Second-line pharmacotherapy intensification after metformin monotherapy in type 2 diabetes: a nationwide register study from Finland during 2011-2022.

BACKGROUND: In this nationwide register study, we examined the initiation of a second-line antidiabetic medicine (ADM) among new patients receiving regular metformin monotherapy in Finland during 2011-2022. We also reflected the second-line treatment patterns on changes in the reimbursement policy, and the national type 2 diabetes (T2D) care guidelines. METHODS: Using register data on all reimbursed ADM purchases during 2010-2022, we defined nine annual cohorts of patients initiating regular metformin monotherapy during 2011-2019, each with a three-year follow-up. Descriptive methods were used to study the patterns of metformin monotherapy and second-line intensification over time. Proportional hazards models were used to analyse the take-up of the second-line ADM. RESULTS: The share of new patients initiating metformin use (11-13% of all metformin users) and regular metformin use (83-85% of all new metformin users) remained stable. In all cohorts, 16-19% of the patients took up a second-line ADM (median time to intensification 1.5 years). With the 2011 cohort as reference, the highest proportion of new regular metformin users taking up a second ADM (hazard ratio 1.12. 95% confidence interval 1.07 ; 1.16, P < .0001) was in the 2019 cohort. In the 2017 cohort, the proportion of patients initiating sodium-glucose cotransporter 2 inhibitors as second-line treatment surpassed those initiating dipeptidyl peptidase-4 inhibitors. The reimbursement policy restricted the use of GLP-1-analogues. CONCLUSIONS: Second-line treatment intensification patterns over time paralleled the changes in the reimbursement system. Thus, our findings suggest that the reimbursement policy may influence the use of ADMs in Finland.

Continued restrictions on nurse practitioners: A qualitative study of the early implementation of full practice authority in Massachusetts.

BACKGROUND: In January 2021, the Commonwealth of Massachusetts granted nurse practitioners (NPs) full practice authority (FPA). Little is known about how care delivery changed after FPA legislation. PURPOSE: To understand the NP perception of early implementation of FPA in Massachusetts. METHODS: Qualitative descriptive design using inductive thematic analysis of open-ended responses to a web-based survey of NPs in Massachusetts from October to December 2021. FINDINGS: Survey response rate was 50.3% (N = 144). Inductive thematic analysis of open-ended responses identified four themes, including: (a) internal and external barriers obstructed FPA implementation, (b) employer communication about scope-of-practice changes was minimal, (c) NPs led initiatives to implement FPA, and (d) some efforts effectively implemented FPA. DISCUSSION: Almost 1 year after FPA was passed, external policies persisted that financially incentivized employers to not change NP scope-of-practice. Concerted efforts are needed to ensure that federal and payer policies, such as incident-to billing, are aligned with state law to encourage the implementation of FPA.

The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy.

BACKGROUND: The reimbursement for expensive medicines poses a growing challenge to healthcare worldwide. In order to increase its control over the costs of medicines, the Dutch government introduced the Coverage Lock (CL) policy in 2015. The CL postpones decisions regarding reimbursement of expensive medicines until detailed advice on i.e., cost-effectiveness has been given. The CL has been in place for six years, has raised many questions and concerns, but currently, no evaluation is known to the authors. A better understanding of the effects of the CL on all stakeholders involved may contribute to reflections on the CL process and help find ways to improve it. An evaluation of Dutch policy will also be relevant for other countries that aim to optimize reimbursement procedures for expensive treatments. To perform this evaluation, we focused on the CL procedure for the medicine nusinersen. Nusinersen is the first treatment for spinal muscular atrophy (SMA). Following EMA approval in May 2017, it was placed in the CL. The analysis of cost-effectiveness and added therapeutic value resulted in an advice for reimbursement limited to children younger than 9.5 years at the start of treatment; this was implemented from August 2018 onwards. METHODS: Qualitative stakeholder perspective analysis of the CL procedure focusing on nusinersen with 15 stakeholders. RESULTS: Stakeholders raised key issues of the CL based on their experience with nusinersen: emotional impact of the CL, duration of the CL procedure, appropriateness of the CL procedure for different types of medicines, transparency of the CL, a wish for patient-centred decision-making and the lack of uniformity of access to expensive treatments. DISCUSSION: Stakeholders supported measures to control healthcare expenses and to ensure reasonable pricing. They considered the delay in access to therapies and lack of procedural transparency to be the main challenges to the CL. Stakeholders also agreed that the interests of patients deserve more attention in the practical implementation of the reimbursement decision. Stakeholders suggested a number of adjustments to improve the CL, such as a faster start with conditional reimbursement programs to ensure access and intensify European collaboration to speed up the assessment of the medicine.

