Access to Oncology Medicines in Canada: Consensus Forum for Recommendations for Improvement.

Patient access to new oncology drugs in Canada is only possible after navigating multiple sequential systemic checkpoints for national regulatory approval, health technology assessment (HTA) and collective government price negotiation. These steps delay access and prevent health care providers from being able to prescribe optimal therapy. Eighteen Canadian oncology clinicians from the medicine, nursing and pharmacy professions met to develop consensus recommendations for defining reasonable government performance standards around process and timeliness to improve Canadian cancer patients' access to best care. A modified Delphi methodology was used to identify consensus on 30 questions involving five themes: accountability, disparities, endpoints, timeliness, and cost-effectiveness. It was agreed that greater transparency is required across regulatory and HTA processes. Health professionals in oncology are frustrated for their patients because they are unable to deliver the modern guideline-supported therapies they want to provide due to delays in approval or funding. Canadian health care providers request improvements in timely access to life-saving therapeutics in line with other comparator countries. Clinicians expect urgent improvements in Canadian health systems to give our patients their best chance of survival.

Drug pricing and transparency in Europe and the United States: what is it and how does it work?

INTRODUCTION: As drug prices are viewed to be opaque, there have been increasing societal demands on policy and decision makers to implement initiatives that promote drug price transparency. AREAS COVERED: This Perspective discusses what drug price transparency is and how it works in theory and in practice. EXPERT OPINION: Transparency on drug prices may target payers, patients and health care professionals; and may relate to prices at each stage in a drug's distribution system. Although proponents claim that drug price transparency will reduce prices and increase patient access, others expect the opposite effect. Nevertheless, a number of international organizations, countries and consumer groups have taken steps to enhance drug price transparency. This has occurred despite a lack of theoretical clarity and of evidence about its likely impact. Policy and decision makers need to consider how payers and pharmaceutical companies are likely to react to drug price transparency and need to be aware that transparency may produce different effects depending on the country to which it is applied. Even though we believe that full drug price transparency is elusive, various incremental measures can be taken to move toward it.

Association between changes in prices and out-of-pocket costs for brand-name clinician-administered drugs.

OBJECTIVE: To determine whether annual changes in prices for clinician-administered drugs are associated with changes in patient out-of-pocket costs. DATA SOURCES AND STUDY SETTING: National commercial claims database, 2009 to 2018. STUDY DESIGN: In a serial, cross-sectional study, we calculated the annual percent change in manufacturer list prices and net prices after rebates. We used two-part generalized linear models to assess the relationship between annual changes in price with (1) the percentage of individuals incurring any out-of-pocket costs and (2) the percent change in median non-zero out-of-pocket costs. DATA COLLECTION/EXTRACTION METHODS: We created annual cohorts of privately insured individuals who used one of 52 brand-name clinician-administered drugs. PRINCIPAL FINDINGS: List prices increased 4.4%/yr (interquartile range [IQR], 1.1% to 6.0%) and net prices 3.3%/yr (IQR, 0.3% to 5.5%). The median percentage of patients with any out-of-pocket costs increased from 38% in 2009 to 48% in 2018, and median non-zero annual out-of-pocket costs increased by 9.6%/yr (IQR, 4.1% to 15.4%). There was no association between changes in prices and out-of-pocket costs for individual drugs. CONCLUSIONS: From 2009 to 2018, prices and out-of-pocket costs for brand-name clinician-administered drugs increased, but these were not directly related for individual drugs. This may be due to changes to insurance benefit design and private insurer drug reimbursement rates.

Trends in price for topical corticosteroids from 2017 to 2021 and the opportunity for cost savings identifiable at the point of care: A retrospective cross-sectional study.

BACKGROUND: Topical corticosteroids possess numerous generics and similar-strength substitutes. Affordability can impact obtaining the medication prescribed. OBJECTIVE: To determine recent trends in topical corticosteroid pricing and potential for cost saving. METHODS: A retrospective cross-sectional study analyzing all prescriptions dispensed for topical corticosteroids from January 1, 2017 through December 31, 2021, using a US all-payer pharmacy-claims database and commercial coupon dataset, was performed. RESULTS: Two hundred thirty-seven unique drug products (≥1 claim) were identified. Factors that predicted for higher cost (P < .05) were branded products (105% more expensive than generics) and ultrapotent class (55% more expensive than low potency) while ointments predicted for lower cost (19% less expensive than creams). Cash prices remained relatively stable, except for ultrapotent branded topical corticosteroids (63% increase). Cost savings were available for both brand-to-generic ($14.75 per unit) and generic-to-generic ($6.82 per unit) switching. Coupon prices were consistently lower than cash prices (r = 0.89). LIMITATIONS: Contracted rates through insurance plans were not included. CONCLUSIONS: Topical corticosteroid prices over the past 5 years have stabilized, the exception being branded ultrapotent corticosteroids. Savings from switching among similar-strength substitutes remain significant despite price stabilization. Coupon prices mirror the hierarchy of cash prices and can help assess real-time costs.

