Showing papers by "Tufts Center for the Study of Drug Development published in 2018"

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.

Global Public Attitudes About Clinical Research and Patient Experiences With Clinical Trials.

Abstract: Importance Effective, continuous improvement in patient engagement depends on an intimate understanding of public and patient perceptions and experiences in clinical research. Objectives To identify the views of clinical trial participants and nonparticipants and characterize trends in these views over time. Design, Setting, and Participants In this survey study, a questionnaire was administered online from May 8 to July 24, 2017, by the Center for Information and Study on Clinical Research Participation (CISCRP), and findings were compared with previous studies conducted in 2013 and 2015. The 2017 sample included responses from 12 427 individuals from 68 countries and represents a 10% participation rate. Similar to international assessments conducted by the CISCRP and other organizations, this study drew responses from a convenience sample; any adult older than 18 years who received an email or had online access was eligible to participate. Main Outcomes and Measures Significant changes were observed in the perceptions and clinical trial experiences of the public and study volunteers compared with past CISCRP studies. Results A total of 12 427 individuals (mean [SD] age, 55 [15] years; 7355 [59.2%] female; 10 085 [81.2%] white), 2194 (17.7%) of whom had participated in previous clinical research studies, responded to the survey in 2017. Findings indicated a belief in the importance of clinical research, but limited understanding of the research process persists. In 2017, a total of 10 506 individuals (84.5%) perceived clinical research to be very important to the discovery and development of new medicines; however, 4079 of 6919 respondents (59.0%) were unable to name a place where studies were conducted. A total of 11 182 respondents (90.0%) believed that clinical research is generally safe; however, 5578 of 12 427 individuals (44.9%) reported that clinical trials are rarely considered as an option when discussing treatments or medications with their physician. Clinical trial participation was perceived as inconvenient and burdensome; in the latest survey, 1075 respondents (49.0%) expressed that their clinical trial participation disrupted their daily routine. Conclusions and Relevance The results of this study may provide a foundation from which to build meaningful and effective engagement with the public and patients and revealed roadblocks, including knowledge gaps among the public, limited physician involvement in discussing clinical trials as treatment options, and the inconveniences that patients encounter after they volunteer to participate. These findings may inform patient engagement strategies and tactics and ultimately help accelerate the drug-development process.

New Benchmarks Characterizing Growth in Protocol Design Complexity

Abstract: The Tufts Center for the Study of Drug Development and Medidata Solutions Inc analyzed data from 9737 protocols and 130,601 investigative site contracts associated with these protocols to derive updated benchmarks characterizing protocol complexity. The results of the study indicate that protocol design complexity continues to grow rapidly. Nearly all phase I, II, and III complexity measures associated with protocol execution increased significantly (eg, P < .0001) from 2001-2005 to 2011-2015. These measures include the number of unique and total procedures performed per patient over the course of a study, the site work effort to administer protocol procedures, the number of study volunteer visits, and the total number of procedures performed per study volunteer visit. The total cost per planned study volunteer per visit also increased significantly (eg, P < .0001) as did the total cost per study volunteer across all planned study visits. Phase I protocols remain the most complex and the most demanding to execute. Phase III protocols have seen the most substantial growth in protocol complexity. Phase IV protocols saw only modest increases in executional complexity during the 10-year time horizon. The implications of the study findings are discussed.

Reforming China's drug regulatory system.

Abstract: Since 2015, the Chinese government has introduced numerous reforms with the aim of establishing a more scientific and efficient drug regulatory system, which are highlighted here.

The Use of Real-World Evidence and Data in Clinical Research and Postapproval Safety Studies.

Abstract: Background:The adoption and use of real-world evidence (RWE) is becoming increasingly important to drug development and patient safety.Methods:The Tufts Center for the Study of Drug Development (CSDD) conducted a benchmark survey of pharmaceutical and biotechnology companies and contract research organizations in a number of areas that support real-world data (RWD) and evidence, including operations and performance areas. Data were gathered on organizational functions, staff, roles and responsibilities, and skill sets required. Also, current and future allocation of budgets and spending were examined as well as return on investment measures. A total of 30 unique companies responded to the survey.Results:Nearly all respondents (29/30 companies) reported that their organizations had an RWE function and most companies indicated that their RWE functions were increasing in size (21 companies). From a postapproval regulatory and labeling perspective, there were two primary areas for company use of RWD to genera...

Estimated lifetime survival benefit of tumor treating fields and temozolomide for newly diagnosed glioblastoma patients.

