Clinical Trials Innovation Regulation and Costs - Working Session - World Health Summit 2009 WS-435

Description:

Working Session

Clinical Trials: Innovation, Regulation and Costs

Co-Host: German Research Foundation

Chairs: Jürgen Schölmerich | Vice President | German Research Foundation (Deutsche Forschungsgemeinschaft)

Liselotte Højgaard | Professor & Director & Chair of the European Medical Research Council | Copenhagen

University Hospital

Outline: R&D productivity in the pharmaceutical industry as measured by bringing innovative therapeutics to market is at a low level. Clinical trials, the bedrock of pharmaceuticals R&D, have become the major time-consuming driver of costs and complexity.

The clinical paradigm is suffering from its regulatory burden, especially from complexity of varying national standards. It is not

prepared well to deal effectively with non-pharmacological therapeutics and advances such as personalized medicine and

regenerative medicine. Risk aversiveness is limiting the potential for new designs and statistical methods. The full potential of

patient stratification and use of biomarkers remains to be captured as well.

Furthermore, incentivation of industry on diseases with large patient populations and investigator-/academia-driven clinical

research under severe strain leaves many innovation opportunities untapped and, hence, public health needs unaddressed.

Especially investigator-driven clinical research is suffering from limited funding, lack of capabilities and huge complexity to deal

with (e.g., data sharing, intellectual property issues, regulatory burden).

In summary, we see untapped opportunities for innovation and a worsening trend of more complex regulations and more

complex protocols facing implementation challenges with too large patient populations. Academia, funding agencies, regulatory

agencies, and industry can resolve this multi-factorial issue only jointly.

This interactive working session will provide an insider’s view on the problem from the perspectives of investigators, funding

agencies, industry, and regulatory agencies. The aspiration is to conclude with a strong call for action on how to improve clinical

medical research in Europe and globally: how to limit the regulatory burden for national and multinational trials, how to optimize

targets and patient selection for clinical trials and how to assure trials of optimal statistical power, how to increase funding to

investigator-driven clinical trials, and how to capture benefits of scientific advances through encouraging new designs. This task

calls for collaboration of all relevant stakeholders.

11.00 Problem Statement

Jürgen Schölmerich | Vice President | German Research Foundation (Deutsche Forschungsgemeinschaft)

11.05 The Investigator’s Perspective

Brian Feagan | Director of Robarts Clinical Trials | Robarts Research Institute

Multiple issues should be considered in selecting patients for clinical trials. True experiments in humans must be conducted under optimal ethical and scientific conditions with patient safety being the ultimate consideration. Frequently tension exists between

selecting patients that are most likely to respond to an intervention (efficacy) and assessment of the intervention’s benefit in the

“real world” (effectiveness). Designers of clinical trials must make wise decisions regarding patient selection if meaningful results

are to be obtained. Examples from recent clinical trials will be used to explore these issues.

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