Uniform system for determining manufacturing costs

Therefore, it was decided by the Association to have a Manual prepared outlining a Uniform System for determining Manufacturing Costs, containing, as one of its major features, the Unit Method of calculating Manufacturing Overhead. The system presented herewith was designed after considerable study of the problems and requirements of pharmaceutical manufacturers. It is the result of six years work with approximately twenty pharmaceutical manufacturers, representing a good cross section of the average sized houses. The purpose of this Manual is to present a simple, uniform method of computing manufacturing costs and to act as a guide for those companies desiring to use such a method. Manufacturing Costs only are dealt with here, that is, the cost of producing products and placing them on the shelf in the finished stock room. Distribution Costs have not been studied sufficiently by the Association to produce results which can be included here. This Manual is written with the idea that it will be read and used by people who are thoroughly familiar with the processes involved in the manufacture of pharmaceuticals and by members of their organizations who have a working knowledge of accounting, even though they may not be expert cost accountants. It is prepared with the knowledge that most of the companies in the Association are average sized organizations, who really do not need an elaborate system of cost accounting. Therefore, a simple system is outlined herein requiring very little clerical labor for its maintenance. The adoption of the methods presented herein by the Association will give the membership reliable manufacturing cost data. In addition, it will make the comparison of costs at Association meetings a more valuable and helpful procedure

WHO informal consultation on revision of guidelines on evaluation of similar biotherapeutic products, virtual meeting, 30 June – 2 July 2021

Publisher Copyright: © 2022The WHO informal consultation was held to promote the revision of WHO guidelines on evaluation of similar biotherapeutic products (SBPs) adopted by the Expert Committee on Biological Standardization (ECBS) in 2009. It was agreed in the past consultations that the evaluation principles in the guidelines are still valid, but a review was recommended to provide more clarity and case-by-case flexibility. The opportunity was therefore taken to review the experience and identify areas where the current guidance could be more permissive without compromising its basic principles, and where additional explanation could be provided regarding the possibility of reducing the amount of data needed for regulatory approval. The meeting participants applauded the leading role taken by the WHO in providing a much-needed streamlined approach for development and evaluation of SBPs which will provide efficient and cost-effective product development and increase patient access to treatments. It was recognized that the principles as currently described in the draft WHO guidelines are based on sound science and experience gained over the last fifteen years of biosimilar approvals. However, since these guidelines when finalised will constitute the global standard for biosimilar evaluation and assist national regulatory authorities in establishing revised guidance and regulatory practice in this complex area, it was felt that further revision and clarity on certain perspectives in specific areas was necessary to dispel uncertainties arising in the current revised version. This report describes the principles in the draft guidelines, including topics discussed and consensus reached.Peer reviewe

The status of clinical trials: Cause for concern

<p>Abstract</p> <p>Background</p> <p>Americans see clinical research as important, with over 15 million American residents participating in NIH-sponsored studies in 2008 and growing yearly.</p> <p>Methods</p> <p>Documents reporting NIH supported Clinical Research projects were reviewed.</p> <p>Results</p> <p>When compared with other studies, the number of interventional Phase III and Phase IV trials have decreased from 20% to 4.4% from 1994-2008.</p> <p>Conclusions</p> <p>This finding most likely has occurred for several reasons. One reason is that the physician lacks an infrastructure for designing and carrying out trials. This lack is because of an absence of a coordinated effort to train clinical trialists. It is clear that the Nation needs a more purposeful approach to developing and maintaining the infrastructure for designing and conducting clinical trials. Building it de novo trial by trial is profoundly inefficient, to say nothing about time consuming and error prone.</p

Trends in the Measurement of Health Utilities in Published Cost-Utility Analyses

Objective:  The Panel on Cost-Effectiveness in Health and Medicine recommended the compilation of a catalog of health state utility weights for use in cost-utility analyses (CUAs), and has given methodological recommendations. This study presents an update, through 2001, to our current registry of utility weights (available at http://www.tufts-nemc.org/cearegistry ; previously at http://www.hsph.harvard.edu/cearegistry ), and documents recent changes in methods used for utility weight elicitation. Methods:  We searched the English-language medical literature for original CUAs reporting outcomes as cost per quality-adjusted life-year (QALY). Two trained readers independently audited each article, abstracting data on the health state descriptions, corresponding utility weights, methods of elicitation, and sources of the estimates. The utility elicitation methods from 1998 to 2001 were compared with the methods used to obtain utilities before 1998. Results:  We identified 306 CUAs published after 1998, reporting 1210 separate health-related utility estimates, bringing the total in our catalog to 2159 weights. Most frequently, health states pertained to the circulatory system and oncology. Methods varied substantially: 36% of authors used direct elicitation (standard gamble, time trade-off or rating scale), 23% used generic health status instruments (EQ-5D, Health Utilities Index, etc.), and 25% estimated weights based on clinical judgment. Community preferences were used in 27% of the values. Compared with pre-1998, utilities published from 1998 to 2001 were more likely to be elicited using a generic instrument, more likely elicited from community samples, and less likely derived from expert opinion, with no formally employed methodology. Conclusions:  Increasingly, analysts conducting CUAs are using generic, preference-weighted instruments, and relying on community-based preferences. Our catalog of utility weights provides a useful reference tool for producers and consumers of CUAs, but also highlights the continued need for improvement in methods and transparency.Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/72131/1/j.1524-4733.2006.00116.x.pd

