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PHT Insights — When and How to Justify Electronic                   Patient-Reported Outcomes (ePROs)Trial conditions dict...
2When and How to Justify Electronic Patient-Reported Outcomes (ePROs)M     any clinical trial managers within Sponsors and...
3   When and How to Justify Electronic Patient-Reported Outcomes (ePROs)    Step 2:                                       ...
4When and How to Justify Electronic Patient-Reported Outcomes (ePROs)Table II: How to Quantify Specific ePRO BenefitsBenefit ...
5When and How to Justify Electronic Patient-Reported Outcomes (ePROs)Table II: How to Quantify Specific ePRO Benefits       ...
6                                                                                 Call PHT for these FREE ePRO decision to...
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When ePRO

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The importance of learning how patients feel and function when taking a new clinical therapy has been acknowledged by the FDA, EMA and other global regulatory authorities. Sponsors currently engaged in drug development programs appreciate and leverage the added value of patient-reported outcome (PRO) data. They no longer ask if PROs should be collected, but what phase to begin PRO collection.

This issue of Insights is intended to identify the costs (delays and expenses) of collecting patient-reported outcomes on paper; and compare these against electronic PRO capture. The intention of this issue to provide clinical teams with industry data that can refute the presumption that paper methods are cheaper than ePRO.

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Transcript of "When ePRO"

  1. 1. PHT Insights — When and How to Justify Electronic Patient-Reported Outcomes (ePROs)Trial conditions dictate whether and when ePRO shouldsupersede paper diaries.The importance of learning how patients feel and function But many mid-sized pharmaceutical companies and CROs arewhen taking a new clinical therapy has been acknowledged just beginning to explore ePRO. For them, comparing the costsby the FDA, EMA and other global of paper diaries to ePRO requires assigningregulatory authorities. Sponsors costs to the associated support functionscurrently engaged in drug development ‘…patient reported outcome for paper methods. Such costs (in time andprograms appreciate and leverage evidence is increasingly money) are distributed across departmentsthe added value of patient-reported viewed as an essential (clinical operations, data management,outcome (PRO) data. They no longer complement to traditional submissions, etc.) and it can be nearlyask if PROs should be collected, but objective clinical evidence impossible to fully account for the manywhat phase to begin PRO collection. for establishing a product’s distributed costs of paper.While it is well known that collecting competitive advantage in This issue of Insights is intended to identifysubjective data on electronic devices the marketplace.’ 1 the costs (delays and expenses) of collecting(instead of on paper diaries) reduces patient-reported outcomes on paper; andtrial costs and duration, quantifying compare these against electronic PROthe return on investment ePRO provides by trial phase can capture. The intention of this issue to provide clinical teamsbe elusive. Most major pharmaceutical companies have with industry data that can refute the presumption that paperdeclared ePRO as the de facto standard for PRO endpoints. methods are cheaper than ePRO. Contents Trial conditions dictate whether and when ePRO should p.1 How to Determine if ePRO supersede paper diaries. Suits Your Trial Conditions eClinical Trial Solutions Market, Global, Cost Saving p.2 This issue of Insights assumes Scenario of ePRO Solutions, 2008 that the reader understands that ePRO is a good fit for a Step 1- Identify and select your company’s trial technol- p.2 trial, and needs to justify the ogy adoption stage costs of automation. Step 2- Identify the hidden cost centers for collecting p.3 A previous issue of Insights PRO on paper diaries entitled ‘Collecting Electronic Step 3 - Compare your trial conditions to average or p.3 Patient-Reported Outcomes (ePROs): Comparing the 5 Proven median costs for global trials within Phases 2 & 3 Ways to acquire Attributed Patient-Reported Data’ details Table II: How to Quantify Specific ePRO Benefits p.4 how to determine if ePRO is appropriate for a particular trial. This is available for download at http://www.phtcorp.com Conclusion p.6 /resources/insights_newsletters/
