Access to essential and innovative anti-cancer medicines: a longitudinal study in Nanjing, China.

PURPOSE: To evaluate the availability, cost, affordability of anti-cancer medicines in Nanjing, Jiangsu. METHODS: A longitudinal tracking investigation study was performed to collect information about 24 essential anti-cancer medicines (EAMs) and 17 innovative anti-cancer medicines (IAMs) in 26 healthcare institutions in Nanjing from 2016 to 2020. The availability, cost, drug utilization and affordability of EAMs and IAMs were investigated. RESULTS: The availability of EAMs showed no significant changes in Nanjing, but the availability of IAMs showed a significant increase in 2018 and 2019 and tended to stabilize in 2020. For EAMs, the DDDc(Defined Daily Dose cost) of LPGs (Lowest-Priced Generics) showed no significant changes, and the DDDc of OBs (Originator Brands) and IAMs significantly decreased. The DDDs(Defined Daily Doses) of EAMs (LPGs) showed a decreasing trend since 2016 and rose again in 2019. Overall, the DDDs of EAMs (LPGs) decreased by 25.18% between 2016 and 2020, but the proportion selected for clinical treatment remained at 67.35% in 2020. The DDDs of EAMs (OBs) and IAMs both showed an increasing trend year by year, with a proportional increase of 207.72% and 652.68%, respectively; but the proportion selected for clinical treatment was only 16.09% and 16.56% respectively in 2020. EAMs (LPGs) had good affordability for urban residents but poor affordability for rural residents; the affordability of EAMs (OBs) and IAMs was poor for both urban and rural residents. CONCLUSIONS: There were no significant changes in the availability and cost of EAMs (LPGs), whose lower prices showed better affordability. Although their relative change in drug utilization showed a decreasing trend, they still dominated clinical treatment. Driven by the national drug price negotiation (NDPN) policy, the availability of IAMs was on the rise. It is necessary to further develop and strengthen policies for essential medicines procurement assessment to improve the accessibility of EAMs.

ESMO study on the availability and accessibility of biomolecular technologies in oncology in Europe.

BACKGROUND: Access to biomolecular technologies has become an essential requirement to ensure optimal and timely treatment of patients with cancer. This study sought to provide a comprehensive overview of the availability and accessibility of biomolecular technologies to patients, the status of their use and prescription, barriers to access, and potential economic issues related to cost and reimbursement. MATERIALS AND METHODS: A total of 201 field reporters from 48 European countries submitted data through an electronic survey tool between July and December 2021. The survey methodology mirrored that from previous ESMO studies addressing the availability and accessibility of antineoplastic medicines, in Europe and worldwide. The preliminary data were posted on the ESMO website for open peer-review, and amendments were incorporated into the final report. RESULTS: Overall, basic single-gene techniques are widely available, whereas access to advanced biomolecular technologies, including large next-generation sequencing panels and complete genomic profiles, is highly heterogeneous. In most countries, advanced biomolecular technologies remain largely inaccessible in clinical practice, are limited to clinical trials or basic research, and associated with progressively increasing cost as the technique becomes more advanced. Differences also exist regarding national sequencing initiatives or molecular tumour boards. The most important barriers to multiple versus single-gene sequencing techniques are the reimbursement of the test (59% versus 24%), and the availability of a suitable medicine, either through reimbursement of treatment (48% versus 30%), off-label treatment (52% versus 35%), or clinical trial enrolment (53% versus 39%). CONCLUSIONS: Cost and availability of both treatment and test are the two main factors limiting patients' access to advanced biomolecular technologies and as a consequence to innovative anticancer strategies. In the era of precision medicine, tackling the accessibility to biomolecular technologies is a key step to reduce inequalities to transformative cancer care.

Uptake and availability of new outpatient cancer medicines in 2010-2021 in Nordic countries - survey of competent authorities.

