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In the last few decades, developers of new drugs, biologics, and devices have increasingly leveraged digital health technologies (DHTs) to assess clinical trial digital endpoints. To our knowledge, a comprehensive assessment of the financial net benefits of digital endpoints in clinical trials has not been conducted. We obtained data from the Digital Medicine Society (DiMe) Library of Digital Endpoints and the US clinical trials registry, ClinicalTrials.gov. The benefit metrics are changes in trial phase duration and enrollment associated with the use of digital endpoints. The cost metric was obtained from an industry survey of the costs of including digital endpoints in clinical trials. We developed an expected net present value (eNPV) model of the cash flows for new drug development and commercialization to assess financial value. The value measure is the increment in eNPV that occurs when digital endpoints are employed. We also calculated a return on investment (ROI) as the ratio of the estimated increment in eNPV to the mean digital endpoint implementation cost. For phase II trials, the increase in eNPV varied from $2.2 million to $3.3 million, with ROIs between 32% and 48% per indication. The net benefits were substantially higher for phase III trials, with the increase in eNPV varying from $27 million to $40 million, with ROIs that were four to six times the investment. The use of digital endpoints in clinical trials can provide substantial extra value to sponsors developing new drugs, with high ROIs.

The Tufts Center for the Study of Drug Development conducted a study among practicing physicians and nurses (health care providers) across multiple specialties to assess their attitudes and experiences with referring patients into clinical trials and to supplement the body of scholarly research focused primarily on referral practices among oncology-based health care providers. A total of 755 physicians and 1255 nurses completed online surveys in late 2015 and early 2016. The results of the study indicate that a high percentage of multispecialty nurses and physicians is interested in referring their patients to appropriate clinical trials, are familiar with the clinical trial process, and are comfortable providing clinical trial information to, and discussing clinical trial opportunities with, their patients. Study results also indicate, however, that health care providers refer only a small number of patients each year largely because of the inability to access clinical trial information and the lack of sufficient information and time to evaluate and confidently discuss clinical trial options with their patients. Many reasons for choosing not to refer patients appear addressable, particularly through effective but presently –underused communication leveraging social media communities and using liaisons who can act as a bridge between clinical care and clinical research.

Articles in peer-reviewed journals and the trade press presuppose that strategic outsourcing relationships have been formed to replace preexisting collaborative approaches with contract research organizations. They do not consider that large, fragmented pharmaceutical and biotechnology companies may be supporting competing and conflicting relationship models simultaneously. A recent Tufts Center for the Study of Drug Development study quantifies actual strategic outsourcing practices among drug development companies and sheds new light on why these relationships may be failing. Tufts Center for the Study of Drug Development conducted an in-depth assessment of 43 Phase II and III clinical studies completed since 2012 to examine the outsourcing relationships used by 9 major pharmaceutical and biotechnology companies to support key functional areas. Descriptive statistics were assessed and t tests were performed to characterize outsourcing practices by function and to determine differences in study performance between transactional and strategic outsourcing relationships. The results indicate that sponsor companies are using a variety of outsourcing relationship models to support their studies, mixing and matching the use of internal staff, and using traditional transactional and strategic outsourcing relationships simultaneously. Specifically, despite the fact that each sponsor company had entered into several strategic outsourcing relationships, in no instance did a single contract research organization manage all functional areas supporting an individual Phase II or III study. In addition, sponsor companies vary the types of outsourcing relationship models that they use on a study-by-study basis. The inability of pharmaceutical and biotechnology companies to consistently embrace and coordinate sourcing strategies is creating internal friction and inefficiency. As a result, the expected impact of strategic outsourcing relationships on drug development performance, quality, and cost remains elusive.

The oversight of research involving human participants is widely believed to be inadequate. The U.S. Congress, national commissions, the Department of Health and Human Services, the Institute of Medicine, numerous professional societies, and others are proposing remedies based on the assumption that the main problems are researchers' conflict of interest, lack of institutional review board (IRB) resources, and the volume and complexity of clinical research. Developing appropriate reform proposals requires carefully delineating the problems of the current system to know what reforms are needed. To stimulate a more informed and meaningful debate, we delineate 15 current problems into 3 broad categories. First, structural problems encompass 8 specific problems related to the way the research oversight system is organized. Second, procedural problems constitute 5 specific problems related to the operations of IRB review. Finally, performance assessment problems include 2 problems related to absence of systematic assessment of the outcomes of the oversight system. We critically assess proposed reforms, such as accreditation and central IRBs, according to how well they address these 15 problems. None of the reforms addresses all 15 problems. Indeed, most focus on the procedural problems, failing to address either the structure or the performance assessment problems. Finally, on the basis of the delineation of problems, we outline components of a more effective reform proposal, including bringing all research under federal oversight, a permanent advisory committee to address recurrent ethical issues in clinical research, mandatory single-time review for multicenter research protocols, additional financial support for IRB functions, and a standardized system for collecting and disseminating data on both adverse events and the performance assessment of IRBs.

