Quality use of medicines within universal health coverage: challenges and opportunities

Background: Medicines are a major driver of quality, safety, equity, and cost of care in low and middle-income country health systems. Universal health coverage implementers must explicitly address appropriate use of medicines to realize the health benefits of medicines, avoid wasting scarce resources, and sustain the financial viability of universal health coverage schemes. Discussion Medicines are major contributors to the health and well-being of individuals and populations when used appropriately, and they waste resources and endanger health when used unnecessarily or incorrectly. Stakeholders need to balance inherently competing objectives in the pharmaceutical sector. Emerging and expanding UHC schemes provide potential levers to balance competing system objectives. To use these levers, sustainable universal coverage programs will require a) information systems that can track medicines utilization, expenditures, and quality of medicines use; b) routine monitoring of indicators of medicines availability, access, affordability, and use; c) policies and programs that facilitate appropriate medicines use by prescribers, dispensers, and patients; d) transparency in setting priorities for medicines coverage under resource constraints; and e) a system perspective to engage diverse actors. As they operationalize paths toward universal health coverage and include targeted medicines coverage policies and programs, systems can build on, and innovate, pharmaceutical policy frameworks and management tools from different countries’ settings. Summary Ensuring that medicines which achieve important health outcomes are available, accessible to all, used appropriately, and sustainably affordable is essential for realizing universal health coverage. Stakeholder cooperation and use of information and financing system levers provide opportunities to work toward this goal

Communication of survival data in US Food and Drug Administration-approved labeling of cancer drugs

This cross-sectional study examines how information on overall survival benefits of novel cancer drug indications is communicated in labeling

Costs of hospital care for hypertension in an insured population without an outpatient medicines benefit: an observational study in the Philippines

<p>Abstract</p> <p>Background</p> <p>Hypertension is the number one attributable risk factor for death throughout the world and a major contributor to morbidity, mortality, and increasing health care expenditures in the Philippines. Lack of access to outpatient antihypertensive medicines leads to avoidable disease progression and costly inpatient admissions. We estimated the cost to the Philippine Health Insurance Corporation (PhilHealth), which generally does not cover outpatient medicines, for inpatient care for hypertension and its sequelae.</p> <p>Methods</p> <p>Using PhilHealth inpatient claims for discharges between July 1, 2002 and December 31, 2005, we describe costs to PhilHealth for hospitalizations classified by primary discharge diagnoses into hospitalizations for hypertension; hypertensive heart and/or renal disease; other definite; and other possible consequences of untreated hypertension and assess disease trajectory for patients with more than one admission.</p> <p>Results</p> <p>PhilHealth reimbursed US $56 million for 444,628 hospitalizations for hypertension-related diagnoses incurred by 360,016 patients during 3.5 years; 42% of admissions were for essential or secondary hypertension; 19% for hypertensive heart or renal disease; and 39% for other consequences of untreated hypertension. Among 60,659 patients admitted during the first 18 months of the study with a diagnosis of essential or secondary hypertension, 9% were hospitalized again for treatment of sequelae; older individuals (vs. =< 40 years old), men, dependents (vs. members), and those who were employed (vs. in the private membership category) were more likely to be hospitalized again; as were those whose first admission during the study period was for consequences of hypertension (vs. essential or secondary hypertension).</p> <p>Conclusion</p> <p>Inpatient care for hypertension and its sequelae is expensive. Since many hospitalizations may be avoided with antihypertensive pharmacologic therapy, an outpatient medicines benefit may be one cost-effective policy option for PhilHealth.</p

Access to aff ordable medicines after health reform: evidence from two cross-sectional surveys in Shaanxi Province,western China

