed fundamental disagreements about what should and should not be considered in reimbursement decisions. Part of this disagreement seems related to the equity-efficiency tradeoff, which reflects fundamental differences in priorities. All in all, it is clear that countries aiming to improve UHC policies will have to go beyond the capacity building needed to utilize the available HTA toolbox. Multi-criteria decision analysis (MCDA) offers a more comprehensive tool for reimbursement decisions where different weights of different factors/attributes can give policymakers important insights to consider. Sooner or later, every country will have to develop their own way to carefully combine the results of those tools with their own priorities. In the end, all policymaking is based on a mix of facts and values.

Impact of enhanced reimbursement on provider participation a cancer care quality program and adherence to cancer treatment pathways in a commercial health plan.

6571 Background: Payment for treatment planning and care coordination for cancer care in the U.S. has been largely funded through the margin between the acquisition cost and reimbursement of cancer drugs. The Institute of Medicine has called for new payment models to align reimbursement to support patient-centered, high-quality affordable cancer care. Methods: In July 2014, 6 commercial HPs in the Midwest and Georgia implemented a cancer care quality program that included additional reimbursement for treatment planning and care coordination when oncologists selected regimens that were on a pathway. Cancer treatment pathways were developed using an evidence-based process with expert advisors from academic centers and community practices for breast, lung and colorectal cancer (available at www.cancercarequalityprogram.com). Practices provided clinical data on HP members starting systemic therapy through a web portal, including tumor type, stage, line of therapy, biomarkers, performance status, and planned t...

An exploratory analysis of the factors leading to delays in cancer drug reimbursement in the European Union: The trastuzumab case

BackgroundThe European Union (EU) has adopted a common procedure for granting marketing authorisation for cancer drugs. Nevertheless, pricing and reimbursement decisions are a competency of EU national governments, and their policies are diverse. We aimed to evaluate the time for trastuzumab reimbursement approval and its association to health expenditure, to health policy performance, to the availability of cost-effectiveness studies and to breast cancer outcome. MethodsBreast cancer outcome was estimated by the mortality/incidence (M/I) ratio. Trastuzumab reimbursement approval dates were provided by Roche. Spearman’s rank correlation and Wilcoxon rank-sum test were used to evaluate associations and/or differences between the variables studied. Additional analyses were made by grouping countries according to compliance to the 180day timeframe stipulated in the EU 89/105/EEC Directive for drug pricing and reimbursement. ResultsA statistically significant inverse and strong correlation between breast cancer M/I ratio and health expenditure (rs=–0.730, p<0.001) and health policy performance (rs=–0.711, p<0.001) was found, meaning the better the score and the higher the expenditure, the fewer patients died after a breast cancer diagnosis. Factors associated with trastuzumab faster reimbursement and compliance to the 89/105/EEC Directive were better health policy score, higher health expenditure and availability of cost-effectiveness studies. ConclusionHigher health policy scores and health expenditure are associated with faster reimbursement of trastuzumab and better breast cancer outcome. Although the study design does not allow inference of causal associations, a marked difference is observed between Eastern and Western Europe, with long delays and increased breast cancer mortality identified in Eastern European countries.

The Evaluation and Use of Economic Evidence to Inform Cancer Drug Reimbursement Decisions in Canada

Cost-effectiveness evidence is increasingly considered in the reimbursement decisions of pharmaceuticals. In some jurisdictions such as the UK and Canada, pharmaceutical manufacturers are required to submit economic evaluations when seeking reimbursement. Our objectives were to describe the role of economic evidence in the cancer drug review process in Canada, and to investigate the nature of problems encountered in the review and interpretation of economic evidence used in the process. We conducted a retrospective review of cancer drug review meeting minutes and reviewers' comments on pharmacoeconomic studies submitted to the oncology drug review process in Canada. We used pharmacoeconomic reviewers' reports and relevant cancer drug review expert advisory committee meeting minutes during the first year of the review process (April 2007 to March 2008). Fifteen economic submissions were reviewed. One-third of the studies had flaws significant enough that the advisory committee could not determine the cost effectiveness of the drugs from the results. The common issues outlined by the reviewers and committee were related to the uncertainty of comparative clinical benefits, quality of life and costs. The reviewers felt that few analyses provided sufficient sensitivity analyses around key variables to assess the robustness of results. Most problems identified by reviewers are simple to fix and do not involve advanced methods. Canada has a separate review process for making cancer drug funding recommendations, and this process uses both clinical and economic evidence. The committee could not determine the value for money of the drugs from several of the submitted pharmacoeconomic analyses. Transparent analyses and detailed critique of evidence are crucial to the use of economic evidence in reimbursement decisions. Rigorous evaluation is resource intensive and may benefit from a shared drug review process among several jurisdictions.

International variability in the reimbursement of cancer drugs by publically funded drug programs.