Growth and Quality of the Cost–Utility Literature, 1976–2001

Purpose:  Cost-utility analyses (CUAs) have become increasingly popular, although questions persist about their comparability and credibility. Our objectives were to: 1) describe the growth and characteristics of CUAs published in the peer-reviewed literature through 2001; 2) investigate whether CUA quality has improved over time; 3) examine whether quality varies by the experience of journals in publishing CUAs, or the source of external funding for study investigators; and 4) examine changes in practices in US-based studies following recommendations of the US Panel on Cost-Effectiveness in Health and Medicine (USPCEHM). This study updates and expands our previous work, which examined CUAs through 1997. Methods:  We conducted a systematic search of the English-language medical literature for original CUAs published from 1976 through 2001, using Medline and other databases. Each study was audited independently by two trained readers, who recorded the methodological and reporting practices used. Results:  Our review identified 533 original CUAs. Comparing articles published in 1998 to 2001 (n = 305) with those published in 1976 to 1997 (n = 228), studies improved in almost all categories, including: clearly presenting the study perspective (73% vs. 52%, P  < 0.001); discounting both costs and quality-adjusted life-years (82% vs. 73%, P  = 0.0115); and reporting incremental cost-utility ratios (69% vs. 46%, P  < 0.001). The proportion of studies disclosing funding sources did not change (65% vs. 65%, P  = 0.939). Adherence to recommended practices was greater in more experienced journals, and roughly equal in industry versus non-industry-funded analyses. The data suggest an impact in methodological practices used in US-based CUAs in accordance with recommendations of the USPCEHM. Conclusions:  Adherence to methodological and reporting practices in published CUAs is improving, although many studies still omit basic elements. Medical journals, particularly those with little experience publishing cost-effectiveness analyses, should adopt and enforce standard protocols for conducting and reporting CUAs.Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/73064/1/j.1524-4733.2005.04010.x.pd

Evaluating treatments in health care: The instability of a one-legged stool

<p>Abstract</p> <p>Background</p> <p>Both scientists and the public routinely refer to randomized controlled trials (RCTs) as being the 'gold standard' of scientific evidence. Although there is no question that placebo-controlled RCTs play a significant role in the evaluation of new pharmaceutical treatments, especially when it is important to rule out placebo effects, they have many inherent limitations which constrain their ability to inform medical decision making. The purpose of this paper is to raise questions about <it>over-reliance </it>on RCTs and to point out an additional perspective for evaluating healthcare evidence, as embodied in the Hill criteria. The arguments presented here are generally relevant to all areas of health care, though mental health applications provide the primary context for this essay.</p> <p>Discussion</p> <p>This article first traces the history of RCTs, and then evaluates five of their major limitations: they often lack external validity, they have the potential for increasing health risk in the general population, they are no less likely to overestimate treatment effects than many other methods, they make a relatively weak contribution to clinical practice, and they are excessively expensive (leading to several additional vulnerabilities in the quality of evidence produced). Next, the nine Hill criteria are presented and discussed as a richer approach to the evaluation of health care treatments. Reliance on these multi-faceted criteria requires more analytical thinking than simply examining RCT data, but will also enhance confidence in the evaluation of novel treatments.</p> <p>Summary</p> <p>Excessive reliance on RCTs tends to stifle funding of other types of research, and publication of other forms of evidence. We call upon our research and clinical colleagues to consider additional methods of evaluating data, such as the Hill criteria. Over-reliance on RCTs is similar to resting all of health care evidence on a one-legged stool.</p

The moral economy of the pharmaceutical industry: Legitimising prices

The practices of pharmaceutical companies have been widely criticised by researchers and investigative journalists, yet their conduct has mostly escaped significant moral opprobrium from the wider public, health professionals and governments. This article examines one reason for this by exploring the techniques companies use when seeking to justify and legitimise their conduct – legitimising techniques that help to render their failures to adhere to accepted standards less visible. It explores these techniques by examining four cases involving pricing where the companies’ conduct has, nonetheless, been questioned. It is divided into three parts. The first looks at the various publicly-stated standards that provide the moral context for the industry’s activities. The second examines four cases, each involving pricing, where companies’ prices have been challenged as morally unacceptable, each leading to a US Government investigation. These provide a means of exploring how companies seek to justify their actions in order to maintain the appearance of conformity to accepted moral standards. The third considers some reasons why the industry’s efforts at legitimation have considerable force. The analysis shows not only the character of the claims made by pharmaceutical companies in defence of their practices – claims about the health benefits of the medicine, access to it, and research and development costs, which are all often exaggerated. It also shows why the companies’ legitimising tactics are typically effective

Use of Pharmacoeconomics Information—Report of the ISPOR Task Force on Use of Pharmacoeconomic/Health Economic Information in Health-Care Decision Making

Objectives: Despite the growing number of pharmacoeconomic (PE)/health economic (HE) studies, very little is known about their use by decision makers. The objectives of the Task Force were to ensure that the good research practices of PE/HE studies pay attention to the needs of health-care decision makers and to develop a “toolbox” for the health-care decision maker wanting to interpret and use PE/HE studies. Methods: The membership of the Task Force consisted of individuals involved in making decisions about the availability or use of medicines and researchers into the use of economic evaluations. The group communicated by E-mail and face-to-face meetings. A literature review of decision makers’ attitudes toward PE/HE studies and published economic evaluation guidelines was undertaken. In addition, a focus group discussion was held with opinion leaders in managed care pharmacy. Results: The literature review identified 16 surveys of decision makers’ attitudes toward PE/HE studies and 15 published guidelines that outlined reporting requirements for economic evaluations. These were reviewed and classified. Based on the published literature and comments from decision makers, seven additional reporting requirements for studies were specified. Conclusions: While the Task Force's additional reporting requirements may be helpful to decision makers, they raise a number of issues. These include the feasibility of meeting the additional requirements, whether decision makers should receive more education in economic evaluation, and whether there should be more study of health-care decision-making procedures themselves.Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/73067/1/j.1524-4733.2003.64245.x.pd