How to Price and to Reimburse Publicly Funded Medicines in Latin America? Lessons Learned from Europe.

This paper reviews the main pricing policies in Latin American countries, discussing their shortcomings. It also gives an overview of the most common pricing and reimbursement policies in Europe and describes in detail three well-established approaches - international price referencing, value-based pricing, including setting up of health technology assessment, and generic and biosimilar policies - building on country examples.

The Complex Cancer Care Coverage Environment - What is the Role of Legislation? A Case Study from Massachusetts.

Over the past decades, anti-cancer treatments have evolved rapidly from cytotoxic chemotherapies to targeted therapies including oral targeted medications and injectable immuno-oncology and cell therapies. New anti-cancer medications come to markets at increasingly high prices, and health insurance coverage is crucial for patient access to these therapies. State laws are intended to facilitate insurance coverage of anti-cancer therapies.Using Massachusetts as a case study, we identified five current cancer coverage state laws and interviewed experts on their perceptions of the relevance of the laws and how well they meet the current needs of cancer care given rapid changes in therapies. Interviewees emphasized that cancer therapies, as compared to many other therapeutic areas, are unique because insurance legislation targets their coverage. They identified the oral chemotherapy parity law as contributing to increasing treatment costs in commercial insurance. For commercial insurers, coverage mandates combined with the realities of new cancer medications - including high prices and often limited evidence of efficacy at approval - compound a difficult situation. Respondents recommended policy approaches to address this challenging coverage environment, including the implementation of closed formularies, the use of cost-effectiveness studies to guide coverage decisions, and the application of value-based pricing concepts. Given the evolution of cancer therapeutics, it may be time to evaluate the benefits and challenges of cancer coverage mandates.

Integrating public preferences into national reimbursement decisions: a descriptive comparison of approaches in Belgium and New Zealand.

BACKGROUND: Public health care payer organizations face increasing pressures to make transparent and sustainable coverage decisions about ever more expensive prescription drugs, suggesting a need for public engagement in coverage decisions. However, little is known about countries' approaches to integrating public preferences in existing funding decisions. The aim of this study was to describe how Belgium and New Zealand used deliberative processes to engage the public and to identify lessons learned from these countries' approaches. METHODS: To describe two countries' deliberative processes, we first reviewed key country policy documents and then conducted semi-structured interviews with five leaders of the processes from Belgium and New Zealand. We assessed each country's rationales for and approaches to engaging the public in pharmaceutical coverage decisions and identified lessons learned. We used qualitative content analysis of the interviews to describe key themes and subthemes. RESULTS: In both countries, the national public payer organization initiated and led the process of integrating public preferences into national coverage decision making. Reimbursement criteria considered outdated and changing societal expectations prompted the change. Both countries chose a deliberative process of public engagement with a multi-year commitment of many stakeholders to develop new reimbursement processes. Both countries' new reimbursement processes put a stronger emphasis on quality of life, the separation of individual versus societal perspectives, and the importance of final reimbursement decisions being taken in context rather than based largely on cost-effectiveness thresholds. CONCLUSIONS: To face the growing financial pressure of sustainable funding of medicines, Belgium's and New Zealand's public payers have developed processes to engage the public in defining the reimbursement system's priorities. Although these countries differ in context and geographic location, they came up with overlapping lessons learnt which include the need for 1) political commitment to initiate change, 2) broad involvement of all stakeholders, and 3) commitment of all to engage in a long-term process. To evaluate these changes, further research is required to understand how coverage decisions in systems with and without public engagement differ.

Expanding Coverage of Oncology Drugs in an Aging, Upper-Middle-Income Country: Analyses of Public and Private Expenditures in Chile.