Stakeholder Perspectives of the Inflation Reduction Act's (2022) Impact on Prescription Drugs: A Narrative Review.

In this review, we examine the impact of the Inflation Reduction Act (IRA) of 2022 on pharmaceutical drugs in the United States, drawing on a diverse range of sources to understand the perceptions of multiple stakeholders and professionals. Findings suggest that the Act, while aiming to control price inflation, has had a multifaceted impact on the pharmaceutical sector. Stakeholders, including pharmaceutical companies, healthcare providers, patient advocacy groups, and policymakers, offered varied perspectives: while some laud the Act for its potential in controlling runaway drug prices and making healthcare more accessible, others raise concerns about possible reductions in drug innovation, disruptions to supply chains, and the sustainability of smaller pharmaceutical companies. The review identified four underlying constructs (themes) in the literature surrounding healthcare stakeholders' perceptions of the IRA's impact upon prescription drugs: pricing and/or dictation pricing issues, topics related to patent law and pharmaceuticals, processes surrounding the IRA's (2022) rules and regulations, and potential threats to the pharmaceutical industry concerning the research and development of future medications. The complex interplay of the Act's implications underscores the importance of ongoing assessment and potential iterative policy refinements as implementation endures.

Generic cardiology drug prices: the potential benefits of the Marc Cuban cost plus drug company model.

Introduction: Generic pharmaceuticals account for the majority of the $359 billion US pharmaceutical market, including for cardiology drugs. Amidst a lack of price transparency and administrative inefficiencies, generic drug prices are high, causing an undue burden on patients. Methods: We identified the 50 most used generic cardiology drugs by volume per the 2020 Medicare Part D spending data. We extracted cost per dose of each drug from the Marc Cuban Cost Plus Drug Company (MCCPDC) website and estimated the aggregate cost savings if MCCPDC were employed on a national scale by calculating the difference between this cost and Medicare spending. Results: Medicare spent $7.7 billion on the 50 most used generic cardiology drugs by volume in 2020 according to Medicare Part D data. Pharmacy and shipping costs accounted for a substantial portion of expenditures. Per our most conservative estimate, $1.3 billion (17% of total) savings were available on 16 of 50 drugs. A slightly less conservative estimate suggested $2.9 billion (38%) savings for 35 of 50 drugs. Discussion: There is enormous potential for cost savings in the US market for generic cardiology drugs. By encouraging increased competition, decreasing administrative costs, and advocating for our patients to compare prices between the MCCPDC and other generic pharmaceutical dispensers, we have the potential to improve access to care and corresponding outcomes for cardiology patients.

Therapeutic value of new medicines not submitted to Health Canada 2014-2021: Cross-sectional study.

OBJECTIVES: To examine whether there has been a change in the number of therapeutically important new medicines not being introduced into the Canadian market in light of the December 2017 announcement of regulatory changes to lower Canadian prices. METHODS: A list was compiled of medicines approved by the Food and Drug Administration (FDA) between 2014-2021 but not submitted to Health Canada. The therapeutic value of these medicines was assessed based on evaluations by two independent sources. If no evaluation was available, potential therapeutic value was determined based on the presence of one or more of three medicine characteristics. Interrupted time series was used to determine if there were changes in overall new medicine introductions and therapeutically important new medicines. RESULTS: The FDA approved 364 new medicines of which 116 (31.9%) were not submitted to Health Canada. There was a decrease in overall annual number of submissions but that was not related to the announcement at the end of 2017. There was no change in the introduction of therapeutically important new medicines as a percent of all medicines not marketed in Canada but there was a decrease in the absolute number. CONCLUSIONS: The number of therapeutically important medicines not being introduced into Canada is increasing but that is not related to the proposed price reforms.

Medicines for the People, not the Financial Markets.

Drug company marketing scandals have often made headlines. However, in recent years, a raft of new pharmaceutical industry transgressions has created crises for health care systems of high-income countries as well as poorer nations. Prices of life-saving drugs are soaring; supply shortfalls of essential medications have become common; development of new antibiotics against resistant organisms is lagging; and inequalities in access to medicines are widening. Policymakers must find new solutions to protect health systems and health.