Discovering healthcare provider behavior patterns through the lens of Medicare excess charge.

BACKGROUND: The phenomenon of excess charge, where a healthcare service provider bills Medicare beyond the limit allowed for a medical procedure, is quite common in the United States public healthcare system. For example, in 2014, healthcare providers charged an average of 3.27 times (and up to 528 times) the allowable limit for cataract surgery. Previous research contends that such excess charges may be indicative of the actual amount that providers bill to non-Medicare patients and subsequent cost-shifting behavior, where a healthcare provider tries to recoup underpayment by Medicare from privately insured, self-pay, out-of-network, and uninsured patients. OBJECTIVES: The objective of this study is to examine the drivers of a provider's excess charge patterns, especially the extent to which the degree of excess charges may be associated with physician characteristics, Medicare reimbursement policy, or socioeconomic status and demographics of a provider's patient base. METHODS: Using data from the 2014 Medicare Provider Utilization files, we identify three procedures with the highest variation in Medicare reimbursements to study the excess charge phenomenon. We then employ a two-step cluster analysis within each procedure to identify distinct provider groups. RESULTS: Each procedure code yielded distinct healthcare provider segments with specific patient demographics and related behavior patterns. Cluster silhouette coefficients indicate that these segments are unique. Three random subsamples from each procedure establish the stability of the clusters. CONCLUSIONS: For each of the three procedures investigated in this study, a sizeable number of healthcare providers serving poorer, riskier patients are often paid significantly lower than their peers, and subsequently have the highest excess charges. For some providers, excess charges reveal possible cost-shifting to private insurance. Patterns of excess charges also indicate an imbalance of market power, especially in areas with lower provider competition and access to health care, thus leading to urban-rural healthcare disparities. Our results reinforce the call for price transparency and an upper limit to overbilling.

Policy-induced selection bias in pharmacoepidemiology: The example of coverage for Alzheimer's medications in British Columbia.

PURPOSES: To assess the impact of a government-sponsored reimbursement policy for cholinesterase inhibitors (ChEIs) on trends in physician visits with a diagnosis of Alzheimer's disease (AD). METHODS: Longitudinal population-based study using interrupted time series methods. British Columbia outpatient claims data for individuals aged 65 and older were used to compute monthly AD visit rates and examine the impact of the ChEI reimbursement policy on the coding of AD. We examined trends in the number of patients with AD visits, the number of AD visits per patient, and visits with "competing" diagnoses (mental, neurological, and cerebrovascular disorders and accidental falls). Finally, we described demographic and clinical features of diagnosed patients. RESULTS: We analyzed 1.9 million AD visits. Faster growth in recorded AD visits was observed after the policy was implemented, from monthly growth of 7.5 visits per 100 000 person-months before the policy (95% confidence interval [CI], 6.1-8.9) to monthly growth of 16.5 per 100 000 person-months after the policy (95% CI, 14.8-18.3). After the implementation of the policy, we observed increased growth in the number of patients with recorded AD visits and the number of AD visits per patient, as well as a shift in diagnoses away from mental diseases and accidental falls to AD (diagnosis substitution). CONCLUSIONS: British Columbia's reimbursement policy for ChEIs was associated with a significant acceleration in Alzheimer's visits. Evaluations of health services utilization and clinical outcomes following drug policy changes need to consider policy-induced influences on the reliability of the data used in the analysis.

Delayed Healing in Metatarsal Fractures: Role of Low-Intensity Pulsed Ultrasound Treatment.

