Use Of High-cost Drugs Research Articles

Restrictions on the use of higher cost drugs for rheumatoid arthritis in England: Surveys of Clinical Commissioning Groups; prescribers.

The authors declare no conflicts of interest. The data that support the findings of this study are available from the corresponding author upon reasonable request.

Trends in Use of High-Cost Antihyperglycemic Drugs Among US Adults with Type 2 Diabetes.

Some antihyperglycemic drugs can reduce cardiovascular events, slow the progression of kidney disease, and prevent death, but they are more expensive than older drugs. (1) To estimate trends in use of antihyperglycemic drugs by cost; (2) to examine use of high-cost drugs by race/ethnicity, income, and insurance status DESIGN: Cross-sectional analysis of the 2003-2018 National Health and Nutrition Examination Survey PARTICIPANTS: US adults ≥18 years with type 2 diabetes EXPOSURES: Race/ethnicity, income, and insurance status MAIN MEASURES: Low-cost noninsulin medications included any drugs that had at least one generic version approved by the Food and Drug Administration. Human regular, NPH, and premixed NPH/regular 70/30 insulins were classified as low-cost. All other noninsulin medications and insulins were considered high-cost KEY RESULTS: The sample included 7,394 patients. Prevalence of use of low-cost noninsulin drugs increased from 37% in 2003-2004 to 52% in 2017-2018. Use of high-cost noninsulin drugs decreased from 2003-2004 to 2013-2014 and then slowly increased. Use of low-cost insulin decreased from 7 to 2% while high-cost insulin rose from 4 to 16%. In multivariable analysis, non-White patients had 25-35% lower odds of receiving high-cost drugs than non-Hispanic Whites. Health insurance was associated with more than twice the odds of having high-cost drugs compared to no insurance. Patients with higher HbA1c or moderate obesity were also more likely to use high-cost drugs. Sex, income, and insurance type were not associated with receipt of high-cost drugs. There was a shift in utilization from high- to low-cost noninsulin drugs, but since 2013-2014 the trend has slowly reversed with increased use of newer, more expensive drug classes. High-cost insulin analogs have almost completely replaced lower cost human insulins. Disparities in receipt of diabetes drugs by race/ethnicity and insurance must be addressed to ensure that cost is not a barrier for disadvantaged populations.

P287 The impact of restricted access to targeted therapies for rheumatoid arthritis on rheumatologists and the service they can provide

Abstract Background/Aims Just under half of the clinical commissioning groups (CCGs) in England restrict the number of targeted therapies that can be prescribed for rheumatoid arthritis (RA) before an individual funding request (IFR) is required. We were interested to explore the impact of this on rheumatology services and rheumatologists themselves. Methods A national survey of rheumatologists who prescribe high-cost drugs was developed to explore prescribing restrictions by CCGs, the impacts on the service and the strategies adopted to mitigate this. Results from the survey were then used to develop an interview schedule for semi structured interviews with five rheumatologists to explore this in more detail. Results A total of 53 rheumatologists started the survey. 23 participants (44%) were restricted in their use of high-cost drugs. Restrictions were mainly by total number rather than mode of action. For 46, (88%) restrictions were communicated through local guidelines. 41% had additional local restrictions either through their pharmacy (5%), local pathways (10%) or a multi-disciplinary team meeting (MDT; 27%). 51% routinely discussed high-cost drugs with colleagues, with 21% discussing only complicated patients. The effect on the service was rated: a lot = 43%; moderately =24%; and a little or not = 7%. 95% said it affected their practice e.g., persisting with ineffective therapy. Blueteq was used by 69%; 29% were negative or very negative about it, 29% were neutral, and 23% were positive about it. 19% had it completed by someone else. Strategies for mitigating the restrictions included: 43% of participants had used a foot instead of a hand in the DAS28; 68% had changed diagnostic category to justify treatment; 51% had negotiated a local pathway. Three themes emerged from the semi-structured interviews. Theme 1) Persistence with partially effective therapy was used to avoid running out of options. There was genuine distress about the poor service that was being delivered. Theme 2) IFRs do not work but negotiating alternative pathways with the CCG can. Theme 3) Bluetec is an extra burden in the process but is OK if you do not have to fill it in yourself. Conclusion Restricting the number of targeted therapies that can be prescribed for RA has a detrimental effect on the service and the prescribers involved which often results in persistence with partially effective therapies. Continuing partially effective therapies is not satisfactory if there may be more effective therapies available. Acknowledgements: On behalf of the EVA-RA Joint Working Group. Disclosure D. Walker: Consultancies; Galapagos, Eli Lilly. S. Robinson: None.

