Tufts Center for the Study of Drug Development

About: Tufts Center for the Study of Drug Development is a based out in . It is known for research contribution in the topics: Drug development & Clinical trial. The organization has 78 authors who have published 258 publications receiving 16047 citations.

Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI's Patient Groups and Clinical Trials Project.

Abstract: While patient groups, regulators, and sponsors are increasingly considering engaging with patients in the design and conduct of clinical development programs, sponsors are often reluctant to go beyond pilot programs because of uncertainty in the return on investment. We developed an approach to estimate the financial value of patient engagement. Expected net present value (ENPV) is a common technique that integrates the key business drivers of cost, time, revenue, and risk into a summary metric for project strategy and portfolio decisions. We assessed the impact of patient engagement on ENPV for a typical oncology development program entering phase 2 or phase 3. For a pre-phase 2 project, the cumulative impact of a patient engagement activity that avoids one protocol amendment and improves enrollment, adherence, and retention is an increase in net present value (NPV) of $62MM ($65MM for pre-phase 3) and an increase in ENPV of $35MM ($75MM for pre-phase 3). Compared with an investment of $100,000 in patient engagement, the NPV and ENPV increases can exceed 500-fold the investment. This ENPV increase is the equivalent of accelerating a pre-phase 2 product launch by 2½ years (1½ years for pre-phase 3). Risk-adjusted financial models can assess the impact of patient engagement. A combination of empirical data and subjective parameter estimates shows that engagement activities with the potential to avoid protocol amendments and/or improve enrollment, adherence, and retention may add considerable financial value. This approach can help sponsors assess patient engagement investment decisions.

Racial disparities among clinical research investigators.

Abstract: Evidence shows that minority patients are underrepresented in clinical trials. The development of new drugs and treatments, however, requires that clinical research studies include representative participants, particularly in light of evidence indicating that minority populations sometimes respond differently to prescription medications. Racial disparities among clinical investigators are often cited as a major reason why minority patients are underrepresented in clinical trials. However, there is little to no empirical data to support or refute the prevalence of disparities among clinical investigators. The Tufts Center conducted two online surveys of 1376 physicians. The first survey (N = 859 respondents; 31% response rate) assessed the overall incidence of minority physician involvement in clinical research. The second survey (N = 768 respondents; 20% response rate) assessed the demographics, experience, and infrastructure of minority physicians who have participated in clinical research as a principal investigator or subinvestigator. The results of this study indicate that significant racial disparities exist among clinical investigators. The results also support assertions that physician race influences race of the clinical trial volunteer. The incidence of participation in clinical research among minority physicians is well below that observed among white physicians, more so with regard to U.S. Food & Drug Administration-regulated clinical trials funded by industry. Minority investigators tend to conduct and initiate fewer clinical trials annually. Yet minority and white physician interest in participating in clinical research is similarly high. Minority investigators tend be younger, with more limited clinical research infrastructure and support than their white counterparts. New strategies, policies, incentives, and reforms are needed to address racial disparities among clinical investigators. In addition, disparities among both volunteers and investigators need to be tracked more closely and methodically to monitor and assess the impact of newly implemented programs and reforms.