The most common fracture in primary care is metatarsal fracture, but it is controversial whether to treat this fracture conservatively or surgically. We performed a cohort study to contrast metatarsal fractures that heal normally with fractures that show delayed healing. We analyzed 5% Medicare Standard Analytic Files, selecting all metatarsal fractures in 2011 to 2013, excluding patients with multiple fractures. Delayed healing was defined as treatment >14 days postfracture with either low-intensity pulsed ultrasound or surgery. Treatment for delayed healing was identified using the Current Procedural Terminology and International Classification of Diseases, Revision 9, Clinical Modification codes. Among 9482 metatarsal fractures, 256 (2.7%) showed delayed healing. Patients with delayed healing were younger (p < .0001); more likely to receive specialist referral (p < .001); more likely to have obesity (p = .005), psychosis (p = .003), chronic lung disease (p = .012), or iron deficiency anemia (p = .016); and more likely to receive surgery before ultrasound (p < .0001). Patients more likely to be treated with surgery than ultrasound included younger patients (p < .0001), obese patients (p = .02), and patients who first see a specialist (p < .05).

Policy change is not enough: engaging provider champions on immediate postpartum contraception.

Rates of short-interval pregnancies that result in unintended pregnancies remain high in the United States and contribute to adverse reproductive health outcomes. Long-acting reversible contraception methods have annual failure rates of <1%, compared with 9% for oral contraceptive pills, and are an effective strategy to reduce unintended pregnancies. To increase access to long-acting reversible contraception in the immediate postpartum period, several State Medicaid programs, which include those in Iowa and Louisiana, recently established reimbursement policies to remove the barriers to reimbursement of immediate postpartum long-acting reversible contraception insertion. We used a mixed-methods approach to analyze 2013-2015 linked Medicaid and vital records data from both Iowa and Louisiana and to describe trends in immediate postpartum long-acting reversible contraception provision 1 year before and after the Medicaid reimbursement policy change. We also used data from key informant interviews with state program staff to understand how provider champions affected policy uptake. We found that the monthly average for the number of insertions in Iowa increased from 4.6 per month before the policy to 6.6 per month after the policy; in Louisiana, the average number of insertions increased from 2.6 per month before the policy to 45.2 per month. In both states, the majority of insertions occurred at 1 academic/teaching hospital. In Louisiana, the additional increase may be due to the engagement of a provider champion who worked at both the state and facility level. Recruiting, training, engaging, and supporting provider champions, as facilitators, with influence at state and facility levels, is an important component of a multipart strategy for increasing successful implementation of state-level Medicaid payment reform policies that allow reimbursement for immediate postpartum long-acting reversible contraception insertions.

Small-Bowel Capsule Endoscopy in Clinical Practice: Has Anything Changed Over 13 Years?

BACKGROUND: In Lombardia, one of the 20 Italian administrative Regions, small-bowel capsule endoscopy (SBCE) was introduced in 2001. In January 2011, the Regional Health Authorities established a reimbursement for outpatient SBCE. AIM: To prospectively record data on SBCE between 2011-2013 and compare them to similar data retrospectively collected from the same geographical area (covering the period 2001-2008) and published in 2008. METHODS: Consecutive SBCEs performed between January 2011 and December 2013 in Lombardia were prospectively collected. RESULTS: In 3 years, 3142 SBCEs were collected; the diagnostic yield (DY) and the overall complication rate were 48.4 and 0.9%, respectively. The main indication was suspected small-bowel bleeding (76.6% of patients); complete small-bowel inspection was achieved in 2796 (89.0%) patients. SBCE was performed as an outpatient procedure in 1945 patients (61.9%). A significant increase in the rate of patients undergoing SBCE for suspected small-bowel bleeding was observed from 2001-2008 to 2011-2013 (67.3 vs. 76.1%; p < 0.001). There was an increase in the number of complete small-bowel examinations (81.2 vs. 89.0%; p < 0.001) and of outpatient SBCEs (6.7 vs. 61.9%; p < 0.001). Conversely, both the retention rate (2.1 vs. 0.8%; p < 0.001) and the rate of patients undergoing SBCE for Crohn's disease (11.5 vs. 5.5%; p < 0.001) decreased significantly. The overall DY remained stable (50.6 vs. 48.4%; p = 0.089). CONCLUSION: Our study shows that, over 13 years, the SBCE safety profile and completion rate significantly improved over time; a change in the spectrum of clinical indications was also observed.

EVIDENCE REQUIRED BY HEALTH TECHNOLGY ASSESSMENT AND REIMBURSEMENT BODIES EVALUATING DIAGNOSTIC OR PROGNOSTIC ALGORITHMS THAT INCLUDE OMICS DATA.

