Background:
Rising expenditure for new cancer medicines is accelerating concerns that their costs will become unsustainable for universal healthcare access. Moreover, early market access of new ...oncology medicines lacking appropriate clinical evaluation generates uncertainty over their cost-effectiveness and increases expenditure for unknown health gain. Patient-level data can complement clinical trials and generate better evidence on the effectiveness, safety and outcomes of these new medicines in routine care. This can support policy decisions including funding. Consequently, there is a need for improving datasets for establishing real-world outcomes of newly launched oncology medicines.
Aim:
To outline the types of available datasets for collecting patient-level data for oncology among different European countries. Additionally, to highlight concerns regarding the use and availability of such data from a health authority perspective as well as possibilities for cross-national collaboration to improve data collection and inform decision-making.
Methods:
A mixed methods approach was undertaken through a cross-sectional questionnaire followed-up by a focus group discussion. Participants were selected by purposive sampling to represent stakeholders across different European countries and healthcare settings. Descriptive statistics were used to analyze quantifiable questions, whilst content analysis was employed for open-ended questions.
Results:
25 respondents across 18 European countries provided their insights on the types of datasets collecting oncology data, including hospital records, cancer, prescription and medicine registers. The most available is expenditure data whilst data concerning effectiveness, safety and outcomes is less available, and there are concerns with data validity. A major constraint to data collection is the lack of comprehensive registries and limited data on effectiveness, safety and outcomes of new medicines. Data ownership limits data accessibility as well as possibilities for linkage, and data collection is time-consuming, necessitating dedicated staff and better systems to facilitate the process. Cross-national collaboration is challenging but the engagement of multiple stakeholders is a key step to reach common goals through research.
Conclusion:
This study acts as a starting point for future research on patient-level databases for oncology across Europe. Future recommendations will require continued engagement in research, building on current initiatives and involving multiple stakeholders to establish guidelines and commitments for transparency and data sharing.
Availability and accessibility of anti-cancer medicines is the pillar of cancer management, and it is one of the main concerns in low-income countries including Rwanda. The objective of this study ...was to assess the availability and affordability of anticancer medicines at cancer-treating hospitals in Rwanda.
A descriptive cross-sectional study was conducted at 5 cancer-treating hospitals in Rwanda. Quantitative data were collected from stock cards and software that manage medicines and included the availability of anti-cancer medicines at the time of data collection, their stock status within the last two years, and the selling price.
The study found the availability of anti-cancer medicines at 41% in public hospitals at the time of data collection, and 45% within the last two years. We found the availability of anti-cancer medicines at 45% in private hospitals at the time of data collection, and 61% within the last two years. 80% of anti-cancer medicines in private hospitals were unaffordable while 20% were affordable. The public hospital that had most of the anti-cancer medicines in the public sector provided free services to the patients, and no cost was applied to the anti-cancer medicines.
The availability of anti-cancer medicines in cancer-treating hospitals is low in Rwanda, and most of them are unaffordable. There is a need to design strategies that can increase the availability and affordability of anti-cancer medicines, for the patients to get recommended cancer treatment options.
Abstract
Objectives
The objective of the literature review is to collect data on how cancer medication pricing affects affordability and availability around the world.
Key findings
A literature ...search was carried out between 12 October 2020 and 2 December 2020, articles were selected based on them being available as full texts online and written in English. The keywords used were: ‘cancer’, ‘medicines’, ‘drug’, ‘drugs’, ‘pharmaceuticals’, ‘price’, ‘prices’, ‘pricing’. The collective search produced a total of 10 725 articles. After a refining process, any articles considered unnecessary and potential duplications were eliminated, and 16 research articles were included in the final analysis. The results were included in the following categories: (a) high-income countries, (b) low- and middle-income countries (LMICs), (c) originator cancer drug prices, (d) generic drug prices, (e) breast and colorectal cancer drug pricing. The expensive pricing of oncology medications is typically expected to be an obstacle for developing nations; however, the soaring prices have been found increasingly challenging for high-income countries. Within Europe, variations exist between countries in government expenditure and cancer drug prices. Originator cancer drug prices tend to be most expensive in Sweden, Denmark, Switzerland and Germany whereas Greece, Spain, Portugal and the UK had among the lowest recorded prices. The high cost of cancer drugs coupled with low availability rates have resulted in restricted access for many LMICs as monthly medicinal costs are often greater than yearly incomes.
