Examination of Clinical Trial Costs and Barriers for Drug Development. Appendix B: Medidata Data Element Descriptions

07/25/2014

Data Element Data Element Description Unit Source
Administrative Staff Costs Non-clinical administrative staff cost associated with managing the study at the sites Month CROCAS® (Medidata Solutions, 2012a)
All Other Costs Includes costs of development for the entity being tested, costs for sponsors to run the study (additional internal costs and ancillary administrative costs), and other costs not captured by the other fields. It is equal to 30% of the total costs (according to Medidata, the clinical trial cost fields they have provided to ERG capture 70% of total costs). Study Medidata Solutions calculation
Central Lab Costs [a] Central laboratory cost, if central laboratory used Patient PICAS® (Medidata Solutions, 2012b)
Clinical Procedure Total Total cost of clinical procedures only for one patient Patient PICAS® (Medidata Solutions, 2012b)
Cost Per IRB Amendment Cost of a single IRB amendment IRB amendment PICAS® (Medidata Solutions, 2012b)
Cost Per IRB Approval Cost of a single IRB approval IRB approval PICAS® (Medidata Solutions, 2012b)
Data Collection, Management and Analysis Costs Costs associated with collection, management, and analysis of data for one study/protocol, across all sites and patients Study CROCAS® (Medidata Solutions, 2012a)
FDA Application Fee The Federal Food, Drug, and Cosmetic Act, as amended by the Prescription Drug User Fee Amendments of 2012, which was signed by the President on July 9, 2012 (PDUFA V), authorizes FDA to collect user fees for certain applications for approval of drug and biological products. This document establishes fee rates for fiscal year (FY) 2013 for application fees for an application requiring clinical data ($1,958,800), for establishment fees ($526,500), and for product fees ($98,380). These fees are effective on October 1, 2012, and will remain in effect through September 30, 2013. For applications submitted on or after October 1, 2012, the new fee schedule must be used. Drug / product U.S. Food and Drug Administration, 2012c.
Number of IRB Amendments Average number of amendments for a given therapeutic area and phase, as derived from Getz, et al., 2011. In this study, amendments were defined as “any change to a protocol requiring internal approval followed by approval from the IRB, ethical review board (ERB), or regulatory authority. Only implemented amendments—that is, amendments approved both internally and by the ethics committee—were counted and analyzed in this study.” Per-phase amendment counts were calculated by multiplying the average number of amendments by phase (from Table 3 in Getz, et al., 2011) by a therapeutic area-specific factor (calculated using numbers in Table 4 in Getz, et al., 2011). Study Getz, et al., 2011; calculation.
Number of IRB Approvals Average number of IRB approvals needed for a given study. The default scenario assumes this to be equal to the number of sites in a study Study Assumption based on data from Medidata Insights™ (Medidata Solutions)
Number of Patients The number of planned patients. This is the number of patients a site is expected and contracted to enroll. Site PICAS® (Medidata Solutions, 2012b)
Number of Project Management Months The time period from contract signature to the delivery of the statistical report. Project management month CROCAS® (Medidata Solutions, 2012a)
Number of SDV Fields Number of SDV fields per study Study CROCAS® (Medidata)
Number of Site Management Months Number of months a site was managed; the time period from the first site initiation to the last site close-out Site month CROCAS® (Medidata Solutions, 2012a)
Number of Site Monitoring Days The number of actual days spent at a given site (on-site) for monitoring purposes (not the total period over which monitoring was conducted) Site monitoring day CROCAS® (Medidata Solutions, 2012a)
Number of Sites Number of clinical investigator sites used per study/protocol Study Medidata Insights(Medidata Solutions)
Number of Trials in Phase The number of trials conducted in a given phase for the drug. Multiple trials within a phase might be required to test different dosages, for example. The default is one trial in each phase. Enter a whole number between 1 and 10 for Phases 1-3 and a whole number between 0 and 3 for Phase 4 (zero signifying that there is no Phase 4 trial). Whole number N/A
