Ruth Lopert

Responding to Smith et al. (Nature, 2014), this paper argues that for medical use, faecal microbiota transplantation (FMT) should be considered a sui generis biological drug, rather than a tissue. Smith and colleagues' thesis is based on possible undesirable economic consequences of this designation--not on its scientific and conceptual basis. The faecal transplant (including gut microbiota, metabolites, mucus, human cells, viruses, fungi, etc.) is not a tissue; it is of topographic--not cellular--human origin. We consider the donor a bioreactor, producing the faecal substrate of therapeutic interest. The debate is of singular importance as the FDA considers FMT a drug and released a new guidance for public consultation in February 2014, whereas to date the European Medicines Agency has not promulgated its position. The UK's National Institute for Heath and Care Excellence does not consider FMT to involve the transplantation of body tissue, and in March 2014 the French regul...

Analyzes British, Australian, and German health systems' policies on covering expensive end-of-life drugs while maintaining equity and efficiency, such as requiring evidence of comparative clinical and cost-effectiveness. Outlines challenges for the U.S

Objectives The aim of the study was to estimate the potential savings to the Pharmaceutical Benefits Scheme (PBS) and the Repatriation Pharmaceutical Benefits Scheme (RPBS) in 2015-16 if biosimilar versions of selected biologic medicines (biologics) had been available and listed on the PBS.Methods The research involved retrospective analysis of Australian Medicare expenditure data and PBS price data from 2015-16 for biologics, for which biosimilar competition may be available in future, listed on the PBS.Results Australian Government expenditure on biologics on the PBS and RPBS was estimated at A$2.29 billion dollars in 2015-16. If biosimilar versions of these medicines had been listed on the PBS in 2015-16, at least A$367million dollars would have been saved in PBS and RPBS subsidies. Modelling based on price decreases following listing of biosimilars on the PBS suggests that annual PBS outlays on biologics could be reduced by as much as 24% through the timely introduction of biosi...

Misuse or misunderstanding of medication information is a common and costly problem in the U.S. The risks of misunderstanding medication information are compounded for the large and growing population of individuals with limited English proficiency that often lacks access to this information in their own language. This paper examines practices related to translation of medication information in the European Union that may serve as a model for future U.S. policy efforts to improve the quality and availability of medication information for individuals with limited English proficiency.

In Australia, most prescription drugs are subsidized through the Pharmaceutical Benefits Scheme (PBS), one of several government programs in which evidence-based decision making is applied to the funding of health technologies. PBS processes are intended to ensure "value for money" for the Australian taxpayer and to support affordable, equitable access to prescription medicines; they are not intended as a mechanism for cost containment. The inclusion of a drug on the national formulary depends on the recommendation of the Pharmaceutical Benefits Advisory Committee (PBAC), which considers not only the comparative effectiveness but also the comparative cost-effectiveness of drugs proposed for listing. While some decisions have been controversial, the PBS retains strong public support. Moreover, evidence does not suggest that the consideration of cost-effectiveness has created a negative environment for the drug industry: Australia has a high penetration of patented medicines...

... London Due for review: 2005 Council Report CR102 April 2002 Page 2. 2 Membership of the Working Group Dr Gillian Mezey (Chairman) South-West London and St George's Mental Health NHS Trust, London Dr Madelyn Hicks South London and Maudsley NHS Trust, London ...

The final text of the Trans Pacific Partnership Agreement (TPP), agreed between the 12 negotiating countries in 2016, included a suite of intellectual property provisions intended to expand and extend pharmaceutical company exclusivities on medicines. It drew wide criticism for including such provisions in an agreement that involved developing countries (Vietnam, Peru, Malaysia, Mexico, Chile and Brunei Darussalam) because of the effect on delaying the introduction of low-cost generics. While developing nations negotiated transition periods for implementing some obligations, all parties would have eventually been expected to meet the same standards had the TPP come into force. While the TPP has stalled following US withdrawal, there are moves by some of the remaining countries to reinvigorate the agreement without the United States. The proponents may seek to retain as much as possible of the original text in the hope that the United States will re-join the accord in future. This ar...

Medication use may be a target for quality improvement, cost containment, and research. We aimed to identify medication classes associated with the highest expenditures among pediatric Medicaid enrollees and to characterize the demographic, clinical, and health service use of children prescribed these medications. Retrospective, cross-sectional study of 3 271 081 Medicaid-enrolled children. Outpatient medication spending among high-expenditure medication classes, defined as the 10 most expensive among 261 mutually exclusive medication classes, was determined by using transaction prices paid to pharmacies by Medicaid agencies and managed care plans among prescriptions filled and dispensed in 2013. Outpatient medications accounted for 16.6% of all Medicaid expenditures. The 10 most expensive medication classes accounted for 63.9% of all medication expenditures. Stimulants (amphetamine-type) accounted for both the highest proportion of expenditures (20.6%) and days of medication use (1...