Summary
The literature has shown the increasing trend of cancer drug pricing. The synthesis has also shown that cancer treatments are unaffordable in many developing countries resulting in most cancer deaths occurring in LMICs. Furthermore, governments cannot effectively challenge patented drug prices until the expiry of the patent.
Objective
Anti-cancer medicine shortages are advancing challenges for patients and hospitals. This study aims to evaluate anti-cancer and supportive medicine shortages in a tertiary hospital in ...Pakistan and propose solutions.
Method
A retrospective observational research was performed in a tertiary care hospital in Pakistan from 2016 to 2020. Data was retrieved from the hospital database using a questionnaire regarding short medicines’ generic name, brand, dosage, source, total source, frequency, causes, impact, management, and analyzed by Microsoft Excel 2013.
Results
Between January 2016 and December 2020, 43 individual medicine shortages were observed, with an average of 8.6 shortages per year. There were shortages of 22 medicines, including 8 anti-cancer (36.4%) and 14 supportive agents (63.6%). Total shortage days were 27,100, with an average of 1232 days (SD 757) per medicine. Supportive medicines’ shortages were frequent, but oncology agents’ shortages were constant. The most affected dosage form was injection. Cardiovascular drugs and alkylating agents were the most affected class in supportive and anti-cancer medicines, respectively. The use of “alternative medicine” and “patient needs based importation” were the most common mitigation strategies.
Conclusion
Shortages of oncology medicines are challenging in Pakistan. The most prominent causes are the lack of updated governmental regulations, registration, and import issues. The tertiary care hospital has very few sources of supply, so it imports these drugs on a need basis to manage the shortages. But it is still concerning because of the huge financial burden on patients and institutions due to expensive import, and therapy become delayed as the import process takes time. Moreover, the most affected drug class was alkylating agents, and dosage was both injectable and oral medicines.
To assess whether using surrogate versus patient-relevant endpoints in pivotal trials of cancer drugs was associated with health technology assessment recommendations in England (National Institute ...for Health and Care Excellence NICE) and Canada (Canadian Agency for Drugs and Technologies in Health CADTH).
Cancer drug approvals from 2012 to 2016 were categorized by demonstrating benefit on overall survival (OS), progression-free survival, disease response, or having no comparator. Approvals were analyzed by benefit category and health technology assessment recommendation. The association between benefit (surrogate vs OS) and recommending a drug was examined using descriptive statistics and linear probability models controlling for unmet need, orphan designation, and cost-effectiveness.
Of 42 cancer indications that NICE recommended, 15 (36%) demonstrated OS benefit. Cancer indications with OS benefit were less likely to receive a recommendation from NICE than those without (P = .04). In linear probability models, availability of OS benefit was no longer associated with a recommendation from NICE (P = .32). Cost-effective cancer drugs had a 55.6% (95% CI: 38.9%-72.3%) higher probability of receiving a recommendation from NICE than those that were not. In Canada, 15 of 37 (41%) cancer indications that were recommended showed OS benefit. There was no detectable association between surrogate measures and CADTH recommendations based on descriptive statistics (P = .62) or in linear probability models (P = .73).
When cost-effectiveness was considered, pivotal trial endpoints were not associated with NICE recommendations. Pivotal trial endpoints, unmet need, orphan status, and cost-effectiveness did not explain CADTH recommendations.
•Drug licensing agencies are under increasing pressure to expedite the approval of new cancer medicines. A common strategy to facilitate faster development of therapeutic agents is to use surrogate measures of clinical benefit. The validity of surrogates of overall survival (OS) in many cancer indications has been debated. There is no up-to-date study that evaluates the impact of surrogate measures on health technology assessment recommendations.•This study shows no evidence that the use of surrogate measures in cancer trials is associated with a health technology assessment decision to reject a drug. During our study period, cancer drugs without OS benefit were more likely to be recommended by the National Institute for Health and Care Excellence in England. Nevertheless, when taking into consideration multiple factors, OS benefit did not affect the National Institute for Health and Care Excellence’s decisions, but cost-effectiveness did. Canadian Agency for Drugs and Technologies in Health (CADTH) decisions in Canada were not associated with the use of surrogate endpoints, unmet need, cost-effectiveness, or orphan status.•These findings raise questions about the assumptions made in cost-effectiveness models in England, as multiple systematic reviews in oncology have found no clear relationship between surrogate measures and patient-centered outcomes, namely OS and quality of life.•The observed variation in CADTH decisions is not explained by known and measured factors in our analysis. Further clarity is needed on the framework guiding CADTH decisions on new cancer drugs.