PURPOSE: The population of Chile has aged, and in 2017, cancer became the leading cause of death. Since 2005, a national health program has expanded coverage of drugs for 13 types of cancer and related palliative care. We describe the trends in public and private oncology drug expenditures in Chile and consider how increasing expenditures might be addressed. METHODS: We analyzed total quarterly drug expenditures for 131 oncology drugs from quarter (Q)3 2012 until Q1 2017, including public and private insurance payments and patient out-of-pocket spending. The data were analyzed by drug-mix, sources of funding, growth, and intellectual property status. The Laspeyres Price Index was used to analyze expenditure growth. RESULTS: We found 131 oncology drugs associated with 87,129 observations. Spending on drugs rose 120% from the first period, spanning from the first 3 quarters (Q3, Q4, Q1 2012-2013) to the last period (Q3, Q4, Q1 2016-2017), corresponding to an annualized rate of 19.2% and totaling US$398 million (in 2017 dollars). The public sector accounted for 84.2% of spending, which included 50 drugs in the official treatment protocols, whereas private insurance accounted for 7.3% in on-protocol drugs. The remaining 8.5% was paid out of pocket. In the public sector, more than 90% of growth resulted from increased use. Seven drugs, including 3 with nonexpired patents, accounted for 50% of total expenditures. CONCLUSION: Increased use and access enabled by expanded public expenditures drove most of the growth in oncology drug expenditures. However, the rate of public expenditure growth may be fiscally unsustainable. Policies are urgently needed to promote the use of generic drugs, the appropriate mix of on-protocol versus off-protocol drugs, and the curbing of off-label prescribing.

Access to medicines through health systems in low- and middle-income countries.

Nearly 2 billion people globally have no access to essential medicines. This means essential medicines are unavailable, unaffordable, inaccessible, unacceptable or of low quality for more than a quarter of the population worldwide. This supplement demonstrates the implications of poor medicine access and highlights recent innovations to improve access to essential medicines by presenting new research findings from low- and middle-income countries (LMICs). These studies answer key questions such as: Can performance-based financing improve availability of essential medicines? How affordable are cardiovascular treatments for children? Which countries' legal frameworks promote universal access to medicines? How appropriately are people using medicines? Do poor-quality medicines impact equity? Answers to these questions are important as essential medicines are vital to the Sustainable Development Goals and are central to the goal of achieving Universal Health Coverage. Access to affordable, quality-assured essential medicines is crucial to reducing the financial burden of care, preventing greater pain and suffering, shortening the duration of illness, and averting needless disabilities and deaths worldwide. This supplement was organized by the Medicines in Health Systems Thematic Working Group of Health Systems Global, a membership organization dedicated to promoting health systems research and knowledge translation. The five studies in the supplement further our understanding by showcasing recent successes and challenges of improving access to quality-assured medicines through health systems in LMICs.

An analysis of expenditures on primary care prescription drugs in the United States versus ten comparable countries.

OBJECTIVE: We sought to estimate size and sources of differences in per capita expenditures on primary care medications in the US versus ten comparable countries combined: Australia, Canada, France, Germany, the Netherlands, New Zealand, Norway, Sweden, Switzerland, and the United Kingdom. METHODS: Using market research data on year 2015 volumes and sales of medicines, we measure total per capita expenditures on six categories of primary care prescription drugs: hypertension treatments, pain medications, lipid lowing medicines, non-insulin diabetes treatments, gastrointestinal preparations, and antidepressants. We quantified the contributions of five drivers of the observed differences in per capita expenditures. RESULTS: We estimated that the US spent 203% more per capita on primary care pharmaceuticals than did the ten comparable countries. Despite the difference in spending levels, on average, Americans actually purchased 12% fewer days of related therapies than residents of the comparator countries. Most of the observed difference in expenditures was due to higher transaction prices of medicines and the use of a more expensive mix of medicines in the US. CONCLUSIONS: If utilization patterns and pharmaceutical prices in the US were similar to those in the 10 comparator countries combined, total spending on primary care pharmaceuticals would fall by 30% or more. Such evidence on the level and drivers of US pharmaceutical expenditures should inform policies in this sector.

Total and out-of-pocket expenditures among women with metastatic breast cancer in low-deductible versus high-deductible health plans.

PURPOSE: High-deductible health plan (HDHP) enrollment is expanding rapidly and might substantially increase out-of-pocket (OOP) payment burden. We examined trends in total and OOP health service expenditures overall and by insurance coverage type among women with metastatic breast cancer. METHODS: We used a longitudinal time series design to examine measures among 5364 women with metastatic breast cancer insured by a large US health insurer from 2004 to 2011. We measured outcomes during the 12 months after a first identified metastatic breast cancer diagnosis and required women to have at least 6 months of prior enrollment. We plotted enrollment measures and adjusted total and OOP spending. We fit trend lines using linear autoregressive models. RESULTS: Between 2004 and 2011, the percentage of women with metastatic breast cancer enrolled in employer-mandated HDHPs increased from 8 to 23% while the percentage enrolled in employer-mandated low-deductible plans (LDHPs) decreased from 69 to 37%. Over the same time period, estimated annual inflation-adjusted total health service spending among women with metastatic breast cancer whose employers only offered HDHPs or LDHPS increased from $96,899 to $104,688 (increase of $1197 per year; 95% confidence interval [CI]: $47,$2,348). Corresponding OOP spending values among these women with employer-mandated deductible levels were $4,496 and $5,151 ($91 per year trend; 95% CI -$13,$195). From 2004-2011, women in HDHPs and LDHPs had unchanged annual OOP spending, estimated at of $6642 (95% CI $6,268,$7016) and $4,247 (95% CI $3956,$4538), respectively. Thus, women in HDHPs experienced 55% (44%, 66%) more OOP spending than women in LDHP. CONCLUSIONS: OOP spending among women with metastatic breast cancer and employer-mandated deductible levels was 55% higher among HDHP than LDHP members, and employer-mandated HDHP enrollment increased substantially from 2004 to 2011. Stakeholders and policymakers should design health plans that protect financially vulnerable cancer patients from high OOP costs.