Background Limited access to essential medicines is a global problem. Improving availability and aff ordability of essential medicines is a key objective of the National Essential Medicine Policy (NEMP) in China. In its initial implementation in 2009, the NEMP targeted primary hospitals with policies designed to increase availability of essential medicines and reduce patients’ economic burden from purchasing medicines. We assessed medicine availability and price during the early years of the health reform in Shaanxi Province in underdeveloped western China. Methods We undertook two public (hospitals) and private (pharmacy) sector surveys of prices and availability of medicines, in September, 2010 and April, 2012, by a standard methodology developed by WHO and Health Action International. We measured medicine availability in outlets at the time of the surveys and infl ation-adjusted median unit prices (MUPs), taking 2010 as the base year. We used general estimating equations to calculate the signifi cance of diff erences in availability from 2010 to 2012 and the Wilcoxon signed rank test to calculate the signifi cance of diff erences in adjusted median prices. Findings We collected data from 50 public sector hospitals and 36 private sector retail pharmacies in 2010 and 72 public hospitals and 72 retail pharmacies in 2012. Mean availability of surveyed medicines was low in both the public and private sectors; availability of many essential medicines decreased from 2010 to 2012, particularly in primary hospitals (from 27·4% to 22·3% for lowest priced generics; p<0·0001). The MUPs of originator brands and their generic equivalents decreased signifi cantly from 2010 to 2012 in primary hospitals in comparison with secondary and tertiary hospitals. In the public sector, the median adjusted patient price was signifi cantly lower in 2012 than in 2010 for 16 originator brands (diff erence –11·7%; p=0·0019) and 29 lowest-priced generics (–5·2%; p=0·0015); the median government procurement price for originator brands also decreased signifi cantly (–10·9%; p=0·0004), whereas the decrease in median procurement price for lowest-priced generics was not signifi cant (–4·9%; p=0·17). In the private sector, the median percentage decrease in price between 2010 and 2012 for 38 lowest-priced generics was 4·7% (IQR 6·3–13·2), compared with 7·9% (4·9–13·9) for 16 originator brands. Interpretation Although infl ation-adjusted medicine prices were numerically lower, there were concerning decreases in availability of lowest-priced generic medicines in both the public and private sectors in 2012 from already low availability in 2010. A long-term, stable, and consistent information system is needed to monitor eff ects of further implementation of the Chinese Essential Medicine Policy

Health care payments in the asia pacific: validation of five survey measures of economic burden

Introduction: Many low and middle-income countries rely on out-of-pocket payments to help finance health care. These payments can pose financial hardships for households; valid measurement of this type of economic burden is therefore critical. This study examines the validity of five survey measures of economic burden caused by health care payments. Methods: We analyzed 2002/03 World Health Survey household-level data from four Asia Pacific countries to assess the construct validity of five measures of economic burden due to health care payments: any health expenditure, health expenditure amount, catastrophic health expenditure, indebtedness, and impoverishment. We used generalized linear models to assess the correlations between these measures and other constructs with which they have expected associations, such as health care need, wealth, and risk protection. Results: Measures of impoverishment and indebtedness most often correlated with health care need, wealth, and risk protection as expected. Having any health expenditure, a large health expenditure, or even a catastrophic health expenditure did not consistently predict degree of economic burden. Conclusions: Studies that examine economic burden attributable to health care payments should include measures of impoverishment and indebtedness

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

The Brazilian private pharmaceutical market after the first ten years of the generics law

Objectives: To describe changes in the private market for selected originators, branded generics (\u27similares\u27), and generic products during the 10 years following passage of the Brazilian Generics Law. Methods: We analyzed longitudinal data collected by IQVIA(R) on quarterly sales by wholesalers to retail pharmacies in Brazil from 1998 through 2010, grouped by originators, branded generics, and generic products in three therapeutic classes (antibiotics, antidiabetics, and antihypertensives). Outcomes included market share (proportion of the total private market volume), sales volume per capita, prices and number of manufacturers by group. Results: In the private market share, generics became dominant in each therapeutic class but the speed of uptake varied. Originators consistently lost most market share while branded generics varied over time. By the end of the study period, generics were the most sold product type in all classes, followed by branded generics. The number of generic manufacturers increased in all classes, while branded generics increased just after the policy but then decreased slowly through the end of 2010. For approximately 50% of the antibiotics analyzed, branded generics and generics had lower prices than originators. For antidiabetics, branded generic and generic prices were quite similar during the period analyzed. Price trends for the various subclasses of antihypertensive exhibited very different patterns over time. Conclusion: Sales of branded generics and originators decreased substantially in the three therapeutic classes analysed following the introduction of the generics policy in Brazil, but the time to market dominance of generics varied by class