Evaluate inter-country variability in the reimbursement of publically funded cancer drugs, and identify factors such as cost containment measures that may contribute to variability. As of February 28, 2010, licensed indications for 10 cancer drugs (bevacizumab, bortezomib, cetuximab, erlotinib, imatinib, pemetrexed, rituximab, sorafenib, sunitinib, and trastuzumab) were obtained from the drug registries of 6 licensing authorities corresponding to 13 countries or regions: Australia, Canada (Ontario), England, Finland, France, Italy, Germany, Japan, New Zealand, the Netherlands, Scotland, Sweden, and the United States (Medicare Parts B and D). Number of licensed indications reimbursed by public payers and the use of cost containment measures were obtained by survey of health authorities involved in reimbursement and through public documents. The 48 identified licensed indications varied between agencies (range: 36-44 indications). Finland, France, Germany, Sweden, and the United States reimbursed the highest percentage of indications (range: 90%-100%). Canada (54%), Australia (46%), Scotland (40%), England (38%), and New Zealand (25%) reimbursed the least. All 5 countries with the lowest rate of reimbursement incorporated a cost-effectiveness analysis into reimbursement decisions and rejected submissions for reimbursement mainly because of lack of cost effectiveness; in New Zealand, lack of cost effectiveness was the second leading cause of rejection after excessive cost. In 9 countries, risk-sharing agreements were used to contain costs. Indications initially not recommended for reimbursement (9 in Australia, 5 in Canada, and 3 in England, New Zealand, and Scotland) were subsequently approved with risk-sharing agreements or special pricing arrangements. Reimbursement of publically funded cancer drugs varies globally. The cause is multifactorial.

PCN178 Reimbursement of Cancer Drugs in the UK: New Approach to End-of-Llife Treatments and the Technology Appraisal Programme of NICE

PCN178 Reimbursement of Cancer Drugs in the UK: New Approach to End-of-Llife Treatments and the Technology Appraisal Programme of NICE

The impact of patient advocacy: the case of innovative breast cancer drug reimbursement

Current research into patient advocacy focuses on attempts of patient groups to mobilise resources and to influence researchers, pharmaceutical companies and policy-makers. This paper adopts a 'framing political opportunities' approach to draw attention to other kinds of advocacy strategies. In a case study of breast cancer patient advocacy of Herceptin reimbursement, it is shown how patient groups tried to gain access to policy-making by means of three different opportunity-framing strategies. Articulation aims at creating awareness through public-agenda building. Negotiation aims at frame alignment between interdependent stakeholders by arranging meetings. Politicisation is a strategy to influence the agendas of political arenas. Patient organisations succeeded in creating awareness and support, which had a considerable impact on other stakeholders. These impacts in turn aided the politicisation of the issue. However, the final impact on reimbursement procedures was only partially achieved due to depoliticising counterstrategies based on persistent ideas buttressing a particular division of responsibilities in the organisation of healthcare. According to these ideas cost control in healthcare is a medical responsibility, not a political one.

A survey of U.S. and Canadian oncologists’ attitudes toward the cost, cost-effectiveness (CE), and reimbursement of cancer drugs

9502 Background: Drug costs and reimbursement issues offer significant challenges to U.S. and Canadian oncologists even though they practice in substantially different health care systems. However, little is known about the attitudes of American and Canadian oncologists towards these issues. Methods: We surveyed 1,379 U.S. and 356 Cdn oncologists to assess their attitudes to cancer drug costs, CE and reimbursement policies. Results: Response rate was 57% in the U.S. and 48% in Canada. Oncologists in both countries stated that patients' “out-of-pocket” drug costs influenced their treatment recommendations (84% U.S., 80% Cdn respondents). Most respondents felt that every patient should have access to effective cancer treatments regardless of cost (66% US; 54% Cdn), while 59% of U.S. and 72% of Cdn and respondents believed that patients should only have access to effective cancer treatments that provided “good value for money.” 70% of U.S. and 64% Cdn respondents felt that &lt;$100,000 per life year gained was a reasonable definition of “good value for money” but less than half of respondents (42% US, 49% Cdn) felt well prepared to interpret and use CE information in their treatment decisions. A majority of respondents (57% US, 69% Cdn) felt government price controls for cancer drugs are needed while a minority felt that more cost-sharing by patients was needed (29% US, 37% Cdn). Most oncologists felt that evaluating whether a drug provides “good value” should be overseen by an independent non-profit agency (57% US, 71% Cdn) or physicians (61% US and Cdn); in contrast, few believed that government (21% US, 33% Cdn), patients (36% US, 37% Cdn) or insurance companies (6% US, 10% Cdn) should determine “good value”. 79% of U.S. and 69% of Cdn respondents felt more use of CE data in coverage and reimbursement decisions is needed. Conclusions: Oncologists in the U.S. and Canada share many similar attitudes to cancer drug costs, CE, and reimbursement policies despite differences in their health care systems. In both countries, oncologists favor more use of CE information. No significant financial relationships to disclose.