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This cross-sectional study included a nationally representative sample of U.S. adults aged ≥50 years with self-reported pain in the past 4 weeks from the 2018 Medical Expenditure Panel Survey. Adjusted linear regression analyses accounted for the complex survey design and assessed differences in several types of annual health care expenditures between individuals who reported frequent exercise (≥30 min of moderate–vigorous intensity physical activity ≥5 times per week) and those who did not. Approximately 23,940,144 of 56,979,267 older U.S. adults with pain reported frequent exercise. In adjusted analyses, individuals who reported frequent exercise had 15% lower annual prescription medication expenditures compared with those who did not report frequent exercise (p =.007). There were no statistical differences between frequent exercise status for other health care expenditure types (p >.05). In conclusion, adjusted annual prescription medication expenditures were 15% lower among older U.S. adults with pain who reported frequent exercise versus those who did not. [ABSTRACT FROM AUTHOR]

The article enumerates the available treatments for Alzheimer's disease, including when they were approved by the U.S. Food and Drug Administration (FDA), their effects, and how much they cost to patients. These include the Aducanumab (Aduhelm) that received approval in June 2021, the monoclonal antibody Lecanemab which was approved under the FDA's accelerated pathway based on phase 2 trial data, and the Donepezil (Aricept) which was approved by the FDA in 1996.

Background: In patients with pulmonary arterial hypertension (PAH), the use of disease-specific therapies (i.e., endothelin receptor antagonists, phosphodiesterase type-5 inhibitors, soluble guanylate cyclase stimulators, prostacyclins, and prostanoids) has been associated with disease improvement and decreased mortality risk. We aimed to quantify the adherence and discontinuation rates for patients prescribed PAH-specific therapies. Methods: We performed a systematic review via searching MEDLINE, EMBASE, and the Cochrane Library from their inception to 4 March 2022 for observational studies published in English that reported data on adherence to and persistence with PAH-targeted therapies. Random-effects meta-analysis was performed to explore average adherence and discontinuation rates. Results: In all, 14 studies involving 14,861 individuals prescribed PAH-targeted therapies were included. The overall pooled proportion of patients adherent to their PAH-targeted medications was 60.9% (95% confidence interval [CI] 52.3–69.1%). The pooled proportions of patients adherent in questionnaire-based studies and in studies using prescription/dispensing data were 52.9% (95% CI 48.9–56.9%) and 62.9% (95% CI 53.1–72.2%), respectively. The pooled proportion of patients who discontinued their PAH-targeted medications was 42.3% (95% CI 31.6–53.3). Factors reported to impact adherence included administration frequency, length of time on treatment, co-payment, and occurrence of adverse events. Conclusions: In the real world, a considerable proportion of patients prescribed PAH-specific therapies were non-adherent or discontinued. As diverse factors may influence treatment adherence, multifaceted interventions are needed to address this trend in order to improve patient outcomes. Registration: The systematic review protocol was registered in the PROSPERO database (CRD42022316638). [ABSTRACT FROM AUTHOR]

This study evaluated the early drug prescription patterns as predictors of final workers compensation claim costs and closure based on an updated analysis on a large cohort. The result would provide claim management with tools to estimate final claims outcomes in the first 3- or 6-months post-injury. Objective: The aim of the study is to determine the associations of workers' compensation claim costs and return to work with drugs prescribed for early symptom management. Methods: Claims filed from 1998 to 2007 were followed for 10 years from the injury date. Drugs analyzed included gabapentin, pregabalin, antipsychotics, antidepressants, sedatives, benzodiazepines, carisoprodol, and opioids, controlling for initial reserve, sex, age, physical therapy, attorney involvement, and surgery. Results: Gabapentin, antipsychotics, antidepressants, and sedatives used in the first 3 months after injury were significantly associated with higher claim cost (≥$100,000). All opioid morphine equivalent doses greater than or equal to 5 mg/d for the first 6 months was significantly associated with higher cost (≥$100,000) and not being released to work at end of third year after injury with dose-response relationships. Conclusions: Prescription patterns in the first 3 months or first 6 months of workers' compensation claim development may be used as predictors of claim outcomes. [ABSTRACT FROM AUTHOR]

Assessing opioid prescription patterns is key to understanding the opioid crisis. Some researchers have suggested that work and workplace issues are not adequately represented in efforts to reduce the opioid crisis. This paper addresses this concern by comparing opioid prescription patterns of occupational injury-caused conditions to other injury-caused conditions. Objectives: This study aimed to compare opioid prescription incidence, supply days, and cost associated with occupational injury and other injury-caused conditions. Methods: We used Medical Expenditure Panel Survey (MEPS) data for 2010–2019. The MEPS provides information on medical conditions and associated medical encounters, treatments, and treatment costs, as well as demographic, education, health, working status, income, and insurance coverage information. We used descriptive statistics and logistic and 2-part regressions. Results: Controlling for covariates and compared with other injury-caused conditions, occupational injury–caused conditions resulted in 33% higher odds of opioid prescribing, 32.8 more opioid prescription supply days, and $134 higher average cost. Conclusions: Occupational injuries were associated with higher opioid incidence and costs, and more opioid supply days. These findings point to the need to focus on making work safer and the role employers may play in supporting worker recovery from injury and opioid use disorders. [ABSTRACT FROM AUTHOR]

Consumer cost sharing is widely employed by payers in the United States in an effort to control spending. Most cost-sharing strategies set patient contributions on the basis of costs incurred by payers and often do not consider medical necessity as a coverage criterion. Available evidence suggests that increases in cost sharing worsen health disparities and adversely affect patient-centered outcomes, particularly among economically vulnerable individuals, people of color, and those with chronic conditions. A key question has been how to better engage consumers while balancing appropriate access to essential services with increasing fiscal pressures. Value-based insurance design (VBID) is a promising approach designed to improve desired clinical and financial outcomes, in which out-of-pocket costs are based on the potential for clinical benefit, taking into consideration the patient's clinical condition. For more than two decades, broad multi stakeholder support and multiple federal policy initiatives have led to the implementation of VBID programs that enhance access to vital preventive and chronic disease medications for millions of Americans. A robust evidence base shows that when financial barriers to essential medications are reduced, increased adherence results, leading to improved patient-centered outcomes, reduced health care disparities, and in some (but not most) instances, lower total medical expenditures. [ABSTRACT FROM AUTHOR]

How and why is implicit and explicit human rights language used by World Trade Organization (WTO) negotiators in debates about intellectual property, know-how, and technology needed to manufacture COVID-19 vaccines, and how do these findings compare with negotiators' human rights framing in 2001? Sampling 26 WTO members and two groups of members, this study uses document analysis and six key informant interviews with WTO negotiators, a representative of the WTO Secretariat, and a nonstate actor. In WTO debates about COVID-19 medicines, negotiators scarcely used human rights frames (e.g., "human rights" or "right to health"). Supporters used both human rights frames and implicit language (e.g., "equity," "affordability," and "solidarity") to garner support for the TRIPS waiver proposal, while opponents and WTO members with undetermined positions on the waiver used only implicit language to advocate for alternative proposals. WTO negotiators use human rights frames to appeal to previously agreed language about state obligations; for coherence between their domestic values and policy on one hand, and their global policy positions on the other; and to catalyze public support for the waiver proposal beyond the WTO. This mixed-methods design yields a rich contextual understanding of the modern role of human rights language in trade negotiations relevant for public health. [ABSTRACT FROM AUTHOR]

