The usage and costs of national drug price-negotiated anticancer medicines in a first-tier city in Northeast China: a study based on health insurance data.

BACKGROUND: The National Drug Price Negotiation (NDPN) policy has entered a normalisation stage, aiming to alleviate, to some extent, the disease-related and economic burdens experienced by cancer patients. This study analysed the use and subsequent burden of anticancer medicines among cancer patients in a first-tier city in northeast China. METHODS: We assessed the usage of 64 negotiated anticancer medicines using the data on the actual drug deployment situation, the frequency of medical insurance claims and actual medication costs. The affordability of these medicines was measured using the catastrophic health expenditure (CHE) incidence and intensity of occurrence. Finally, we used the defined daily doses (DDDs) and defined daily doses cost (DDDc) as indicators to evaluate the actual use of these medicines in the region. RESULTS: During the study period, 63 of the 64 medicines were readily available. From the perspective of drug usage, the frequency of medical insurance claims for negotiated anticancer medicines and medication costs showed an increasing trend from 2018 to 2021. Cancer patients typically sought medical treatment at tertiary hospitals and purchased medicines at community pharmacies. The overall quantity and cost of medications for patients covered by the Urban Employee Basic Medical Insurance (UEBMI) were five times higher than those covered by the Urban and Rural Resident Medical Insurance (URRMI). The frequency of medical insurance claims and medication costs were highest for lung and breast cancer patients. Furthermore, from 2018 to 2021, CHE incidence showed a decreasing trend (2.85-1.60%) under urban patients' payment capability level, but an increasing trend (11.94%-18.42) under rural patients' payment capability level. The average occurrence intensities for urban (0.55-1.26 times) and rural (1.27-1.74 times) patients showed an increasing trend. From the perspective of drug utilisation, the overall DDD of negotiated anticancer medicines showed an increasing trend, while the DDDc exhibited a decreasing trend. CONCLUSION: This study demonstrates that access to drugs for urban cancer patients has improved. However, patients' medical behaviours are affected by some factors such as hospital level and type of medical insurance. In the future, the Chinese Department of Health Insurance Management should further improve its work in promoting the fairness of medical resource distribution and strengthen its supervision of the nation's health insurance funds.

Financial impact of integrated specialty pharmacy efforts to avoid oral anticancer medication waste.

BACKGROUND: The growing number of oral anticancer medications represents a significant portion of pharmacy spending and can be costly for patients. Patients taking oral anticancer medications may experience frequent treatment changes following necessary safety and effectiveness monitoring, often resulting in medication waste. Strategies to avoid medication waste could alleviate the financial burden of these costly therapies on the payer and the patient. OBJECTIVE: To evaluate the impact on waste and cost avoidance of reviewing the amount of medication patients have on hand and the presence of upcoming follow-up (ie, provider visit, laboratory testing, or imaging) before requesting a prescription refill renewal for patients taking oral anticancer medications through an integrated health system specialty pharmacy. METHODS: We performed a retrospective review of patients filling oral anticancer medications prescribed by a Vanderbilt University Medical Center provider and dispensed by Vanderbilt Specialty Pharmacy between January 1, 2020, and December 31, 2020. Specialty pharmacists received a system-generated refill renewal request for oral anticancer medications when the final prescription refill was dispensed, prompting the pharmacist to review the patient's medical record for continued therapy appropriateness and to request a new prescription. If the patient had a sufficient supply on hand to last until an upcoming follow-up (ie, provider visit, imaging, or laboratory assessment), the pharmacist postponed the renewal until after the scheduled follow-up. Patients were included in the analysis if the refill renewal request was postponed after review of the amount of medication on hand and the presence of an upcoming follow-up. Medication outcomes (ie, continued, dose changed, held, medication changed to a different oral anticancer medication, or discontinued) resulting from the follow-up were collected. Cost avoidance in US dollars was assigned based on the outcome of follow-up by calculating the price per unit times the number of units that would have been unused or in excess of what was needed if the medication had been dispensed before the scheduled follow-up. The average wholesale price minus 20% (AWP-20%) and wholesale acquisition cost (WAC) were used to report a range of costs avoided over 12 months. RESULTS: The total cost avoidance over 12 months associated with postponing refill renewal requests in a large academic health system with an integrated specialty pharmacy ranged from $549,187.03 using WAC pricing to $751,994.99 using AWP-20% pricing, with a median cost avoidance per fill of $366.04 (WAC) to $1,931.18 (AWP-20%). Refill renewal requests were postponed in 159 instances for 135 unique patients. After follow-up, medications were continued unchanged in only 2% of postponed renewals, 56% of follow-ups resulted in medication discontinuations, 32% in dose changes, 5% in medication changes, and 5% in medication holds. CONCLUSIONS: Integrated health system specialty pharmacist postponement of refill requests after review of the amount of medication on hand and upcoming follow-up proved effective in avoiding waste and unnecessary medication costs in patients treated with oral anticancer medications at a large academic health system.

