Increased Drug Costs Research Articles

The impact of pricing strategy on the costs of oral anti-cancer drugs.

BackgroundThe soaring costs of anti‐cancer drugs pose a threat to the sustainability of cancer care. The pricing strategy chosen by manufacturers can impact the costs of oral anti‐cancer drugs during dose modifications, but this issue remains under‐recognized in the literature. In general, with the flat pricing strategy, there is a single fixed price for each tablet regardless of dosage strength, whereas with linear pricing, the price of each tablet increases with its dose. We hypothesize that flat pricing will have increased drug costs compared to linear pricing during dose reductions since the cost remains fixed despite decreased dose requirements. This practice may have significant financial implications considering the high costs, extensive utilization, and frequent occurence of dose reductions with anti‐cancer drugs. MethodsOral anti‐cancer drugs reviewed by the pan‐Canadian Oncology Drug Review program between 2011 and 2018 were identified. The cost per mg and cost per 28‐day cycle were calculated for dose levels −2 to +2. The percent change in cost per mg and cost per cycle during dose modifications from the standard dose were calculated. We conducted Mann‐Whitney U and Fisher‐exact tests to compare the association between drug costs during dose reductions and pricing strategy.ResultsIn this study, 30 oral anti‐cancer drugs for use in 41 indications were analyzed; 44% of drugs used linear pricing and 56% used flat pricing. Dose reductions increased the mean cost per mg for drugs with linear pricing by 14.7% (range: 0%‐50%) at dose level −1 and 17.2% (range: 0%‐50%) at dose level −2 and flat pricing by 60.8% (range: 19%‐100%) at dose level −1 and 99.1% (range: 0%‐300%) at dose level −2. The cost per mg was significantly increased in drugs using flat pricing compared to linear pricing when dose reduction to either level ‐1 (P = 0.010) or level ‐2 (P = 0.006) occurred. The mean cost per cycle was decreased for drugs using linear pricing by 20.9% (range: −40% to 0%) at dose level −1 and 48.7% (range: −60% to −25%) at dose level −2 and flat pricing by 0.8% (range −6% to 0%) at dose level −1 and 11.0% (range: −50% to 100%) at dose level −2. The cost per cycle was significantly decreased in drugs with linear pricing compared to flat pricing when the standard dose is reduced to either dose level ‐1 (P = 0.005) or dose level ‐2 (P = 0.026).ConclusionsOverall, flat pricing had significantly greater costs compared to linear pricing during dose reductions of anti‐cancer drugs. While there is a general expectation that the cost of drugs should decrease with dose reduction, drugs with flat pricing were generally found to have increased cost per mg and no change in the cost per cycle. The resultant increased spending on drug acquisition (despite purchasing lower doses) lead to financial wastage, which has significant implications on cost‐effectiveness considerations and budgets. Future economic evaluations should take into consideration the hidden costs associated with dose reductions of flat priced drugs.

PCN107 BUDGET IMPACT ANALYSIS OF LAROTRECTINIB FOR 8 TUMORS IN THE UNITED STATES

Larotrectinib is an oral tropomyosin receptor kinase (TRK) inhibitor indicated for cancers where the tumor expresses neurotrophic tyrosine receptor kinase (NTRK) gene fusions. We assessed potential budget impact of the introduction of larotrectinib. In accordance with the expected pan-tumor indication, we assessed the financial implications of covering larotrectinib across 8 tumors: colorectal, non-small cell lung, melanoma, thyroid, gastrointestinal stromal tumor, infantile fibrosarcoma, soft tissue sarcoma, and salivary gland cancer. We created a budget impact model with a flexible time horizon. The model framework centralizes parameter control while separately modeling the available treatments, dosing, and market share for each of the 8 tumor types. The base case assumed a commercial U.S. health plan of 1,000,000 enrollees. The treated population for each tumor type was estimated from SEER, TRK fusion testing rate assumptions, fusion mutation prevalence, and treatment uptake assumptions. The budget impact was calculated as total and per member per month (PMPM) cost. Scenario analyses were performed in order to approximate the uncertainty in the base case results and identify likely financial determinants. We estimated that less than one patient would be treated with larotrectinib across the eight tumor types in the first year after accounting for the frequency of NTRK gene fusion in each tumor type. The total costs in year 1 without larotrectinib were $41,300 compared to $68,000 with the first year of larotrectinib uptake, an increase of $26,700 (less than $0.001 PMPM). Over three years, we estimated 1 patient was treated with larotrectinib, a PMPM increase of $0.01. The increase in costs was due proportionally to increased costs of genetic testing and increased drug costs. These results were most sensitive to the parameters that define the treated population. Adding larotrectinib is likely to have minimal additional costs when added to a health plan formulary.

