Private payers' varicose vein policies are inaccurate, disparate, and not evidence based, which mandates a proposal for a reasonable and responsible policy for the treatment of venous disease.

Varicose veins afflict more than one in five Americans, and although varicose veins may be an asymptomatic cosmetic concern in some, many others experience symptoms of pain, aching, heaviness, itching, and swelling. More advanced venous disease can result from untreated venous insufficiency. The complications of chronic venous disease, including bleeding, thrombosis, and ulceration, are seen in up to 2 million Americans annually. Numerous reports have documented venous disease adversely affects quality of life and that treatment of venous disease can improve quality of life. It has previously been documented that private insurers, and Centers for Medicare & Medicaid Services subcontractors for that matter, have disparate policies that in many instances are self-serving, contain mistakes, use outdated evidence, and disregard evidence-based guidelines. The two leading venous medical societies, the American Venous Forum and the American Venous and Lymphatic Society, have come together to review the varicose vein coverage policies of seven major U.S. private medical insurance carriers whose policies cover more than 150 million Americans. The authors reviewed the policies for venous disease and, if significant gaps or inconsistencies are found, we hope to point them out, and, finally, to propose a thoughtful and reasonable policy based on the best available evidence.

Evidence Supporting the Value of Surgical Procedures: Can We Do Better?

There is an acknowledged need for higher-quality evidence to quantify the benefit of surgical procedures, yet not enough has been done to improve the evidence base. This lack of evidence can prevent fully informed decision-making, lead to unnecessary or even harmful treatment, and contribute to wasteful expenditures of scare health care resources. Barriers to evidence generation include not only the long-recognized technical difficulties and ethical challenges of conducting randomized surgical trials, but also legal challenges that limit incentives to conduct surgical research as well as market-based challenges that make it difficult for those funding surgical research to recoup investment costs. These legal and market dynamics differ substantially from those surrounding new drug or device development. Nevertheless, obstacles could be overcome and overall expenditures could be reduced if a share of federal health care agency budgets were reallocated to generating randomized trial data, standardizing outcome measures, and conducting observational studies analogous to those that have been facilitated for drugs via the Food and Drug Administration's Sentinel Initiative. Until better quality evidence is available, ethical principles require adequate disclosure of the limited evidence base supporting current surgical procedures.

Virtual Fracture Clinics in Orthopaedic Surgery - A Systematic Review of Current Evidence.

AIMS: Approximately 75% of fractures are simple, stable injuries which are often unnecessarily immobilised with subsequent repeated radiographs at numerous fracture clinic visits. In 2014, the Glasgow Fracture Pathway offered an alternative virtual fracture clinic (VFC) pathway with the potential to reduce traditional fracture clinic visits, waiting times and overall costs. Many units have implemented this style of pathway in the non-operative management of simple, undisplaced fractures. This study aims to systematically review the clinical outcomes, patient reported outcomes and cost analyses for VFCs. MATERIALS AND METHODS: Two independent reviewers performed the literature search based on PRISMA guidelines, utilizing the MEDLINE, EMBASE and COCHRANE Library databases. Studies reporting outcomes following the use of VFC were included. Outcomes analysed were: 1) clinical outcomes, 2) patient reported outcomes, and 3) cost analysis. RESULTS: Overall, 15 studies involving 11,921 patients with a mean age of 41.1 years and mean follow-up of 12.6 months were included. In total, 65.7% of patients were directly virtually discharged with protocol derived conservative management, with 9.1% using the Helpline and 15.6% contacting their general practitioner for advice or reassurance. A total of 1.2% of patients experienced fracture non-unions and 0.4% required surgical intervention. The overall patient satisfaction rate was 81.0%, with only 1.3% experiencing residual pain at the fracture site. Additionally, the mean cost per patient for VFC was £71, with a mean saving of £53 when compared to traditional clinic models. Subgroup analysis found that for undisplaced fifth metatarsal or radial head/neck fractures, the rates of discharge from VFC to physiotherapy or general practitioners were 81.2% and 93.7% respectively. DISCUSSION AND CONCLUSION: This study established that there is excellent evidence to support virtual fracture clinic for non-operative management of fifth metatarsal fractures, with moderate evidence for radial head and neck fractures. However, the routine use of virtual fracture clinics is presently not validated for all stable, undisplaced fracture patterns. LEVEL OF EVIDENCE: IV; Systematic Review of all Levels of Evidence.