Access denied: CMS' action hurts patients with cancer in rural America.

In 2020, cancer claimed more than 600,000 lives in the US. Cancer is an unyielding public health crisis. Cancer treatments typically involve multidisciplinary approaches, including surgery, radiation therapy, chemotherapy, and oral medications. For patients, especially those in rural areas, obtaining multiple oral medications can be inconvenient. The adoption of delivering cancer medications from medically integrated pharmacies (MIPs) has become popular in recent years. On May 12, 2023, CMS introduced guidelines restricting MIPs from delivering cancer-specific medications by mail or to oncology satellite offices and also requiring patients themselves to pick up the medications in person. This regulatory change has had a devastating impact on patients with cancer in rural and underserved communities, exacerbating existing health care disparities. This commentary explores the negative impacts of the policy change by CMS in rural America.

Breakthrough Therapy Cancer Drugs and Indications With FDA Approval: Development Time, Innovation, Trials, Clinical Benefit, Epidemiology, and Price.

BACKGROUND: The breakthrough therapy designation (BTD) facilitates the development of drugs with a large preliminary benefit in treating serious or life-threatening diseases. This study analyzes the FDA approval, trials, benefits, unmet needs, and pricing of breakthrough and nonbreakthrough therapy cancer drugs and indications. PATIENTS AND METHODS: We analyzed 355 cancer indications with FDA approval (2012-2022). Breakthrough and nonbreakthrough indications were compared regarding their FDA approval, innovativeness, clinical trials, epidemiology, and price. Data were extracted from FDA labels, the Global Burden of Disease study, and the Centers for Medicare & Medicaid Services. Hazard ratios (HRs) for overall survival (OS), progression-free survival (PFS), and relative risk (RR) of tumor response were meta-analyzed across randomized controlled trials. Objective response rates (ORRs) were meta-analyzed for single-arm trials. RESULTS: We identified 137 breakthrough and 218 nonbreakthrough cancer indications. The median clinical development time was 3.2 years shorter for breakthrough drugs than for nonbreakthrough drugs (5.6 vs 8.8 years; P=.002). The BTD was more frequently granted to biomarker-directed indications (46% vs 34%; P=.025) supported by smaller trials (median, 149 vs 326 patients; P<.001) of single-arm (53% vs 27%; P<.001) and phase I or II design (61% vs 31%; P<.001). Breakthrough indications offered a greater OS (HR, 0.69 vs 0.74; P=.031) and tumor response (RR, 1.48 vs 1.32; P=.006; ORR, 52% vs 40%; P=.004), but not a PFS benefit (HR, 0.53 vs 0.58; P=.212). Median improvements in OS (4.8 vs 3.2 months; P=.002) and PFS (5.4 vs 3.3 months; P=.005) but not duration of response (8.7 vs 4.7 months; P=.245) were higher for breakthrough than for nonbreakthrough indications. The BTD was more frequently granted to first-in-class drugs (42% vs 28%; P=.001) and first-in-indication treatments (43% vs 29%; P<.001). There were no differences in treatment and epidemiologic characteristics between breakthrough and nonbreakthrough drugs. Breakthrough drugs were more expensive than nonbreakthrough drugs (mean monthly price, $38,971 vs $22,591; P=.0592). CONCLUSIONS: The BTD expedites patient access to effective and innovative, but also expensive, new cancer drugs and indications.

The impact of willingness-to-pay threshold on price reduction recommendations for oncology drugs: a review of assessments conducted by the Canadian Agency for Drugs and Technologies in Health.

