Showing papers by "Louis P. Garrison published in 2010"

Can't get no satisfaction? Will pay for performance help?: toward an economic framework for understanding performance-based risk-sharing agreements for innovative medical products.

Abstract: This article examines performance-based risk-sharing agreements for pharmaceuticals from a theoretical economic perspective. We position these agreements as a form of coverage with evidence development. New performance-based risk sharing could produce a more efficient market equilibrium, achieved by adjustment of the price post-launch to reflect outcomes combined with a new approach to the post-launch costs of evidence collection. For this to happen, the party best able to manage or to bear specific risks must do so. Willingness to bear risk will depend not only on ability to manage it, but on the degree of risk aversion. We identify three related frameworks that provide relevant insights: value of information, real option theory and money-back guarantees. We identify four categories of risk sharing: budget impact, price discounting, outcomes uncertainty and subgroup uncertainty. We conclude that a value of information/real option framework is likely to be the most helpful approach for understanding the costs and benefits of risk sharing. There are a number of factors that are likely to be crucial in determining if performance-based or risk-sharing agreements are efficient and likely to become more important in the future: (i) the cost and practicality of post-launch evidence collection relative to pre-launch; (ii) the feasibility of coverage with evidence development without a pre-agreed contract as to how the evidence will be used to adjust price, revenues or use, in which uncertainty around the pay-off to additional research will reduce the incentive for the manufacturer to collect the information; (iii) the difficulty of writing and policing risk-sharing agreements; (iv) the degree of risk aversion (and therefore opportunity to trade) on the part of payers and manufacturers; and (v) the extent of transferability of data from one country setting to another to support coverage with evidence development in a risk-sharing framework. There is no doubt that--in principle--risk sharing can provide manufacturers and payers additional real options that increase overall efficiency. Given the lack of empirical evidence on the success of schemes already agreed and on the issues we set out above, it is too early to tell if the recent surge of interest in these arrangements is likely to be a trend or only a fad.

Rewarding Value Creation to Promote Innovation in Oncology: The Importance of Considering the Global Product Life Cycle

Abstract: Disclosures: Louis P. Garrison, Jr.: Honoraria: Roche Pharmaceuticals, Novartis Pharmaceuticals; Research funding/contracted research: Genentech, Inc. The content of this article has been reviewed by independent peer reviewers to ensure that it is balanced, objective, and free from commercial bias. No financial relationships relevant to the content of this article have been disclosed by the independent peer reviewers.

Preference values associated with stage III colon cancer and adjuvant chemotherapy

Abstract: To elicit preference values for health states associated with Stage III colon cancer (CRC) and to explore the effect of neuropathy associated with current adjuvant treatment. We used time trade-off (TTO) techniques to elicit preferences from 49 CRC patients and 49 community members. We elicited preferences for 7 health states: remission; adjuvant therapy with no, mild, moderate, and severe neuropathy; metastatic stable; and metastatic progressive disease. Mean TTO values were adjusted for the covariates age, education, and current health. Patients’ adjusted mean TTO value for remission was 0.83; adjuvant chemotherapy health states ranged from 0.48 to 0.61. Significant differences were observed for both patient and community groups between TTO for remission and all adjuvant health states (P < 0.001), and between adjuvant therapy with no neuropathy and metastatic health states (P ≤ 0.001). Across all health states, patients’ values were on average 0.12 higher than community members (P < 0.05). The findings highlight the trade-offs between the disutility of adjuvant treatment, the higher utility of remission, and the severe utility loss during metastatic disease. The preference values obtained from this study will be useful for informing patients’ treatment decisions and payer cost-utility analyses of adjuvant treatment for colon cancer.

