Perspectives on Biomarker and Surrogate Endpoint Evaluation Discussion Forum Summary (2011) / Chapter Skim
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5 Industry Perspectives
5 Industry Perspectives
Pages 51-68
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From page 51... ...
• Will a prospective biomarker evaluation process add clarity to product development and consumer understanding? • To the degree it is possible to respond, if at all, what are your key concerns for implementation of the recommendations?
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Another concern raised by several discussants was the possibility that the standards established by the recommended biomarker evaluation framework will have a chilling effect on research in this area.
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These are complicated diseases, and even when they are straightforward, the variation between patients, between individuals, between … population[s] , is massive." However, researchers believe that it will eventually be possible to identify biomarkers as surrogate endpoints for chronic diseases.
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that drugs cure and mitigate disease, while healthy foods reduce the risk of developing disease, Thomas Fleming noted that some drugs also serve the latter purpose; for example, interventions to prevent HIV transmission, type 2 diabetes, and Alzheimer's disease. Such drugs are rigorously scrutinized to ensure their efficacy, he continued, and if such claims are based on surrogate endpoints, those biomarkers must be validated.
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From page 55... ...
or pharmaceutical industry conduct can benefit the food industry, which can apply their results to help determine health claims for foods. Drugs are often a specific compound that is used to produce specific effects, whereas foods consist of many compounds which may produce many different effects.
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represents the world's leading food, beverage, and consumer products companies, by promoting sound policy, championing initiatives that increase productivity and growth, and helping to ensure the safety and security of consumer pack aged goods through scientific excellence, said Melissa Musiker, senior manager of science policy, nutrition, and health, at the Grocery Manufac turers Association. She noted that the report's publication had sparked discussion in the popular press as to the validity and appropriateness of all types of voluntary nutrition claims used on food product labels.
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From page 57... ...
However, since health claims are preventive in nature, the type of study needed to assess such a claim should differ from that used to support a disease treatment, she argued. "The scientific research needed to assess the validity of a biomarker for the purpose of voluntary nutrition claims presents unique challenges for foods," she said.
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From page 58... ...
"This, in turn, limits the ability to derive answers to important questions relating to the ability of diet, food, and food components to promote health and reduce the risk of chronic disease." The Council for Responsible Nutrition (CRN) has commented on the need for additional biomarkers as surrogate endpoints to both the FDA and the NIH, he reported.
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Williams said that in regard to biomarkers, the pharmaceutical industry wants the same thing as the FDA, as reflected in their charge to the committee: "to establish a framework for the level of evidence and the nature of evidence for biomarker utilization." However, such a framework should also enable drug companies and their regulators to reach a decision as to what level of evidence is needed for a particular biomarker application. He added that the pharmaceutical industry wants "consis tency and transparency in biomarker evaluation, rather than something … unpredictable and opaque," as reflected in the report.
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For example, the report said that "tumor shrinkage is not acceptable as a surrogate end point … partly because the analytic validation is not very good." However, within individual clinical trials, Dr. Williams noted that tumor size can be gauged accurately, "so the analytic validation can be controlled." In addition, he suggested that tumor shrinkage could have been used as an example to demonstrate evidence thresholds.
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Dr. Fleming responded, "I believe the reason you didn't see that is when we have 35 years of experience using this measure and now have extensive data regarding the relationship between the effects on tumor shrinkage and the effects on clinical endpoints, we're finding that we're not seeing a reliable assessment of efficacy using tumor shrinkage."
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However, if you don't recognize the outcome of a short trial in terms of lives saved, you wouldn't tolerate the risks inherent in using surrogate endpoints. Focusing on risk and downplaying benefit leads to "a kind of precautionary view of surrogate endpoints," much as Michael Lauer had expressed, Dr.
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Ball noted that the committee recommended that such expert panels comprise people representing a range of perspectives, because those are the people with the greatest expertise on the topic; he also noted that the FDA typically includes experts with these types of conflicts of interest on such panels.
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In considering whether a prospective biomarker evaluation process would add clarity to product development or consumer understanding with respect to IVD biomarker assays, Dr. Zakowski noted that such clarity is achieved through the following routes, most of which were also noted by Dr.
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• Recognize the interdependence of analytical validation, qualifica tion, and utilization. PRESENTATION By RICHARD kuNTz, MEDTRONIC Richard Kuntz, senior vice president and chief scientific, clinical, and regulatory officer at Medtronic, Inc., noted that his remarks reflected a quick review of the report from his personal perspective and not as a representative of the medical device industry.
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They also found that perturbing this system with treatments resulted in correlated responses by TLR and late loss. After more than 15 publications on their methodol ogy over the course of nearly a decade, he and his colleagues felt they had a established a simple relationship between late loss and TLR and a relatively simple mechanical model explaining the relationship, which would be useful in studying late loss (Mauri et al., 2005)
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From page 67... ...
new operators and new patients, with more efficient systems in the postmarket." He added that the development of patient registries, along with advances in computational models and observational statistics, should encourage such efforts. The concept of surrogate endpoints could be extended to several device design elements such as computational bioengineering modeling, Dr.
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