Showing papers by "Tufts Center for the Study of Drug Development published in 2007"

The cost of biopharmaceutical R&D: is biotech different?

Abstract: The costs of developing the types of new drugs that have been pursued by traditional pharmaceutical firms have been estimated in a number of studies. However, similar analyses have not been published on the costs of developing the types of molecules on which biotech firms have focused. This study represents a first attempt to get a sense for the magnitude of the R&D costs associated with the discovery and development of new therapeutic biopharmaceuticals (specifically, recombinant proteins and monoclonal antibodies [mAbs]). We utilize drug-specific data on cash outlays, development times, and success in obtaining regulatory marketing approval to estimate the average pre-tax R&D resource cost for biopharmaceuticals up to the point of initial US marketing approval (in year 2005 dollars). We found average out-of-pocket (cash outlay) cost estimates per approved biopharmaceutical of $198 million, $361 million, and $559 million for the preclinical period, the clinical period, and in total, respectively. Including the time costs associated with biopharmaceutical R&D, we found average capitalized cost estimates per approved biopharmaceutical of $615 million, $626 million, and $1241 million for the preclinical period, the clinical period, and in total, respectively. Adjusting previously published estimates of R&D costs for traditional pharmaceutical firms by using past growth rates for pharmaceutical company costs to correspond to the more recent period to which our biopharmaceutical data apply, we found that total out-of-pocket cost per approved biopharmaceutical was somewhat lower than for the pharmaceutical company data ($559 million vs $672 million). However, estimated total capitalized cost per approved new molecule was nearly the same for biopharmaceuticals as for the adjusted pharmaceutical company data ($1241 million versus $1318 million). The results should be viewed with some caution for now given a limited number of biopharmaceutical molecules with data on cash outlays, different therapeutic class distributions for biopharmaceuticals and for pharmaceutical company drugs, and uncertainty about whether recent growth rates in pharmaceutical company costs are different from immediate past growth rates. Copyright © 2007 John Wiley & Sons, Ltd.

Development trends for monoclonal antibody cancer therapeutics

Abstract: Monoclonal antibodies are now established as a key therapeutic modality for a range of diseases. Owing to the ability of these agents to selectively target tumour cells, cancer has been a major focus of development programmes for monoclonal antibodies so far. Here, we overview trends in the clinical development and regulatory approval of monoclonal antibodies for cancer since 1980, with the aim of informing future research and development for this class of therapeutics.

Economics of New Oncology Drug Development

Abstract: Purpose Review existing studies and provide new results on the development, regulatory, and market aspects of new oncology drug development. Methods We utilized data from the US Food and Drug Administration (FDA), company surveys, and publicly available commercial business intelligence databases on new oncology drugs approved in the United States and on investigational oncology drugs to estimate average development and regulatory approval times, clinical approval success rates, first-in-class status, and global market diffusion. Results We found that approved new oncology drugs to have a disproportionately high share of FDA priority review ratings, of orphan drug designations at approval, and of drugs that were granted inclusion in at least one of the FDA's expedited access programs. US regulatory approval times were shorter, on average, for oncology drugs (0.5 years), but US clinical development times were longer on average (1.5 years). Clinical approval success rates were similar for oncology and other ...

Patient access to pharmaceuticals: an international comparison

Abstract: We have identified eight sub-dimensions of patient access to pharmaceuticals: marketing approvals, time of marketing approval, coverage, cost sharing, conditions of reimbursement, speed from marketing approval to reimbursement, extent to which beneficiaries control choice of their drug benefit, and evenness of the availability of drugs to the population. For a sample of commonly used best-selling drugs in the United States (US), we measured these eight access sub-dimensions across four health systems: France, the Netherlands, the United Kingdom (UK), and the US. Although the US approved between 15 and 18% more drugs than the other three countries, the US was slower than France and the UK to approve drugs licensed in all four countries. The percentage of drugs covered is approximately the same for all four countries. For covered drugs, we observe the least cost sharing by patients in the Netherlands. The Netherlands imposes conditions of reimbursement on a much larger percentage of drugs. France seems to be the slowest in respect of speed from marketing approval to reimbursement. The US is the most flexible in terms of the extent to which beneficiaries control their choice of drug benefit but it is the least universal in terms of evenness of the availability of drugs to the population. Our study confirms the frequently cited problems of access in European countries: lag between marketing approval and reimbursement, and inflexibility in respect of the extent to which beneficiaries control their choice of drug benefit. At the same time, our study confirms, qualitatively, different kinds of access problems in the US: relatively high patient cost sharing for pharmaceuticals, and wide variation in coverage.

The increasingly complex fourth hurdle for pharmaceuticals

Abstract: There are three known criteria that underlie drug reimbursement decisions: therapeutic value, cost effectiveness and burden of disease. However, evidence from recent reimbursement decisions in several jurisdictions points to residual unexplained variables, one of which may be budget impact. An economic rationale for carrying out budget impact analyses is opportunity cost, measured by the economic benefits foregone by using resources in one way rather than another. Under certain assumptions, cost-effectiveness analysis accounts for opportunity cost while conveying to the decision maker the price of maximising health gains, subject to a budget constraint. However, the underlying assumptions are implausible, particularly in the context of pharmaceutical care. Although drugs that are cost effective may lead to unambiguous health gains among patient groups for whom the drugs are indicated, the opportunity costs could conceivably lead to a reduction in aggregate health gains, or failure to meet different kinds of equity considerations. The pertinent policy question is where to find the resources to fund new innovations, such as cost-effective pharmaceuticals, or drugs targeting severe diseases. It may be a matter of redeployment of resources across healthcare sectors, cancelling the funding of (older) pharmaceuticals that are less cost effective, or delisting drugs that are cost effective but target less burdensome conditions.

