Pharmaceutical drug development: high drug prices and the hidden role of public funding.

In 2019, the record for the most expensive drug was broken at US$2.1 million per patient. The high costs of new drugs are justified by the pharmaceutical industry as the expense required for maintaining research and development (R&D) pipelines. However, this does not take into account that globally the public pays for between one to two-thirds of upfront R&D costs through taxpayers or charitable donations. Governments are effectively paying twice for medicines; first through R&D, and then paying the high prices upon approval. High drug prices distort research priorities, emphasising financial gains and not health gains. In this manuscript, issues surrounding the current patent-based drug development model, public funding of research and pharmaceutical lobbying will be addressed. Finally, innovations in drug development to improve public health needs and guaranteeing medication access to patients will be explored.

A comparison between the effects of drug costs and share of family income on drug costs in determining drug price.

ABSTRACT: High health care and medication expenditures pose a financial burden on Americans seeking care. It is imperative to determine the role of affordability in influencing access to health care and medications.To investigate the association between financial burden and health care access by comparing the effects of absolute and relative financial burdens, measured by total health care/medication expenditure (Expenditure) and health care/medication expenditure as a share of annual family income (Expenditure Share), respectively.Delay in receiving health care services and delay in obtaining prescription medications.A cross-sectional analysis of the 2017 Medical Expenditure Panel Survey using multivariate logistic regressions with Expenditure and Expenditure Share variables standardized to facilitate comparison.While both absolute and relative financial burdens were found to be positively associated with the outcomes, the relative measure had a significantly higher association that was about twice as much as the absolute one. For the outcome of delay in getting health care, the standardized odds ratios (OR) for health care expenditure and health care expenditure as a share of family income were 1.13 (95% confidence interval [CI] = 1.09-1.18) and 1.25 (95% CI = 1.20-1.32), respectively. For the outcome of delay in getting medications, the standardized OR for medication expenditure and medication expenditure as a share of family income were 1.11 (95% CI = 1.08-1.15) and 1.23 (95% CI = 1.18-1.29), respectively.The study illustrated the importance of including income in policy considerations intended to balance value, access, and affordability. Specifically, income should be included in measures assessing the value of medications.

Availability of Cost-effectiveness Studies for Drugs With High Medicare Part D Expenditures.

Importance: Prescription drug spending in the US requires policy intervention to control costs and improve the value obtained from pharmaceutical spending. One such intervention is to apply cost-effectiveness evidence to decisions regarding drug coverage and pricing, but this intervention depends on the existence of such evidence to guide decisions. Objective: To characterize the availability and quality of cost-effectiveness studies for prescription drugs with the greatest Medicare Part D spending. Design, Setting, and Participants: In this national cross-sectional analysis, publicly available 2016 Medicare drug spending records were merged with 2016 US Food & Drug Administration Orange Book data and the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry. All studies published through 2015 that evaluated the cost-effectiveness of the 250 drugs for which Medicare Part D spending was the greatest in US-based adult patient populations were included. Data were analyzed from September 2018 to June 2020. Main Outcomes and Measures: The presence and quality of published cost-effectiveness analyses for the 250 drugs for which Medicare Part D spending was greatest in 2016 were assessed based on the inclusion of key cost-effectiveness analysis elements and global ratings by independent reviewers for the Tufts CEA Registry. Results: Medicare Part D spending on the 250 drugs in the sample totaled $122.8 billion in 2016 (84.1% of total spending). Of these 250 drugs, 91 (36.4%) had a generic equivalent and 159 (63.6%) retained some patent exclusivity. There were 280 unique cost-effectiveness analyses for these drugs, representing data on 135 (54.0%) of the 250 drugs included and 67.0% of Part D spending on the top 250 drugs. The 115 drugs (46.0%) without cost-effectiveness studies accounted for 33.0% of Part D spending on the top 250 drugs. Of the 280 available studies, 128 (45.7%) were industry sponsored. A large proportion of the studies (250 [89.3%]) did not meet the minimum quality requirements. Conclusions and Relevance: In this cross-sectional study, a substantial proportion of 2016 Medicare Part D spending was for drugs with absent or low-quality cost-effectiveness analyses. The lack of quality analyses may present a challenge in efforts to develop policies addressing drug spending in terms of value.

Evaluating essential medicines for treating childhood cancers: availability, price and affordability study in Ghana.

INTRODUCTION: Access to childhood cancer medicines is a critical global health challenge. There is a lack of sufficient context-specific data in Ghana on access to essential medicines for treating childhood cancers. Here, we present an analysis of essential cancer medicine availability, pricing, and affordability using the pediatric oncology unit of a tertiary hospital as the reference point. METHOD: Data on prices and availability of 20 strength-specific essential cancer medicines and eight non-cancer medicines were evaluated using the modified World Health Organization (WHO)/Health Action International method. Two pharmacies in the hospital and four private pharmacies around the hospital were surveyed. We assessed their median price ratio using the WHO international reference price guide. The number of days wages per the government daily wage salary was used to calculate the affordability of medicines. RESULTS: The mean availability of essential cancer medicines and non-cancer medicines at the hospital pharmacies were 27 and 38% respectively, and 75 and 84% respectively for private pharmacies. The median price ratio of cancer medicines was 1.85, and non-cancer medicines was 3.75. The estimated cost of medicines for treating a 30 kg child with Acute lymphoblastic leukaemia was GHÈ» 4928.04 (US$907.56) and GHÈ» 4878.00 (US$902.62) for Retinoblastoma, requiring 417 and 413-days wages respectively for the lowest-paid unskilled worker in Ghana. CONCLUSION: The mean availability of cancer medicines at the public and private pharmacies were less than the WHO target of 80%. The median price ratio for cancer and non-cancer medicines was less than 4, yet the cost of medicines appears unaffordable in the local setting. A review of policies and the establishment of price control could improve availability and reduce medicines prices for the low-income population.