Introduction: Neglected tropical diseases (NTDs) are an important global health challenge, however, little is known about how to effectively finance NTD related services. Integrated management in particular, is put forward as an efficient and effective treatment modality. This is a background study to a broader health economic evaluation, seeking to document the costs of integrated case management of NTDs versus standard care in Liberia. In the current study, we document barriers and facilitators to NTD care from a health financing perspective. Methods: We carried out key informant interviews with 86 health professionals and 16 national health system policymakers. 46 participants were active in counties implementing integrated case management and 40 participants were active in counties implementing standard care. We also interviewed 16 patients and community members. All interviews were transcribed and analysed using the thematic framework approach. Findings: We found that decentralization for NTD financing is not yet achieved – financing and reporting for NTDs is still centralized and largely donor-driven as a vertical programme; government involvement in NTD financing is still minimal, focused mainly on staffing, but non-governmental organisations (NGOs) or international agencies are supporting supply and procurement of medications. Donor support and involvement in NTDs are largely coordinated around the integrated case management. Quantification for goods and budget estimations are specific challenges, given the high donor dependence, particularly for NTD related costs and the government's limited financial role at present. These challenges contribute to stockouts of medications and supplies at clinic level, while delays in payments of salaries from the government compromise staff attendance and retention. For patients, the main challenges are high transportation costs, with inflated charges due to fear and stigma amongst motorbike taxi riders, and out-of-pocket payments for medication during stockouts and food/toiletries (for in-patients). Conclusion: Our findings contribute to the limited work on financing of SSSD services in West African settings and provide insight on challenges and opportunities for financing and large costs in accessing care by households, which is also being exacerbated by stigma. [ABSTRACT FROM AUTHOR]

Background: Commonly recognized childhood conduct problems often lead to costly problems in adulthood. This study aimed to evaluate the long‐term cumulative cost of childhood conduct problems until the age of 30. The costs included inpatient care, nervous system medicine purchases, and criminal offences. Methods: The study used population‐based nationwide 1981 birth cohort data. Families and teachers assessed the conduct problems of the eight‐year‐olds based on Rutter questionnaires. We grouped 5,011 children into low‐level of conduct problems (52%), intermediate‐level of conduct problems (37%), and high‐level of conduct problems (11%) groups, based on combined conduct symptoms scores. The analysis included the cohort data with the Care Register for Health Care, the Drug Prescription Register, and the Finnish Police Register. The cost valuation of service use applied national unit costs in 2016 prices. We used Wilcoxon rank‐sum test to test the differences between groups and gender. Results: During 1989–2011, average cumulative costs of the high‐level (€44,348, p <.001) and the intermediate‐level (€19,405, p <.001) of conduct problems groups were higher than the low‐level of conduct problems group's (€10,547) costs. In all three groups, the boys' costs were higher than girls' costs. Conclusions: The costs associated with conduct problems in childhood are substantial, showing a clear need for cost‐effective interventions. Implementation decisions of interventions benefit from long‐term cost‐effectiveness modelling studies. Costing studies, like this, provide cost and cost offset information for modelling studies. [ABSTRACT FROM AUTHOR]

The purpose of this study was to examine the temporal trends and dynamics of financial hardship among older adults in the U.S. between 2006 and 2016 using the Health and Retirement Study. Sample included a total of 13,537 eligible person observations with a median age of 68 years. Financial hardship included measures of difficulty paying bills, food insecurity, taking less medication due to cost, and ongoing financial strain. Regression analyses were performed using a three-wave quadrennial model to estimate the prevalence of financial hardship over time, to explore temporal patterns and identify persistent hardship. Findings reveal that 51% of respondents who experienced food insecurity at one or more waves were transient. This pattern was similar to respondents who experienced ongoing financial strain (52% transient). Respondents who reported difficulty paying bills (68%) and reduced medications due to cost (62%) were also transient. Significant predictors across all four domains of financial hardship include age, years of education, marital status, self-rated health. Being African American was positively associated with reduced medication use and food insecurity. This study provides insight into the temporal dynamics of financial hardship in later life. It also highlights the contiguous, intermediate and transient nature of financial hardship among older adult populations. [ABSTRACT FROM AUTHOR]

There is an alarming delay in Europe for anticancer medicines becoming accessible for children. Following a paediatric European Union marketing authorisation, national Health Technology Assessment (HTA) agencies evaluate effectiveness, and safety of medicines to support decision on their cost and reimbursement. This study (a SIOPE Access to Medicines project) aimed to evaluate how these HTA evaluations take place for anticancer medicines indicated for paediatric use in Europe and to explore where the delays for market access originate. We obtained HTA reports from the public domain for nine European countries for blinatumomab, dinutuximab beta and tisagenlecleucel. We evaluated the time elapsed between marketing authorisation for a paediatric indication and a national HTA decision and the nature of the decision. Out of 23 HTA decisions (four countries without blinatumomab report), 18 were positive, two with restrictions, three negative. For blinatumomab, tisagenlecleucel and dinutuximab beta, the median time to an HTA decision after regulatory approval for paediatric use was 353 days (range 193–751), 141 days (range 77–517) and 515 days (range 0–780), respectively, with variability between countries. Dinutuximab beta and tisagenlecleucel were first introduced in children, but did not result in shorter time to HTA decision. For blinatumomab, marketing authorisation followed 1008 days after the indication in adults, with HTA applications submitted a median of 167 days later, and a recommendation after 145 days. This study reveals ample variability in HTA decision making in nine European Union countries. Collaboration and alignment of required evidence is needed to facilitate robust scientific HTA assessments, also considering methodological challenges in paediatric oncology. • Health technology assessments of paediatric oncology drugs vary greatly within Europe. • The timing of HTA evaluation adds to the delay of patient access. • Differences between adult and paediatric trial methodology should be considered. • Multistakeholder collaboration between EMA, academia, pharmaceutical companies, patients advocates and HTAs is needed. [ABSTRACT FROM AUTHOR]

The proliferation of international and regional organisations in the last decades led to increasing overlap of memberships and mandates in social policy areas. Whereas the literature has explored the benefits and perils of institutional overlap though neglecting the social policy dimension of such processes, studies on regionalism have focused on single cases of regional organisations. This paper breaks new ground by examining the effects of the overlap in membership, health mandates and institutional mechanisms between the Common Market of the South (MERCOSUR) and the Union of South American Nations (UNASUR) between 2008 and 2018. It focuses on two cases of access to medicines: the creation of the medicine price bank and the price negotiation of high-cost medicines. Our argument is that the overlap was positive, leading to an incipient trend towards cooperation. This was facilitated by the membership structure, the expertise already accumulated in the region and the relations and networks among those involved in health regional cooperation. Overall, the article deepens our understanding of the conditions under which regional organisations, even in the context of institutional overlap, can contribute to adequately respond to transnational challenges, which, as global health, are not only persistent, but also profoundly affect our societies. [ABSTRACT FROM AUTHOR]

