Work-Sampling Study of an Innovative Care Coordination Program Aimed at Children With Chronic Health Conditions.

PURPOSE OF STUDY: To estimate time allocation and labor cost for care coordinators (CCs), community health workers (CHWs), and mental health workers (MHWs) to conduct care coordination tasks in a pediatric care coordination program. PRIMARY PRACTICE SETTING: A public tertiary academic medical center in Chicago, IL. METHODOLOGY AND SAMPLE: A work-sampling study was conducted using a text message-based survey on 5 CCs, 20 CHWs, and 4 MHWs who volunteered to participate. Workers were randomly sampled within working hours to collect information on who was the subject of interaction and what service was being delivered over a 6-month period. Time allocation of workers to different subjects and services was summarized using descriptive statistics. RESULTS: Care coordinators allocated 41% of their time to managing CHW teams. Community health workers allocated 37% of time providing services directly to children and 26% to the parent/caregiver. Mental health workers allocated 16% of time providing services to children and 29% to the parent/caregiver. The care coordination program serviced 5,965 patients, with a total annual labor cost of $1,455,353. IMPLICATIONS FOR CASE MANAGEMENT PRACTICE: Community health workers spent the majority of time working with patients and their families to conduct assessments. Mental health workers primarily addressed children's needs through their caregivers. Care coordinators primarily supported CHWs in coordinating care. Results may be used to inform development of such programs by determining services most often utilized, and labor cost may be used to inform program implementation and reimbursement.

The Introduction of New Non-Drug Health Technologies into Canadian Healthcare Institutions: Opportunities and Challenges.

INTRODUCTION: A recent pan-Canadian survey of 48 health organizations concluded that structures, processes, factors and information used to support funding decisions on new non-drug health technologies (NDTs) vary within and across jurisdictions in Canada. METHODS: A self-administered survey was used to determine demographic and financial characteristics of organizations, followed by in-depth interviews with senior leadership of consenting organizations to understand the processes for making funding decisions on NDTs. RESULTS: Seventy-three and 48 organizations completed self-administered surveys and telephone interviews, respectively (with 45 participating in both ways). Fifty-five different processes were identified, the majority of which addressed capital equipment. Most involved multidisciplinary committees (with medical and non-medical representation), but the types of information used to inform deliberations varied. Across all processes, decision-making criteria included local considerations such as alignment with organizational priorities. CONCLUSIONS: NDT decision-making processes vary in complexity, depending on characteristics of the healthcare organization and context.

Almost half of references in reports on new and emerging nondrug health technologies are grey literature.

OBJECTIVE: The research investigated how frequently grey literature is used in reports on new and emerging nondrug health technologies, which sources are most cited, and how grey literature searching is reported. METHODS: A retrospective review of references cited in horizon scanning reports on nondrug health technologies-including medical devices, laboratory tests, and procedures-was conducted. A quasi-random sample of up to three reports per agency was selected from a compilation of reports published in 2014 by international horizon scanning services and health organizations. RESULTS: Twenty-two reports from 8 agencies were included in the analysis. On average, 47% (288/617) of references listed in the bibliographies of the horizon scanning reports were grey literature. The most frequently cited type of grey literature was information from manufacturers (30% of all grey literature references), regulatory agencies (10%), clinical trial registries (9%), and other horizon scans or evidence synthesis reports (9%). The US Food and Drug Administration (FDA) and ClincalTrials.gov were the most frequently cited specific sources, constituting 7% and 8% of grey literature references, respectively. Over two-thirds (15/22) of the analyzed reports provided some details on search methodology; all 15 of these reported searching some grey literature. CONCLUSIONS: In this sample, grey literature represented almost half of the references cited in reports on new and emerging nondrug health technologies. Of these grey literature references, almost half came from three sources: the manufacturers, ClincalTrials.gov, and the FDA. There was wide variation in the other sources cited. Literature search methodology was often insufficiently reported for analysis.

Update of survival and cost of metastatic melanoma with new drugs: Estimations from the MelBase cohort.