THE MOST FAVORED NATIONS MODEL INTERIM FINAL RULE IS FATALLY FLAWED AND MUST BE RESCINDED-YET THE PROBLEM IT ADDRESSES DEMANDS A SOLUTION.

DISCLOSURES: No funding supported the writing of this letter. The author has nothing to disclose.

Análisis del cumplimiento del plan de consumo de los medicamentos de alto costo. Matanzas. 2012-2017 / Analysis of the fulfillment of the consumption plan of high cost medications. Matanzas. 2012 - 2017

RESUMEN Introducción: los medicamentos de alto costo son medicamentos nuevos, altamente específicos y utilizados en condiciones clínicas complejas, como el tratamiento de algunos tipos de cáncer; enfermedades que comprometen el sistema inmunológico, enfermedades inflamatorias o infecciosas. Objetivo: determinar cumplimiento del plan de consumo de los medicamentos de alto costo en la provincia de Matanzas, en el quinquenio 2012-2017. Materiales y métodos: estudio descriptivo, observacional de seguimiento sobre el cumplimiento del plan de consumo de los medicamentos de alto costo, en la población matancera del año 2012 al 2017. Se analizó el universo de medicamentos incluidos en esta categoría, a partir de la base de datos de suministro de medicamentos que emplea la Empresa Comercializadora de Medicamentos. Se identificaron las variables estudiadas. Resultados: en todo el período de estudio se observó un incremento creciente de los medicamentos de alto costo, en el 88,9 % de ellos el consumo ha estado por encima de la planificación realizada por la provincia. Los costos se incrementaron en un 233 % y además en las prescripciones realizadas de estos productos, se encontraron problemas como escaques vacíos, antibióticos sin impresión diagnóstica y omisión de la forma de presentación del medicamento y/o dosis indicada. Conclusiones: el consumo de muchos medicamentos de alto costo fue mayor que la planificación realizada en la provincia de Matanzas, durante el período analizado. Implicó un incremento significativo del presupuesto destinado a estos fines y se detectaron dificultades en el cumplimiento de lo establecido en las prescripciones de dichos medicamentos (AU).

ABSTRACT Introduction: high cost medications (HCM) are new highly specific medications and used in complex clinical conditions as in treatment of some types of cancer, diseases that compromise the immunological system, inflammatory or infections disorders. Objective: to determine the fulfillment of the consumption plan of high-cost medications in the province of Matanzas in the period 2012-2017. Materials and methods: a descriptive, observational, follow up study on the fulfillment of the consumption plan of high-cost medication by the population of Matanzas2012 year to 2017. The universe of drugs included in this category was analyzed on the bases of the drug-supplying database used by the Drug Commercializing Enterprise (ENCOMED in Spanish). The studied variables were identified. Results: it was observed a growing increase of high-cost drugs use during all the period; in 88.9 % of them the consumption has been above the planning made in the province. The costs increased in 233 %, and besides that in the prescriptions made of these drugs there were found problems like empty boxes, antibiotics without diagnostic impression and omissions of the drug presentation forms and/or the prescribed doses. Conclusions: the consumption of many high-cost drugs was higher than the planning made in the province of Matanzas for the analyzed period. It implied a significant increase of the budget destined for these aims and difficulties were found in the fulfillment of the terms for prescribing these drugs (AU).

Treatment With Liposomal Amphotericin B for All Confirmed Cases of Human Visceral Leishmaniasis in Brazil: A Budget Impact Analysis.

OBJECTIVES: To estimate the budget impact of the expansion of liposomal amphotericin B use for all confirmed cases of visceral leishmaniasis (VL) in Brazil. Currently, the first-line medicine for VL treatment is meglumine antimoniate. Liposomal amphotericin B is indicated only for patients with a greater risk of severity by the disease. METHODS: The analysis was performed from the perspective of the Brazilian public healthcare system over 3 years, considering the following 2 scenarios: the reference scenario with the current recommendations for VL treatment and the alternative scenario based on the use of liposomal amphotericin B for all patients. A diffusion rate of 60% was used in the first year, 80% in the second year, and 100% in the third year. The epidemiological parameters used in the analysis came from the Notifiable Diseases Information System and from a clinical trial that evaluated the efficacy and safety of medicines for the treatment of VL in the country. The costs were related to the treatment of VL and to hospital and outpatient care. RESULTS: In the reference scenario, the total cost for treatment of the 3453 VL confirmed cases in 2014 was $1 447 611.75. The incremental budget impact with the use of liposomal amphotericin B for all the VL confirmed cases was $299 646.43 in the third year. CONCLUSIONS: The analysis presented will support the decision process for the use and expansion of liposomal amphotericin B for all VL confirmed cases in Brazil.