Comparison of claims from high-drug cost beneficiaries in Ontario, Canada, and Australia: a cross-sectional analysis.

Background:Globally, payers are struggling with rising drug costs, driven primarily by the increasing number of high-cost medications used by their beneficiaries. We aimed to compare the annual drug spending on claims from high–drug cost beneficiaries in the province of Ontario, Canada, and Australia.Methods:We conducted a cross-sectional analysis of public drug claims in Ontario and Australia from fiscal years 2006 to 2017. We identified the total government costs for prescribed medications per beneficiary. During the study period, public drug coverage in Ontario was provided to all residents 65 years of age and older, those with financial needs, and those living in long-term care or in need of home care. Australia maintains a publicly funded, universal system covering all citizens. Based on annual spending, we divided beneficiaries into 4 cost groups, representing the top 1%, top 5%, top 10% and the remaining 90%. We reported the following for each cost group: medication cost and proportion of total government spending, number of unique drugs dispensed per person and the top 10 most costly drug classes.Results:In Ontario and Australia, the top 1% of beneficiaries accounted for a large and increasing proportion of all government drug costs, growing from 12% ($405 946 197) to 24% ($1 345 977 248) in Ontario, and from 14% ($86 565 586) to 34% ($416 097 984) in Australia between 2006 and 2017. The most costly drug classes among high-drug cost beneficiaries in both jurisdictions were biologics and hepatitis C treatments.Interpretation:In both Ontario and Australia, a small number of beneficiaries accounted for a large proportion of public drug spending, driven largely by the use of expensive medications. The current development of potential national pharmacare strategies in Canada must optimize the use of high-cost drugs to ensure the sustainability of the program.

The current situation regarding the availability and accessibility of anticancer drugs for breast cancer in the Peruvian public health systems.

The availability of effective, accessible, safe and high-quality anticancer drugs for the medical treatment of cancer is fundamental in ensuring optimal healthcare in the public health system in Peru. The main objective is to assess the current situation regarding anticancer drugs (termed ‘high-cost’) for breast cancer, as well as an analysis of the possible factors which negatively impact access to the Peruvian public health systems.There are similarities in the availability of anticancer drugs, since most treatments are covered by the Peruvian Ministry of Health. EsSalud offers an extra monoclonal antibody (pertuzumab) in the metastatic treatment stage. Meanwhile, the National Institute of Neoplastic Diseases (INEN), mainly for metastatic disease, has relied on more tested treatments over the last year. An agreement has been reached with the armed forces, which will enable patients to receive oncology care at the INEN and, thereby, benefit from the use of high-cost drugs.

P142 Restriction of use of high cost drugs for the treatment of rheumatoid arthritis by CCGs in England

Abstract Background/Aims It became apparent at a session at the 2019 BSR Annual Conferene that some CCGs were restricting the number of high cost drugs (HCDs) that were permitted to be prescribed for an individual with rheumatoid arthritis. Further HCDs could only then be prescribed by utilising an Individual Funding Request. We were interested to explore how common this was and what the restrictions were in different areas. Methods The websites of all CCGs in England were scrutinised between March and July 2020 for local guidelines for the use of HCDs in patients with rheumatoid arthritis (RA). The results were tabulated and mapped. Results Of 134 CCGs in England, 69 (51%) had no expressed restriction on numbers of HCDs provided that NICE thresholds were observed. 10 (7%) had a pathway limiting the drugs to 6, 49 (33%) restricted to 4 and a further 9 (7%) restricted to 3. Dates of guidance implementation varied from 07/2017 to 07/2020 with the vast majority being 2018 and 2019. Geographically, the restrictions were not uniform. The CCGs restricting to 3 HCDs were all in London except for one, Oxford. Those restricting to 4 were mainly from the South East, North West and East. Those restricting to 6 HCDs were in the Liverpool area. Largely unrestricted areas were the North East, the South West, the Midlands and the majority of Yorkshire. The majority of CCGs gave no explanation for the restriction of HCDs. Where stated, the reason given was that it had not been shown to be cost effective. Conclusion Restricting the number of HCDs may deprive some patients of effective drugs that have been NICE approved contrary to NICE guidance. There are currently 5 different modes of action (MOA) of HCDs (TNFi, B cell depletion, IL6i, CTLA4 and JAKi) for the treatment of moderately or severely active RA, therefore in some areas not even all MOAs may have been tried. There is no reliable way of predicting a patient's response to an individual drug, so it is necessary to trial and observe. Non-responders will require more treatment changes. Many patients have secondary failure of drugs after initial good response, so trying other drugs with the same MOA is a logical progression. Over a long disease course, patients will need many different interventions and access to the widest possible range is important. Whilst there are lower response rates for HCDs in biologic inadequate response phase III studies, responders can be observed after only 3 months of treatment. Restricting the number of drug choices is also likely to affect clinical practice, as less effective drugs could be continued unnecessarily for fear of running out of choices. Disclosure D. Walker: Honoraria; Gilead Sciences Ltd, Ely Lilly Pharmaceuticals, Pfizer Pharmaceuticals. Grants/research support; Gilead Sciences Ltd. S. Robinson: None. J. Barry: Corporate appointments; Gilead Sciences Ltd. P. Punter: Corporate appointments; Gilead Sciences Ltd. S. Kearns: Corporate appointments; Gilead Sciences Ltd.