Overall survival benefits of cancer drugs approved in China from 2005 to 2020

Importance: Of approximately 9 million patients with cancer in China in 2020, more than half were diagnosed with late-stage cancers. Recent regulatory reforms in China have focused on improving the availability of new cancer drugs. However, evidence on the clinical benefits of new cancer therapies authorized in China is not available. Objective: To characterize the clinical benefits of cancer drugs approved in China, as defined by the availability and magnitude of statistically significant overall survival (OS) results. Design, Setting, and Participants: This mixed-methods study comprising a systematic review and cross-sectional analysis identified antineoplastic agents approved in China between January 1, 2005, and December 31, 2020, using publicly available data and regulatory review documents issued by the National Medical Products Administration. The literature published up to June 30, 2021, was reviewed to collect results on end points used in pivotal trials supporting cancer drug approvals. Main Outcomes and Measures: The primary outcome measure was a documented statistically significant positive OS difference between a new cancer therapy and a comparator treatment. Secondary outcome measures were the magnitude of OS benefit and other primary efficacy measures in pivotal trials. Results: Between 2005 and 2020, 78 cancer drugs corresponding to 141 indications were authorized in China, including 20 drugs (25.6%) (for 30 indications) approved in China only. Of all indications, 26 (18.4%) were evaluated in single-arm or dose-optimization trials, most of which were authorized after 2017. By June 30, 2021, 34 drug indications (24.1%) had a documented lack of OS gain. For 68 indications (48.2%) that had documented evidence of OS benefit, the median magnitude of OS improvement was 4.1 (range, 1.0-35.0) months. After a median follow-up of 1.9 (range, 1.0-11.1) years from approval, OS data for 13 indications (9.2%) were either not reported or were still not mature. Fewer than one-third of cancer drug indications approved in China only had documented evidence of OS benefits (9 of 30 [30.0%]), whereas more than one-half of the cancer drug indications also available in the US or Europe had OS benefits (59 of 111 [53.1%]). Conclusions and Relevance: In this study, almost half of cancer drug indications approved in China had demonstrated OS gain. With the increase of cancer drug approvals based on single-arm trials or immature survival data in recent years, these findings highlight the need to routinely monitor the clinical benefits of new cancer therapies in Chin

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

Abstract 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

Communication of anticancer drug benefits and related uncertainties to patients and clinicians: document analysis of regulated information on prescription drugs in Europe

Objective To evaluate the frequency with which relevant and accurate information about the benefits and related uncertainties of anticancer drugs are communicated to patients and clinicians in regulated information sources in Europe. Design Document content analysis. Setting European Medicines Agency. Participants Anticancer drugs granted a first marketing authorisation by the European Medicines Agency, 2017-19. Main outcome measures Whether written information on a product addressed patients’ commonly asked questions about: who and what the drug is used for; how the drug was studied; types of drug benefit expected; and the extent of weak, uncertain, or missing evidence for drug benefits. Information on drug benefits in written sources for clinicians (summaries of product characteristics), patients (patient information leaflets), and the public (public summaries) was compared with information reported in regulatory assessment documents (European public assessment reports). Results 29 anticancer drugs that received a first marketing authorisation for 32 separate cancer indications in 2017-19 were included. General information about the drug (including information on approved indications and how the drug works) was frequently reported across regulated information sources aimed at both clinicians and patients. Nearly all summaries of product characteristics communicated full information to clinicians about the number and design of the main studies, the control arm (if any), study sample size, and primary measures of drug benefit. None of the patient information leaflets communicated information to patients about how drugs were studied. 31 (97%) summaries of product characteristics and 25 (78%) public summaries contained information about drug benefits that was accurate and consistent with information in regulatory assessment documents. The presence or absence of evidence that a drug extended survival was reported in 23 (72%) summaries of product characteristics and four (13%) public summaries. None of the patient information leaflets communicated information about the drug benefits that patients might expect based on study findings. Scientific concerns about the reliability of evidence on drug benefits, which were raised by European regulatory assessors for almost all drugs in the study sample, were rarely communicated to clinicians, patients, or the public. Conclusions The findings of this study highlight the need to improve the communication of the benefits and related uncertainties of anticancer drugs in regulated information sources in Europe to support evidence informed decision making by patients and their clinicians