Defining Value of Cancer Therapeutics-A Health System Perspective.

Because of the rising costs of cancer care and ongoing challenges in ensuring access to quality care, there is an increasing need to prioritize spending and define the benefits of therapy in proportion to costs. The term "value" has gained favor as means to define the relative utility of a medical intervention in terms of benefits, risks, and financial costs, which in turn can help clinicians, patients, and policy makers prioritize "high-value" care. While numerous value concepts have been proposed, a comprehensive discussion of value initiatives along the care continuum is missing. In this Commentary, we propose a health system taxonomy of value initiatives in cancer care to discuss what the field needs to progress.

Drivers of expenditure on primary care prescription drugs in 10 high-income countries with universal health coverage.

BACKGROUND: Managing expenditures on pharmaceuticals is important for health systems to sustain universal access to necessary medicines. We sought to estimate the size and sources of differences in expenditures on primary care medications among high-income countries with universal health care systems. METHODS: We compared data on the 2015 volume and cost per day of primary care prescription drug therapies purchased in 10 high-income countries with various systems of universal health care coverage (7 from Europe, in addition to Australia, Canada and New Zealand). We measured total per capita expenditure on 6 categories of primary care prescription drugs: hypertension treatments, pain medications, lipid-lowering medicines, noninsulin diabetes treatments, gastrointestinal preparations and antidepressants. We quantified the contributions of 5 drivers of the observed differences in per capita expenditures. RESULTS: Across countries, the average annual per capita expenditure on the primary care medicines studied varied by more than 600%: from $23 in New Zealand to $171 in Switzerland. The volume of therapies purchased varied by 41%: from 198 days per capita in Norway to 279 days per capita in Germany. Most of the differences in average expenditures per capita were driven by a combination of differences in the average mix of drugs selected within therapeutic categories and differences in the prices paid for medicines prescribed. INTERPRETATION: Significant international differences in average expenditures on primary care medications are driven primarily by factors that contribute to the average daily cost of therapy, rather than differences in the volume of therapy used. Average expenditures were lower among single-payer financing systems that appeared to promote lower prices and the selection of lower-cost treatment options.

The Impact of the Affordable Care Act on Cancer Survivorship.

In 2010, the Patient Protection and Affordable Care Act (ACA) was implemented with the aim of expanding access to quality, affordable care. In this review, we describe the ACA provisions that are most relevant for cancer survivors, provide available published evidence, and offer insights for future research. We found that provisions focusing on access to preventive care, access to quality and coordinated care, and coverage expansion and increased affordability suggest beneficial effects. However, we identified research gaps specifically addressing the intended and unintended consequences of the ACA on cancer survivorship care. Whether or not the ACA continues in its current form, research should address the effects of enhanced preventive services, innovative models of care, and payment structures that promote quality of care, as well as access to affordable, equitable care for a growing population of cancer survivors.

A rapidly changing global medicines environment: How adaptable are funding decision-making systems?

BACKGROUND: With the launch of very highly priced therapies and sudden price increases of generics, pressures on health systems have drastically increased. OBJECTIVES: We aimed to elicit opinions of key decision makers responsible for national assessment and funding decisions on their experiences to adapt to these new realities. METHODS/SETTING: Through interviews with decision makers of pharmaceutical assessment and/or funding agencies, we describe the challenges systems are currently facing, systems' responses and systems' characteristics facilitating or hindering responses to changes and overarching topics for the future. RESULTS: Among the most common challenges are increased funding pressures, increased uncertainty and lack of transparency in decision-making. Systems' responses include utilization management, changing of assessment processes, stakeholder engagement and a focus on outcomes and on coordinated negotiations. Integrated delivery systems, fixed health care budgets and geographic and historical characteristics facilitate or sometimes hinder responses to change. Future policy emphasis lays on expanding data structures, managing the exit of drugs funded early, and implementing processes for communications with patients and the public. CONCLUSIONS: Going forward emphasis has to be given to structured communications with all stakeholders with a specific emphasis on the broader public and patients about financial limits and priority setting in health care.