P091 Can use of high cost drugs for rare rheumatic diseases be reliably identified within routinely collected NHS data? Results from a pilot study of rituximab use in vasculitis using data from the National Disease Registration Service (NDRS) and the Registration of Complex Rare Diseases - Exemplars in Rheumatology (RECORDER) project

Abstract Background/Aims Understanding real-world usage of high cost drugs and subsequent outcomes are crucial to support high quality care, adoption of innovation, and reduce unwarranted variation in treatment. Hospital Episode Statistics (HES) contain diagnoses (coded by ICD-10) and procedures/treatment (coded by OPCS) for admissions in England. However, OPCS codes are not specific for individual drugs, for example X921 (cytokine inhibitors band 1) includes rituximab and 15 other drugs. We aimed to validate the accurate identification of patients treated with rituximab for ANCA-associated vasculitis (AAV) using HES data. Methods We used data available to the National Congenital Anomaly and Rare Disease Registration Service (NCARDRS), part of NDRS at Public Health England and their legal permissions (CAG 10-02(d)/2015). We extracted from HES all patients treated at one hospital who ever had a coded diagnosis of either granulomatosis with polyangiitis, eosinophilic granulomatosis with polyangiitis, microscopic polyangiitis, polyarteritis nodosa or 'arteritis unspecified’, which is applied by coders to several clinical terms including “systemic vasculitis” and “ANCA associated vasculitis”. Enabled by data sharing agreements, we reviewed hospital records of those patients who had a Finished Consultant Episode (FCE) during 2018/19, to validate diagnoses and whether X921 reliably identified rituximab. Results Initially using codes only for granulomatosis with polyangiitis, eosinophilic granulomatosis with polyangiitis, or microscopic polyangiitis we identified 65 people, 60 of whom had AAV confirmed in their medical notes (Positive predictive value (PPV) 92.3%; 95% CI 83.0-97.5). When we expanded our search to include in addition people with codes for polyarteritis nodosa or ‘arteritis unspecified’ who had rheumatology or renal follow up, we identified an additional 13 people with AAV and 10 who had other types of systemic vasculitis (PPV for AAV 79.5, 95% CI 69.6-87.4; PPV for systemic vasculitis 90.9%, 95% CI 82.9-96.0). Among patients identified only by the specific ICD-10 codes for the 3 subtypes of AAV there were 51 episodes coded as X921, of which 50 correctly identified a rituximab infusion (and 1 was tocilizumab). No additional infusions for these patients were missed by the coding. The PPV was 98.0% (95% CI 89.6-100). Among patients identified by the more inclusive algorithm, there were 61 episodes coded as X921 in that year. Of these, 59 were for rituximab treatment for AAV and an additional 4 rituximab infusions occurred but were not coded. The PPV was 96.7% (95% CI 88.7-99.6), and the sensitivity was 93.7% (95% CI 84.5-98.2). Conclusion This pilot study demonstrates how the use of novel algorithms within the NDRS RECORDER project can enable the identification and registration of people with AAV and their high-cost treatment at whole-population level. Further work is now underway to study this national cohort, and to apply this methodology to other rare autoimmune diseases and high cost drugs. Disclosure M. Chakravorty: None. F. Pearce: Grants/research support; Recipient of research grant from Vifor Phama. Vifor Pharma had no influence on the design, interpretation or reporting of this work. M. Rutter: None. P. Lanyon: Grants/research support; Recipient of research grant from Vifor Phama. Vifor Pharma had no influence on the design, interpretation or reporting of this work. J. Aston: None. M. Bythell: None. S. Stevens: None.