Cancer Drug Prices in the United States: Efficacy, Innovation, Clinical Trial Evidence, and Epidemiology.

OBJECTIVES: Rising cancer drug prices challenge patients and healthcare systems. Although prices are routinely assigned to original drug indications receiving US Food and Drug Administration (FDA) approval, the pricing of supplemental indication approvals remains uncertain. This study identifies and quantifies factors associated with cancer drug prices, distinctly analyzing original and supplemental indications. METHODS: Clinical trial evidence and epidemiologic data supporting new indications' FDA approval (2003-2022) were collected from the Drugs@FDA database, ClinicalTrials.gov, and Global Burden of Disease study. Indication-specific monthly treatment costs were calculated for Medicare patients. The association between log-prices and collected variables were assessed in regression analyses. RESULTS: We identified 145 drugs approved across 373 cancer indications. Drugs were priced at $24 444 per month on average (median = $16 013). For original indications, prices weakly correlated to improvements in overall survival (ß = 0.28, P = .037) and progression-free survival (ß = 0.16, P = .001). Original indications' prices were as follows: (1) negatively associated with disease incidence (ß = -0.21, P < .001) and prevalence; (2) positively correlated with first-in-class drugs (26%, P = .057), gene and cell therapies (176%, P < .001), hematologic cancers (62%, P < .001), and severe diseases with substantial unmet needs (6% per disability-adjusted life-year, P < .001); and (3) negatively correlated to indications with randomized-controlled phase 3 trials. Prices were poorly associated with supplemental indications' efficacy, clinical evidence, and epidemiology. CONCLUSIONS: Cancer drug prices are set based on the original indication's characteristics, thereby omitting the value of supplemental indications. Indication-specific pricing, coverage, and reimbursement policies considering each indication's safety, efficacy, innovativeness, and unmet needs are necessary to align a drug's value and price.

Pharmaceutical Purchasing: a Review of the Landscape and Implications for Antidotal Therapies.

The management of the poisoned patient often requires the utilization of uncommonly used pharmaceutical interventions. These interventions can be associated with significant costs to both the patient and treating institution. Pharmaceutical supply shortages and issues with accessibility of antidotal therapies complicate the management of many toxic exposures. These challenges are an inherent property of the pharmaceutical purchasing infrastructure in the United States, which is a complicated network of public and private intra-institutional agreements. The cost and availability of any given therapy is dependent on the individual contracting agreements between the treating institution, payer, pharmacy benefit manager, manufacturer or wholesaler, and in some cases a specialty pharmacy. Small or remote hospitals may experience greater challenges related to insufficient patient volume to achieve predicable prescribing patterns of rare and expensive medications, necessitating consignment purchasing arrangements. Although pharmaceutical costs are the focus of recent legislative attention, these reforms are not expected to significantly alter the cost or availability of antidotal therapies.

A Pharmacoeconomic Outlook of the Biological Drugs Marketed in India: A Cost Variance Analysis.

Introduction Biologic drugs are used to treat various illnesses like autoimmune diseases, cancers, hormonal irregularities, anemia, etc., and to prevent various diseases as vaccines. Though various biologic drugs are already available, they are still not within reach of the common man due to financial constraints. Through many search engines, studies evaluating the cost variation of different brands of biologics were investigated; however, only a few studies that address this problem were found. Hence, this study was planned with the objective of addressing the cost variation of various brands of biologic medicines available in the Indian market. Methods The website for the Current Index of Medical Specialties (CIMS) for India's location was used to obtain the prices of the different brands of biologic medicines in Indian National Rupee (INR) currency, which different manufacturers market with identical forms in strength and dosage. The percentage cost variation and cost ratio were calculated with the help of the minimum and maximum prices of various brands of biologic drugs. Results The prices of biologics belonging to six different classes that are available in 23 formulations were analyzed. The highest cost variability was shown by pegfilgrastim 6 mg at 1,022.92%, and the minimum-cost variation was shown by darbepoetin alfa 200 mcg at 13.07%. Conclusion Our research found a vast variance in the costs of various brands of biologic medicines in India. The government should address this cost variation problem by developing various policies, such as breaking the monopoly of manufacturers, providing tax incentives to nonprofit generic medicine manufacturers, and incorporating more biologic drugs under the protection of the Drugs Prices Control Order (DPCO).

Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement.