Use of real-world evidence in cancer drug funding decisions in Canada: a qualitative study of stakeholders' perspectives.

BACKGROUND: Real-world evidence (RWE) can provide postmarket data to inform whether funded cancer drugs yield expected outcomes and value for money, but it is unclear how to incorporate RWE into Canadian cancer drug funding decisions. As part of the Canadian Real-World Evidence Value for Cancer Drugs (CanREValue) Collaboration, this study aimed to explore stakeholder perspectives on the current state of RWE in Canada to inform a Canadian framework for use of RWE in cancer drug funding decisions. METHODS: This was a qualitative descriptive study. Qualitative semistructured interviews were conducted from April to July 2018. Participants were Canadian and international stakeholders who had experience with RWE and drug funding decision-making. Thematic analysis was used to analyze data. RESULTS: Thirty stakeholders participated in the study. Five themes were identified. Stakeholders indicated that RWE had value in cancer drug funding decisions. However, a cultural shift is needed to adopt RWE in decision-making. Further, the Canadian infrastructure for real-world data is currently inadequate for decision-making, and there is a need for committed investment in building capacity to collect and analyze RWE. Finally, there is a need for increased collaboration among key stakeholders. INTERPRETATION: The findings of this study suggest that if RWE is to be used in drug funding decisions, there is a need for a cultural shift, improved data infrastructure, committed investment in capacity building and increased stakeholder collaboration. Together with local stakeholder engagement, application of these findings may contribute to optimizing implementation of RWE.

Association Between a National Insurer's Pay-for-Performance Program for Oncology and Changes in Prescribing of Evidence-Based Cancer Drugs and Spending.

PURPOSE: Cancer drug prescribing by medical oncologists accounts for the greatest variation in practice and the largest portion of spending on cancer care. We evaluated the association between a national commercial insurer's ongoing pay-for-performance (P4P) program for oncology and changes in the prescribing of evidence-based cancer drugs and spending. METHODS: We conducted an observational difference-in-differences study using administrative claims data covering 6.7% of US adults. We leveraged the geographically staggered, time-varying rollout of the P4P program to simulate a stepped-wedge study design. We included patients age 18 years or older with breast, colon, or lung cancer who were prescribed cancer drug regimens by 1,867 participating oncologists between 2013 and 2017. The exposure was a time-varying dichotomous variable equal to 1 for patients who were prescribed a cancer drug regimen after the P4P program was offered. The primary outcome was whether a patient's drug regimen was a program-endorsed, evidence-based regimen. We also evaluated spending over a 6-month episode period. RESULTS: The P4P program was associated with an increase in evidence-based regimen prescribing from 57.1% of patients in the preintervention period to 62.2% in the intervention period, for a difference of +5.1 percentage point (95% CI, 3.0 percentage points to 7.2 percentage points; P < .001). The P4P program was also associated with a differential $3,339 (95% CI, $1,121 to $5,557; P = .003) increase in cancer drug spending and a differential $253 (95% CI, $100 to $406; P = .001) increase in patient out-of-pocket spending, but no significant changes in total health care spending ($2,772; 95% CI, -$181 to $5,725; P = .07) over the 6-month episode period. CONCLUSION: P4P programs may be effective in increasing evidence-based cancer drug prescribing, but may not yield cost savings.

Navigating Ethical Practices in the Era of High Cost Hematology.

PURPOSE OF REVIEW: In this review article, we will highlight ethical issues faced by hematologists due to a growing constellation of expensive diagnostics and therapeutics in hematology. We outline the important issues surrounding this topic including stakeholders, cost considerations, and various ethical challenges surrounding access to care, communication about costs, and individual vs. societal responsibilities. We review available tools to navigate these ethical themes and offer potential solutions. RECENT FINDINGS: We identified several gaps in the literature on the topic of ethical issues in hematology treatment and supplement by non-hematological cancer and general medical literature. We propose proactive solutions to address these problems to include cost transparency, utilization of evidence-based decision making tools, application of the four quadrant approach to ethical care, and advanced systems-based practice curriculum for physician trainees.