Payment incentives and the use of higher-cost drugs: a retrospective cohort analysis of intravenous iron in the Medicare population.

Medicare Part B payment methods incentivize the use of more expensive injectable and infused drugs. We examined prescribing patterns in the context of intravenous (IV) iron, for which multiple similarly safe and efficacious formulations exist, with wide variations in price. We conducted a retrospective cohort analysis of IV iron utilization and payment in the Medicare population between 2015 and 2017. This analysis used a national, random 20% sample of Medicare fee-for-service beneficiaries with Part B claims for IV iron between January 2015 and December 2017-a period before, during, and after a national shortage of iron dextran. This sample included 66,710 Medicare fee-for-service beneficiaries with at least 1 Part B claim for IV iron. The greatest increase in utilization occurred in the most expensive iron formulation, ferric carboxymaltose; its market share rose from 27.4% of use in 2015 to 47.7% in 2017. The use of a less expensive formulation, iron dextran, decreased from 26.7% to 18.7% over the same period. An alternative payment model in Maryland hospitals was associated with markedly less utilization of ferric carboxymaltose, accounting for 4.7% of IV iron utilization in Maryland hospitals. There was an increase in the dispensing of a higher-priced IV iron formulation associated with a shortage of a less expensive drug that persisted once the shortage ended. These findings in IV iron have broader implications for Part B drug payment policy because the price of the drug determines the physician and health system payment.

1604P Aggregated analysis of treatment and survival outcome of all metastatic renal cell carcinoma patients treated from 2014 to 2018 in the Veneto region, Italy

Monitoring the use of high cost drugs at a population level is becoming increasingly relevant for regional Healthcare systems in order to appropriately program the allocation of resources and conduct budget impact analyses whenever a novel drug is approved. We planned a retrospective analysis of all prescriptions of high cost drugs to mRCC pts in the Veneto Region of Italy, years 2014-2018. Aggregated data of type of drug, line of treatment, treatment duration-TD, OS and costs were extracted from Veneto Healthcare database covering 4.9 million people. Average monthly cost of therapy was calculated dividing the cost of all drug resupplies dispensed to each patient by TD in mo of the same treatment. Managed entry agreements and clinical trials were not considered. We identified 1135 pts who started at least one line of systemic treatment for mRCC, and a total of 1665 treatments: 58% in first line, 26% second, 12% third, 4% fourth or further line. First-line pazopanib and sunitinib had a median DT of 10.5 and 9.7 mo (32.3% censored, p=0.51), and median OS of 28.6 and 26.2 mo (54.6% censored, p=0.61), respectively. Average monthly cost was estimated to be 3,583 and 2,679 euros for sunitinib and pazopanib, respectively (p<0.0001) (Table). Nivolumab and cabozantinib had a longer DT both in second line (compared to axitinib, everolimus and sorafenib (p<0.0001)) and third line (compared to everolimus or sorafenib, (p<0.0001)), although medians were not reached with all drugs (Table). Cost of second or third line treatment per patient was significantly higher for Nivolumab compared to all other drugs, p<0.0001 (Table). Our pupulation-based analysis confirms that first line pazopanib and sunitinib have comparable DT and OS, although cost of sunitinib without managed entry agreements is higher. Nivolumab and cabozantinib have superior DT both in second and third line setting, with nivolumab being the most expensive drug.Table 1604PDT, OS and average monthly cost of drugs per patient (NR=not reached)LineNDT (mo)pOS (mo)Pcost (euros)pFIRSTPazopanibSunitinib34461510.5 (8.7-12.9)9.7 (8.4-12.0)0.512.9.0 (21.7-35.3)26.8 (21.4-33.4)0.6126793583<0.0001SECONDAxitinibCabozantinibEverolimusNivolumabSorafenib11734123118285.9 (3.9-7.2)NR4.5 (3.1-5.0)8.7 (5.2-13.7)2.8 (1.6-3.7)<0.000113.8 (10.8-24.6)NR12.2 (9.0-15.6)NR12.5 (5.4-19.4)<0.00013.0442860335755593261<0.0001THIRDCabozantinibEverolimusNivolumabSorafenib30585261NR3.8 (2.9-4.5)4.8 (2.8-10.0)3.0 (2.2-5.6)<0.0001NR8.3 (6.9-14.3)NR8.9 (5.7-12.2)<0.00012939360253482920<0.0001 Open table in a new tab