Racial disparities in all-cause mortality among younger commercially insured women with incident metastatic breast cancer.

Racial disparities in breast cancer mortality persist and are likely related to multiple factors. Over the past decade, progress has been made in treating metastatic breast cancer, particularly in younger women. Whether disparities exist in this population is unknown. Using administrative claims data between 2000 and 2011 (OptumInsight, Eden Prairie, MN) of members insured through a large national US health insurer, we identified women aged 25-64 years diagnosed with incident metastatic breast cancer diagnosed between November 1, 2000, and December 31, 2008. We examined time from diagnosis to death, with up to 3 years of follow-up. We stratified analyses by geocoded race and socio-economic status, age-at-diagnosis, morbidity score, US region of residence, urban/non-urban, and years of diagnosis. We constructed Kaplan-Meier survival plots and analyzed all-cause mortality using multivariate Cox proportional hazard models. Among 6694 women with incident metastatic breast cancer (78 % Caucasian, 4 % African American, and 18 % other), we found higher mortality rates among women residing in predominantly African American versus Caucasian neighborhoods (hazard ratio (HR) 1.84; 95 % confidence interval, CI 1.39-2.45), women with high versus lower morbidity (HR 1.30 [1.12-1.51]), and women whose incident metastatic diagnosis was during 2000-2004 versus 2005-2008 (HR 1.60 [1.39-1.83]). Caucasian (HR 0.61 [0.52-0.71]) but not African American women (HR not significant) experienced improved mortality in 2005-2008 versus 2000-2004. Despite insured status, African American women and women with multi-morbidity had poorer survival. Only Caucasian women had improved mortality over time. Modifiable risk factors for increased mortality need to be addressed in order to reduce disparities.

Thirty Years of Media Coverage on High Drug Prices in the United States--A Never-Ending Story or a Time for Change?

In recent years drug prices have increasingly become a topic of debate for patients, providers, payers and policy makers. To place the current drug price debate into historical context, we searched the New York Times and Wall Street Journal from 1985 - 2015 and found that concerns about drug prices have commonly featured in the press over the study period with recently stronger calls for change. Price levels, types of innovations, stakeholder responses, and strategies to address high prices discussed in the media suggest that concerted efforts are required to enable affordable and high-value innovations.

Effect of the economic recession on pharmaceutical policy and medicine sales in eight European countries.

OBJECTIVE: To identify pharmaceutical policy changes during the economic recession in eight European countries and to determine whether policy measures resulted in lower sales of, and less expenditure on, pharmaceuticals. METHODS: Information on pharmaceutical policy changes between 2008 and 2011 in eight European countries was obtained from publications and pharmaceutical policy databases. Data on the volume and value of the quarterly sales of products between 2006 and 2011 in the 10 highest-selling therapeutic classes in each country were obtained from a pharmaceutical market research database. We compared these indicators in economically stable countries; Austria, Estonia and Finland, to those in economically less stable countries, Greece, Ireland, Portugal, Slovakia and Spain. FINDINGS: Economically stable countries implemented two to seven policy changes each, whereas less stable countries implemented 10 to 22 each. Of the 88 policy changes identified, 33 occurred in 2010 and 40 in 2011. They involved changing out-of-pocket payments for patients in 16 cases, price mark-up schemes in 13 and price cuts in 11. Sales volumes increased moderately in all countries except Greece and Portugal, which experienced slight declines after 2009. Sales values decreased in both groups of countries, but fell more in less stable countries. CONCLUSION: Less economically stable countries implemented more pharmaceutical policy changes during the recession than economically stable countries. Unexpectedly, pharmaceutical sales volumes increased in almost all countries, whereas sales values declined, especially in less stable countries.

Impact of pharmaceutical policy interventions on utilization of antipsychotic medicines in Finland and Portugal in times of economic recession: interrupted time series analyses.

