Perspectives on Biomarker and Surrogate Endpoint Evaluation Discussion Forum Summary (2011) / Chapter Skim
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8 Key Themes, Challenges, and Opportunities
8 Key Themes, Challenges, and Opportunities
Pages 87-96
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From page 87... ...
BIOMARkER EVALuATION FRAMEWORk Many speakers noted the importance of a biomarker evaluation framework across product areas. For example, Andrew Shao said that an absence of an accepted framework has limited the amount of research conducted on the role of diet and nutrition in health promotion and dis ease prevention: "We anticipate that a formal biomarker evaluation pro cess will add clarity to product development, as companies that choose to invest in research will have a better understanding a priori that the research will have broader acceptability and applicability to public health recommendations." Stephen Williams said that the pharmaceutical industry wants a consistent, transparent biomarker evaluation framework that
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From page 88... ...
Thomas Fleming suggested that a lack of clarity, both from the regulatory and scientific perspective, on biomarker evaluation standards is much more likely to inhibit innovation. Implementation of Recommendation 3 of the report could help FDA to bring scientific and regulatory clarity to biomarker evaluation across the FDA's centers and regulated product categories.
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From page 89... ...
purpose first, then I don't think you can define how good is good enough, and that's what validation and qualification are about." Maria Lopes-Virella said that the committee recognized that there must be a rationale for embarking on the evaluation of a biomarker, which can, in some sense, be seen as utilization, which is the reason the framework was depicted as circular. In organizing the evaluation framework, the committee placed analytical validation, qualification, and utilization in order of the decisions that would be made, understanding that the biomarker evaluation process would be initiated on the basis of an initial motivation or context of use.
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From page 90... ...
Kathleen Ellwood noted that reliance on long-term clinical trials is not always feasible, and that the FDA requested the IOM to undertake this study to address the absence of an agreed-upon, systematic, and transparent process for qualifying surrogate endpoints. Several speakers from the food and nutrition industries said that they rely on biomarkers and surrogate endpoints to conduct trials on nutritional interventions, because it is too costly or logistically challenging to conduct trials with clinical endpoints.
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From page 91... ...
Douglas Balentine said that the process by which food and nutrition companies identify promising biomarkers is fundamentally different from the way the pharmaceutical industry develops biomarkers. Many food biomarker leads come from epidemiological or observational studies that examine "intake markers" such as beta-carotene or the consumption of certain foods.
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From page 92... ...
Thus, he had hoped that the committee would recommend "ways to use surrogate endpoints to inform shorter-term clinical trials that the food industry could do with an endpoint that made sense." Victor De Gruttola responded that "if you can only do the randomized study on the biomarker, but not the clinical endpoint, then you are in the world of observational studies." Such studies show a causal effect on the biomarker, but to conclude that there is a causal impact on the clinical endpoint requires a randomized controlled trial, he said -- unless the biomarker has been validated as a surrogate endpoint. If evidence from a controlled trial of foods shows an effect on a biomarker, and epidemiologic evidence suggests that the effect on the biomarker may be correlated with the effect on the clinical endpoint, this information leads to a hypothesis, Dr.
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From page 93... ...
If the claim is not based on clinical endpoints, "don't say that it reduces heart disease," she insisted. Some speakers noted that there is a lack of incentives for the food and nutrition industries to conduct rigorous studies to support the biomarker evaluation framework.
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From page 94... ...
For example, there are multiple ways food scientists might approach a surrogate endpoint such as blood pressure. Biologists and medical scientists need to agree among themselves what a biomarker means, rather than present conflicting or confusing information to the public or the media, she said.
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From page 95... ...
Communication was one of the main topics the committee discussed, according to Dr. Lopes-Virella, and committee members were concerned that their message be clear and not frightening.
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