Obinutuzumab plus chemotherapy followed by obinutuzumab monotherapy is cost-effective vs. rituximab plus chemotherapy followed by rituximab monotherapy for previously untreated follicular lymphoma patients in the United States

The GALLIUM trial compared obinutuzumab (GA101, G)-based chemotherapy followed by G monotherapy (G + chemo) for up to two years to rituximab (R)-based chemotherapy followed by R monotherapy (R + chemo) for up to two years in previously untreated follicular lymphoma (FL) patients. We estimated the cost-effectiveness of G + chemo versus R + chemo utilizing GALLIUM trial data and published literature. G + chemo had increased drug costs (undiscounted: $135,200 versus $127,700 for R + chemo), representing a relative increase of 5.9%. However, this was offset by a $6,400 lower cost for disease progression. G + chemo led to increased quality-adjusted life years (QALYs) relative to R + chemo of 0.81 (95% credible range, [CR]: 0.22–1.37), and the overall discounted incremental cost was $1,900 (95% CR: −$7,400 to $8,900). The incremental cost-effectiveness ratio was ∼$2,300 per QALY gained, and the results were highly robust to sensitivity analyses. Treatment with G + chemo compared to R + chemo is cost-effective in previously untreated FL patients in the US.

Impact of Brexit on UK and EU Drug Regulation and Patient Access.

The chronology of the United Kingdom (UK) leaving the European Union (EU) is presented together with its implications for medicines regulation, including the move of the European Medicines Agency from London (UK) to Amsterdam (the Netherlands). The legal and political options for the UK and the EU are discussed, which at the time of writing (October 2018) are both uncertain. Of importance is the response of the pharmaceutical industry and the possible consequences for UK patients, including delays in access to innovative medicines and an increase in drug costs. Although there may be some possible advantages for UK medicines regulation, these will depend on the outcome of the ongoing negotiations.

Estimated Cost of Anticancer Therapy Directed by Comprehensive Genomic Profiling in a Single-Center Study.

PurposeComprehensive genomic profiling (CGP) detects several classes of genomic alterations across numerous genes simultaneously and can match more patients with genomically targeted therapies than conventional molecular profiling. The current study estimated the costs of anticancer drugs and overall survival (OS) for patients who were treated with matched and unmatched therapy.MethodsCosts were estimated for patients with complete data (188 of 500 patients) from a prospective, nonrandomized study of patients with diverse refractory cancers who underwent CGP and were treated with matched or unmatched therapy. We assessed mean time to treatment failure (TTF) and mean observed OS. Patient-specific drug and administration costs were imputed for the first regimen after CGP on the basis of drug classes, unit costs, and time on treatment.ResultsPatients on matched (n = 122) versus unmatched (n = 66) therapy had longer mean TTF (+1.5 months) and observed OS (+2.4 months) and higher drug costs (+$38,065; all P < .01). Increased drug costs were largely attributable to the longer duration of therapy associated with extended TTF (66.3%) rather than higher monthly drug costs (33.7%). Incremental increases in TTF (+1.9 months v +1.2 months) and observed OS (+2.5 months v +2.1 months) between matched and unmatched therapies were larger for those who underwent CGP in earlier- versus later-line therapy. Incremental increases in drug costs between matched and unmatched therapies were lower for earlier- compared with later-line therapy (+$27,000 v +$43,000, respectively).ConclusionMatched therapy was associated with longer TTF, increased OS, and manageable incremental cost increases compared with unmatched therapy. Most of these increased costs were a result of the longer duration of therapy rather than higher monthly drug costs. The benefits of matching were numerically greater in earlier versus later lines of therapy, which is consistent with the value of early use of CGP.