An Evidence-Based Care Protocol Improves Outcomes and Decreases Cost in Pediatric Appendicitis.

BACKGROUND: Appendicitis is a common indication for urgent abdominal surgery in the pediatric population. The postoperative management varies significantly in time to discharge and cost of care. The objective of this study was to investigate whether implementation of an evidence-based protocol after an appendectomy would lead to decreased length of stay and cost of care. METHODS: In 2014 at the Children's Hospital of Pittsburgh, an initiative to develop an evidenced-based protocol to treat appendicitis was undertaken. A work group was formed of pediatric surgeons and other important personnel to determine best practices. Treatment pathways were created. Pathways differed with recommendation on postoperative antibiotic choice and duration, diet initiation, and discharge criteria. Data were prospectively gathered from all patients (ages 0-18 y) with acute appendicitis from January 2015 to December 2016. Primary outcomes were length of stay and cost of care. Secondary outcomes were surgical site infection, readmission rate, and duration of postoperative antibiotics. RESULTS: Among the 1289 patients, 481 patients were in the preprotocol cohort and 808 patients were in the postprotocol cohort. 27% of patients had an intraoperative diagnosis of complicated appendicitis. There was a significantly shorter length of stay in the postprotocol cohort (P < 0.001). Median costs for the whole cohort decreased 0.6% and 24.6% for patients with complicated appendicitis after protocol initiation (P < 0.01). CONCLUSIONS: This study has demonstrated that introduction of an evidence-based clinical care protocol for pediatric patients with appendicitis leads to shorter hospital stay and decreased hospital costs.

Trigger Finger Treatment: Identifying Predictors of Nonadherence and Cost.

BACKGROUND: Evidence-based practices in medicine are linked with a higher quality of care and lower health care cost. For trigger finger, identifying patient factors associated with nonadherence to evidence-based practices will aid physicians in treatment decisions. The objectives were to (1) determine patient factors associated with treatment nonadherence, (2) examine the success rates of steroid injections, and (3) evaluate the economic consequences of nonadherence to treatment recommendations. METHODS: The authors used data from the Clinformatics DataMart database from 2010 to 2017 to conduct a population-based analysis of patients with single-digit trigger finger. The authors calculated rates of steroid injection success and examined associations between injection success and patient factors using chi-square tests. In addition, the authors analyzed differences in the cost to the insurer, the cost to the patient, and total cost. RESULTS: A total of 29,722 patients were included in this analysis. Injection success rates were similar for diabetic (72 percent) and nondiabetic patients (73 percent), women (73 percent), and men (73 percent). Nonetheless, diabetics (OR, 1.4; 95 percent CI, 1.4 to 1.5; p < 0.001) and women (OR, 1.2; 95 percent CI, 1.1 to 1.2; p < 0.001) were significantly more likely to receive nonadherent treatment. In total, $23 million (U.S. dollars) were spent on nonadherent trigger finger care. CONCLUSIONS: Diabetics and women have increased odds of having surgery without a prior steroid injection, despite similar success rates of steroid injections compared to nondiabetics and men. Because performing surgical release before any steroid injections may represent a higher cost treatment option, providers should provide steroid injections before surgery for all patients regardless of diabetes status or sex to minimize overtreatment. CLINICAL QUESTION/LEVEL OF EVIDENCE: Risk, III.

Choosing Wisely® in Hematology: Have We Made a Difference?

PURPOSE OF REVIEW: The Choosing Wisely® initiative, led by the American Board of Internal Medicine Foundation in collaboration with national professional medical societies, aims to help patients choose care that is essential, free from harm, and evidence-based. The American Society of Hematology has advocated practices specific to hematology for physicians and patients to examine carefully. Here, we summarize various barriers to adopting these practices, interventions used to improve adoption, and challenges in measuring the effectiveness of these interventions. RECENT FINDINGS: The Choosing Wisely® campaign has become an international effort with more than 20 countries worldwide having embraced it. Such widespread interest indicates that the campaign initiated an important dialog between patients and physicians about overutilization of resources. Evidence showing the positive impact of interventions on adopting these practices is accumulating, but their effect on improving clinical outcomes is uncertain. Decreasing overuse of resources is a cultural change in perspective for practitioners and patients alike. We believe that healthcare delivery is transitioning from being volume-based to value-based. As we continue to support the Choosing Wisely® campaign, we need to implement strategies to document and measure the influence of our value-based recommendations on physician practices, patient care and attitudes, and healthcare costs.