Since late 2020, the Canadian Agency of Drugs and Technologies in Health (CADTH) has been using a threshold of $50,000 (CAD) per quality-adjusted life-year (QALY) for both oncology and non-oncology drugs. When used for oncology products, this threshold is hypothesized to have a higher impact on the time to access these drugs in Canada. We studied the impact of price reductions on time to engagement and negotiation with the pan-Canadian Pharmaceutical Alliance for oncology drugs reviewed by CADTH between January 2020 and December 2022. Overall, 103 assessments reported data on price reductions recommended by CADTH to meet the cost-effectiveness threshold for reimbursement. Of these assessments, 57% (59/103) recommendations included a price reduction of greater than 70% off the list price. Eight percent (8/103) were not cost-effective even at a 100% price reduction. Of the 47 assessments that had a clear benefit, in 21 (45%) CADTH recommended a price reduction of at least 70%. The median time to price negotiation (not including time to engagement) for assessments that received at least 70% vs >70% price reduction was 2.6 vs 4.8 months. This study showed that there is a divergence between drug sponsor's incremental cost-effectiveness ratio (ICER) and CADTH revised ICER leading to a price reduction to meet the $50,000/QALY threshold. For the submissions with clear clinical benefit the median length of engagement (2.5 vs 3.3 months) and median length of negotiation (3.1 vs 3.6 months) were slightly shorter compared with the submissions where uncertainties were noted in the clinical benefit according to CADTH. This study shows that using a $50,000 per QALY threshold for oncology products potentially impacts timely access to life saving medications.

Access to innovative anticancer medicines in China: a national survey on availability, price and affordability.

OBJECTIVES: This study aimed to investigate the availability, price, and affordability of nationally negotiated innovative anticancer medicines in China. DESIGN: Retrospective observational study based on data from a nationwide medical database. DATA SOURCES/SETTING: Quarterly data about the use of innovative anticancer medicines from 2020 to 2022 were collected from the Chinese Medicine Economic Information Network. This study covered 895 public general hospitals in 30 provincial administrative regions in China. Of the total hospitals, 299 (33.41%) were secondary and 596 (66.59%) were tertiary. MAIN OUTCOME MEASURES: The adjusted WHO and Health Action International methodology was used to calculate the availability and affordability of 33 nationally negotiated innovative anticancer medicines in the investigated hospitals. Price is expressed as the defined daily dose cost. RESULTS: On average, the total availability of 33 innovative anticancer medicines increased annually from 2020 to 2022. The median availability of all investigated medicines in tertiary hospitals from 2020 to 2022 was 24.04%, 33.60% and 37.61%, respectively, while the indicators in secondary hospitals were 4.90%, 12.54% and 16.48%, respectively. The adjusted prices of the medicines newly put in Medicare (in March 2021) decreased noticeably, with the decline rate ranging from 39.98% to 82.45% in 2021 compared with those in 2020. Most generic brands were priced much lower than the originator brands. The affordability of anticancer medicines has improved year by year from 2020 to 2022. In comparison, rural residents had lower affordability than urban residents. CONCLUSIONS: The overall accessibility of 33 nationally negotiated innovative anticancer medicines improved from 2020 to 2022. However, the overall availability of most anticancer medicines in China remained at a low level (less than 50%). Further efforts should be made to sufficiently and equally benefit patients with cancer.

Impact of Early Access Reform on Oncology Innovation in France: Approvals, Patients, and Costs.