A review of data needed to parameterize a dynamic model of measles in developing countries

Abstract: Dynamic models of infection transmission can project future disease burden within a population. Few dynamic measles models have been developed for low-income countries, where measles disease burden is highest. Our objective was to review the literature on measles epidemiology in low-income countries, with a particular focus on data that are needed to parameterize dynamic models. We included age-stratified case reporting and seroprevalence studies with fair to good sample sizes for mostly urban African and Indian populations. We emphasized studies conducted before widespread immunization. We summarized age-stratified attack rates and seroprevalence profiles across these populations. Using the study data, we fitted a "representative" seroprevalence profile for African and Indian settings. We also used a catalytic model to estimate the age-dependent force of infection for individual African and Indian studies where seroprevalence was surveyed. We used these data to quantify the effects of population density on the basic reproductive number R0. The peak attack rate usually occurred at age 1 year in Africa, and 1 to 2 years in India, which is earlier than in developed countries before mass vaccination. Approximately 60% of children were seropositive for measles antibody by age 2 in Africa and India, according to the representative seroprevalence profiles. A statistically significant decline in the force of infection with age was found in 4 of 6 Indian seroprevalence studies, but not in 2 African studies. This implies that the classic threshold result describing the critical proportion immune (pc) required to eradicate an infectious disease, pc = 1-1/R0, may overestimate the required proportion immune to eradicate measles in some developing country populations. A possible, though not statistically significant, positive relation between population density and R0 for various Indian and African populations was also found. These populations also showed a similar pattern of waning of maternal antibodies. Attack rates in rural Indian populations show little dependence on vaccine coverage or population density compared to urban Indian populations. Estimated R0 values varied widely across populations which has further implications for measles elimination. It is possible to develop a broadly informative dynamic model of measles transmission in low-income country settings based on existing literature, though it may be difficult to develop a model that is closely tailored to any given country. Greater efforts to collect data specific to low-income countries would aid in control efforts by allowing highly population-specific models to be developed.

A Flexible Approach To Evidentiary Standards For Comparative Effectiveness Research

Abstract: The Patient Protection and Affordable Care Act established a new Patient-Centered Outcomes Research Institute to identify and address research priorities for comparative effectiveness research. Among its many responsibilities, the institute has been charged with setting priorities, developing methodological standards, and communicating research results to decision makers. In this paper we consider how the institute can support the different standards for acceptable evidence used by various government agencies, providers, patients, and other decision makers. We argue that the public interest will best be served if the institute develops a balanced and flexible approach to deciding which types of studies to fund.

Regulatory Benefit-Risk Assessment and Comparative Effectiveness Research

Abstract: Over the past 5 years, we have witnessed growing interest in both comparative effectiveness research (CER) and regulatory benefit-risk assessment (BRA) Both deal with benefits and harms, although at different stages of the product lifecycle There are growing pressures for a more systematic and quantitative approach to regulatory BRA However, there is also a need for CER–beyond the evidence that can reasonably be generated during prelaunch product development Important regulatory and policy questions include the following: What would be a level playing field across disease areas and companies? Who should bear the costs of these studies? What role can benefit-risk modelling play? What is the value of research and how is it related to the prevalence of disease? What is the relationship between uncertainty and the value of evidence? We need to recognize the lifecycle nature of evidence generation, moving from the regulatory setting to the real world and affecting potentially hundreds of thousands, or even millions, of patients worldwide We need to emphasize not only the public goods nature of information embedded in innovations, but also that it is global Finally, we need to more systematically explore the benefits and costs of gathering further information–the value of research–recognizing that doing this requires a model or methodology, which we have, for systematically appraising our current state of knowledge and what could be gained from further research All said, it would seem that BRA and CER should be neither strangers nor strange bedfellows, but may need to be coaxed into being bedfellows

Pharmaceutical interventions for mitigating an influenza pandemic: modeling the risks and health-economic impacts