BACKGROUND: Nordic countries excel in cancer care, but studies on uptake, costs, or managed entry agreements of cancer medicines have not been conducted recently. The aim of this study was to examine the uptake and availability of orally administered new cancer medicines in Nordic countries. Orally administered cancer medicines enable and are used in the community as part of outpatient care. Firstly, we studied the distribution, costs and adoption of managed entry agreements of these medicines, and secondly, uptake of and managed entry agreements for cancer medicines used in outpatient care that were granted marketing authorization in Europe in 2010-2021. METHODS: An E-mail survey of competent authorities, meaning pharmaceutical service organizers, payers or other government or non-government actors developing pharmaceutical service operations, in Denmark, Finland, Iceland, Norway, and Sweden in April-June 2022. The data were analysed using frequencies and percentages for descriptive analysis. RESULTS: The distribution of cancer medicines has similarities in Finland, Iceland, Norway, and Sweden, where cancer medicines can be distributed both via hospitals or hospital pharmacies for inpatient use, and via community pharmacies for outpatient use. In Denmark, cancer medicines are predominantly distributed via publicly funded hospitals. In all countries that provided data on the costs, the costs of cancer medicines had notably gone up from 2010 to 2021. The number of reimbursable medicines out of new cancer medicines varied from 36 products in Denmark and Iceland to 51 products in Sweden, out of 67 studied products. Managed entry agreements, often with confidential discounts, were in use in all Nordic countries. The number of agreements and the cancer types for which agreements were most often made varied from three agreements made in Iceland to 35 agreements made in Finland, out of 67 studied products. Average days from authorization to reimbursement of new cancer medicines varied from an average of 416 to 895 days. CONCLUSIONS: Nordic countries share similar characteristics but also differ in terms of the details in distribution, adopted managed entry agreements, market entry, and availability of new orally administered cancer medicines used in the outpatient care. The costs of cancer medicines have increased in all Nordic countries during the last decade. Due to differences in health care and because orally administered cancer medicines can be dispensed at community and hospital pharmacies in all studied countries other than Denmark, the number of reimbursable medicines and managed entry agreements vary between countries. However, Nordic countries show good agreement for 2010 to 2021 in entry and reimbursement decisions of novel cancer medicines.

Availability and affordability of anticancer medicines at cancer treating hospitals in Rwanda.

BACKGROUND: 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. METHODOLOGY: 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. RESULTS: 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. CONCLUSION: 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.

Is the quality of evidence in health technology assessment deteriorating over time? A case study on cancer drugs in Australia.

OBJECTIVE: This study aimed to assess whether there have been changes in the quality of clinical evidence submitted for government subsidy decisions on cancer medicines over the past 15 years. METHODS: We reviewed public summary documents (PSDs) reporting on subsidy decisions made by the Pharmaceutical Benefits Advisory Committee (PBAC) from July 2005 to July 2020. Information was extracted on the study design, directness of comparison, sample size, and risk of bias (RoB). Changes in the quality of evidence were assessed using regression analysis. RESULTS: Overall, 214 PSDs were included in the analysis. Thirty-seven percent lacked direct comparative evidence. Thirteen percent presented observational or single-arm studies as the basis for decisions. Among PSDs presenting indirect comparisons, 78 percent reported transitivity issues. Nearly half (41 percent) of PSDs reporting on medicines supported by head-to-head studies noted there was a moderate/high/unclear RoB. PSDs reporting concerns with RoB increased by a third over the past 7 years, even after adjusting for disease rarity and trial data maturity (OR 1.30, 95% CI: 0.99, 1.70). No time trends were observed regarding the directness of clinical evidence, study design, transitivity issues, or sample size during any of the analyzed periods. CONCLUSION: Our findings indicate that the clinical evidence supplied to inform funding decisions for cancer medicines is often of poor quality and has been deteriorating over time. This is concerning as it introduces greater uncertainty in decision making. This is particularly important as the evidence supplied to the PBAC is often the same as that supplied to other global decision-making bodies.

Anti-cancer medicine shortages in an oncology tertiary hospital of Pakistan: A five-year retrospective study.

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.

How government health insurance coverage of novel anti-cancer medicines benefited patients in China - a retrospective analysis of hospital clinical data.