OBJECTIVES: To analyze the impacts of pharmaceutical sector policies implemented to contain country spending during the economic recession--a reference price system in Finland and a mix of policies including changes in reimbursement rates, a generic promotion campaign and discounts granted to the public payer in Portugal - on utilization of, as a proxy for access to, antipsychotic medicines. METHODOLOGY: We obtained monthly IMS Health sales data in standard units of antipsychotic medicines in Portugal and Finland for the period January 2007 to December 2011. We used an interrupted time series design to estimate changes in overall use and generic market shares by comparing pre-policy and post-policy levels and trends. RESULTS: Both countries' policy approaches were associated with slight, likely unintended, decreases in overall use of antipsychotic medicines and with increases in generic market shares of major antipsychotic products. In Finland, quetiapine and risperidone generic market shares increased substantially (estimates one year post-policy compared to before, quetiapine: 6.80% [3.92%, 9.68%]; risperidone: 11.13% [6.79%, 15.48%]. The policy interventions in Portugal resulted in a substantially increased generic market share for amisulpride (estimate one year post-policy compared to before: 22.95% [21.01%, 24.90%]; generic risperidone already dominated the market prior to the policy interventions. CONCLUSIONS: Different policy approaches to contain pharmaceutical expenditures in times of the economic recession in Finland and Portugal had intended--increased use of generics--and likely unintended--slightly decreased overall sales, possibly consistent with decreased access to needed medicines--impacts. These findings highlight the importance of monitoring and evaluating the effects of pharmaceutical policy interventions on use of medicines and health outcomes.

Medicines discarded in household garbage: analysis of a pharmaceutical waste sample in Vienna.

OBJECTIVES: To analyze a sample of pharmaceutical waste drawn from household garbage in Vienna, with the aim to learn whether and which medicines end up unused in normal household waste. METHODS: We obtained a pharmaceutical waste sample from the Vienna Municipal Waste Department. This was drawn by their staff in a representative search in October and November 2009. We did a manual investigation of the sample which contained packs and loose blisters, excluded medical devices and traced loose blisters back to medicines packs. We reported information on the prescription status, origin, therapeutic group, dose form, contents and expiry date. We performed descriptive statistics for the total data set and for sub-groups (e.g. items still containing some of original content). RESULTS: In total, 152 packs were identified, of which the majority was prescription-only medicines (74%). Cardiovascular medicines accounted for the highest share (24%). 87% of the packs were in oral form. 95% of the packs had not expired. 14.5% of the total data set contained contents but the range of content left in the packs varied. Results on the packs with contents differed from the total: the shares of Over-the Counter medicines (36%), of medicines of the respiratory system (18%) and of the musculo-skeletal system (18%), for dermal use (23%) and of expired medicines (19%) were higher compared to the full data set. CONCLUSIONS: The study showed that some medicines end up unused or partially used in normal household garbage in Vienna. Our results did not confirm speculations about a high percentage of unused medicines improperly discarded. There is room for improved patient information and counseling to enhance medication adherence and a proper discharge of medicines.

Is Europe still heading to a common price level for on-patent medicines? An exploratory study among 15 Western European countries.

BACKGROUND: Previous studies have suggested that medicines prices in Europe converge over time as a result of policy measures such as external price referencing. OBJECTIVE: To explore whether ex-factory prices of on-patented medicines in Western European countries have converged over a recent period of time. METHODS: Prices of ten on-patent medicines in five years (2007, 2008, 2010, 2011, 2012) of 15 European countries were analyzed. The unit of analysis was the ex-factory price in Euro per defined daily dose (exchange rate indexed to 2007). A score (deviation from the average price) per country as well as the ranges were calculated for all medicines. RESULTS: The prices between countries and selected products varied to a great extent from as low as an average price of € 1.3/DDD for sitagliptin in 2010-2012 to an average of € 221.5/DDD for alemtuzumab in 2011. Between 2008 and 2012, a price divergence was seen which was fully driven by two countries, Germany (up to 27% more expensive than the average) and Greece (up to 32% cheaper than the average). All other countries had stable prices and centered around the country average. Prices of less expensive as well as expensive medicines remained relatively stable or decreased over time, while only the price of sirolimus relatively increased. CONCLUSIONS: Our study period included the time of the recession and several pricing policy measures may have affected the prices of medicines. Instead of the expected price convergence we observed a price divergence driven by price changes in only two of the 15 countries. All other European countries remained stable around the country average. Further research is needed to expand the study to a bigger sample size, and include prescribing data and Eastern European countries.