Abstract: Model-based analyses built on burden-of-disease and cost-effectiveness theory predict that pharmaceutical interventions may efficiently mitigate both the epidemiologic and economic impact of an influenza pandemic. Pharmaceutical interventions typically encompass the application of (pre)pandemic influenza vaccines, other vaccines (notably pneumococcal), antiviral treatments and other drug treatment (e.g., antibiotics to target potential complications of influenza). However, these models may be too limited to capture the full macro-economic impact of pandemic influenza. The aim of this article is to summarize current health-economic modeling approaches to recognize the strengths and weaknesses of these approaches, and to compare these with more recently proposed alternative methods. We conclude that it is useful, particularly for policy and planning purposes, to extend modeling concepts through the application of alternative approaches, including insurers' risk theories, human capital approaches and sectoral and full macro-economic modeling. This article builds on a roundtable meeting of the Pandemic Influenza Economic Impact Group that was held in Boston, MA, USA, in December 2008.

Economic evaluation of capecitabine as adjuvant or metastatic therapy in colorectal cancer

Abstract: Capecitabine, an oral prodrug of 5-fluorouracil, is indicated for adjuvant treatment in patients with Dukes' C colon cancer and for subsequent lines in metastatic colorectal cancer. The aim of this article is to review the literature on the economics of capecitabine for the treatment of colon cancer. A systematic review was conducted to search for articles published from January 2003 to December 2009 that met the inclusion criteria. For abstracts that were considered acceptable, full-text articles were then reviewed. Of the 42 potential studies that were identified, 13 original studies (16 publications) met the inclusion criteria. To date, the economic evaluation literature has consistently projected or found that capecitabine is not only a cost-effective treatment for adjuvant or for metastatic colorectal cancer (i.e., providing good value for money) but, furthermore, would actually be cost saving in the majority of country settings.

The economic value of reducing medication dosing frequency with drug delivery technologies: an evidence assessment

Abstract: Objective: To critically evaluate published cost-effectiveness studies of novel drug products requiring less-frequent medication dosing compared to conventional formulations of the same drug substance.Methods: A search was conducted in the Medline and Embase databases for cost-effectiveness studies published before May 2009 that compared two or more drug delivery technologies formulated with the same active drug substance. The Quality of Health Economic Studies (QHES) grading criteria for cost-effectiveness studies was applied to the selected publications.Results: The literature search identified approximately 907 articles of which six cost-effectiveness studies met the inclusion criteria. The studies spanned four chronic conditions, were conducted from various international perspectives and used decision-analytic models to project economic outcomes. The base-case results of all six studies indicated that the drug product with sustained therapeutic efficacy was either more effective and less costly (‘domi...

Economic Issues and Genomic Medicine

Abstract: The era of genomic and personalized medicine offers significant promise for the development of novel healthcare approaches and technologies that will ultimately improve patients’ quality of life and life expectancy. As with any new healthcare technology, questions arise as to their potential budgetary impact and cost-effectiveness. Furthermore, questions have arisen in regard to the economic incentives to develop these technologies. In this chapter, we discuss economic evaluation of genomic medicine, particularly in light of next-generation technologies that promise to drastically reduce whole-genome sequencing costs. We examine the challenges in pharmacogenomics of economic incentives for developing diagnostics linked to therapeutics. The content of this chapter will help readers understand the multidisciplinary nature of the factors that will influence the value of genomic medicine, provide a framework for assessing these factors in a systematic, quantitative fashion, and highlight the importance of value-based reimbursement for genomic technologies for the future of genomic and personalized medicine.

Data Investigation of Bariatric Surgery Outcome and Economic Savings

Abstract: : This study sought to (1) define the clinical impact and economic burden of bariatric surgical procedures, and (2) estimate the cost-effectiveness and budgetary impact of obesity treatments when compared to no surgical intervention. We developed a cost-effectiveness model and a payer-based budget and fiscal impact tool to compare bariatric surgical procedures to non-operative approaches for maorbid obesity. Use of these economic models based on data from the Department of Defense (DOD) population found that all evaluated surgical interventions were cost-effective compared to non-surgical interventions. These economic assessments models can inform helath policy decisions related to obesity.