O10 Combining clinical and ultrasound assessment to taper biologic therapies in patients with RA in routine clinical practice

Abstract Background There is growing evidence for tapering biologic therapies in patients with rheumatoid arthritis in sustained clinical remission to avoid overtreatment and minimise side-effects. Ultrasound assessment for subclinical synovitis adds to clinical assessment of patients with rheumatoid arthritis suitable for tapering biologic therapies. Our primary objective was to combine clinical and ultrasound assessment to select patients with rheumatoid arthritis for tapering biologic therapies in routine clinical practice. The secondary objectives were to identify predictors for successful tapering and assess the cost savings to the local health economy by optimising the use of high cost drugs. Methods All patients with rheumatoid arthritis on a biologic therapy for 2 years and in sustained clinical remission (DAS28≤2.6) over the previous year were seen in the remission clinic. They had an Ultrasound scan of the small joints of the hands, wrists and other symptomatic joints. Patients with no activity on Power Doppler were advised to lengthen the interval of their biologic therapy gradually and were followed once every 3 months. Patients were not on oral steroids but continued conventional DMARDs. Patients had a dedicated helpline if they had a flare. Results Ninety-three of the 120 patients with rheumatoid arthritis on biologic therapy seen in the biologic remission clinic between January and October 2019 were eligible and all but one agreed to taper. They were 70% female with a mean age of 62.8 years and mean duration of disease 14.6 years. Their mean duration of biologic therapy was 6.3 years; mean baseline DAS28 was 6.3 pre-biologic therapy and 1.7 before tapering. Fifty-seven of the patients were on a TNF inhibitor and 35 were on other biologic therapies. Forty of the ninety-two patients were co-prescribed DMARDs. Screening failure was due to clinical activity in 13 patients, Ultrasound Power Doppler activity in 23 patients, interstitial lung disease in 2 patients and shoulder surgery in one. Only two of the 40 patients who had completed 6 months had a flare and reverted to the baseline frequency. Of the remaining 52 patients, 22 patients had completed 3 months at the tapered dose and 3 patients who were in the initial 3 months had a flare and reverted to the baseline frequency. Initial drug-cost savings at 6 months was approximately £45,000. Conclusion Tapering of biologic therapies in patients with rheumatoid arthritis is feasible in routine clinical practice. Ultrasound is helpful to stratify patients for biologic tapering and has enabled a higher proportion of patients to remain in remission after tapering. Disclosures H. Pillai: None. N. Nolkha: None. A. Yau: None. S. Matthews: None. A. Hall: None. G. Hirsch: None. S. Venkatachalam: None.

P13 An analysis of multidisciplinary care in systemic auto-immune rheumatic disease in a single centre