Legislations incentivising orphan drug development and scientific advances have made orphan drugs pharma's high-end favourite for the past two decades. Currently, around 50% of new marketing authorizations are for orphan drugs. For third-party healthcare payers ("payers") the rise of orphan drugs presents new challenges, including a high degree of uncertainty around clinical benefits and harms, a moderate effect size (for many orphan drugs), and a high price tag. The association of high clinical uncertainty and moderate effect sizes is not surprising in small target populations but in combination with high prices creates the risk of allocative and technical inefficiencies for payers. We here discuss and illustrate these risks. A combination of policies is needed for mitigation of allocative inefficiency: while there may be a rationale for higher prices for orphan than non-orphan drugs, a focus of pricing and reimbursement negotiations should include considerations of product profitability and of the consequences of orphan drug costs on the distribution inequality of medication costs for individual insured persons, coupled to knowledge generation from reimbursement contracts covering high-price orphan drugs that would benefit the wider patient community. Performance-based managed entry agreements could help to de-risk the economic consequences of clinical uncertainty and to mitigate technical inefficiency.

Drug Cost-Effectiveness Assessments Require Standards for Rigor and Inclusion.

The Institute for Clinical and Economic Review (ICER), a nonprofit, nongovernmental organization, is the predominant independent price assessor in the United States. ICER's cost effectiveness assessments are increasingly being used to support health insurance coverage and healthcare policy decisions. ICER often does not apply rigorous data quality and inclusion criteria to either the assumptions embedded within their cost-effectiveness models or the data inputted into the models. Poor quality assumptions and data can lead to poor quality assessments. ICER should re-evaluate their reliance on quality adjusted life-years and equal value of life years gained as measures of drug effectiveness, establish data quality and inclusiveness minimum standards, produce cost-effectiveness assessments only when the minimum data is available, and prominently report data quality and inclusion limitations. These changes will increase the rigor and inclusiveness of drug price assessments and support sustainable access to high-value care for all Americans.

The Effect of the Drug Life Cycle Price on Cost-Effectiveness: Case Studies Using Real-World Pricing Data.

OBJECTIVES: Cost-effectiveness analyses (CEAs) generally assume constant drug prices throughout the model time horizon, yet it is known that prices are not constant, often with price decreases near loss of exclusivity (LOE). This study explores the impact of using dynamic drug-specific prices on the incremental cost-effectiveness ratio (ICER) using selected reproduced case studies. METHODS: Case studies were selected following explicit criteria to reflect a variety of drug characteristics. For each drug, a published CEA model was identified, replicated, and modified with dynamic real-world pricing data, to compare ICERs based on constant drug prices with estimates obtained when including drug life cycle pricing. The impact of dynamic real-world pricing-inclusive LOE-was analyzed using a single patient cohort and multiple cohorts over time. RESULTS: Fluvastatin, alendronic acid + colecalciferol combination therapy, letrozole and clopidogrel were selected as case studies. Inclusion of real-world pricing data compared with applying constant prices reduced the ICER in a single-cohort setting up to 43%. In the multicohort analyses, further reductions of the ICERs were observed of up to 113%. The ICERs were sensitive to the period of drug usage relative to the models' time horizons, the relative proportions of drug costs in the overall treatment costs, and timing of LOE compared with the cost year of the original analysis. CONCLUSIONS: Assuming dynamic drug prices may lead to more representative ICER estimates. Future CEAs for drugs could account for predicted and disaggregated life cycle price developments based on retrospective data.

Addressing High Drug Prices by Reforming Pharmacy Benefit Managers.

Recently, Congress has focused on reforms to address pharmacy benefit managers' (PBMs) role in high drug prices for patients. Congress must not excessively restrict PBMs' ability to negotiate with manufacturers; alternatively, reforms could be paired with other policies that address the high prices of brand-name drugs.

Pharmacy Benefit Management: The Cost of Drug Price Rebates.

Pharmacy Benefit Managers (PBM) induce drug manufacturers to offer rebates to insurers and employers by denying coverage through formulary exclusions, impeding physician prescription through prior authorization, and reducing patient drug use through cost sharing. As they tighten these access obstacles, PBMs reduce the net prices received by the manufacturers.

The least costly pharmacy for cancer supportive care medications over time: the logistic toxicity of playing catch up.

