Commissioner Hamburg on R&D Productivity

The following is an excerpt from FDA Commissioner Margaret Hamburg's keynote address to the Food & Drug Law Institute annual meeting in Washington April 4.

As you know, the FDA plays an essential role in the development of new products and therapies for patients and consumers. But that pipeline is at risk: despite unprecedented spending on basic research and development by the government as well as the biotechnology and pharmaceutical industries—to the tune of more than $80 billion this past year—the number of submissions of new molecular entities to the FDA for review is on the decline.

In addition, the rate of failures in drug development remains high: recent reports suggest that any given drug studied in early human clinical trials has less than a 10 percent chance of making it successfully all the way through to approval and marketing, with most of the attrition occurring during clinical trials, well before the actual FDA review process begins. And as medical devices and other products become increasingly complex, the standards and tools we use to evaluate their safety and efficacy are not keeping pace.

In other words, the return on investment for biomedical products continues to drop.

Unfortunately, FDA has been identified in many analyses as a key factor in this decline—largely because rising product development times and costs are attributed to delays and uncertainty in the regulatory approval process.

We certainly get a lot of criticism—some of it deserved—but in reality the situation is a lot more complex. There are many factors involved and, looking to the future, we must, as a nation, develop meaningful strategies to address what is a real and serious problem. Looking at FDA, it is important to recognize the proactive role we need to play, and also to address some myths and misunderstandings. In fact, our rate of approvals has not slowed for many years, even as products become more complex.

And despite what you may be hearing, we have a strong track record for meeting and often exceeding tough deadlines for review times established by industry in conjunction with FDA as part of the user fee process. We have also made progress in speeding products to market. In the past few years, 90 percent of the 3,000-plus 510(k) medical device submissions we receive each year were reviewed in 90 days or less.

In addition, there are a lot of criticisms out there that we are slower than Europe in approving new products—and are therefore driving companies away.

In fact, FDA consistently approves new drugs and devices as fast or faster than other countries, while maintaining the safety standards that Americans count on. For example, of the 57 novel drugs approved by both FDA and the EMA, the FDA's European counterpart, from 2006 through 2010, 43 were approved first in the United States. Of the 27 drugs in this group that were given priority review because of their special therapeutic potential, 24 were approved first by the FDA…and the FDA's median time to approve these priority drugs was 183 days, some 220 days faster than the EMA.

Similarly, a study recently commissioned by the device industry showed that FDA approves low risk medical devices faster than Europe, and higher risk as fast as Europe.

Regardless, we certainly don't view our agency as "in competition" with the EMA or any other sister regulatory agency—our scientists work closely with theirs and share information on a regular basis. And as we strive to meet the challenges of globalization, as I mentioned before, this is more important than ever.

The truth is that on the world stage, the FDA is still regarded as the premier regulatory agency, the one that other nations look to as a standard... and one that opens up international markets for FDA-approved products.

And we continue to have a unique perspective as a regulator, but also as a science-based public health agency. So, at this critical moment of need for patients and consumers in our country and around the world, we have a real responsibility to take advantage of this unique position and deliver new, innovative products to the people who need them most. And this is an opportunity for us to strengthen and modernize our scientific capacity to support the review and approval of products for the 21st century.

Much of regulatory uncertainty is rooted in scientific uncertainty: a lack of clear, reliable data that supports decision making for product safety and efficacy. There are many reasons for this scientific uncertainty, including increasingly complex medical products and an incomplete understanding of human biology. But most importantly, it stems from a lack of attention and investment in regulatory science—the science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of FDA-regulated products.

This, in my view, is unacceptable. Investments in regulatory science can drive sound, smart regulatory policies that lead to safer, more innovative products for people who need them, while at the same time contributing to the economic growth and health of the device and pharmaceutical industries.

Which is why advancing regulatory science on behalf of the public health has been a top priority since I became Commissioner. Through our Advancing Regulatory Science Initiative, we are devoting time and resources to leading the effort to ensure these investments are made. In addition to strengthening the science base at the FDA, we are building the partnerships—across government and with academia, industry, and the non-profit community—that will drive the development of innovative medical products and the delivery of better, safer products to the American people.