Use of Real-World Evidence in US Payer Coverage Decision-Making for Next-Generation Sequencing-Based Tests: Challenges, Opportunities, and Potential Solutions.

OBJECTIVES: Given the potential of real-world evidence (RWE) to inform understanding of the risk-benefit profile of next-generation sequencing (NGS)-based testing, we undertook a study to describe the current landscape of whether and how payers use RWE as part of their coverage decision making and potential solutions for overcoming barriers. METHODS: We performed a scoping literature review of existing RWE evidentiary frameworks for evaluating new technologies and identified barriers to clinical integration and evidence gaps for NGS. We synthesized findings as potential solutions for improving the relevance and utility of RWE for payer decision-making. RESULTS: Payers require evidence of clinical utility to inform coverage decisions, yet we found a relatively small number of published RWE studies, and these are predominately focused on oncology, pharmacogenomics, and perinatal/pediatric testing. We identified 3 categories of innovation that may help address the current undersupply of RWE studies for NGS: (1) increasing use of RWE to inform outcomes-based contracting for new technologies, (2) precision medicine initiatives that integrate clinical and genomic data and enable data sharing, and (3) Food and Drug Administration reforms to encourage the use of RWE. Potential solutions include development of data and evidence review standards, payer engagement in RWE study design, use of incentives and partnerships to lower the barriers to RWE generation, education of payers and providers concerning the use of RWE and NGS, and frameworks for conducting outcomes-based contracting for NGS. CONCLUSIONS: We provide numerous suggestions to overcome the data, methodologic, infrastructure, and policy challenges constraining greater integration of RWE in assessments of NGS.

Publicly accessible evidence of health-related quality of life benefits associated with cancer drugs approved by the European Medicines Agency between 2009 and 2015.

OBJECTIVE: Health-related quality of life (HRQoL) is one of the most important patient-relevant study end-points for the direct measurement of the benefit of cancer drugs. Therefore, our aim is to detect cancer indications with no published information on HRQoL at the time of European Medicines Agency (EMA) approval and monitor any reported HRQoL evidence updates after at least three years of follow-up. METHODS: We included all cancer indications that were approved by the EMA between January 2009 and October 2015. Our main sources of information were the EMA website, clinicaltrials.gov and a systematic literature search in PubMed. Information on HRQoL outcomes was extracted alongside evidence on median overall survival. RESULTS: In total, we identified 110 indications, of which more than half (n = 58, 53%) were lacking available information on HRQoL assessments at the time of EMA approval. After a monitoring period of at least three years, 24 updates were identified, resulting in 34 (31%) therapies where information on HRQoL was still not available. For the 76 therapies with reported information on HRQoL, cancer-specific instruments were mostly used (n = 49/76). Regarding cumulative evidence on median overall survival and HRQoL, 33 (n = 33/110, 30%) as well as 15 (n = 15/110, 14%) cancer drugs were lacking information on both study end-points at the time of approval and after monitoring, respectively. CONCLUSION: Our results demonstrate that there is an urgent need of routine re-evaluation of reimbursed cancer drugs with initially missing information on major outcomes. Standardisation of the typology and quality of HRQoL assessments need to be improved to allow better comparability of results.

Stereotactic Ablative Body Radiotherapy for Intermediate- or High-Risk Prostate Cancer.

Stereotactic ablative radiotherapy (SABR) is a relatively novel form of high precision radiotherapy. For low- and intermediate risk patients, ultrahypofractionation (UHF - more than 5 Gy per day) has been compared to conventionally fractionated or moderately hypofractionated radiotherapy in two large randomized studies. A third smaller randomized study examined the question of the optimal frequency of treatments. The results of these studies will be reviewed. SABR for high risk prostate cancer has been shown to be feasible and is well tolerated with careful planning and setup techniques. However, there is currently insufficient data supporting its use for high-risk patients to offer SABR outside of a clinical trial. SABR costs less to the radiotherapydepartments and, the patient, as well as increasing system capacity. Therefore, it has the potential to be widely adopted in the next few years.