Poractant Alfa Versus Beractant for Neonatal Respiratory Distress Syndrome: A Retrospective Cost Analysis.

The objectives of this study were to determine the average medication cost per patient of poractant alfa and beractant, and to compare the outcomes of treatment with these agents. We conducted a retrospective, observational, cohort study of patients who received surfactant, before and after an institutional formulary change from beractant to poractant alfa. The primary outcome was the average medication cost per case. Secondary measures were clinical outcomes, including duration of respiratory support, length of hospital stay, and the occurrence of complications. A total of 114 patients were enrolled; 38 were treated with poractant alfa and 76 patients were treated with beractant. Baseline characteristics were similar between groups. The average medication cost per patient was $1756.44 ± $1030.06 and $1329.78 ± $705.64 for poractant alfa and beractant, respectively (p = 0.011). Patients treated with poractant alfa had a shorter length of stay (45.0 ± 30.5 days) than patients treated with beractant (65.1 ± 37.1 days) (p = 0.010). Rates of pneumothoraces, pulmonary hemorrhage, necrotizing enterocolitis, intraventricular hemorrhage, and mortality did not differ significantly between groups. We found an unanticipated increase in drug cost with poractant alfa compared to beractant.

Modelling the cost-effectiveness of pay-for-performance in primary care in the UK

BackgroundIntroduced in 2004, the United Kingdom’s (UK) Quality and Outcomes Framework (QOF) is the world’s largest primary-care pay-for-performance programme. Given some evidence of the benefits and the substantial costs associated with the QOF, it remains unclear whether the programme is cost-effective. Therefore, we assessed the cost-effectiveness of continuing versus stopping the QOF.MethodsWe developed a lifetime simulation model to estimate quality-adjusted life years (QALYs) and costs for a UK population cohort aged 40–74 years (n = 27,070,862) exposed to the QOF and for a counterfactual scenario without exposure. Based on a previous retrospective cross-country analysis using data from 1994 to 2010, we assumed the benefits of the QOF to be a change in age-adjusted mortality of −3.68 per 100,000 population (95% confidence interval –8.16 to 0.80). We used cost-effectiveness thresholds of £30,000/QALY, £20,000/QALY and £13,000/QALY to determine the optimal strategy in base-case and sensitivity analyses.ResultsIn the base-case analysis, continuing the QOF increased population-level QALYs and health-care costs yielding an incremental cost-effectiveness ratio (ICER) of £49,362/QALY. The ICER remained >£30,000/QALY in scenarios with and without non-fatal outcomes or increased drug costs, and under differing assumptions about the duration of QOF benefit following its hypothetical discontinuation. The ICER for continuing the programme fell below £30,000/QALY when QOF incentive payments were 36% lower (while preserving QOF mortality benefits), and in scenarios where the QOF resulted in substantial reductions in health-care spending or non-fatal cardiovascular disease events. Continuing the QOF was cost-effective in 18%, 3% and 0% of probabilistic sensitivity analysis iterations using thresholds of £30,000/QALY, £20,000/QALY and £13,000/QALY, respectively.ConclusionsCompared to stopping the QOF and returning all associated incentive payments to the National Health Service, continuing the QOF is not cost-effective. To improve population health efficiently, the UK should redesign the QOF or pursue alternative interventions.

PHP12 - Is the Orphan Drug Act Being used as a Loophole to Extend Periods of Drug Exclusivity?

