Statement of Jay P. Siegel, M.D.

Statement of Jay P. Siegel, M.D.

Johnson & Johnson
EXECUTIVE SUMMARY
March 7, 2007

Entire testimony available here. (PDF format)

Johnson & Johnson welcomes the opportunity to participate constructively in the development of a sound path forward for follow-on biologics. Any legislative framework for follow-on biologic products should, of course, be well-grounded in science, with paramount attention to patient safety and well-being. To that end, there are a number of principles critical to address in any abbreviated approval system for follow-on products, five of which are as follows:

First, there will always be a need for appropriate pre-marketing clinical data to help ensure that a follow-on biologic is safe and effective. The complexities of biologics make them particularly susceptible to clinically significant changes when changes are made to the process by which they are manufactured. As the production of follow-on biologics will necessarily involve many changes in process, there will always be the potential for subtle, but clinically meaningful differences for follow-on products. Since laboratory testing has proven insufficient to detect many clinically important differences in biologics, testing in humans will be necessary to help ensure their safety and efficacy.

Second, there cannot be allowance for determinations of “comparability” for products that are so different in structure that they should be considered different products entirely, or for products that may have clinically meaningful differences. Products that are substantially different in structure should be regulated as different products requiring full testing for safety and efficacy.

Third, a follow-on biologic product should not be considered interchangeable with its reference product. Follow-on biologics can be similar, but never identical to an innovator biologic. Any application of “interchangeability” status to a follow-on could lead to inappropriate assumptions of sameness and substitution of one for the other, with potentially serious adverse health consequences. Such substitution of biologics should be discouraged.

Fourth, FDA must be empowered to require post-marketing clinical studies and post- marketing safety surveillance to ensure safety. Some risks associated with a follow-on biologic, like all pharmaceuticals, will become apparent only in the post-marketing period. To optimize patient safety and control such risks, the safety of follow-on biologics, like other drugs and biologics, should be monitored by the license holder through a robust post-marketing safety surveillance program. Also, FDA must not be limited in its ability to request post-marketing clinical studies when appropriate.

Fifth, there should be no constraints placed on the FDA for ensuring the safety of follow-on products. As we enter this new field with new safety risks, the FDA should be unhampered in its ability to request and receive additional data from a manufacturer as the need becomes apparent.

A Path Forward: We are fortunate that the EU has already made substantial progress in developing and implementing a policy based in good science and public health and consistent with their unique regulatory and healthcare framework. We should be able to leverage that work to have a frank, transparent and scientific debate here in the United States, and thereby develop a model which will be compatible with our own regulatory and healthcare environment. It is the hope of Johnson & Johnson that a scientifically-based public process will provide a framework and pathway for follow- on biologics in the US√Ďa pathway which reflects an overriding concern for patient safety and which provides appropriate incentives for innovation so that the promise of new and innovative biologic therapies can continue to be realized for patients for generations to come.