Introduction The under-reporting of adverse drug events (ADEs) is an international health concern. A number of studies have assessed the root causes but, to our knowledge, little information exists relating under-reporting to practices and systems used for the recording and tracking of drug‐related adverse event observations in ambulatory settings, institutional settings, and retail pharmacies. Objectives Our objective was to explore the process for reporting ADEs in US hospitals, ambulatory settings, and retail pharmacies; to explore gaps and inconsistencies in the reporting process; and to identify the causes of under-reporting ADEs in these settings. Methods The Tufts Center for the Study of Drug Development (Tufts CSDD) interviewed 11 thought leaders and conducted a survey between May and August 2014 among US-based healthcare providers (HCPs) in diverse settings to assess their experiences with, and processes for, reporting ADEs. Results A total of 123 individuals completed the survey (42 % were pharmacists; 27 % were nurses; 15 % were physicians; and 16 % were classified as ‘other’). HCPs indicated that the main reasons for under-reporting were difficulty in determining the cause of the ADE, given that most patients receive multiple therapies simultaneously (66 % of respondents); that HCPs lack sufficient time to report ADEs (63 % of respondents); poor integration of ADE-reporting systems (53 % of respondents); and uncertainty about reporting procedures (52 % of respondents). Discussion The results of this pilot study identify that key factors contributing to the under-reporting of ADEs relate to a lack of standardized process, a lack of training and education, and a lack of integrated health information technologies.

Background Deployment of remote and virtual clinical trial methods and technologies, referred to collectively as decentralized clinical trials (DCTs), represents a profound shift in clinical trial practice. To our knowledge, a comprehensive assessment of the financial net benefits of DCTs has not been conducted. Methods We developed an expected net present value (eNPV) model of the cash flows for new drug development and commercialization to assess the financial impact of DCTs. The measure of DCT value is the increment in eNPV that occurs, on average, when DCT methods are employed in comparison to when they are not. The model is populated with parameter values taken from published studies, Tufts CSDD benchmark data, and Medable Inc. data on DCT projects. We also calculated the return on investment (ROI) in DCTs as the ratio of the increment in eNPV to the DCT implementation cost. Results We found substantial value from employing DCT methods in phase II and phase III trials. If we assume that DCT methods are applied to both phase II and phase III trials the increase in value is $20 million per drug that enters phase II, with a seven-fold ROI. Conclusions DCTs can provide substantial extra value to sponsors developing new drugs, with high returns to investment in these technologies. Future research on this topic should focus on expanding the data to larger datasets and on additional aspects of clinical trial operations not currently measured.

The Tufts Center for the Study of Drug Development analyzed 8,317 clinical trial protocols to benchmark protocol complexity by phase and therapeutic area and to characterize trends in clinical trial complexity and the burden placed on study staff to execute protocol procedures between 2000 and 2007. Wide variability in protocol complexity and work burden was observed across therapeutic areas, within and between clinical research phases. Phase 1 protocols are the most complex and the most demanding to execute. The mean number of total procedures per protocol in phase 4 studies and the work burden associated with phase 1 protocols grew the fastest between the periods 2000-2003 and 2004–2007. The complexity and work burden of phase 3 protocols also grew rapidly during this period. Protocols in anti-infectives, immunomodulation, CNS, and oncology are consistently the most complex and burdensome to execute. Reasons for the wide variability in protocol complexity and work burden by phase and therapeutic area are discussed. This study provides insight into areas where protocol design improvements should be targeted.

The extent to which new drug developers can benefit financially from shorter development times has implications for development efficiency and innovation incentives. We provided a real-world example of such gains by using recent estimates of drug development costs and returns. Time and fee data were obtained on 5 single-source manufacturing projects. Time and fees were modeled for these projects as if the drug substance and drug product processes had been contracted separately from 2 vendors. The multi-vendor model was taken as the base case, and financial impacts from single-source contracting were determined relative to the base case. The mean and median after-tax financial benefits of shorter development times from single-source contracting were $44.7 million and $34.9 million, respectively (2016 dollars). The after-tax increases in sponsor fees from single-source contracting were small in comparison (mean and median of $0.65 million and $0.25 million). For the data we examined, single-source contracting yielded substantial financial benefits over multi-source contracting, even after accounting for somewhat higher sponsor fees.

Two frequently cited figures by clinical research insiders and observers – the cost of missing a day to generate prescription drug sales and the cost of a day to conduct a clinical trial – are outdated and based on anecdotal evidence. In late 2023, the Tufts Center for the Study of Drug Development conducted empirical research to gather more accurate and granular estimates and to test whether average sales per day have changed over time. 645 drugs launched since 2000, and 409 clinical trial budgets were drawn from commercially available and proprietary data sets and analyzed. The results indicate that a single day equals approximately $500,000 in lost prescription drug or biologic sales, with daily prescription sales for infectious, hematologic, cardiovascular, and gastrointestinal diseases among the highest. The results also show that each year, the average sales per day of prescription drugs and biologics has decreased by approximately $80,000—$100,000. The estimated direct daily cost to conduct a clinical trial is approximately $40,000 per day for phase II and III clinical trials, with those in respiratory, rheumatology, and dermatology having the highest relative daily direct costs.