The Orphan Drug Act of 1983 provides branded drugs with extended exclusivity up to 7 years per approved rare disease indication. There is concern that the Act may negatively impact long-term availability of affordable alternatives because some manufacturers apply for numerous orphan approvals to prevent competition for rare disease indications. Our objective was to determine the amount of additional time that approved orphan drugs have been able to maintain market exclusivity through multiple rare disease indications. We conducted a retrospective cohort analysis of FDA Orphan Drug Designations and Approvals between 1983-2017. The mean time of exclusivity gained in years per additional orphan drug approval was calculated to determine whether certain specialty drugs for rare diseases were gaining extensive amounts of exclusivity with multiple orphan approvals. A fixed-effects model regressed the extended duration of exclusivity per additional approval over time, controlling potential policy interruptions over 34 years. We identified 413 drugs approved for 615 rare disease indications in the U.S. under the Orphan Drug Act. Of these drugs, those approved for a second orphan indication had 4.7 years of average increase in market exclusivity (p<0.05) beyond the initial 7 years. The third approval gained 3.1 additional years; the fourth gained 2.7 additional years; and the fifth gained 2.9 additional years (p<0.05). Taken together, five orphan drug approvals resulted in an average of 13.4 additional years of market exclusivity across all indications. Since the beginning of the Act, exclusivity has increased in all orphan drugs over time (p<0.01). The Orphan Drug Act may be used to help manufacturers maintain branded status longer than initial periods and may contribute to increasing drug costs. Limiting the number of additional exclusivity periods a single drug can receive could increase access and affordability, and motivate competition among rare disease treatments.

Treatment decisions, clinical outcomes, and pharmacoeconomics in the treatment of patients with EGFR mutated stage III/IV NSCLC in Germany: an observational study

BackgroundWe evaluated treatment decisions and outcomes in a cohort of predominately Caucasian patients with EGFR mutation-positive (EGFR Mut+) non-small-cell lung cancer (NSCLC).MethodsREASON (NCT00997230) was a non-interventional study in German patients with stage IIIB/IV NSCLC. Secondary endpoints for EGFR Mut + NSCLC included progression-free survival (PFS), overall survival (OS), adverse event (AE) management, and pharmacoeconomic outcomes.ResultsAmong 334 patients with EGFR Mut + NSCLC, tyrosine kinase inhibitors (TKIs) were the most common first-line therapy (56.6%, 53.0% gefitinib). Among patients who received TKIs/gefitinib before first disease progression, PFS was longer compared with those who did not receive a TKI (median 10.1/10.0 vs. 7.0 months; HR 0.67/0.69; log-rank p = 0.012/p = 0.022). OS was longer for those patients who ever received a TKI/gefitinib during their complete therapy course compared with those who never received a TKI (median 18.4/18.1 vs. 13.6 months; HR 0.53/0.55; p = 0.003/p = 0.005). Total mean first-line treatment healthcare costs per person were higher for those receiving TKIs (€46,443) compared with those who received chemotherapy (€27,182). Mean outpatient and inpatient costs were highest with chemotherapy. Rash, diarrhea, and dry skin were the most commonly reported AEs for patients receiving gefitinib.ConclusionsIn REASON, TKI therapy was the most common first- and second-line treatment for EGFR Mut + NSCLC, associated with increased drug costs compared with chemotherapy. Patients who received gefitinib or a TKI ever during their complete therapy course had prolonged PFS and OS compared with patients who did not receive a TKI.Trial registrationThe trial was registered on October, 2009 with ClinicalTrials.gov: https://clinicaltrials.gov/ct2/show/NCT00997230?term=NCT00997230&rank=1

Trajectories of Injectable Cancer Drug Costs After Launch in the United States.

Purpose Cancer drug prices at launch have increased in recent years. It is unclear how individual drug prices change over time after launch and what market determinants influence these changes. We measured the price trajectories of a cohort of cancer drugs after their launch into the US market and assessed the influence of market structure on price changes. Methods We studied the changes in mean monthly costs for a cohort of 24 patented, injectable anticancer drugs that were approved by the US Food and Drug Administration between 1996 and 2012. To account for discounts and rebates, we used the average sales prices published by the Centers for Medicare and Medicaid Services. Costs were adjusted to US general and health-related inflation rates. For each drug, we calculated the cumulative and annual drug cost changes. We then used a multivariable regression model to evaluate the association between market and cost changes over time. Results With a mean follow-up period of 8 years, the mean percent change in cost for all drugs was +25% (range, -14% to +96%). After adjusting for inflation, the mean cost change was +18% (range, -16% to +59%). Rituximab and trastuzumab followed a similar pattern in cost increases over time, and the inflation-adjusted monthly costs rose since approval by 49% and 44%, respectively. New supplemental US Food and Drug Administration approvals, new off-label indications, and new competitors did not influence the annual cost change rates. Conclusion Anticancer drug costs may change substantially after launch. Regardless of competition or supplemental indications, there is a steady increase in costs of patented anticancer agents over time. New regulations may be needed to prevent additional increases in drug costs after launch.