BACKGROUND: An ambitious reform of the early access (EA) process was set up in July 2021 in France, aiming to simplify procedures and accelerate access to innovative drugs. OBJECTIVE: This study analyzes the characteristics of oncology drug approvals through the EA process and its impact on real-life data for oncology patients. METHODS: The number and characteristics of EA demands concerning oncology drugs submitted to the National Health Authority (HAS, Haute Autorité de Santé) were reviewed until 31 December 2022. A longitudinal retrospective study on patients treated with an EA oncology drug between 1 January 2019 and 31 December 2022 was also performed using the French nationwide claims database (Systeme National des Données de Santé [SNDS]) to assess the impact of the reform on the number of indications and patients, and the costs. RESULTS: Among 110 published decisions, the HAS granted 88 (80%) EA indications within 70 days of assessment on average, including 46 (52%) in oncology (67% in solid tumors and 33% in hematological malignancies). Approved indications were mostly supported by randomized phase III trials (67%), whereas refused EA relied more on non-randomized (57%) trials. Overall survival was the primary endpoint of 28% of EA approvals versus none of denied EAs. In the SNDS data, the annual number of patients with cancer treated with an EA drug increased from 3137 patients in 2019 to 18,341 in 2022 (+ 484%), whereas the number of indications rose from 12 to 62, mainly in oncohematology (n = 17), lung (n = 12), digestive (n = 9) and breast cancer (n = 9). Reimbursement costs for EA treatments surged from €42 to €526 million (+ 1159%). CONCLUSION: The French EA reform contributed to enabling rapid access to innovations in a wide range of indications for oncology patients. However, the findings highlight ongoing challenges in financial sustainability, warranting continued evaluation and adjustments.

Global representativeness and impact of funding sources in cost-effectiveness research on systemic therapies for advanced breast cancer: A systematic review.

BACKGROUND: Breast cancer (BC) is the most incident tumor and, consequently, any new intervention can potentially promote a considerable budget impact if incorporated. Cost-effectiveness (CE) studies assist in the decision-making process but may be influenced by the country's perspective of analysis and pharmaceutical industry funding. METHODS: A systematic review of Medline, Scopus, and Web of Science from January 1st, 2012 to July 8th, 2022 was conducted to identify CE studies of tumor-targeted systemic-therapies for advanced BC. Articles without incremental cost-effectiveness ratio calculations were excluded. We extracted information on the country and class of drug studied, comparator type, authors' conflicts of interest (COI), pharmaceutical industry funding, and authors' conclusions. RESULTS: 71 studies comprising 204 CE assessments were included. The majority of studies were from the United States and Canada (44%), Asia (32%) and Europe (20%). Only 8% were from Latin America and none from Africa. 31% had pharmaceutical industry funding. The most studied drug classes were cyclin-dependent-kinase inhibitors (29%), anti-HER2 therapy (23%), anti-PD(L)1 (11%) and hormone therapy (11%). Overall, 34% of CE assessments had favorable conclusions. Pharmaceutical industry-funded articles had a higher proportion of at least one favorable conclusion (82% vs. 24%, p-value<0.001), European countries analyzed (45% vs. 9%, p-value = 0.003), and CE assessments with same class drug comparators (56% vs. 33%, p-value = 0.004). CONCLUSIONS: Breast cancer CE literature scarcely represents low-and-middle-income countries' perspectives and is influenced by pharmaceutical industry funding which targets European countries', frequently utilizes comparisons within same-drug class, and is more likely to have favorable conclusions.

Critical Examination of Modeling Approaches Used in Economic Evaluations of First-Line Treatments for Locally Advanced or Metastatic Non-Small Cell Lung Cancer Harboring Epidermal Growth Factor Receptor Mutations: A Systematic Literature Review.

BACKGROUND: Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, with up to 32% of patients with NSCLC harboring an epidermal growth factor receptor (EGFR) mutation. NSCLC harboring an EGFR mutation has a dedicated treatment pathway, with EGFR tyrosine kinase inhibitors and platinum-based chemotherapy often being the therapy of choice. OBJECTIVE: The aim of this study was to systemically review and summarize economic models of first-line treatments used for locally advanced or metastatic NSCLC harboring EGFR mutations, as well as to identify areas for improvement for future models. METHODS: Literature searches were conducted via Ovid in PubMed, MEDLINE, MEDLINE In-Process, Embase, Evidence-Based Medicine Reviews: Health Technology Assessment, Evidence-Based Medicine Reviews: National Health Service Economic Evaluation Database, and EconLit. An initial search was conducted on 19 December 2022 and updated on 11 April 2023. Studies were selected according to predefined criteria using the Population, Intervention, Comparator, Outcome and Study design (PICOS) framework. RESULTS: Sixty-seven articles were included in the review, representing 59 unique studies. The majority of included models were cost-utility analyses (n = 52), with the remaining studies being cost-effectiveness analyses (n = 4) and a cost-minimization analysis (n = 1). Two studies incorporated both a cost-utility and cost-minimization analysis. Although the model structure across studies was consistently reported, justification for this choice was often lacking. CONCLUSIONS: Although the reporting of economic models in NSCLC harboring EGFR mutations is generally good, many of these studies lacked sufficient reporting of justification for structural choices, performing extensive sensitivity analyses and validation in economic evaluations. In resolving such gaps, the validity of future models can be increased to guide healthcare decision making in rare indications.