PURPOSE: Since 2011, significant progress was observed in metastatic melanoma (MM), with the commercialisation of seven immunotherapies or targeted therapies, which showed significant improvement in survival. In France, in 2004, the cost of MM was estimated at €1634 per patient; this cost has not been re-estimated since. This study provided an update on survival and cost in real-life clinical practice. METHODS: Clinical and economic data (treatments, hospitalisations, radiotherapy sessions, visits, imaging and biological exams) were extracted from the prospective MelBase cohort, collecting individual data in 955 patients in 26 hospitals, from diagnosis of metastatic disease until death. Survival was estimated by the Kaplan-Meier method. Costs were calculated from the health insurance perspective using French tariffs. For live patients, survival and costs were extrapolated using a multistate model, describing the 5-year course of the disease according to patient prognostic factors and number of treatment lines. RESULTS: Since the availability of new drugs, the mean survival time of MM patients has increased to 23.6 months (95%confidence interval [CI] :21.2;26.6), with 58% of patients receiving a second line of treatment. Mean management costs increased to €269,682 (95%CI:244,196;304,916) per patient. Drugs accounted for 80% of the total cost. CONCLUSION: This study is the first that evaluated the impact of immunotherapies and targeted therapies both on survival and cost in real-life conditions. Alongside the introduction of breakthrough therapies in the first and subsequent lines, MM has been associated with a significant increase in survival but also in costs, raising the question of financial sustainability.

Funding breakthrough therapies: A systematic review and recommendation.

BACKGROUND: Advanced therapy medicinal products (ATMPs) are innovative therapies likely associated with high prices. Payers need guidance to create a balance between ensuring patient access to breakthrough therapies and maintaining the financial sustainability of the healthcare system. OBJECTIVE: The aims of this study were to identify, define, classify and compare the approaches to funding high-cost medicines proposed in the literature, to analyze their appropriateness for ATMP funding and to suggest an optimal funding model for ATMPs. RESULTS: Forty-eight articles suggesting new funding models for innovative high-cost therapies were identified. The models were classified into 3 groups: financial agreement, health outcomes-based agreement and healthcoin. Financial agreement encompassed: discounts, rebates, price and volume caps, price-volume agreements, loans, cost-plus price, intellectual-based payment and fund-based payment. Health outcomes-based agreements were defined as agreements between manufacturers and payers based on drug performance, and were divided into performance-based payment and coverage with evidence development. Healthcoin described a new suggested tradeable currency used to assign monetary value to incremental outcomes. CONCLUSION: With a large number of ATMPs in development, it is time for stakeholders to start thinking about new pathways and funding strategies for these innovative high-cost therapies. An "ATMP-specific fund" may constitute a reasonable solution to ensure rapid patient access to innovation without threatening the sustainability of the health care system.

Promoting innovation in small markets: Evidence from the market for rare and intractable diseases.

In many medical care markets with limited profit potential, firms often have little incentive to innovate. These include the market for rare diseases, "neglected" tropical diseases, and personalized medicine. Governments and not-for-profit organizations promote innovation in such markets but empirical evidence on the policy effect is limited. We study this issue by analyzing the impact of a demand-side policy in Japan, which reduces the cost sharing of patients with some rare and intractable diseases and attempts to establish and promote the treatment of those diseases. Using clinical trials data taken from public registries, we identify the effect of the policy using a difference-in-difference approach. We find that the demand-side policy increased firms' incentive to innovate: firm-sponsored clinical trials increased 181% (0.16 per disease per year) when covered by the policy. This result indicates that the demand-side policy can be an important part of innovation policies in markets with limited profit potential.

More than 5000 patients with metastatic melanoma in Europe per year do not have access to recommended first-line innovative treatments.

BACKGROUND: Despite the efficacy of innovative treatments for metastatic melanoma, their high costs has led to disparities in cancer care among different European countries. We analysed the availability of these innovative therapies in Europe and estimated the number of patients without access to first-line recommended treatment per current guidelines of professional entities such as the European Society for Medical Oncology (ESMO), the European Organisation for Research and Treatment of Cancer (EORTC), the European Association of Dermato-Oncology (EADO), and European Dermatology Forum (EDF). MATERIALS AND METHODS: Web-based online survey was conducted in 30 European countries with questions about the treatment schedules from 1st May 2015 to 1st May 2016: number of metastatic melanoma patients, registration and reimbursement of innovative medicines (updated data, as of 1st October 2016), percentage of patients treated and availability of clinical studies and compassionate-use programmes. RESULTS: The recommended BRAF inhibitor (BRAFi) + MEK inhibitor (MEKi) combination was both registered and fully reimbursed in 9/30 (30%) countries, and in 13/30 (43%) (all from Eastern Europe) not reimbursed. First-line immunotherapy with anti-PD1 antibodies was registered and fully reimbursed in 14/30 (47%) countries, while in 13/30 (43%) (all from Eastern Europe) not reimbursed. It was estimated that in Europe 19,600 patients with metastatic melanoma are treated, and 5238 (27%) do not have access to recommended first-line therapy. Significant correlation was found between human development index (HDI, UNDP report 2015), (r = 0.662; p < 0.001), health expenditure per capita (r = 0.695; p < 0.001) and the Mackenbach score of health policy performance (r = 0.765; p < 0.001) with the percentage of patients treated with innovative medicines and a number of reimbursed medicines. CONCLUSIONS: Great discrepancy exists in metastatic melanoma treatment across Europe. It is crucial to increase the awareness of national and European policymakers, oncological societies, melanoma patients' associations and pharma industry.