Developing a framework to incorporate real-world evidence in cancer drug funding decisions: the Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration.

BACKGROUND: Oncology therapy is becoming increasingly more expensive and challenging the affordability and sustainability of drug programmes around the world. When new drugs are evaluated, health technology assessment organisations rely on clinical trials to inform funding decisions. However, clinical trials are not able to assess overall survival and generalises evidence in a real-world setting. As a result, policy makers have little information on whether drug funding decisions based on clinical trials ultimately yield the outcomes and value for money that might be expected. OBJECTIVE: The Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration, consisting of researchers, recommendation-makers, decision makers, payers, patients and caregivers, are developing and testing a framework for Canadian provinces to generate and use real-world evidence (RWE) for cancer drug funding in a consistent and integrated manner. STRATEGY: The CanREValue collaboration has established five formal working groups (WGs) to focus on specific processes in the generation and use of RWE for cancer drug funding decisions in Canada. The different RWE WGs are: (1) Planning and Drug Selection; (2) Methods; (3) Data; (4) Reassessment and Uptake; (5) Engagement. These WGs are acting collaboratively to develop a framework for RWE evaluation, validate the framework through the multiprovince RWE projects and help to integrate the final RWE framework into the Canadian healthcare system. OUTCOMES: The framework will enable the reassessment of cancer drugs, refinement of funding recommendations and use of novel funding mechanisms by decision-makers/payers across Canada to ensure the healthcare system is providing clinical benefits and value for money.

Contrasting evidence to reimbursement reality for off-label use (OLU) of drug treatments in cancer care: rationale and design of the CEIT-OLU project.

Background: Off-label use (OLU) of a drug reflects a perceived unmet medical need, which is common in oncology. Cancer drugs are often highly expensive and their reimbursement is a challenge for many healthcare systems. OLU is frequently regulated by reimbursement restrictions. For evidence-based healthcare, treatment ought to be reimbursed if there is sufficient clinical evidence for treatment benefit independently of patient factors not related to the treatment indication. However, little is known about the reality of OLU reimbursement and its association with the underlying clinical evidence. Here, we aim to investigate the relationship of reimbursement decisions with the underlying clinical evidence. Methods/ design: We will extract patient characteristics and details on treatment and reimbursement of cancer drugs from over 3000 patients treated in three Swiss hospitals. We will systematically search for clinical trial evidence on benefits associated with OLU in the most common indications. We will describe the prevalence of OLU in Switzerland and its reimbursement in cancer care, and use multivariable logistic regression techniques to investigate the association of approval/rejection of a reimbursement requests to the evidence on treatment effects and to further factors, including type of drug, molecular predictive markers and the health insurer. Discussion: Our study will provide a systematic overview and assessment of OLU and its reimbursement reality in Switzerland. We may provide a better understanding of the access to cancer care that is regulated by health insurers and we hope to identify factors that determine the level of evidence-based cancer care in a highly diverse western healthcare system.

Value-based medicine and palliative care: how do they converge?

Introduction: Sick persons need doctors who understand their pathology, know how to treat their problem, and accompany them through their illness. This study aimed to synthesize the state of knowledge regarding the concept of value-based medicine (VBM) through an integrative literature review, and establish how VBM can be applied in palliative care. Areas covered: An integrative review was conducted with the keywords 'value-based medicine,' 'patient-centered care,' and 'medicina baseada em valor' (Portuguese for VBM) in PubMed and Virtual Health Library, identifying 17,189 articles in total. Of these, 10 articles met the eligibility criteria. VBM combines the highest level of technical-scientific data with patients' values. It is defined as the combination of evidence-based medicine, patient-centered care, and cost-effectiveness. Patients' values are a set of preferences, concerns, and expectations that contribute toward accommodating their needs in the treatment clinic. Expert opinion: Like VBM, palliative care focuses on patients' values and quality of life, respecting natural limits. The early development of a care plan with active participation of the patient in the face of life-threatening diseases should be encouraged and can bring peace and comfort in a person's final moments.