Cost effectiveness of vildagliptin versus glimepiride as add-on treatment to metformin for the treatment of diabetes mellitus type 2 patients in Greece

ObjectivesThis study was designed to assess the cost-effectiveness of vildagliptin versus glimepiride as add-on to metformin in the management of type 2 diabetes mellitus (T2DM) patients in the Greek healthcare setting.MethodsA cost-effectiveness model was designed, using MS Excel, to compare two treatment strategies. Strategy 1 consisted of first-line metformin, followed by metformin + vildagliptin in second-line, while strategy 2 consisted of first line metformin, followed by metformin + glimepiride in second line. Subsequent lines were the same in both strategies and consisted of metformin + basal insulin and metformin + basal + rapid insulin. Clinical data and utility decrements relating to diabetes complications were taken from the published literature. Only direct medical costs were included in the analysis (cost base year 2014), and consisted of drug, adverse events and comorbidity costs (taken from local officially published sources and the literature). The perspective adopted was that of the Social Insurance Fund. The time horizon was lifetime, and future costs and outcomes were discounted at 3.5% per annum.ResultsAdding vildagliptin to metformin increased drug costs compared with adding glimepiride to metformin (€2853 vs. €2427, respectively). However, this increase was offset by a decrease in the costs of associated comorbidities (€4393 vs. €4539) and adverse events (€2757 vs. €3111), resulting in a lower total cost of €74 in strategy 1 compared with strategy 2. Comorbidities were the largest cost component in both strategies, accounting for 43.9 and 45.0% in strategies 1 and 2, respectively. Strategy 1 was also associated with increased life-years (LYs, 0.11) and quality-adjusted life-years (QALYs, 0.11) compared with strategy 2. Strategy 1 is therefore dominant, as it is associated with both lower overall costs and increased effectiveness.ConclusionsVildagliptin as add-on treatment to metformin in the management of T2DM in Greece appears to be dominant versus. glimepiride in terms of both cost per LY and cost per QALY gained.

Economic impact of the use of rifaximin 550 mg twice daily for the treatment of overt hepatic encephalopathy in Italy.

PurposeHepatic encephalopathy (HE) is associated with a reduced survival, an increased risk of hospitalization for recurrences, and a reduced health-related quality of life. The purpose of the present economic analysis was to evaluate the impact on the Italian National Health Service (INHS) expenditure of the treatment with rifaximin 550 mg twice daily (Tixteller®/Tixtar®) for the reduction of the recurrences of overt HE, with respect to the current treatment approach.Patients and methodsCosts associated with patients treated with rifaximin 550 mg twice daily were estimated considering the reduction in hospitalizations for HE recurrences revealed by registrative clinical trial (−50%) applied to the hospitalization rate (42.5%) emerging from an Italian observational real-world study; costs associated with patients not treated with rifaximin were estimated based on the hospitalization rate, resulting from the same Italian observational study. Sensitivity analyses considering possible different discount levels to INHS structures for rifaximin were performed. The INHS perspective for a period of 3 years was considered.ResultsThe treatment with rifaximin 550 mg twice daily, although increasing drug costs, is associated with a reduction in hospitalizations for HE recurrences that leads to an overall reduction of total costs charged to INHS, which could be estimated, based on the forecasted uptake of the treatment, at about €130,000 in the first year, reaching ~€260,000 in the third year. Considering a possible discount for rifaximin 550 mg to INHS structure of 20%, the total saving at the third year accounts for ~€3,000,000. Moreover, a relevant reduction in the number of hospitalizations and bed days is associated with rifaximin treatment.ConclusionThe treatment with rifaximin 550 mg twice daily, even if associated with an increase in drug expenditure, results in a reduction in total health care costs charged to INHS due to a reduction in hospitalizations for HE recurrences.