Innovative strategies to improve diabetes outcomes in disadvantaged populations.

Diabetes disproportionately affects disadvantaged populations. Eighty percent of deaths directly caused by diabetes occurred in low- and middle-income countries. In high-income countries, there are marked disparities in diabetes control among racial/ethnic minorities and those with low socio-economic status. Innovative, effective and cost-effective strategies are needed to improve diabetes outcomes in these populations. Technological advances, peer educators and community health workers have expanded methodologies to reach, educate and monitor individuals with diabetes. In the present manuscript we review the outcomes of these strategies, and describe the barriers to and facilitators of these approaches for improving diabetes outcomes.

Quantitative analysis of technological innovation in minimally invasive surgery.

BACKGROUND: In the past 30 years surgical practice has changed considerably owing to the advent of minimally invasive surgery (MIS). This paper investigates the changing surgical landscape chronologically and quantitatively, examining the technologies that have played, and are forecast to play, the largest part in this shift in surgical practice. METHODS: Electronic patent and publication databases were searched over the interval 1980-2011 for ('minimally invasive' OR laparoscopic OR laparoscopy OR 'minimal access' OR 'key hole') AND (surgery OR surgical OR surgeon). The resulting patent codes were allocated into technology clusters. Technology clusters referred to repeatedly in the contemporary surgical literature were also included in the analysis. Growth curves of patents and publications for the resulting technology clusters were then plotted. RESULTS: The initial search revealed 27,920 patents and 95,420 publications meeting the search criteria. The clusters meeting the criteria for in-depth analysis were: instruments, image guidance, surgical robotics, sutures, single-incision laparoscopic surgery (SILS) and natural-orifice transluminal endoscopic surgery (NOTES). Three patterns of growth were observed among these technology clusters: an S-shape (instruments and sutures), a gradual exponential rise (surgical robotics and image guidance), and a rapid contemporaneous exponential rise (NOTES and SILS). CONCLUSION: Technological innovation in MIS has been largely stagnant since its initial inception nearly 30 years ago, with few novel technologies emerging. The present study adds objective data to the previous claims that SILS, a surgical technique currently adopted by very few, represents an important part of the future of MIS.

Global update: USA.

The NIH, which has an annual budget of over US$31 billion, is the world's largest biomedical research agency and is a major strength for science in the USA. Despite the political nature of stem cell research, this area of science has flourished across the country. In 2010, the NIH funded approximately US$1.3 billion in stem cell research. According to the ISI Web of Science, more than 4000 US-authored stem cell publications were produced in 2010 - approximately 40% of the world total. The average citation rate was 4.12 per article, with six articles amassing 100 citations in less than 18 months after release.

Global update: Scotland.

Scotland is shaping up as Europe's 'go-to' place to translate cell therapy research from the laboratory into the clinic. While continuing to punch above its weight in fundamental research, the resources for translating research to clinical application are perhaps its main attraction for the international stem cell research community.

The Society for Clinical Trials opposes US legislation to permit marketing of unproven medical therapies for seriously ill patients.

The proposed Bill S.1956 is a bad law. Wide early access to minimally tested treatments cannot be expected to lead to better or more compassionate care of the seriously ill patient. The long history of medicine is replete with treatments that initially seemed promising to patients, doctors, and especially to their own inventors, but which careful study revealed to be worthless or harmful. We understand the desperate plight of these patients and the importance of hope for them and their families. However, although the proposed law is portrayed as an effort to help patients in desperate need of treatment, in fact its effect would be to undermine the system of scientifically valid testing of new drugs that has been a bulwark of health care for several decades. The effect of the law would be to provide many more possible choices of treatment but much less information upon which to make the choice. Patients would be very unlikely to end up receiving an effective treatment. They would be much more likely to receive useless or possibly harmful treatments in the last days of their lives. The Society for Clinical Trials strongly opposes this legislation.