Policymakers in high-, low-, and middle-income countries alike face challenging choices about resource allocation in health. Economic evaluation can be useful in providing decision makers with the best evidence of the anticipated benefits of new investments, as well as their expected opportunity costs-the benefits forgone of the options not chosen. To guide the decisions of health systems effectively, it is important that the methods of economic evaluation are founded on clear principles, are applied systematically, and are appropriate to the decision problems they seek to inform. The Bill and Melinda Gates Foundation, a major funder of economic evaluations of health technologies in low- and middle-income countries (LMICs), commissioned a "reference case" through the International Decision Support Initiative (iDSI) to guide future evaluations, and improve both the consistency and usefulness to decision makers. The iDSI Reference Case draws on previous insights from the Wor...

The inclusion of elevated standards of intellectual property (IP) protection in the recently negotiated Trans-Pacific Partnership (TPP) agreement has raised serious public health concerns regarding access to medicines. A lesser-known trade agreement under negotiation in the Asia-Pacific region is the Regional Comprehensive Economic Partnership (RCEP). Framed as an attempt to reassert ASEAN's position in response to the United States-led TPP, RCEP includes key players China and India as well as several low- and middle-income countries (LMICs). Leaked drafts of IP provisions proposed by Japan and South Korea raise similar concerns in the Asia-Pacific region. This article identifies TRIPS (Trade Related Aspects of Intellectual Property Rights agreement)-Plus provisions in leaked negotiating texts and examines their implications for LMICs that are not also parties to the TPP: Cambodia, Indonesia, Laos, Myanmar, the Philippines, Thailand, China, and India. We find that higher levels of IP protection delay the market entry of generic medicines, giving rise to increased costs to governments and reduced access to essential medicines. The article concludes that the public health community should recognize risks inherent in trade agreements that promote expansions of IP rights and engage with governments to ensure that public health is adequately and explicitly protected in trade and investment negotiations.

In the Trans Pacific Partnership (TPP) Agreement negotiations, the USA successfully pursued intellectual property (IP) provisions that will affect the affordability of medicines, including anti-retrovirals (ARV) for HIV. Vietnam has the lowest GDP per capita of the 12 TPP countries and in 2013 provided ARVs for only 68% of eligible people living with HIV. Using the current Vietnamese IP regime as our base case, we analysed the potential impact of a regime making full use of legal IP flexibilities, and one based on the IP provisions of the final, agreed TPP text. Results indicate that at current funding levels 82% of Vietnam's eligible people living with HIV would receive ARVs if legal flexibilities were fully utilised, while as few as 30% may have access to ARVs under the TPP Agreement - more than halving the proportion currently treated.

We examined potential cost savings to the Medicare program associated with a “value based” approach to prescription drug benefit design (VBBD). Adherence to statin therapy has been demonstrated to reduce medical costs for Medicare beneficiaries with diabetes. Reductions in drug plan out of pocket (OOP) prices may increase medication adherence, increasing total and plan spending, but with cost offsets for

Drugs may be the most frequent single cause of delirium, and very often they are a critical element in a multifactorial aetiology. While delirium may be precipitated by virtually any drug, certain classes of drugs are more commonly implicated. Effective management of drug-induced delirium involves recognition, cessation or dosage reduction of the causative drug(s), and initiation of reorientation strategies and supportive medical care. Specific "antidotes' are appropriate in only a few limited cases. Drug treatment aimed at sedation should be introduced for specific indications, such as aggression, risk of harm to self or others, hallucinations, patient distress, and where compliance with therapy or procedures is essential. Certain benzodiazepines (diazepam, lorazepam, midazolam) and/or haloperidol may be the most appropriate choices in these circumstances. Primary prevention requires the prescription of alternative lower risk medications and the minimisation of polypharmacy. Secondary prevention may be achieved through improved recognition of the condition.

ABSTRACT Health policy makers concerned with explosive growth in US Medicare expenditures generally belong to the Willy Sutton school of management—go where the money is. But focusing on costly patients and expensive services may divert attention from potentially significant savings at the opposite end of the spectrum. The aim of this paper is to identify mutable factors that are predictive of low spending without compromising receipt of needed services. Data for the study come from the 2002 Medicare Current Beneficiary Survey. The sample N=8,455 after exclusion of respondents with missing values for key variables. We divide the sample into 5 equal size quintiles based on cumulative medical spending and characterize beneficiaries in each decile by the following domains: (1) health indicators (self reported health, ADL limitations, presence of common chronic diseases), (2) demographic factors (age, gender, race, marital status, education), (3) access indicators (income, rural residence, Medicare supplemental health insurance, prescription coverage, usual source of care), (4) health habits (smoking, preventive health measures, obesity), (5) health care seeking/avoidance behaviors (attitudes toward providers), and (6) health shocks (health significantly worse or better than a year ago). As expected, the health indicators reflect few health problems and high levels of self-reported health in the lowest spending quintile with steadily worsening health status thereafter. The opposite is seen in health insurance and drug coverage, with the lowest rates in quintile 1 (84% and 60%, respectively) rising to 95% and 78%, respectively, in quintile 5. However, the most startling findings relate to a cluster of sociodemographic factors (never married, nonwhite and/or Hispanic, poverty level income, and less than high school education) that are disproportionately represented in the lowest and highest deciles. Never married beneficiaries represent 22% of quintile 1, 14% of quintile 3, and 17% of quintile 5. Nonwhites are 25% of quintile 1, 16% in quintile 3, and 21% in quintile 5. Beneficiaries with no high school education are 17% of quintile 1, 13% of quintile 3, and 18% of quintile 5. These U shaped distributions suggest the presence of strong mitigating factors that initially predict low spending but after a tipping point work in the opposite direction. To better understand these relationships we subset two socioeconomic status groups defined, respectively, as low SES (under 150% of FPL and less than a high school education), and high SES (above 300% FPL and some post-graduate education). We then estimated piecewise regression models of medical spending by quintile to accommodate sign switches among the sociodemographic variables. The high SES group had a much higher level of positive health investments (flu shot, usual source of care) and lower levels of negative health investments (smoking, obesity, care avoidance behaviors) compared to the low SES group throughout the spending range. The regression results showed that positive health investments were associated with higher spending in the lowest quintile but then reduced spending in the upper quintiles. The opposite was true for negative health investments. These findings suggest that improving positive health behaviors will generate future savings in Medicare, but longitudinal analysis is necessary to prove the point.