  2. 2. 2When and How to Justify Electronic Patient-Reported Outcomes (ePROs)M any clinical trial managers within Sponsors and CROs are familiar with the data quality and integrity benefits of ePRO, Step 1:and recommend ePRO for phase 3 trials where PRO measures serve asendpoint data. Documented advantages of ePRO include This document is intended to assist in calculating the• Simplification of site tasks (storing, correcting, shipping paper diaries), financial advantages of ePRO over Paper in 3 steps• Reductions in data variance, Step 1: Identify and select your company’s trial technology adoption stage;• Shorter study duration, Step 2: Identify the hidden cost centers for collecting PRO on paper diaries; and• Improved protocol compliance through real-time monitoring (via time-stamped data entry reports), and Step 3: Compare your trial conditions to average or median costs for trials within Phases 2 & 3.• Reduced time to database lock. Identify and select your company’s trialCost Saving Scenario of ePRO Solutions technology adoption stage: To frame the context of your decision, clinical trial managers should Paper Diaries ePRO acknowledge the current level of ePRO adoption at their organization. PHT has found the stage of ePRO adoption, as defined below, helps frame the Subject decision and ROI for using ePRO in a particular trial. The adoption stage helps specify which ePRO attributes will be most important to a given trial Site Review and ROI evaluation. The stages: Colection Site Monitor X Saved Cost Stage1 Sent to DM Sponsors are starting to use a basic ePRO system for simple uses, such as integrated voice response (IVR) for patient qualification, enrollment Data Entry and/or randomization. Paper is the most frequently utilized method of data collection. Sponsor DB Stage2 Medical Monitor and Statistician IT groups within Sponsors are adopting EDC technologies to gain trial data efficiencies. Having recognized value from simple IVR ePRO use, these Source: GBI Research sponsors are testing other ePRO modalities such as mobile data capture devices for the subject and/or for the site. Since ePRO systems drive cross-The paper diary method is a lengthier process than ePRO, resulting functional benefits, data management and health outcome professionalsin increases in cost and time. If Pharmaceutical Companies are collaborate on ePRO evaluations.adopting ePRO solutions, they save considerable cost and time.2 Stage3 These Sponsors have shifted from paper to EDC. For those trials whereSponsors and CROs recognize that the ‘…efficiencies linked to new primary and secondary endpoints will be drawn from data collected directlytechnologies generally increase over time as end users gain familiarity with from patients, the clinical teams expect to use ePRO. Clinical and data teamsthem through repeated use.’3 As a result of the quantifiable efficiencies and prefer ePRO over paper although in some instances management may requiredownstream savings of ePRO, more clinical trial managers are also electing them to use paper for certain trials. For sponsors at this stage, outsourcingePRO for their phase 2 trials. Given the critical proceed or stop decisions professionals are often responsible for selecting CROs and ePRO systems thatthat are often made based on Phase 2 results, this makes sense even when are proven to be successful for large international projects and who have anthere are relatively fewer subjects and a shorter timeline. Such decisions unblemished record with regulatory authorities.benefit from a full understanding of the experience of each of the subjects,and ePRO consistently delivers more pertinent information per subject. Stage4Unlike their counterparts within the top ten pharmaceutical giants who All Sponsor clinical management systems are fully integrated with one or morehave come to regard paper diary collection as the exception, clinical trial EDC systems. All note taking, signatures, lab results, imaging and ePROs aremanagers within mid-sized pharmas and biotechs must demonstrate electronic. ePRO Systems are used for trials in phases 2 and 3, and in phasethe financial advantages of collecting PRO electronically, since their 4 (post marketing surveillance) trials. Sponsors seek long-term partnershipsorganizations are still accustomed to paper diaries. with vendors, especially those willing to share trial risks.
  3. 3. 3 When and How to Justify Electronic Patient-Reported Outcomes (ePROs) Step 2: Step 3: Identify the hidden cost centers for Compare your trial conditions to collecting PRO on paper diaries: average or median costs for global After identifying your adoption stage, the next step is to identify trials within Phases 2 & 3: which ePRO benefit(s) are most relevant to the evaluation of paper Refer to Table II–How to Quantify Specific ePRO Benefits on vs. ePRO data collection. page 4 to identify which ePRO benefit to quantify, based on your For example, clinical trial managers whose organization or organization’s technology phase: therapeutic division is in Stage1 for technology adoption are advised Example1: Sponsor in Stage1 technology adoption to focus on reduced site burden, increased compliance, confirmed time-stamped data entry, increased data availability, reduced data Phase 2 trial with 160 subjects and 19 sites. loss and decreased enrollment tracking time: Average cost $4,160,000. ePRO will conservatively save 10% ($416,000.) of trial costs for these reasons: Technology Stage • Reduced site burden $300,000.     ePRO Benefits • Increased compliance 67,000. Stage1 Stage2 Stage3 Stage4 • Confirmed time-stamped data entry 141,000.A.   Reduced  site   burden   X X X X • Increased data availability 162,000.B.   Timeliness  of   diary  presentation    X X X X • Reduced data loss 52,000.C.   Confirmed  time stamped data entry     X X X X • Decreased enrollment tracking time 15,000.D.  