MCC_Beyond EDC_20150622_Medidata_White-Paper

06/2015 WHITE PAPER

Medidata and other marks used herein are trademarks of Medidata Solutions, Inc. All other trademarks are the property of their respective owners.Copyright © 2015 Medidata Solutions, Inc.

Beyond EDC: Speeding Treatments to Patients with the Medidata Clinical Cloud

206/2015 WHITE PAPER BEYOND EDC: SPEEDING TREATMENTS TO PATIENTS WITH THE MEDIDATA CLINICAL CLOUD

Meeting the Challenge

Historically, scientific innovation alone in the absence of any significant pricing pressures was sufficient to maintain return on investment (ROI) and deliver on unmet medical needs. Now, the game has changed: scientific innovation must go hand in hand with operational innovation to meet the needs of patients and investors. Innovative drug development is not a luxury, but a societal and market necessity. New market leaders will be those sponsors that are able to achieve the efficiencies required to cost effectively support their scientific and operational delivery needs. To this end, one efficiency will be invaluable—an integrated clinical development technology platform will play a major role in the strategy of each leader.

Using Medidata Clinical Cloud to Deliver Enhanced Value to the Clinical Development Lifecycle

Life sciences organizations globally spend $90+ billion per year on clinical development, including $51 billion on processes and operations1 and approximately $2 billion2 on clinical trial technology. Leveraging the benefits of platform technology, as opposed to loosely connected point solutions, impacts these numbers, substantially reducing sponsors’ overhead.

When sponsors use Medidata’s approach to optimizing the development process—or turn to clinical research organizations (CROs) who do—they gain superior operational efficiency because Medidata uniquely integrates technology and science. This operational innovation frees sponsors to concentrate on their core competencies.

Medidata’s integrated technology approach allows sponsors to optimize every component of the clinical trial process, including:

• Study Planning

• Site Engagement

• Patient Engagement

• Study Conduct

With the Medidata Clinical Cloud®, sponsors can efficiently roll out the entire clinical research workflow—rather than focusing simply on discrete activities—allowing them to realize the full operational and business value that clinical trial technology promises.

Patient Data

Monitoring

Site Payments

Data Analytics Data Capture &

Management

Study Planning

Figure 1: Medidata Clinical Cloud Capabilities


Best Practices Supporting Clinical Trial Implementation Innovation

To understand how a sponsor can obtain superior operational efficiencies with Medidata, let’s examine each category of the clinical trial process.

Improve Study Planning

The ChallengeGrowing rates of avoidable protocol amendments and excessive procedures in study plans compromise trial efficiency. Almost 25 percent of procedures in a protocol are unconnected to any key endpoint: primary, secondary, screening, safety, treatment or exploratory.3 While the average case report form (CRF) length has increased by 300 percent over the past 10 years4, up to 30 percent of data collected by sponsors is never used for an FDA submission.5 Moreover, nearly 40 percent of protocol amendments occur before the first subject first dose.6 The Tufts Center for the Study of Drug Development has found that excess procedures have helped increase the execution burden on sites by 11 percent per year.7 For sponsors and CROs, this means longer trial times, lower patient recruitment and retention, and inefficient data collection and analysis.

Best PracticeOptimize study design, first, by aligning a protocol to a set of defined study objectives, eliminating study activities that do not support defined endpoints. Second, benchmark protocol design against industry procedure costs, usage and frequency to reduce unneeded complexity and costs.

Medidata: Best Practice, in Action Medidata’s Clinical Cloud allows sponsors to optimize study and protocol design, resulting in higher quality protocols. The platform provides industry benchmark data for the cost and frequency of individual procedures. This helps trial planners understand the impact of different protocol designs on the critical areas of patient enrollment, overall trial cost and timeline. Shaping a trial to focus on only relevant patient data can boost patient enrollment and safety by reducing invasive procedures. As well, it can significantly reduce total trial cost. From there, the Medidata Clinical Cloud provides budgeting, contracting and outsourced (i.e., CRO) planning to negotiate with the sites for study execution.

Proof points Analyses done by Medidata’s Data Science and Operations Team in conjunction with sponsors show the Clinical Cloud has had the following impacts on trial costs and data quality:

• Reduced procedures in a protocol by up to 50 percent without impacting scientific excellence, creating savings of $450,000 to $9 million per study.

• Reduced the number of avoidable amendments by up to 44 percent.

• Lowered the average site grant per patient by 2-5 percent, contributing to savings of $20 million for one sponsor.

