Citizens' views on prices of medicines reimbursed by the National Health Service: Findings from Italian online focus groups.

INTRODUCTION: Access to medicines is one of the biggest challenges to health systems, affecting society and individuals. This study aims to explore citizens' opinions, perceptions and attitudes on the model of medicines' research and development (R&D) and price setting of medicines reimbursed by the Italian National Health Service. MATERIALS AND METHODS: We run four online focus groups, analysed through thematic analysis. INCLUSION CRITERIA: people aged 30-70 years, who had completed at least compulsory schooling (8-10 years), with no specialised knowledge about the subject. EXCLUSION CRITERIA: healthcare workers, pharmaceutical and device industry employees, researchers and medicine policy board members. We aimed to include a purposive sample of 20 participants, variable in terms of age, educational level and place of residence. RESULTS: Eleven women and six men participated. The mean age was 53 years (range: 28-73). Most (n = 15) had a university degree or attended secondary schools. Eight had a job, five were not employed, and four were retired. In general, participants supported the role of the public health service. Almost all had limited knowledge of medicines' R&D and price setting. Most asked for transparency on medicine prices and negotiation criteria. Participants considered revenues of pharmaceutical companies disproportionate and most called for containment measures of profits. Most were in favour of a stronger public intervention in R&D and prices' negotiations. Few were sceptical of the public sector's ability to play this role. DISCUSSION: Medicines' prices were discussed as a health matter. Increasing citizens' awareness of these topics is needed by providing spaces and conditions to participate in the discussion, including different perspectives and interests. PATIENT OR PUBLIC CONTRIBUTION: Members of BEUC-the European consumer organisation-proposed the project. Altroconsumo, an independent consumer organisation and OCU, a Spanish consumer organisation, participated in developing the project and the main topics to discuss. The Mario Negri Institute and Aplica cooperative-the Spanish methodological team-were involved by BEUC and their national organisations to define the methodology, organisational aspects and contents and conducted the focus groups.

Why are our medicines so expensive? Spoiler: Not for the reasons you are being told .

Often described as a natural economic trend, the prices that pharmaceutical companies charge for new medicines have skyrocketed in recent years. Companies claim these prices are justified because of the 'value' new treatments represent or that they reflect the high costs and risks associated with the research and development process. They also claim that the revenues generated through these high prices are required to pay for continued innovation.This paper argues that high prices are not inevitable but the result of a societal and political choice to rely on a for-profit business model for medical innovation, selling medicines at the highest price possible. Instead of focusing on therapeutic advances, it prioritises profit maximisation to benefit shareholders and investors over improving people's health outcomes or equitable access.As a result, people and health systems worldwide struggle to pay for the increasingly expensive health products, with growing inequities in access to even life-saving medicines while the biopharmaceutical industry and its financiers are the most lucrative business sectors.As the extreme COVID-19 vaccine inequities once again highlighted, we urgently need to reform the social contract between governments, the biopharmaceutical industry, and the public and restore its original health purpose. Policymakers must redesign policies and financing of the pharmaceutical research and development ecosystem such that public and private sectors work together towards the shared objective of responding to public health and patients' needs, rather than maximising financial return because medicines should not be a luxury.

Hospital Prices for Physician-Administered Drugs for Patients with Private Insurance.

BACKGROUND: Hospitals can leverage their position between the ultimate buyers and sellers of drugs to retain a substantial share of insurer pharmaceutical expenditures. METHODS: In this study, we used 2020-2021 national Blue Cross Blue Shield claims data regarding patients in the United States who had drug-infusion visits for oncologic conditions, inflammatory conditions, or blood-cell deficiency disorders. Markups of the reimbursement prices were measured in terms of amounts paid by Blue Cross Blue Shield plans to hospitals and physician practices relative to the amounts paid by these providers to drug manufacturers. Acquisition-price reductions in hospital payments to drug manufacturers were measured in terms of discounts under the federal 340B Drug Pricing Program. We estimated the percentage of Blue Cross Blue Shield drug spending that was received by drug manufacturers and the percentage retained by provider organizations. RESULTS: The study included 404,443 patients in the United States who had 4,727,189 drug-infusion visits. The median price markup (defined as the ratio of the reimbursement price to the acquisition price) for hospitals eligible for 340B discounts was 3.08 (interquartile range, 1.87 to 6.38). After adjustment for drug, patient, and geographic factors, price markups at hospitals eligible for 340B discounts were 6.59 times (95% confidence interval [CI], 6.02 to 7.16) as high as those in independent physician practices, and price markups at noneligible hospitals were 4.34 times (95% CI, 3.77 to 4.90) as high as those in physician practices. Hospitals eligible for 340B discounts retained 64.3% of insurer drug expenditures, whereas hospitals not eligible for 340B discounts retained 44.8% and independent physician practices retained 19.1%. CONCLUSIONS: This study showed that hospitals imposed large price markups and retained a substantial share of total insurer spending on physician-administered drugs for patients with private insurance. The effects were especially large for hospitals eligible for discounts under the federal 340B Drug Pricing Program on acquisition costs paid to manufacturers. (Funded by Arnold Ventures and the National Institute for Health Care Management.).