OBJECTIVES: Multi-analyte assays with algorithmic analyses (MAAAs) use combinations of circulating and clinical markers including omics-based sources for diagnostic and/or prognostic purposes. Assessing MAAAs is challenging under existing health technology assessment (HTA) methods or practices. We undertook a scoping review to explore the HTA methods used for MAAAs to identify the criteria used for clinical research and reimbursement purposes. METHODS: This review included only non-companion (stand-alone) tests that are actionable and that have been evaluated by leading HTA or insurer/reimbursement bodies up to September 2017. RESULTS: Twenty-five reports and articles evaluating seventeen MAAAs were examined, most of which have been developed in oncology. The two main models used were the EUnetHTA Core model and the Evaluation of Genomic Applications in Practice and Prevention ACCE framework. Clinical validity and utility criteria were used, as were economic, ethical, legal, and social aspects. Economic evidence on MAAAs was scarce, and there is no consensus on whether the perspectives used are sufficiently broad to include all relevant stakeholders. CONCLUSIONS: Clinical utility and efficiency were the most used criteria, with stronger evidence needed linking the use of the algorithm with the clinical outcomes in real-life practice. HTA bodies must as well consider questions related to the analytical validity of MAAAs or with organizational aspects. The two main models, the EUnetHTA Core model and the ACCE framework, could be adapted to the assessment of MAAAs.

It's Time! The Path for Nursing Reimbursement Reform.

Since the COVID-19 pandemic, nurses and nurse leaders are increasingly vocal about chronic understaffing and the impact the staffing crisis continues to have on nurses' well-being and patient outcomes. The American Nurses Association's Nurse Staffing Task Force addressed the importance of staffing standards as a critically needed step toward improving patient and population health outcomes. Against the backdrop of ongoing nursing shortages, hospital leaders have been hesitant to embrace staffing ratios, expressing concerns about their ability to hire and retain sufficient nursing staff, as operational revenue margins remain thin and nursing labor is costly. This article explicates structural issues within the current nursing reimbursement model that harms hospitals' business case for investments in nurse staffing and work environments. We argue that nurses must advocate for nursing reimbursement reform to increase the nursing workforce and improve nurse staffing and work environments. Such reform is necessary to support sustained hospital investments, financial philosophies, and approaches to meaningfully address and improve nurse staffing.

Impact of Site-Neutral Payments for Commercial and Employer-Sponsored Plans.

Site-neutral payment is a policy created by federal rule making and implemented by the Centers for Medicare and Medicaid Services (CMS) that aims to reduce healthcare costs by aligning payment rates for certain services provided in multiple care settings. Site-neutral payments are intended to eliminate the incentive for providers to acquire facilities, such as physician offices or ambulatory surgical centers (ASCs), that Medicare reimburses at the lower non-facility rate and convert those settings into hospital outpatient departments (HOPDs), where Medicare reimburses at the higher facility rate. Although initiated by Congress to address payment disparities in Medicare, similar payment discrepancies can be seen in the commercial market where individual and employer-sponsored health plans often pay more for certain outpatient services depending on their location. This analysis presents a simulation of the impact of applying site-neutral payments to the commercial market with respect to overall potential savings for consumers, health plans and the federal government. To conduct the analysis, we use an all-payer claims data base generalizable to the United States. The analysis focused on a select group of outpatient services identified by the Medicare Payment Advisory Commission (MedPAC). We mapped the MedPAC identified 68 Ambulatory Payment Classifications (APCs), the codes Medicare uses to reimburse facilities for outpatient services, to the relevant CPT4/HCPCS codes, which the commercial market uses for billing. The potential cost savings of applying the site-neutral payment policy to the commercial insurance market to be $58 billion for year 2022. We estimate the 10-year total (2024-2033) employer market premium reduction ranges from 5.35% to 5.0% and found that those premium reductions would result in employer-sponsored insurance (ESI) tax subsidy savings of $140 billion to the federal government over a 10-year period (2024-2033).

Promoting the adoption of local governmental policy on the reimbursement of chronic disease medicines (PAPMed): study protocol of a field-based cluster randomized trial in rural Nantong, China.

