Pharmaceuticals cost-effectiveness analysis

In Chapter 4 Aidan Hollis examines three proposals in considerable detail. The first is an Advanced Purchase Commitment by sponsors, who offer an explicit subsidy in advance for innovative products. The subsidy offer includes a fixed-dollar amount per unit as well as a commitment to purchase a specific number of units at that price. The second proposal is that sponsors pay annual rewards based on the therapeutic effectiveness of innovative drugs. The third approach is to offer a patent extension on patented products to pharmaceutical companies if they successfully developed a vaccine for a disease such as HIV/AIDS that is highly prevalent, particularly in some low-income countries. Hollis concludes that the third approach is an extremely inefficient way to reward innovation. By contrast, the second approach could correct the market failure directly by rewarding innovative drugs according to their therapeutic effectiveness, which is measurable by cost-effectiveness analysis, a topic discussed later in greater detail in Chapters 10 and 11. 

To decide on the allocation of public and private resources, some form of cost-effectiveness analysis is used in many countries, large and small (Drummond, Chapter 11). The single most common type of medical care to which this method is applied is the use of pharmaceutical products. Sometimes the decision involves new uses for existing products developed and produced in the country making the decision, and sometimes it involves products imported from abroad. Sometimes as well the decision involves the potential allocation of resources to a new product if brought to market, and often public funds play a role in such innovation. 

George, B., A. Harris, andA. Mitchell. 2001. Cost-Effectiveness Analysis and the Consistency of Decision-Making Evidence from Pharmaceutical Reimbursement in Australia (1991 to 1996). PharmacoEconomics 19(11) 1103-1109. 

Second, a full analysis should go beyond the identification of cost. Only if the safety and effectiveness of two pharmaceutical agents are equivalent will cost alone determine the choice of therapy. Cost-effectiveness analysis requires that cost be weighed against effectiveness and that when two or more alternatives are being compared, the additional cost... 

U.S. pharmaceutical industry s challenge to cost-effectiveness analysis... 

Such a treaty or provisions also facilitate recognition that the evolving process of scientific cost-effectiveness analysis is best placed to normatively legitimize claims for pharmaceutical "innovation," by gatekeeping, as a global public good, entry to the class of truly "essential" medicines. 

Etemad LR, Hay JW. 2003. Cost-effectiveness analysis of pharmaceutical care in a medicare dmg benefit program. Value Health 6 425. 

Hyphenated analytical techniques such as LC-MS, which combines liquid chromatography and mass spectrometry, are well-developed laboratory tools that are widely used in the pharmaceutical industry. Eor some compounds, mass spectrometry alone is insufficient for complete structural elucidation of unknown compounds nuclear magnetic resonance spectroscopy (NMR) can help elucidate the structure of these compounds (see Chapter 20). Traditionally, NMR experiments are performed on more or less pure samples, in which the signals of a single component dominate. Therefore, the structural analysis of individual components of complex mixtures is normally time-consuming and less cost-effective. The... 

My claim is that, because virtually all analyses of drugs from a societal perspective use a cost that is too high (relative to the appropriate one), more pharmaceuticals may be cost effective than is generally concluded under current measurement methods. The usual methods can, with some modifications, be made to more closely approximate the correct analysis. 

The basic approach for performing economic assessments of pharmaceutical products, as discussed above, has been adapted from the general methodology for cost-effectiveness and cost-benefit analysis. 

Establishing the value of a new pharmaceutical can be done through a cost-effectiveness ratio, where the costs are compared with currently accepted therapy and the effect is expressed in natural units such as life-years gained or disability-free days. A cost-utility analysis uses QALYs as the expression of the drug s effect, which is a measure that incorporates all the outcomes as well as all the costs of the drug treatment. Such a broad-based measure captures how much improved the patient s life becomes as a result of the treatment and at what cost. Quality-adjusted life-years can be viewed as life-years gained,... 

The NIR region is of great interest for pharmaceutical applications. NIRS is fast, nondestructive, and cost effective. Samples require no preparation and can be measured as such, intact and available for further analysis. NIRS can be performed in-, on-, and offline. Also, glass fiber optics can be used to perform remote analysis, thus bringing radiation directly to the sample. Many more advantages can be cited when considering the practical use of NIR in a pharmaceutical process, depending on the particular objective. 

Liquid chromatography/mass spectrometry (LC/MS)-based techniques provide unique capabilities for pharmaceutical analysis. LC/MS methods are applicable to a wide range of compounds of pharmaceutical interest, and they feature powerful analytical figures of merit (sensitivity, selectivity, speed of analysis, and cost-effectiveness). These analytical features have continually improved, resulting in easier-to-use and more reliable instruments. These developments coincided with the pharmaceutical industry s focus on describing the collective properties of novel compounds in a rapid, precise, and quantitative way. As a result, the predominant pharmaceutical sample type shifted from nontrace/pure samples to trace mixtures (i.e., protein digests, natural products, automated synthesis, bile, plasma, urine). The results of these developments have been sig-... 

An IPC-ESI-MS/MS method using volatile perfluorinated carboxylic adds as IPRs added directly to the sample solution (not incorporated into the mobile phase) was developed for the quantitative assay of methadone in human plasma. This cost-effective strategy enhanced the efficiency of separation and minimized ion suppression because no IPR was present in the eluent [103]. Table 13.1 lists other potential uses of IPC in the pharmaceutical and clinical analysis fields. 

An impressive diversity of mass analyzers are utilized in modem analytical instrumentation. An overview of the common mass spectrometer analyzers follows, with particular emphasis on linear quadrupole mass analyzers, quadrupole ion traps, and time-of-flight mass analyzers, as they arguably constitute the quantitative MS workhorses of the pharmaceutical industry. The description of alternate analyzer systems should provide a framework in which the utility of these three particular systems provides the most cost-effective analytical mass spectrometer systems for pharmaceutical analysis. 

Profile This privately held company was founded in 1993 to integrate advanced technologies in structure-based design, combinatorial chemistry, and chemi-informatics for the cost-effective discovery of orally active pharmaceuticals. The company has developed a system capable of generating new drugs through computer-controlled robotic synthesis and analysis of chemical libraries. Current drug discovery efforts are focused on cardiovascular disease and cancer. 

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