Many collaborations occur in the precompetitive space where all partners can benefit from new knowledge without losing competitive advantage. Foundations can play a central role in such collaborations, Capone said, because the absence of a profit motive can keep the focus on the science and on the benefits to patients. In that regard the parties to a collaboration need to recognize the potential threats to a collaborative enterprise. For example, efforts to protect intellectual property, either by industry or academic partners, can stymie partnerships and thereby limit scientific advances. If all the information generated by a partnership is available, no party feels that it is being disadvantaged versus other parties in the group. Transparency requires effective governance structures and accountability. If these cannot be achieved, Capone said, it may be necessary to find different partners.

FDA INITIATIVES

A particularly important partner in many collaborations is the FDA, which was represented at the workshop by its commissioner, Margaret Hamburg. The FDA has been working with sponsors, patient groups, and academia to get into the marketplace new products that represent the opportunities of personalized medicine, she said. These collaborative efforts have generated real benefits for patients with treatments being developed that affect the underlying mechanism of disease rather than treating symptoms, such as with ivacaftor, and that affect the appropriate use of medications, as represented by the more than 110 drugs that now have genetic information on their labels.

Research collaborations will be increasingly important to the FDA in the future, because it does not have the resources to do all of the research needed to develop the regulatory tools and knowledge needed for the agency to do its job as efficiently and effectively as possible, Hamburg said. Biomarkers need to be identified, characterized, and validated. Standards for whole-genome sequencing and SNP panels need to be established. Innovative clinical trial designs need to be developed that are faster, cheaper, more adaptive, and use smaller populations of patients, particularly in projects to identify subpopulations of patients that can benefit from a drug or that would react poorly to a drug.

One complication for the FDA is that many potential therapies cut across its traditional domains of product activity. The combination of a diagnostic with a therapeutic intervention falls into two centers within the FDA with different regulatory frameworks. The FDA’s challenge is to deal with such products in ways that “are efficient and reflective of where the science is and where these products are,” Hamburg said. The FDA will continue to work with its sponsors to break down barriers to co-development and to help create an effective scientific and business framework.



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