BACKGROUND: China started to cover novel medicines for the treatment of major cancers, such as trastuzumab for breast cancer by the government health insurance programs since 2016. Limited data have been published on the use of cancer medications and little is known about how government health insurance coverage of novel anti-cancer medicines benefited patients in the real world. This study aimed to generate evidence to inform the health security authorities to optimize the government health insurance coverage of novel anti-cancer medicines as a more inclusive and equal policy, through which each of the needed patient can get access to the novel anti-cancer medicines regardless of the ability to pay. METHODS: The study targeted one of the government health insurance newly covered novel medicines for breast cancer and the breast cancer patients. The analyses were based on the data collected from one tertiary public hospital in Fujian province of China. We conducted interrupted time series analysis with a segmented regression model and multivariate analyses with a binary logistic regression model to analyze the impact of the government health insurance coverage on medicines utilization and the determinants of patient's medication choice. RESULTS: The average proportion of patients who initiated medication with novel medicines increased from 37.4% before the government health insurance coverage to 69.2% afterwards. Such an increase was observed in all patient sub-groups. The monthly proportion of patients who initiated medication with novel medicines increased sharply by 18.3 % (95 %CI,10.4-34.0 %, p = 0.01) in September 2017, the afterwards trend continuously increased (95 %CI,1.03-3.60, p = 0.02). The critical determinants of patient's medication choice were mostly connected with the patient's health insurance benefits packages. CONCLUSIONS: The government health insurance coverage of novel anti-breast-cancer medicines benefited the patients generally. The utilization of novel medicines such as trastuzumab continuously increased. The insurance coverage benefited well the patients in the high-risk age groups. However, rural patients, patients enrolled in the "resident program", and patients from low-income residential areas and non-local patients benefited less from this policy. Improving the benefits package of the low-income patients and the "resident program" beneficiary would be of considerable significance for a more inclusive and equal health insurance coverage of novel anti-cancer medicines.

Association Between the Use of Surrogate Measures in Pivotal Trials and Health Technology Assessment Decisions: A Retrospective Analysis of NICE and CADTH Reviews of Cancer Drugs.

OBJECTIVE: 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]). METHODS: 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. RESULTS: 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). CONCLUSION: 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.

Availability, prices, and affordability of selected essential cancer medicines in a middle-income country - the case of Mexico.

BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: 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.

Improved anticancer drug response prediction in cell lines using matrix factorization with similarity regularization.

BACKGROUND: Human cancer cell lines are used in research to study the biology of cancer and to test cancer treatments. Recently there are already some large panels of several hundred human cancer cell lines which are characterized with genomic and pharmacological data. The ability to predict drug responses using these pharmacogenomics data can facilitate the development of precision cancer medicines. Although several methods have been developed to address the drug response prediction, there are many challenges in obtaining accurate prediction. METHODS: Based on the fact that similar cell lines and similar drugs exhibit similar drug responses, we adopted a similarity-regularized matrix factorization (SRMF) method to predict anticancer drug responses of cell lines using chemical structures of drugs and baseline gene expression levels in cell lines. Specifically, chemical structural similarity of drugs and gene expression profile similarity of cell lines were considered as regularization terms, which were incorporated to the drug response matrix factorization model. RESULTS: We first demonstrated the effectiveness of SRMF using a set of simulation data and compared it with two typical similarity-based methods. Furthermore, we applied it to the Genomics of Drug Sensitivity in Cancer (GDSC) and Cancer Cell Line Encyclopedia (CCLE) datasets, and performance of SRMF exceeds three state-of-the-art methods. We also applied SRMF to estimate the missing drug response values in the GDSC dataset. Even though SRMF does not specifically model mutation information, it could correctly predict drug-cancer gene associations that are consistent with existing data, and identify novel drug-cancer gene associations that are not found in existing data as well. SRMF can also aid in drug repositioning. The newly predicted drug responses of GDSC dataset suggest that mTOR inhibitor rapamycin was sensitive to non-small cell lung cancer (NSCLC), and expression of AK1RC3 and HINT1 may be adjunct markers of cell line sensitivity to rapamycin. CONCLUSIONS: Our analysis showed that the proposed data integration method is able to improve the accuracy of prediction of anticancer drug responses in cell lines, and can identify consistent and novel drug-cancer gene associations compared to existing data as well as aid in drug repositioning.

Cancer control-A global perspective.

Disparities in cancer control exist in low- and middle-income countries (LMICs). Many countries do not have cancer registries to record incidence, mortality and prevalence and are reliant on Globocan estimates of their cancer burden. Poorer cancer control within and between countries occurs in those living remotely from urban centres, those in a low socioeconomic group and some ethnic groups who have lifestyle and belief systems which impact on cancer control. High-income countries generally have population screening programmes for cervix, breast and bowel cancer. However, simpler forms of screening for cancer of the cervix like visual inspection with acetic acid have been shown to be feasible in developing nations. The widespread use of vaccines to prevent cancer has been achieved with the Hepatitis B vaccine but the human papilloma virus vaccine to prevent cancer of the cervix is largely only available in high-income countries. Access to and training of oncological surgeons in LMICs is limited, while 70% of patients in these countries cannot access radiotherapy. The World Health Organization has developed a list of essential medicines although access remains poor in LMICs. The United Nations has set targets for the control of non-communicable diseases to improve global cancer control.