What is known and objective: Drug development is generally a long and expensive process. Regulatory authorities have established several expedited regulatory pathways, such as accelerated approval designation in the United States (1992) and conditional approval regulation in Japan (2017). In Japan, prior to 2017, the Pharmaceutical and Medical Device Agency (PMDA) granted marketing approval without requesting the results of confirmatory clinical trials case by case basis, and even after 2018, only three drugs have been approved via the conditional approval pathway, although many drugs without confirmatory clinical trials have been approved without this pathway; therefore, it is difficult to predict the circumstances under which confirmatory clinical trials may be waived. The aim of this study was to investigate the characteristics of drugs for which the requirement of confirmatory clinical trials for approval was waived in Japan. We also aimed to identify factors and formulae to predict a waiver of confirmatory clinical trials. Methods: Data on approved drugs and their characteristics were mainly extracted from the Japan Pharmaceuticals and Medical Device Agency database. The seriousness of the disease, existence of available treatments and number of patients were considered as candidate factors. The influence of each factor on receiving a waiver was determined using logistic regression analysis comparing drugs approved with and without confirmatory clinical trials as the binary response variable. The predictive formula was derived from the results of the logistic regression analysis. A receiver operating characteristic curve was used to evaluate the accuracy of the prediction. Results and discussion: Categorization of drugs as antineoplastic agents, use of the cost accounting method in the drug pricing system, "orphan" designation and accelerated approval designation in the United States emerged as significant factors in the logistic regression analysis, predicting a waiver for confirmatory clinical trials (p ≤ 0.05). These factors were then used to establish a predictive model to ascertain whether confirmatory clinical trials would be necessary for a new drug, exhibiting good sensitivity (0.8) and specificity (0.8) and high accuracy for newly approved drugs. What is new and conclusion: The identification of key factors that can predict waivers of confirmatory clinical trials may accelerate the development of clinically important drugs and improve patient access globally. This study investigated the characteristics of drugs that confirmatory clinical trials were waived for approval in Japan, and identified key factors and formulae to predict the waiver of confirmatory clinical trials. [ABSTRACT FROM AUTHOR]

The article discusses the impact of the intervention of pharmacists on drug related expenses due to COVID-19 for hospitalized patients within a health care system. Topics mentioned include the role of pharmacists in providing clinical expertise with drug therapy of COVID-19, the reduction in medication costs and medication waste due to the implementation of hospital-approved criteria, and the advantages of implementing pharmacist documentation at order verification.

Purpose Results of the first ASHP National Survey of Health-System Specialty Pharmacy Practice are presented. Methods A sample of 230 leaders in health-system specialty pharmacies were contacted by email and invited to participate in a survey hosted using an online survey application. The survey sample was compiled from ASHP member lists, through review of data from other ASHP surveys indicating the presence of specialty pharmacies, and by outreach to ASHP member organizational leaders. Results The response rate was 53.0%. Most health-system specialty pharmacies dispense 30,000 or fewer specialty prescriptions per year, have an annual revenue of $100 million or less, are part of an entity eligible to participate in the 340B Drug Pricing Program, operate 1 specialty pharmacy location, have at least 1 specialty pharmacy accreditation, dispense nonspecialty medications in addition to specialty medications, and employ an average of 13 pharmacists and 15 technicians. More than two-thirds of health-system specialty pharmacies (68.8%) dispense no more than half of the prescriptions written by their providers due to payer network restrictions or limited distribution drugs. The health-system specialty pharmacy practice model includes access to the electronic health record (100% of respondents), pharmacists and technicians dedicated to specific clinics (64.9% and 57.7%, respectively), specialty pharmacist involvement in treatment decisions and drug therapy selection prior to the prescription being written (64.9%), and documenting recommendations and progress notes in patients' electronic health record (93.4%). Most health-system specialty pharmacies (83.3%) offer experiential or formal education in specialty pharmacy. Top challenges that survey respondents expected to face in the next year included restricted access to payer networks and limited distribution drugs, 340B Drug Pricing Program changes, and shrinking reimbursement from payers. Conclusion The health-system specialty pharmacy represents an integrated advanced practice model that incorporates specialty medication-use management across the continuum of care. [ABSTRACT FROM AUTHOR]

Background: Successful diabetes treatment requires commitment and understanding of disease management by the patients. Objective: This trial aimed to evaluate the programme effectiveness of home medication review by community pharmacists (HMR-CP) in optimising diabetes care and reducing medication wastage. Methods: A randomised controlled trial was conducted on 166 patients with Type 2 Diabetes Mellitus (T2DM) who were randomly assigned to the intervention or control groups. The intervention group received HMR-CP at 0-month, 3-month, and 6-month. The primary outcome was haemoglobin A1c (HbA1c) while clinical outcomes, anthropometric data, and humanistic outcomes were the secondary outcomes. For the intervention group, drug-related problems (DRP) were classified according to the Pharmaceutical Care Network Europe Foundation (PCNE). Medication adherence was determined based on the Pill Counting Adherence Ratio (PCAR). The cost of medication wastage was calculated based on the total missed dose by the T2DM patients multiplied by the cost of medication. General linear model and generalised estimating equations were used to compare data across the different time-points within and between the groups, respectively. Results: No significant difference was observed in the demographic and anthropometric data at baseline between the two groups except for fasting blood glucose (FBG). There was a significant reduction in the HbA1c (-0.91%) and FBG (-1.62mmol/L) over the study period (p<0.05). A similar observation was noted in diastolic blood pressure (DBP) and total cholesterol (TC) but not in high-density lipoprotein (HDL), and anthropometric parameters. Both utility value and Michigan Diabetes Knowledge Test (MDKT) scores increased significantly over time. As for the intervention group, significant changes in PCAR (p<0.001) and the number of DRP (p<0.001) were noted. Conclusions: HMR-CP significantly improved the glycaemic control, QoL, medication adherence, and knowledge of T2DM patients as well as reduced the number of DRP and cost of medication wastage. However, the impact of HMR-CP on certain clinical and anthropometric parameters remains inconclusive and further investigation is warranted. [ABSTRACT FROM AUTHOR]

OBJECTIVES: To determine whether elimination of co-pays for prescription drugs affects medication adherence and total health care spending. STUDY DESIGN: Retrospective comparative study. METHODS: We conducted a difference-in-differences comparison in the year before and after expansion of a Zero Dollar Co-pay (ZDC) prescription drug benefit in commercially insured Louisiana residents. Blue Cross and Blue Shield of Louisiana members with continuous disease management program enrollment were analyzed, of whom 6463 were enrolled in the ZDC program and 1821 were controls who were ineligible because their employers did not opt in. RESULTS: After ZDC expansion, medication adherence fell in the control group and rose in the ZDC group, with a relative increase of 2.1 percentage points (P = .002). Medical spending fell by $71 per member per month (PMPM) (P = .027) in the ZDC group relative to controls. Overall, there was no significant increase in the cost of drugs between treatment and controls. However, when drugs were further categorized, there was a significant increase of $8 PMPM for generic drugs and no significant difference for brand name drugs. Comparisons of medication adherence rates by household income showed the largest relative increase post ZDC expansion among low-income members. CONCLUSIONS: Elimination of co-pays for drugs indicated to treat chronic illnesses was associated with increases in medication adherence and reductions in overall spending of $63. Benefit designs that eliminate co-pays for patients with chronic illnesses may improve adherence and reduce the total cost of care. [ABSTRACT FROM AUTHOR]

The author reflects on the proposed changes to the Aotearoa health system in New Zealand. Topics discussed include the significance of the disestablishment of District Health Boards and creation of a public health agency and an independent Māri Health Authority, role of the absence of adequate partnership with Māri in the monocultural biomedicine in the country, and the need for Māri representation in drafting legislation to support the changes.