IN ANY DISCUSSION of key pharmaceutical policy issues, Australia's National Medicines Policy (NMP) is an important touchstone of which Australians can be justly proud. Those familiar with the stalled evolution of the Canadian National Pharmaceuticals Strategy and the uneven provincial patchwork of pharmaceutical coverage in Canada for example, may wonder why it is that a country with longstanding universal health

To (1) measure utilization of and adherence to heart failure medications and (2) assess whether better adherence is associated with lower Medicare spending. Pooled cross-sectional design using six 3-year cohorts of Medicare beneficiaries with congestive heart failure (CHF) from 1997 through 2005 (N = 2204). Adherence to treatment was measured using average daily pill counts. Bivariate and multivariate methods were used to examine the relationship between medication adherence and Medicare spending. Multivariate analyses included extensive variables to control for confounding, including healthy adherer bias. Approximately 58% of the cohort were taking an angiotensin-converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB), 72% a diuretic, 37% a beta-blocker, and 34% a cardiac glycoside. Unadjusted results showed that a 10% increase in average daily pill count for ACE inhibitors or ARBs, beta-blockers, diuretics, or cardiac glycosides was associated with reductions in Me...

Several Central and Eastern European (CEE) countries require cost-utility analyses (CUAs) to support reimbursement formulary listing. However, CUAs informed by local evidence are often unavailable, and the cost-effectiveness of the several currently reimbursed biologicals is unclear. To estimate the cost-effectiveness as multiples of per capita GDP/quality adjusted life years (QALY) of four biologicals (infliximab, etanercept, adalimumab, golimumab) currently reimbursed in six CEE countries in six inflammatory rheumatoid and bowel disease conditions. Systematic literature review of published cost-utility analyses in the selected conditions, using the United Kingdom (UK) as reference country and with study selection criteria set to optimize the transfer of results to the CEEs. Prices in each CEE country were pro-rated against UK prices using purchasing power parity (PPP)-adjusted per capita GDP, and local GDP per capita/QALY ratios estimated. Central and Eastern European countries list prices were 144-333% higher than pro rata prices. Out of 85 CUAs identified by previous systematic literature reviews, 15 were selected as a convenience sample for estimating the cost-effectiveness of biologicals in the CEE countries in terms of per capita GDP/QALY. Per capita GDP/QALY values varied from 0.42 to 6.4 across countries and conditions (Bulgaria: 0.97-6.38; Czech Republic: 0.42-2.76; Hungary: 0.54-3.54; Poland: 0.59-3.90; Romania: 0.77-5.07; Slovakia: 0.55-3.61). While results must be interpreted with caution, calculating pro rata (cost-effective) prices and per capita GDP/QALY ratios based on CUAs can aid reimbursement decision-making in the absence of analyses using local data.

For medicines – especially biologics – competition dynamics are heavily dependent on the extent to which substi- tution is permitted at pharmacy (both for the initial pres- cription and for any subsequent refill). Today the choice of a particular brand at treatment initiation has significant consequences. Are the patient and the public program that funds their care bound to a particular treatment? Is the market really ‘open’ once patents have expired? Pro- moting competition in the world of often very expensive biologics raises many issues in regulating practice while at the same time avoiding both public health and political risks, and anti-competitive practices. While the intrinsic quality of biosimilars approved and used in Europe is no longer a point of discussion, as regulations are developed and implemented in the United States, the debate between competing manufacturers has moved onto substitution and, with it, the potential for intensive price competition. This paper propose...

2007112034laboratorio_20071121 Los debates en curso en el sector farmacéutico acerca de la propiedad intelectual, 1, 2 de fijación de precios y reembolso, 3, 4 y las inversiones públicas de investigación 5 tienen un denominador común: la búsqueda de la innovación. Sin ...