Using the Medidata Clinical Cloud, a sponsor can optimize protocol design to reduce unnecessary procedures and amendments, quickly plan resource allocation and provide precise expenditure projections.

Patient Data

Monitoring

Site Payments

Data Analytics Data Capture &Management

Study Planning • Protocol design• Budgeting• Contracting• Outsourced planning

• Study cost benchmarks

• Reduce procedures by 50%• Reduce number of avoidable amendments by 44%• Lower site grant per patient by 2-5%

Figure 2: The Value of Improving Study Planning


Improve Site Engagement

The ChallengeSite and subject enrollment is a key component to meeting study timelines, but alarming statistics illustrate how difficult it can be to meet these study objectives. One survey of trials active between 2003 and 2006 shows that only about a quarter of patients initially screened actually completed a trial, a near 50 percent drop from just a few years prior.8 Additionally, almost a quarter of sites never enrolled a single patient,9 and approximately 25 percent of all principal investigators interviewed said they would never participate in a clinical trial again.10 While more recent information is unavailable, enrolling sites and subjects for a trial remains difficult and continues to be a top challenge for sponsors today.

Best Practice Optimize site and subject enrollment based on key study measures given in real time to identify any potential enrollment risks. Seeing measurements in real time, sponsors can modify the study or implement a recruitment rescue strategy before recruitment and retention rates fall low enough to negatively impact study timelines.

Medidata: Best Practice, in ActionSite enrollment success and data quality are not determined by any one factor. That’s why the Medidata Clinical Cloud’s capabilities span risk-based monitoring, global payments, site visit reporting and site document management. With this platform, sponsors are able to reduce operational resource overhead while leaving more

time for site training and relationship building. Most of all, the platform allows investigators to stay focused on enrollment and study protocol by lowering site burden. Additionally, the study will benefit from lower costs through automated global risk-based monitoring; reinforced milestone attainment resulting from timely, accurate and automated site payments; and shorter timelines to author, review, approve and publish monitoring reports.11

Proof points Sponsors and CROs report that by unifying their clinical trial management system (CTMS) and EDC processes in the Medidata Clinical Cloud they achieved:

• a 42 percent reduction in time to file and approve a monitoring visit report

• a 40 percent increase in clinical research associate (CRA) productivity.

• a 10 percent reduction in CRA turnover

• a 60 percent reduction in site payment processing time

By unifying clinical trial management, monitoring and payments with electronic data capture (EDC), the Medidata Clinical Cloud allows sponsors to rapidly manage, track and report on key study activities and timed deliverables from study start-up through site close-out.

Patient Data

Monitoring

Site Payments

Data Analytics

Data Capture& Management

• EDC / CDMS• Medical coding• Randomization• Trial drug supply optimization• Safety reporting

• Site selection• Milestone tracking• Visit reports• Statistical analysis• Targeted source data verification• KRIs

• Site enrollment benchmarks

• Payment request• Cost forecasting• Accrual tracking

• 42% faster monitoring visit report approval• 40% increase in CRA productivity • 10% reduction in CRA turnover • 60% faster site payment processing

Study Planning

Figure 3: The Value of Improving Site Engagement


Improve Patient Engagement

The ChallengeAn effective method to increase patient engagement, for areas like quality of life improvements of a treatment, is to involve more patient-reported outcomes (PROs). The rising importance of PROs and the greater availability of digital technology to support clinical trials have led to greater use of ePRO in place of paper-based PRO systems.12 A recent Tufts survey shows the majority of respondents preferred using ePRO over paper; however, more than 60 percent stated they believe paper is less expensive.13 An analysis published in Applied Clinical Trials finds that the break-even point for traditional ePRO solutions over paper is at 40,000 pages of data.14 Special-purpose ePRO systems have been designed to support trials with less than 40,000 pages, but they come at an even higher cost burden, meaning poor ROI still restricts a swath of trials from accessing the advantages of ePRO.

Best PracticeAccording to Tufts CDSS 2014 research, the top benefits of ePRO over paper-based systems are increased data quality, patient compliance and efficiency of data collection.15

ePRO also frees sites from the logistics, transcription and archiving of the physical assets, and allows sponsors to cut the costs of these and related activities from their budgets. What’s more, the FDA notes that “data quality in general needs industry’s attention,” and to this end promotes capturing source data in electronic form.16,17 Now, with the majority of patients connected 24×7 via smartphones and ePRO apps, clinical trials do not need to rely solely on special-purpose ePRO systems.