Increased  data  availability  X X X X Using calculations from Table II, theE.   Reduced  data  loss  X X X X projected savings would be $737,000. (18%)F.  Decreased  enrollment   tracking  time  X  X X  X G.  Decreased Data Variance       X  X X  X  Example2: Sponsor in Stage2 technology adoptionH.  Decreased Database Lock time       X X X Phase 3 trial with 800 subjects and 50 sites.I.   Fewer  queries  and  faster  resolution  time X X XJ.   Reduced  trial  duration  X X X Average cost $15,440,000.K.   Decreased  monitoring  visits  X X ePRO will conservatively save 10% ($1,544,000.) of trial costs for these reasons:L.  Increased reuse  of systems & components         X X • Reduced site burden $600,000. • Increased compliance 247,000. • Confirmed time-stamped data entry 525,000. • Increased data availability 602,000. “Using an e-diary is always recommendable, • Reduced data loss 193,000. when assessing principal criteria, whatever the • Decreased enrollment tracking time 56,000. phase (II or III). In this case, the improvement • Decrease in data variance 154,000. in data quality with ePRO far exceeds the • Decreased database lock 1,315,000. • Fewer queries and faster investment in technology.” resolution time 309,000. Mrs. Silvia Munoz Chimeno Using calculations from Table II, the Clinical Project Manager projected savings would be $3,901,000. (25%) Servier Spain
  4. 4. 4When and How to Justify Electronic Patient-Reported Outcomes (ePROs)Table II: How to Quantify Specific ePRO BenefitsBenefit of ePRO How to Calculate7 ePRO SavingsA. Reduced site burden When enrollment criteria for studies is calculated by ePRO, a significant burden from staff is removed. ePRO also ensures that only patients who belong in the study are actually enrolled.4 Cost benefit of Reduced Site Burden: • It costs $20,000 to activate a site and $2,000/mo to maintain one • Average number of sites in Phase 2 & 3 global trials is 35 • Average duration of Phase 2 & 3 global trials is 33 months • Average site activation and maintenance is $3,000,000 with paper PRO ePRO reduces site burden of paper administration by 20% $600,000B. Increased compliance The worthlessness (inaccuracy, untimeliness) of [paper diary] data is now well accepted, beginning with anecdotal evidence experienced by researchers decades ago and proven in controlled examination like that published in the British Medical Journal in 2002.5 Cost benefit of Increased Compliance: • Data Cleaning consumes 8% of trial time and cost • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Data Cleaning costs $784,000 with paper PRO ePRO improves compliance by 20%, saving that in Data Cleaning $157,000C. Confirmed time-stamped data entry When evaluating PRO-based claims, we intend to review the clinical trial protocol to determine what steps were taken to ensure that patients understood the instrument recall period.6 Cost benefit of Timely Diary Presentation: • Patient treatment after enrollment costs 17% of the trial • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Patient treatment after enrollment costs $1,700,000 with paper PRO ePRO reduces patient treatment costs by 20%, eliminating the cost of diary data that cannot be submitted due to illegible, untimely or irrelevant paper entries $340,000D. Increased Data Availability Cost benefit of Increased Data Availability: • Site Monitoring consumes 7% of trial time and cost • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Site Monitoring costs $685,000 with paper PRO • Increased data availability with ePRO reduces site visits by 20% $137,000 • Subject Monitoring consumes 10% of trial time and cost • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Subject Monitoring costs $980,000 with paper PRO • ePRO reduces study coordinator time required to collect, review and assign new paper Daily Diaries at each visit, by 25% $245,000 Total ePRO savings of increased data availability $382,000
  5. 5. 5When and How to Justify Electronic Patient-Reported Outcomes (ePROs)Table II: How to Quantify Specific ePRO Benefits continuedBenefit of ePRO How to Calculate ePRO SavingsE. Decreased Data Loss Cost benefit of Decreased Data Loss: • Statistical Analysis consumes 5% of trial time and cost • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Statistical Analysis costs $490,000 with paper PRO ePRO reduces statistical analysis by reducing paper data loss by 25% $122,500F. Decreased Enrollment Tracking Time Cost benefit of Decreased Enrollment Tracking Time: • Site Selection/Enrollment/Contract consumes 12% of trial time and cost • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Site Selection/Enrollment/Contract consumes $1,176,000 with paper PRO • Enrollment tracking consumes 6% or $588,000 with paper PRO ePRO decreases study coordinator enrollment tracking time by 50% $294,000G. Decrease in Data Variance Cost benefit of Decreased Data Variance: • Statistical Analysis consumes 5% of trial time and cost • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Statistical analysis costs $490,000 with paper PRO ePRO reduces data variance by 20% $98,000H. Decreased Database Lock Time Cost benefit of Decreased Datase Lock Time [valid for Phase 3 trials]: • Data Cleaning consumes 8% of trial time and cost • Average cost of