Cancer medicines: a private vice for public benefit?

Cancer medicines have become one of the most dominant global medical technologies. They generate huge profits for the biopharmaceutical industry as well as fuel the research and advocacy activities of public funders, patient organisations, clinical and scientific communities and entire federal political ecosystems. The mismatch between the price, affordability and value of many cancer medicines and global need has generated significant policy debate, yet we see little change in behaviours from any of the major actors from public research funders through to regulatory authorities. In this policy analysis we examine whether, considering the money and power inherent in this system, any rationale global consensus and policy can be achieved to deliver affordable and equitable cancer medicines that consistently deliver clinically meaningful benefit.

Access to novel cancer medicines in four countries in Central and Eastern Europe in relation to clinical benefit.

BACKGROUND: 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). MATERIALS AND METHODS: 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). RESULTS: 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). CONCLUSIONS: 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.

Expanding access to high-cost medicines under the Universal Health Coverage scheme in Thailand: review of current practices and recommendations.

BACKGROUND: There has been an increasing demand to reimburse high-cost medicines, through public health insurance schemes in Thailand. METHODS: A mixed method approach was employed. First, a rapid review of select high-income countries was conducted, followed by expert consultations and an in-depth review of three countries: Australia, England and Republic of Korea to understand reimbursement mechanisms of high-cost medicines. In Thailand, current pathways for reimbursing high-cost medicines reviewed, the potential opportunity cost estimated, and stakeholder consultations were conducted to identify context specific considerations. RESULTS: High-income countries reviewed have implemented a variety of pathways and mechanisms for reimbursing high-cost medicines under specific eligibility criteria, listing processes, varying cost-effectiveness thresholds and special funding arrangements. In Thailand, high-cost medicines that do not offer good value-for-money are excluded from the reimbursement process. A framework for reimbursing high-cost medicines that are not cost-effective at the current willingness-to-pay threshold was proposed for Thailand. Under this framework, specific criteria are proposed to determine their eligibility for reimbursement such life-saving nature, treatment of conditions with no alternative treatment options, and affordability. CONCLUSION: High-cost medicines may become eligible for reimbursement through alternative mechanisms based on specific criteria which depend on each context. The application of HTA methods and processes is important in guiding these decisions to support sustainable access to affordable healthcare in pursuit of Universal Health Coverage (UHC).

The reality of cancer care for many.

The global literature is heavy with technical documents on how we should deliver cancer care, as well as policy discourses and studies on cancer services and systems. But it is woefully short on the reality of lived experiences. "Adaptations to Deliver Chemotherapy in a District Hospital: Successes and Challenges" from Malawian colleagues based in the Kumuzu University of Health Sciences in the District Hospital of the same name shines a much needed light of realism on the lived experiences of cancer. Why is this letter so important? Because it is much more than a narrative about a district hospital in a low resource country struggling to deliver basic cancer care, it is about the intricate relationship and trade-offs between patients and cancer carers in all resource constrained settings. It bears witness to a reality that feels very far away from the shining bright lights of modern cancer care with all its attendant technological trappings and choices.

Essential cancer medicines: adding feasibility to the magnitude of clinical benefit value chain.

BACKGROUND: 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. MATERIALS AND METHODS: 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. RESULTS: 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. CONCLUSION: 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 systemic cancer therapies applying ESMO's tools and other resources to assist in improving cancer care globally: the Kazakh experience.

BACKGROUND: In Kazakhstan, cancer is the second leading cause of death with a major public health and economic burden. In the last decade, cancer care and cancer medicine costs have significantly increased. To improve the efficiency and efficacy of cancer care expenditure and planning, the Kazakhstan Ministry of Health requested assistance from the World Health Organization (WHO) and the European Society for Medical Oncology (ESMO) to review its systemic cancer treatment protocols and essential medicines list and identify high-impact, effective regimens. MATERIALS AND METHODS: ESMO developed a four-phase approach to review Kazakhstan cancer treatment protocols: (i) perform a systematic analysis of the country's cancer medicines and treatment protocols; (ii) cross-reference the country's cancer protocols with the WHO Model List of Essential Medicines, the ESMO-Magnitude of Clinical Benefit Scale and the European Medicines Agency's medicine availability and indications database; (iii) extract treatment recommendations from the ESMO Clinical Practice Guidelines; (iv) expert review for all cancer medicines not on the WHO Model List of Essential Medicines and the country treatment protocols. RESULTS: This ESMO four-phase approach led to the update of the Kazakhstan national essential cancer medicines list and the list of cancer treatment protocols. This review has led to the withdrawal of several low-value or non-evidence-based medicines and a budget increase for cancer care to include all essential and highly effective medicines and treatment options. CONCLUSION: When applied effectively, this four-phase approach can improve access to medicines, efficiency of expenditure and sustainability of cancer systems. The WHO-ESMO collaboration illustrated how, by sharing best practices, tools and resources, we can address access to cancer medicines and positively impact patient care.