More alternatives have become available for the diagnosis and treatment of cancer in low- and middle-income countries. Because of increasing demands, governments are now facing a problem of limited ...affordability and availability of essential cancer medicines. Yet, precise information about the access to these medicines is limited, and the methodology is not very well developed. We assessed the availability and affordability of essential cancer medicines in Mexico, and compared their prices against those in other countries of the region.
We surveyed 21 public hospitals and 19 private pharmacies in 8 states of Mexico. Data were collected on the availability and prices of 49 essential cancer medicines. Prices were compared against those in Chile, Peru, Brazil, Colombia and PAHO's Strategic Fund.
Of the various medicines, mean availability in public and private sector outlets was 61.2 and 67.5%, respectively. In the public sector, medicines covered by the public health insurance "People's Health Insurance" were more available. Only seven (public sector) and five (private sector) out of the 49 medicines were considered affordable. Public sector procurement prices were 41% lower than in other countries of the region.
The availability of essential cancer medicines, in the public and private sector, falls below World Health Organization's 80% target. The affordability remains suboptimal as well. A national health insurance scheme could serve as a mechanism to improve access to cancer medicines in the public sector. Comprehensive pricing policies are warranted to improve the affordability of cancer medicines in the private sector.
Background
In order to establish a long-term strategy for bearing the costs of anti-cancer drugs, the state had organized five rounds of national-level pricing negotiations and introduced the ...National Health Insurance Coverage (NHIC) policy since 2016. In addition, the National Healthcare Security Administration (NHSA) introduced the volume-based purchasing (VBP) pilot program to Nanjing in September 2019. Taking non-small cell lung cancer as an example, the aim of the study was to verify whether national pricing negotiations, the NHIC policy and the VBP pilot program had a positive impact on the accessibility of three targeted anti-cancer drugs.
Methods
Based on the hospital procurement data, interrupted time series (ITS) design was used to analyze the effect of the health policy on the accessibility and affordability of gefitinib, bevacizumab and recombinant human endostatin from January 2013 to December 2020 in Nanjing, China.
Results
The DDDs of the three drugs increased significantly after the policy implementation (
P
< 0.001,
P
< 0.001,
P
= 0.008). The trend of DDDc showed a significant decrease (
P
< 0.001,
P
< 0.001,
P
< 0.001). The mean availability of these drugs before the national pricing negotiation was <30% in the surveyed hospitals, and increased significantly to 60.33% after 2020 (
P
< 0.001,
P
= 0.001,
P
< 0.001). The affordability of these drugs has also increased every year after the implementation of the insurance coverage policy. The financial burden is higher for the rural patients compared with the urban patients, although the gap is narrowing.
Conclusion
The accessibility of targeted anti-cancer drugs has increased significantly after the implementation of centralized prices, the NHIC policy and the VBP pilot program, and has shown sustained long-term growth. Multi-pronged supplementary measures and policy approaches by multiple stakeholders will facilitate equitable access to effective and affordable anti-cancer drugs.
Almost 100 novel cancer medicines have been approved in Europe over the last decade. Limited public health care resources in countries in Central and Eastern Europe (CEE) call for a prioritization of ...access to effective medicines. We investigated how both reimbursement status and waiting time to reimbursement correlate with the magnitude of clinical benefit provided by novel medicines in four selected countries (Czechia, Hungary, Poland, and Slovakia).
A total of 124 indications of 51 cancer medicines with marketing authorization by the European Medicines Agency in 2011-2020 were included and followed up until 2022. Data on reimbursement status and waiting time to reimbursement (i.e. time from marketing authorization to national reimbursement approval) were collected for each country. Data were analyzed in relation to clinical benefit status (i.e. substantial versus nonsubstantial clinical benefit) of indications according to the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS).
The degree of reimbursement differed between countries with 64% of indications with reimbursement in Czechia, 40% in Hungary, 51% in Poland, and 19% in Slovakia. In all countries, a significantly greater proportion of indications with a substantial clinical benefit was reimbursed (P < 0.05). The median waiting time to reimbursement ranged from 27 months in Poland to 37 months in Hungary. No significant differences in waiting time in relation to clinical benefit were observed in any country (P = 0.25-0.84).
Cancer medicines with a substantial clinical benefit are more likely to be reimbursed in all four CEE countries. Waiting times to reimbursement are equally long for medicines with or without a substantial clinical benefit, indicating a lack of prioritization of fast access to medicines delivering a substantial benefit. Incorporation of the ESMO-MCBS in reimbursement assessments and decisions could aid in better utilization of limited resources to deliver more effective cancer care.
