Showing papers by "Tufts Center for the Study of Drug Development published in 2003"

Trends in development and approval times for new therapeutics in the United States.

Abstract: The process of clinical development and regulatory review of new therapeutics in the United States was significantly changed by a number of legislative acts passed in the 1980s and 1990s. These acts were designed to encourage the development of innovative products, especially for rare, serious or life-threatening diseases, and to ensure that patients had timely access to these treatments. To assess the effects of the various modifications to the process, the Tufts Center for the Study of Drug Development analysed clinical development and approval data for 554 therapeutics (504 small molecules, 40 recombinant proteins and 10 monoclonal antibodies) approved in the United States from 1980–2001. Trends in the number of approved products and the clinical development and approval times indicated that the effects of these changes were generally beneficial as of the mid- to late-1990s, but that the gains have not been sustained in the early 2000s. Current efforts by the FDA, and the pharmaceutical and biopharmaceutical industry, to reverse the recent tendency toward fewer new approvals and longer approval times are discussed.

Therapeutic recombinant proteins: trends in US approvals 1982 to 2002.

Abstract: Recombinant proteins have become an important class of therapeutics since the first product was approved for marketing in the US in 1982. To determine trends in the clinical development and approval processes for therapeutic recombinant proteins, data for 270 products that entered clinical study in the period 1980 to 2002 were collected and analyzed. Mean clinical and approval phase lengths for 54 US-approved products stratified by time period, therapeutic indication, protein type, review rating and FDA review center are compared. Approval success rates, calculated using three methods that differ only in the definition of success, and the variation of approval success rates over five time periods are presented.

Canadian and US Drug Approval Times and Safety Considerations

Abstract: BackgroundApproval times of new drugs are frequently longer in Canada than in the US, but it has been argued that reducing approval times might lead to unsafe drugs receiving marketing approval.ObjectiveTo compare new drug approval times in Canada and the US over a 10-year period and to relate them to safety discontinuations.MethodsApplication and approval dates of all new drugs except diagnostic products, new salts, esters, isomers, and dosage forms of already-marketed drugs, as well as combinations containing previously approved substances approved in the US and Canada between January 1992 and December 2001 were obtained from the respective drug regulatory agencies and other sources. Information about drugs discontinued for safety reasons was obtained from the agencies' publications and Web sites and from journal articles.ResultsNew drug approval times were significantly longer in Canada than in the US. The difference occurs in all drug categories and by review type (priority/standard). However, the pro...

The New Drug Approvals of 1999, 2000, and 2001: Drug Development Trends a Decade after Passage of the Prescription Drug User Fee Act of 1992:

Abstract: This report is the sixth in a triennial series by the Tufts Center for the Study of Drug Development examining various aspects of recent new drug approvals in the United States. In 1999, 2000, and 2001 the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research approved 86 new drugs, 82 of which met the Tufts Center for the Study of Drug Development’s definition of a new chemical entity (NCE). Of the 82, 34 (41%) received priority review, while 48 (59%) had standard review. The mean length of the clinical phase (Investigational New Drug Application [IND] filing to New Drug Application [NDA] submission) for the 82 NCEs was 5.5 years, and the mean approval phase (NDA submission to approval) was 1.4 years. Whereas the clinical phase represents a 14% decrease from that value for the previous three-year period, the approval phase remained unchanged. The mean approval phase for priority NCEs (0.9 years) was half that for standard NCEs (1.8 years). Of the 75 NCEs for which foreign marketing data were available, 51 % were first approved for marketing in the United States, while 27% were available in foreign markets one or more years prior to United States approval, with a mean of 6.5 years of prior foreign marketing. The percentage of products first approved in the United States represents a continuation of a trend since passage of the Prescription Drug User Fee Act of 1992 of drug sponsors seeking marketing approval in the United States prior to approval in other markets.

Switching omeprazole in Sweden and the United States.