Medicare Part D has significantly enhanced access to prescription drugs among Medicare beneficiaries. However, the recent rapid rise of utilization management policies in the Medicare Part D program may have adversely affected access to prescription drugs. I study the effects of expected and observed exposure to utilization management in prescription drug utilization using Medicare Part D claims data from 2009 to 2016 and an instrumental variables strategy based on the interaction of lagged health status and the set of plans available to each beneficiary. I find that the expected share of spending subject to utilization management increases the observed share, with the smallest effect for prior authorization. Increases in the expected share of drug spending subject to prior authorization increases Part D spending by $122.27 per percentage point, with almost three-quarters of this increase being paid by the Medicare program, rather than beneficiaries or plans. Comparable increases in step therapy and quantity limit exposure increase spending by $46 and decrease spending by $31, respectively. Interestingly, increased exposure to prior authorization and quantity limits increases the average price per 30-day prescription. [ABSTRACT FROM AUTHOR]

Objectives: Many countries use international (or external) reference pricing-benchmarking prices against those in other countries-to manage spending on prescription drugs. By contrast, the United States (US) allows manufacturers to set drug prices freely. In December 2019, a major bill passed the House of Representatives that would introduce international reference pricing to reduce US drug spending. In September 2020, President Trump issued an executive order to apply international reference pricing for drugs purchased under Medicare. As US policymakers consider adopting reference pricing, it is important to recognize four key administrative issues that have complicated other countries' experiences.Methods: We analyzed the US policy proposals and literature on international experience with international reference pricing to identify implementation challenges and potential effects of US adoption of international reference pricing.Results: Four key administrative issues were identified: lack of price transparency, delays in market approvals, the frequency of price revisions, and the prevalence of cross-referencing.Conclusions: Failure to account for the key issues in the emerging US approach will lead to overspending from overestimation of prices. Policymakers also need to recognize the collateral effects that the US adoption of international reference pricing may have on other countries' prices. Given the size of the pharmaceutical market in the US and other market issues, US reference pricing will likely increase drug list and net prices in other countries. Because of limitations in implementation and collateral effects, US policymakers should consider international reference pricing as a supportive tool alongside other cost containment policies, such as value-based pricing or volume agreements. International reference pricing could limit drug spending in the US but faces implementation challenges and will negatively affect other countries. [ABSTRACT FROM AUTHOR]

Background: Millions worth of unused drugs particularly those indicated for chronic diseases such as diabetes were returned and disposed leading to substantial wastage. Use of patients' own medications (POMs) in the inpatient setting has reduced wastage and saved cost. The impact of utilizing POMs in the outpatient setting has hitherto not been determined. Purpose: This study aims to compare the cost, medication adherence and glycaemic control of utilizing POMs versus usual dispensing. Methods: Prospective randomized controlled study was conducted among diabetic patients that required monthly medication refill in the Outpatient Pharmacy in 2017. Patients who consented were equally divided into POMs and control groups. Both groups brought excess medications from home at week-0 and week-12. Patients in the POMs group brought excess medications monthly and sufficient amount of drugs were added until the next refill date. Drugs were dispensed as usual in the control group. Total cost consisting of the cost of drugs, staff and building was calculated. Glycosylated haemoglobin (HbA1c) was measured at baseline and week-12. Adherence was measured based on pill counting. Results: Thirty patients aged 56.77 ± 14.67 years with 13.37 ± 7.36 years of diabetes participated. Baseline characteristics were similar between the groups. POMs minimized the total cost by 38.96% which translated to a cost saving of USD 42.76 ± 6.98, significantly different versus USD 0.02 ± 0.52 in the control group, p = 0.025. Mean HbA1c reduced significantly (−0.79%, p = 0.016) in the POMs group but not significant in the control group (−0.11%, p = 0.740). Medication adherence improved significantly in both groups at week-12 (p < 0.010). Nevertheless, patients in the POMs group were more adherent, 87.20% vs. 66.32%, p = 0.034. Conclusion: Utilizing POMs resulted in cost saving, improved adherence and better glycaemic control. Use of POMs should be practiced in the outpatient pharmacy to reduce wastage and cost. [ABSTRACT FROM AUTHOR]

Objectives: Adverse drug events (ADE) are a common cause of morbidity and mortality in elderly patients. In this study, we assessed the impact of multidisciplinary medication review (MMR) for nursing home residents on patient safety and costs incurred by the hospital and the national health service. Methods: Medical files of residents were retrospectively assessed for medications prescribed in the previous six months. A pharmacist reviewed the prescriptions and suggested modifications to the patient's medical team. Patients were followed for six months. Trivalle's ADE geriatric risk score was calculated before and after MMR, as were number of potentially inappropriate medications, and economic impact from the perspective of the health care system and the nursing home. Results: Forty-nine patients were recruited. ADE score dropped one risk level (median score of 4 before versus 1 after, p < 0.0001). The number of patients taking at least one potentially inappropriate medication decreased from 30.6% before to 6.1% after MMR (p = 0.005). A mean saving of €232 per patient was made from the nursing home perspective following MMR (p = 0.008). Conclusion: The MMR reduced the iatrogenic drug risk for elderly residents and costs from the nursing home perspective, particularly drug expenditure. [ABSTRACT FROM AUTHOR]

Purpose This study aimed to develop a process to reduce waste through redistribution of short-dated medications in emergency drug boxes and assess associated cost avoidance. Methods Short-dated medication items (defined as those expiring within 4 months) from more than 200 emergency drug boxes located throughout a large academic medical center were collected, sorted, and counted during two 3-month time periods. Medications expiring within 2 weeks were discarded. Remaining short-dated medications were redistributed to satellite pharmacies and the emergency department based on historical utilization patterns. The counts and costs of redistributed medications were collected and totaled. Results were compared between study periods to assess differences in numbers of drugs redistributed and the associated cost avoidance. Results A total of 4,415 short-dated medication items were collected during the study periods. The medication items associated with the highest cost avoidance were epinephrine 1 mg/mL, 30-mL vial (cost avoidance of $25,764), phenylephrine 10 mg/mL, 1-mL vial (cost avoidance of $8,626), and naloxone 0.4 mg/mL, 1-mL vial (cost avoidance of $5,382). The estimated total annualized cost avoidance was $104,357 for the first period and $144,674 for the second period. Of the 16 unique medications stocked in emergency drug boxes, 12 were in short supply at the institution at the time of the project, and about 67% of the redistributed items were subject to national drug shortages. Conclusion A process that facilitates appropriate redistribution of short-dated emergency drug box medications can reduce medication waste and lead to substantial cost avoidance. [ABSTRACT FROM AUTHOR]