BACKGROUND: Among rural Chinese patients with non-communicable diseases (NCDs), low socioeconomic status increases the risk of developing NCDs and associated financial burdens in paying for medicines and treatments. Despite the chronic disease medicine reimbursement policy of the local government in Nantong City, China, various barriers prevent patients from registering for and benefitting from the policy. This study aims to develop a behavior science-based intervention program for promoting the adoption of the policy and to evaluate the effectiveness of the program compared with usual practices. METHODS: Barriers and opportunities affecting stakeholders in adopting the policy were identified through contextual research and summarized through behavior mapping. The intervention is designed to target these barriers and opportunities through behavior science theories and will be evaluated through a 6-month cluster randomized controlled trial in Tongzhou District, Nantong, China. A total of 30 villages from two townships are randomized in a 1:1 ratio to either the intervention or the control arm (usual practices). Village doctors in the intervention arm (1) receive systematic training on policy details, registration procedures, and intervention protocol, (2) promote the policy and encourage registration, (3) follow up with patients in the first, third, and sixth months after the intervention, and (4) receive financial incentives based on performance. The primary outcome is policy registration rate and the secondary outcomes include the number of patients registering for the policy, medical costs saved, frequency of village doctor visits, and health measures such as blood pressure and glucose levels. DISCUSSION: This study is one of very few that aims to promote adoption of NCDs outpatient medication reimbursement policies, and the first study to evaluate the impact of these policies on patients' financial and physical wellbeing in China. The simple, feasible, and scalable intervention is designed based on the theories of behavior science and is applicable to similar low-income regions nationwide where outpatient medical costs remain a financial burden for patients. TRIAL REGISTRATION: ClinicalTrials.gov NCT04731194 , registered on 29 January 2021; Chinese Clinical Trial Registry ChiCTR2100042152 , registered on 14 January 14 2021.

Review of Publicly Available State Policies for Long-Acting Reversible Contraception Device Reimbursement.

Background: Provider challenges to accessing long-acting reversible contraception (LARC) include level of reimbursement for LARC device acquisition and cost to stock. State-level LARC device reimbursement policies that cover a greater proportion of the cost of the LARC device and enable providers to purchase LARC upfront may improve contraceptive access. Materials and Methods: To summarize state-level policies that include language on LARC device reimbursement in the outpatient setting, we conducted a systematic, web-based review among all 50 states of publicly available LARC device reimbursement policies that include coverage of LARC devices as a medical or pharmacy benefit, the use of the 340B Drug Pricing Program to purchase LARC devices, and separate payment for LARC devices outside of the Medicaid Prospective Payment System (PPS) payment rate for Federally Qualified Health Centers or Rural Health Clinics. Results: Forty-two percent (21/50) of states with publicly available state-level policies included language on LARC device reimbursement. Among the states, 24% (5/21) had coverage policies as a medical benefit, 33% (7/21) as a pharmacy benefit, and 19% (4/21) as both a medical benefit and pharmacy benefit; 38% (8/21) used the 340B Program to purchase LARC devices; and 62% (13/21) indicated separate payment for LARC devices outside of the Medicaid PPS payment rate. Conclusion: State-level policies for LARC device reimbursement vary, highlighting differences in reimbursement strategies across the U.S. Future research could explore how the implementation of these payment methods may impact LARC device reimbursement and whether increased reimbursement may improve access to the full range of contraceptive methods.

Impact of reimbursement restriction on drug market sales under the National Health Insurance in Japan.

AIM: National health care expenditures have been increasing each year, although the Japanese government has annually revised official drug prices. Managing the health care system to pay for expensive drugs is a major concern. The reimbursement restriction, which is the only way that a drug can be implemented before market entry in Japan, is crucial for managing expenditures. Therefore, this study identifies the impact of the reimbursement restriction on drug market sales in Japan, particularly in the situation where health technology assessment or other market access regulations are not applicable before market entry. METHOD: All new drugs listed in fiscal years 2011-2019, along with their market size forecast, were identified using the materials from the Central Social Insurance Medical Council. We then calculated the percentage rate of reimbursement amounts based on the National Database of Health Insurance Claims relative to the predicted market size as a dependent variable. Using the reimbursement restriction for each drug as an independent variable, we performed descriptive and univariate analyses on each variable, followed by generalized linear mixed-effects model regression analysis. RESULTS: We identified 211 drugs. The mean rates of drugs that required physicians, facilities, and patients to meet criteria for use were 30.85% (n = 2), 31.42% (n = 2), and 72.11% (n = 6), respectively. The mean rate of drugs that required diagnostic testing was 22.99% (n = 7), which was 3.7 times lower than the rate of drugs that did not require such testing (p < .05). CONCLUSION: Our results indicate that the reimbursement restriction requiring diagnostic testing has a substantial impact on decreasing market sales. As the number of cases for each requirement is small, further study is needed to measure the impact of the other reimbursement restrictions.