Obstacles and Opportunities in New Drug Development

Abstract: Obstacles on the road to new drug development A host of formidable obstacles confront pharmaceutical and biopharmaceutical companies in their quest to bring innovative new medicines to market. Rapidly growing R&D costs, waning investor confidence, increasing competitive pressures, an uncertain regulatory environment, public mistrust, and a highly volatile political climate are significant threats to the research-based industry. Let us look at each of these threats more closely. R&D spending on new drugs in the United States continues to spiral upward, exceeding $40 billion in 2006. At the same time, the number of new molecular and biological entities approved by the US Food and Drug Administration (FDA) has declined. Growing R&D investment combined with diminishing output strongly suggests that the cost to bring one new drug to market is increasing at a very rapid pace. Based on data newly published by the Tufts Center for the Study of Drug Development (Tufts CSDD), the average capitalized cost to bring one new biopharmaceutical product to market, including the cost of failures, is $1.24 billion in 2005 dollars. High R&D costs reflect the enormous challenge of developing products for ever more chronic and complex diseases, for example, neuropharmacologic and oncology indications. High development costs can also be attributed to the rapid growth in the size of clinical studies, the difficulty in recruiting and retaining subjects for these studies, high candidate attrition rates, and late-stage failures in the drug development process. Current Tufts CSDD data show that the average time to bring a new pharmaceutical product to market, from the start of clinical testing to FDA approval, is 8.5 years, and the clinical success rate is 21.5%. It is worth noting that in the neuropharmacologic area those figures are 10.8 years and 14% and in the oncology area they are 9.3 years and 8%. Rising drug development costs, long time lines, and low clinical success rates in the research-based industry have led to an erosion in investor confidence, resulting in an alarming decline in pharmaceutical company stock prices and market capitalization. Unquestionably, “blockbuster R&D strategies,” which many firms adopted in the 1990s, have contributed to declining late-stage success rates and high development costs. Such models focus on the development of products in therapeutic areas with large potential markets. These tend to be chronic disease areas, such as hypertension, arthritis, and depression. Unfortunately, these often represent crowded pharmaceutical markets, and thus the likelihood of failure during premarket clinical testing due to economic reasons (i.e., the inability to compete successfully in the marketplace) increases. Tufts CSDD analyses have shown that these economic failures tend to occur late in the clinical development process when R&D costs and resource demands are at their highest. Drug developers must also deal with increasing competition in the marketplace. The current worldwide focus on containing health-care costs and restrictive price-control policies in many industrialized countries, combined with dwindling market exclusivity periods for new therapeutic products and the expiration of patent terms for a relatively high number of major blockbuster drugs, has led to a reassessment of R&D strategies in many pharmaceutical companies. These new strategies emphasize value, both economic and therapeutic, in the search for new medicines. Regulatory hurdles create additional obstacles for drug developers, especially with the current political climate in the United States, where the highly publicized withdrawal from the market of several high-profile drugs for safety reasons has created a risk-averse environment for drug review at the FDA. The FDA Amendments Act of 2007 (FDAAA), signed into law on 27 September 2007, reauthorizes the collection of industry user fees to pay for new drug and biologic application review by the FDA and includes a host of provisions that focus on product safety. For example,

R&d Costs, Innovative Output and Firm Size in the Pharmaceutical Industry

Abstract: This study examines the relationships between firm size, R&D costs and output in the pharmaceutical industry. Project–level data from a survey of 12 US–owned pharmaceutical firms on drug development costs, development phase lengths and failure rates are used to determine estimates of the R&D cost of new drug development by firm size. Firms in the sample are grouped into three size categories, according to their pharmaceutical sales at the beginning of the study period. The R&D cost per new drug approved in the US is shown to decrease with firm size, while sales per new drug approved are shown to increase markedly with firm size. Sales distributions are highly skewed and suggest that firms need to search for blockbuster drugs with above–average returns. The results are consistent with substantial economies of scale in pharmaceutical R&D, particularly at the discovery and preclinical development phases.

New Drug Innovation and Pharmaceutical Industry Structure: Trends in the Output of Pharmaceutical Firms:

Abstract: This study examines what is generally regarded to be the most important measure of innovation in the pharmaceutical industry—the extent to which new drugs are developed and marketed by pharmaceutical firms. Pharmaceutical industry output, as measured by new chemical entity (NCE) approvals in the United States since the 1962 Amendments to the Federal Food, Drug, and Cosmetic Act of 1938, is examined at the firm level. This long-term historical perspective permits us to observe the extent to which this industry has been concentrated with respect to innovative output and how stable company leadership positions have been over time. Databases containing detailed information on all NCEs approved in the United States from 1963 to 1999 and on a large sample of investigational NCEs taken into clinical testing since 1963 were utilized to examine productivity in developing new products at the firm level according to the following stratifications: period of approval, therapeutic class, whether the compounds were self...