Assessment of the Global Fund-supported procurement and supply chain reforms at the Ethiopian Pharmaceuticals Supply Agency: a mixed-methods study.

OBJECTIVE: The Global Fund to Fight AIDS, Tuberculosis and Malaria (Global Fund) partnered with the Ethiopian Pharmaceutical Supply Agency (EPSA) in 2018-2019 to reform procurement and supply chain management (PSCM) procedures within the Ethiopian healthcare system. This assessment sought to determine the impact of the reforms and document the lessons learnt. DESIGN: Mixed-methods study incorporating qualitative and quantitative analysis. Purposive and snowballing sampling techniques were applied for the qualitative methods, and the data collected was transcribed in full and subjected to thematic content analysis. Descriptive analysis was applied to quantitative data. SETTING: The study was based in Ethiopia and focused on the EPSA operations nationally between 2017 and 2021. PARTICIPANTS: Twenty-five Ethiopian healthcare decision-makers and health workers. INTERVENTION: Global Fund training programme for health workers and infrastructural improvements OUTCOMES: Operational and financial measures for healthcare PSCM. RESULTS: The availability of antiretrovirals, tuberculosis and malaria medicines, and other related commodities, remained consistently high. Line fill rate and forecast accuracy were average. Between 2018 and 2021, procurement lead times for HIV and malaria-related orders reduced by 43.0% relative to other commodities that reported an increase. Many interview respondents recognised the important role of the Global Fund support in improving the performance of EPSA and provided specific attributions to the observed successes. However, they were also clear that more needs to be done in specific critical areas such as financing, strategic reorganisation, data and information management systems. CONCLUSION: The Global Fund-supported initiatives led to improvements in the EPSA performance, despite several persistent challenges. To sustain and secure the gains achieved so far through Global Fund support and make progress, it is important that various stakeholders, including the government and the donor community, work together to support EPSA in delivering on its core mandate within the Ethiopian health system.

Impact of China's National Centralized Drug Procurement Policy on pharmaceutical enterprises' financial performance: a quasi-natural experimental study.

Introduction: In China, the interest relationship between pharmaceutical enterprises and medical institutions has harmed the healthy development of pharmaceutical enterprises. In November 2018, the National Centralized Drug Procurement (NCDP) policy was published. The NCDP policy severs the interest relationship and significantly impacts on pharmaceutical enterprises's financial performance. Methods: Using the implementation of China's National Centralized Drug Procurement (NCDP) policy as a quasi-natural experiment, this study evaluated the impact of participation in the NCDP policy on pharmaceutical enterprises' financial performance. We developed a difference-in-difference model to estimate the change in financial performance after NCDP implementation, based on financial data on Chinese listed pharmaceutical enterprises. Results: We found that the bid-winning enterprises' financial performance significantly improved after participating in NCDP. This may be related to lower costs, market share expansion, and increased research and development investment by the bid-winning enterprises. Discussion: To further promote the high-quality development of pharmaceutical enterprises in China, the government should expand the variety of drugs on the NCDP list (NCDP drugs), while improving the drug patent protection system and the policies to support the bid-winning enterprises.

The Problem of Limited-Supply Agreements for Medicare Price Negotiation.

This Viewpoint discusses how limited-supply agreements (introduction of generic products in reduced volumes) might thwart efforts to negotiate lower prices on prescription drugs in the US.

Patents and Regulatory Exclusivities on GLP-1 Receptor Agonists.