Multimodal Pain Management in Older Elective Arthroplasty Patients.

Background:Pain management after elective arthroplasty in older adults is complicated due to the risk of undertreatment of postoperative pain and potential adverse effects from analgesics, notably opioids. Using combinations of analgesics has been proposed as potentially beneficial to achieve pain control with lower opioid doses.Objective:We compared a multimodal pain protocol with a traditional one, in older elective arthroplasty patients, measuring self-rated pain, incidence of postoperative delirium, quantity and cost of opioid analgesics consumed.Methods:One hundred fifty-eight patients, 70 years and older, admitted to tertiary care for elective arthroplasty were prospectively assessed postoperative days 1–3. Patients received either traditional postoperative analgesia (acetaminophen plus opioids) or a multimodal pain protocol (acetaminophen, opioids, gabapentin, celecoxib), depending on surgeon preference. Self-rated pain, postoperative delirium, and time to achieve standby-assist ambulation were compared, as were total opioid doses and analgesic costs.Results:Despite receiving significantly more opioid analgesics (traditional: 166.4 mg morphine-equivalents; multimodal: 442 mg morphine equivalents; t = 10.64, P < .0001), there was no difference in self-rated pain, delirium, or mobility on postoperative days 1–3. Costs were significantly higher in the multimodal group (t = 9.15, P < .0001). Knee arthroplasty was associated with higher pain scores than hip arthroplasty, with no significant difference in opioid usage.Conclusion:A multimodal approach to pain control demonstrated no benefit over traditional postoperative analgesia in elective arthroplasty patients, but with significantly higher amounts of opioid consumed. This poses a potential risk regarding tolerability in frail older adults and results in increased drug costs.

Can biosimilars help achieve the goals of US health care reform?

The US Patient Protection and Affordable Care Act (ACA) aims to expand health care coverage, contain costs, and improve health care quality. Accessibility and affordability of innovative biopharmaceuticals are important to the success of the ACA. As it is substantially more difficult to manufacture them compared with small-molecule drugs, many of which have generic alternatives, biologics may increase drug costs. However, biologics offer demonstrated improvements in patient care that can reduce expensive interventions, thus lowering net health care costs. Biosimilars, which are highly similar to their reference biologics, cost less than the originators, potentially increasing access through reduced prescription drug costs while providing equivalent therapeutic results. This review evaluates 1) the progress made toward enacting health care reform since the passage of the ACA and 2) the role of biosimilars, including the potential impact of expanded biosimilar use on access, health care costs, patient management, and outcomes. Barriers to biosimilar adoption in the USA are noted, including low awareness and financial disincentives relating to reimbursement. The evaluated evidence suggests that the ACA has partly achieved some of its aims; however, the opportunity remains to transform health care to fully achieve reform. Although the future is uncertain, increased use of biosimilars in the US health care system could help achieve expanded access, control costs, and improve the quality of care.

The impact of pricing strategy on the cost of oral anti-cancer drugs during dose reductions.

