A few of the proposed changes “might make a critical difference,” says Washington attorney John Cohrssen, an Organisation for Economic Co-operation and Development (OECD; Paris) advisor who once worked on Capitol Hill. Planning for this innovation package began last summer when BIO commissioned Elias Zerhouni, the former director of the US National Institutes of Health (subsequently appointed president of global R&D at Paris-based Sanofi in January), to gather comments from key opinion-makers in academia, medical institutions and biotech firms. The merits of the resulting proposals were debated internally and shaped into an eight-page summary document, which was presented to the Health Subcommittee last month by Paul Hastings, BIO's vice-chairman of emerging companies and the president and CEO of Redwood City, California–based OncoMed Pharmaceuticals. Also invited to the meeting was Jonathan Leff, managing director of New York–based Warburg Pincus, who testified on behalf of the Arlington, Virginia–based National Venture Capital Association (NVCA). The NVCA's proposals, developed by its Medical Innovation and Competitiveness Coalition, which includes life science venture capitalists, were mostly aligned with those drawn up by BIO. Patient advocacy groups were also present, represented by Marc Boutin, executive vice president and CEO of the National Health Council in Washington, DC.
The shining part of BIO's document, according to Greg Conko, senior fellow at Washington's Competitive Enterprise Institute, is the proposal to create a progressive or conditional approval pathway. This approach allows all data and information associated with a product to be taken into account during regulatory review, whatever its value. This would allow the US Food and Drug Administration (FDA) to gradually introduce new drugs to the market for defined patient populations after phase 2 studies, while phase 3 testing is still ongoing. Although this possibility has been discussed before, this new version of progressive approval would apply only to innovative products for unmet needs, those that offer significant improvements in standard of care, targeted therapies or those that have been approved by other regulators, such as the London-based European Medicines Agency.
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