Determinants of market prices for drugs under Japan's national health insurance.

AIMS: The Japanese government reimburses patients for drugs at prices specified in the Drug Price Standard (DPS) published by the National Health Insurance (NHI) scheme. It revises reimbursements for most drugs on the basis of their market prices. This study thereby identifies factors related to drugs or disease that impact market prices for drugs using the DPS list. MATERIALS AND METHODS: This study first examined the 2018 DPS list to identify all listed drugs, their prices, and their stipulated reimbursements. We then excluded from this study all the drugs for which prices are set per alternate rules. We calculated the percentage divergence between market prices and DPS prices and designated it our dependent variable. We performed descriptive and a univariate analysis on each variable and constructed multivariate regression models featuring independent variables for drug characteristics that might affect market prices. RESULTS: We identified 1,775 drugs with prices revised only by the market. We observed higher percentage divergences between DPS and market prices for drugs with generic alternatives (p < 0.001), drugs listed in the Japanese Pharmacopoeia (p < 0.001), and drugs for which at least two new drugs entered the same therapeutic category (p < 0.001). Injectable drugs exhibited a more significant and negative correlation with percentage divergences (p = 0.009) than ingestible drugs. Drugs that treat specific organs (p < 0.001), affect metabolism (p = 0.001), and those prescribed for non-therapeutic purposes (p < 0.001) display significantly higher percentage divergence than drugs affecting the nervous system and sensory organs. Divergences are less for narcotics (p < 0.001) and drugs that counter pathologic microorganisms and parasites (p = 0.004). CONCLUSIONS: Factors that elevate competition among pharmaceutical companies likely lower market prices for drugs, and the direction of prices under NHI in Japan is affected by the category of diseases a drug treats.

Success of Big Infectious Disease Reimbursement Policy in China.

Big infectious diseases do harm to the whole society, and it is highly crucial to control them on time. The major purpose of this article is to theoretically demonstrate that the Chinese government's intervention in large-scale infectious diseases is successful and efficient. Two potential strategies were considered: strategy 1 was infectious disease without government intervention, and strategy 2 was infectious disease with government intervention. By evolution model, this article illustrates the efficiency of big infectious disease reimbursement policy in China. Without government reimbursement, this article finds that high expenditures accelerate the disease infection. The number of infected persons decreases under big infectious disease reimbursement policy in China. The higher the treatment costs, the more important the government intervention. Big infectious disease reimbursement policy in China can serve as an efficient example to cope with big infectious diseases.

Validity of the Best Practice Tariff in paediatric major trauma: A retrospective cohort study from a level 1 children's major trauma centre.

INTRODUCTION: The Best Practice Tariff (BPT) in major trauma awards Major Trauma Centres (MTCs) a financial incentive when predefined standards of care are met. However, no tailored criteria exist with regards to the reimbursement policy in paediatric major trauma. In this study, we aim to examine the utility of the paediatric Major Trauma BPT and identify predictors of additional resource utilisation. MATERIALS AND METHODS: This cohort study encompassed all paediatric major trauma calls (N = 682) presenting to a designated combined adult and paediatric MTC between July 2014 and June 2017. Patient demographics, admission pattern, injury parameters, length of stay (LOS) and the need for operative management were collected. Patients approved for the BPT uplift payment (BPT group) were compared with the cohort of children not qualifying (non-BPT group). RESULTS: Overall, less than a quarter (23.2%) of the trauma population qualified for the BPT. The proportion of patients requiring operative intervention and CT scanning in the BPT group was significantly higher (p<0.001). These children also attained a higher ISS (median, 13.5 vs. 0, p <0.001) and required longer hospitalisation. Following a Receiver Operator Characteristic (ROC) curve analysis, a cut off ISS score > 8 demonstrated an excellent predictive value in identifying children qualifying for BPT (true positive and false positive rates: 90% and 10.7%). However, a subgroup analysis including the more severely injured children (ISS >8) not qualifying for the uplift payment revealed that equally substantial resource went into their management - 42.9% needed surgical intervention and 57.1% a CT scan. DISCUSSION: This study demonstrated that BPT in paediatric major trauma is a valuable reimbursement; however, our findings also unveiled a cohort deemed ineligible for BPT despite the high costs accrued. Re-evaluation of the remuneration criteria of paediatric major trauma networks with an alternative, more inclusive reimbursement policy is needed.