Abstract: This article compares the Swedish Medical Products Agency's (MPA) decision to switch omeprazole from prescription (Rx) to over-the-counter (OTC) status with the US Food and Drug Administration (FDA) advisory panel's decision not to authorize the switch. The agencies' differing perspectives on efficacy, safety, labeling, and clinical trial requirements are evaluated with regard to the Rx-to-OTC switch process in general and omeprazole's case in particular. The FDA and MPA regulatory policies on switches are substantially divergent. The FDA maintains a stricter set of switch guidelines and requirements than the MPA. One could infer from this that the FDA is more risk-averse than the MPA. Nevertheless, the omeprazole switch in Sweden appears to be an exception in that it contrasts with the MPA's historical reluctance to switch the Rx status of medications. Cost considerations appear to have triggered the omeprazole switch, making it a special case. The lessons to be drawn from this case study are both specific and general. At the specific level, this case study suggests the MPA's decision to switch omeprazole was prompted by economic considerations, whereas the FDA's mandate did not allow cost to affect its decision on omeprazole. At a general level, this case study indicates that the differences between the FDA and MPA with respect to their regulatory policies on switches and their mandates apply not only to omeprazole but also to the dozens of switches currently under consideration by the respective regulatory agencies.

Clinical development of therapeutic recombinant proteins.

Abstract: Only a small subset of the therapeutics that enter clinical studies will prove to be safe and effective in humans and gain approval for marketing. The success of the products and, by inference, the sponsoring companies can be measured by tracking advancement through the clinical phase and review transitions to marketing approval. To determine phase transition probabilities and approval success rates for recombinant protein (rDNA) therapeutics, the Tufts Center for the Study of Drug Development collected data for 271 rDNA therapeutics that entered clinical study between 1980 and 2002. The data were stratified into eight therapeutic categories. Approval success rates were calculated for rDNA therapeutics with two possible fates: (i) approval in any country and (ii) U.S. approval only. Global approval success rates ranged from 23% to 63%, and U.S. approval success rates ranged from 17% to 58%. Trends in clinical phase lengths over five time periods and an overview of the rDNA therapeutics currently under Food and Drug Administration review are discussed.

On the possibility of a positive-sum game in the distribution of health care resources.

Abstract: Health care resource distribution is a subject of debate among health policy analysts, economists, and philosophers. In the United States, there is a widening gap between the more-and less-advantaged socioeconomic sub-populations in terms of both health care resource distribution and outcomes. Conventional wisdom suggests that there is a tradeoff, a zero-sum game, between efficiency and fairness in the distribution of health care resources. Promoting fairness in the distribution of health care resources and outcomes is not efficient in terms of maximization of a health outcome production function. On the other side of the coin, improving efficiency comes at the expense of fairness. Such conventional wisdom is supported in part by standard static Paretian welfare analysis. However, in this paper it is shown that in a dynamic setting in which there are efficiency gains in the health production function, fairness in distribution of health care resources can improve simultaneously.

Impact of Disease Management on Cost-Effectiveness of Medicare Spending

Abstract: Findings from the third annual Tufts Center for the Study of Drug Development survey of 22 leading disease and pharmacy benefits managers suggest that incorporation of disease management into Medicare would lower hospital inpatient costs. However, it is unclear whether hospital cost savings would be sufficient to offset increases in pharmacy, physician, and outpatient expenditures as a result of an added combination disease management and pharmacy benefit. Furthermore, the survey indicates that the Centers for Medicare & Medicaid Services (CMS) would likely struggle in recruiting disease managers due to their limited enrollment of Medicare beneficiaries, relative inexperience with contracts that put disease managers at risk, and only sporadic use of randomized design studies to test and prove the efficacy of disease management interventions. Thirteen of the 22 survey respondents are currently considered early compilers per accreditation standards developed by the National Committee on Quality Assurance. However, none of the 22 survey respondents compty with the stringent CMS requirements described in the solicitation for the 2002 disease management demonstration.

State-based versus federal-based approaches to reducing the medicare pharmaceutical coverage gap

Abstract: A state-based approach, such as the Immediate Helping Hand proposal put forward by the Bush administration, is likely to be less effective than a federal-based approach at reducing the Medicare pharmaceutical coverage gap. In addition, the voluntary nature of a state-based approach, coupled with variations across states in existing coverage benefits and the limited reach of state pharmacy assistance programs, would likely lead to a perpetuation of uneven coverage. This article argues in favor of adding a federal-based universal prescription drug benefit to Medicare on the grounds of both equity and empirical evidence. Adding a universal drug benefit to the currently existing Medicare program would extend application of the social insurance concept across hospital care, physician service, and prescription drug coverage components of Medicare. As a result, a more equitable distribution of prescription drug coverage would be promoted while mitigating the effects of selection risk.