Large Simple Trials and Knowledge Generation in a Learning Health System Workshop Summary (2013) / Chapter Skim
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4 Medical Product Regulatory Issues
4 Medical Product Regulatory Issues
Pages 27-34
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From page 27... ...
• Closer attention to each element in the trial design could help to alleviate the complexity associated with unnecessary proce dures, protocol amendments, and irrelevant data collection. Granger • Trial quality is driven by whether it answers an important ques tion that will change clinical practice and improve outcomes.
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From page 28... ...
INTRODUCTION Clinical trials of all designs, including large simple trials, are often overly complex and costly. This chapter summarizes panel discussions on the current state of trial complexity, various strategies for reducing the complexity of clinical trials and increasing their efficacy, and the U.S.
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From page 29... ...
Moreover, many current studies are collecting more genetic material and biomarker data and involve a combination treatment or a diagnostic procedure, both of which add complexity and additional procedures to the clinical trial protocol. More data are being collected at all phases of clinical trials, Getz said, possibly because of pressure from regulatory agencies to collect more safety data or to determine whether a project should be terminated early.
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From page 30... ...
Possible reasons for such a high proportion of noncore procedures, Getz elaborated, include a desire to collect additional data to gain insight into a particular mechanism of action of a drug or even a new area of development, along with efforts to mitigate the risk that regulatory agencies will request additional data or resistance by purchasers and payers to pay for the drug once it is approved and on the market. Additionally, protocol designs from earlier phases of clinical trials are often reused for later studies, without consideration of whether all procedures from the former studies are necessary for the latter ones.
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From page 31... ...
Granger described the work of Lisa Berdan and the Duke Clinical Research Institute showing that the top 10 percent of trial sites enroll about 40 percent of all patients. Moreover, the cost of starting up the trial at each site is significant, estimated to be a minimum of $14,000, so the removal of those sites that will not be productive is a critical source BOX 4-1 Elements Determining the Quality of a Clinical Trial The trial must be performed • ith an adequate number of events to answer the question with w confidence; • in a practice setting to make results generalizable; • with proper randomization; • ith reasonably complete follow-up and definitive ascertainment of the w primary outcome; • with an aggregate safety assessment; • ith a plan for ongoing measurement, feedback, and improvement of w quality measures during the conduct of the trial; • ith safeguards against bias in determining clinically relevant outcomes w (like blinding)
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From page 32... ...
Additionally, further extension of these changes to include a 50 percent lower per patient study award reduced costs by a dramatic 60 percent. The model of the cost of a radically streamlined trial, which was limited to 100 high-volume sites, eliminated on-site evaluations and source data verification, used highly focused case report forms, and dramatically reduced payments to the study sites.
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From page 33... ...
Sherman continued by underscoring the idea that the collection of extraneous data points attributed to a fear of regulatory agencies is not supported by the reality of FDA's regulatory practices; what truly matters is meeting the primary endpoint. Of the utmost importance to FDA is data quality, Sherman explained, and only high-quality data can provide substantial evidence.
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From page 34... ...
If these characteristics do not apply to the trial in question, concerns about FDA inspection or requests for additional data points may be unnecessary. In terms of harmonization, Sherman continued, FDA has been focused on the streamlining of clinical trials to modernize and harmonize trial design.
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