Effects of removing reimbursement restrictions on targeted therapy accessibility for non-small cell lung cancer treatment in Taiwan: an interrupted time series study.

INTERVENTIONS: Targeted therapies have been proven to provide clinical benefits to patients with metastatic non-small cell lung cancer (NSCLC). Gefitinib was initially approved and reimbursed as a third-line therapy for patients with advanced NSCLC by the Taiwan National Health Insurance (NHI) in 2004; subsequently it became a second-line therapy (in 2007) and further a first-line therapy (in 2011) for patients with epidermal growth factor receptor mutation-positive advanced NSCLC. Another targeted therapy, erlotinib, was initially approved as a third-line therapy in 2007, and it became a second-line therapy in 2008. OBJECTIVES: This study is aimed towards an exploration of the impacts of the Taiwan NHI reimbursement policies (removing reimbursement restrictions) related to accessibility of targeted therapies. SETTING: We retrieved 2004-2013 claims data for all patients with lung cancer diagnoses from the NHI Research Database. DESIGN AND OUTCOME MEASURES: Using an interrupted time series design and segmented regression, we estimated changes in the monthly prescribing rate by patient number and market shares by cost following each modification of the reimbursement policy for gefitinib and erlotinib for NSCLC treatment. RESULTS: Totally 92 220 patients with NSCLC were identified. The prescribing rate of the targeted therapies increased by 15.58%, decreased by 10.98% and increased by 6.31% following the introduction of gefitinib as a second-line treatment in 2007, erlotinib as a second-line treatment in 2008 and gefitinib as as first line treatment in 2011, respectively. The average time to prescription reduced by 65.84% and 41.59% following coverage of erlotinib by insurance and gefitinib/erlotinib as second-line treatments in 2007-2008 and following gefitinib as the first-line treatment in 2011. CONCLUSIONS: The changes in reimbursement policies had a significant impact on the accessibility of targeted therapies for NSCLC treatment. Removing reimbursement restrictions can significantly increase the level and the speed of drug accessibility.

Pre-approval incentives to promote adoption of personalized medicine: a theoretical approach.

BACKGROUND: Currently, personalised medicine is becoming more frequently used and many drug companies are including this strategy to gain market access for very specialized therapies. In this article, in order to understand the relationships between the health authority and the drug company when deciding upon the implementation of personalized medicines, we take a theoretical perspective to model it when the price and reimbursement policy follows a pay-for-performance scheme. During the development of a new drug, the firm must decide whether to generate additional knowledge by investing in additional resources to stratify the target population based on a biomarker or directly apply for marketing authorization for the new treatment without information on the characteristics of patients who could respond to it. In this context, we assume that the pricing policy is set by the health authority, and then we characterize the pricing and investment decisions contingent on the rate of response to the treatment. RESULTS: We find that the price when the firm carries out R&D leading to the personalized treatments is not necessarily higher than the price if the firm does not carry out the R&D investment. When the rate of response to the treatment is too low, then the new drug is not marketed. If the rate of response is too high, personalized medicine is not implemented. For intermediate values of the rate of response, the adoption of personalized medicine may occur if the investment costs are sufficiently low; otherwise, the treatment is given to all patients without additional information on their characteristics. The higher the quality of the genetic test (in terms of its sensitivity and specificity), the wider the interval for the values of the proportional responders for which personalized medicine may be implemented. CONCLUSIONS: Our findings show that pre-approval incentives (prices) to promote the personalized treatments depend on the specific characteristics of the disease and the efficacy of the treatment. The model gives an intuitive idea about what to expect in terms of price incentives when the possibility of personalizing treatments becomes a strategic decision for the stakeholders.