Abstract Background Auto-immune rheumatic diseases are multisystem conditions with high morbidity and mortality, where co-ordinated multidisciplinary (MDT) care is necessary to ensure effective and timely treatment. An NHS England Commissioning for Quality and Innovation was implemented in 2016 to support development of MDTs to achieve earlier diagnosis and intervention and standardise quality of care and access to high-cost drugs. Methods A regional network for MDTs was established in one centre, led by Rheumatology with input from other specialities. MDT outcomes are recorded in a bespoke database. We analysed MDT outcomes over a two-year period from September 2017 to August 2019 for the following: disease activity assessments, investigations requested, referrals to other specialists, treatment changes and the reasons for these. Results During this period, 2,750 MDT discussions were recorded for 1,270 patients. The most common diagnoses were giant cell arteritis (30%), systemic lupus erythematosus (9%) and granulomatosis with polyangiitis (6%). Disease activity scores were recorded on 2,744 (99.8%) occasions with BVAS (n = 1,658, mean 0.82, range 0-21) and SLEDAI (n = 379, mean 2.04, range 0-20) used most. This underlines that decisions were consistently based on validated outcome measures. Investigations were ordered on 1165 (42%) occasions. Imaging was requested in 1,124 cases, biopsy in 37, functional tests in 221 and other tests in 18. Ultrasound was requested most often (614). Onward referrals were made in 237 (9%), most frequently to respiratory (32) and ENT (32). On 1550 occasions (56%) a treatment change was made: 1049 (38%) had a new change and 501 (18%) had a previously planned change. The most common reasons were good response to therapy (354 [23%]), inefficacy (345 [22%]) or starting additional therapy (214 [14%]). This shows that a significant number of treatment changes were approved by the MDT, both for treatment reduction as well as escalation. A biologic was added on 65 occasions (4% of treatment changes), most commonly in GCA (8 started on tocilizumab), SLE (5) and GPA (5). Biologics were repeated in 121 (8%) and stopped in 25 (2%). Where biologics were added, mean pre-treatment BVAS was 4.7 and post-treatment was 0.6. Mean reduction in BVAS after treatment was 5.2. The equivalent mean scores for SLEDAI were 7 (pre-treatment), 2 (post-treatment) and 7.7 (reduction). On average, the addition of biologics resulted in reductions in disease activity scores, which provides justification for their use. Conclusion Our data suggest that the large number of MDT reviews over this two-year period contributed significantly to management of systemic auto-immune rheumatic diseases by approving investigations, referrals and treatment changes. This facilitated timely intervention and avoidance of overtreatment. The auditable recording of these reviews and disease activity scores ensured decisions were guided by objective assessments and the use of high-cost drugs was standardised and justifiable. Disclosures K. Sandhu None. N. Ahmad None. A. Verdiyeva None. R. Luqmani None.

Trends in Diabetes Treatment and Monitoring among Medicare Beneficiaries.

Diabetes is a costly and common condition, but little is known about recent trends in diabetes management among Medicare beneficiaries. To evaluate the use of diabetes medications and testing supplies among Medicare beneficiaries. Retrospective cohort analysis of Medicare claims from 2007 to 2014. Traditional Medicare beneficiaries with a diagnosis of diabetes in the current or any prior year. We analyzed choices of first diabetes medication for those new to medication and patterns of adding medications. We also examined the use of testing supplies, use of statins and ACE inhibitors/angiotensin receptor blockers, and spending. Diagnosed diabetes increased from 28.7% to 30.2% of beneficiaries from 2007 to 2014. The use of metformin as the most commonly prescribed first medication increased from 50.2% in 2007 to 70.2% in 2014, whereas long-acting sulfonylureas decreased from 16.6% to 8.2%. The use of thiazolidinediones fell considerably, while the use of new diabetes medication classes increased. Among patients prescribed insulin, long-acting insulin as the first choice increased substantially, from 38.9% to 56.8%, but short-acting or combination regimens remained common, particularly among older or sicker beneficiaries. Prescriptions of testing supplies for more than once-daily testing were also common. The mean total cost of diabetes medications per patient increased over the period due to the increasing use of high-cost drugs, particularly by those patients with costs above the 90th percentile of spending, although the median costs decreased for both medications and testing supplies. The use of metformin and long-acting insulin have increased substantially among elderly Medicare patients with diabetes, but a substantial subgroup continues to receive costly and complex treatment regimens.

Growth Hormone Utilization Review in a Pediatric Primary Care Setting.

Objective:One of the main problems facing public health providers and administrators in many countries is ensuring the rational use of high-cost drugs. In this regard, on-going process of medication use evaluation can be considered as a useful tool. In this study, we evaluated certain usage aspects of a highly-cost medication, that is, recombinant growth hormone (GH).Methods:This cross-sectional study conducted from August 2012 to August 2014. Children receiving GH ± gonadotropin releasing hormone (GnRH) analogs were included in the study. A researcher-designed checklist was developed to evaluate the GH utilization in these patients. Baseline demographic characteristics and background clinical and growth data, as well as any aspects of drug therapy including indications, dosing, monitoring, and discontinuation were collected from the patients' medical records.Findings:Seventy children receiving GH entered the study, of which 23 patients (32.85%) received GH and GnRH analogs simultaneously. At the baseline, 67 children (95.7%) had GH stimulation test, whereas serum insulin-like growth factor-1 (IGF-1) levels were measured in 63 (90%) patients. Sixty-seven patients (95.71%) had thyroid function test, whereas bone age was determined in 68 children (97.14%). The mean ± standard deviation of GH dose for idiopathic short stature, GH deficiency, Turner's syndrome and born small for gestational age in our study was 0.22 ± 0.025 mg/kg/week, 0.23 ± 0.04 mg/kg/week, 0.22 ± 0.015 mg/kg/week, and 0.23 ± 0.02 mg/kg/week, respectively. Height and weight of all patients were followed every 3–6 months, regularly. Thirty patients were treated with GH for at least 1 year, of which thyroid hormones and IGF-1 levels were measured annually in 25 (83.33%) and 26 (86.66%) patients, respectively; while bone age was evaluated in 13 (43.33%) children, annually. GH treatment was discontinued in 15 patients (21.42%), while financial problem was the major reason.Conclusion:Diagnostic tests and monitoring of height, weight, IGF-1 level and thyroid function was properly performed in this setting. However, a number of patients with ISS and Turner's syndrome were under-dosed.