Medidata: Best Practice, in ActionSpecial-purpose ePRO systems are simply too costly to consider for the majority of studies with smaller volumes of assessments—under 40,000 pages—and tighter budgets. The alternative, Medidata Patient Cloud® ePRO integrated with Medidata Rave®, provides an ROI that allows sponsors to eliminate paper-based PROs at extremely low volumes of assessments.

Proof pointSponsors and CROs that connect Medidata Rave to Medidata Patient Cloud ePRO deliver the benefits of ePRO to studies that contain more than 50,000 pages of assessments to as few as 100 pages.

Improve Study Conduct

The ChallengeClinical trial costs have dramatically increased over the past decade. This reflects how complex trials have become in order to meet clinical development requirements. Ten years ago, the average Phase III clinical program comprised more than 1,400 patients and 67 sites. It cost $86.3 million out-of-pocket and had nearly 70 percent chance of reaching market successfully.18,19 Today, the average Phase

Unifying Medidata Rave’s EDC with randomization, safety monitoring, trial supply and electronic trial master file capabilities provides a “single source of truth” that accelerates study conduct and close-out

Medidata’s unified electronic patient-reported outcomes (ePRO) and EDC provides a single source of patient data and allows a sponsor to extend Rave studies to engage more patients on more assessments.

Pages of Assessments Patient Cloud Traditional ePRO

100 No

1,000 No

5,000 No

10,000 No

50,000+

Figure 4: Patient Cloud Improves Patient Engagement at All Levels


III clinical program comprises over 2,800 patients at more than 100 sites. It generates out-of-pocket costs in excess of $235 million and has 50–60 percent chance of bringing a treatment to market.20,21 One of the best ways to reign in the growing costs of trials is to simplify their complexity with smart technology. Some sponsors turn to loosely connected point solution technologies. While these point solutions address specific areas, they inversely relate to trial complexity. As complexity increases, their efficacy as tools for managing the trial, from both the business and scientific perspectives, decreases. To continue to be effective and reduce costs, management tools must have a broader and more unified scope of capabilities.

Best PracticeOptimize study conduct by tightly coupling processes to enable a sponsor to proactively identify risks before they impact study timelines, data integrity and cost.

Medidata: Best Practice, in Action Today’s clinical development environment requires the holistic integration of technologies to drive continuous process improvement and gains in productivity. Comprehensive integration that unites business with scientific needs transforms the value proposition of the clinical research process itself.22 Medidata Clinical Cloud is a unified technology platform consisting of pre-configured modules, clinical libraries and centrally managed templates that efficiently capture, report, process and analyze trial data.

• Integrated EDC and randomization brings simplified planning, setup and implementation to a range of allocation methodologies. Automatically integrating with Rave EDC provides a single sign-on, integrated experience for investigative sites, eliminating the need for separate web or telephone-based systems.

• Integrated medical coding supports internal and outsourced clinical researchers in a secure cloud environment. MedDRA®, MedDRA/J®, WHO Drug and JDrug dictionaries come pre-configured, and specialty, company and other dictionaries are easily imported and migrated, with streamlined dictionary upgrades.

• Safety reporting system integration eliminates redundant paper collection and minimizes reconciliation processes. It leverages the clinical data already captured in Rave for serious adverse events (SAEs) and adverse events (AEs) and automatically forwards it to a safety system, with or without manual intervention and with notification to designated personnel, as configured.

Proof point

• Using common design standards, sponsors were able to re-use forms, edit checks and other study design elements, reducing database build times from 11–12 weeks down to 7–8 weeks. For one sponsor, the median cycle time improvement from last patient, last visit to database lock went from 30 days to just 10.

• Sponsors and CROs that use Medidata Rave with Medidata Balance® report reduced data entry cycle time by up to 35 percent.

• CROs and sponsors that connect Medidata Rave to Medidata Rave Safety Gateway cut SAE reconciliation time by up to 81 percent.

Patient Data

Monitoring

Site Payments

Data Analytics

Data Capture& Management

• EDC / CDMS• Medical coding• Randomization• Trial drug supply optimization• Safety reporting

• Trial cycle time benchmarks• Shorten database build times by 41%• Reduce cycle time from LPLV to database lock by 67% • Lower data entry cycle time by 35% • Cut SAE reconciliation time by 81%

Study Planning

Figure 5: The Value of Improving Study Conduct


The Bottom Line

Today’s complex biopharmaceutical, diagnostic and medical device development environment requires the holistic integration of technologies delivered as a platform to drive continuous process improvement and exponential gains in productivity.