Patient access to a drug after US regulatory approval is controlled by complex interactions between governmental and third-party payers, pharmacy benefit managers, distributers, manufacturers, health systems, and pharmacies that together mediate the receipt of goods by patients after prescription by clinicians. Recent medication approvals highlight why and how the distribution of clinically beneficial novel therapies is controlled. Although imposed limitations on availability may be rational considering the fiduciary responsibilities of payers and escalating spending on health care and pharmaceuticals, transparency and communication are lacking, and some utilization management may disproportionately affect vulnerable populations. Analysis of the current health insurance landscape suggests mechanisms by which patient access to appropriate medications can be improved and patient and clinician frustration reduced while acknowledging the financial realities of the pharmaceutical marketplace. We propose creation of a shared, standardized, and transparent process for coverage decisions that minimizes administrative barriers and is defensible on the basis of clinical and cost-effectiveness evidence. These reforms would benefit patients and improve the efficiency of the pharmaceutical system. [ABSTRACT FROM AUTHOR]

Cost-related nonadherence to prescription medicines is a common problem with important implications for population health. Relative to men, women may be more vulnerable to cost-related nonadherence because of higher health needs and lower financial resources. Using data from the Commonwealth Fund International Health Policy Survey, we compared cost-related nonadherence among younger (ages 18-64) and older (ages 65 and older) women and men in eleven high-income countries. Among younger adults, the unadjusted female-male disparity was larger in the US compared with other countries: One in four younger women reported cost-related nonadherence compared with one in seven younger men. This large disparity persisted after adjustment for age, income, and chronic conditions. We also found smaller but significant female-male differences among younger women in Australia and Canada. We did not find significant female-male differences among older adults in adjusted analyses in any country. Higher rates of cost-related nonadherence among younger women, and US women in particular, may produce important sex-related disparities in health outcomes that should be further explored. [ABSTRACT FROM AUTHOR]

Background: Predictions of drug sales play an important role in setting drug prices in Japan, where drug prices are highly regulated. One of 2 primary Japanese drug-pricing methodologies—the cost calculation method— refers extensively to the prediction of drug sales in establishing drug prices. Deviations from predicted sales can induce disproportionate drug prices and unexpected pharmaceutical expenditures. This study aimed to estimate the upward deviation of actual sales from predicted sales and to explore the predictors of such upward deviation. Methods: Estimates of upward deviation in 2015 were produced for new molecular entities (NMEs) approved in 2006-2015. To identify the predictors associated with upward deviation through binary logistic regression analyses, sales within 3 years of launch and in the predicted year in which peak sales would be reached were used. Results: Estimated upward deviation was more than 1220 billion yen in 2015 for the targeted drugs. Drugs priced by the cost calculation method or by obtaining additional indications were significantly more likely to show an upward deviation from predicted peak sales. Conclusions: There is substantial upward deviation between actual and predicted drug sales in Japan. So long as drug sales predictions are used in drug price calculations, a flexible repricing system is needed to buffer unexpected pharmaceutical expenditures. [ABSTRACT FROM AUTHOR]

Background: Generic substitution (GS) was introduced in Finland in 2003 and supplemented with a reference price system (RPS) in 2009. Patients play a vital role in the acceptance of GS and the use of less expensive generic medicines. The objective of this study was to explore Finnish pharmacy customers' experience with allowing and refusing GS. Specific aims were to investigate the reasons for (1) allowing and (2) refusing GS and (3) to determine the prescription medicine-related factors influencing the customer's choice of an interchangeable prescription medicine.Methods: A questionnaire survey was conducted in February 2018. Questionnaires were handed out from 18 community pharmacies across Finland to customers ≥18 years who purchased for themselves a prescription medicine included in the RPS. A descriptive approach was used in the analysis using frequencies, the Chi-square test and Fisher's exact test.Results: The final study material consisted of 1043 questionnaires (response rate 40.0%). Of the customers, 47.9% had both allowed and refused GS, 41.2% had only allowed GS and 6.0% had only refused GS. Customers had allowed GS because they wanted to lower their medicine expenses (75.5%), or because the prescribed medicine (30.8%) or medicine they had used before (27.4%) was unavailable at the pharmacy. The main reasons for refusing GS were an insignificant price difference between interchangeable medicines (63.3%) and satisfaction with the medicine used before (60.2%). The main factors influencing customers' choice of an interchangeable prescription medicine were price (81.1%), familiarity (38.4%) and availability (32.8%). Customers who had allowed GS chose the medicine based on price. Customers who had only refused GS appreciated familiarity more than the price of the medicine.Conclusions: GS is a common practice in Finnish community pharmacies. The price of the medicine was the most important factor affecting customers' decision to allow or refuse GS and the choice of an interchangeable prescription medicine. Thus, customers should receive information about medicine prices at the pharmacy in order to help them make their decision. However, individual needs should also be taken into account in counselling because customers regard several factors as important in their choice of an interchangeable medicine. [ABSTRACT FROM AUTHOR]

Background: Recently, the Executive Branch and Judiciary in Brazil increased spending due to larger numbers of lawsuits that forced the State to provide health goods and services. This phenomenon, known as health judicialization, has created challenges and required the Executive Branch and Judiciary to create institutional strategies such as technical chambers and departments to reduce the social, economic and political distortions caused by this phenomenon. This study aims to evaluate the effects of two institutional strategies deployed by a Brazilian municipality in order to cope with the economic, social and political distortions caused by the phenomenon of health judicialization regarding access to medicines.Methods: A longitudinal study was carried out in a capital in the Central-West Region of Brazil. A sample of 511 lawsuits was analyzed. The variables were placed into three groups: the sociodemographic characteristics and the plaintiffs' disease, the characteristics of the claimed medical products and the institutional strategies. To analyze the effect of the interventions on the total cost of the medicines in the lawsuits, bivariate and multivariate linear regressions with variance were performed. For the categorical outcomes, Poisson regressions were performed with robust variance, using a significance level of 5%.Results: A reduction in the costs of medicines in the lawsuits and of the requests for medicines within the SUS formulary was verified after the deployment of the Department of Assessment of Nonstandardized Medicines (DAMNP) and the Technical Chamber of Health Assessment (CATS); an increase in processed prescriptions from the Brazilian Universal Health System was observed after the deployment of the CATS; and an increase in medicines outside the SUS formulary without a therapeutic alternative was verified after the CATS.Conclusion: The institutional strategies deployed were important tools to reduce the high costs of the medicines in the lawsuits. In addition, they represented a step forward for the State, provided a benefit to society and indicated a potential path for the health and justice systems of other countries that also face problems caused by the judicialization of health. [ABSTRACT FROM AUTHOR]

Introduction: There are positive aspects regarding the prescribing of fixed dose combinations (FDCs) versus prescribing the medicines separately. However, these have to be balanced against concerns including increased costs and their irrationality in some cases. Consequently, there is a need to review their value among lower- and middle-income countries (LMICs) which have the greatest prevalence of both infectious and noninfectious diseases and issues of affordability.Areas covered: Review of potential advantages, disadvantages, cost-effectiveness, and availability of FDCs in high priority disease areas in LMICs and possible initiatives to enhance the prescribing of valued FDCs and limit their use where there are concerns with their value.Expert commentary: FDCs are valued across LMICs. Advantages include potentially improved response rates, reduced adverse reactions, increased adherence rates, and reduced costs. Concerns include increased chances of drug:drug interactions, reduced effectiveness, potential for imprecise diagnoses and higher unjustified prices. Overall certain FDCs including those for malaria, tuberculosis, and hypertension are valued and listed in the country's essential medicine lists, with initiatives needed to enhance their prescribing where currently low prescribing rates. Proposed initiatives include robust clinical and economic data to address the current paucity of pharmacoeconomic data. Irrational FDCs persists in some countries which are being addressed. [ABSTRACT FROM AUTHOR]