Budget Impact of Introducing Fixed-Duration Mosunetuzumab for the Treatment of Relapsed or Refractory Follicular Lymphoma After Two or More Lines of Systemic Therapy in the USA.

OBJECTIVE: This study aimed to assess the budget impact of introducing fixed-duration mosunetuzumab as a treatment option for adult patients with relapsed or refractory follicular lymphoma after at least two prior systemic therapies and to estimate the total cumulative costs per patient in the USA. METHODS: A 3-year budget impact model was developed for a hypothetical 1-million-member cohort enrolled in a mixed commercial/Medicare health plan. Comparators were: axicabtagene ciloleucel, tisagenlecleucel, tazemetostat, rituximab plus lenalidomide, copanlisib, and older therapies (rituximab or obinutuzumab ± chemotherapy). Costs per patient comprised treatment-associated costs including the drug, its administration, adverse events, and routine care. Dosing and safety data were ascertained from respective package inserts and clinical trial data. Drug costs (March 2023) were estimated based on the average wholesale acquisition cost reported in AnalySource®, and all other costs were based on published sources and inflated to 2022 US dollars. Market shares were obtained from Genentech internal projections and expert opinion. Budget impact outcomes were presented on a per-member per-month basis. RESULTS: Compared with a scenario without mosunetuzumab, its introduction over 3 years resulted in a budget increase of $69,812 (1% increase) and an average per-member per-month budget impact of $0.0019. Among the newer therapies, mosunetuzumab had the second-lowest cumulative per patient cost (mosunetuzumab = $202,039; axicabtagene ciloleucel = $505,845; tisagenlecleucel = $476,293; rituximab plus lenalidomide = $263,520; tazemetostat = $250,665; copanlisib = $127,293) and drug costs, and its introduction only increased total drug costs by 0.1%. By year 3, the cumulative difference in the per patient cost with mosunetuzumab was -$303,805 versus axicabtagene ciloleucel, -$274,254 versus tisagenlecleucel, -$61,481 versus rituximab plus lenalidomide, -$48,625 versus tazemetostat, and $74,747 versus copanlisib. Older therapies were less costly with 3-year cumulative costs that ranged from $36,512 to $147,885. CONCLUSIONS: Over 3 years, the estimated cumulative per patient cost of mosunetuzumab is lower than most available newer therapies, resulting in a small increase in the budget after its formulary adoption for the treatment of relapsed or refractory follicular lymphoma.

Budget impact analysis of venetoclax for the management of acute myeloid leukemia from the perspective of the social security and the private sector in Argentina.

OBJECTIVE: This study aimed to estimate the budget impact of the incorporation of venetoclax for the treatment of patients with Acute Myeloid Leukemia (AML) over 75 years of age or those with comorbidities and contraindications for the use of intensive chemotherapy, from the perspective of the social security and the private third-party payers in Argentina. METHODS: A budget impact model was adapted to estimate the cost difference between the current scenario (azacitidine, decitabine and low doses of cytarabine) and the new scenario (incorporation of venetoclax) for a third-party payer over a time horizon of three years. Input parameters were obtained from a literature review, validated or complemented by expert opinion using a modified Panel Delphi approach. All direct medical costs were estimated by the micro-costing approach and were expressed in US dollars (USD) as of September 2020 (1 USD = 76.18 Argentine pesos). RESULTS: For a third-party payer with a cohort of 1,000,000 individuals covered, incorporating venetoclax was associated with an average budget impact per-member per-month (PMPM) of $0.11 USD for the social security sector and $0.07 USD for the private sector. The duration of treatment with venetoclax was the most influential parameter in the budget impact results. CONCLUSION: The introduction of venetoclax was associated with a positive and slight budget impact. These findings are informative to support policy decisions aimed to expand the current treatment landscape of AML.