Importance: Glucagon-like peptide 1 (GLP-1) receptor agonists were first approved for the treatment of type 2 diabetes in 2005. Demand for these drugs has increased rapidly in recent years, as indications have expanded, but they remain expensive. Objective: To analyze how manufacturers of brand-name GLP-1 receptor agonists have used the patent and regulatory systems to extend periods of market exclusivity. Evidence Review: The annual US Food and Drug Administration's (FDA) Approved Drug Products With Therapeutic Equivalence Evaluations was used to identify GLP-1 receptor agonists approved from 2005 to 2021 and to record patents and nonpatent statutory exclusivities listed for each product. Google Patents was used to extract additional data on patents, including whether each was obtained on the delivery device or another aspect of the product. The primary outcome was the duration of expected protection from generic competition, defined as the time elapsed from FDA approval until expiration of the last-to-expire patent or regulatory exclusivity. Findings: On the 10 GLP-1 receptor agonists included in the cohort, drug manufacturers listed with the FDA a median of 19.5 patents (IQR, 9.0-25.8) per product, including a median of 17 patents (IQR, 8.3-22.8) filed before FDA approval and 1.5 (IQR, 0-2.8) filed after FDA approval. Fifty-four percent of all patents listed on GLP-1 receptor agonists were on the delivery devices rather than active ingredients. Manufacturers augmented patent protection with a median of 2 regulatory exclusivities (IQR, 0-3) obtained at approval and 1 (IQR, 0.3-4.3) added after approval. The median total duration of expected protection after FDA approval, when accounting for both preapproval and postapproval patents and regulatory exclusivities, was 18.3 years (IQR, 16.0-19.4). No generic firm has successfully challenged patents on GLP-1 receptor agonists to gain FDA approval. Conclusions and Relevance: Patent and regulatory reform is needed to ensure timely generic entry of GLP-1 receptor agonists to the market.

Commentary: Reconsidering Pharmaceutical Research and Development Investments.

Following Lee and colleagues' (2023) article explaining how Canadians are being shortchanged by drug companies when it comes to investments in research and development (R&D), this rejoinder adds context and appends two other very problematic elements in the debate between wishful narratives over the industry's contribution in R&D and actual numbers. First, even the current stricter definition of R&D investment might simply be too large considering that elements such as seeding trials - a well-known marketing device - can be accounted for as R&D expenditures. Second, this rejoinder identifies how Statistics Canada acted in concert with Innovative Medicines Canada to reinforce the industry's preferred narratives around R&D expenditures. This situation puts into question the trustworthiness of Canada's statistical agency.

Política de medicamentos de alto costo en un hospital público pediátrico: Análisis de utilización y costos / High-cost medication policy at a public pediatric hospital: utilization and cost analysis

Objetivos. Analizar el circuito de utilización de los medicamentos de alto costo (MAC) y los resultados clínicos obtenidos en un hospital de pediatría público de alta complejidad de Argentina y presentar una estrategia de selección replicable para otras instituciones de similares características de la región. Métodos: Estudio prospectivo, descriptivo, aleatorizado, conducido en el Hospital de Pediatría Juan P. Garrahan de la Ciudad Autónoma de Buenos Aires en el período entre el 1 de setiembre de 2018 y el 31 de marzo de 2019. Se evaluaron dos unidades de estudio, la unidad paciente y la unidad MAC. Resultados: Los MAC consumen 7.921.200 dólares estadounidenses (USD) anuales y representan el 41% del costo de los medicamentos del hospital de alta complejidad. El 50% del costo de los MAC estuvo representado por la gammaglobulina (medicamento utilizado en diferentes enfermedades). Los pacientes proceden de toda la Argentina y otros países y un 44% tiene cobertura de salud. Los diagnósticos para los que se prescribieron MAC con mayor frecuencia fueron los relacionados con patología oncológica (leucemia linfoide aguda, leucemia mieloblástica aguda). El 54% de los pacientes presentó mejoría atribuible directamente a la administración de los MAC, 39% no presentó cambios y el 7% empeoró. Conclusiones: La efectividad en los resultados clínicos y el análisis de los circuitos de aprobación indican que, además de la aprobación por las entidades nacional e internacionales, la evaluación responsable por parte de las instituciones efectoras, mediante la discusión interdisciplinaria basada en la mejor evidencia, contribuye a optimizar la utilización de los MAC y la seguridad de los pacientes (AU)