S60 - Libro Blu: a tool to coordinate Oncology practice and to rationalise resource employment

Background: AOVV was born in 2003, covering a huge area with 4 hospitals, each working autonomously until then. The reform of Lombardy public health in 2015 changed the AOVV in ASST-VAL, including one more hospital. Such a broad area, with the conglomeration of different experiences, brought a need for harmonization of therapeutic choices to ensure all patients the same standard of care. Moreover, the increasing appearance of high cost drugs and the simultaneous restrictions in public health budget resulted in a need for a more rational allocation of expense. In this context, we took inspiration, and used its name, from “Libro Blu”, a tool previously adopted by IOSI. Material and methods: Our effort to coordinate Oncology practice resulted in the first edition of our “Libro Blu” in 2012. After periodic updates, in 2015 a major revision took place. Besides the updates in care of specific cancers, carried out by Oncology, Hematology and Radiation Therapy specialists, much attention has been paid to supportive care, involving consultants from Internal Medicine, Geriatrics, Endocrinology, Cardiology, Palliative Care and Psycho-Oncology. Recommendations in “Libro Blu” derive from Italian and international guidelines; after a thorough discussion in multidisciplinary meetings, they have been adapted to the specific needs of our local reality and approved after a final revision. Results: The implementation of “Libro Blu” resulted in a more consistent application of treatment protocols in our institution, ensuring all residents the same standard of care; notably, the facilitation of multidisciplinary discussions during this process allowed to raise the level of our cancer care, focusing both on treatment of the disease and on patients' needs in supportive care. Furthermore, establishing institutional guidelines for a rational use of high cost drugs allowed to reduce costs by about 400000 €/year. Conclusions: “Libro Blu” has allowed to establish a homogeneous standard of care in a broad area in which health professionals were previously working autonomously. The cost reduction deriving from our guidelines is particularly relevant in this moment, with Oncology facing the appearance of many high cost drugs in the context of a spending review that also involves the Italian health system. We think that “Libro Blu”, with the implementation of guidelines adapted to the specific needs of a local institution, could be reproduced in fields other than Oncology and in other local realities.

A clinical audit of high-cost and off-label drug use in dermatology.

The use of high-cost, off-label, unsubsidised drugs has become valuable in the management of dermatology patients with challenging conditions unresponsive to conventional therapy. Currently, there is no dedicated funding and a paucity of evidence for such drugs. The aim of this audit was to review outcomes and costs. A retrospective clinical audit of high-cost off-label dermatology drug applications to the High Cost Drug's Subcommittee was undertaken at a tertiary public hospital in Brisbane, Queensland, between 2002 and 2013. Of 28 applications, 25 were approved. The medications included thalidomide, mycophenolate mofetil, cyclosporin, infliximab, rituximab and adalimumab. Over 70% of patients responded to treatment. Individual annual costs for these medications ranged from $2068 to $36 984. Adverse effects resulted in drug withdrawal in five cases. Despite the absence of dedicated funding for high-cost drugs and a rigorous committee approval process, most applications were approved, of which >70% benefited from treatment. This audit provides useful clinical experience with off-label use of high-cost drugs in difficult dermatological conditions.

