CEO of Aastrom Biosciences calls for conditional approval framework at FDA

Over at Xconomy, Tim Mayleben, president and CEO of Aastrom Biosciences, makes a passionate plea for regulatory reform and changes at the NIH to support companies moving through the "valley of death", i.e., that can't get access to the capital they need to move from preclinical research to clinical proof of concept studies. (Once you have proof of concept data, it gets much easier to raise capital.)

Without these changes, Mayleben warns, the U.S. may abdicate its leadership of the global biomedical industry:

The most important issue facing Aastrom and many other companies in biotechnology is the lack of adequate funding to support innovative new companies, technologies and therapies. The challenges in drug development, especially in the early stages, are only compounded by the current regulatory environment with a primary focus on risk reduction. ...

...the current situation has to change if the United States is going to remain the global leader in the discovery, development, and commercialization of innovative new therapies in the next two decades. If we don't improve the current conditions, we will continue on a path to increasing irrelevance in biomedical research and innovation, especially given the fact that other countries are rapidly recognizing the potential of our industry to improve human health and create jobs, and they are in many ways ahead of the U.S. in supporting these companies.

Mayleben believes that the NIH should shift more funding from basic research to translational research, perhaps even supporting companies with the most promising new therapies. It certainly would make sense for the NIH - with a budget of over $30 billion dollars, to develop more of the basic tools for advancing new drug candidates through to proof of concept studies, either by standardizing biomarker assays and diagnostics on a disease by disease basis, and working with the FDA to validate new regulatory science tools, like adaptive clinical trial designs.

While this might be seen as conflicting with the NIH's mandate for supporting basic science research, without better translational tools, NIH-funded basic research will wither on the vine far too often because companies can't get to the point where their treatments are candidates for FDA approval.

But my favorite recommendation that Mayleben makes is for the FDA to become more flexible about when - and how - it evaluates efficacy and safety for new drug candidates. Essentially, Mayleben is suggesting that the FDA move to a conditional approval framework.

The ability to review and approve new therapies with great speed and, equally importantly, the ability to "disapprove" and remove therapies from the market or alter the product indication post approval, could significantly improve the competitive position of the U.S. in biotechnology in the years ahead. Industry will have to accept the fact that FDA decisions might not mean approval forever. They will build a model where marketing can begin while additional data is collected to provide further confirmation that benefits outweigh risks.

(For some overview on how this might work in practice, see here.)

I think this is exactly what needs to happen, and a development that's long overdue. Of course, the FDA's current accelerated approval process is basically what Mayleben is calling for; but the FDA typically uses accelerated approval only for drugs for cancer or other life threatening or orphan indications. However, from a disease burden perspective, conditional approval is also needed for widespread diseases or conditions that affect tens of millions of Americans, like diabetes and obesity.

Our drug development system is set up to prevent the next Thalidomide or Vioxx, rather than accelerate the search for cure for Parkinson's or diabetes. Right now, most of the industries' and the FDA's money, time, and expertise are focused on gathering information in the pre-market environment, where at least some of what we learn will later turn out to be incomplete or flat out wrong. It also ignores the potential of new tools like electronic medical records, and new social networking sites to improve how medicines are used "in the real world".

To give the most innovative biotech companies a better chance of bringing truly innovative new therapies to market, we need a new paradigm - a postmarket paradigm that would match the rest of the U.S.'s fluid, information-based economy.

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