Site Overhead Percent Site overhead charged on contracts by the site estimated at 25 percent of total per-study costs Percent PICAS® (Medidata Solutions, 2012b)
Patient Recruitment Costs Advertising costs associated with recruitment of patients at the per-patient level Patient PICAS® (Medidata Solutions, 2012b)
Patient Retention Costs Amount paid to the patient for study participation, which might include financial compensation, reimbursement for travel, meals, etc Patient PICAS® (Medidata Solutions, 2012b)
Phase Time Total length of study phase, in years. For Phases 1, 2, and 3, this variable is equal to the maximum of the phase lengths from DiMasi, et al. (2003), the Number of Site Management Months from Medidata, and the Number of Project Management Months from Medidata. For Phase 4, this variable is equal to the higher of the Number of Site Management Months and the Number of Project Management Months. From DiMasi, et al. (2003): “The timeline is constructed from information on average phase lengths and the average gaps and overlaps between successive phases in a Tufts Center for the Study of Drug Development database of approved new drugs and in our cost survey.” The NDA/BLA review time includes the time from first submission of an NDA/BLA to regulatory marketing approval, and comes from DiMasi, Grabowski, & Vernon (2004). Trial phase times do not reflect differences between therapeutic areas; however, therapeutic areaspecific NDA/BLA review times were available and used for a select list of therapeutic areas. When the user specifies that there are multiple trials within a given phase, total phase time is defined as the average of the maximum phase time entered in any of the trials and the sum of all phase times entered in all of the trials. This is intended to account for the fact that trials may either be concurrent or sequential, depending on the circumstances. Years DiMasi, et al., 2003; DiMasi, Grabowski, & Vernon, 2004; CROCAS® (Medidata Solutions, 2012a)
Physician Costs Physician salary cost for one patient (physician salaries divided by the number of patients at site) Patient PICAS® (Medidata Solutions, 2012b)
Real Annual Discount Rate The rate at which clinical trial costs are discounted over the time period of the study/development process. Custom discount rates may be entered as decimals between 0 and 1 with leading zero (e.g., 0.15). Percent Drug sponsor interviews
RN/CRA Costs Clinical site staff cost (staff salaries divided by the number of patients at site) Patient PICAS® (Medidata Solutions, 2012b)
SDV Cost Data clean-up cost for one case report form (CRF) field Data field CROCAS® (Medidata Solutions, 2012a)
Site Monitoring Costs On-site monitoring cost for a single day Day CROCAS® (Medidata Solutions, 2012a)
Site Recruitment Costs Cost for CRO to evaluate and recruit one site (which may or may not involve a site visit) Site CROCAS® (Medidata Solutions, 2012a)
Site Retention Costs Cost for CRO to manage one site for one month Month CROCAS® (Medidata Solutions, 2012a)
Success Probability The percent chance that a trial will be successful in a given phase and progress to the next phase (or, in the case of the NDA/BLA review phase, the percent chance that the drug will be granted approval). The BioMedTracker success probabilities used represent ERG's best guess for most relevant therapeutic area; if figures were not available for a similar therapeutic area, general/overall percentages were used. Custom success probabilities may be entered as decimals between 0 and 1 with leading zero (e.g., 0.80). Only one success probability value may be specified for the entire set of trials within a given phase. Percent per phase Hay et al., 2011.
Worldwide Sales Revenues (millions of 2008 dollars) Worldwide sales revenues (in millions of dollars) over the product life cycle for new drugs approved in the United States during the period from 1990 to 1994 (net present values, discounted at 11% to the launch year). The revenue figures have been inflated from 2000 dollars to 2008 dollars (midpoint between 2004 and 2012, the range covered by the itemized cost data) using the producer price index for commodities in the category “Drugs and pharmaceuticals” from the Bureau of Labor Statistics (BLS). Drug / product (millions of 2008 dollars) DiMasi, Grabowski, & Vernon, 2004; Consumer Price Index (CPI) Inflation Calculator. (2012).

[a] Phase 1 study sites tend to have in-house or local labs as opposed to central labs.

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