e18312 Background: The pricing strategy of oral medications can affect their costs. The strategy of flat pricing per tablet may increase drug costs in the event of dose reductions requiring more tablets, as there is a single price for different tablet strengths, but the impact is largely unknown. With the strategy of linear pricing, the tablet price increases with its strength. We sought to determine the impact of pricing strategy on the cost of oral anti-cancer drugs during dose reductions. Methods: Oral anti-cancer drugs reviewed by the pan-Canadian Oncology Drug Review were identified between July 2011 to January 2015. The pricing strategy of these drugs was reviewed. We examined the percentage change in cost per mg and cost per 28 days as a result of dose reduction from dose level 0 to -1 and -2 for each drug. Results: Seventeen drugs for use in 20 indications were included in the analysis. There were 3 drugs for hematological malignancies and 14 drugs for solid cancers. Fifty-nine percent (10/17) of these drugs were available in multiple strengths; five of them utilized fixed pricing per tablet strategy and the other 5 utilized linear pricing. The remaining drugs (7/17) were available in a single strength tablet. Dose reductions generally increased the cost per mg for drugs using flat pricing per tablet, with a mean increase of 82% (range: 25%-200%) at dose level -1 and 100% (range: 0%-200%) at dose level -2. Dose reduction had no effect on the cost per mg of drug for drugs using linear pricing apart from lenalidomide, which had increased costs due to minimal price variation between the highest and lowest tablet strengths. In general, dose reduction did not decrease the cost per 28 days of drug for drugs using flat pricing per tablet, but was proportionally reduced in drugs using linear pricing. Conclusions: While there is a general expectation that the cost of drugs should decrease with dose reduction, oral anti-cancer drugs using flat pricing per tablet have increased cost per mg and no decrease in cost per 28 days despite dose reduction. Future economic evaluations should account for the impact of dose reductions for oral drugs using the flat pricing per tablet strategy on cost-effectiveness and budget.

Impact of intravenous cancer drug wastage on economic evaluations.

6607 Background: Intravenous drugs administered through body-surface area (BSA) or weight-based dosing may cause wastage due to large and/or limited fixed vial sizes, and vial sharing restrictions. Drug wastage leads to incremental costs without incremental value to patients. Bach et al. (2016) estimated 10% of revenue ($1.8 billion) from cancer drugs would result from wastage in 2016. The pan-Canadian Oncology Drug Review (pCODR) committee provides recommendations on which drugs to publicly reimburse by reviewing clinical and economic evidence. There is considerable potential that drug wastage could impact the economic evaluations. We sought to determine the impact of modeling cancer drug wastage on the results of economic evaluations. Methods: Economic evaluations submitted by drug manufacturers and reviewed by the pCODR Economic Guidance Panel (EGP) were assessed for frequency of wastage reporting and modeling. Cost-effectiveness analyses and budget impact analyses were conducted for scenarios in which “no wastage” and “wastage” of drugs occurred. Sensitivity analyses were performed to determine the effects of BSA and weight variation. Results: 12 drugs for use in 17 indications were analyzed. Wastage was reported in 71% and incorporated in 53% of manufacturer’s models, resulting in a mean incremental cost-effectiveness ratio (ICER) increase of 6.1% (range: 1.3% to 14.6%). EGP reported and incorporated wastage for 59% of models, resulting in a mean ICER increase of 15.0% (2.6% to 48.2%). When maximum wastage (i.e. the entire unused portion of each vial is discarded) was incorporated in our independent analysis, the mean ICER increased by 24.0% (0.0% to 97.2%) and the mean 3-year total incremental costs increased by 26.0% (0.0% to 83.1%). Over a 3-year period, wastage can increase the total incremental drug budget cost by CAD $102 million nationally. Changing the mean BSA or body weight caused 45% of the drugs to use a different vial size (if available) and/or quantity, resulting in further increased drug costs. Conclusions: Wastage can have an under-recognized and significant impact on economic evaluations of intravenous chemotherapy drugs. Guidelines are needed to promote uniform and optimal modeling of drug wastage in economic evaluations.

Estimated cost of anticancer therapy directed by comprehensive genomic profiling (CGP) in a single-center study.