Phase 3 global trials is $15,440,000 • Data Cleaning consumes 8% , or $1,235,000 with paper PRO • ePRO reduces data cleaning by 20% $247,000 • Average cost of Phase 3 global trials is $15,440,000 • Database lock consumes 37% of the trial time and cost, or $5,712,800 • ePRO reduces database lock time by 18.7% $1,068,000 Total ePRO savings of decreased database lock time. $1,315,000I. Fewer Queries & Faster Resolution Time Cost benefit of Reduced Queries: • Subject Monitoring & Administration consumes 10% of trial time and cost • Average cost of Phase 2 & Phase 3 global trials is $9,800,000 • Subject Monitoring & Administration costs $980,000 with paper PRO • Queries and data cleaning consumes 25% of Subject Monitoring & Administration, or $245,000 ePRO reduces queries by 80%, saving 2% of trial time and cost $196,000
  6. 6. 6 Call PHT for these FREE ePRO decision tools: Conclusion There are many tangible economic reasons to utilize PRO Selection Tool: ePRO instead of paper diaries in a clinical trial. Data • Which collection method for PRO data will best supports the solid business case to elect ePRO for trials support your trial—Paper or ePRO? across phases whenever the patient perspective is critically important to prove the safety and efficacy of a therapy. Even if the patients’ perspective is NOT critical, Sponsors may elect ePRO to remain competitive and elicit maximum data regarding patient experiences, side 1. Will PRO data be used to support 2. Is the PRO data intended for an 3. Are you collecting sensitive, personal 4. Is subject recruitment difficult? PAPER a claim? FDA/EMEA/other data? • Paper is practical for supervised effects, outcomes and the overall benefits a given drug PRO data collection where the agency Fast Track, subject population is abundant, Accelerated Approval PRO data are not personal, or when there are no plans for PRO NO or Priority Review? NO NO NO data submission. YES YES YES YES offers vs. the competition. ePRO • Regulatory agencies recommend that the PRO data collection method be capable of ensuring that reports are done on schedule, as defined by ePRO • ePRO on-demand data enables rapid access for independent evaluation or analysis, and is appropriate for any regulatory body that will want to meet with ePRO • Subjects welcome the opportunity to record without embarrassment, and appreciate that caregivers and family lack access to their personally revealing ePRO • ePRO data has been shown to cluster to the mean value (lower standard deviation) compared to paper data, enabling smaller sample sizes in Phase 2 ePRO • Provides regulatory agencies the protocol time requirement. “If a patient diary the sponsor regularly to review the data (possibly for information. trials and more rapid, conclusive Phase 3 trials. with proof that the protocol or some other form of unsupervised data entry is an expedited process). time requirements were met used, we plan to review the clinical trial protocol • Subjects are more willing to provide more to determine what steps are taken to ensure that • Rapid access to data enables adaptive design and complete data. • Provides on-demand data for other trial efficiencies. evaluation or analysis patients make entries according to the clinical trial design and not, for example, just before a • Improves subject compliance More robust online ROI and ePRO Decision Tools are clinic visit when their reports will be collected.” when private or sensitive data is collected • Enables smaller sample sizes Insist on ePRO data for rapid analysis. available from your PHT Account Executive. These Tools are designed to guide clinical trial managers through www.phtcorp.com ©2010 PHT Corporation v12010 Trust Your Patient Data US 1.877.360.2901 EUR 41.22.879.91.00 a short series of questions about their trial, in order to summarize which ePRO modality is best suited for the trial, and how to quantify and justify collecting trial data Online ePRO Decision Tool: on ePRO vs. paper diaries. • Which ePRO collection method will best support your protocol? PHT SitePad Tablet –The mobile touch-screen tablet for ePRO data collected at sites US 1.877.360.2901 • EUR 41.22.879.91.00 1 Doward et al, “Patient reported outcomes: looking beyond the label claim”, August 2010. PHT LogPad– 2 GBI Research, “The Future of eClinical Trial Solutions - Market Forecasts to 2015, Competitive Hand Held Benchmarking and Case Studies,” GBI Research, GBIHC021MR (December 2009): p.3. for patients 3 Applied Clinical Trials, “The Upfront Cost Hurdle of EDC”, April 2010: p. 52 on-the-go 4 Pompa et al, GlaxoSmithKline, July 2008 Powerpoint presentation “ePRO Awareness Session”. 5 Stone, et al., Patient non-compliance with paper diaries, BMJ 324: 1193, 18 May 2002. 6 FDA, Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims, p.14, December 2009” 7 Paraxel’s Bio/Pharmaceutical R&D Statistical Sourcebook 2009/2010, with extrapolations.US HEADQUARTERS: EUROPEAN HEADQUARTERS:PHT Corporation PHT Corporation Sàrl www.phtcorp.com500 Rutherford Avenue 2, chemin Louis-Hubert Copyright © 2010 PHT CorporationBoston, MA 02129 USA 1213 Petit-Lancy, Geneva, Switzerland Rev 092010Toll-Free: US 1.877-360-2901 Phone: 41.22.879.91.00

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