PURPOSE: No single pharmacy in an urban zip code is consistently the least expensive across medications. If medication prices change differently across pharmacies, patients and clinicians will face challenges accessing affordable medications when refilling medications. This is especially pertinent to people with cancer with multiple fills of supportive care medications over time. We evaluated if the lowest-priced pharmacy for a formulation remains the lowest-priced over time. METHODS: We compiled generic medications used to manage nausea/vomiting (14 formulations) and anorexia/cachexia (12 formulations). We extracted discounted prices in October 2021 and again in March 2022 for a typical fill at 8 pharmacies in Minneapolis, Minnesota, USA (zip code 55,414) using GoodRx.com. We examined how prices changed across formulations and pharmacies over time. RESULTS: Data were available for all 208 possible pharmacy-formulation combinations (8 pharmacies × 26 formulations). For 172 (83%) of the 208 pharmacy-formulation combinations, the March 2022 price was within 20% of the October 2021 price. Across pharmacy-formulation combinations, the price change over time ranged from - 76 to + 292%. For 12 (46%) of the 26 formulations, at least one pharmacy with the lowest price in October 2021 no longer was the least costly in March 2022. For one formulation (dronabinol tablets), the least expensive pharmacy became the most expensive, with an absolute and relative price increase of a fill of $22 and 85%. CONCLUSION: For almost half of formulations studied, at least one pharmacy with the lowest price was no longer the least costly a few months later. The lowest price for a formulation (across pharmacies) could also change considerably. Thus, even if a patient accesses the least expensive pharmacy for a medication, they may need to re-check prices across all pharmacies with each subsequent fill to access the lowest prices. In addition to safety concerns, directing medications to and accessing medications at multiple pharmacies can add time and logistic toxicity to patients with cancer, their care partners, prescribers, and pharmacy teams.

Self-Medication Behaviors of Chinese Residents and Consideration Related to Drug Prices and Medical Insurance Reimbursement When Self-Medicating: A Cross-Sectional Study.

BACKGROUND: Self-medication has become a common phenomenon. Economic factors are important factors that affect the self-medication of residents. This study aimed to investigate the current status of self-medication behaviors in China and explored the related factors affecting considerations associated with medical insurance reimbursement or drug price in self-medication. METHODS: A national cross-sectional investigation was conducted among Chinese people over 18 years old under a multi-stage sampling method through a questionnaire, which includes demographic sociological characteristics, self-medication behaviors and scales. The Chi-square test was used to analyze whether the respondents consider medical insurance reimbursement or drug price as an important factor when purchasing over-the-counter (OTC) drugs. Logistic regression was used to examine the associated factors of considering medical insurance reimbursement or drug price. RESULTS: In total, 9256 respondents were included in this study; 37.52% of the respondents regarded drug prices as an important consideration, and 28.53% of the respondents attached great importance to medical insurance reimbursement. Elderly respondents who lived in the central region, had medical insurance, and had lower levels of health literacy were more likely to consider the medical insurance reimbursement, while respondents with high monthly family income as well as students were less likely to consider the same issue (p < 0.05). Respondents settled in the central and western regions, students, those without fixed occupations, those who suffered from chronic diseases, or those with lower health literacy were more likely to consider drug prices, while the respondents with bachelor degrees, urban population and high per capita monthly income were less likely to consider the drug prices (p < 0.05). CONCLUSION: Self-medication behaviors with OTC drugs were prevalent in China, and consideration factors of medical insurance reimbursement or drug prices were related to socio-demographic characteristics and health literacy. There is a need to take measures to reduce the economic burden of self-medication, improve the health literacy of residents and strengthen public health education.

Impact of the Drug Prices Control Order (2013) on the Utilization of Anticancer Medicines in India: An Interrupted Time-Series Analysis.

Objectives The National Pharmaceutical Pricing Authority introduced a series of Drug Prices Control Orders since 1970 to regulate the prices of essential medicines in India. This study evaluated the impact of the Drug Prices Control Order of 2013 on the utilization of anticancer medicines in the Indian private sector. Methods We used monthly sales audit data for a period of 2012-15, provided by Intercontinental Medical Statistics (IMS) Health. Through interrupted time series design and segmented regression models, we estimated the change in utilization of anticancer medicines following the drug pricing policy implementation. Results Of 1556 anticancer drug packs, 22.3% (n= 347) were price-controlled. The policy led to an immediate monthly reduction of 27.3% (95% CI -38.6%, -13.9%; p=0.001) and a long-term monthly reduction of 0.7% (95% CI -1.6%, 0.3%; p=0.16) in price-controlled formulation's utilization. In the final study month, the price-controlled formulation's utilization was 5.03 thousand standard units lower than what would have been expected without the policy. Melphalan showed the highest immediate reduction, and alpha-interferon showed the highest long-term reduction in utilization. Conclusion Drug prices control order 2013 caused an immediate and long-term decline in the utilization of anticancer medicines in the Indian private sector. However, study data was limited to a specific part of the Indian anticancer drug market, which must be considered when interpreting findings.