From the Pink Sheet ...
FDA is making the case for increased data-sharing and disclosure about drug development failures in the name of bolstering regulatory science.
In a speech to the Food and Drug Law Institute’s annual meeting on April 5, FDA Commissioner Margaret Hamburg said agency disclosure of reasons why a drug failed to win approval would strengthen the scientific underpinnings of drug development and regulatory review.
In addition, the agency is examining how it can mine its databases of submitted applications and approved products to help address scientific uncertainties in the development process.
Hamburg used the FDLI speech to link two of her key initiatives since joining the agency in May 2009 – strengthening regulatory science and increasing transparency of agency actions.
Despite the agency’s stance that increased data-sharing and disclosure would speed the drug development process, such concepts are sure to see pushback from pharmaceutical companies concerned about protecting proprietary information.
Spurring Innovation …
Hamburg’s speech to FDLI focused on the theme of innovation and what she said was FDA’s responsibility “to take advantage of our unique position” to help deliver new, ground-breaking medical products.
“Innovative products that are truly transformative create unique scientific and regulatory challenges, so we’re rolling up our sleeves, developing FDA’s regulatory science and innovation initiative strategy and doing everything that we can to rise to the challenges and to ensure that FDA is a consistently powerful catalyst for innovation,” Hamburg said.
“Moving forward, we’ll focus our efforts on several distinct yet inter-related areas of activity, each of which must be addressed if we are to make a meaningful difference in supporting innovation.”
The first area she touched on is regulatory reform, saying, “We’ll continue to work hard to streamline and modernize regulatory pathways and make them more predictable and transparent.” She cited several examples of FDA action in this regard, including its ongoing implementation of the “21st Century Review Process,” also known as good review management practices, and encouraging a quality-by-design approach to drug manufacturing.
“In each of these cases regulatory reform involved changing practices but also developing and applying better scientific understanding and tools to the review process, which brings me to the second area, strengthening science and reducing scientific uncertainty,” Hamburg said.
Hamburg unveiled her regulatory science platform in October. Among the initiative’s goals are modernizing product development; improving the speed, efficiency and predictability of the development, application review and manufacturing process; and using informatics to enhance health and drug safety
FDA’s fiscal 2012 budget request seeks $48.7 million in new funding for the program
Regulatory uncertainty is rooted both in scientific uncertainty and the failure to capitalize on existing knowledge, she said.
“At FDA, we have all of the data for every medical product ever submitted and approved in the U.S. There are huge opportunities to mine this information for the benefit of everybody to learn how to more effectively design drugs, to learn more about why products fail and how to better predict failure, and perhaps how to repurpose certain … drugs as we learn more about how to better assess subpopulations of responders.”
Developing new ways to share data and enhance communications with companies, without compromising trade secrets and other important commercial confidential information, is an important priority for FDA and should be for industry as well, Hamburg said. “This is part of what I mean by changing systems. We need to replace what is outmoded or not working with stronger, more effective patterns of practices.”
…Instead Of Waiting For Failure
During the question-and-answer session following her speech, Hamburg was asked how the concept of data-sharing would work “on a nuts and bolts basis” given industry concerns with such a proposal.
“This is an area that we’re obviously looking at carefully and cautiously and any decisions going forward would be done in partnership … with our key stakeholders,” she said.
She noted that agency scientists already have begun looking at data-mining opportunities with external partners, and the agency’s Transparency Initiative is examining some of the issues pertaining to disclosure.
FDA’s Transparency Task Force issued draft recommendations in May aimed at increasing public disclosure about agency actions. These included disclosing the existence and contents of “complete response” and “refuse to file” letters for NDAs and BLAs, as well as summary safety and effectiveness information for investigational applications or pending marketing applications if doing so would benefit public health
Many of the disclosure proposals drew strong objections from the Pharmaceutical Research and Manufacturers of America and the Biotechnology Industry Organization, which raised concerns about protecting trade secrets and the recommendations’ legality
However, Hamburg pointed to the benefits of disclosing information about why a drug was not approved.
“That can contain very, very important information to the R&D field more broadly,” she said, noting that there might be “multiple sponsors developing drugs following scientific pathways that, based on earlier work done, we know will fail. It’s not in the interest of those companies or the broader public good to just sit and watch them fail.”
Hamburg suggested that industry and individual companies would have to be on board with the disclosure concept. “A given company would have to be willing to say, ‘I understand there is a common good here … even though it might be demonstrating my dirty laundry just a little bit.’”
FDA is not alone among regulators in seeking to encourage such data-sharing and disclosure on drugs in development and clinical failures, she said.
“This is something that other regulatory authorities are also thinking about and working on as well because it’s an issue of really helping to build out the underlying science as we think about how can we make both the medical product development process as targeted and as effective and as efficient as possible, and how can we utilize the information that we have as regulators to support that process.”
Hamburg was asked whether FDA is discussing unilateral disclosure of information about drug development programs even if a sponsor objects. “It’s a discussion that we’re having, and I think it’s a partnership working with industry,” she said. “We need to move in directions that make sense, that will have value added, and where everyone understands the expectations and the opportunities.”
By Sue Sutter