High and rising prescription drug costs have become a preoccupying policy problem in the United States. Notwithstanding broad, bipartisan interest in finding effective policy solutions, several aspects of the drug affordability problem make it an uncommonly difficult one to solve. This article reviews the moral, market, and political factors contributing to the difficulty. Among the moral problems is lack of agreement about how to weigh the fundamental tradeoff involved in regulating drug prices—affordability versus incentives for innovation—and about what constitutes a fair price. Market-related factors include the lack of price transparency and a myriad of perverse incentives in the system through which prescription drugs are supplied to patients. Finally, current policy choices are constrained by past political compromises, and an atmosphere of scandal focusing on egregious instances of price gouging has made rational deliberation about fixes to deeper problems in the system difficult. [ABSTRACT FROM AUTHOR]

The article suggests ways for clinicians to help reduce medicines wastage and improve patient health. It discusses the importance of targeting intervention needs of patients to support adherence via communication, decision-making and provision of information resources. Emphasis is also given on the significance of detecting hidden changes in patients to improve care. The option to provide non-medicinal treatment based on patient choice and clinical appropriateness is considered.

Background: Cash prices for prescription drugs vary widely in the United States.Objective: To describe cash price variation by retail pharmacy type for 10 generic and 6 brand-name drugs throughout the United States and stratified by ZIP code.Design: Cross-sectional study.Setting: Drug pricing data from GoodRx, an online tool for comparing drug prices, representing more than 60 000 U.S. pharmacies (fall 2015).Measurements: Cash prices for a 1-month supply of generic and brand-name drugs were ascertained. Stratified by ZIP code, relative cash prices for groups of generic and brand-name drugs were estimated for big box, grocery-based, small chain, and independent pharmacies compared with a reference group of large chain pharmacies.Results: Across 16 325 ZIP codes, 68 353 unique pharmacy stores contributed cash prices. When stratified by 5-digit ZIP code, the relative cash prices for generic drugs at big box, grocery-based, small chain, and independent pharmacies compared with those at large chain pharmacies were 0.52 (95% CI, 0.51 to 0.53), 0.82 (CI, 0.81 to 0.83), 1.51 (CI, 1.45 to 1.56), and 1.61 (CI, 1.58 to 1.64), respectively. The relative cash prices for brand-name drugs were 0.97 (CI, 0.96 to 0.97), 1.00 (CI, 0.99 to 1.00), 1.06 (CI, 1.05 to 1.08), and 1.03 (CI, 1.02 to 1.04), respectively.Limitation: Results may not reflect current drug prices and do not account for point-of-sale discounts or price matching that may be offered by smaller pharmacies.Conclusion: Compared with large chains, independent pharmacies and small chains had the highest cash prices for generic drugs and big box pharmacies the lowest. Relative differences in cash prices for brand-name drugs were modest across types of retail pharmacies.Primary Funding Source: Arnold Ventures. [ABSTRACT FROM AUTHOR]

Background: Dose banding parenteral chemotherapy has the potential to optimise aseptic unit capacity and reduce drug expenditure without compromising the service provided. Methods: Dose banding tables from NHS England were implemented into the electronic chemotherapy prescribing system. Compliance to the dose bands was analysed and submitted quarterly. Analysis of drug expenditure, drug use and cost per milligram data was also collected. Results: Expenditure on the 17 drugs identified in the 2016/17 dose standardisation CQUIN reduced by approximately £100,000 per month over the CQUIN despite an increase in the number of prescribed doses of these drugs. At the beginning of the year, the percentage of work compounded in house was 60%, which was reduced to 51% of total workload at the end of the year due to outsourcing commonly prescribed doses from commercial pharmaceutical aseptic manufacturers. Conclusion: Dose banding parenteral chemotherapy is an efficient cost-saving strategy which also can help to increase the capacity of the aseptic unit. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study was to describe the effect of a therapeutic equivalence program (TEP) in achieving financial sustainability from 2010–11 to 2014–15. Methods: A TEP was introduced at Monash Health in 2006–07. Therapeutic medicine classes for inclusion were selected by stakeholder consensus and a preferred medicine for each class was chosen based upon therapeutic equivalence and cost considerations. New patients were commenced on a preferred medicine, but patients already prescribed another medicine from the same therapeutic class were not automatically switched to the preferred medicine. Data was obtained retrospectively from the pharmacy dispensing system, including the purchasing and issuing of all medicines from the preferred medicine classes. The prescribing patterns for preferred and comparator medicines were used as a measure of acceptance of the TEP, along with the savings produced by the program. Results: Over the 5-year evaluation period, 18 therapeutic classes were targeted, including seven new classes. Six therapeutic classes from the 11 included in the TEP before 2010–11 were removed throughout the evaluation period when the comparative economic benefits were no longer present. The use of all preferred medicines increased following implementation and a total of AU$7.38 million was saved from 2010–11 to 2014–15 and AU$10.54 million across 2006–07 to 2014–15. Conclusions: This paper provides an update on the progress of the TEP at Monash Health and outlines additional learnings gained. The market dynamics for pharmaceuticals means ongoing maintenance and review of the therapeutic medicine classes targeted is important to enable continued economic benefits. What is known about the topic?: There is continued and increasing focus on efficient, cost-effective and financially sustainable medication management. There is limited information available on strategies that can be implemented at a health service level. What does this paper add?: The TEP has resulted in sustained savings. The market dynamics for pharmaceuticals means ongoing maintenance and review of the therapeutic classes targeted is important to enable continued economic benefits. What are the implications for practitioners?: TEP is a process of genuine disinvestment. Identification and resolution of critical factors in the success of the program may assist implementation at other health services. [ABSTRACT FROM AUTHOR]

Objective: To analyze factors associated with changes in prescription drug use and expenditures in the United States from 1999 to 2016, a period of rapid growth, deceleration, and resumed above-average growth.Data Sources/study Setting: The Medical Expenditure Panel Survey (MEPS), containing household and pharmacy information on over five million prescription drug fills.Study Design: We use nonparametric decomposition to analyze drug use, average payment per fill, and per capita expenditure, tracking the contributions over time of socioeconomic characteristics, health status and treated conditions, insurance coverage, and market factors surrounding the patent cycle.Data Collection/extraction Methods: Medical Expenditure Panel Survey data were combined with information on drug approval dates and patent status.Principal Findings: Per capita utilization increased by nearly half during 1999-2016, with changes in health status and treated conditions accounting for four-fifths of the increase. In contrast, per capita expenditures more than doubled, with individual characteristics only explaining one-third of the change. Other drivers of spending during this period include the changing pipeline of new drugs, drugs losing exclusivity, and changes in generic competition.Conclusions: Long-term trends in treated conditions were the fundamental drivers of medication use, whereas factors involving the patent cycle accelerated and decelerated spending growth relative to trends in use. [ABSTRACT FROM AUTHOR]