Abstract: Aim To estimate the mean lifetime survival benefit, an essential component of health economic evaluations in oncology, of adding tumor treating fields (TTFields) to maintenance temozolomide (TMZ) for newly diagnosed glioblastoma patients. Methods We integrated EF-14 trial data with glioblastoma epidemiology data. The model provided for an evidence-based approach to estimate lifetime survival for the material number of EF-14 trial patients still alive at 5 years. Results & conclusion Patients treated with TTFields and TMZ had an incremental mean lifetime survival of 1.8 years (TTFields/TMZ: 4.2 vs TMZ alone: 2.4). Patients alive at year 2 after starting TTFields had a 20.7% probability of surviving to year 10. The results presented here provide the required incremental survival benefit necessary for a future assessment of the incremental cost-effectiveness of TTFields.

Cost Drivers of a Hospital-Acquired Bacterial Pneumonia and Ventilator-Associated Bacterial Pneumonia Phase 3 Clinical Trial.

Abstract: Background Studies indicate that the prevalence of multidrug-resistant infections, including hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP), has been rising. There are many challenges associated with these disease conditions and the ability to develop new treatments. Additionally, HABP/VABP clinical trials are very costly to conduct given their complex protocol designs and the difficulty in recruiting and retaining patients. Methods With input from clinicians, representatives from industry, and the US Food and Drug Administration, we conducted a study to (1) evaluate the drivers of HABP/VABP phase 3 direct and indirect clinical trial costs; (2) to identify opportunities to lower these costs; and (3) to compare (1) and (2) to endocrine and oncology clinical trials. Benchmark data were gathered from proprietary and commercial databases and used to create a model that calculates the fully loaded (direct and indirect) cost of typical phase 3 HABP/VABP endocrine and oncology clinical trials. Results Results indicate that the cost per patient for a 200-site, 1000-patient phase 3 HABP/VABP study is $89600 per patient. The cost of screen failures and screen failure rates are the main cost drivers. Conclusions Results indicate that biopharmaceutical companies and regulatory agencies should consider strategies to improve screening and recruitment to decrease HABP/VABP clinical trial costs.

Assessing Study Start-up Practices, Performance, and Perceptions Among Sponsors and Contract Research Organizations:

Abstract: Background:Site identification, site selection, and study start-up have become the focus of improvement by organizations conducting clinical trials.Methods:To examine and measure the process from site identification through site activation, Tufts Center for the Study of Drug Development (CSDD) conducted a comprehensive survey among pharmaceutical organizations, biotech companies, and contract research organizations (CROs). Responses from over 400 unique companies were gathered and analyzed.Results:The results indicate that the start-up process is on average 5 to 6 months in total duration, and cycle times across all activities, including site identification, site selection, and study start-up, are faster for repeat sites than for new sites. Comparisons between sponsor and CROs indicate that CROs completed all site-related activities 6 to 11 weeks faster than sponsors. Other areas impacting cycle times were examined, including centralized versus decentralized functions, investment in technology, and organi...

Rising Drug Costs Drives the Growth of Pharmacy Benefit Managers Exclusion Lists: Are Exclusion Decisions Value-Based?

Abstract: Objective We examine whether drugs’ excluded versus recommended status on pharmacy benefit manager exclusion lists corresponds to evidence from cost-effectiveness analyses, lack of evidence, or rebates. Data Sources To find cost-effectiveness data for drugs on 2016 exclusion lists of CVS Caremark and Express Scripts, we searched the Tufts Cost-Effectiveness Analysis Registry and the peer-reviewed literature. Study Design For each excluded and recommended drug, we compared the mean cost-per-QALY, and we calculated the difference between the numbers of excluded and recommended drugs for which we could find no cost-effectiveness evidence. Data Collection As keywords in our searches, we used the brand and generic drug name and “cost-effectiveness” and “cost-per-quality-adjusted life-year.” Of 240 retrieved studies, 110 were selected for analysis. Principal Findings The mean cost-per-QALY for excluded drugs was higher ($51,611) than the cost-per-QALY for recommended drugs ($49,474), but not statistically significant. We could find no cost-effectiveness evidence in the Registry or peer-reviewed literature for 23 of the excluded drugs, and no evidence for 5 of the recommended drugs. Conclusions Cost-effectiveness does not correlate with a drug's excluded or recommended status. Lack of cost-effectiveness evidence favors a drug's excluded status.