The 2009 PDA-FDA Joint Regulatory Conference opened this morning in Washington, DC, with a keynote address by Dr. Joshua Sharfstein, principal deputy commissioner at the US Food and Drug Administration.

Before Dr. Sharfstein’s address, Richard Johnson was introduced as the new PDA president. Former association president Bob Myers retired earlier this year. “I’m very excited about challenges we face as an association going forward and am committed to continuing the tradition of PDA,” said Johnson, whose term became effective September 1. “Our commitment to our mission is equally clear and so we will be continuing to live up to that mission as we go forward…. We are going to continue connecting people in science and regulation.”

Next, FDA’s Rick Friedman, co-chair of the conference along with Martin Van Trieste of Amgen, briefly discussed the focus of the conference, “Securing the Future of Medical Product Quality: A 2020 Vision.” He noted that after a series of contamination events, including diethyl glycol [DEG] and heparin, industry and regulators have learned many lessons. “We’ve learned together in the last years that as your unknowns in the supply chain increase, so does cumulative uncertainly and risk. We’ve also learned that integrity can be compromised either by those lacking understanding of how to properly manufacture or handle drugs or by those with bad intentions. The question we have to collectively deal with is how do we improve the system to prevent these grave risks?”

Sharfstein began his address by noting the title of the conference and how it “takes a little courage in the world of medical product technology to look ahead,” he said. “It’s so important to look forward to the next stage in innovation and science for product quality…. Helping to shape the future is one important reason why [FDA Commissioner Dr. Hamburg and I] accepted these positions.” Sharfstein and Hamburg started their positions at FDA this past spring.

Referring to the May 2009 New England Journal of Medicine article in which he and Dr. Hamburg outlined their vision of the agency as a public health agency, Sharfstein outlined four principles guiding their definition of a public health agency.

First, a primary principle of public health is prevention, he said. “Rick Friedman mentioned economic adulteration as a problem, diethylene glycol, supply-chain management, and others, and I think all of these things are amenable to a preventive approach,” he explained. “When one company falters because of a quality problem, it’s their problem to fix and prevent. But when a problem occurs across the industry, when it causes the kind of quality-system problems…it’s everyone’s problem and it’s FDA’s problem to help think about solutions.”  

Sharfstein also brought up the quality trio guidelines finalized by the International Conference on Harmonization, ICH Q8, Q9, and Q10, as an example. “The goal of these efforts is to understand from the start what makes a product of high quality rather than waiting to discover a problem after the fact,” he said. “We would like to foster more such productive collaboration that leads to concrete results in preventing problems.”

The second principle of a public health approach requires the best available science, said Sharfstein. FDA is working to identify key projects that can help product quality, streamline approval pathways, and improve scientific training. “We’d like to see more guidance documents emerge out of these scientific efforts to provide a clear and transparent process to address long-standing quality issues and to help bring important products to the market.”

Sharfstein pointed out that both basic science and regulatory science are needed to improve industry, and then provided a rowboat analogy. If a rower has one massive right arm and a puny left arm, it’s no surprise that the result is not forward moving, he said. “Industry has an important responsibility for helping to move regulatory science forward.”

Third, a public agency by definition involves the public, he said, pointing to the new transparency initiatives at FDA such as its website, public meetings, and blog. He encouraged industry to improve its transparency as well, noting that sometimes, companies are reluctant to issue a press release or conduct a recall when things go wrong.

“In the next decade, with speed of information only accelerating, it’s a good assumption that product quality problems will eventually come to light. It’s better for companies to develop effective and transparent communication systems now rather than stumble when others are providing key messages about your products for you,” he said.

Sharfstein emphasized his point by stating, “When public health is at stake, FDA is going to take this basic position: You can explain this problem to the public or we will.”

He discussed the fact that, based on his experience as the Baltimore City Health Commissioner, the public seems to understand two basic messages: this product is totally safe or totally unsafe. But he noted that companies can’t not react for fear of scaring the public. “Having that challenge of risk communication is not a reason to not to tell the public what they need to know…. We need to work together to establish messages besides ‘totally safe’ and ‘totally unsafe.’ …One such message would be that there are both benefits and risk and that we believe the benefits exceed the risks.”

Fourth and finally, Sharfstein discussed the premise of public health as one in which the health of the public matters. “In evaluating product quality issues for 2020, I would imagine there are many ways to resolve key challenges as you face them, however, I urge you to consider the health of the public as the North Pole for your compass.”

A brief question-and-answer session followed (Sharfstein had to head to the White House for a budget meeting). One audience participant asked about FDA’s new enforcement approach, which Dr. Hamburg discussed recently at the Food and Drug Law Institute.

“Enforcement is an important tool and for it to be effective tool, it has to be timely and meaningful and it has to address issues that are important. What Dr. Hamburg mentioned [in the Food and Drug Law Institute talk is that…] the enforcement system at FDA has not always met that goal all the time.” With this in mind, when FDA sees the public health in danger, it will be acting more quickly. “We may not even send a Warning Letter if we think that it’s that serious. At the same time, we want to give an incentive for people to fix it as quickly as possible…. One incentive would be a letter from the agency that the problem has been resolved.”

He pointed out that when he was the Baltimore City Health Commissioner, if there was a problem in the city, the mayor expected him to fix it right away, not exchange memos about it for several months. “Our hope [at FDA] is that we’re able to send a message that there are certain product quality things to be avoided in the first place, but if they happen, the agency can act very quickly on them and also have them resolved quickly.” 

In a bit of a candid statement during the Q&A session, Sharfstein admitted that he’s been reading the trade press and watching TV reports about FDA since he and Dr. Hamburg began their transition at the agency. He’s been quite interested in the media’s take on whether the agency will be pro-industry or anti-industry. “Dr. Hamburg and I feel very strongly that a public health approach is not pro- or anti- industry. To the extent that we can articulate that something is worth it, we think it’s okay to take the risk….. At meetings that I’m at, we’re saying, what would make something great possible? Let’s not be so risk-averse that we lose opportunities for really important innovation and progress….. In the end, we’ll be judged on whether we can make those changes happen at FDA…and that’s something that we’re working on.”