The benefits of supporting the entire clinical research workflow with a single platform go beyond eliminating redundant tasks with multiple associated reconciliations. A truly optimized clinical trial has enhanced transparency, workflow and collaboration, which results in lower total costs, shorter timelines and reduced risk.

Medidata has unified the clinical development and trial execution process through a single cloud-based platform that addresses clinical research needs from trial concept to conclusion.

About MedidataMedidata Solutions is the leading global provider of cloud-based solutions for clinical research in life sciences, transforming clinical development through its advanced applications and intelligent data analytics. The Medidata Clinical Cloud™ brings new levels of productivity and quality to the clinical testing of promising medical treatments, from study design and planning through execution, management and reporting. We are committed to advancing the competitive and scientific goals of global customers, which include over 90% of the top 25 global pharmaceutical companies; innovative biotech, diagnostic and device firms; leading academic medical centers; and contract research organizations.

[email protected] | mdsol.com | +1 866 515 6044

Medidata Clinical Cloud™

Cloud-based clinical research solutions | Innovative technology Data-driven analytics | Reduced costs | Improved time to market Faster decisions | Minimized risk


Endnotes1. K. Getz et al., “Assessing the Impact of Protocol Design Changes on Clinical Trial

Performance,” American Journal of Therapeutics, vol. 15, issue 5 (Sep-Oct 2008): 450–7.

2. PAREXEL’s Bio/Pharmaceutical R&D Statistical Sourcebook 2009/2010 (Waltham: PAREXEL International Corporation, 2009).

3. American Journal of Therapeutics (October 2012).

4. K. Getz et al., “Assessing the Impact of Protocol Design Changes on Clinical Trial Performance,” American Journal of Therapeutics, vol. 15, issue 5 (Sep-Oct 2008): 450–7.

5. “Trends and Implications of Increasingly Complex Protocol Designs,” Tufts Center for Study of Drug Development (CSDD), 2009 AMIA Spring Congress.

6. K. Getz, “Protocol Amendments: A Costly Solution,” Applied Clinical Trials, May 1, 2011.

7. K Getz et al., “Assessing the Impact of Protocol Design Changes on Clinical Trial Performance,” American Journal of Therapeutics, vol. 15, issue 5 (Sep-Oct 2008): 450–7.

8. “Trends and Implications of Increasingly Complex Protocol Design,” Tufts Center for Study of Drug Development (CSDD), 2009 AMIA Spring Congress.

9. Medidata Insights Database Analysis (2011).

10. Centerwatch Analysis (2005).

11. For additional savings calculations, see Medidata Solutions white paper, “Accelerate Time to Revenue with Less Risk and Cost Using the Medidata Clinical Cloud” © 2013 Medidata Solutions, Inc.

12. CC.B. Forrest, “Digitization of Patient-Reported Outcomes,” Value in Health, vol. 16, issue 4 (June 2013): 459-460.

13. “Study of ePRO Usage in Clinical Trials,” Tufts Center for Study of Drug Development (CSDD), June, 2014.

14. José, Nadina C., Langell, Kai. “ePRO vs. Paper,” Applied Clinical Trials, vol. 19, issue 6 (June 2010).

15. “Study of ePRO Usage in Clinical Trials,” Tufts Center for Study of Drug Development (CSDD), June, 2014.


16. Food and Drug Administration, Center for Drug Evaluation and Research et al., Guidance for Industry Electronic Source Data in Clinical Investigations, September 2013. Available at http://www.fda.gov/downloads/drugs/guidancecom plianceregulatoryinformation/guidances/ucm328691.pdf

17. Wayne Koberstein, “Janet Woodcock’s Quality Agenda At CDER,” Pharmaceutical Online, February 2014. Available at http://www.pharmaceuticalonline.com/doc/ janet-woodcock-s-quality-agenda-at-cder-0001

18. J. A. DiMasi, R. W. Hansen, H. G. Grabowski, “The Price of Innovation: New Estimates of Drug Development Costs,” Journal of Health Economics, vol. 22, issue 2 (March 2003): 151–85/

19. PAREXEL’s Bio/Pharmaceutical R&D Statistical Sourcebook 2008/2009 (Waltham: PAREXEL International Corporation, 2008).

20. A. Malani and T. Philipson, “Clinical Trials, the Market for Observations, and the Cost of Medical R&D,” University of Chicago, July 11, 2008.12/

21. S.M. Paul et al., “How to Improve R&D Productivity: The Pharmaceutical Industry’s Grand Challenge,” Nature Reviews Drug Discovery 9, (March 2010): 203–14/

22. Centerwatch Analysis (2007)/

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