A David and Goliath set-up: a qualitative study of the challenges of ensuring the introduction of cost-effective new cancer medicines in Finland.

BACKGROUND: To combat the global challenge of cancer, priority has been placed on the research and development of new cancer medicines (NCMs). NCMs are often approved for marketing in accelerated processes. Despite significant advances in treating cancer, the overall added value and high prices of NCMs has been questioned. While market authorisations for NCMs are granted at the EU level, the assessment of added value, price negotiations and purchase or reimbursement decisions are made by member states. This article explores the practices in Finland for assessing and deciding on purchasing or reimbursing NCMs. METHODS: Semi-structured interviews were conducted with 26 civil servants, hospital employees, scientists, and representatives of cancer NGOs and of the pharmaceutical industry in 2019 and 2020. The transcribed interviews were coded inductively using Atlas.ti software and analysed thematically under 3 major themes and 11 sub-themes. RESULTS: The clinical value of NCMs is considered to be high, especially regarding NCMs for certain types of cancer. Proper patient selection is important but difficult and not all NCMs can be considered as adding value. The prices are considered to often be very high, leading to concerns about the sustainability and equity of health systems. Equity concerns among cancer patients are raised concerning differences in the availability of NCMs between hospital districts and cost differences for patients between those receiving outpatient and inpatient treatment. The systems and processes in Finland for deciding on the introduction of NCMs are fragmentary, involving separate approaches for outpatient care and hospital medicines by under-resourced evaluation bodies. The scientific evidence available is often limited for evidence-based decisions on introduction. Individual hospital districts sometimes introduce NCMs without assessment by national bodies. This can hamper the proper assessment of some NCMs before their uptake and lead to unequal access to NCMs by hospitals. There is an increasing lack of transparency about pricing, due to the rapid increase of market entry agreements. Lack of transparency on information on prices poses a challenge for authorities responsible for equitable access to cost-effective care within the available resources. CONCLUSIONS: Robust reform of the national introductory systems is needed. Internationally, efforts are needed to increase price transparency, to revise incentives within the system of market approval and to accumulate and assess evidence of comparable value and cost-effectiveness after the market approval of NCMs.

Trends in accessibility of negotiated targeted anti-cancer medicines in Nanjing, China: An interrupted time series analysis.

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.

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments.

BACKGROUND: Advances in cancer medicines have resulted in tangible health impacts, but the magnitude of benefits of approved cancer medicines could vary greatly. Health Technology Assessment (HTA) is a multidisciplinary process used to inform resource allocation through a systematic value assessment of health technology. This paper reviews the challenges in conducting HTA for cancer medicines arising from oncology trial designs and uncertainties of safety-efficacy data. METHODS: Multiple databases (PubMed, Scopus and Google Scholar) and grey literature (public health agencies and governmental reports) were searched to inform this policy narrative review. RESULTS: A lack of robust efficacy-safety data from clinical trials and other relevant sources of evidence has made HTA for cancer medicines challenging. The approval of cancer medicines through expedited pathways has increased in recent years, in which surrogate endpoints or biomarkers for patient selection have been widely used. Using these surrogate endpoints has created uncertainties in translating surrogate measures into patient-centric clinically (survival and quality of life) and economically (cost-effectiveness and budget impact) meaningful outcomes, with potential effects on diverting scarce health resources to low-value or detrimental interventions. Potential solutions include policy harmonization between regulatory and HTA authorities, commitment to generating robust post-marketing efficacy-safety data, managing uncertainties through risk-sharing agreements, and using value frameworks. CONCLUSION: A lack of robust efficacy-safety data is a central problem for conducting HTA of cancer medicines, potentially resulting in misinformed resource allocation.