6605 Background: Accumulating evidence supports the clinical benefit of targeted therapies matched to cancer patients based on genomic alterations. CGP, which detects all classes of alterations (base pair substitutions, copy number, insertions/deletions, and rearrangements), can match more patients with available and investigational therapies. This study estimated anti-cancer drug costs and overall survival (OS) for matched vs. unmatched therapy. Methods: Costs were estimated for patients with complete data (N = 188/500) from a prospective, nonrandomized, phase I oncology center study of patients with diverse refractory cancers who underwent CGP and were treated with matched or unmatched therapy (PMID: 27197177). Average time to treatment failure and average OS were assessed during the observation period. Patient-specific drug and administration costs were imputed for the first regimen after CGP based on drug classes, unit costs, and times to treatment failure. Results: Patients onmatched (N = 122) vs. unmatched (N = 66) therapy had, on average, longer time on treatment (+1.5 mos), longer observed survival (+2.4 mos), and higher anti-cancer drug costs (+$38K) (all p &lt; 0.01); 66% of increased drug costs were attributable to longer time on treatment as opposed to higher monthly drug costs. Combination therapy was used for 71% of matched and 53% of unmatched patients. Those undergoing CGP in earlier-line (1-3; N = 58) vs. later-line (4+; N = 130) therapy had numerically larger incremental increases in average times on treatment (+1.9 vs. +1.2 mos) and survival (+2.5 vs. +2.1 mos), and numerically lower incremental drug costs (+$27K vs. +$43K), with matched vs. unmatched therapy. Conclusions: For patients cared for in a phase I clinic, matched vs. unmatched therapy was associated with longer treatment durations, longer survival times, and manageable incremental costs. Despite frequent use of combination therapy, most of the increased costs of matched therapy were due to longer treatment times rather than higher monthly drug costs. Benefits of matching were numerically greater in earlier- vs. later-lines, consistent with the value of earlier-line use of CGP to guide treatment.

Improving access to high-cost cancer drugs in Latin America: Much to be done.

Lack of access to high-cost medications is a complex issue at the intersection of economics, medicine, politics, and ethics, and it poses a significant threat to global health care. The problem is even more significant in low- and middle-income countries, such as those in Latin America, where governments and individuals struggle to pay for products that are priced at several times the level of their per capita gross domestic product. In this review, we examine the determinants for increasing drug costs and how Latin American countries face this burgeoning crisis. We emphasize that a number of opportunities and strategies to reduce costs and improve access exist and should be identified and implemented, ideally within a regional approach with multiple stakeholders involved and based on systematic and transparent cost-effectiveness analyses. Cancer 2017;123:1313-1323. © 2016 American Cancer Society.

Increasing drug costs are challenging hospital pharmacy budgets

Increasing drug costs are challenging hospital pharmacy budgets

Cost and cost-effectiveness of tuberculosis treatment shortening: a model-based analysis.

BackgroundDespite improvements in treatment success rates for tuberculosis (TB), current six-month regimen duration remains a challenge for many National TB Programmes, health systems, and patients. There is increasing investment in the development of shortened regimens with a number of candidates in phase 3 trials.MethodsWe developed an individual-based decision analytic model to assess the cost-effectiveness of a hypothetical four-month regimen for first-line treatment of TB, assuming non-inferiority to current regimens of six-month duration. The model was populated using extensive, empirically-collected data to estimate the economic impact on both health systems and patients of regimen shortening for first-line TB treatment in South Africa, Brazil, Bangladesh, and Tanzania. We explicitly considered ‘real world’ constraints such as sub-optimal guideline adherence.ResultsFrom a societal perspective, a shortened regimen, priced at USD1 per day, could be a cost-saving option in South Africa, Brazil, and Tanzania, but would not be cost-effective in Bangladesh when compared to one gross domestic product (GDP) per capita. Incorporating ‘real world’ constraints reduces cost-effectiveness. Patient-incurred costs could be reduced in all settings. From a health service perspective, increased drug costs need to be balanced against decreased delivery costs. The new regimen would remain a cost-effective option, when compared to each countries’ GDP per capita, even if new drugs cost up to USD7.5 and USD53.8 per day in South Africa and Brazil; this threshold was above USD1 in Tanzania and under USD1 in Bangladesh.ConclusionReducing the duration of first-line TB treatment has the potential for substantial economic gains from a patient perspective. The potential economic gains for health services may also be important, but will be context-specific and dependent on the appropriate pricing of any new regimen.Electronic supplementary materialThe online version of this article (doi:10.1186/s12879-016-2064-3) contains supplementary material, which is available to authorized users.