Lengthy and Variable Delays in Oncology Drug Coverage Determination.

This cohort study examines the variability in time to pharmacy and therapeutics committees' determinations of coverage of approved oncology drugs across multiple payers.

Biogenic Silver Nanoparticles for Targeted Cancer Therapy and Enhancing Photodynamic Therapy.

Different conventional therapeutic procedures are utilized globally to manage cancer cases, yet the mortality rate in patients with cancer remains considerably high. Developments in the field of nanotechnology have included novel therapeutic strategies to deal with cancer. Biogenic (green) metallic silver nanoparticles (AgNPs) obtained using plant-mediated protocols are attractive to researchers exploring cancer treatment. Biogenic AgNPs present advantages, since they are cost-effective, easy to obtain, energy efficient, and less toxic compared to chemically and physically obtained AgNPs. Also, they present excellent anticancer abilities thanks to their unique sizes, shapes, and optical properties. This review provides recent advancements in exploring biogenic AgNPs as a drug or agent for cancer treatment. Thus, great attention was paid to the anticancer efficacy of biogenic AgNPs, their anticancer mechanisms, their efficacy in cancer photodynamic therapy (PDT), their efficacy in targeted cancer therapy, and their toxicity.

Utilization Management Trends in Medicare Part D Oncology Drugs, 2010-2020.

This study examines the exposure of Medicare Part D beneficiaries to utilization management, such as prior authorization, for oral oncology drugs.

[New, more expensive - Not necessarily better]. / Nouveau et (beaucoup) plus cher ­ Meilleur ? - Chroniques génomiques.

The prices of new oncology drugs are frequently above 100,000 US dollars, and this does not generally correlate with significantly improved clinical efficacy. In the absence of effective regulation and of real competition, companies tend to charge « what the market can bear ¼. Regulatory intervention is required, notably at the EU level.

Treatment patterns and economic burden among newly diagnosed cervical and endometrial cancer patients.

Aim: This study evaluated treatment patterns, healthcare resource use and healthcare costs among newly diagnosed US patients with cervical or endometrial cancer. Materials & methods: The authors identified patients diagnosed between 2015 and 2018, described them by line of therapy (LOT), then summarized all-cause per patient per month healthcare resource use and healthcare costs per LOT. Results: Among 1004 patients with cervical cancer and 2006 patients with endometrial cancer, 65.2 and 71.4%, respectively, received at least LOT1. Common treatment modalities in LOT1 were surgery (cervical, 58.0%; endometrial, 92.6%), radiation therapy (cervical, 49.8%; 24.7%) and systemic therapy (cervical, 53.3%; endometrial, 26.1%). Mean per patient per month costs per LOT were pre-treatment (cervical, US$17,210; endometrial, US$14,601), LOT1 (cervical, US$10,929; endometrial, US$6859), LOT2 (cervical, US$15,183; endometrial, US$10,649) and LOT3+ (cervical, US$19,681; endometrial, US$9206). Conclusion: Overall, newly diagnosed patients with cervical or endometrial cancer received guideline-recommended treatment. Outpatient visits mainly drove healthcare costs across LOTs.

Treatment patterns and economic burden among cervical and endometrial cancer patients newly initiating systemic therapy.

Aim: To evaluate treatment patterns, healthcare resource use (HCRU) and all-cause healthcare costs among patients with cervical or endometrial cancer newly initiating systemic therapy. Methods: We identified patients with cervical or endometrial cancer newly initiating systemic therapy - a claims-based proxy for advanced disease - between 2014 and 2019, described them by line of therapy (LOT), and summarized the per patient per month (PPPM) HCRU and healthcare costs per LOT. Results: Among 1229 patients with cervical cancer and 2659 patients with endometrial cancer, LOT1 therapies included systemic only (cervical, 50.1%; endometrial, 83.2%) and systemic with radiation therapy (cervical, 49.9%; endometrial, 16.8%). Mean PPPM total costs were: LOT1 (cervical, US$15,892; endometrial, US$11,363), LOT2 (US$20,193; US$14,019) and LOT3+ (US$16,576; US$14,645). Conclusions: Overall, patients received guideline-concordant care and experienced significant economic burden, which increased with LOT.