Background and Objective: Unplanned pregnancies can lead to poorer maternal and child health outcomes. The Australian Therapeutic Goods Administration committee rejected reclassifying a range of oral contraceptive pills (OCPs) from prescription to pharmacist-only medicines in 2015, mainly based on safety concerns. Improving access to OCPs may encourage some women to use contraceptives or switch from other contraceptive methods. However, some adverse events may increase and some women may stop using condoms, increasing their risk of sexually transmitted infections. This study aimed to estimate the cost effectiveness of reclassifying OCPs from prescription to pharmacist-only.Perspective: Healthcare system.Setting: Australian primary care.Methods: A Markov model was used to synthesise data from a variety of sources. The model included all Australian women aged 15-49 years (N = 5,644,701). The time horizon was 35 years. Contraceptive use before reclassification was estimated using data from the Household, Income and Labour Dynamics in Australia (HILDA) survey, while survey data informed use after reclassification. Health outcomes included pregnancies, pregnancy outcomes (live birth, miscarriage, stillbirth, ectopic pregnancy and abortion), sexually transmitted infections, adverse events (venous thromboembolism, depression, myocardial infarction and stroke), ovarian cancer cases and quality-adjusted life-years. Costs included those related to general practitioner and specialist consultations, contraceptives and other medicines, pharmacist time, hospitalisations and adverse events. All costs were reported in 2016 Australian Dollars. A 5% discount rate was applied to health outcomes and costs.Results: Reclassifying OCPs resulted in 85.70 million quality-adjusted life-years experienced and costs of $46,910.14 million over 35 years, vs. 85.68 million quality-adjusted life-years experienced and costs of $50,274.95 million with OCPs remaining prescription-only. Thus, reclassifying OCPs was more effective and cost saving. However, a sensitivity analysis found that more research on the probability of pregnancy in women not using contraception and not trying to conceive is needed.Conclusion: Reclassifying OCPs is likely to be considered cost effective by Australian decision makers. [ABSTRACT FROM AUTHOR]

Background Clinicians prescribe high-cost medicines for rare diseases and nonapproved indications when conventional therapies have failed. Objective To examine the use of non-formulary high-cost medicines at an Australian public hospital. Methods Retrospective audit of individual patient use applications for nonformulary medicines costing more than $5000 AUD per year at a large tertiary referral hospital in Adelaide, South Australia over a 12-month study period from January 2015 to December 2015. Main outcome measures Total cost of non-formulary high-cost medicines, medication class, indications for use, level of supporting evidence and proposed monitoring outcomes. Results Eighty-seven individual patient use applications were examined. All except one were approved, at a total cost of $1,339,203 AUD. The most common drug classes were anti-CD20 (n = 33, 38%), combined antiretrovirals (n = 10, 11%) and TNF-alpha antagonists (n = 10, 11%). There were 56 indications for these medicines with the majority being inflammatory conditions (n = 52, 60%), followed by infections (n = 14, 16%) and malignancies (n = 14, 16%). Of the first-time individual patient use applications (n = 63), there were 25 applications (40%) that provided a case series as supporting evidence. Approximately half of new individual patient use applications (n = 32) proposed an objective monitoring outcome, but few (n = 13, 21%) contained sufficient information to allow a third party to determine efficacy of the medication. Conclusions Non-formulary high-cost medicines are being used for a broad range of indications based largely on low levels of evidence. Prospective definition of an adequate response to treatment and reporting of these outcomes is required to improve the evidence-base and to aid decision-making for subsequent treatment courses. [ABSTRACT FROM AUTHOR]

Background: Estimates of drug spending are often central to the public policy debate on how to manage healthcare spending in the United States. Nevertheless, common estimates of prescription drug spending vary substantially by source, which can inhibit productive policy dialogue.Objectives: To review publicly reported estimates of drug spending and uncover the underlying methodological inputs that drive the substantial variation in estimates of prescription drug spending.Methods: We systematically evaluated 5 estimates of drug spending to identify differences in the underlying methodological inputs and approaches. To uniformly assess and compare estimates, we developed a model to identify the inputs of 3 primary components associated with each estimate: numerator (How is drug cost measured?), denominator (How is healthcare cost measured?), and population (What group of individuals is included in the measurement?). We then applied standardized methodological inputs to each estimate to assess whether variation among estimates could be reconciled. We then conducted a sensitivity analysis to address important limitations.Results: We found that the 18.8 percentage point range in the publicly reported estimates is predominately attributed to methodological differences. Reconciling estimates using a standardized methodological approach reduces this range to 4.0 percentage points.Conclusions: Because variation in estimates of drug spending is primarily driven by methodological differences, stakeholders should seek to establish a mutually agreed upon methodological approach that is appropriate for the policy question at hand to provide a sound basis for health spending policy discussions. [ABSTRACT FROM AUTHOR]

Introduction: Poor prescription drug adherence is common, jeopardizing the benefits of treatment and increasing the costs of health care in the United States. A frequently reported barrier to adherence is patient out-of-pocket (OOP) costs. Areas Covered: This systematic review examines interventions that address patient cost-sharing to improve adherence to prescription drugs and reduce costs of care. Twenty-eight published studies were identified with 22 distinct interventions. Most papers were published in or after 2010, and nearly a third were published after 2014. Expert Opinion: Many of the interventions were associated with improved adherence compared to controls, but effects were modest and varied across drug classes. In some studies, adherence remained stable in the intervention group, but declined in the control group. Patient OOP costs generally declined following the intervention, usually as a direct result of the financial structure of the intervention, such as elimination of copayments, and costs to health plans for prescription drugs increased accordingly. For those studies that reported drug and nondrug costs, lower health plan nondrug medical spending generally compensated for increased spending on prescription drugs. With increasing health-care spending, especially for prescription drugs, efforts to improve prescription drug adherence in the United States are important. Federal policies regarding prescription drug prices may have an impact on cost-related nonadherence, but the content and timing of any policies are hard to predict. As such, employers and health plans will face greater pressure to explore innovative approaches to lowering costs and increasing access for beneficiaries. Value-based financial incentive models have the potential to be a part of this effort; research should continue to evaluate their effectiveness. [ABSTRACT FROM AUTHOR]

Many countries use external reference pricing to help determine drug prices. However, external reference pricing has received little attention in the US--perhaps because the US is often the first adopter of drugs. External reference pricing could be used to set prices for drugs that were already established in the market. We compared the price differentials between the US and the UK, Japan, and Ontario (Canada) for single-source brand-name drugs that had been on the market for at least three years. We found that the prices averaged 3.2-4.1 times higher in the US after rebates were considered. The price differential for individual drugs varied from 1.3 to 70.1. The longer a drug remained on the market, the greater the differential. The estimated savings to Medicare Part D of adopting the average price of drugs in the reference countries was $72.9 billion in 2018. Medicare could use external reference pricing in Part D to improve affordability for patients. [ABSTRACT FROM AUTHOR]

A single screening question and an arsenal of cost-reducing strategies can help you help your patients afford their medications. [ABSTRACT FROM AUTHOR]