•Limited public health care resources call for a prioritization of access to effective cancer medicines.•The degree of reimbursement of newer cancer medicines differs greatly between four countries in Central and Eastern Europe.•In all countries, a significantly greater proportion of indications with substantial ESMO-MCBS was reimbursed.•No significant differences in time to reimbursement from EMA approval in relation to ESMO-MCBS was observed in any country.•Incorporation of the ESMO-MCBS in reimbursement assessments and decisions could aid in better utilization of resources.
Objective:
The study aimed to evaluate the impact of the National Health Insurance Coverage (NHIC) policy on the utilisation and accessibility of innovative anti-cancer medicines in Nanjing, China.
...Methods:
We used the adjusted World Health Organisation and Health Action International methodology to calculate the price and availability of 15 innovative anti-cancer medicines included in the National Health Insurance drug list in 20 tertiary hospitals and six secondary hospitals in Nanjing before and after NHIC policy implementation. Interrupted time-series regression was used to analyse the changes in the utilisation of the study medicines.
Results:
The price reduction rates of innovative anti-cancer medicines ranged between 34 and 65%. The mean availability rate was 27.44% before policy implementation and increased to 47.33% after policy implementation. The utilisation of anti-cancer medicines suddenly increased with a slope of 33.19–2,628.39 when the policy was implemented. Moreover, the usage rate of bevacizumab, bortezomib, and apatinib significantly increased (
p
< 0.001,
p
= 0.009, and
p
< 0.001, respectively) after policy implementation. With regard to price reduction and medical insurance reimbursement, the medicines became more affordable after policy implementation (0.06–1.90 times the per capita annual disposable income for urban patients and 0.13–4.46 times the per capita annual disposable income for rural patients).
Conclusion:
The NHIC policy, which was released by the central government, effectively improved the utilisation and affordability of innovative anti-cancer medicines. However, the availability of innovative anti-cancer medicines in hospitals remained low and the utilisation of innovative anti-cancer medicines was affected by some factors, including the incidence of cancer, limitation of indications within the insurance program, and the rational use of innovative anti-cancer medicines. It is necessary to improve relevant supporting policies to promote the affordability of patients. The government should speed up the process of price negotiation to include more innovative anti-cancer medicines in the medical insurance coverage, consider including both medical examinations and adjuvant chemotherapy in the medical insurance, and increase investment in health care.
Cancer is a global public health problem, requiring efficient health system investments to deliver sustainable impact on population health. Access to medicines is a critical component of health ...systems, having a crucial role in delivering therapeutic benefits. Since 1977, the World Health Organization (WHO) has published a Model List of Essential Medicines (EML) that includes key health interventions for the prevention and control of conditions of public health relevance. Essential medicines are selected for inclusion in the EML based on the evidence of efficacy, safety, therapeutic value, and the potential to impact population health. With the rapid changes in the therapeutic landscape of cancer treatment with new medicine approvals, there is a critical need to select and prioritise specific cancer interventions based on their intrinsic value.
The European Society for Medical Oncology (ESMO) has developed a decisional methodology based on a threshold with a minimum set of technical specifications and a consensus-based procedure for decisions to select candidate cancer medicines to be submitted to the WHO for consideration for the WHO EML.
ESMO recognises the WHO EML as an important reference guide for medicines that all countries should include in their national EMLs. Cancer medicines on the WHO EML are used in the treatment of the majority of cancers, and are recommended in the evidence-based ESMO Clinical Practice Guidelines that medical oncologists use to treat patients. ESMO’s submissions to the WHO EML in 2019 and 2021 and their respective outcomes are presented in the manuscript.
Due to the rising costs associated with newly available therapies, structured, reproducible, and field-tested tools to evaluate the added clinical benefit from these therapies need to be implemented in pre-selecting potential candidate medicines to be included in the WHO EML. ESMO is proud to collaborate closely with WHO on this important global public health initiative.
•Prioritising access to safe, effective, quality, and affordable essential medicines is key to improve cancer care.•The WHO is instrumental in recognising medicines with public health relevance through the WHO Essential Medicines’ List.•EML’s selection is based on disease prevalence, evidence of efficacy and safety, and comparative cost-effectiveness.•The WHO EML Expert Committee recognises the ESMO-MCBS as a screening tool for inclusion consideration in the EML.•ESMO supports the update of the WHO EML, a reference for countries to ensure essential cancer care to all.
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