Predicción del riesgo individual de alto coste sanitario para la identificación de pacientes crónicos complejos

ObjectiveTo develop a predictive model for the risk of high consumption of healthcare resources, and assess the ability of the model to identify complex chronic patients. MethodsA cross-sectional study was performed within a healthcare management organization by using individual data from 2 consecutive years (88,795 people). The dependent variable consisted of healthcare costs above the 95th percentile (P95), including all services provided by the organization and pharmaceutical consumption outside of the institution. The predictive variables were age, sex, morbidity—based on clinical risk groups (CRG)—and selected data from previous utilization (use of hospitalization, use of high-cost drugs in ambulatory care, pharmaceutical expenditure). A univariate descriptive analysis was performed. We constructed a logistic regression model with a 95% confidence level and analyzed sensitivity, specificity, positive predictive values (PPV), and the area under the ROC curve (AUC). ResultsIndividuals incurring costs >P95 accumulated 44% of total healthcare costs and were concentrated in ACRG3 (aggregated CRG level 3) categories related to multiple chronic diseases. All variables were statistically significant except for sex. The model had a sensitivity of 48.4% (CI: 46.9%-49.8%), specificity of 97.2% (CI: 97.0%-97.3%), PPV of 46.5% (CI: 45.0%-47.9%), and an AUC of 0.897 (CI: 0.892 to 0.902). ConclusionsHigh consumption of healthcare resources is associated with complex chronic morbidity. A model based on age, morbidity, and prior utilization is able to predict high-cost risk and identify a target population requiring proactive care.

Managing myelodysplastic syndromes in very old patients: a teaching case report

The introduction of hypomethylating agents in the treatment of myelodysplastic syndromes (MDS) has significantly changed the clinical scenario of these diseases, which afflict predominantly older individuals. However, some concerns regarding the optimal application of these innovative and costly agents in the treatment of geriatric high-risk MDS remain. We report here the case of a nonagenarian treated with hypomethylating agents achieving a long-lasting clinical response and a significant improvement in her functional status. Our case confirmed that functional status and biological status, rather than the chronological age alone, can substantially guide the plan of an appropriate treatment strategy in high-risk MDS patients; moreover, the current case emphasizes the need for targeted studies in the field of geriatric MDS in order to formulate guidelines on the appropriate use of these costly agents, so that candidate patients can receive adequate treatment to preserve their quality of life and life expectancy, but at the same time avoiding unnecessary costs deriving from the use of high-cost drugs for those in whom a significant therapeutic result cannot be reasonably expected.

Leveraging drug-utilization and external benchmarking data to drive change in prescribing behaviors

Improved outcomes and cost savings achieved at a large hospital through a drug utilization benchmarking and reporting initiative are described. Using the University HealthSystem Consortium (UHC) Clinical Resource Manager (CRM) database, the University of Kansas Hospital identified nine target areas (based on Medicare Severity Diagnosis-Related Group) in which the hospital's drug-utilization practices were deemed suboptimal relative to those of other UHC member facilities with similar caseloads. The pharmacy department developed a CRM template for generating customized reports comparing the hospital's performance on various drug-utilization metrics with that of top-performing peers (i.e., institutions achieving the best patient care outcomes in terms of mortality and length of stay) in the nine target areas. A pre-post comparison of drug-utilization data collected before and after implementation of the reporting initiative indicated improved outcomes in all nine initially selected target areas, with estimated cumulative annualized cost savings of about $900,000. The CRM-generated reports are now distributed semiannually to attending physicians and other hospital leaders via electronic and hard-copy means, focusing on variances from UHC top-performer and overall UHC averages in the use of higher-cost drugs. The reporting initiative has generally fostered enhanced physician-pharmacist collaboration in the investigation of identified drug-utilization variances and implementation of practice changes. By evaluating service-specific trends of internal drug utilization against external benchmarks and emulating prescribing practices at top-performing institutions, an academic medical center has achieved improved patient care outcomes and cost savings.

Benefits and risks of increasing restrictions on access to costly drugs in Medicaid.

States are reacting to increased Medicaid drug costs by implementing cost-control policies, such as preferred drug lists (PDLs) and prior authorization. PDLs have risks as well as benefits. Targeting essential drug classes with heterogeneous patient responses and side effects could reduce appropriate care, adversely affect health status, and cause shifts to more costly types of care. Assessing inappropriate use of high-cost drugs before implementing regulations and instituting simple mechanisms to exempt high-risk patients could maximize savings and minimize harm. The current exponential growth in such policies and the limited evidence base justifies investment in research to identify which policies can achieve savings without unintended consequences.

Using pharmacoeconomics to justify use of higher-cost drugs

Using pharmacoeconomics to justify use of higher-cost drugs