Universal Health Coverage is key to reach the overall health-related Sustainable Development Goal, and within this, access to safe, effective, quality, and affordable essential medicines is critical. Currently, medicines for noncommunicable diseases in many countries are not available when needed and if they are present, are unaffordable. Countries face the challenges of rising prevalence of noncommunicable diseases due to increasing risk factors and ageing populations, along with under-diagnosis and under-treatment. Providing noncommunicable disease medicines is only one piece of a complex picture of providing care within Universal Health Coverage that requires strengthening health-care systems, as well as financial resources, priority setting, and monitoring and evaluation systems. Financing for Universal Health Coverage needs to enable adequate resources to be allocated for medicines with a focus on equity as well as priority setting for noncommunicable diseases medicines for reimbursement in benefits packages, efficient procurement and distribution of these medicines, supported by price regulation. These processes need to be evidence-based, transparent and grounded on national values and priorities. Monitoring and evaluation of availability and affordability are key components of sustainable reimbursement systems. With the current Universal Health Coverage agenda, the World Health Organization and countries can no longer ignore the issue of access to medicines for noncommunicable disease and need to develop the appropriate responses in order to guarantee equitable access. [ABSTRACT FROM AUTHOR]

Spinal muscular atrophy (SMA) is a recessive disorder caused by a mutation in the survival motor neuron 1 gene (SMN1); it affects 1 in 11 000 newborn infants. The most severe and most common form, type 1 SMA, is associated with early mortality in most cases and severe disability in survivors. Nusinersen, an antisense oligonucleotide, promotes production of full-length protein from the pseudogene SMN2. Nusinersen treatment prolongs survival of patients with type 1 SMA and allows motor milestone acquisition. Patients with type 2 SMA also show progress on different motor scales after nusinersen treatment. Nusinersen was recently approved by the European Medicines Agency and the US Food and Drug Administration; it is now reimbursed in several European countries and in the USA. In Australia, the transition from expanded access programme to commercial availability is coming soon. In New Zealand, an expanded access programme is opened, and in Canada price negotiation for the treatment is in progress. In this review we exemplify the clinical benefit of nusinersen in subgroups of patients with SMA. Nusinersen represents the first efficacious marked approved drug in type 1 and type 2 SMA. Different knowledge gaps, such as results in older patients, in patients with permanent ventilation, in patients with neonatal forms, or in patients after spinal fusion, still need to be addressed. WHAT THIS PAPER ADDS: Identifies gaps in knowledge about the efficacy of nusinersen in broader populations of patients with spinal muscular atrophy. Identifies open questions in populations of patients where proof of efficacy is available. [ABSTRACT FROM AUTHOR]

Abstract Objective We investigated the South African tendering system for medicines to (a) evaluate its impact on prices and market concentration over a 14-year period and (b) analyze the accuracy of government forecasts of drug demand. Methods We calculated Herfindahl-Hirschman indexes to measure market concentration levels based on all pharmaceutical tender contracts issued by the South African government between 2003 and 2016 (n = 8701). We estimated price indexes to track changes in medicine costs over this period. We compared prices set through tenders in the public health care system to the corresponding prices in the private system. We also analyzed government data on procurement in selected drug classes to assess the accuracy of demand forecasts. Findings Between 2003 and 2016, the prices of medicines in most tender categories in the public health care system dropped by an average of around 40% or more. The prices of medicines procured for the public system through tenders were almost always lower than those sold in the private system. Tenders generally remained moderately to highly competitive over time (i.e., Herfindahl-Hirschman indexes < 2500), although the number of different firms winning contracts decreased in many categories. There were large discrepancies between the drug need estimates by the government and the quantities it went on to procure, with estimates off by more than 50% in most drug classes (9/16 observations). Conclusion Tendering may be an effective measure to lower drug costs. Because most tenders remained competitive over time, price decreases may be durable. South African government officials should monitor the availability and prices of medicines to ensure continued access to affordable medicines for patients, as it may be undermined by the decreasing number of firms winning contracts over time. Given the large discrepancy between forecasts and procurements, the government would benefit from improving the accuracy of its demand forecasts. Highlights • Tendering was linked to large price cuts for many medicines in South Africa. • It did not appear to have an adverse effect on market concentration. • Yet there has been a drop in the number of firms winning contracts over time. • The South African government should improve its forecasts of drug demand. [ABSTRACT FROM AUTHOR]

Objectives: To develop and test a method for approximating generic entry of top-selling drugs.Methods: The procedure involved 1) identifying products' key patents as those with a patent term restoration extension (whenever relevant) or otherwise as the first expiring patent listed in the US Food and Drug Administration's patent register, 2) determining whether the key patent had been extended through an associated pediatric extension, 3) identifying other regulatory exclusivities associated with the drug, and 4) categorizing key patents as active ingredient (or extended) patents versus secondary patents. The accuracy and precision of the procedure's predictions were then tested against a database containing the timing of generic entry for 170 top-selling drugs that lost market exclusivity between 2000 and 2012, on the basis of Medicaid data.Results: Overall, the procedure predicted a median market exclusivity period of 12.5 years (interquartile range [IQR] 7.25-14.5) compared with the median actual period of 12.5 years (IQR 8.5-14.75 years). Among the 131 drugs (77%) with active ingredient patents, the median predicted market exclusivity was 12.25 years (IQR 7.5-14.5) compared with a median actual period of 13.0 years (IQR 10.0-14.75). Among the 38 (22%) drugs protected only by secondary patents, the median predicted market exclusivity was 16.0 years (IQR 6.75-19.5), but the median actual market exclusivity was only 8.25 years (IQR 6.25-13.5).Conclusions: The procedure approximated median actual exclusivity with reasonable accuracy and precision for drugs with active ingredient patents, but substantially overestimated exclusivity for drugs with only secondary patents. [ABSTRACT FROM AUTHOR]

Introduction: Multiple Criteria Decision Analysis (MCDA) is increasingly used in health care mainly because it moves decision-making from ad hoc to an evidence-based and comprehensive process. Developing countries with more restricted financial and human research capacities, however, should consider their own methods of MCDA development and implementation. Areas covered: An MCDA framework to improve procurement decisions of off-patent pharmaceuticals was developed for developing countries and adapted to Indonesia, Kazakhstan and Vietnam during three policy workshops. Based on the experience of these workshops and one joint workshop with international experts and decision makers from multiple developing countries, general recommendations were formulated on how to implement MCDA specifically in developing countries. We provide 17 practical MCDA implementation recommendations in four major areas, including (1) MCDA objectives; (2) technical considerations of MCDA tool; (3) development and customization of MCDA tool and (4) policy implementation of MCDA in decision-making. Expert commentary: These practical MCDA recommendations for developing countries contribute to feasible, transparent, stepwise, iterative and standardized decision-making in health care. [ABSTRACT FROM AUTHOR]

The increasing price of prescription drugs is an ongoing concern for Medicare and Medicaid, particularly for patients with chronic health conditions who are using multiple medications and patients in these programs taking high-priced brand-name specialty drugs. Shifts in benefit design, including higher deductibles and a movement away from copayments to coinsurance, have increased patient out-of-pocket costs and put pressure on program budgets. In this paper, the American College of Physicians expands on its position paper from 2016 and offers additional recommendations to decrease out-of-pocket costs for patients, enhance the government's purchasing power, and address existing policies that add costs to the health care system. [ABSTRACT FROM AUTHOR]