Objectives. To analyze the utilization circuit of high-cost medications (HCM) and the clinical results obtained in a tertiarycare public pediatric hospital in Argentina and to present a selection strategy that may be disseminated to other institutions of similar characteristics in the region. Methods: A prospective, descriptive, randomized study was conducted at Hospital de Pediatría Juan P. Garrahan in Buenos Aires between September 1, 2018 and March 31, 2019. Two study units were evaluated, the patient and the HCM. Results: HCMs account for 7,921,200 US dollars (USD) per year and represent 41% of the cost of drugs in this tertiary-care hospital. Gamma globulin (a drug used for different diseases) accounted for 50% of the cost of HCMs. Patients came from Argentina and other countries and 44% had a health insurance. Cancer (acute lymphoid leukemia, acute myeloblastic leukemia) was the diagnosis for which HCMs were most frequently prescribed. Fifty-four percent of patients showed improvement directly attributable to the administration of HCMs, 39% showed no change, and 7% worsened. Conclusions: The effectiveness in clinical outcomes and the analysis of approval circuits show that, in addition to approval by national and international entities, responsible evaluation by the effector institutions through interdisciplinary discussion based on the best evidence contributes to optimizing the use of HCMs and patient safety (AU)

Association Between Drug Characteristics and Manufacturer Spending on Direct-to-Consumer Advertising.

Importance: Some drugs are heavily marketed through direct-to-consumer advertising. Objective: To identify drug characteristics associated with a greater share of promotional spending on advertising directly to consumers. Design, Setting, and Participants: Exploratory cross-sectional analysis of drug characteristics and promotional spending for the 150 top-selling branded prescription drugs in the US in 2020 as identified from IQVIA National Sales Perspectives data. Promotional spending data were provided by IQVIA ChannelDynamics. Exposures: Drug characteristics (total 2020 sales; total 2020 promotional spending; clinical benefit ratings; number of indications, off-label use; molecule type; nature of condition treated; administration type; generic availability; US Food and Drug Administration [FDA] approval year, World Health Organization anatomical therapeutic chemical classification; Medicare annual mean spending per beneficiary; percent sales attributable to the drug; market size; market competitiveness) assessed from health technology assessment agencies (France's Haute Autorité de Santé and Canada's Patented Medicine Prices Review Board) and drug data sources (Drugs@FDA, the FDA Purple Book, Lexicomp, Merative Marketscan Research Databases, and Medicare Spending by Drug data). Main Outcomes and Measures: Proportion of total promotional spending allocated to direct-to-consumer-advertising for each drug. Results: The 2020 median proportion of promotional spending allocated to direct-to-consumer advertising was 13.5% (IQR, 1.96%-36.6%); median promotional spending, $20.9 million (IQR, $2.72-$131 million); and median total sales, $1.51 billion (IQR, $0.97-$2.26 billion). Of the 150 best-selling drugs, 16 were missing data and key covariates; therefore, the primary study sample comprised 134 drugs. After adjustment for multiple drug characteristics, the mean proportion of total promotional spending allocated to direct-to-consumer advertising for the remaining 134 drugs was an absolute 14.3% (95% CI, 1.43%-27.2%; P = .03) higher for those with low added clinical benefit than for those with high added clinical benefit and an absolute 1.5% (95% CI, 0.44%-2.56%; P = .005) higher for each 10% increase in total sales. Conclusions and Relevance: Among top-selling US drugs in 2020, a rating of lower added benefit and higher total drug sales were associated with a higher proportion of manufacturer total promotional spending allocated to direct-to-consumer advertising. Further research is needed to understand the implications of these findings.

Expensive seems better: The price of a non-effective drug modulates its perceived efficacy.

Previous studies have shown that the price of a given product impacts the perceived quality of such product. This finding was also observed in medical contexts, showing that expensive drugs increase the placebo effect compared to inexpensive ones. However, addressing a drug's efficacy requires making causal inferences between the drug and the healing. These inferences rely on the contingency between these two events, a factor that is difficult to control in the placebo research. The present study aimed to test whether the price of a given drug modulates its perceived efficacy using a proper (though fictitious) non-effective drug, so that not only the objective contingency, but also the probability of the cause and the probability of the effect could be adequately controlled for. We expected higher efficacy judgements for the expensive non-effective drug than for the inexpensive one. To test this hypothesis, 60 volunteers participated in a contingency learning task that was programmed so that 72% of the patients healed regardless of whether they took the drug. Approximately one-half of the participants were told that the drug was expensive, whereas the other half were told that it was inexpensive. As expected, the efficacy judgements of participants who saw the expensive drug were significantly higher than those who saw the inexpensive one. Overall, our results showed that the price of a non-effective drug modulates its perceived efficacy, an effect that seems to be mediated by the estimated number of doses administered. This result parallels findings in the placebo literature but using a laboratory methodology that allows stronger control of the variables, suggesting that the illusory overestimation produced by the more expensive treatments might be on the basis of the greater efficacy of the more expensive placebos.