Eyeworld

EW NEWS & OPINION 12 The FDA is the gateway for new therapies. As a vigilant gatekeeper, the regulatory process has to ensure that new treatments are safe and ef- fective. But it has to be a facilitator for the development of new thera- pies. The process of medical innova- tion is complex, but in my experience there are examples of best practices right in the halls of the FDA. As a drug developer who leads the clinical research and devel- opment of one of the most success- ful approved biologic therapies for eye disease (Lucentis for macular de- generation), I have the added com- parative experience with the CEDR, whose input and oversight were crit- ical in the execution of this highly complex, rigorous therapeutic pro- gram and resulted in the commer- cialization of a groundbreaking therapeutic which now helps hun- dreds of thousands of patients who would otherwise go blind from mac- ular degeneration. This program was not only successful but exemplary in many ways of how the regulatory process should work and was refer- enced by the FDA reviewers in a published guidance to industry for best-in-class drug development. The key learning from this expe- rience were clear, explicit guidance to companies and investigators, con- sistency, and transparency of feed- back in the review process and a new level of in-house expertise from the FDA reviewers. My experience with the devel- opment of new technologies is that the pathway to innovation is chal- lenging and it is necessary to take calculated risks in a thoughtful and deliberate way in order to protect patients. We need safe and effective treatments for our patients, and it is critical that we have the best-in-class regulatory process to do justice to the high level of passion, talent, and resources this country invests in the innovation process to help patients. EW November 2011 Dr. Chang speaks with Dr. Ianchulev about the hearing Dr. Chang: Tell us about the congres- sional hearing and who was invited to testify. Dr. Ianchulev: In Washington, as elsewhere in this country, there is increasing concern and attention paid to the global leadership posi- tion of the U.S., particularly with re- spect to science, technology, and innovation. This comes on the heels of a recent report by the World Eco- nomic Forum, which ranked the U.S. number six in terms of innova- tion and number 40 for health and primary education. So it is not sur- residents get when they observe a cataract case for the first time. First and foremost, it requires a lot of preparation. You cannot afford to testify to Congress as an expert pan- elist without verifying all the facts, understanding your role, and know- ing your limitations. For me the challenge was to distill the key mes- sages and focus the debate on spe- cific examples and "symptoms." As a physician, I had to tell the clinical and academic story of the impact I see on patient care and resident edu- cation; as an innovator who works with companies to develop the next medical therapy (from Lucentis to refractive cataract devices and mini- mally invasive glaucoma micro- stents), I had to share the dilemmas we face today in getting technology to patients quickly and efficiently. Finally, as a venture partner who participates in the funding and in- vestment process of new start-ups, I had to discuss some of the recent challenges in raising capital to fund the development of new drugs and devices. Given the allotted time, it is important to get the key points across fast. The hearing also im- pressed me with the high level of Congressional continued from page 11 Fischell, a distinguished physicist and one of the great medical inno- vators credited for the development of milestone technologies such as the implantable cardiac defibrillator, the insulin pump, and numerous coronary stents; Dr. Curfman, execu- tive director of the NEJM; Michael Mandel, chief economic strategist for the Progressive Policy Institute; as well as several patient advocates. As a clinician, innovator, and devel- oper of medical therapies, my role as an expert on the panel was to in- form Congress about the sympto- matic state of the regulatory process in the U.S., and the impact on small and large companies as well as on clinicians and patients with respect to choice and access to (or lack thereof) different treatment options and technologies. Dr. Chang: What was the experience of testifying like? Dr. Ianchulev: From the side, a con- gressional hearing feels like what we see on C-SPAN. But that is pretty much where it ends. C-SPAN pro- vides as much insight into the con- gressional process as first year A case of unbalanced incentives and no chance for appeal by R. Doyle Stulting, M.D., Ph.D. D r. Ianchulev's testimony is accurate and eloquent. The requirements for approval of drugs and devices in this country have be- come so burdensome that sponsors do not seek approval unless the technology is al- ready proven and projected to be sufficiently prof- itable to cover the high cost of regulatory efforts. Our patients more frequently must travel overseas for treatment that has been proven safe and effective by scientifically valid clinical data published in the peer-reviewed literature. Why does this situ- ation exist, and what can be done to reverse this trend? Having been involved with the regulatory process as a consultant for both the FDA and product sponsors over the past 23 years, I see well-in- tentioned, dedicated FDA reviewers who are highly motivated to avoid approval of products with even the most remote chance of undetected ad- verse reactions. As a result, they micro-manage clinical protocols, de- manding clinical data that might have little or no bearing on the safety of innovative products—with no regard for the cost of those demands. They actually pride themselves on their lack of concern for cost, delay in ap- proval, or the financial survival of the companies they regulate. Sponsors of devices and drugs have no advocates in the FDA—no one who recognizes the detrimental effect on U.S. citizens caused by the lack of access to new technologies. The press in this country is quick to report complications of an approved device or drug, but they don't publish sto- ries about the lack of access to treatments and how it harms our citizens. There is no avenue for appeal that does not require an inordinate amount of time and money—or make sponsors fear retribution from the reviewers with whom they must work in the future. When additional funding is provided to the FDA, through the federal budget or user fees, it is typically used to hire more reviewers, statisticians, and regulatory personnel, supplying additional manpower to create even more burdensome requirements for approval. Why not use these funds to add sponsor advocates to the FDA, empowered to question unnecessary protocol demands by primary reviewers and respond to sponsor com- plaints? We don't need more regulatory personnel in the FDA. We need more reasonable, properly incentivized, scientifically knowledgeable advocates for industry who can shepherd proven innovative technologies that are available to the rest of the world through our regulatory system. prising that Congress is paying at- tention to and trying to understand the innovation debate that has heated up recently. On July 20, 2011, the Energy and Commerce Committee (which ultimately over- sees the FDA) held a hearing on the impact of FDA regulation on innova- tion, patients, and jobs. Congress wanted to hear a multi-faceted view on the topic and invited expert wit- nesses for the seven-person panel. It was a very impactful group repre- senting different stakeholders in the process of innovation and patient care. The group included Dr. Robert Dr. Ianchulev

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