Trends in the development and approval of monoclonal antibodies for viral infections.

Abstract: Monoclonal antibodies (mAbs) developed for either the prevention or treatment of viral diseases represent a small, but valuable, class of products. Since 1985, commercial firms have initiated clinical studies involving a total of 28 mAbs. To date, one product (palivizumab) has been approved and eight candidates are currently in clinical study. Most commercial mAbs studied as antiviral agents in the clinic have either directly or indirectly targeted human immunodeficiency virus, respiratory syncytial virus, or hepatitis C virus infections. However, the ability of mAbs to bind to specific targets and utilize various anti-infective modes of action would seem to make them well suited for the prevention and/or treatment of a wider variety of viral diseases. A number of factors, including the continuing need for innovative medicines for viral infections, the global spread of viral infections, and increased government funding for the study of pathogen countermeasures, have prompted companies to reconsider mAbs as antiviral agents. Public sector research into the use of mAbs against emerging pathogens, such as severe acute respiratory syndrome coronavirus, may have already provided candidates for further development.

Should the patent system for new medicines be abolished

Abstract: “It is hard to think of many industries that have contributed as much to human welfare as the pharmaceutical industry.” This statement was made not by a pharmaceutical industry chief executive officer but by two antitrust regulators, Roy Levy and Abraham Wickelgren, with the Federal Trade Commission.1 There is indeed evidence from the medical and economics literature that new drugs have played a central role in increased longevity, enhanced quality of life, and improved labor-force participation and productivity. Recent studies have attributed as much as half of all welfare gains worldwide during the 20th century to the introduction of new medicines and technologies.2,3 Despite these noteworthy accomplishments, the industry has been subjected to hostile criticism and radical policy proposals. One of the major targets of these proposals is the patent system, in which inventors are awarded exclusive rights to develop and commercialize new medicines. A variety of alternative schemes have been offered, but virtually all come down to greater reliance on the government to fund the discovery and development of new drugs. This would be done through either a prize fund for private inventors of new medical technologies or direct government funding of the research through grants and contracts. The advantage claimed for these proposals is that they would lower drug prices and allow greater access to new medicines, because without patents all medicines would be “genericized” at the time of introduction.

Can Medicare Draw Lessons from Dutch Experience with a National Formulary

Abstract: Objective: Using seven therapeutic subclasses of cardiovascular drugs as a case study, we investigated how different systems of formulary practice have an impact on access to prescription drugs, as well as the likely repercussions of the recently passed Medicare legislation on Medicare beneficiaries. We identified four dimensions of access: availability, coverage, equity, and flexibility. We examined a fifth item, transparency, unrelated to access but relevant to the formulary decision-making process as a whole. Study Design and Methods: Drawing from public data from the US and Dutch drug regulatory agencies, three leading US managed care plans participating in the Medicare Advantage program, Michigan's Medicaid agency, and the Dutch board of insurers, we determined for the period 1991–2004 (1) which cardiovascular drugs were approved for marketing in the United States and the Netherlands and approval dates; (2) formulary status of the approved drugs; and (3) copayments for on-formulary drugs. For each in...

The new Dutch health insurance system and its implications for pharmaceutical innovation

Abstract: Purpose – The purpose of this paper is to provide commentary on the state of affairs regarding implementation of Dutch health insurance reform, focusing on whether such reform is conducive to pharmaceutical innovation.Design/methodology/approach – The general characteristics of the Dutch healthcare system is outlined, together with a brief synopsis of the 2006 health insurance reform initiative. This is followed by a description of the four market intervention mechanisms and their implications for pharmaceutical innovation. Finally, these implications and the potential for policy transfer to other European countries are discussed.Findings – The new Dutch health insurance system represents a novel approach that closely follows Enthoven's managed competition model. Certain features of the new system are conducive to pharmaceutical innovation. These positive features include more flexibility on the part of private insurers to deviate from the national formulary, speedier reimbursement appraisals, and more ea...

Mandatory comparator trials for therapeutically similar drugs: an assessment of the facts

Abstract: Legislative proposals intended to compel industry to perform comparator trials are currently under consideration as Congress begins discussions on the reauthorization of the Prescription Drug User Fee Act. The proposals vary from ones that simply mandate head-tohead trials by statute to those that would provide specific incentives, either positive ones such as extending periods of market protection or negative ones such as a prohibition on advertising until trials are done. Whereas the use of mandating head-to-head trials postapproval is open to debate, the more contentious scenario is one in which head-to-head trials are required preapproval. So far, that discussion has been driven primarily by presumption, perception, and rhetoric. What has been missing from the debate is a careful assessment of the realities of the research and development (R&D) environment and the marketplace for new medicines, the feasibility of actually conducting such trials, and how useful the data would be relative to the burden on industry and the US Food and Drug Administration (FDA). Before moving forward politically on this issue, a few facts should be brought to light. We attempt to provide answers to some of the questions pertinent to the debate.